General Information

Age Group

Adults

Status

Recruiting

Protocol Number

NCT06023199

Background Information

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.

We intend to examine the effect of voxelotor treatment on performance using the 6-Minute Walk Test (6MWT).
The study also hopes to observe the effect of voxelotor on pulmonary hypertension parameters using echocardiography/ brain natriuretic peptide, grip strength improvement as well as subjective improvement in quality-of-life measures.

For more information visit:  https://clinicaltrials.gov/study/NCT06023199

Offered At

Inova Schar Cancer
A Division of Inova Fairfax Hospital
8081 Innovation Park Drive
Fairfax, VA 22031
Principal Investigator

Sheinei Alan, MD

Eligibility Information

  • 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
  • Additional inclusion criteria in protocol

Ineligibility Information

  • Hemoglobin results < 6 g/dL or > 10.5 g/dL
  • Participants receiving hydroxyurea who have had a dose change within the prior 3 months
  • Females who are breastfeeding or pregnant
  • Participants 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
  • Prior hypersensitivity to voxelotor or its excipients
  • Additional ineligibility in protocol