We're building a better ClinicalTrials.gov. Check it out and tell us what you think!
Try the New Site
We're building a modernized ClinicalTrials.gov! Visit Beta.ClinicalTrials.gov to try the new functionality.
Working…
ClinicalTrials.gov
ClinicalTrials.gov Menu

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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT05036512
Recruitment Status : Completed
First Posted : September 5, 2021
Last Update Posted : May 26, 2023
Sponsor:
Collaborator:
Pfizer
Information provided by (Responsible Party):
Global Blood Therapeutics

Study Description
Go to 
Top of Page Study Description Study Design Arms and Interventions Outcome Measures Eligibility Criteria Contacts and Locations More Information
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.

Condition or disease Intervention/treatment Phase
Sickle Cell Disease Drug: GBT021601 Phase 1

Detailed Description:
This is a randomized, double-blind, placebo controlled, single and multiple ascending dose study in healthy participants.
Study Design
Go to 
Top of Page Study Description Study Design Arms and Interventions Outcome Measures Eligibility Criteria Contacts and Locations More Information

Layout table for study information
Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 129 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
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.
Actual Study Start Date : December 9, 2020
Actual Primary Completion Date : November 2, 2022
Actual Study Completion Date : February 7, 2023

Resource links provided by the National Library of Medicine


Arms and Interventions
Go to 
Top of Page Study Description Study Design Arms and Interventions Outcome Measures Eligibility Criteria Contacts and Locations More Information

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 Name: 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 Name: Placebo


Outcome Measures
Go to 
Top of Page Study Description Study Design Arms and Interventions Outcome Measures Eligibility Criteria Contacts and Locations More Information

Primary Outcome Measures :
  1. Safety, as assessed by frequency and severity of adverse events (AEs) [ Time Frame: 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.

  2. Safety, as assessed by changes in Heart Rate. [ Time Frame: 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.

  3. Safety, as assessed by changes in eGFR [ Time Frame: 119 days from screening Part A, 134 days from screening Part B ]
    Number of participants with changes in eGFR from baseline

  4. Safety, as assessed by changes in alanine aminotransferase (ALT) [ Time Frame: 119 days from screening Part A, 134 days from screening Part B ]
    Number of participants with changes in alanine aminotransferase (ALT)

  5. Safety, as assessed by changes in Blood pressure [ Time Frame: 119 days from screening Part A, 134 days from screening Part B ]
    Number of participants with changes in systolic (mmHg) and diastolic (mmHg) blood

  6. Plasma concentration [ Time Frame: 119 days from screening Part A ]
    Time of Cmax

  7. Plasma concentration [ Time Frame: 134 days from screening Part B ]
    Cmax on D1-D15


Secondary Outcome Measures :
  1. Determine whole blood concentration of GBT021601 [ Time Frame: 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.

  2. Determine plasma concentration of GBT021601. [ Time Frame: 134 days from screening Part B ]
    With dosing data from each cohort determine the steady-state maximum plasma/whole blood concentration (Cmax).

  3. Safety, as assessed by changes in QTcF [ Time Frame: 119 days from screening Part A, 134 days from screening Part B ]
    Number of participants with changes in the QTcF interval from baseline


Eligibility Criteria
Go to 
Top of Page Study Description Study Design Arms and Interventions Outcome Measures Eligibility Criteria Contacts and Locations More Information

Information from the National Library of Medicine

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.


Layout table for eligibility information
Ages Eligible for Study:   18 Years to 55 Years   (Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   Yes
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.

Contacts and Locations
Go to 
Top of Page Study Description Study Design Arms and Interventions Outcome Measures Eligibility Criteria Contacts and Locations More Information

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT05036512


Locations
Layout table for location information
United States, Texas
ICON Early Phase Services, LLC
San Antonio, Texas, United States, 78209
Australia, Western Australia
Linear Clinical Research
Nedlands, Western Australia, Australia, 6009
Sponsors and Collaborators
Global Blood Therapeutics
Pfizer
Investigators
Layout table for investigator information
Study Director: Pfizer CT.gov Call Center Pfizer
More Information
Go to 
Top of Page Study Description Study Design Arms and Interventions Outcome Measures Eligibility Criteria Contacts and Locations More Information
Layout table for additonal information
Responsible Party: Global Blood Therapeutics
ClinicalTrials.gov Identifier: NCT05036512    
Other Study ID Numbers: GBT021601-011
C5351001 ( Other Identifier: Pfizer )
First Posted: September 5, 2021    Key Record Dates
Last Update Posted: May 26, 2023
Last Verified: May 2023
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

Layout table for additional information
Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Additional relevant MeSH terms:
Layout table for MeSH terms
Anemia, Sickle Cell
Anemia, Hemolytic, Congenital
Anemia, Hemolytic
Anemia
 Hematologic Diseases
Hemoglobinopathies
Genetic Diseases, Inborn