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Hemoglobin S Polymerization Inhibitor

A Study to Evaluate GBT021601 in Single and Multiple Doses in Healthy Participants

Phase 1
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
Be between 18 and 65 years old
Timeline
Screening 3 weeks
Treatment Varies
Follow Up 119 days from screening part a, 134 days from screening part b

Summary

This trial tests a new drug, GBT021601, in healthy people to check its safety and behavior in the body. The drug works by stopping a protein in red blood cells from clumping together.

Eligible Conditions
  • Sickle Cell Disease

Timeline

Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~119 days from screening part a, 134 days from screening part b
This trial's timeline: 3 weeks for screening, Varies for treatment, and 119 days from screening part a, 134 days from screening part b for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.
Primary study objectives
Plasma concentration
Safety, as assessed by changes in Blood pressure
Safety, as assessed by changes in Heart Rate.
+3 more
Secondary study objectives
Determine plasma concentration of GBT021601.
Determine whole blood concentration of GBT021601
Safety, as assessed by changes in QTcF

Side effects data

From 2022 Phase 1 trial • 6 Patients • NCT04983264
17%
Sickle cell anaemia with crisis
100%
80%
60%
40%
20%
0%
Study treatment Arm
Part A: Single Dose Period (SCD Related)
Part B: Multiple Ascending- Dose Period (SCD Related)
Part C: Extended Treatment Period (SCD Related)
Part A: Single Dose Period (Non-SCD Related)
Part B: Multiple Ascending- Dose Period (Non-SCD Related)
Part C: Extended Treatment Period (Non-SCD Related)

Trial Design

2Treatment groups
Experimental Treatment
Placebo Group
Group I: GBT021601Experimental Treatment1 Intervention
GBT021601 as a tablet or capsule with dose based off of preceding cohort's data.
Group II: PlaceboPlacebo Group1 Intervention
Placebo as a tablet or capsule with dose based off of preceding cohort's data.
Treatment
First Studied
Drug Approval Stage
How many patients have taken this drug
GBT021601
2021
Completed Phase 1
~150

Find a Location

Logistics

Participation is compensated

You will be compensated for participating in this trial.

Who is running the clinical trial?

Global Blood TherapeuticsLead Sponsor
35 Previous Clinical Trials
3,087 Total Patients Enrolled
PfizerLead Sponsor
4,669 Previous Clinical Trials
17,861,795 Total Patients Enrolled
Eleanor Lisbon, MD, MPHStudy DirectorGBT
5 Previous Clinical Trials
541 Total Patients Enrolled
Pfizer CT.gov Call CenterStudy DirectorPfizer
3,553 Previous Clinical Trials
14,902,797 Total Patients Enrolled
~26 spots leftby Dec 2025