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

Sickle Cell Disease Clinical Trial

Official title:

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.

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT05036512
• Other study ID #: GBT021601-011
• Secondary ID: C5351001
• Status: Completed
• Phase: Phase 1
• Start date: December 9, 2020
• Est. completion date: February 7, 2023

Study information

• Verified date: July 2023
• Source: Pfizer
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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.

Description:

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

Recruitment information / eligibility

• Status: Completed
• Enrollment: 129
• Est. completion date: February 7, 2023
• Est. primary completion date: November 2, 2022
• Accepts healthy volunteers: Accepts Healthy Volunteers
• Gender: All
• Age group: 18 Years to 55 Years

Eligibility

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.

Related Conditions & MeSH terms

• Anemia, Sickle Cell
• Sickle Cell Disease

Intervention

Drug:
• GBT021601
Administered orally with water as a single dose in the morning.

Locations

Country	Name	City	State
Australia	Harry Perkins Institute of Medical Research	Nedlands	Western Australia
Australia	Linear Clinical Research	Nedlands	Western Australia
Australia	Oxford Compounding	North Perth	Western Australia
United States	ICON Early Phase Services, LLC	San Antonio	Texas

Sponsors (1)

Lead Sponsor	Collaborator
Pfizer

Countries where clinical trial is conducted

United States,  Australia, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	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
Primary	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
Primary	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
Primary	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
Primary	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
Primary	Plasma concentration	Time of Cmax	119 days from screening Part A
Primary	Plasma concentration	Cmax on D1-D15	134 days from screening Part B
Secondary	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
Secondary	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
Secondary	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

External Links

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