TerminatedPhase 2/3

GBT021601-022: A Study of GBT021601 in Participants With Sickle Cell Disease (SCD)

Stopped: Study terminated as data obtained from this non-placebo-controlled trial may not be informative for the interpretation of the benefits and risks of osivelotor

United States47 participantsStarted 2023-01-05

Plain-language summary

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

Who can participate

Age range6 Months

SexALL

See this in plain English?

AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

Inclusion criteria

✓. Male or female aged 6 months or older with SCD who participated and received study drug or placebo in a previous osivelotor clinical study and completed the end of treatment visit.
✓. Females of childbearing potential are required to have a negative urine pregnancy test prior to dosing on Day 1.
✓. If sexually active, females of childbearing potential must consistently use highly effective methods of contraception consistently throughout the study and for at least 120 days after the last dose of study drug. If sexually active, male participants must use barrier methods of contraception until 84 days after the last dose of study drug. Male participants are eligible to participate if they agree to the following requirements during the study intervention period and for 84 days after the last dose of study intervention:
✓. 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.

What they're measuring

Number of Participants With Treatment Emergent Adverse Events (TEAEs)

Timeframe: From first dose of study drug up to 56 days after last dose of study drug (approximately up to 736 days)

Change From Baseline in Hematocrit at Week 12

Timeframe: Baseline, Week 12

Change From Baseline in Hematocrit at Week 48

Timeframe: Baseline, Week 48

Change From Baseline in Leukocytes at Week 12

Timeframe: Baseline, Week 12

Change From Baseline in Leukocytes Week 48

Timeframe: Baseline, Week 48

Change From Baseline in Supine Blood Pressure (SBP) at Week 12

Timeframe: Baseline, Week 12

Change From Baseline in SBP at Week 48

Timeframe: Baseline, Week 48

Change From Baseline in Diastolic Blood Pressure (DBP) at Week 12

Timeframe: Baseline, Week 12

Trial details

NCT IDNCT05632354

SponsorPfizer

Sponsor typeINDUSTRY

Study typeINTERVENTIONAL

Primary completion2025-02-13

Results submitted2026-02-10

View on ClinicalTrials.gov More Sickle Cell Disease trials

Who can participate

Age range6 Months

SexALL

See this in plain English?

AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

Inclusion criteria

✓. Male or female aged 6 months or older with SCD who participated and received study drug or placebo in a previous osivelotor clinical study and completed the end of treatment visit.
✓. Females of childbearing potential are required to have a negative urine pregnancy test prior to dosing on Day 1.
✓. If sexually active, females of childbearing potential must consistently use highly effective methods of contraception consistently throughout the study and for at least 120 days after the last dose of study drug. If sexually active, male participants must use barrier methods of contraception until 84 days after the last dose of study drug. Male participants are eligible to participate if they agree to the following requirements during the study intervention period and for 84 days after the last dose of study intervention:
✓. 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.

What they're measuring

Number of Participants With Treatment Emergent Adverse Events (TEAEs)

Timeframe: From first dose of study drug up to 56 days after last dose of study drug (approximately up to 736 days)

Change From Baseline in Hematocrit at Week 12

Timeframe: Baseline, Week 12

Change From Baseline in Hematocrit at Week 48

Timeframe: Baseline, Week 48

Change From Baseline in Leukocytes at Week 12

Timeframe: Baseline, Week 12

Change From Baseline in Leukocytes Week 48

Timeframe: Baseline, Week 48

Change From Baseline in Supine Blood Pressure (SBP) at Week 12

Timeframe: Baseline, Week 12

Change From Baseline in SBP at Week 48

Timeframe: Baseline, Week 48

Change From Baseline in Diastolic Blood Pressure (DBP) at Week 12

Timeframe: Baseline, Week 12