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

Phase 1

Completed

• Conditions: Sickle Cell Disease
• Interventions: Drug: GBT021601

• Registration Number: NCT05036512
• Lead Sponsor: Pfizer

Brief Summary

This first in human study is designed to evaluate the safety, tolerability, pharmacokinetics (PK), and food effect of GBT021601, a hemoglobin S (HbS) polymerization inhibitor, in healthy participants.

Detailed Description

This is a randomized, double-blind, placebo controlled, single and multiple ascending dose study in healthy participants.

Basic Information
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Recruitment & Eligibility
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Study & Design
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Trial Locations

Study Timeline

• Study Start: 2020-12-09
• Study First Submitted: 2021-08-13
• Study First Submitted QC: 2021-08-31
• Study First Posted: 2021-09-05
• Primary Completion: 2022-11-02
• Study Completion: 2023-02-07
• Status Verified: 2023-07
• Last Update Submitted: 2023-07-22
• Last Update Posted: 2023-07-25

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 129

Inclusion Criteria

• Healthy males and females ≥ 18 to ≤ 55 years of age
• Body mass index ≥ 18.0 to ≤ 30.0 kg/m2
• Body weight ≥ 50 kg at screening and Day -1

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Exclusion Criteria

• Positive pregnancy test or currently breastfeeding.

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Study & Design

• Study Type: INTERVENTIONAL
• Study Design: PARALLEL

Arm && Interventions

Group	Intervention	Description
GBT021601	GBT021601	GBT021601 as a tablet or capsule with dose based off of preceding cohort's data.
Placebo	GBT021601	Placebo as a tablet or capsule with dose based off of preceding cohort's data.

Primary Outcome Measures

Name	Time	Method
Safety, as assessed by frequency and severity of adverse events (AEs)	119 days from screening Part A, 134 days from screening Part B	AEs will be coded to system organ class and preferred term using the Medical Dictionary for Regulatory Activities (MedDRA) and summarized.
Safety, as assessed by changes in Heart Rate.	119 days from screening Part A, 134 days from screening Part B	Number of participants with changes in heart rate (bpm) as compared to baseline.
Safety, as assessed by changes in eGFR	119 days from screening Part A, 134 days from screening Part B	Number of participants with changes in eGFR from baseline
Safety, as assessed by changes in alanine aminotransferase (ALT)	119 days from screening Part A, 134 days from screening Part B	Number of participants with changes in alanine aminotransferase (ALT)
Safety, as assessed by changes in Blood pressure	119 days from screening Part A, 134 days from screening Part B	Number of participants with changes in systolic (mmHg) and diastolic (mmHg) blood
Plasma concentration	134 days from screening Part B	Cmax on D1-D15

Secondary Outcome Measures

Name	Time	Method
Determine whole blood concentration of GBT021601	119 days from screening Part A	Hemoximetry will be used to assess oxygen saturation in whole blood by generating oxygen equilibrium curves (OECs) which relate the extent of Hb-O2 saturation to the partial pressure of O2 (pO2) and measure the binding affinity of O2 to Hb.
Safety, as assessed by changes in QTcF	119 days from screening Part A, 134 days from screening Part B	Number of participants with changes in the QTcF interval from baseline
Determine plasma concentration of GBT021601.	134 days from screening Part B	With dosing data from each cohort determine the steady-state maximum plasma/whole blood concentration (Cmax).

Trial Locations

Locations (4)

Harry Perkins Institute of Medical Research; 🇦🇺 Nedlands, Western Australia, Australia

Linear Clinical Research; 🇦🇺 Nedlands, Western Australia, Australia

ICON Early Phase Services, LLC; 🇺🇸 San Antonio, Texas, United States

Oxford Compounding; 🇦🇺 North Perth, Western Australia, Australia