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Assessing Physical Function in Sickle Cell Patients Taking Voxelotor

Sponsor
Inova Health Care Services (Other)
Overall Status
Not yet recruiting
CT.gov ID
NCT06023199
Collaborator
Pfizer (Industry)
10
Enrollment
1
Location
9
Anticipated Duration (Months)
1.1
Patients Per Site Per Month

Study Details

Study Description

Brief Summary

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.

Condition or Disease Intervention/Treatment Phase

Study Design

Study Type:
Observational
Anticipated Enrollment :
10 participants
Observational Model:
Cohort
Time Perspective:
Prospective
Official Title:
Assessing Functional Capacity in Sickle Cell Patients Receiving Voxelotor
Anticipated Study Start Date :
Sep 15, 2023
Anticipated Primary Completion Date :
Mar 15, 2024
Anticipated Study Completion Date :
Jun 15, 2024

Outcome Measures

Primary Outcome Measures

  1. Participant's Physical Performance Using 6 Minutes Walk Test (6MWT) Pre and Post Treatment with Voxelotor. [6 months from the treatment initiation]

    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.

Other Outcome Measures

  1. The Change in the Quality of Life as assessed by Sheehan Disability Scale (SDS) During the 6 Months Trial Period. [6 months from the treatment initiation]

    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).

Eligibility Criteria

Criteria

Ages Eligible for Study:
18 Years to 65 Years
Sexes Eligible for Study:
All
Accepts Healthy Volunteers:
No
Inclusion Criteria:

  1. Provision of signed and dated informed consent form

  2. Stated willingness to comply with all study procedures and availability for the duration of the study

  3. Male or female,

  4. aged 18-65 years

  5. Diagnosis of sickle cell disease with HbSS, HbSc, HbS-beta thalassemia genotypes

  6. Hemoglobin level of 6 g/dL - 10.5 g/dL

  7. Between 0 and 10 vaso-occlusive (VOC) episodes in previous 12 months

  8. Ability to take oral medication and be willing to adhere to the voxelotor regimen

  9. For females of reproductive potential, they will be educated to avoid pregnancy during study period and for one month after due to limited data surrounding pregnancy and voxelotor

  10. For males of reproductive potential: use of condoms or other methods to ensure effective contraception with partner

  11. Agreement to adhere to Lifestyle Considerations (see section 5.3) throughout study duration

Exclusion Criteria:

  1. Hemoglobin results < 6 g/dL or > 10.5 g/dL

  2. Patients receiving hydroxyurea who have had a dose change within the prior 3 months

  3. Females who are breastfeeding or pregnant

  4. Patients who are receiving regularly scheduled blood (RBC) transfusion therapy (also termed chronic, prophylactic, or preventive transfusion) within 30 days of 6MWT

  5. Any form of hepatic dysfunction as demonstrated by alanine aminotransferase (ALT) >4 × ULN or Child-Pugh Class C

  6. Acute kidney injury or chronic renal disease with estimated glomerular filtration rate<30mL/min/1.73 m2

  7. Current or previous diagnosis of malignancy within the last two years excluding local therapy for non-melanoma skin malignancy

  8. Unstable cardiopulmonary disease within the last 6 months prior to study enrollment

  9. Any conditions including behavioral or medical that in the opinion of the investigator, may negatively impact informed consent, participation and study interpretation

  10. Current use of commercially available voxelotor

  11. Prior hypersensitivity to voxelotor or its excipients

  12. Patients with baseline gait and other physical impairments that would limit 6MWT and other functional assessment at discretion of the investigator

  13. Patients who on day of assessment present with vaso-occlusive crisis or rate their pain greater than 6 on a scale of 1-10 using the Numeric Rating Scale will not be assessed that day and re-screened at the investigator's discretion.

Contacts and Locations

Locations

Site City State Country Postal Code
1 Inova Schar Cancer Institute Falls Church Virginia United States 22042

Sponsors and Collaborators

  • Inova Health Care Services
  • Pfizer

Investigators

  • Principal Investigator: Sheinei Alan, Inova Schar Cancer Institute

Study Documents (Full-Text)

None provided.

More Information

Publications

Responsible Party:
Inova Health Care Services
ClinicalTrials.gov Identifier:
NCT06023199
Other Study ID Numbers:
  • U23-02-4988
First Posted:
Sep 5, 2023
Last Update Posted:
Sep 5, 2023
Last Verified:
Aug 1, 2023
Individual Participant Data (IPD) Sharing Statement:
No
Plan to Share IPD:
No
Studies a U.S. FDA-regulated Drug Product:
Yes
Studies a U.S. FDA-regulated Device Product:
No
Product Manufactured in and Exported from the U.S.:
No
Additional relevant MeSH terms:
Anemia, Sickle Cell

Study Results

No Results Posted as of Sep 5, 2023