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Hemoglobin Modifiers

Voxelotor for Sickle Cell Disease (HOPE Kids 2 Trial)

Phase 3
Waitlist Available
Research Sponsored by Pfizer
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial
Must have
Male or female participants with Sickle Cell Anemia (SCA)
Be younger than 18 years old
Must not have
Known history or findings suggestive of significant cerebral vasculopathy (eg, moyamoya or significant vasculopathy)
History of seizure disorder
Timeline
Screening 3 weeks
Treatment Varies
Follow Up 24, 48, and 96 weeks
Awards & highlights
Pivotal Trial

Summary

This trial is testing a new medication to see if it can help kids with sickle cell disease. The goal is to see if it can reduce problems with blood flow in the brain.

Who is the study for?
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.
What is being tested?
The 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.
What are the potential side effects?
Possible side effects of Voxelotor may include headache, diarrhea, abdominal pain, nausea, fatigue, rash and fever. The severity of these side effects can vary from one individual to another.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below
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I have Sickle Cell Anemia.

Exclusion Criteria

You may be eligible for the trial if you check “No” for criteria below:
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I have a history of serious brain blood vessel problems.
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I have a history of seizures.
Select...
I've had over 10 severe pain crises needing hospital or clinic visits in the last year.
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I weigh less than 10kg.
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I have had a stroke that caused specific physical or mental problems.
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I have received a red blood cell transfusion within the last 90 days.
Select...
I was hospitalized for a pain crisis or lung issue related to my condition within the last 14 days.

Timeline

Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~24, 48, and 96 weeks
This trial's timeline: 3 weeks for screening, Varies for treatment, and 24, 48, and 96 weeks for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.
Primary study objectives
TCD Measurement
Secondary study objectives
TCD Flow Velocity Reduction
Time to Conversion to Abnormal TCD Flow Velocity
Time to Reversion to Normal TCD Flow Velocity

Side effects data

From 2022 Phase 4 trial • 25 Patients • NCT04400487
32%
Sickle cell anaemia with crisis
4%
Acute chest syndrome
100%
80%
60%
40%
20%
0%
Study treatment Arm
Voxelotor: SCD Related
Voxelotor: Non-SCD Related

Awards & Highlights

Pivotal Trial
The final step before approval, pivotal trials feature drugs that have already shown basic safety & efficacy.

Trial Design

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.
Treatment
First Studied
Drug Approval Stage
How many patients have taken this drug
Voxelotor
2023
Completed Phase 4
~90

Find a Location

Who is running the clinical trial?

PfizerLead Sponsor
4,669 Previous Clinical Trials
17,861,688 Total Patients Enrolled
Global Blood TherapeuticsLead Sponsor
35 Previous Clinical Trials
2,980 Total Patients Enrolled
Pfizer CT.gov Call CenterStudy DirectorPfizer
3,553 Previous Clinical Trials
14,902,690 Total Patients Enrolled

Media Library

Voxelotor (Hemoglobin Modifiers) Clinical Trial Eligibility Overview. Trial Name: NCT04218084 — Phase 3
Sickle Cell Disease Research Study Groups: Voxelotor, Placebo
Sickle Cell Disease Clinical Trial 2023: Voxelotor Highlights & Side Effects. Trial Name: NCT04218084 — Phase 3
Voxelotor (Hemoglobin Modifiers) 2023 Treatment Timeline for Medical Study. Trial Name: NCT04218084 — Phase 3
~46 spots leftby Dec 2025
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