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

Primary Purpose

Sickle Cell Disease

Status
Completed
Phase
Phase 1
Locations
International
Study Type
Interventional
Intervention
GBT021601
Sponsored by
Pfizer
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Sickle Cell Disease

Eligibility Criteria

18 Years - 55 Years (Adult)All SexesAccepts Healthy Volunteers

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.

Sites / Locations

  • ICON Early Phase Services, LLC
  • Harry Perkins Institute of Medical Research
  • Linear Clinical Research
  • Oxford Compounding

Arms of the Study

Arm 1

Arm 2

Arm Type

Placebo Comparator

Experimental

Arm Label

Placebo

GBT021601

Arm Description

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

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

Outcomes

Primary Outcome Measures

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.
Safety, as assessed by changes in Heart Rate.
Number of participants with changes in heart rate (bpm) as compared to baseline.
Safety, as assessed by changes in eGFR
Number of participants with changes in eGFR from baseline
Safety, as assessed by changes in alanine aminotransferase (ALT)
Number of participants with changes in alanine aminotransferase (ALT)
Safety, as assessed by changes in Blood pressure
Number of participants with changes in systolic (mmHg) and diastolic (mmHg) blood
Plasma concentration
Time of Cmax
Plasma concentration
Cmax on D1-D15

Secondary Outcome Measures

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.
Determine plasma concentration of GBT021601.
With dosing data from each cohort determine the steady-state maximum plasma/whole blood concentration (Cmax).
Safety, as assessed by changes in QTcF
Number of participants with changes in the QTcF interval from baseline

Full Information

First Posted
August 13, 2021
Last Updated
July 22, 2023
Sponsor
Pfizer
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1. Study Identification

Unique Protocol Identification Number
NCT05036512
Brief Title
A Study to Evaluate GBT021601 in Single and Multiple Doses in Healthy Participants
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.
Study Type
Interventional

2. Study Status

Record Verification Date
July 2023
Overall Recruitment Status
Completed
Study Start Date
December 9, 2020 (Actual)
Primary Completion Date
November 2, 2022 (Actual)
Study Completion Date
February 7, 2023 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Pfizer

4. Oversight

Studies a U.S. FDA-regulated Drug Product
Yes
Studies a U.S. FDA-regulated Device Product
No
Data Monitoring Committee
No

5. Study Description

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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Sickle Cell Disease

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 1
Interventional Study Model
Parallel Assignment
Masking
ParticipantCare ProviderInvestigatorOutcomes Assessor
Allocation
Randomized
Enrollment
129 (Actual)

8. Arms, Groups, and Interventions

Arm Title
Placebo
Arm Type
Placebo Comparator
Arm Description
Placebo as a tablet or capsule with dose based off of preceding cohort's data.
Arm Title
GBT021601
Arm Type
Experimental
Arm Description
GBT021601 as a tablet or capsule with dose based off of preceding cohort's data.
Intervention Type
Drug
Intervention Name(s)
GBT021601
Other Intervention Name(s)
Placebo
Intervention Description
Administered orally with water as a single dose in the morning.
Primary Outcome Measure Information:
Title
Safety, as assessed by frequency and severity of adverse events (AEs)
Description
AEs will be coded to system organ class and preferred term using the Medical Dictionary for Regulatory Activities (MedDRA) and summarized.
Time Frame
119 days from screening Part A, 134 days from screening Part B
Title
Safety, as assessed by changes in Heart Rate.
Description
Number of participants with changes in heart rate (bpm) as compared to baseline.
Time Frame
119 days from screening Part A, 134 days from screening Part B
Title
Safety, as assessed by changes in eGFR
Description
Number of participants with changes in eGFR from baseline
Time Frame
119 days from screening Part A, 134 days from screening Part B
Title
Safety, as assessed by changes in alanine aminotransferase (ALT)
Description
Number of participants with changes in alanine aminotransferase (ALT)
Time Frame
119 days from screening Part A, 134 days from screening Part B
Title
Safety, as assessed by changes in Blood pressure
Description
Number of participants with changes in systolic (mmHg) and diastolic (mmHg) blood
Time Frame
119 days from screening Part A, 134 days from screening Part B
Title
Plasma concentration
Description
Time of Cmax
Time Frame
119 days from screening Part A
Title
Plasma concentration
Description
Cmax on D1-D15
Time Frame
134 days from screening Part B
Secondary Outcome Measure Information:
Title
Determine whole blood concentration of GBT021601
Description
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.
Time Frame
119 days from screening Part A
Title
Determine plasma concentration of GBT021601.
Description
With dosing data from each cohort determine the steady-state maximum plasma/whole blood concentration (Cmax).
Time Frame
134 days from screening Part B
Title
Safety, as assessed by changes in QTcF
Description
Number of participants with changes in the QTcF interval from baseline
Time Frame
119 days from screening Part A, 134 days from screening Part B

10. Eligibility

Sex
All
Minimum Age & Unit of Time
18 Years
Maximum Age & Unit of Time
55 Years
Accepts Healthy Volunteers
Accepts Healthy Volunteers
Eligibility Criteria
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.
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Pfizer CT.gov Call Center
Organizational Affiliation
Pfizer
Official's Role
Study Director
Facility Information:
Facility Name
ICON Early Phase Services, LLC
City
San Antonio
State/Province
Texas
ZIP/Postal Code
78209
Country
United States
Facility Name
Harry Perkins Institute of Medical Research
City
Nedlands
State/Province
Western Australia
ZIP/Postal Code
6009
Country
Australia
Facility Name
Linear Clinical Research
City
Nedlands
State/Province
Western Australia
ZIP/Postal Code
6009
Country
Australia
Facility Name
Oxford Compounding
City
North Perth
State/Province
Western Australia
ZIP/Postal Code
6006
Country
Australia

12. IPD Sharing Statement

Plan to Share IPD
No
IPD Sharing 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.
Links:
URL
https://pmiform.com/clinical-trial-info-request?StudyID=GBT021601-011
Description
To obtain contact information for a study center near you, click here.

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

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