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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
Timeline
Screening 3 weeks
Treatment Varies
Follow Up 24, 48, and 96 weeks
Awards & highlights

HOPE Kids 2 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.Check my eligibility
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.See study design
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.

HOPE Kids 2 Trial Eligibility Criteria

Inclusion Criteria

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

HOPE Kids 2 Trial 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

Outcome measures can provide a clearer picture of what you can expect from a treatment.
Primary outcome measures
TCD Measurement
Secondary outcome measures
Annualized incidence rate of vaso-occlusive crises (VOCs)
Change in Absolute Reticulocyte from baseline
Change in Lactate Dehydrogenase (LDH) from baseline
+7 more

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

HOPE Kids 2 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
4,580 Previous Clinical Trials
14,633,967 Total Patients Enrolled
Global Blood TherapeuticsLead Sponsor
35 Previous Clinical Trials
4,641 Total Patients Enrolled
Pfizer CT.gov Call CenterStudy DirectorPfizer
3,485 Previous Clinical Trials
11,810,842 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
~52 spots leftby Jun 2025
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