GBT021601-021: A Study in Adult and Pediatric Participants With SCD
Sickle Cell Disease
About this trial
This is an interventional treatment trial for Sickle Cell Disease
Eligibility 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
Sites / Locations
- Lynn Institute of the Ozarks
- Lundquist Institute for Biomedical Innovation at Harbor- UCLA Medical Center
- Edward Jenner Research Group LLC.Recruiting
- Kidz Hematology / Oncology a division of Kidz Medical
- St. Mary's Medical Center
- Alpha Clinical Research GeorgiaRecruiting
- University of Illinois Hospital and Health Sciences System - Hospital Pharmacy Services
- University of Illinois Hospital and Health Sciences System
- University of Illinois at Chicago Clinical Research Center
- LSU Health Baton Rouge-North ClinicRecruiting
- Our lady of the Lake Hospital
- Our Lady of the Lake Hopistal, Inc
- Our Lady of the Lake Hopistal, IncRecruiting
- Our Lady of the Lake Hospital, Inc In-Patient PharmacyRecruiting
- Our Lady of the Lake Physicians Group, Medical OncologyRecruiting
- University Medical Center New OrleansRecruiting
- Mississippi Center for Advanced MedicineRecruiting
- UT Physicians Comprehensive Sickle Cell ClinicRecruiting
- McGovern Medical School/Health Science Center HoustonRecruiting
- Memorial Hermann - TMC Investigational Drugs, IDS PharmacyRecruiting
- Memorial Hermann Hospital, Texas Medical Center - Clinical Research Unit (CRU)Recruiting
- UT Physicians Comprehensive Sickle Cell ClinicRecruiting
- Inova Schar Cancer Institute Infusion PharmacyRecruiting
- Inova Schar Cancer InstituteRecruiting
- Virginia Commonwealth University- Clinical Research Services Unit
- Virginia Commonwealth University
- Gertrude's Children's Hospital
- Nini Hospital s.a:l
- University College Hospital IbadanRecruiting
- Aminu Kano Teaching HospitalRecruiting
- Lagos University Teaching HospitalRecruiting
Arms of the Study
Arm 1
Arm 2
Arm 3
Active Comparator
Placebo Comparator
Experimental
Part A
Part B
Part C
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.
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
100 mg dose in cohort C1, dose level for cohorts C2 to C4 to be determined based on emerging data