Status:
ACTIVE_NOT_RECRUITING
Assessing Physical Function in Sickle Cell Patients Taking Voxelotor
Lead Sponsor:
Inova Health Care Services
Collaborating Sponsors:
Pfizer
Conditions:
Sickle Cell Disease
Eligibility:
All Genders
18-65 years
Phase:
PHASE2
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 o...
Eligibility Criteria
Inclusion
- Provision of signed and dated informed consent form
- Stated willingness to comply with all study procedures and availability for the duration of the study
- Male or female,
- aged 18-65 years
- Diagnosis of sickle cell disease with HbSS, HbSc, HbS-beta thalassemia genotypes
- Hemoglobin level of 6 g/dL - 10.5 g/dL
- Between 0 and 10 vaso-occlusive (VOC) episodes in previous 12 months
- Ability to take oral medication and be willing to adhere to the voxelotor regimen
- 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
- For males of reproductive potential: use of condoms or other methods to ensure effective contraception with partner
- Agreement to adhere to Lifestyle Considerations (see section 5.3) throughout study duration
Exclusion
- Hemoglobin results \< 6 g/dL or \> 10.5 g/dL
- Patients receiving hydroxyurea who have had a dose change within the prior 3 months
- Females who are breastfeeding or pregnant
- Patients who are receiving regularly scheduled blood (RBC) transfusion therapy (also termed chronic, prophylactic, or preventive transfusion) within 30 days of 6MWT
- Any form of hepatic dysfunction as demonstrated by alanine aminotransferase (ALT) \>4 × ULN or Child-Pugh Class C
- Acute kidney injury or chronic renal disease with estimated glomerular filtration rate\<30mL/min/1.73 m2
- Current or previous diagnosis of malignancy within the last two years excluding local therapy for non-melanoma skin malignancy
- Unstable cardiopulmonary disease within the last 6 months prior to study enrollment
- Any conditions including behavioral or medical that in the opinion of the investigator, may negatively impact informed consent, participation and study interpretation
- Current use of commercially available voxelotor
- Prior hypersensitivity to voxelotor or its excipients
- Patients with baseline gait and other physical impairments that would limit 6MWT and other functional assessment at discretion of the investigator
- 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.
Key Trial Info
Start Date :
October 23 2023
Trial Type :
INTERVENTIONAL
Allocation :
ACTUAL
End Date :
December 23 2025
Estimated Enrollment :
6 Patients enrolled
Trial Details
Trial ID
NCT06023199
Start Date
October 23 2023
End Date
December 23 2025
Last Update
November 26 2025
Active Locations (1)
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1
Inova Schar Cancer Institute
Falls Church, Virginia, United States, 22042