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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 weeks3 visits

Treatment ~ Varies

Follow Up ~ 24, 48, and 96 weeks

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

Acute chest syndrome

100%

80%

60%

40%

20%

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

0 / 1Eligibility criteria met

Find a Location

Who is running the clinical trial?

PfizerLead Sponsor

4,570 Previous Clinical Trials

10,910,204 Total Patients Enrolled

Global Blood TherapeuticsLead Sponsor

35 Previous Clinical Trials

4,641 Total Patients Enrolled

Pfizer CT.gov Call CenterStudy DirectorPfizer

3,475 Previous Clinical Trials

8,091,318 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

Frequently Asked Questions

These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.

Are there other instances of Voxelotor being used in medical research?

"Currently, there are 7 different active studies investigating Voxelotor with 3 of them in Phase 3. Some of the clinical trials related to Voxelotor are happening in Philadelphia, Pennsylvania; however, there are a total of 139 locations running trials for Voxelotor."

Answered by AI

How many people are currently testing this treatment?

"In total, 224 patients that meet the specific inclusion criteria are needed for this clinical trial. Patients have the choice to participate in this trial at University of Miami Medical Center in Miami, Florida or University of Mississippi Medical Center in Jackson, Mississippi."

Answered by AI

Are people of an older age bracket still being allowed to enroll in this research?

"Eligible participants for this clinical study must be aged 2-14, as stated in the inclusion criteria."

Answered by AI

Who would be an ideal candidate to take part in this clinical trial?

"To qualify for this trial, applicants must be aged 2-14, have anemia, and sickle cell. Additionally, the applicant must be either male or female."

Answered by AI

Are there any available spots left for participants in this research?

"The information available on clinicaltrials.gov seems to suggest that this study is currently looking for participants. The trial was first posted on 4/7/2021 and was last updated on 8/16/2022. The study is admitting a total of 224 participants at 10 sites."

Answered by AI

Have other research teams looked into this same topic previously?

"Research into Voxelotor began in 2016. The first, sponsored by Global Blood Therapeutics, 155-person study was completed that year. Based on the data from that initial study, Voxelotor received approval for Phase 2 clinical trials. There are currently 7 active studies involving Voxelotor taking place in 39 cities across 14 countries."

Answered by AI