Voxelotor for Sickle Cell Disease

Phase-Based Progress Estimates
Azienda Ospedaliera di Padova, Padova, ItalySickle Cell DiseaseVoxelotor - Drug
2 - 14
All Sexes
What conditions do you have?

Study 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.

Eligible Conditions
  • Sickle Cell Disease

Treatment Effectiveness

Effectiveness Progress

2 of 3
This is further along than 85% of similar trials

Study Objectives

1 Primary · 9 Secondary · Reporting Duration: 24, 48 and 96 weeks

24 weeks
TCD Measurement
Week 96
Change in Unconjugated Bilirubin
Week 96
Change in Absolute Reticulocyte
Change in Lactate Dehydrogenase (LDH)
Change in Percent of Reticulocyte
Conversion to Abnormal TCD Flow Velocity
Number of Participants with Change in Hemoglobin (Hb)
Reversion to Normal TCD Flow Velocity
TCD Flow Velocity Reduction
48 and 96 weeks
Change in TCD Flow Velocity

Trial Safety

Safety Progress

3 of 3
This is further along than 85% of similar trials

Trial Design

2 Treatment Groups

1 of 2
1 of 2

Experimental Treatment

Non-Treatment Group

236 Total Participants · 2 Treatment Groups

Primary Treatment: Voxelotor · Has Placebo Group · Phase 3

Experimental Group · 1 Intervention: Voxelotor · Intervention Types: Drug
PlaceboComparator Group · 1 Intervention: Placebos · Intervention Types: Drug
First Studied
Drug Approval Stage
How many patients have taken this drug
FDA approved

Trial Logistics

Trial Timeline

Screening: ~3 weeks
Treatment: Varies
Reporting: 24, 48 and 96 weeks

Who is running the clinical trial?

Global Blood TherapeuticsLead Sponsor
34 Previous Clinical Trials
5,305 Total Patients Enrolled

Eligibility Criteria

Age 2 - 14 · All Participants · 5 Total Inclusion Criteria

Mark “Yes” if the following statements are true for you:

Frequently Asked Questions

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." - Anonymous Online Contributor

Unverified Answer

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." - Anonymous Online Contributor

Unverified Answer

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." - Anonymous Online Contributor

Unverified Answer

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." - Anonymous Online Contributor

Unverified Answer

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." - Anonymous Online Contributor

Unverified Answer

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." - Anonymous Online Contributor

Unverified Answer

Are there Voxelotor side effects that patients should be made aware of?

"Given that this is a Phase 3 trial with some efficacy data and multiple rounds of safety data, we at Power rate the safety of Voxelotor as a 3." - Anonymous Online Contributor

Unverified Answer
Please Note: These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.