NCT05036512

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.

Trial Health

90
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
129

participants targeted

Target at P75+ for phase_1

Timeline
Completed

Started Dec 2020

Typical duration for phase_1

Geographic Reach
2 countries

4 active sites

Status
completed

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

Study Start

First participant enrolled

December 9, 2020

Completed
8 months until next milestone

First Submitted

Initial submission to the registry

August 13, 2021

Completed
23 days until next milestone

First Posted

Study publicly available on registry

September 5, 2021

Completed
1.2 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

November 2, 2022

Completed
3 months until next milestone

Study Completion

Last participant's last visit for all outcomes

February 7, 2023

Completed
Last Updated

July 25, 2023

Status Verified

July 1, 2023

Enrollment Period

1.9 years

First QC Date

August 13, 2021

Last Update Submit

July 22, 2023

Conditions

Sickle Cell Disease

Outcome Measures

Primary Outcomes (7)

  • 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 Outcomes (3)

  • Determine whole blood concentration of GBT021601

    119 days from screening Part A

  • Determine plasma concentration of GBT021601.

    134 days from screening Part B

  • Safety, as assessed by changes in QTcF

    119 days from screening Part A, 134 days from screening Part B

Study Arms (2)

Placebo

PLACEBO COMPARATOR

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

Drug: GBT021601

GBT021601

EXPERIMENTAL

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

Drug: GBT021601

Interventions

GBT021601DRUG

Administered orally with water as a single dose in the morning.

Also known as: Placebo
GBT021601Placebo

Eligibility Criteria

Age18 Years - 55 Years
Sexall
Healthy VolunteersYes
Age GroupsAdult (18-64)

You may qualify if:

  • 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

You may not qualify if:

  • \- Positive pregnancy test or currently breastfeeding.

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (4)

ICON Early Phase Services, LLC

San Antonio, Texas, 78209, United States

Location

Harry Perkins Institute of Medical Research

Nedlands, Western Australia, 6009, Australia

Location

Linear Clinical Research

Nedlands, Western Australia, 6009, Australia

Location

Oxford Compounding

North Perth, Western Australia, 6006, Australia

Location

Related Links

MeSH Terms

Conditions

Anemia, Sickle Cell

Condition Hierarchy (Ancestors)

Anemia, Hemolytic, CongenitalAnemia, HemolyticAnemiaHematologic DiseasesHemic and Lymphatic DiseasesHemoglobinopathiesGenetic Diseases, InbornCongenital, Hereditary, and Neonatal Diseases and Abnormalities

Study Officials

  • Pfizer CT.gov Call Center

    Pfizer

    STUDY DIRECTOR

Study Design

Study Type
interventional
Phase
phase 1
Allocation
RANDOMIZED
Masking
QUADRUPLE
Who Masked
PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
Purpose
TREATMENT
Intervention Model
PARALLEL
Sponsor Type
INDUSTRY
Responsible Party
SPONSOR

Study Record Dates

First Submitted

August 13, 2021

First Posted

September 5, 2021

Study Start

December 9, 2020

Primary Completion

November 2, 2022

Study Completion

February 7, 2023

Last Updated

July 25, 2023

Record last verified: 2023-07

Data Sharing

IPD Sharing
Will not share

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.

Locations

AU(3)US(1)