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

A Double-Blind, Randomized, Placebo-Controlled, Single and Multiple Ascending Dose (SAD/MAD) Study to Evaluate the Safety, Tolerability, PK, and Food Effect of GBT021601, a Hemoglobin S Polymerization Inhibitor, in Healthy Participants.

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.

Study Overview

• Status: Completed
• Conditions: Sickle Cell Disease
• Intervention / Treatment: Drug: GBT021601

Detailed Description

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

• Study Type: Interventional
• Enrollment (Actual): 129
• Phase: Phase 1

Contacts and Locations

Study Locations

• Australia
  • Western Australia
    • Nedlands, Western Australia, Australia, 6009
      • Linear Clinical Research
    • Nedlands, Western Australia, Australia, 6009
      • Harry Perkins Institute of Medical Research
    • North Perth, Western Australia, Australia, 6006
      • Oxford Compounding
• United States
  • Texas
    • San Antonio, Texas, United States, 78209
      • ICON Early Phase Services, LLC

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 18 years to 55 years (Adult)
• Accepts Healthy Volunteers: Yes

Description

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

Exclusion Criteria:

- Positive pregnancy test or currently breastfeeding.

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: Randomized
• Interventional Model: Parallel Assignment
• Masking: Quadruple

Number of Arms

2

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Placebo Comparator: Placebo; Placebo as a tablet or capsule with dose based off of preceding cohort's data.	Drug: GBT021601; Administered orally with water as a single dose in the morning.; Other Names: Placebo
Experimental: GBT021601; GBT021601 as a tablet or capsule with dose based off of preceding cohort's data.	Drug: GBT021601; Administered orally with water as a single dose in the morning.; Other Names: Placebo

What is the study measuring?

Primary Outcome Measures

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

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Determine whole blood concentration of GBT021601	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.	119 days from screening Part A
Determine plasma concentration of GBT021601.	With dosing data from each cohort determine the steady-state maximum plasma/whole blood concentration (Cmax).	134 days from screening Part B
Safety, as assessed by changes in QTcF	Number of participants with changes in the QTcF interval from baseline	119 days from screening Part A, 134 days from screening Part B

Collaborators and Investigators

• Sponsor: Pfizer
• Collaborators: Pfizer
• Investigators: Study Director: Pfizer CT.gov Call Center, Pfizer

Publications and helpful links

Helpful Links

• To obtain contact information for a study center near you, click here.

Study record dates

Study Major Dates

• Study Start (Actual): December 9, 2020
• Primary Completion (Actual): November 2, 2022
• Study Completion (Actual): February 7, 2023

Study Registration Dates

• First Submitted: August 13, 2021
• First Submitted That Met QC Criteria: August 31, 2021
• First Posted (Actual): September 5, 2021

Study Record Updates

• Last Update Posted (Actual): July 25, 2023
• Last Update Submitted That Met QC Criteria: July 22, 2023
• Last Verified: July 1, 2023

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Hematologic Diseases; Genetic Diseases, Inborn; Anemia; Anemia, Hemolytic, Congenital; Anemia, Hemolytic; Hemoglobinopathies; Anemia, Sickle Cell
• Other Study ID Numbers: GBT021601-011; C5351001 (Other Identifier: Pfizer)

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO
• IPD Plan Description: Pfizer will provide access to individual de-identified participant data and related study documents (e.g. protocol, Statistical Analysis Plan (SAP), Clinical Study Report (CSR)) upon request from qualified researchers, and subject to certain criteria, conditions, and exceptions. Further details on Pfizer's data sharing criteria and process for requesting access can be found at: https://www.pfizer.com/science/clinical_trials/trial_data_and_results/data_requests.

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: Yes
• Studies a U.S. FDA-regulated device product: No