Voxelotor Brain Oxygenation and Neurocognitive Study

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

Get a concise snapshot of the trial, including recruitment status, study phase, enrollment targets, and key timeline milestones.

Recruitment Status

WITHDRAWN

Clinical Phase

PHASE4

Study Classification

INTERVENTIONAL

Study Start Date

2023-08-09

Study Completion Date

2023-08-23

Brief Summary

Review the sponsor-provided synopsis that highlights what the study is about and why it is being conducted.

This is an open label, single arm multicenter trial to evaluate the effect of voxelotor treatment on cerebral blood flow (CBF) and neurocognitive function in adolescent and young adult participants (12-30 years of age) with sickle cell disease (SCD).

Related Clinical Trials

Explore similar clinical trials based on study characteristics and research focus.

Voxelotor Neurocognitive Function Study

NCT05228834

Sickle Cell Disease

TERMINATED PHASE3

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

NCT05018728

Sickle Cell Anemia in Children

TERMINATED PHASE2

A Voxelotor for Sickle Cell Anemia Patients at Highest Risk for Progression of Chronic Kidney Disease

NCT04335721

Sickle Cell Disease Sickle Cell Nephropathy

TERMINATED PHASE1/PHASE2

Voxelotor Sickle Cell Exercise Study

NCT04581356

Sickle Cell Anemia

COMPLETED PHASE4

Assessing Physical Function in Sickle Cell Patients Taking Voxelotor

NCT06023199

Sickle Cell Disease

ACTIVE_NOT_RECRUITING PHASE2

Detailed Description

Dive into the extended narrative that explains the scientific background, objectives, and procedures in greater depth.

Eligible participants will receive daily treatment with 1500 mg voxelotor for 24 weeks. During screening, at 12 and 24 weeks, participants will undergo an MRI for evaluation of cerebral blood flow and oxygen extraction fraction as well as NIH toolbox testing for evaluation of executive function, processing speed, and nonexecutive function.

Conditions

See the medical conditions and disease areas that this research is targeting or investigating.

Sickle Cell Disease

Study Design

Understand how the trial is structured, including allocation methods, masking strategies, primary purpose, and other design elements.

Allocation Method

NA

Intervention Model

SINGLE_GROUP

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

Review each arm or cohort in the study, along with the interventions and objectives associated with them.

Active Drug

Generic Name: Voxelotor Dosage Form: tablet Dosage: 1500mg Frequency: QD Duration: 24 weeks

Group Type EXPERIMENTAL

Voxelotor Oral Tablet

Intervention Type DRUG

During the Treatment Period, participants will receive 1500 mg of voxelotor once daily (administered as tablets) for 24 weeks in addition to ongoing current standard of care (SOC) treatment

Interventions

Learn about the drugs, procedures, or behavioral strategies being tested and how they are applied within this trial.

Voxelotor Oral Tablet

During the Treatment Period, participants will receive 1500 mg of voxelotor once daily (administered as tablets) for 24 weeks in addition to ongoing current standard of care (SOC) treatment

Intervention Type DRUG

Other Intervention Names

Discover alternative or legacy names that may be used to describe the listed interventions across different sources.

Oxbryta

Eligibility Criteria

Check the participation requirements, including inclusion and exclusion rules, age limits, and whether healthy volunteers are accepted.

Inclusion Criteria

1. Male or female participants with confirmed diagnosis of SCD with HbSS or Hbβ0 thalassemia genotype. Documentation of SCD genotype is required and may be based on documented history of laboratory testing or confirmed by laboratory testing during Screening.
2. Aged 12 to 30 years.
3. Screening Hb level ≥ 5.5 and ≤ 10.5 g/dL.
4. Must meet site-specific compliance requirements for a diagnostic MRI scan.
5. Able to answer NIH Toolbox Module questions in English
6. If participant is receiving hydroxyurea (HU) they must have been on a stable dose for at least 90 days prior to signing the ICF/AF, with no dose modifications or initiation of HU planned or anticipated by the Investigator.
7. If participant is receiving erythropoiesis-stimulating agents (ESAs) they must have been on a stable dose for at least 12 weeks before enrollment with no dose modifications planned or anticipated by the Investigator.
8. Participants, who if female and of child-bearing potential, agree to use highly effective methods of contraception from study start to 30 days after the last dose of study drug and who if male, agree to use barrier methods of contraception and refrain from donating sperm from study start to 30 days after the last dose of study drug.
9. Females of child-bearing potential must have a negative pregnancy test before the administration of study drug.
10. Written informed consent (≥ 18 years) or parental/guardian consent and participant assent (≥ 12-17 years) per Institutional Review Board (IRB) policy and requirements, consistent with ICH guidelines.
11. Capable of complying with the requirements and restrictions in the protocol, and willing to participate in the study.

Exclusion Criteria

1. History of overt stroke including hemorrhagic stroke, transient ischemic attacks, or spinal cord injury.
2. Grade 4 vasculopathy defined as moderate stenosis (50% to 69%) in more than 2 major cerebral arteries or severe stenosis (\> 70%) in any major cerebral artery.
3. Non-MRI compatible metal hardware and/or metal braces.
4. Congenital brain malformation, previously diagnosed severe developmental disability (eg autism and/or intelligence quotient \[IQ\] \<60, and/or severe attention deficit hyperactivity disorder \[ADHD\]), or impairment that would prevent the use of a computer tablet.
5. Participant is taking or has received voxelotor (Oxbryta®) within 90 days prior to the Screening Visit.
6. Participant is taking or has received crizanlizumab (Adakveo®) within 90 days prior to the Screening Visit.
7. Vaso-occlusive event requiring intravenous opioids within 28 days prior to Day 1.
8. Red blood cell (RBC) transfusion within 3 months before initiation of study drug or receives scheduled RBC transfusion therapy (also termed chronic, prophylactic, or preventive transfusion).
9. Surgery within 8 weeks before Day 1 or planned elective surgery during the study.
10. Anemia due to bone marrow failure (eg, myelodysplasia).
11. Absolute reticulocyte count (ARC) \< 100 × 10\^9/L.
12. Screening alanine aminotransferase or aspartate aminotransferase \> 4× upper limit of normal (ULN).
13. Severe renal dysfunction (estimated glomerular filtration rate \[eGFR\] \<45 mL/min/1.73 m\^2) or on chronic dialysis.
14. Clinically significant bacterial, fungal, parasitic, or viral infection which requires therapy

1. Acute bacterial infection requiring antibiotic use should delay Screening/enrollment until the course of antibiotic therapy has been completed.
2. Known active hepatitis A, B, or C or are known to be human immunodeficiency virus (HIV) positive.
15. Symptomatic coronavirus disease of 2019 (COVID-19) infection.
16. Females who are breast-feeding or pregnant.
17. History of hematopoietic stem cell transplant or gene therapy.
18. Participants taking concomitant medications such as sensitive CYP3A4 substrates with a narrow therapeutic range, or strong CYP3A4 inducers.
19. Participated in another clinical trial of an investigational product (or medical device) within 30 days or 5 half-lives of date of informed consent, whichever is longer, or is currently participating in another trial of an investigational product (or medical device).
20. Medical, psychological, or behavioral condition that, in the opinion of the Investigator, would confound or interfere with evaluation of safety and/or efficacy of the study drug, prevent compliance with the study protocol; preclude informed consent; or render the participant unable/unlikely to comply with the study procedures (particularly the MRI scan).

Minimum Eligible Age

12 Years

Maximum Eligible Age

30 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No