<clinical_study>
  <!-- This xml conforms to an XML Schema at:
    https://clinicaltrials.gov/ct2/html/images/info/public.xsd -->
  <required_header>
    <download_date>ClinicalTrials.gov processed this data on September 20, 2023</download_date>
    <link_text>Link to the current ClinicalTrials.gov record.</link_text>
    <url>https://clinicaltrials.gov/ct2/show/NCT01704079</url>
  </required_header>
  <id_info>
    <org_study_id>CTAP101-CL-3002</org_study_id>
    <nct_id>NCT01704079</nct_id>
  </id_info>
  <brief_title>Safety and Efficacy of CTAP101 to Treat Secondary Hyperparathyroidism in Stage 3 or 4 CKD and Vitamin D Insufficiency</brief_title>
  <official_title>A Multi-Center, Randomized, Double-Blind, Placebo-Controlled Study to Evaluate Efficacy and Safety of CTAP101 Capsules to Treat Secondary Hyperparathyroidism in Subjects With Stage 3 or 4 Chronic Kidney Disease and Vitamin D Insufficiency</official_title>
  <sponsors>
    <lead_sponsor>
      <agency>OPKO IP Holdings II, Inc.</agency>
      <agency_class>Industry</agency_class>
    </lead_sponsor>
  </sponsors>
  <source>OPKO Health, Inc.</source>
  <oversight_info>
    <has_dmc>Yes</has_dmc>
  </oversight_info>
  <brief_summary>
    <textblock>
      This study will evaluate the efficacy of CTAP101 Capsules versus placebo in reducing intact&#xD;
      parathyroid hormone (iPTH) by at least 30% from pretreatment baseline; safety and&#xD;
      tolerability of CTAP101 will also be evaluated&#xD;
    </textblock>
  </brief_summary>
  <detailed_description>
    <textblock>
      This is a phase 3, multi-center, randomized, double-blind, placebo-controlled study to&#xD;
      evaluate the efficacy and safety of CTAP101 Capsules administered for 26 weeks to treat SHPT&#xD;
      in subjects ≥18 years of age with stage 3 or 4 CKD and vitamin D insufficiency. The study&#xD;
      will be conducted at approximately 40 sites within the United States (US). Approximately 550&#xD;
      subjects will be screened to randomize approximately 210 eligible subjects, stratified by CKD&#xD;
      stage (approximately 105 subjects in each stage) in a 2:1 ratio to receive either CTAP101&#xD;
      Capsules or matching placebo. An Interactive Voice Response System (IVRS) will provide study&#xD;
      treatment group assignments using the computer-generated randomization code provided by the&#xD;
      IVRS vendor. Subjects will receive an initial daily dose of 1 capsule (CTAP101 Capsules, 30&#xD;
      µg, or matching placebo) for the first 12 weeks. After l2 weeks (visit 8) and per pre-defined&#xD;
      criteria, the dose may be increased in a blinded fashion by the IVRS with oversight by an&#xD;
      independent medical monitor.&#xD;
    </textblock>
  </detailed_description>
  <overall_status>Completed</overall_status>
  <start_date>November 2012</start_date>
  <completion_date type="Actual">September 2014</completion_date>
  <primary_completion_date type="Actual">August 2014</primary_completion_date>
  <phase>Phase 3</phase>
  <study_type>Interventional</study_type>
  <has_expanded_access>No</has_expanded_access>
  <study_design_info>
    <allocation>Randomized</allocation>
    <intervention_model>Parallel Assignment</intervention_model>
    <primary_purpose>Treatment</primary_purpose>
    <masking>Double (Participant, Investigator)</masking>
  </study_design_info>
  <primary_outcome>
    <measure>Number of Participants in the Intent to Treat Population With Decrease in Plasma Intact Parathyroid Hormone (iPTH) of ≥30% From Pre-treatment Baseline</measure>
    <time_frame>Approximately 6 months</time_frame>
    <description>Number of subjects in the intent to treat population attaining mean decrease in plasma intact Parathyroid Hormone (iPTH) of ≥30% from P\pre-treatment baseline in the efficacy assessment phase (EAP) referred to as responders</description>
  </primary_outcome>
  <secondary_outcome>
    <measure>Number of Participants in the Per Protocol Population With Decrease in Plasma Intact Parathyroid Hormone (iPTH) of ≥30% From Pre-treatment Baseline</measure>
    <time_frame>Approximately 6 months</time_frame>
    <description>Number of subjects in the per protocol population attaining a mean decrease in plasma intact parathyroid hormone (iPTH) of ≥30% from pre-treatment baseline in the efficacy assessment phase (EAP), referred to as responders</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Subjects in the Intent to Treat Population With Normal Serum Total 25-hydroxyvitamin D</measure>
    <time_frame>Approximately 6 months</time_frame>
    <description>Subjects in the intent to treat population with normal serum total 25-hydroxyvitamin D (&gt;/= 30 ng/dL)</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Subjects in the Per Protocol Population With Normal Serum Total 25-hydroxyvitamin D</measure>
    <time_frame>Approximately 6 months</time_frame>
    <description>Subjects in the per protocol population with normal serum total 25-hydroxyvitamin D (&gt;/= 30 ng/mL)</description>
  </secondary_outcome>
  <number_of_arms>2</number_of_arms>
  <enrollment type="Actual">216</enrollment>
  <condition>Chronic Kidney Disease</condition>
  <condition>Hyperparathyroidism, Secondary</condition>
  <condition>Vitamin D Deficiency</condition>
  <arm_group>
    <arm_group_label>CTAP101 30 μg capsules</arm_group_label>
    <arm_group_type>Active Comparator</arm_group_type>
    <description>1 CTAP101 30 μg capsule daily at bedtime for 12 weeks, with a possible increase to 2 CTAP101 30 μg capsules daily at bedtime for study duration, if needed (26 weeks total). Subjects not requiring a dose increase after 12 weeks would receive 1 CTAP101 30 μg capsule and one sugar pill (to CTAP101 30 μg capsule) for weeks 13-26.</description>
  </arm_group>
  <arm_group>
    <arm_group_label>Sugar pill to CTAP101 30 μg</arm_group_label>
    <arm_group_type>Placebo Comparator</arm_group_type>
    <description>1 sugar pill to CTAP101 30 μg capsule daily at bedtime for 12 weeks, with an increase to 2 sugar pills to CTAP101 30 μg capsules daily at bedtime for weeks 13-26.</description>
  </arm_group>
  <intervention>
    <intervention_type>Drug</intervention_type>
    <intervention_name>CTAP101 30 μg capsules</intervention_name>
    <description>CTAP101 30 μg capsule taken daily at bedtime</description>
    <arm_group_label>CTAP101 30 μg capsules</arm_group_label>
    <other_name>Calcifediol</other_name>
  </intervention>
  <intervention>
    <intervention_type>Other</intervention_type>
    <intervention_name>Sugar pill to CTAP101 30 μg capsules</intervention_name>
    <description>Sugar pill capsule (placebo to CTAP101 30 μg capsule) taken daily at bedtime.</description>
    <arm_group_label>CTAP101 30 μg capsules</arm_group_label>
    <arm_group_label>Sugar pill to CTAP101 30 μg</arm_group_label>
    <other_name>Placebo</other_name>
  </intervention>
  <eligibility>
    <criteria>
      <textblock>
        Inclusion Criteria:&#xD;
&#xD;
          1. Urinary albumin excretion ≤ 3000 mcg/mg of creatinine&#xD;
&#xD;
          2. Stage 3 or 4 CKD&#xD;
&#xD;
          3. Plasma iPTH: ≥ 85 pg/mL and &lt; 500 pg/mL&#xD;
&#xD;
          4. Serum Ca: ≥ 8.4 mg/dL and &lt; 9.8 mg/dL&#xD;
&#xD;
          5. Serum P: ≥ 2.0 mg/dL and &lt; 5.0 mg/dL&#xD;
&#xD;
          6. Serum 25-hydroxyvitamin D: ≥ 10 ng/mL and &lt; 30 ng/mL.&#xD;
&#xD;
          7. Stable dose of Vitamin D therapy ≤ 1600 IU/day and receiving same dose for at least 2&#xD;
             months&#xD;
&#xD;
        Exclusion Criteria:&#xD;
&#xD;
          1. History of kidney transplant or parathyroidectomy&#xD;
&#xD;
          2. Spot urine calcium:creatinine ratio &gt; 0.2 (&gt;200 mg/g Cr)&#xD;
&#xD;
          3. Current serious illness, such as malignancy, HIV, liver disease, cardiovascular event&#xD;
             or hepatitis&#xD;
&#xD;
          4. Currently on dialysis&#xD;
&#xD;
          5. Use of pharmacological dose of ergocalciferol or cholecalciferol (≥ 50,000 IU mcg per&#xD;
             month) during the study&#xD;
      </textblock>
    </criteria>
    <gender>All</gender>
    <minimum_age>18 Years</minimum_age>
    <maximum_age>N/A</maximum_age>
    <healthy_volunteers>No</healthy_volunteers>
  </eligibility>
  <overall_official>
    <last_name>Joel Melnick, MD</last_name>
    <role>Study Director</role>
    <affiliation>OPKO Renal</affiliation>
  </overall_official>
  <location>
    <facility>
      <name>OPKO Renal</name>
      <address>
        <city>Bannockburn</city>
        <state>Illinois</state>
        <zip>60015</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location_countries>
    <country>United States</country>
  </location_countries>
  <verification_date>December 2019</verification_date>
  <study_first_submitted>October 6, 2012</study_first_submitted>
  <study_first_submitted_qc>October 9, 2012</study_first_submitted_qc>
  <study_first_posted type="Estimate">October 11, 2012</study_first_posted>
  <results_first_submitted>July 8, 2016</results_first_submitted>
  <results_first_submitted_qc>July 8, 2016</results_first_submitted_qc>
  <results_first_posted type="Estimate">August 17, 2016</results_first_posted>
  <disposition_first_submitted>October 22, 2015</disposition_first_submitted>
  <disposition_first_submitted_qc>October 22, 2015</disposition_first_submitted_qc>
  <disposition_first_posted type="Estimate">November 16, 2015</disposition_first_posted>
  <last_update_submitted>December 17, 2019</last_update_submitted>
  <last_update_submitted_qc>December 17, 2019</last_update_submitted_qc>
  <last_update_posted type="Actual">December 18, 2019</last_update_posted>
  <responsible_party>
    <responsible_party_type>Sponsor</responsible_party_type>
  </responsible_party>
  <keyword>Parathyroid Diseases</keyword>
  <keyword>Renal Insufficiency</keyword>
  <keyword>Kidney Failure, Chronic</keyword>
  <keyword>Hyperparathyroidism, Secondary</keyword>
  <keyword>Vitamin D</keyword>
  <keyword>Hyperparathyroidism</keyword>
  <keyword>Kidney Diseases</keyword>
  <keyword>Kidney Failure</keyword>
  <keyword>Renal Insufficiency, Chronic</keyword>
  <condition_browse>
    <!-- CAUTION:  The following MeSH terms are assigned with an imperfect algorithm            -->
    <mesh_term>Neoplasm Metastasis</mesh_term>
    <mesh_term>Kidney Diseases</mesh_term>
    <mesh_term>Renal Insufficiency, Chronic</mesh_term>
    <mesh_term>Vitamin D Deficiency</mesh_term>
    <mesh_term>Hyperparathyroidism</mesh_term>
    <mesh_term>Hyperparathyroidism, Secondary</mesh_term>
  </condition_browse>
  <intervention_browse>
    <!-- CAUTION:  The following MeSH terms are assigned with an imperfect algorithm            -->
    <mesh_term>Calcifediol</mesh_term>
  </intervention_browse>

  <clinical_results>

    <participant_flow>
      <recruitment_details>This multicenter study was conducted at 45 investigational sites within the US. Subjects were enrolled and treated from 04 February 2013 through 13 August 2014.</recruitment_details>
      <pre_assignment_details>Subjects were stratified by CKD stage and were randomized in a 2:1 ratio to receive a daily 30 μg oral dose of CTAP101 capsules (or matching placebo) for 12 weeks at bedtime. Subjects presenting on a regimen of bone metabolism therapy were to discontinue their prior treatment for at least 28 days' washout (except for bisphosphonates).</pre_assignment_details>
      <group_list>
        <group group_id="P1">
          <title>Sugar Pill to CTAP101 30 μg</title>
          <description>1 sugar pill to CTAP101 30 μg capsule daily at bedtime for 12 weeks, with an increase to 2 sugar pills to CTAP101 30 μg capsules daily at bedtime for weeks 13-26.&#xD;
Sugar pill to CTAP101 30 μg capsules: Sugar pill capsule (placebo to CTAP101 30 μg capsule) taken daily at bedtime.</description>
        </group>
        <group group_id="P2">
          <title>CTAP101 30 μg Capsules</title>
          <description>1 CTAP101 30 μg capsule daily at bedtime for 12 weeks, with a possible increase to 2 CTAP101 30 μg capsules daily at bedtime for study duration, if needed (26 weeks total). Subjects not requiring a dose increase after 12 weeks would receive 1 CTAP101 30 μg capsule and one sugar pill (to CTAP101 30 μg capsule) for weeks 13-26.&#xD;
CTAP101 30 μg capsules: CTAP101 30 μg capsule taken daily at bedtime&#xD;
Sugar pill to CTAP101 30 μg capsules: Sugar pill capsule (placebo to CTAP101 30 μg capsule) taken daily at bedtime.</description>
        </group>
      </group_list>
      <period_list>
        <period>
          <title>Overall Study</title>
          <milestone_list>
            <milestone>
              <title>STARTED</title>
              <participants_list>
                <participants group_id="P1" count="72"/>
                <participants group_id="P2" count="144"/>
              </participants_list>
            </milestone>
            <milestone>
              <title>Entered Extension Study</title>
              <participants_list>
                <participants group_id="P1" count="53"/>
                <participants group_id="P2" count="102"/>
              </participants_list>
            </milestone>
            <milestone>
              <title>COMPLETED</title>
              <participants_list>
                <participants group_id="P1" count="58"/>
                <participants group_id="P2" count="121"/>
              </participants_list>
            </milestone>
            <milestone>
              <title>NOT COMPLETED</title>
              <participants_list>
                <participants group_id="P1" count="14"/>
                <participants group_id="P2" count="23"/>
              </participants_list>
            </milestone>
          </milestone_list>
          <drop_withdraw_reason_list>
            <drop_withdraw_reason>
              <title>Withdrawal by Subject</title>
              <participants_list>
                <participants group_id="P1" count="5"/>
                <participants group_id="P2" count="7"/>
              </participants_list>
            </drop_withdraw_reason>
            <drop_withdraw_reason>
              <title>Lost to Follow-up</title>
              <participants_list>
                <participants group_id="P1" count="2"/>
                <participants group_id="P2" count="5"/>
              </participants_list>
            </drop_withdraw_reason>
            <drop_withdraw_reason>
              <title>Adverse Event</title>
              <participants_list>
                <participants group_id="P1" count="4"/>
                <participants group_id="P2" count="7"/>
              </participants_list>
            </drop_withdraw_reason>
            <drop_withdraw_reason>
              <title>Protocol Violation</title>
              <participants_list>
                <participants group_id="P1" count="1"/>
                <participants group_id="P2" count="0"/>
              </participants_list>
            </drop_withdraw_reason>
            <drop_withdraw_reason>
              <title>Subject noncompliance</title>
              <participants_list>
                <participants group_id="P1" count="2"/>
                <participants group_id="P2" count="3"/>
              </participants_list>
            </drop_withdraw_reason>
            <drop_withdraw_reason>
              <title>Serum Calcium &gt;/= 11.0 mg/dL, repeat con</title>
              <participants_list>
                <participants group_id="P1" count="0"/>
                <participants group_id="P2" count="1"/>
              </participants_list>
            </drop_withdraw_reason>
          </drop_withdraw_reason_list>
        </period>
      </period_list>
    </participant_flow>

    <baseline>
      <group_list>
        <group group_id="B1">
          <title>Sugar Pill to CTAP101 30 μg</title>
          <description>1 sugar pill to CTAP101 30 μg capsule daily at bedtime for 12 weeks, with an increase to 2 sugar pills to CTAP101 30 μg capsules daily at bedtime for weeks 13-26.&#xD;
Sugar pill to CTAP101 30 μg capsules: Sugar pill capsule (placebo to CTAP101 30 μg capsule) taken daily at bedtime.</description>
        </group>
        <group group_id="B2">
          <title>CTAP101 30 μg Capsules</title>
          <description>1 CTAP101 30 μg capsule daily at bedtime for 12 weeks, with a possible increase to 2 CTAP101 30 μg capsules daily at bedtime for study duration, if needed (26 weeks total). Subjects not requiring a dose increase after 12 weeks would receive 1 CTAP101 30 μg capsule and one sugar pill (to CTAP101 30 μg capsule) for weeks 13-26.&#xD;
CTAP101 30 μg capsules: CTAP101 30 μg capsule taken daily at bedtime&#xD;
Sugar pill to CTAP101 30 μg capsules: Sugar pill capsule (placebo to CTAP101 30 μg capsule) taken daily at bedtime.</description>
        </group>
        <group group_id="B3">
          <title>Total</title>
          <description>Total of all reporting groups</description>
        </group>
      </group_list>
      <analyzed_list>
        <analyzed>
          <units>Participants</units>
          <scope>Overall</scope>
          <count_list>
            <count group_id="B1" value="72"/>
            <count group_id="B2" value="144"/>
            <count group_id="B3" value="216"/>
          </count_list>
        </analyzed>
      </analyzed_list>
      <measure_list>
        <measure>
          <title>Age</title>
          <units>years</units>
          <param>Mean</param>
          <dispersion>Standard Deviation</dispersion>
          <class_list>
            <class>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="B1" value="65.3" spread="10.06"/>
                    <measurement group_id="B2" value="66.8" spread="10.90"/>
                    <measurement group_id="B3" value="66.3" spread="10.63"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
          </class_list>
        </measure>
        <measure>
          <title>Sex: Female, Male</title>
          <units>Participants</units>
          <param>Count of Participants</param>
          <class_list>
            <class>
              <category_list>
                <category>
                  <title>Female</title>
                  <measurement_list>
                    <measurement group_id="B1" value="39"/>
                    <measurement group_id="B2" value="71"/>
                    <measurement group_id="B3" value="110"/>
                  </measurement_list>
                </category>
                <category>
                  <title>Male</title>
                  <measurement_list>
                    <measurement group_id="B1" value="33"/>
                    <measurement group_id="B2" value="73"/>
                    <measurement group_id="B3" value="106"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
          </class_list>
        </measure>
        <measure>
          <title>Region of Enrollment</title>
          <units>participants</units>
          <param>Number</param>
          <class_list>
            <class>
              <title>United States</title>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="B1" value="72"/>
                    <measurement group_id="B2" value="144"/>
                    <measurement group_id="B3" value="216"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
          </class_list>
        </measure>
      </measure_list>
    </baseline>

    <outcome_list>
      <outcome>
        <type>Primary</type>
        <title>Number of Participants in the Intent to Treat Population With Decrease in Plasma Intact Parathyroid Hormone (iPTH) of ≥30% From Pre-treatment Baseline</title>
        <description>Number of subjects in the intent to treat population attaining mean decrease in plasma intact Parathyroid Hormone (iPTH) of ≥30% from P\pre-treatment baseline in the efficacy assessment phase (EAP) referred to as responders</description>
        <time_frame>Approximately 6 months</time_frame>
        <population>Intent to treat</population>
        <group_list>
          <group group_id="O1">
            <title>Sugar Pill to CTAP101 30 μg</title>
            <description>1 sugar pill to CTAP101 30 μg capsule daily at bedtime for 12 weeks, with an increase to 2 sugar pills to CTAP101 30 μg capsules daily at bedtime for weeks 13-26.&#xD;
Sugar pill to CTAP101 30 μg capsules: Sugar pill capsule (placebo to CTAP101 30 μg capsule) taken daily at bedtime.</description>
          </group>
          <group group_id="O2">
            <title>CTAP101 30 μg Capsules</title>
            <description>1 CTAP101 30 μg capsule daily at bedtime for 12 weeks, with a possible increase to 2 CTAP101 30 μg capsules daily at bedtime for study duration, if needed (26 weeks total). Subjects not requiring a dose increase after 12 weeks would receive 1 CTAP101 30 μg capsule and one sugar pill (to CTAP101 30 μg capsule) for weeks 13-26.&#xD;
CTAP101 30 μg capsules: CTAP101 30 μg capsule taken daily at bedtime&#xD;
Sugar pill to CTAP101 30 μg capsules: Sugar pill capsule (placebo to CTAP101 30 μg capsule) taken daily at bedtime.</description>
          </group>
        </group_list>
        <measure>
          <title>Number of Participants in the Intent to Treat Population With Decrease in Plasma Intact Parathyroid Hormone (iPTH) of ≥30% From Pre-treatment Baseline</title>
          <description>Number of subjects in the intent to treat population attaining mean decrease in plasma intact Parathyroid Hormone (iPTH) of ≥30% from P\pre-treatment baseline in the efficacy assessment phase (EAP) referred to as responders</description>
          <population>Intent to treat</population>
          <units>participants</units>
          <param>Number</param>
          <analyzed_list>
            <analyzed>
              <units>Participants</units>
              <scope>Measure</scope>
              <count_list>
                <count group_id="O1" value="72"/>
                <count group_id="O2" value="144"/>
              </count_list>
            </analyzed>
          </analyzed_list>
          <class_list>
            <class>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="5"/>
                    <measurement group_id="O2" value="49"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
          </class_list>
        </measure>
        <analysis_list>
          <analysis>
            <group_id_list>
              <group_id>O1</group_id>
              <group_id>O2</group_id>
            </group_id_list>
            <non_inferiority_type>Superiority or Other (legacy)</non_inferiority_type>
            <p_value>&lt;0.0001</p_value>
            <method>Cochran-Mantel-Haenszel</method>
          </analysis>
        </analysis_list>
      </outcome>
      <outcome>
        <type>Secondary</type>
        <title>Number of Participants in the Per Protocol Population With Decrease in Plasma Intact Parathyroid Hormone (iPTH) of ≥30% From Pre-treatment Baseline</title>
        <description>Number of subjects in the per protocol population attaining a mean decrease in plasma intact parathyroid hormone (iPTH) of ≥30% from pre-treatment baseline in the efficacy assessment phase (EAP), referred to as responders</description>
        <time_frame>Approximately 6 months</time_frame>
        <population>Per protocol</population>
        <group_list>
          <group group_id="O1">
            <title>Sugar Pill to CTAP101 30 μg</title>
            <description>1 sugar pill to CTAP101 30 μg capsule daily at bedtime for 12 weeks, with an increase to 2 sugar pills to CTAP101 30 μg capsules daily at bedtime for weeks 13-26.&#xD;
Sugar pill to CTAP101 30 μg capsules: Sugar pill capsule (placebo to CTAP101 30 μg capsule) taken daily at bedtime.</description>
          </group>
          <group group_id="O2">
            <title>CTAP101 30 μg Capsules</title>
            <description>1 CTAP101 30 μg capsule daily at bedtime for 12 weeks, with a possible increase to 2 CTAP101 30 μg capsules daily at bedtime for study duration, if needed (26 weeks total). Subjects not requiring a dose increase after 12 weeks would receive 1 CTAP101 30 μg capsule and one sugar pill (to CTAP101 30 μg capsule) for weeks 13-26.&#xD;
CTAP101 30 μg capsules: CTAP101 30 μg capsule taken daily at bedtime&#xD;
Sugar pill to CTAP101 30 μg capsules: Sugar pill capsule (placebo to CTAP101 30 μg capsule) taken daily at bedtime.</description>
          </group>
        </group_list>
        <measure>
          <title>Number of Participants in the Per Protocol Population With Decrease in Plasma Intact Parathyroid Hormone (iPTH) of ≥30% From Pre-treatment Baseline</title>
          <description>Number of subjects in the per protocol population attaining a mean decrease in plasma intact parathyroid hormone (iPTH) of ≥30% from pre-treatment baseline in the efficacy assessment phase (EAP), referred to as responders</description>
          <population>Per protocol</population>
          <units>participants</units>
          <param>Number</param>
          <analyzed_list>
            <analyzed>
              <units>Participants</units>
              <scope>Measure</scope>
              <count_list>
                <count group_id="O1" value="60"/>
                <count group_id="O2" value="119"/>
              </count_list>
            </analyzed>
          </analyzed_list>
          <class_list>
            <class>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="5"/>
                    <measurement group_id="O2" value="47"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
          </class_list>
        </measure>
        <analysis_list>
          <analysis>
            <group_id_list>
              <group_id>O1</group_id>
            </group_id_list>
            <non_inferiority_type>Superiority or Other (legacy)</non_inferiority_type>
            <p_value>&lt;0.0001</p_value>
            <method>Cochran-Mantel-Haenszel</method>
          </analysis>
        </analysis_list>
      </outcome>
      <outcome>
        <type>Secondary</type>
        <title>Subjects in the Intent to Treat Population With Normal Serum Total 25-hydroxyvitamin D</title>
        <description>Subjects in the intent to treat population with normal serum total 25-hydroxyvitamin D (&gt;/= 30 ng/dL)</description>
        <time_frame>Approximately 6 months</time_frame>
        <population>Intent to treat</population>
        <group_list>
          <group group_id="O1">
            <title>Sugar Pill to CTAP101 30 μg</title>
            <description>1 sugar pill to CTAP101 30 μg capsule daily at bedtime for 12 weeks, with an increase to 2 sugar pills to CTAP101 30 μg capsules daily at bedtime for weeks 13-26.&#xD;
Sugar pill to CTAP101 30 μg capsules: Sugar pill capsule (placebo to CTAP101 30 μg capsule) taken daily at bedtime.</description>
          </group>
          <group group_id="O2">
            <title>CTAP101 30 μg Capsules</title>
            <description>1 CTAP101 30 μg capsule daily at bedtime for 12 weeks, with a possible increase to 2 CTAP101 30 μg capsules daily at bedtime for study duration, if needed (26 weeks total). Subjects not requiring a dose increase after 12 weeks would receive 1 CTAP101 30 μg capsule and one sugar pill (to CTAP101 30 μg capsule) for weeks 13-26.&#xD;
CTAP101 30 μg capsules: CTAP101 30 μg capsule taken daily at bedtime&#xD;
Sugar pill to CTAP101 30 μg capsules: Sugar pill capsule (placebo to CTAP101 30 μg capsule) taken daily at bedtime.</description>
          </group>
        </group_list>
        <measure>
          <title>Subjects in the Intent to Treat Population With Normal Serum Total 25-hydroxyvitamin D</title>
          <description>Subjects in the intent to treat population with normal serum total 25-hydroxyvitamin D (&gt;/= 30 ng/dL)</description>
          <population>Intent to treat</population>
          <units>participants</units>
          <param>Number</param>
          <analyzed_list>
            <analyzed>
              <units>Participants</units>
              <scope>Measure</scope>
              <count_list>
                <count group_id="O1" value="72"/>
                <count group_id="O2" value="144"/>
              </count_list>
            </analyzed>
          </analyzed_list>
          <class_list>
            <class>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="5"/>
                    <measurement group_id="O2" value="120"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
          </class_list>
        </measure>
        <analysis_list>
          <analysis>
            <group_id_list>
              <group_id>O1</group_id>
              <group_id>O2</group_id>
            </group_id_list>
            <non_inferiority_type>Superiority or Other (legacy)</non_inferiority_type>
            <p_value>&lt;0.0001</p_value>
            <method>Cochran-Mantel-Haenszel</method>
          </analysis>
        </analysis_list>
      </outcome>
      <outcome>
        <type>Secondary</type>
        <title>Subjects in the Per Protocol Population With Normal Serum Total 25-hydroxyvitamin D</title>
        <description>Subjects in the per protocol population with normal serum total 25-hydroxyvitamin D (&gt;/= 30 ng/mL)</description>
        <time_frame>Approximately 6 months</time_frame>
        <population>Per protocol</population>
        <group_list>
          <group group_id="O1">
            <title>Sugar Pill to CTAP101 30 μg</title>
            <description>1 sugar pill to CTAP101 30 μg capsule daily at bedtime for 12 weeks, with an increase to 2 sugar pills to CTAP101 30 μg capsules daily at bedtime for weeks 13-26.&#xD;
Sugar pill to CTAP101 30 μg capsules: Sugar pill capsule (placebo to CTAP101 30 μg capsule) taken daily at bedtime.</description>
          </group>
          <group group_id="O2">
            <title>CTAP101 30 μg Capsules</title>
            <description>1 CTAP101 30 μg capsule daily at bedtime for 12 weeks, with a possible increase to 2 CTAP101 30 μg capsules daily at bedtime for study duration, if needed (26 weeks total). Subjects not requiring a dose increase after 12 weeks would receive 1 CTAP101 30 μg capsule and one sugar pill (to CTAP101 30 μg capsule) for weeks 13-26.&#xD;
CTAP101 30 μg capsules: CTAP101 30 μg capsule taken daily at bedtime&#xD;
Sugar pill to CTAP101 30 μg capsules: Sugar pill capsule (placebo to CTAP101 30 μg capsule) taken daily at bedtime.</description>
          </group>
        </group_list>
        <measure>
          <title>Subjects in the Per Protocol Population With Normal Serum Total 25-hydroxyvitamin D</title>
          <description>Subjects in the per protocol population with normal serum total 25-hydroxyvitamin D (&gt;/= 30 ng/mL)</description>
          <population>Per protocol</population>
          <units>participants</units>
          <param>Number</param>
          <analyzed_list>
            <analyzed>
              <units>Participants</units>
              <scope>Measure</scope>
              <count_list>
                <count group_id="O1" value="60"/>
                <count group_id="O2" value="119"/>
              </count_list>
            </analyzed>
          </analyzed_list>
          <class_list>
            <class>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="5"/>
                    <measurement group_id="O2" value="116"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
          </class_list>
        </measure>
        <analysis_list>
          <analysis>
            <group_id_list>
              <group_id>O1</group_id>
              <group_id>O2</group_id>
            </group_id_list>
            <non_inferiority_type>Superiority or Other (legacy)</non_inferiority_type>
            <p_value>&lt;0.0001</p_value>
            <method>Cochran-Mantel-Haenszel</method>
          </analysis>
        </analysis_list>
      </outcome>
    </outcome_list>

    <reported_events>
      <time_frame>6 months from randomization to end of therapy</time_frame>
      <group_list>
        <group group_id="E1">
          <title>Sugar Pill to CTAP101 30 μg</title>
          <description>1 sugar pill to CTAP101 30 μg capsule daily at bedtime for 12 weeks, with an increase to 2 sugar pills to CTAP101 30 μg capsules daily at bedtime for weeks 13-26.&#xD;
Sugar pill to CTAP101 30 μg capsules: Sugar pill capsule (placebo to CTAP101 30 μg capsule) taken daily at bedtime.</description>
        </group>
        <group group_id="E2">
          <title>CTAP101 30 μg Capsules</title>
          <description>1 CTAP101 30 μg capsule daily at bedtime for 12 weeks, with a possible increase to 2 CTAP101 30 μg capsules daily at bedtime for study duration, if needed (26 weeks total). Subjects not requiring a dose increase after 12 weeks would receive 1 CTAP101 30 μg capsule and one sugar pill (to CTAP101 30 μg capsule) for weeks 13-26.&#xD;
CTAP101 30 μg capsules: CTAP101 30 μg capsule taken daily at bedtime&#xD;
Sugar pill to CTAP101 30 μg capsules: Sugar pill capsule (placebo to CTAP101 30 μg capsule) taken daily at bedtime.</description>
        </group>
      </group_list>
      <serious_events>
        <default_vocab>MedDRA 15.0</default_vocab>
        <default_assessment>Non-systematic Assessment</default_assessment>
        <category_list>
          <category>
            <title>Total</title>
            <event_list>
              <event>
                <sub_title>Total, serious adverse events</sub_title>
                <counts group_id="E1" subjects_affected="11" subjects_at_risk="72"/>
                <counts group_id="E2" subjects_affected="22" subjects_at_risk="144"/>
              </event>
            </event_list>
          </category>
          <category>
            <title>Blood and lymphatic system disorders</title>
            <event_list>
              <event>
                <sub_title>Anaemia</sub_title>
                <counts group_id="E1" events="0" subjects_affected="0" subjects_at_risk="72"/>
                <counts group_id="E2" events="2" subjects_affected="2" subjects_at_risk="144"/>
              </event>
            </event_list>
          </category>
          <category>
            <title>Cardiac disorders</title>
            <event_list>
              <event>
                <sub_title>Acute myocardial infarction</sub_title>
                <counts group_id="E1" events="1" subjects_affected="1" subjects_at_risk="72"/>
                <counts group_id="E2" events="1" subjects_affected="1" subjects_at_risk="144"/>
              </event>
              <event>
                <sub_title>Cardiac arrest</sub_title>
                <counts group_id="E1" events="0" subjects_affected="0" subjects_at_risk="72"/>
                <counts group_id="E2" events="1" subjects_affected="1" subjects_at_risk="144"/>
              </event>
              <event>
                <sub_title>Cardiac disorder</sub_title>
                <counts group_id="E1" events="0" subjects_affected="0" subjects_at_risk="72"/>
                <counts group_id="E2" events="1" subjects_affected="1" subjects_at_risk="144"/>
              </event>
              <event>
                <sub_title>Cardiac failure</sub_title>
                <counts group_id="E1" events="1" subjects_affected="1" subjects_at_risk="72"/>
                <counts group_id="E2" events="1" subjects_affected="1" subjects_at_risk="144"/>
              </event>
              <event>
                <sub_title>Cardiac failure acute</sub_title>
                <counts group_id="E1" events="0" subjects_affected="0" subjects_at_risk="72"/>
                <counts group_id="E2" events="1" subjects_affected="1" subjects_at_risk="144"/>
              </event>
              <event>
                <sub_title>Cardiac failure congestive</sub_title>
                <counts group_id="E1" events="0" subjects_affected="0" subjects_at_risk="72"/>
                <counts group_id="E2" events="3" subjects_affected="3" subjects_at_risk="144"/>
              </event>
              <event>
                <sub_title>Myocardial infarction</sub_title>
                <counts group_id="E1" events="0" subjects_affected="0" subjects_at_risk="72"/>
                <counts group_id="E2" events="1" subjects_affected="1" subjects_at_risk="144"/>
              </event>
            </event_list>
          </category>
          <category>
            <title>General disorders</title>
            <event_list>
              <event>
                <sub_title>Asthenia</sub_title>
                <counts group_id="E1" events="1" subjects_affected="1" subjects_at_risk="72"/>
                <counts group_id="E2" events="0" subjects_affected="0" subjects_at_risk="144"/>
              </event>
              <event>
                <sub_title>Chest pain</sub_title>
                <counts group_id="E1" events="1" subjects_affected="1" subjects_at_risk="72"/>
                <counts group_id="E2" events="1" subjects_affected="1" subjects_at_risk="144"/>
              </event>
              <event>
                <sub_title>Device malfunction</sub_title>
                <counts group_id="E1" events="0" subjects_affected="0" subjects_at_risk="72"/>
                <counts group_id="E2" events="1" subjects_affected="1" subjects_at_risk="144"/>
              </event>
            </event_list>
          </category>
          <category>
            <title>Hepatobiliary disorders</title>
            <event_list>
              <event>
                <sub_title>Cholelitiasis</sub_title>
                <counts group_id="E1" events="0" subjects_affected="0" subjects_at_risk="72"/>
                <counts group_id="E2" events="1" subjects_affected="1" subjects_at_risk="144"/>
              </event>
            </event_list>
          </category>
          <category>
            <title>Infections and infestations</title>
            <event_list>
              <event>
                <sub_title>Cellulitis</sub_title>
                <counts group_id="E1" events="1" subjects_affected="1" subjects_at_risk="72"/>
                <counts group_id="E2" events="0" subjects_affected="0" subjects_at_risk="144"/>
              </event>
              <event>
                <sub_title>Localized infection</sub_title>
                <counts group_id="E1" events="0" subjects_affected="0" subjects_at_risk="72"/>
                <counts group_id="E2" events="1" subjects_affected="1" subjects_at_risk="144"/>
              </event>
              <event>
                <sub_title>Pneumonia</sub_title>
                <counts group_id="E1" events="1" subjects_affected="1" subjects_at_risk="72"/>
                <counts group_id="E2" events="3" subjects_affected="3" subjects_at_risk="144"/>
              </event>
              <event>
                <sub_title>Pneumonia bacterial</sub_title>
                <counts group_id="E1" events="1" subjects_affected="1" subjects_at_risk="72"/>
                <counts group_id="E2" events="0" subjects_affected="0" subjects_at_risk="144"/>
              </event>
              <event>
                <sub_title>Sepsis</sub_title>
                <counts group_id="E1" events="2" subjects_affected="2" subjects_at_risk="72"/>
                <counts group_id="E2" events="0" subjects_affected="0" subjects_at_risk="144"/>
              </event>
              <event>
                <sub_title>Urinary tract infection</sub_title>
                <counts group_id="E1" events="0" subjects_affected="0" subjects_at_risk="72"/>
                <counts group_id="E2" events="1" subjects_affected="1" subjects_at_risk="144"/>
              </event>
            </event_list>
          </category>
          <category>
            <title>Injury, poisoning and procedural complications</title>
            <event_list>
              <event>
                <sub_title>Fall</sub_title>
                <counts group_id="E1" events="0" subjects_affected="0" subjects_at_risk="72"/>
                <counts group_id="E2" events="1" subjects_affected="1" subjects_at_risk="144"/>
              </event>
            </event_list>
          </category>
          <category>
            <title>Investigations</title>
            <event_list>
              <event>
                <sub_title>Blood creatinine increased</sub_title>
                <counts group_id="E1" events="0" subjects_affected="0" subjects_at_risk="72"/>
                <counts group_id="E2" events="3" subjects_affected="3" subjects_at_risk="144"/>
              </event>
            </event_list>
          </category>
          <category>
            <title>Metabolism and nutrition disorders</title>
            <event_list>
              <event>
                <sub_title>Dehydration</sub_title>
                <counts group_id="E1" events="2" subjects_affected="2" subjects_at_risk="72"/>
                <counts group_id="E2" events="1" subjects_affected="1" subjects_at_risk="144"/>
              </event>
              <event>
                <sub_title>Fluid overload</sub_title>
                <counts group_id="E1" events="0" subjects_affected="0" subjects_at_risk="72"/>
                <counts group_id="E2" events="1" subjects_affected="1" subjects_at_risk="144"/>
              </event>
              <event>
                <sub_title>Hyperkalaemia</sub_title>
                <counts group_id="E1" events="1" subjects_affected="1" subjects_at_risk="72"/>
                <counts group_id="E2" events="2" subjects_affected="2" subjects_at_risk="144"/>
              </event>
              <event>
                <sub_title>Hypoglycaemia</sub_title>
                <counts group_id="E1" events="0" subjects_affected="0" subjects_at_risk="72"/>
                <counts group_id="E2" events="2" subjects_affected="2" subjects_at_risk="144"/>
              </event>
            </event_list>
          </category>
          <category>
            <title>Musculoskeletal and connective tissue disorders</title>
            <event_list>
              <event>
                <sub_title>Musculoskeletal chest pain</sub_title>
                <counts group_id="E1" events="1" subjects_affected="1" subjects_at_risk="72"/>
                <counts group_id="E2" events="0" subjects_affected="0" subjects_at_risk="144"/>
              </event>
              <event>
                <sub_title>Rhabdomyolysis</sub_title>
                <counts group_id="E1" events="0" subjects_affected="0" subjects_at_risk="72"/>
                <counts group_id="E2" events="1" subjects_affected="1" subjects_at_risk="144"/>
              </event>
            </event_list>
          </category>
          <category>
            <title>Nervous system disorders</title>
            <event_list>
              <event>
                <sub_title>Cerebrovascular accident</sub_title>
                <counts group_id="E1" events="1" subjects_affected="1" subjects_at_risk="72"/>
                <counts group_id="E2" events="1" subjects_affected="1" subjects_at_risk="144"/>
              </event>
              <event>
                <sub_title>Dizziness</sub_title>
                <counts group_id="E1" events="0" subjects_affected="0" subjects_at_risk="72"/>
                <counts group_id="E2" events="1" subjects_affected="1" subjects_at_risk="144"/>
              </event>
              <event>
                <sub_title>Encephalopathy</sub_title>
                <counts group_id="E1" events="0" subjects_affected="0" subjects_at_risk="72"/>
                <counts group_id="E2" events="1" subjects_affected="1" subjects_at_risk="144"/>
              </event>
              <event>
                <sub_title>Myasthenia gravis crisis</sub_title>
                <counts group_id="E1" events="0" subjects_affected="0" subjects_at_risk="72"/>
                <counts group_id="E2" events="1" subjects_affected="1" subjects_at_risk="144"/>
              </event>
              <event>
                <sub_title>Status epilepticus</sub_title>
                <counts group_id="E1" events="1" subjects_affected="1" subjects_at_risk="72"/>
                <counts group_id="E2" events="0" subjects_affected="0" subjects_at_risk="144"/>
              </event>
              <event>
                <sub_title>Syncope</sub_title>
                <counts group_id="E1" events="0" subjects_affected="0" subjects_at_risk="72"/>
                <counts group_id="E2" events="2" subjects_affected="2" subjects_at_risk="144"/>
              </event>
              <event>
                <sub_title>Thalamic infarction</sub_title>
                <counts group_id="E1" events="1" subjects_affected="1" subjects_at_risk="72"/>
                <counts group_id="E2" events="0" subjects_affected="0" subjects_at_risk="144"/>
              </event>
            </event_list>
          </category>
          <category>
            <title>Psychiatric disorders</title>
            <event_list>
              <event>
                <sub_title>Mental status changes</sub_title>
                <counts group_id="E1" events="1" subjects_affected="1" subjects_at_risk="72"/>
                <counts group_id="E2" events="0" subjects_affected="0" subjects_at_risk="144"/>
              </event>
            </event_list>
          </category>
          <category>
            <title>Renal and urinary disorders</title>
            <event_list>
              <event>
                <sub_title>Urinary tract obstruction</sub_title>
                <counts group_id="E1" events="0" subjects_affected="0" subjects_at_risk="72"/>
                <counts group_id="E2" events="1" subjects_affected="1" subjects_at_risk="144"/>
              </event>
            </event_list>
          </category>
          <category>
            <title>Respiratory, thoracic and mediastinal disorders</title>
            <event_list>
              <event>
                <sub_title>Acute pulmonary oedema</sub_title>
                <counts group_id="E1" events="1" subjects_affected="1" subjects_at_risk="72"/>
                <counts group_id="E2" events="0" subjects_affected="0" subjects_at_risk="144"/>
              </event>
              <event>
                <sub_title>Acute respiratory failure</sub_title>
                <counts group_id="E1" events="0" subjects_affected="0" subjects_at_risk="72"/>
                <counts group_id="E2" events="1" subjects_affected="1" subjects_at_risk="144"/>
              </event>
              <event>
                <sub_title>Respiratory failure</sub_title>
                <counts group_id="E1" events="1" subjects_affected="1" subjects_at_risk="72"/>
                <counts group_id="E2" events="0" subjects_affected="0" subjects_at_risk="144"/>
              </event>
            </event_list>
          </category>
          <category>
            <title>Skin and subcutaneous tissue disorders</title>
            <event_list>
              <event>
                <sub_title>Angioedema</sub_title>
                <counts group_id="E1" events="0" subjects_affected="0" subjects_at_risk="72"/>
                <counts group_id="E2" events="1" subjects_affected="1" subjects_at_risk="144"/>
              </event>
            </event_list>
          </category>
          <category>
            <title>Vascular disorders</title>
            <event_list>
              <event>
                <sub_title>Deep vein thrombosis</sub_title>
                <counts group_id="E1" events="0" subjects_affected="0" subjects_at_risk="72"/>
                <counts group_id="E2" events="1" subjects_affected="1" subjects_at_risk="144"/>
              </event>
              <event>
                <sub_title>Hypertensive emergency</sub_title>
                <counts group_id="E1" events="0" subjects_affected="0" subjects_at_risk="72"/>
                <counts group_id="E2" events="1" subjects_affected="1" subjects_at_risk="144"/>
              </event>
            </event_list>
          </category>
        </category_list>
      </serious_events>
      <other_events>
        <frequency_threshold>5</frequency_threshold>
        <default_vocab>MedDRA 15.0</default_vocab>
        <default_assessment>Non-systematic Assessment</default_assessment>
        <category_list>
          <category>
            <title>Total</title>
            <event_list>
              <event>
                <sub_title>Total, other adverse events</sub_title>
                <counts group_id="E1" subjects_affected="18" subjects_at_risk="72"/>
                <counts group_id="E2" subjects_affected="32" subjects_at_risk="144"/>
              </event>
            </event_list>
          </category>
          <category>
            <title>Gastrointestinal disorders</title>
            <event_list>
              <event>
                <sub_title>Constipation</sub_title>
                <counts group_id="E1" events="4" subjects_affected="4" subjects_at_risk="72"/>
                <counts group_id="E2" events="5" subjects_affected="5" subjects_at_risk="144"/>
              </event>
            </event_list>
          </category>
          <category>
            <title>General disorders</title>
            <event_list>
              <event>
                <sub_title>Oedema peripheral</sub_title>
                <counts group_id="E1" events="3" subjects_affected="3" subjects_at_risk="72"/>
                <counts group_id="E2" events="9" subjects_affected="9" subjects_at_risk="144"/>
              </event>
            </event_list>
          </category>
          <category>
            <title>Infections and infestations</title>
            <event_list>
              <event>
                <sub_title>Nasopharyngitis</sub_title>
                <counts group_id="E1" events="1" subjects_affected="1" subjects_at_risk="72"/>
                <counts group_id="E2" events="8" subjects_affected="8" subjects_at_risk="144"/>
              </event>
            </event_list>
          </category>
          <category>
            <title>Metabolism and nutrition disorders</title>
            <event_list>
              <event>
                <sub_title>Gout</sub_title>
                <counts group_id="E1" events="5" subjects_affected="5" subjects_at_risk="72"/>
                <counts group_id="E2" events="6" subjects_affected="6" subjects_at_risk="144"/>
              </event>
              <event>
                <sub_title>Metabolic acidosis</sub_title>
                <counts group_id="E1" events="4" subjects_affected="4" subjects_at_risk="72"/>
                <counts group_id="E2" events="1" subjects_affected="1" subjects_at_risk="144"/>
              </event>
            </event_list>
          </category>
          <category>
            <title>Musculoskeletal and connective tissue disorders</title>
            <event_list>
              <event>
                <sub_title>Pain in extremity</sub_title>
                <counts group_id="E1" events="4" subjects_affected="4" subjects_at_risk="72"/>
                <counts group_id="E2" events="3" subjects_affected="3" subjects_at_risk="144"/>
              </event>
            </event_list>
          </category>
          <category>
            <title>Vascular disorders</title>
            <event_list>
              <event>
                <sub_title>Hypertension</sub_title>
                <counts group_id="E1" events="6" subjects_affected="6" subjects_at_risk="72"/>
                <counts group_id="E2" events="8" subjects_affected="8" subjects_at_risk="144"/>
              </event>
            </event_list>
          </category>
        </category_list>
      </other_events>
    </reported_events>

    <certain_agreements>
      <pi_employee>Principal Investigators are NOT employed by the organization sponsoring the study.</pi_employee>
      <restrictive_agreement>The only disclosure restriction on the PI is that the sponsor can review results communications prior to public release and can embargo communications regarding trial results for a period that is less than or equal to 60 days. The sponsor cannot require changes to the communication and cannot extend the embargo.</restrictive_agreement>
    </certain_agreements>
    <point_of_contact>
      <name_or_title>Douglass Laidlaw, PhD, Vice President, Medical Affairs</name_or_title>
      <organization>OPKO Health, Inc.</organization>
      <phone>305-575-4172</phone>
      <email>dlaidlaw@opko.com</email>
    </point_of_contact>
  </clinical_results>
</clinical_study>


download date: ClinicalTrials.gov processed this data on September 20, 2023
link text: Link to the current ClinicalTrials.gov record.
url: https://clinicaltrials.gov/ct2/show/NCT01704079
org study id: CTAP101-CL-3002
nct id: NCT01704079
lead sponsor: 
has dmc: Yes
This study will evaluate the efficacy of CTAP101 Capsules versus placebo in reducing intact
      parathyroid hormone (iPTH) by at least 30% from pretreatment baseline; safety and
      tolerability of CTAP101 will also be evaluated
This is a phase 3, multi-center, randomized, double-blind, placebo-controlled study to
      evaluate the efficacy and safety of CTAP101 Capsules administered for 26 weeks to treat SHPT
      in subjects ≥18 years of age with stage 3 or 4 CKD and vitamin D insufficiency. The study
      will be conducted at approximately 40 sites within the United States (US). Approximately 550
      subjects will be screened to randomize approximately 210 eligible subjects, stratified by CKD
      stage (approximately 105 subjects in each stage) in a 2:1 ratio to receive either CTAP101
      Capsules or matching placebo. An Interactive Voice Response System (IVRS) will provide study
      treatment group assignments using the computer-generated randomization code provided by the
      IVRS vendor. Subjects will receive an initial daily dose of 1 capsule (CTAP101 Capsules, 30
      µg, or matching placebo) for the first 12 weeks. After l2 weeks (visit 8) and per pre-defined
      criteria, the dose may be increased in a blinded fashion by the IVRS with oversight by an
      independent medical monitor.
allocation: Randomized
intervention model: Parallel Assignment
primary purpose: Treatment
masking: Double (Participant, Investigator)
measure: Number of Participants in the Intent to Treat Population With Decrease in Plasma Intact Parathyroid Hormone (iPTH) of ≥30% From Pre-treatment Baseline
time frame: Approximately 6 months
description: Number of subjects in the intent to treat population attaining mean decrease in plasma intact Parathyroid Hormone (iPTH) of ≥30% from P\pre-treatment baseline in the efficacy assessment phase (EAP) referred to as responders
measure: Number of Participants in the Per Protocol Population With Decrease in Plasma Intact Parathyroid Hormone (iPTH) of ≥30% From Pre-treatment Baseline
time frame: Approximately 6 months
description: Number of subjects in the per protocol population attaining a mean decrease in plasma intact parathyroid hormone (iPTH) of ≥30% from pre-treatment baseline in the efficacy assessment phase (EAP), referred to as responders
measure: Subjects in the Intent to Treat Population With Normal Serum Total 25-hydroxyvitamin D
time frame: Approximately 6 months
description: Subjects in the intent to treat population with normal serum total 25-hydroxyvitamin D (>/= 30 ng/dL)
measure: Subjects in the Per Protocol Population With Normal Serum Total 25-hydroxyvitamin D
time frame: Approximately 6 months
description: Subjects in the per protocol population with normal serum total 25-hydroxyvitamin D (>/= 30 ng/mL)
arm group label: CTAP101 30 μg capsules
arm group type: Active Comparator
description: 1 CTAP101 30 μg capsule daily at bedtime for 12 weeks, with a possible increase to 2 CTAP101 30 μg capsules daily at bedtime for study duration, if needed (26 weeks total). Subjects not requiring a dose increase after 12 weeks would receive 1 CTAP101 30 μg capsule and one sugar pill (to CTAP101 30 μg capsule) for weeks 13-26.
arm group label: Sugar pill to CTAP101 30 μg
arm group type: Placebo Comparator
description: 1 sugar pill to CTAP101 30 μg capsule daily at bedtime for 12 weeks, with an increase to 2 sugar pills to CTAP101 30 μg capsules daily at bedtime for weeks 13-26.
intervention type: Drug
intervention name: CTAP101 30 μg capsules
description: CTAP101 30 μg capsule taken daily at bedtime
arm group label: CTAP101 30 μg capsules
other name: Calcifediol
intervention type: Other
intervention name: Sugar pill to CTAP101 30 μg capsules
description: Sugar pill capsule (placebo to CTAP101 30 μg capsule) taken daily at bedtime.
arm group label: CTAP101 30 μg capsules
arm group label: Sugar pill to CTAP101 30 μg
other name: Placebo
criteria: 
gender: All
minimum age: 18 Years
maximum age: N/A
healthy volunteers: No
last name: Joel Melnick, MD
role: Study Director
affiliation: OPKO Renal
facility: 
country: United States
responsible party type: Sponsor
mesh term: Neoplasm Metastasis
mesh term: Kidney Diseases
mesh term: Renal Insufficiency, Chronic
mesh term: Vitamin D Deficiency
mesh term: Hyperparathyroidism
mesh term: Hyperparathyroidism, Secondary
mesh term: Calcifediol
participant flow: 
baseline: 
outcome list: 
reported events: 
certain agreements: 
point of contact: 