Voxelotor for Sickle Cell Disease
(HOPE Kids 2 Trial)

Recruiting in Palo Alto (17 mi)

+77 other locations

Age: < 18

Sex: Any

Travel: May Be Covered

Time Reimbursement: Varies

Trial Phase: Phase 3

Waitlist Available

Sponsor: Pfizer

Pivotal Trial (Near Approval)

Prior Safety Data

Breakthrough Therapy

Approved in 2 Jurisdictions

Trial Summary

What is the purpose of this trial?

This study is a Phase 3, randomized, double-blind, placebo-controlled study of voxelotor in pediatric participants, aged ≥ 2 to \< 15 years old, with Sickle Cell Disease. The primary objective is to evaluate the effect of voxelotor on the TCD (Transcranial Doppler Ultrasound) measurements in SCD participants in this age range.

Research Team

Pfizer CT.gov Call Center

Principal Investigator

Pfizer

Eligibility Criteria

This trial is for children aged 2 to less than 15 with Sickle Cell Disease who have specific blood flow rates in their brains and stable hemoglobin levels. They must not have had frequent hospital visits for pain, recent blood transfusions, or weigh under 10kg. Those on hydroxyurea need a stable dose for at least 90 days.

Inclusion Criteria

I have been on a stable dose of HU for at least 90 days without expecting changes.

I have Sickle Cell Anemia.

TCD time averaged maximum of the mean velocity (TAMMV) arterial cerebral blood flow ≥ 170 to < 200cm/sec during the Screening Period

See 2 more

Exclusion Criteria

I have a history of serious brain blood vessel problems.

I have a history of seizures.

I've had over 10 severe pain crises needing hospital or clinic visits in the last year.

See 5 more

Treatment Details

Interventions

Placebos (Other)
Voxelotor (Hemoglobin Modifiers)

Trial OverviewThe study tests Voxelotor, a medication intended to help manage Sickle Cell Disease, against a placebo (a substance with no therapeutic effect). It's designed to see if Voxelotor can improve blood flow in the brain as measured by Transcranial Doppler Ultrasound.

Participant Groups

2Treatment groups

Experimental Treatment

Placebo Group

Group I: VoxelotorExperimental Treatment1 Intervention

Voxelotor 1500mg or equivalent daily as a tablet, dispersible tablet, or as powder for oral suspension.

Group II: PlaceboPlacebo Group1 Intervention

Matching placebo.

Voxelotor is already approved in United States, European Union for the following indications:

🇺🇸 Approved in United States as Oxbryta for:

Sickle Cell Disease in patients aged 4 years and older

🇪🇺 Approved in European Union as Oxbryta for:

Hemolytic anemia due to sickle cell disease in adults and pediatric patients 12 years of age and older as monotherapy or in combination with hydroxyurea