← Back to Search

Other

Voxelotor for Sickle Cell Disease

Phase 3

Waitlist Available

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

Must not have

Female participant who is breastfeeding or pregnant

Use of St. John's wort, sensitive cytochrome P450 (CYP) 3A4 substrates with a narrow therapeutic index, or moderate or strong CYP3A4 inducers within 30 days of Day 1

Timeline

Screening 3 weeks

Treatment Varies

Follow Up throughout entire study

Awards & highlights

Pivotal Trial

No Placebo-Only Group

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.

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.

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.

Exclusion Criteria

You may be eligible for the trial if you check “No” for criteria below:

Select...

I am currently pregnant or breastfeeding.

Select...

I haven't taken St. John's wort or certain strong medications in the last 30 days.

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

Study objectives can provide a clearer picture of what you can expect from a treatment.

Primary study objectives

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

Awards & Highlights

Pivotal Trial

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

No Placebo-Only Group

All patients enrolled in this study will receive some form of active treatment.

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 / 3Eligibility criteria met