← Back to Search

Other

Voxelotor for Sickle Cell Disease

Q: Does this research extend to geriatric patients?

In order to be included in this study, participants must meet the age requirement of being between 6 months to 18 years old.

Q: Are there other healthcare facilities conducting this research in our state?

Although there are 9 other locations, the recruiting patients for this study are at Our Lady of the Lake Regional Medical Center in Baton Rouge, Children's National Medical Center in <a href="https://www.withpower.com/clinical-trials/washington">Washington</a>, and Children's Mercy Hospital <a href="https://www.withpower.com/clinical-trials/kansas">Kansas</a> City in Kansas City.

Q: Have there been any similar studies to this one previously?

Voxelotor has been under investigation since 2016. The first clinical trial, sponsored by Global Blood Therapeutics, completed in that same year. With 155 participants, the study yielded positive results and led to the drug's Phase 2 approval. Currently, there are 7 active trials taking place in 39 cities and 14 countries.

Q: Could you please share what research has been done on Voxelotor in the past?

There are a total of 7 different clinical trials still recruiting patients for Voxelotor. Out of these, 3 are in Phase 3. Many of these trial sites are located in or around Philadelphia, with a total of 139 locations running trials for Voxelotor.

Q: For which demographics is this trial seeking volunteers?

Anemia and <a href="https://www.withpower.com/clinical-trials/sickle-cell">sickle cell</a> are the primary qualifications for this study. Additionally, children between the ages of 6 months to 18 years old are accepted. In total, this clinical trial is looking for 600 participants.

Q: Has Voxelotor undergone drug testing by the FDA?

There is some evidence to support the efficacy of Voxelotor and it has undergone multiple rounds of testing, making it a safe drug with a score of 3.

Q: Are there any unfilled roles for participants in this research project?

Data from clinicaltrials.gov suggests that this trial is not currently looking for patients. This information is accurate as of September 27th, 2022. Although this study is no longer enrolling patients, there are 208 other trials that are.

Phase 3

Recruiting

Research Sponsored by Global Blood Therapeutics

Eligibility Criteria Checklist

Specific guidelines that determine who can or cannot participate in a clinical trial

Must have

Male or female participant with SCD, who participated and received study drug in a GBT-sponsored voxelotor clinical study

Be younger than 65 years old

Timeline

Screening 3 weeks

Treatment Varies

Follow Up throughout entire study

Awards & highlights

Study Summary

This trial is testing a new drug for sickle cell disease in children up to 18 years old who have already participated in other trials for the drug.

Who is the study for?

This trial is for men and women with Sickle Cell Disease who were previously in a voxelotor study. They must agree to the study's terms, not be pregnant or breastfeeding, and haven't used certain drugs that affect enzymes in the liver within the last 30 days.Check my eligibility

What is being tested?

The trial is an open-label extension which means everyone gets Voxelotor. It's for those who've already taken it in past trials to continue observing its effects over a longer period without any placebo control.See study design

What are the potential side effects?

While specific side effects aren't listed here, Voxelotor could potentially cause issues like headache, nausea, fatigue, or allergic reactions based on how it affects individuals with Sickle Cell Disease.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below

Select...

I have sickle cell disease and was part of a voxelotor study.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ throughout entire study

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~throughout entire study

This trial's timeline: 3 weeks for screening, Varies for treatment, and throughout entire study for reporting.

Treatment Details

Study Objectives

Outcome measures can provide a clearer picture of what you can expect from a treatment.

Primary outcome measures

Sickle Cell Disease-Related Complications

TEAEs and SAEs

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

Trial Design

1Treatment groups

Experimental Treatment

Group I: VoxelotorExperimental Treatment1 Intervention

All participants will receive voxelotor once daily (QD), administered orally as tablets, dispersible tablets, or a stick pack formulation (powder blend formulation packaged as stick packs). Participants aged ≥ 12 years and/or ≥ 40 kgs will receive a voxelotor dose of 1500 mg QD. Participants aged < 12 years and < 40 kgs will receive weight based dosing of voxelotor. The participant's weight at study entry will be used to determine the starting voxelotor dose in this study. Participants may receive study drug as long they continue to receive clinical benefit that outweighs risk as determined by the investigator and/or until the participant has access to voxelotor from an alternative source.

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?

Global Blood TherapeuticsLead Sponsor

35 Previous Clinical Trials

4,577 Total Patients Enrolled

PfizerLead Sponsor

4,570 Previous Clinical Trials

10,910,685 Total Patients Enrolled

Margaret Tonda, PharmDStudy DirectorGlobal Blood Therapeutics, Inc.

3 Previous Clinical Trials

776 Total Patients Enrolled

Media Library

Voxelotor (Other) Clinical Trial Eligibility Overview. Trial Name: NCT04188509 — Phase 3

Sickle Cell Disease Research Study Groups: Voxelotor

Sickle Cell Disease Clinical Trial 2023: Voxelotor Highlights & Side Effects. Trial Name: NCT04188509 — Phase 3

Voxelotor (Other) 2023 Treatment Timeline for Medical Study. Trial Name: NCT04188509 — Phase 3

Frequently Asked Questions

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

Does this research extend to geriatric patients?

"In order to be included in this study, participants must meet the age requirement of being between 6 months to 18 years old."

Answered by AI

Are there other healthcare facilities conducting this research in our state?

"Although there are 9 other locations, the recruiting patients for this study are at Our Lady of the Lake Regional Medical Center in Baton Rouge, Children's National Medical Center in Washington, and Children's Mercy Hospital Kansas City in Kansas City."

Answered by AI

Have there been any similar studies to this one previously?

"Voxelotor has been under investigation since 2016. The first clinical trial, sponsored by Global Blood Therapeutics, completed in that same year. With 155 participants, the study yielded positive results and led to the drug's Phase 2 approval. Currently, there are 7 active trials taking place in 39 cities and 14 countries."

Answered by AI

Could you please share what research has been done on Voxelotor in the past?

"There are a total of 7 different clinical trials still recruiting patients for Voxelotor. Out of these, 3 are in Phase 3. Many of these trial sites are located in or around Philadelphia, with a total of 139 locations running trials for Voxelotor."

Answered by AI

For which demographics is this trial seeking volunteers?

"Anemia and sickle cell are the primary qualifications for this study. Additionally, children between the ages of 6 months to 18 years old are accepted. In total, this clinical trial is looking for 600 participants."

Answered by AI

Has Voxelotor undergone drug testing by the FDA?

"There is some evidence to support the efficacy of Voxelotor and it has undergone multiple rounds of testing, making it a safe drug with a score of 3."

Answered by AI

Are there any unfilled roles for participants in this research project?

"Data from clinicaltrials.gov suggests that this trial is not currently looking for patients. This information is accurate as of September 27th, 2022. Although this study is no longer enrolling patients, there are 208 other trials that are."

Answered by AI

Recent research and studies

clinicaltrials.govStudy

Open-Label Extension of Voxelotor

2019. "Open-Label Extension of Voxelotor". ClinicalTrials.gov. https://clinicaltrials.gov/ct2/show/NCT04188509.

All > Sickle Cell Anemia