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Unknown

GBT021601 for Sickle Cell Disease

Phase 2 & 3

Recruiting

Research Sponsored by Pfizer

Eligibility Criteria Checklist

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

Must have

Participants with SCD ages 12 to 65 years, inclusive

Male or female with SCD

Timeline

Screening 3 weeks

Treatment Varies

Follow Up through week 48

Awards & highlights

Study Summary

This trial is testing a new drug, GBT021601, to see if it is safe and effective. The study will also look at how the body processes the drug and how it affects the body.

Who is the study for?

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.Check my eligibility

What is being tested?

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.See study design

What are the potential side effects?

While specific side effects of GBT021601 are not listed, common ones may include reactions at injection sites, potential liver issues, headaches, nausea, fatigue, and possible changes in blood counts.

Eligibility Criteria

Inclusion Criteria

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

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

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I have sickle cell disease.

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I've had 2 to 10 severe pain crises in the last year.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ through week 48

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~through week 48

This trial's timeline: 3 weeks for screening, Varies for treatment, and through week 48 for reporting.

Treatment Details

Study Objectives

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

Primary outcome measures

Part A

Part B

Part C

Trial Design

3Treatment groups

Experimental Treatment

Active Control

Placebo Group

Group I: Part CExperimental Treatment1 Intervention

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

Group II: Part AActive Control1 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 BPlacebo Group1 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

0 / 3Eligibility criteria met

Find a Location

Who is running the clinical trial?

PfizerLead Sponsor

4,570 Previous Clinical Trials

10,915,469 Total Patients Enrolled

Global Blood TherapeuticsLead Sponsor

35 Previous Clinical Trials

4,360 Total Patients Enrolled

Pfizer CT.gov Call CenterStudy DirectorPfizer

3,476 Previous Clinical Trials

8,091,664 Total Patients Enrolled

Media Library

GBT021601 (Unknown) Clinical Trial Eligibility Overview. Trial Name: NCT05431088 — Phase 2 & 3

Sickle Cell Disease Research Study Groups: Part B, Part C, Part A

Sickle Cell Disease Clinical Trial 2023: GBT021601 Highlights & Side Effects. Trial Name: NCT05431088 — Phase 2 & 3

GBT021601 (Unknown) 2023 Treatment Timeline for Medical Study. Trial Name: NCT05431088 — Phase 2 & 3

Frequently Asked Questions

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

Does this trial have an age cap and, if so, what is it?

"The age requirements for this study are that participants must be under 65 years old and have surpassed 6 months of age."

Answered by AI

Could you inform me as to how many people are enrolled in this clinical trial?

"480 volunteers are necessary to carry out this clinical trial. These participants must meet the pre-specified inclusion criteria and can come from various locations, such as Lundquist Institute for Biomedical Innovation at Harbor UCLA Medical Center in Torrance, California and Lynn Institute of the Ozarks in Little Rock, Arkansas."

Answered by AI

Are there any open positions in this clinical trial for new participants?

"The clinical trial is presently looking for participants, according to the information on clinicaltrials.gov. The study was first posted on 10/10/2022 and was last edited on 11/7/2022."

Answered by AI

Do I fit the bill to join this clinical trial?

"To participate in this research, participants must have anemia and sickle cell. Furthermore, they can only be within the specified age range of 6 months to 65 years old. In total, 480 people are needed for this clinical trial."

Answered by AI

Recent research and studies

clinicaltrials.govStudy

A Phase 2/3 Study in Adult and Pediatric Participants With SCD

2022. "A Phase 2/3 Study in Adult and Pediatric Participants With SCD". ClinicalTrials.gov. https://clinicaltrials.gov/ct2/show/NCT05431088.