GBT021601 for Sickle Cell Disease

Recruiting at 10 trial locations

Overseen By Pfizer Pfizer CT.gov Call Center

Age: Any Age

Sex: Any

Trial Phase: Phase 2 & 3

Sponsor: Pfizer

Must not be taking: Voxelotor, Crizanlizumab

Disqualifiers: Noncompliance, Investigational drugs, others

No Placebo Group All trial participants will receive the active study treatment (no placebo)

Prior Safety Data This treatment has passed at least one previous human trial

Compensation: Varies

Number of Visits: Unknown

Duration: Up to 5 years

47 Participants Needed

Trial Summary

What is the purpose of this trial?

An Open-label Extension Study of GBT021601 in Participants with Sickle Cell Disease

Research Team

Pfizer CT.gov Call Center

Principal Investigator

Pfizer

Eligibility Criteria

This trial is for males and females aged 6 months or older with Sickle Cell Disease who were part of a previous GBT021601 study. They must use effective contraception if sexually active, have provided consent, and not have withdrawn from the earlier study. Those who recently used voxelotor or were noncompliant are excluded.

Inclusion Criteria

I will use barrier contraception methods for 120 days after my last dose.

Participant has provided written informed consent/assent. For underage participants, both the consent of the participant's legal representative or legal guardian and the participant's assent (where applicable) must be obtained based on local requirements

I am using reliable birth control methods during and for 4 months after the study.

Exclusion Criteria

Participant withdrew consent or was noncompliant from the originating GBT021601 clinical study

I have taken voxelotor within the last 10 days.

Timeline

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive GBT021601 to evaluate long-term safety and efficacy

Up to 5 years

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

Open-label extension

Participants continue to receive GBT021601 to assess long-term safety

Up to 5 years

Treatment Details

Interventions

• GBT021601

Trial Overview The trial is testing an open-label drug called GBT021601 in participants with Sickle Cell Disease to see its long-term effects. It's an extension of a prior study where participants continue receiving this medication.

Participant Groups

Group I: Treatment Experimental Treatment 1 Intervention

Osivelotor

Who Is Running the Clinical Trial?

Pfizer

Lead Sponsor

Trials

4,712

Recruited

50,980,000+

Known For

Vaccine Innovations

Top Products

Viagra, Zoloft, Lipitor, Prevnar 13

Albert Bourla

Pfizer

Chief Executive Officer since 2019

PhD in Biotechnology of Reproduction, Aristotle University of Thessaloniki

Patrizia Cavazzoni

Pfizer

Chief Medical Officer

MD from McGill University

Global Blood Therapeutics

Lead Sponsor

Trials

36

Recruited

3,200+