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GBT021601-021: A Study in Adult and Pediatric Participants With SCD

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ClinicalTrials.gov Identifier: NCT05431088
Recruitment Status : Recruiting
First Posted : June 24, 2022
Last Update Posted : November 10, 2022
Sponsor:
Information provided by (Responsible Party):
Global Blood Therapeutics

Study Description
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Brief Summary:
The purpose of this study is to evaluate the safety, tolerability, efficacy, pharmacokinetics and pharmacodynamics of GBT021601.

Condition or disease Intervention/treatment Phase
Sickle Cell Disease Drug: GBT021601 Phase 2 Phase 3

Detailed Description:
This is a three-part, multicenter, Phase 2/3 study of orally administered GBT021601 in participants with sickle cell disease (SCD). Part A will evaluate the safety, tolerability, and efficacy of GBT021601 in adult participants with SCD to determine an optimal dose. Part B will evaluate the efficacy of GBT021601 versus placebo in adult and pediatric participants with SCD for 48 weeks. Part C will evaluate the pharmacokinetics (PK) and safety of single and multiple doses (MD) of open-label single arm GBT021601 administered to pediatric participants.
Study Design
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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 480 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Masking Description: Part B only
Primary Purpose: Treatment
Official Title: A Phase 2/3 Randomized, Multicenter Study of GBT021601 Administered Orally to Participants With Sickle Cell Disease and an Open-Label Pharmacokinetics Study in Pediatric Participants With Sickle Cell Disease
Actual Study Start Date : October 10, 2022
Estimated Primary Completion Date : October 2026
Estimated Study Completion Date : April 2027

Resource links provided by the National Library of Medicine


Arms and Interventions
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Arm Intervention/treatment
Active Comparator: Part A

Initially, participants will be randomized 1:1 to 100 mg and 150 mg daily. Upon review of the 150 mg safety data from at least 6 participants, there will be 1:1:1 randomization: 100 mg, 150 mg, and up to 200 mg.

Participants will then receive maintenance once daily doses through Week 12.

 Drug: GBT021601
Tablets which contain GBT021601 drug substance
Placebo Comparator: Part B
Following the selection of the optimal safe and effective dose from Part A of the study, Part B of the study will assess the efficacy and safety of 48 weeks of the optimal dose, compared to placebo
 Drug: GBT021601
Tablets which contain GBT021601 drug substance
Experimental: Part C
100 mg dose in cohort C1, dose level for cohorts C2 to C4 to be determined based on emerging data
 Drug: GBT021601
Tablets which contain GBT021601 drug substance


Outcome Measures
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Primary Outcome Measures :
  1. Part A [ Time Frame: Through week 12 ]
    Number of adult participants with change from baseline in hemoglobin (Hb) through week 12 as measured by change in GBT021601 concentrations from baseline or percentage change from baseline of clinical measures of anemia Hb and hemolysis (including indirect bilirubin, reticulocytes and lactate dehydrogenase).

  2. Part B [ Time Frame: Through week 48 ]
    Proportion of participants with an increase from baseline of >1 g/dL in Hb at week 48 as measured by change in GBT021601 concentrations from baseline or percentage change from baseline of clinical measures of anemia (hemoglobin) and hemolysis (including indirect bilirubin, reticulocytes and lactate dehydrogenase).

  3. Part C [ Time Frame: Through Week 6 ]
    Assess the pharmacokinetics (PK), while observing maximum concentration (Cmax) after a single dose as measured by a noncompartmental PK analysis or population PK analysis using nonlinear mixed-effect modeling. To characterize GBT021601 PK in plasma and whole blood following a single dose.

  4. Part C [ Time Frame: Through Week 2 ]
    Assess the PK, while observing minimum concentration (Cmin) and Cmax after multiple dose administration as measured by a noncompartmental PK analysis or population PK analysis using nonlinear mixed-effect modeling will be performed to characterize GBT021601 PK in plasma and whole blood following multiple doses.


Eligibility Criteria
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Ages Eligible for Study:   6 Months to 65 Years   (Child, Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

Part A, Part B, and Part C:

  • Male or female with SCD
  • Participants with stable Hb value as judged by the Investigator
  • For participants taking hydroxyurea and/or L-glutamine, the dose must be stable for at least 90 days prior to signing the ICF or assent and with no anticipated need for dose adjustments during the study in the opinion of the Investigator.

Part B:

  • Participants with SCD ages 12 to 65 years, inclusive
  • Participants with more than or equal to 2 and ≤ 10 VOCs within 12 months of Screening.

Exclusion Criteria:

Part A, Part B, and Part C:

  • Participants who had more than 10 VOC within 12 months of screening
  • Female participant who is breastfeeding or pregnant
  • Participants who receive RBC transfusion therapy regularly or received an RBC transfusion ---for any reason within 90 days of Day 1
  • Participants hospitalized for sickle cell crisis or other vaso-occlusive event within 14 days of signing the ICF
Contacts and Locations
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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): NCT05431088


Contacts
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Contact: Eleanor Lisbon, MD, MPH +1-650-781-1765 elisbon@gbt.com
Contact: Jessica Guider +1-630-364-2275 jguider@gbt.com

Locations
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United States, Arkansas
Lynn Institute of the Ozarks Recruiting
Little Rock, Arkansas, United States, 72204
Contact: Derek Lewis, MD       dlewismd.lhsi@gmail.com   
United States, California
Lundquist Institute for Biomedical Innovation at Harbor UCLA Medical Center Recruiting
Torrance, California, United States, 90502
Contact: Moran Gotesman, MD       moran.gotesman@lundquist.org   
United States, Louisiana
University Medical Center, New Orleans Recruiting
New Orleans, Louisiana, United States, 70112
Contact: Jyotsna Fuloria, MD       jyotsna.fuloria@lcmchealth.org   
Sponsors and Collaborators
Global Blood Therapeutics
Investigators
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Study Director: Eleanor Lisbon, MD, MPH Global Blood Therapeutics
More Information
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Responsible Party: Global Blood Therapeutics
ClinicalTrials.gov Identifier: NCT05431088    
Other Study ID Numbers: GBT021601-021
First Posted: June 24, 2022    Key Record Dates
Last Update Posted: November 10, 2022
Last Verified: August 2022
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