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

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ClinicalTrials.gov Identifier: NCT05036512
Recruitment Status : Recruiting
First Posted : September 5, 2021
Last Update Posted : September 5, 2021
Sponsor:
Information provided by (Responsible Party):
Global Blood Therapeutics

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.

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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 124 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
Estimated Primary Completion Date : January 2022
Estimated Study Completion Date : April 2022

Resource links provided by the National Library of Medicine


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




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



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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.


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


Contacts
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Contact: Jessica Guider, MS, MPH, MBA 630-364-3275 jguider@gbt.com
Contact: Eleanor Lisbon, MD, MPH 650-781-1765 elisbon@gbt.com

Locations
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United States, Texas
ICON Early Phase Services, LLC Recruiting
San Antonio, Texas, United States, 78209
Contact: Cassandra Key, MD    210-283-4120    Cassandra.Key@iconplc.com   
Australia, Western Australia
Linear Clinical Research Recruiting
Nedlands, Western Australia, Australia, 6009
Contact: Andrew Redfern, MD    +61 (0) 433 078 719    aredfern@linear.org.au   
Sponsors and Collaborators
Global Blood Therapeutics
Investigators
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Study Director: Eleanor Lisbon, MD, MPH GBT
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Responsible Party: Global Blood Therapeutics
ClinicalTrials.gov Identifier: NCT05036512    
Other Study ID Numbers: GBT021601-011
First Posted: September 5, 2021    Key Record Dates
Last Update Posted: September 5, 2021
Last Verified: August 2021
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

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