Overview

The Effect of Voxelotor on Cerebral Hemodynamic Response in Children With Sickle Cell Anemia

Status:
Not yet recruiting

Trial end date:
2022-10-01

Target enrollment:
0

Participant gender:
All

Summary

Voxelotor is a new drug for adolescents and adults with sickle cell disease that improves hemoglobin levels and reduces the incidence of worsening anemia. However, it is unclear whether this increase in hemoglobin is associated with a reduction in cerebral metabolic stress. This study will measure the effects of voxelotor on cerebral hemodynamics.

Phase:

Phase 2

Accepts Healthy Volunteers?

Details

Lead Sponsor:

Emory University

Collaborator:

Global Blood Therapeutics

Criteria

Inclusion Criteria:

1. Male or female participants, ages 4 to 17 years, inclusive

2. Homozygous hemoglobin SS (HbSS) or hemoglobin S/beta0 thalassemia (HbS/β0 thal)

3. Hemoglobin (Hb): Hb ≤10.5 g/dL at baseline

4. Concomitant hydroxyurea (HU) therapy is allowed if the dose has been stable for at
least 3 months with no anticipated need for dose adjustments during the study and no
sign of hematological toxicity

Exclusion Criteria:

1. Any one of the following requiring medical attention within 14 days prior to signing
the informed consent form (ICF):

- Vaso-occlusive crisis (VOC)

- Acute chest syndrome (ACS)

- Splenic sequestration crisis

- Dactylitis

2. Red blood cell (RBC) transfusion within 60 days of signing the ICF

3. Evidence of abnormal high blood flow velocities on transcranial doppler (TCD) of 200
cm/sec or more

4. Received an investigational drug within 30 days or 5 half-lives, whichever is longer,
of signing the ICF