GBT021601 for Sickle Cell Disease

Recruiting at61 trial locations

Age: Any Age

Sex: Any

Travel: May Be Covered

Time Reimbursement: Varies

Trial Phase: Phase 2 & 3

Waitlist Available

Sponsor: Pfizer

Prior Safety Data

Trial Summary

What is the purpose of this trial?

This trial is testing an oral medication called osivelotor to see if it can safely help people with sickle cell disease feel better. It will also study how the medication works in the body.

Research Team

Pfizer CT.gov Call Center

Principal Investigator

Pfizer

Eligibility Criteria

This trial is for individuals aged 12-65 with Sickle Cell Disease (SCD) who have had 2 to 10 pain episodes related to the disease in the past year. They must not be pregnant or breastfeeding, and should not need regular blood transfusions or have received one within the last 90 days.

Inclusion Criteria

Participants with stable Hb value as judged by the Investigator

Part B:

I am between 12 and 65 years old and have sickle cell disease.

Exclusion Criteria

I am currently pregnant or breastfeeding.

I have had more than 10 pain crises in the last year.

I regularly get or have had a red blood cell transfusion in the last 3 months.

Treatment Details

Interventions

• GBT021601 (Unknown)

Trial Overview The study is testing GBT021601's safety and effectiveness in treating SCD. It will look at how the body processes the drug and its impact on health outcomes for both adults and children with stable hemoglobin levels.

Participant Groups

Group I: Part C Experimental Treatment 1 Intervention

100 mg dose in cohort C1, dose level for cohorts C2 to C4 to be determined based on emerging data

Group II: Part A Active Control 1 Intervention

Initially, participants will be randomized 1:1 to 100 mg and 150 mg daily. Upon review of the 150 mg safety data from at least 6 participants, there will be 1:1:1 randomization: 100 mg, 150 mg, and up to 200 mg. Participants will then receive maintenance once daily doses through Week 12.

Group III: Part B Placebo Group 1 Intervention

Following the selection of the optimal safe and effective dose from Part A of the study, Part B of the study will assess the efficacy and safety of 48 weeks of the optimal dose, compared to placebo

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+