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/NCT06023199</url>
    </required_header>
    <id_info>
      <org_study_id>U23-02-4988</org_study_id>
      <nct_id>NCT06023199</nct_id>
    </id_info>
    <brief_title>Assessing Physical Function in Sickle Cell Patients Taking Voxelotor</brief_title>
    <official_title>Assessing Functional Capacity in Sickle Cell Patients Receiving Voxelotor</official_title>
    <sponsors>
      <lead_sponsor>
        <agency>Inova Health Care Services</agency>
        <agency_class>Other</agency_class>
      </lead_sponsor>
      <collaborator>
        <agency>Pfizer</agency>
        <agency_class>Industry</agency_class>
      </collaborator>
    </sponsors>
    <source>Inova Health Care Services</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>
      <is_us_export>No</is_us_export>
    </oversight_info>
    <brief_summary>
      <textblock>
        Voxelotor is a novel hemoglobin polymerization inhibitor which has been demonstrated to&#xD;
        reduce hemolysis and improve hemoglobin levels. There have been numerous studies examining&#xD;
        the clinical impact of voxelotor in sickle cell disease (SCD) patients, but there are few&#xD;
        published reports on the effects of treatment on physical function in patients with SCD. The&#xD;
        hypothesis to be tested is that anemic SCD patients will have improvements in performance&#xD;
        after 6 months of voxelotor treatment.&#xD;
      </textblock>
    </brief_summary>
    <overall_status>Not yet recruiting</overall_status>
    <start_date type="Anticipated">September 15, 2023</start_date>
    <completion_date type="Anticipated">June 15, 2024</completion_date>
    <primary_completion_date type="Anticipated">March 15, 2024</primary_completion_date>
    <study_type>Observational</study_type>
    <has_expanded_access>No</has_expanded_access>
    <study_design_info>
      <observational_model>Cohort</observational_model>
      <time_perspective>Prospective</time_perspective>
    </study_design_info>
    <primary_outcome>
      <measure>Participant's Physical Performance Using 6 Minutes Walk Test (6MWT) Pre and Post Treatment with Voxelotor.</measure>
      <time_frame>6 months from the treatment initiation</time_frame>
      <description>The 6MWT is a sensitive objective assessment of functional exercise capacity which assess distance walked over 6 minutes.This test detects minor and moderate functional impairment resulting from treatment with Voxelotor.</description>
    </primary_outcome>
    <other_outcome>
      <measure>The Change in the Quality of Life as assessed by Sheehan Disability Scale (SDS) During the 6 Months Trial Period.</measure>
      <time_frame>6 months from the treatment initiation</time_frame>
      <description>The SDS is a short self-reporting questionnaire that assess functional impairment in work/school, social and family life. The assessment scale has a total score of 30 (0 unimpaired and 30 extremely impaired).</description>
    </other_outcome>
    <enrollment type="Anticipated">10</enrollment>
    <condition>Sickle Cell Disease</condition>
    <intervention>
      <intervention_type>Drug</intervention_type>
      <intervention_name>Voxelotor</intervention_name>
      <description>Voxelotor is administered orally at a dose of 1500mg daily.</description>
      <other_name>Oxbryta</other_name>
    </intervention>
    <eligibility>
      <study_pop>
        <textblock>
          This study will enroll adults diagnosed with various genotypes of sickle cell disease&#xD;
          including HbSS, HbSC or HbS beta thalassemia.&#xD;
        </textblock>
      </study_pop>
      <sampling_method>Non-Probability Sample</sampling_method>
      <criteria>
        <textblock>
          Inclusion Criteria:&#xD;
  &#xD;
            1. Provision of signed and dated informed consent form&#xD;
  &#xD;
            2. Stated willingness to comply with all study procedures and availability for the&#xD;
               duration of the study&#xD;
  &#xD;
            3. Male or female,&#xD;
  &#xD;
            4. aged 18-65 years&#xD;
  &#xD;
            5. Diagnosis of sickle cell disease with HbSS, HbSc, HbS-beta thalassemia genotypes&#xD;
  &#xD;
            6. Hemoglobin level of 6 g/dL - 10.5 g/dL&#xD;
  &#xD;
            7. Between 0 and 10 vaso-occlusive (VOC) episodes in previous 12 months&#xD;
  &#xD;
            8. Ability to take oral medication and be willing to adhere to the voxelotor regimen&#xD;
  &#xD;
            9. For females of reproductive potential, they will be educated to avoid pregnancy during&#xD;
               study period and for one month after due to limited data surrounding pregnancy and&#xD;
               voxelotor&#xD;
  &#xD;
           10. For males of reproductive potential: use of condoms or other methods to ensure&#xD;
               effective contraception with partner&#xD;
  &#xD;
           11. Agreement to adhere to Lifestyle Considerations (see section 5.3) throughout study&#xD;
               duration&#xD;
  &#xD;
          Exclusion Criteria:&#xD;
  &#xD;
            1. Hemoglobin results &lt; 6 g/dL or &gt; 10.5 g/dL&#xD;
  &#xD;
            2. Patients receiving hydroxyurea who have had a dose change within the prior 3 months&#xD;
  &#xD;
            3. Females who are breastfeeding or pregnant&#xD;
  &#xD;
            4. Patients who are receiving regularly scheduled blood (RBC) transfusion therapy (also&#xD;
               termed chronic, prophylactic, or preventive transfusion) within 30 days of 6MWT&#xD;
  &#xD;
            5. Any form of hepatic dysfunction as demonstrated by alanine aminotransferase (ALT) &gt;4 ×&#xD;
               ULN or Child-Pugh Class C&#xD;
  &#xD;
            6. Acute kidney injury or chronic renal disease with estimated glomerular filtration&#xD;
               rate&lt;30mL/min/1.73 m2&#xD;
  &#xD;
            7. Current or previous diagnosis of malignancy within the last two years excluding local&#xD;
               therapy for non-melanoma skin malignancy&#xD;
  &#xD;
            8. Unstable cardiopulmonary disease within the last 6 months prior to study enrollment&#xD;
  &#xD;
            9. Any conditions including behavioral or medical that in the opinion of the&#xD;
               investigator, may negatively impact informed consent, participation and study&#xD;
               interpretation&#xD;
  &#xD;
           10. Current use of commercially available voxelotor&#xD;
  &#xD;
           11. Prior hypersensitivity to voxelotor or its excipients&#xD;
  &#xD;
           12. Patients with baseline gait and other physical impairments that would limit 6MWT and&#xD;
               other functional assessment at discretion of the investigator&#xD;
  &#xD;
           13. Patients who on day of assessment present with vaso-occlusive crisis or rate their&#xD;
               pain greater than 6 on a scale of 1-10 using the Numeric Rating Scale will not be&#xD;
               assessed that day and re-screened at the investigator's discretion.&#xD;
        </textblock>
      </criteria>
      <gender>All</gender>
      <minimum_age>18 Years</minimum_age>
      <maximum_age>65 Years</maximum_age>
      <healthy_volunteers>No</healthy_volunteers>
    </eligibility>
    <overall_official>
      <last_name>Sheinei Alan</last_name>
      <role>Principal Investigator</role>
      <affiliation>Inova Schar Cancer Institute</affiliation>
    </overall_official>
    <overall_contact>
      <last_name>Elahe Mollapour</last_name>
      <phone>571-472-0615</phone>
      <email>elahe.mollapour@inova.org</email>
    </overall_contact>
    <overall_contact_backup>
      <last_name>Keary Jane't</last_name>
      <phone>571-472-0234</phone>
      <email>keary.janet@inova.org</email>
    </overall_contact_backup>
    <location>
      <facility>
        <name>Inova Schar Cancer Institute</name>
        <address>
          <city>Falls Church</city>
          <state>Virginia</state>
          <zip>22042</zip>
          <country>United States</country>
        </address>
      </facility>
      <contact>
        <last_name>Elahe Mollapour</last_name>
        <phone>517-472-0615</phone>
        <email>elahe.mollapour@inova.org</email>
      </contact>
      <contact_backup>
        <last_name>Keary Jane't</last_name>
        <phone>571-472-0234</phone>
        <email>keary.janet@inova.org</email>
      </contact_backup>
      <investigator>
        <last_name>Sheinei Alan, MD</last_name>
        <role>Principal Investigator</role>
      </investigator>
    </location>
    <location_countries>
      <country>United States</country>
    </location_countries>
    <reference>
      <citation>Eisenstaedt R, Penninx BW, Woodman RC. Anemia in the elderly: current understanding and emerging concepts. Blood Rev. 2006 Jul;20(4):213-26. doi: 10.1016/j.blre.2005.12.002. Epub 2006 Feb 10.</citation>
      <PMID>16472893</PMID>
    </reference>
    <reference>
      <citation>Vichinsky E, Hoppe CC, Ataga KI, Ware RE, Nduba V, El-Beshlawy A, Hassab H, Achebe MM, Alkindi S, Brown RC, Diuguid DL, Telfer P, Tsitsikas DA, Elghandour A, Gordeuk VR, Kanter J, Abboud MR, Lehrer-Graiwer J, Tonda M, Intondi A, Tong B, Howard J; HOPE Trial Investigators. A Phase 3 Randomized Trial of Voxelotor in Sickle Cell Disease. N Engl J Med. 2019 Aug 8;381(6):509-519. doi: 10.1056/NEJMoa1903212. Epub 2019 Jun 14.</citation>
      <PMID>31199090</PMID>
    </reference>
    <reference>
      <citation>Howard J, Ataga KI, Brown RC, Achebe M, Nduba V, El-Beshlawy A, Hassab H, Agodoa I, Tonda M, Gray S, Lehrer-Graiwer J, Vichinsky E. Voxelotor in adolescents and adults with sickle cell disease (HOPE): long-term follow-up results of an international, randomised, double-blind, placebo-controlled, phase 3 trial. Lancet Haematol. 2021 May;8(5):e323-e333. doi: 10.1016/S2352-3026(21)00059-4. Epub 2021 Apr 7.</citation>
      <PMID>33838113</PMID>
    </reference>
    <reference>
      <citation>Howard J, Hemmaway CJ, Telfer P, Layton DM, Porter J, Awogbade M, Mant T, Gretler DD, Dufu K, Hutchaleelaha A, Patel M, Siu V, Dixon S, Landsman N, Tonda M, Lehrer-Graiwer J. A phase 1/2 ascending dose study and open-label extension study of voxelotor in patients with sickle cell disease. Blood. 2019 Apr 25;133(17):1865-1875. doi: 10.1182/blood-2018-08-868893. Epub 2019 Jan 17.</citation>
      <PMID>30655275</PMID>
    </reference>
    <reference>
      <citation>Inusa BPD, Hsu LL, Kohli N, Patel A, Ominu-Evbota K, Anie KA, Atoyebi W. Sickle Cell Disease-Genetics, Pathophysiology, Clinical Presentation and Treatment. Int J Neonatal Screen. 2019 May 7;5(2):20. doi: 10.3390/ijns5020020. eCollection 2019 Jun.</citation>
      <PMID>33072979</PMID>
    </reference>
    <reference>
      <citation>Grosse SD, Odame I, Atrash HK, Amendah DD, Piel FB, Williams TN. Sickle cell disease in Africa: a neglected cause of early childhood mortality. Am J Prev Med. 2011 Dec;41(6 Suppl 4):S398-405. doi: 10.1016/j.amepre.2011.09.013.</citation>
      <PMID>22099364</PMID>
    </reference>
    <reference>
      <citation>Piel FB, Patil AP, Howes RE, Nyangiri OA, Gething PW, Dewi M, Temperley WH, Williams TN, Weatherall DJ, Hay SI. Global epidemiology of sickle haemoglobin in neonates: a contemporary geostatistical model-based map and population estimates. Lancet. 2013 Jan 12;381(9861):142-51. doi: 10.1016/S0140-6736(12)61229-X. Epub 2012 Oct 25.</citation>
      <PMID>23103089</PMID>
    </reference>
    <reference>
      <citation>Anie KA, Green J. Psychological therapies for sickle cell disease and pain. Cochrane Database Syst Rev. 2002;(2):CD001916. doi: 10.1002/14651858.CD001916.</citation>
      <PMID>12076428</PMID>
    </reference>
    <reference>
      <citation>Crawford J, Cella D, Cleeland CS, Cremieux PY, Demetri GD, Sarokhan BJ, Slavin MB, Glaspy JA. Relationship between changes in hemoglobin level and quality of life during chemotherapy in anemic cancer patients receiving epoetin alfa therapy. Cancer. 2002 Aug 15;95(4):888-95. doi: 10.1002/cncr.10763.</citation>
      <PMID>12209734</PMID>
    </reference>
    <reference>
      <citation>Corona LP, Andrade FCD, da Silva Alexandre T, de Brito TRP, Nunes DP, de Oliveira Duarte YA. Higher hemoglobin levels are associated with better physical performance among older adults without anemia: a longitudinal analysis. BMC Geriatr. 2022 Mar 21;22(1):233. doi: 10.1186/s12877-022-02937-4.</citation>
      <PMID>35313814</PMID>
    </reference>
    <reference>
      <citation>Yenamandra A, Marjoncu D. Voxelotor: A Hemoglobin S Polymerization Inhibitor for the Treatment of Sickle Cell Disease. J Adv Pract Oncol. 2020 Nov-Dec;11(8):873-877. doi: 10.6004/jadpro.2020.11.8.7. Epub 2020 Nov 1.</citation>
      <PMID>33489427</PMID>
    </reference>
    <reference>
      <citation>Agarwala P, Salzman SH. Six-Minute Walk Test: Clinical Role, Technique, Coding, and Reimbursement. Chest. 2020 Mar;157(3):603-611. doi: 10.1016/j.chest.2019.10.014. Epub 2019 Nov 2.</citation>
      <PMID>31689414</PMID>
    </reference>
    <verification_date>August 2023</verification_date>
    <study_first_submitted>August 7, 2023</study_first_submitted>
    <study_first_submitted_qc>September 1, 2023</study_first_submitted_qc>
    <study_first_posted type="Actual">September 5, 2023</study_first_posted>
    <last_update_submitted>September 1, 2023</last_update_submitted>
    <last_update_submitted_qc>September 1, 2023</last_update_submitted_qc>
    <last_update_posted type="Actual">September 5, 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>Anemia, Sickle Cell</mesh_term>
    </condition_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/NCT06023199
  org study id: U23-02-4988
  nct id: NCT06023199
  lead sponsor: 
  collaborator: 
  has dmc: Yes
  is fda regulated drug: Yes
  is fda regulated device: No
  is us export: No
  Voxelotor is a novel hemoglobin polymerization inhibitor which has been demonstrated to
        reduce hemolysis and improve hemoglobin levels. There have been numerous studies examining
        the clinical impact of voxelotor in sickle cell disease (SCD) patients, but there are few
        published reports on the effects of treatment on physical function in patients with SCD. The
        hypothesis to be tested is that anemic SCD patients will have improvements in performance
        after 6 months of voxelotor treatment.
  observational model: Cohort
  time perspective: Prospective
  measure: Participant's Physical Performance Using 6 Minutes Walk Test (6MWT) Pre and Post Treatment with Voxelotor.
  time frame: 6 months from the treatment initiation
  description: The 6MWT is a sensitive objective assessment of functional exercise capacity which assess distance walked over 6 minutes.This test detects minor and moderate functional impairment resulting from treatment with Voxelotor.
  measure: The Change in the Quality of Life as assessed by Sheehan Disability Scale (SDS) During the 6 Months Trial Period.
  time frame: 6 months from the treatment initiation
  description: The SDS is a short self-reporting questionnaire that assess functional impairment in work/school, social and family life. The assessment scale has a total score of 30 (0 unimpaired and 30 extremely impaired).
  intervention type: Drug
  intervention name: Voxelotor
  description: Voxelotor is administered orally at a dose of 1500mg daily.
  other name: Oxbryta
  study pop: 
  sampling method: Non-Probability Sample
  criteria: 
  gender: All
  minimum age: 18 Years
  maximum age: 65 Years
  healthy volunteers: No
  last name: Sheinei Alan
  role: Principal Investigator
  affiliation: Inova Schar Cancer Institute
  last name: Elahe Mollapour
  phone: 571-472-0615
  email: elahe.mollapour@inova.org
  last name: Keary Jane't
  phone: 571-472-0234
  email: keary.janet@inova.org
  facility: 
  contact: 
  contact backup: 
  investigator: 
  country: United States
  citation: Eisenstaedt R, Penninx BW, Woodman RC. Anemia in the elderly: current understanding and emerging concepts. Blood Rev. 2006 Jul;20(4):213-26. doi: 10.1016/j.blre.2005.12.002. Epub 2006 Feb 10.
  PMID: 16472893
  citation: Vichinsky E, Hoppe CC, Ataga KI, Ware RE, Nduba V, El-Beshlawy A, Hassab H, Achebe MM, Alkindi S, Brown RC, Diuguid DL, Telfer P, Tsitsikas DA, Elghandour A, Gordeuk VR, Kanter J, Abboud MR, Lehrer-Graiwer J, Tonda M, Intondi A, Tong B, Howard J; HOPE Trial Investigators. A Phase 3 Randomized Trial of Voxelotor in Sickle Cell Disease. N Engl J Med. 2019 Aug 8;381(6):509-519. doi: 10.1056/NEJMoa1903212. Epub 2019 Jun 14.
  PMID: 31199090
  citation: Howard J, Ataga KI, Brown RC, Achebe M, Nduba V, El-Beshlawy A, Hassab H, Agodoa I, Tonda M, Gray S, Lehrer-Graiwer J, Vichinsky E. Voxelotor in adolescents and adults with sickle cell disease (HOPE): long-term follow-up results of an international, randomised, double-blind, placebo-controlled, phase 3 trial. Lancet Haematol. 2021 May;8(5):e323-e333. doi: 10.1016/S2352-3026(21)00059-4. Epub 2021 Apr 7.
  PMID: 33838113
  citation: Howard J, Hemmaway CJ, Telfer P, Layton DM, Porter J, Awogbade M, Mant T, Gretler DD, Dufu K, Hutchaleelaha A, Patel M, Siu V, Dixon S, Landsman N, Tonda M, Lehrer-Graiwer J. A phase 1/2 ascending dose study and open-label extension study of voxelotor in patients with sickle cell disease. Blood. 2019 Apr 25;133(17):1865-1875. doi: 10.1182/blood-2018-08-868893. Epub 2019 Jan 17.
  PMID: 30655275
  citation: Inusa BPD, Hsu LL, Kohli N, Patel A, Ominu-Evbota K, Anie KA, Atoyebi W. Sickle Cell Disease-Genetics, Pathophysiology, Clinical Presentation and Treatment. Int J Neonatal Screen. 2019 May 7;5(2):20. doi: 10.3390/ijns5020020. eCollection 2019 Jun.
  PMID: 33072979
  citation: Grosse SD, Odame I, Atrash HK, Amendah DD, Piel FB, Williams TN. Sickle cell disease in Africa: a neglected cause of early childhood mortality. Am J Prev Med. 2011 Dec;41(6 Suppl 4):S398-405. doi: 10.1016/j.amepre.2011.09.013.
  PMID: 22099364
  citation: Piel FB, Patil AP, Howes RE, Nyangiri OA, Gething PW, Dewi M, Temperley WH, Williams TN, Weatherall DJ, Hay SI. Global epidemiology of sickle haemoglobin in neonates: a contemporary geostatistical model-based map and population estimates. Lancet. 2013 Jan 12;381(9861):142-51. doi: 10.1016/S0140-6736(12)61229-X. Epub 2012 Oct 25.
  PMID: 23103089
  citation: Anie KA, Green J. Psychological therapies for sickle cell disease and pain. Cochrane Database Syst Rev. 2002;(2):CD001916. doi: 10.1002/14651858.CD001916.
  PMID: 12076428
  citation: Crawford J, Cella D, Cleeland CS, Cremieux PY, Demetri GD, Sarokhan BJ, Slavin MB, Glaspy JA. Relationship between changes in hemoglobin level and quality of life during chemotherapy in anemic cancer patients receiving epoetin alfa therapy. Cancer. 2002 Aug 15;95(4):888-95. doi: 10.1002/cncr.10763.
  PMID: 12209734
  citation: Corona LP, Andrade FCD, da Silva Alexandre T, de Brito TRP, Nunes DP, de Oliveira Duarte YA. Higher hemoglobin levels are associated with better physical performance among older adults without anemia: a longitudinal analysis. BMC Geriatr. 2022 Mar 21;22(1):233. doi: 10.1186/s12877-022-02937-4.
  PMID: 35313814
  citation: Yenamandra A, Marjoncu D. Voxelotor: A Hemoglobin S Polymerization Inhibitor for the Treatment of Sickle Cell Disease. J Adv Pract Oncol. 2020 Nov-Dec;11(8):873-877. doi: 10.6004/jadpro.2020.11.8.7. Epub 2020 Nov 1.
  PMID: 33489427
  citation: Agarwala P, Salzman SH. Six-Minute Walk Test: Clinical Role, Technique, Coding, and Reimbursement. Chest. 2020 Mar;157(3):603-611. doi: 10.1016/j.chest.2019.10.014. Epub 2019 Nov 2.
  PMID: 31689414
  responsible party type: Sponsor
  mesh term: Anemia, Sickle Cell
  sharing ipd: No