<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/NCT04490915</url>
  </required_header>
  <id_info>
    <org_study_id>NBI-74788-CAH3003</org_study_id>
    <secondary_id>2019-004873-17</secondary_id>
    <nct_id>NCT04490915</nct_id>
  </id_info>
  <brief_title>Global Safety and Efficacy Registration Study of Crinecerfont for Congenital Adrenal Hyperplasia</brief_title>
  <acronym>CAHtalyst</acronym>
  <official_title>A Randomized, Double-Blind, Placebo-Controlled Study to Evaluate the Safety and Efficacy of Crinecerfont (NBI-74788) in Adult Subjects With Classic Congenital Adrenal Hyperplasia, Followed by Open-Label Treatment</official_title>
  <sponsors>
    <lead_sponsor>
      <agency>Neurocrine Biosciences</agency>
      <agency_class>Industry</agency_class>
    </lead_sponsor>
  </sponsors>
  <source>Neurocrine Biosciences</source>
  <oversight_info>
    <has_dmc>Yes</has_dmc>
    <is_fda_regulated_drug>Yes</is_fda_regulated_drug>
    <is_fda_regulated_device>No</is_fda_regulated_device>
  </oversight_info>
  <brief_summary>
    <textblock>
      This is a Phase 3 study to evaluate the efficacy, safety, and tolerability of crinecerfont&#xD;
      versus placebo administered for 24 weeks in approximately 165 adult participants with classic&#xD;
      CAH due to 21-hydroxylase deficiency. The study consists of a 6-month randomized,&#xD;
      double-blind, placebo-controlled period, followed by 1 year of open-label treatment with&#xD;
      crinecerfont. Subsequently, participants may elect to participate in the open-label extension&#xD;
      (OLE) period. The duration of participation in the study is approximately 20 months for the&#xD;
      core study and will be a variable amount of time per subject for the OLE (estimated to be&#xD;
      approximately 3 years).&#xD;
    </textblock>
  </brief_summary>
  <overall_status>Active, not recruiting</overall_status>
  <start_date type="Actual">July 23, 2020</start_date>
  <completion_date type="Anticipated">August 2027</completion_date>
  <primary_completion_date type="Actual">July 19, 2023</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>Triple (Participant, Care Provider, Investigator)</masking>
  </study_design_info>
  <primary_outcome>
    <measure>Percent change from baseline in glucocorticoid daily dose at Week 24</measure>
    <time_frame>Baseline and Week 24</time_frame>
  </primary_outcome>
  <secondary_outcome>
    <measure>Change from baseline in serum androstenedione at Week 4</measure>
    <time_frame>Baseline and Week 4</time_frame>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Achievement of a reduction in glucocorticoid daily dose to physiologic levels at Week 24</measure>
    <time_frame>Baseline and Week 24</time_frame>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Change from baseline in homeostatic model assessment of insulin resistance (HOMA-IR) index at Week 24</measure>
    <time_frame>Baseline and Week 24</time_frame>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Change from baseline in body weight at Week 24</measure>
    <time_frame>Baseline and Week 24</time_frame>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Change from baseline in fat mass at Week 24</measure>
    <time_frame>Baseline and Week 24</time_frame>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Change from baseline in blood pressure at Week 24</measure>
    <time_frame>Baseline and Week 24</time_frame>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Change from baseline in glucose tolerance at Week 24</measure>
    <time_frame>Baseline and Week 24</time_frame>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Change from baseline in waist circumference at Week 24</measure>
    <time_frame>Baseline and Week 24</time_frame>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Change from baseline in menstrual regularity at Week 24</measure>
    <time_frame>Baseline and Week 24</time_frame>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Change from baseline in testicular adrenal rest tumor size at Week 24</measure>
    <time_frame>Baseline and Week 24</time_frame>
  </secondary_outcome>
  <number_of_arms>2</number_of_arms>
  <enrollment type="Actual">182</enrollment>
  <condition>Congenital Adrenal Hyperplasia</condition>
  <arm_group>
    <arm_group_label>Crinecerfont</arm_group_label>
    <arm_group_type>Experimental</arm_group_type>
    <description>Crinecerfont capsule, administered orally, twice daily for 24 weeks during the placebo-controlled treatment period, followed by active treatment with crinecerfont for at least 1 year.</description>
  </arm_group>
  <arm_group>
    <arm_group_label>Placebo</arm_group_label>
    <arm_group_type>Placebo Comparator</arm_group_type>
    <description>Placebo capsule, administered orally, twice daily for 24 weeks, followed by active treatment with crinecerfont for at least 1 year.</description>
  </arm_group>
  <intervention>
    <intervention_type>Drug</intervention_type>
    <intervention_name>Crinecerfont</intervention_name>
    <description>CRF1-receptor antagonist</description>
    <arm_group_label>Crinecerfont</arm_group_label>
    <arm_group_label>Placebo</arm_group_label>
    <other_name>NBI-74788</other_name>
  </intervention>
  <intervention>
    <intervention_type>Drug</intervention_type>
    <intervention_name>Placebo</intervention_name>
    <description>Non-active dosage form</description>
    <arm_group_label>Placebo</arm_group_label>
  </intervention>
  <eligibility>
    <criteria>
      <textblock>
        Inclusion Criteria:&#xD;
&#xD;
          1. Be willing and able to adhere to the study procedures, including all requirements at&#xD;
             the study center and return for the follow-up visit.&#xD;
&#xD;
          2. Have a medically confirmed diagnosis of classic 21-hydroxylase deficiency CAH.&#xD;
&#xD;
          3. Be on a stable regimen of steroidal treatment for CAH.&#xD;
&#xD;
          4. Participants of childbearing potential must agree to use an acceptable method of&#xD;
             contraception during the study.&#xD;
&#xD;
        Exclusion Criteria:&#xD;
&#xD;
          1. Have a diagnosis of any of the other known forms of classic CAH.&#xD;
&#xD;
          2. Have a history of bilateral adrenalectomy, hypopituitarism, or other condition&#xD;
             requiring chronic glucocorticoid therapy.&#xD;
&#xD;
          3. Have a clinically significant unstable medical condition or chronic disease other than&#xD;
             CAH.&#xD;
&#xD;
          4. Have a history of cancer unless considered cured.&#xD;
&#xD;
          5. Are pregnant.&#xD;
&#xD;
          6. Have a known history of clinically significant arrhythmia or abnormalities on ECG.&#xD;
&#xD;
          7. Have a known hypersensitivity to any corticotropin releasing hormone antagonists.&#xD;
&#xD;
          8. Have received any other investigational drug within 30 days before initial screening&#xD;
             or plan to use an investigational drug (other than the study drug) during the study.&#xD;
&#xD;
          9. Have current substance dependence, or current substance (drug) or alcohol abuse.&#xD;
&#xD;
         10. Have had a blood loss ≥550 mL or donated blood or blood products within 8 weeks prior&#xD;
             to 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>Clinical Development Lead</last_name>
    <role>Study Director</role>
    <affiliation>Neurocrine Biosciences</affiliation>
  </overall_official>
  <location>
    <facility>
      <name>Neurocrine Clinical Site</name>
      <address>
        <city>Los Angeles</city>
        <state>California</state>
        <zip>90027</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Neurocrine Clinical Site</name>
      <address>
        <city>San Diego</city>
        <state>California</state>
        <zip>92123</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Neurocrine Clinical Site</name>
      <address>
        <city>San Francisco</city>
        <state>California</state>
        <zip>94143</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Neurocrine Clinical Site</name>
      <address>
        <city>Aurora</city>
        <state>Colorado</state>
        <zip>80045</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Neurocrine Clinical Site</name>
      <address>
        <city>Atlanta</city>
        <state>Georgia</state>
        <zip>30329</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Neurocrine Clinical Site</name>
      <address>
        <city>Indianapolis</city>
        <state>Indiana</state>
        <zip>46202</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Neurocrine Clinical Site</name>
      <address>
        <city>Bethesda</city>
        <state>Maryland</state>
        <zip>20982</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Neurocrine Clinical Site</name>
      <address>
        <city>Boston</city>
        <state>Massachusetts</state>
        <zip>02115</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Neurocrine Clinical Site</name>
      <address>
        <city>Ann Arbor</city>
        <state>Michigan</state>
        <zip>48109</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Neurocrine Clinical Site</name>
      <address>
        <city>Minneapolis</city>
        <state>Minnesota</state>
        <zip>55454</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Neurocrine Clinical Site</name>
      <address>
        <city>Rochester</city>
        <state>Minnesota</state>
        <zip>55905</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Neurocrine Clinical Site</name>
      <address>
        <city>Saint Louis</city>
        <state>Missouri</state>
        <zip>63110</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Neurocrine Clinical Site</name>
      <address>
        <city>Great Neck</city>
        <state>New York</state>
        <zip>11021</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Neurocrine Clinical Site</name>
      <address>
        <city>New York</city>
        <state>New York</state>
        <zip>10029</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Neurocrine Clinical Site</name>
      <address>
        <city>Winston-Salem</city>
        <state>North Carolina</state>
        <zip>27157</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Neurocrine Clinical Site</name>
      <address>
        <city>Tulsa</city>
        <state>Oklahoma</state>
        <zip>74135</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Neurocrine Clinical Site</name>
      <address>
        <city>Philadelphia</city>
        <state>Pennsylvania</state>
        <zip>19104</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Neurocrine Clinical Site</name>
      <address>
        <city>Pittsburgh</city>
        <state>Pennsylvania</state>
        <zip>15224</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Neurocrine Clinical Site</name>
      <address>
        <city>Dallas</city>
        <state>Texas</state>
        <zip>75390</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Neurocrine Clinical Site</name>
      <address>
        <city>Seattle</city>
        <state>Washington</state>
        <zip>98105</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Neurocrine Clinical Site</name>
      <address>
        <city>Graz</city>
        <zip>8036</zip>
        <country>Austria</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Neurocrine Clinical Site</name>
      <address>
        <city>Vienna</city>
        <zip>1090</zip>
        <country>Austria</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Neurocrine Clinical Site</name>
      <address>
        <city>Brussels</city>
        <zip>1070</zip>
        <country>Belgium</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Neurocrine Clinical Site</name>
      <address>
        <city>Leuven</city>
        <zip>3000</zip>
        <country>Belgium</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Neurocrine Clinical Site</name>
      <address>
        <city>Sofia</city>
        <zip>1431</zip>
        <country>Bulgaria</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Neurocrine Clinical Site</name>
      <address>
        <city>Sofia</city>
        <zip>1606</zip>
        <country>Bulgaria</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Neurocrine Clinical Site</name>
      <address>
        <city>Halifax</city>
        <state>Nova Scotia</state>
        <zip>B3H 2Y9</zip>
        <country>Canada</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Neurocrine Clinical Site</name>
      <address>
        <city>Hradec Králové</city>
        <zip>50005</zip>
        <country>Czechia</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Neurocrine Clinical Site</name>
      <address>
        <city>Angers</city>
        <zip>49933</zip>
        <country>France</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Neurocrine Clinical Site</name>
      <address>
        <city>Grenoble</city>
        <zip>38700</zip>
        <country>France</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Neurocrine Clinical Site</name>
      <address>
        <city>Le Kremlin-Bicêtre</city>
        <zip>94270</zip>
        <country>France</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Neurocrine Clinical Site</name>
      <address>
        <city>Nantes</city>
        <zip>44093</zip>
        <country>France</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Neurocrine Clinical Site</name>
      <address>
        <city>Paris</city>
        <zip>75651</zip>
        <country>France</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Neurocrine Clinical Site</name>
      <address>
        <city>Paris</city>
        <zip>75679</zip>
        <country>France</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Neurocrine Clinical Site</name>
      <address>
        <city>Dresden</city>
        <zip>01307</zip>
        <country>Germany</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Neurocrine Clinical Site</name>
      <address>
        <city>Essen</city>
        <zip>45147</zip>
        <country>Germany</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Neurocrine Clinical Site</name>
      <address>
        <city>Frankfurt</city>
        <zip>60590</zip>
        <country>Germany</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Neurocrine Clinical Site</name>
      <address>
        <city>Leipzig</city>
        <zip>04103</zip>
        <country>Germany</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Neurocrine Clinical Site</name>
      <address>
        <city>Munich</city>
        <zip>80336</zip>
        <country>Germany</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Neurocrine Clinical Site</name>
      <address>
        <city>Athens</city>
        <zip>106 76</zip>
        <country>Greece</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Neurocrine Clinical Site</name>
      <address>
        <city>Athens</city>
        <zip>115 27</zip>
        <country>Greece</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Neurocrine Clinical Site</name>
      <address>
        <city>Athens</city>
        <zip>11527</zip>
        <country>Greece</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Neurocrine Clinical Site</name>
      <address>
        <city>Thessaloníki</city>
        <zip>54642</zip>
        <country>Greece</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Neurocrine Clinical Site</name>
      <address>
        <city>Afula</city>
        <zip>1834111</zip>
        <country>Israel</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Neurocrine Clinical Site</name>
      <address>
        <city>Beer Sheva</city>
        <zip>8410101</zip>
        <country>Israel</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Neurocrine Clinical Site</name>
      <address>
        <city>Petah Tikva</city>
        <zip>4941480</zip>
        <country>Israel</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Neurocrine Clinical Site</name>
      <address>
        <city>Tel Aviv</city>
        <zip>64239</zip>
        <country>Israel</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Neurocrine Clinical Site</name>
      <address>
        <city>Ancona</city>
        <zip>60126</zip>
        <country>Italy</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Neurocrine Clinical Site</name>
      <address>
        <city>Bologna</city>
        <zip>40138</zip>
        <country>Italy</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Neurocrine Clinical Site</name>
      <address>
        <city>Florence</city>
        <zip>50139</zip>
        <country>Italy</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Neurocrine Clinical Site</name>
      <address>
        <city>Messina</city>
        <zip>98125</zip>
        <country>Italy</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Neurocrine Clinical Site</name>
      <address>
        <city>Milan</city>
        <zip>20132</zip>
        <country>Italy</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Neurocrine Clinical Site</name>
      <address>
        <city>Milan</city>
        <zip>20149</zip>
        <country>Italy</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Neurocrine Clinical Site</name>
      <address>
        <city>Naples</city>
        <zip>80131</zip>
        <country>Italy</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Neurocrine Clinical Site</name>
      <address>
        <city>Padova</city>
        <zip>35128</zip>
        <country>Italy</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Neurocrine Clinical Site</name>
      <address>
        <city>Roma</city>
        <zip>00161</zip>
        <country>Italy</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Neurocrine Clinical Site</name>
      <address>
        <city>Leiden</city>
        <zip>2333</zip>
        <country>Netherlands</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Neurocrine Clinical Site</name>
      <address>
        <city>Kraków</city>
        <zip>31-011</zip>
        <country>Poland</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Neurocrine Clinical Site</name>
      <address>
        <city>Poznań</city>
        <zip>60-355</zip>
        <country>Poland</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Neurocrine Clinical Site</name>
      <address>
        <city>Warszawa</city>
        <zip>01-809</zip>
        <country>Poland</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Neurocrine Clinical Site</name>
      <address>
        <city>Porto</city>
        <zip>4200-319</zip>
        <country>Portugal</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Neurocrine Clinical Site</name>
      <address>
        <city>Belgrade</city>
        <zip>11000</zip>
        <country>Serbia</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Neurocrine Clinical Site</name>
      <address>
        <city>Madrid</city>
        <zip>28034</zip>
        <country>Spain</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Neurocrine Clinical Site</name>
      <address>
        <city>Sevilla</city>
        <zip>41013</zip>
        <country>Spain</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Neurocrine Clinical Site</name>
      <address>
        <city>Gothenburg</city>
        <zip>41345</zip>
        <country>Sweden</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Neurocrine Clinical Site</name>
      <address>
        <city>Stockholm</city>
        <zip>17176</zip>
        <country>Sweden</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Neurocrine Clinical Site</name>
      <address>
        <city>Cardiff</city>
        <zip>CF14 4HH</zip>
        <country>United Kingdom</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Neurocrine Clinical Site</name>
      <address>
        <city>London</city>
        <zip>WC1B 5EH</zip>
        <country>United Kingdom</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Neurocrine Clinical Site</name>
      <address>
        <city>Manchester</city>
        <zip>M20 4BX</zip>
        <country>United Kingdom</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Neurocrine Clinical Site</name>
      <address>
        <city>Salford</city>
        <zip>M6 8HD</zip>
        <country>United Kingdom</country>
      </address>
    </facility>
  </location>
  <location_countries>
    <country>Austria</country>
    <country>Belgium</country>
    <country>Bulgaria</country>
    <country>Canada</country>
    <country>Czechia</country>
    <country>France</country>
    <country>Germany</country>
    <country>Greece</country>
    <country>Israel</country>
    <country>Italy</country>
    <country>Netherlands</country>
    <country>Poland</country>
    <country>Portugal</country>
    <country>Serbia</country>
    <country>Spain</country>
    <country>Sweden</country>
    <country>United Kingdom</country>
    <country>United States</country>
  </location_countries>
  <link>
    <url>https://www.cahtalyststudy.com/</url>
    <description>Study website - Cahtalyst Study</description>
  </link>
  <verification_date>August 2023</verification_date>
  <study_first_submitted>July 24, 2020</study_first_submitted>
  <study_first_submitted_qc>July 24, 2020</study_first_submitted_qc>
  <study_first_posted type="Actual">July 29, 2020</study_first_posted>
  <last_update_submitted>August 17, 2023</last_update_submitted>
  <last_update_submitted_qc>August 17, 2023</last_update_submitted_qc>
  <last_update_posted type="Actual">August 21, 2023</last_update_posted>
  <responsible_party>
    <responsible_party_type>Sponsor</responsible_party_type>
  </responsible_party>
  <condition_browse>
    <!-- CAUTION:  The following MeSH terms are assigned with an imperfect algorithm            -->
    <mesh_term>Adrenal Hyperplasia, Congenital</mesh_term>
    <mesh_term>Adrenogenital Syndrome</mesh_term>
    <mesh_term>Adrenocortical Hyperfunction</mesh_term>
    <mesh_term>Hyperplasia</mesh_term>
  </condition_browse>
  <patient_data>
    <sharing_ipd>No</sharing_ipd>
  </patient_data>
  <!-- Results have not yet been posted for this study                                          -->
</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/NCT04490915
org study id: NBI-74788-CAH3003
secondary id: 2019-004873-17
nct id: NCT04490915
lead sponsor: 
has dmc: Yes
is fda regulated drug: Yes
is fda regulated device: No
This is a Phase 3 study to evaluate the efficacy, safety, and tolerability of crinecerfont
      versus placebo administered for 24 weeks in approximately 165 adult participants with classic
      CAH due to 21-hydroxylase deficiency. The study consists of a 6-month randomized,
      double-blind, placebo-controlled period, followed by 1 year of open-label treatment with
      crinecerfont. Subsequently, participants may elect to participate in the open-label extension
      (OLE) period. The duration of participation in the study is approximately 20 months for the
      core study and will be a variable amount of time per subject for the OLE (estimated to be
      approximately 3 years).
allocation: Randomized
intervention model: Parallel Assignment
primary purpose: Treatment
masking: Triple (Participant, Care Provider, Investigator)
measure: Percent change from baseline in glucocorticoid daily dose at Week 24
time frame: Baseline and Week 24
measure: Change from baseline in serum androstenedione at Week 4
time frame: Baseline and Week 4
measure: Achievement of a reduction in glucocorticoid daily dose to physiologic levels at Week 24
time frame: Baseline and Week 24
measure: Change from baseline in homeostatic model assessment of insulin resistance (HOMA-IR) index at Week 24
time frame: Baseline and Week 24
measure: Change from baseline in body weight at Week 24
time frame: Baseline and Week 24
measure: Change from baseline in fat mass at Week 24
time frame: Baseline and Week 24
measure: Change from baseline in blood pressure at Week 24
time frame: Baseline and Week 24
measure: Change from baseline in glucose tolerance at Week 24
time frame: Baseline and Week 24
measure: Change from baseline in waist circumference at Week 24
time frame: Baseline and Week 24
measure: Change from baseline in menstrual regularity at Week 24
time frame: Baseline and Week 24
measure: Change from baseline in testicular adrenal rest tumor size at Week 24
time frame: Baseline and Week 24
arm group label: Crinecerfont
arm group type: Experimental
description: Crinecerfont capsule, administered orally, twice daily for 24 weeks during the placebo-controlled treatment period, followed by active treatment with crinecerfont for at least 1 year.
arm group label: Placebo
arm group type: Placebo Comparator
description: Placebo capsule, administered orally, twice daily for 24 weeks, followed by active treatment with crinecerfont for at least 1 year.
intervention type: Drug
intervention name: Crinecerfont
description: CRF1-receptor antagonist
arm group label: Crinecerfont
arm group label: Placebo
other name: NBI-74788
intervention type: Drug
intervention name: Placebo
description: Non-active dosage form
arm group label: Placebo
criteria: 
gender: All
minimum age: 18 Years
maximum age: N/A
healthy volunteers: No
last name: Clinical Development Lead
role: Study Director
affiliation: Neurocrine Biosciences
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country: Austria
country: Belgium
country: Bulgaria
country: Canada
country: Czechia
country: France
country: Germany
country: Greece
country: Israel
country: Italy
country: Netherlands
country: Poland
country: Portugal
country: Serbia
country: Spain
country: Sweden
country: United Kingdom
country: United States
url: https://www.cahtalyststudy.com/
description: Study website - Cahtalyst Study
responsible party type: Sponsor
mesh term: Adrenal Hyperplasia, Congenital
mesh term: Adrenogenital Syndrome
mesh term: Adrenocortical Hyperfunction
mesh term: Hyperplasia
sharing ipd: No