Assessing Physical Function in Sickle Cell Patients Taking Voxelotor

Running, not enrolling · Phase 2

Conditions studied: Sickle Cell Disease

In brief

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.

Key facts

Study ID

NCT06023199

Run by

Inova Health Care Services

People needed

6

Starts

2023-10-23

Expected to finish

2026-06-15

Last updated by the study team

2026-02-02

Who can join

Age: 18 and older, up to 65. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

• 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

You may not qualify if…

• 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.

Where it is running

• Inova Schar Cancer Institute — Falls Church, Virginia, United States

Full record on ClinicalTrials.gov

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