xml: <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/NCT06016634</url>
    </required_header>
    <id_info>
      <org_study_id>1693339</org_study_id>
      <secondary_id>5K23HL148310</secondary_id>
      <secondary_id>2020095</secondary_id>
      <nct_id>NCT06016634</nct_id>
    </id_info>
    <brief_title>Alendronate for Hip Osteonecrosis in Adults With Sickle Cell Disease</brief_title>
    <official_title>A Feasibility Study of Alendronate as Treatment for Femoral Head Osteonecrosis in Adults With Sickle Cell Disease</official_title>
    <sponsors>
      <lead_sponsor>
        <agency>University of California, Davis</agency>
        <agency_class>Other</agency_class>
      </lead_sponsor>
      <collaborator>
        <agency>National Heart, Lung, and Blood Institute (NHLBI)</agency>
        <agency_class>NIH</agency_class>
      </collaborator>
      <collaborator>
        <agency>Doris Duke Charitable Foundation</agency>
        <agency_class>Other</agency_class>
      </collaborator>
    </sponsors>
    <source>University of California, Davis</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>
        A prospective, single-arm, intervention study of oral alendronate in adults with sickle cell&#xD;
        disease and hip osteonecrosis&#xD;
      </textblock>
    </brief_summary>
    <detailed_description>
      <textblock>
        The investigators hypothesize that adults with sickle cell disease (SCD) and osteonecrosis of&#xD;
        the femoral head (ONFH) will tolerate oral alendronate 70 mg administered once a week for 24&#xD;
        weeks (6 months). In addition to collecting safety and tolerability data on alendronate in&#xD;
        study participants, the investigators will also measure the preliminary efficacy of&#xD;
        alendronate using changes in the Adult Sickle Cell Quality of Life Measurement System&#xD;
        (ASCQ-Me) Pain Impact scores from baseline to 3-months and 6-months after alendronate&#xD;
        initiation. Serum and urine specimen will also be collected to assess changes in bone&#xD;
        biomarkers at baseline, 3-months, and 6-months.&#xD;
  &#xD;
        The investigators plan to recruit 24 adults with SCD from the University of California Davis&#xD;
        Medical Center to this single-arm, open label, interventional study. The investigators&#xD;
        anticipate enrolling 1-2 participants per month between Sep 2023- Dec 2024.&#xD;
  &#xD;
        The study endpoints are summarized below:&#xD;
  &#xD;
          -  To determine the recruitment and retention rates of adults with SCD-related ONFH&#xD;
             enrolled in this prospective, single-arm, open label alendronate interventional study&#xD;
  &#xD;
          -  To measure the safety, tolerability, and preliminary efficacy of oral alendronate in&#xD;
             adults with SCD-related ONFH over a 6-month treatment duration&#xD;
  &#xD;
          -  To measure changes in bone biomarkers in the serum and urine of study participants not&#xD;
             receiving chronic red blood cell transfusions at 3 time points: baseline, 3-months, and&#xD;
             6-months after initiation of alendronate&#xD;
  &#xD;
        The investigators' goal is to complete primary data analysis by May 2025.&#xD;
      </textblock>
    </detailed_description>
    <overall_status>Not yet recruiting</overall_status>
    <start_date type="Anticipated">October 2023</start_date>
    <completion_date type="Anticipated">June 2025</completion_date>
    <primary_completion_date type="Anticipated">December 2024</primary_completion_date>
    <phase>Phase 2</phase>
    <study_type>Interventional</study_type>
    <has_expanded_access>No</has_expanded_access>
    <study_design_info>
      <allocation>N/A</allocation>
      <intervention_model>Single Group Assignment</intervention_model>
      <intervention_model_description>Prospective cohort of 24 adults with sickle cell disease and hip osteonecrosis</intervention_model_description>
      <primary_purpose>Treatment</primary_purpose>
      <masking>None (Open Label)</masking>
    </study_design_info>
    <primary_outcome>
      <measure>Recruitment and retention rates</measure>
      <time_frame>up to 28 weeks</time_frame>
      <description>Proportion of eligible patients who get recruited to the study; number of enrolled patients who complete 6-month study</description>
    </primary_outcome>
    <primary_outcome>
      <measure>Incidence of Treatment-Emergent Adverse Events as assessed by participant report</measure>
      <time_frame>up to 28 weeks</time_frame>
      <description>Collect all adverse events reported by patients and determine if related to study drug</description>
    </primary_outcome>
    <primary_outcome>
      <measure>Pain assessed by the Adult Sickle Cell Quality of Life Measurement System (ASCQ-Me) Pain Impact questionnaire</measure>
      <time_frame>up to 28 weeks</time_frame>
      <description>The Adult Sickle Cell Quality of Life Measurement System (ASCQ-Me) Pain Impact questionnaire is a patient-reported outcome measure of pain level in the past 7 days. The ASCQ-Me pain scale ranges from 0-100, with a standardized mean of 50 and a standard deviation of 10, where lower scores signify worse disease impact.</description>
    </primary_outcome>
    <secondary_outcome>
      <measure>Serum CTX-1</measure>
      <time_frame>baseline, 3 months, 6 months</time_frame>
      <description>Measurement of C-terminal telopeptide of type I collagen (CTX) in serum as a biomarker for osteoclast activity (bone resorption)</description>
    </secondary_outcome>
    <secondary_outcome>
      <measure>Serum P1NP</measure>
      <time_frame>baseline, 3 months, 6 months</time_frame>
      <description>Measurement of Procollagen type I N-terminal propeptide (P1NP) in serum as a biomarker for osteoblast activity (bone formation)</description>
    </secondary_outcome>
    <number_of_arms>1</number_of_arms>
    <enrollment type="Anticipated">24</enrollment>
    <condition>Sickle Cell Disease</condition>
    <condition>Sickle Cell Anemia</condition>
    <condition>Osteonecrosis</condition>
    <condition>Ischemic Necrosis</condition>
    <condition>Avascular Necrosis</condition>
    <arm_group>
      <arm_group_label>Alendronate group</arm_group_label>
      <arm_group_type>Experimental</arm_group_type>
      <description>Single-arm prospective cohort of 24 adult with SCD</description>
    </arm_group>
    <intervention>
      <intervention_type>Drug</intervention_type>
      <intervention_name>Alendronate Sodium</intervention_name>
      <description>Administer oral alendronate 70 mg once a week x 24 weeks to all study participants</description>
      <arm_group_label>Alendronate group</arm_group_label>
      <other_name>alendronate tablet</other_name>
      <other_name>oral alendronate</other_name>
      <other_name>Fosamax</other_name>
      <other_name>Binosto</other_name>
    </intervention>
    <eligibility>
      <criteria>
        <textblock>
          Inclusion Criteria:&#xD;
  &#xD;
            -  Age 18-80 years with SCD (any genotype, confirmed by hemoglobin electrophoresis or&#xD;
               high performance liquid chromatography)&#xD;
  &#xD;
            -  Ability to provide written informed consent&#xD;
  &#xD;
            -  Ability to lay on a dual-energy X-ray absorptiometry (DXA) scanner&#xD;
  &#xD;
            -  Negative urine pregnancy test for anyone of childbearing potential at study entry&#xD;
  &#xD;
          Exclusion Criteria:&#xD;
  &#xD;
            -  Pregnant women&#xD;
  &#xD;
            -  Adults unable to consent&#xD;
  &#xD;
            -  Individuals who are not yet adults (infants, children, teenagers)&#xD;
  &#xD;
            -  Prisoners&#xD;
  &#xD;
            -  Hospitalizations (for any cause) within 2 weeks of study entry&#xD;
        </textblock>
      </criteria>
      <gender>All</gender>
      <minimum_age>18 Years</minimum_age>
      <maximum_age>80 Years</maximum_age>
      <healthy_volunteers>No</healthy_volunteers>
    </eligibility>
    <overall_official>
      <last_name>Oyebimpe O Adesina, MD, MS</last_name>
      <role>Principal Investigator</role>
      <affiliation>UC Davis School of Medicine</affiliation>
    </overall_official>
    <overall_contact>
      <last_name>Leyla Y Teos, PhD</last_name>
      <phone>(916) 460-2749</phone>
      <email>lyteos@ucdavis.edu</email>
    </overall_contact>
    <verification_date>September 2023</verification_date>
    <study_first_submitted>August 7, 2023</study_first_submitted>
    <study_first_submitted_qc>August 22, 2023</study_first_submitted_qc>
    <study_first_posted type="Actual">August 30, 2023</study_first_posted>
    <last_update_submitted>September 6, 2023</last_update_submitted>
    <last_update_submitted_qc>September 6, 2023</last_update_submitted_qc>
    <last_update_posted type="Actual">September 7, 2023</last_update_posted>
    <responsible_party>
      <responsible_party_type>Sponsor</responsible_party_type>
    </responsible_party>
    <keyword>sickle cell disease</keyword>
    <keyword>sickle cell anemia</keyword>
    <keyword>osteonecrosis of the femoral head</keyword>
    <keyword>hip osteonecrosis</keyword>
    <keyword>ischemic necrosis of the femur</keyword>
    <keyword>avascular necrosis of the femur</keyword>
    <condition_browse>
      <!-- CAUTION:  The following MeSH terms are assigned with an imperfect algorithm            -->
      <mesh_term>Osteonecrosis</mesh_term>
      <mesh_term>Anemia, Sickle Cell</mesh_term>
      <mesh_term>Necrosis</mesh_term>
    </condition_browse>
    <intervention_browse>
      <!-- CAUTION:  The following MeSH terms are assigned with an imperfect algorithm            -->
      <mesh_term>Alendronate</mesh_term>
    </intervention_browse>
    <patient_data>
      <sharing_ipd>No</sharing_ipd>
    </patient_data>
    <!-- Results have not yet been posted for this study                                          -->
  </clinical_study>
  
  
text: 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/NCT06016634
  org study id: 1693339
  secondary id: 5K23HL148310
  secondary id: 2020095
  nct id: NCT06016634
  lead sponsor: 
  collaborator: 
  collaborator: 
  has dmc: Yes
  is fda regulated drug: Yes
  is fda regulated device: No
  A prospective, single-arm, intervention study of oral alendronate in adults with sickle cell
        disease and hip osteonecrosis
  The investigators hypothesize that adults with sickle cell disease (SCD) and osteonecrosis of
        the femoral head (ONFH) will tolerate oral alendronate 70 mg administered once a week for 24
        weeks (6 months). In addition to collecting safety and tolerability data on alendronate in
        study participants, the investigators will also measure the preliminary efficacy of
        alendronate using changes in the Adult Sickle Cell Quality of Life Measurement System
        (ASCQ-Me) Pain Impact scores from baseline to 3-months and 6-months after alendronate
        initiation. Serum and urine specimen will also be collected to assess changes in bone
        biomarkers at baseline, 3-months, and 6-months.
  
        The investigators plan to recruit 24 adults with SCD from the University of California Davis
        Medical Center to this single-arm, open label, interventional study. The investigators
        anticipate enrolling 1-2 participants per month between Sep 2023- Dec 2024.
  
        The study endpoints are summarized below:
  
          -  To determine the recruitment and retention rates of adults with SCD-related ONFH
             enrolled in this prospective, single-arm, open label alendronate interventional study
  
          -  To measure the safety, tolerability, and preliminary efficacy of oral alendronate in
             adults with SCD-related ONFH over a 6-month treatment duration
  
          -  To measure changes in bone biomarkers in the serum and urine of study participants not
             receiving chronic red blood cell transfusions at 3 time points: baseline, 3-months, and
             6-months after initiation of alendronate
  
        The investigators' goal is to complete primary data analysis by May 2025.
  allocation: N/A
  intervention model: Single Group Assignment
  intervention model description: Prospective cohort of 24 adults with sickle cell disease and hip osteonecrosis
  primary purpose: Treatment
  masking: None (Open Label)
  measure: Recruitment and retention rates
  time frame: up to 28 weeks
  description: Proportion of eligible patients who get recruited to the study; number of enrolled patients who complete 6-month study
  measure: Incidence of Treatment-Emergent Adverse Events as assessed by participant report
  time frame: up to 28 weeks
  description: Collect all adverse events reported by patients and determine if related to study drug
  measure: Pain assessed by the Adult Sickle Cell Quality of Life Measurement System (ASCQ-Me) Pain Impact questionnaire
  time frame: up to 28 weeks
  description: The Adult Sickle Cell Quality of Life Measurement System (ASCQ-Me) Pain Impact questionnaire is a patient-reported outcome measure of pain level in the past 7 days. The ASCQ-Me pain scale ranges from 0-100, with a standardized mean of 50 and a standard deviation of 10, where lower scores signify worse disease impact.
  measure: Serum CTX-1
  time frame: baseline, 3 months, 6 months
  description: Measurement of C-terminal telopeptide of type I collagen (CTX) in serum as a biomarker for osteoclast activity (bone resorption)
  measure: Serum P1NP
  time frame: baseline, 3 months, 6 months
  description: Measurement of Procollagen type I N-terminal propeptide (P1NP) in serum as a biomarker for osteoblast activity (bone formation)
  arm group label: Alendronate group
  arm group type: Experimental
  description: Single-arm prospective cohort of 24 adult with SCD
  intervention type: Drug
  intervention name: Alendronate Sodium
  description: Administer oral alendronate 70 mg once a week x 24 weeks to all study participants
  arm group label: Alendronate group
  other name: alendronate tablet
  other name: oral alendronate
  other name: Fosamax
  other name: Binosto
  criteria: 
  gender: All
  minimum age: 18 Years
  maximum age: 80 Years
  healthy volunteers: No
  last name: Oyebimpe O Adesina, MD, MS
  role: Principal Investigator
  affiliation: UC Davis School of Medicine
  last name: Leyla Y Teos, PhD
  phone: (916) 460-2749
  email: lyteos@ucdavis.edu
  responsible party type: Sponsor
  mesh term: Osteonecrosis
  mesh term: Anemia, Sickle Cell
  mesh term: Necrosis
  mesh term: Alendronate
  sharing ipd: No