Sickle Cell Disease MedDRA version: 21.0 Level: PT Classification code 10040644 Term: Sickle cell disease System Organ Class: 10010331 - Congenital, familial and genetic disorders
Conditions
Interventions
Product Name: voxelotor 500 mg tablets
Product Code: GBT440
Pharmaceutical Form: Tablet
INN or Proposed INN: voxelotor
CAS Number: 1446321-46-5
Current Sponsor code: GBT440
Concentration unit: mg mill
Sponsors
Global Blood Therapeutics, Inc.
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: 1. Age = 2 to =65 years) no F.1.3.1 Number of subjects for this age range 0
Exclusion criteria
Exclusion criteria: 1. Body weight 4× upper limit of normal (ULN) for age 12. History of unstable or deteriorating cardiac or pulmonary disease within 6 months prior to consent including severe or unstable pulmonary hypertension 13. Clinically significant bacterial, fungal, parasitic, or viral infection currently requiring or will require therapy. a. Participants with acute bacterial infection requiring antibiotic use should delay screening until the course of antibiotic therapy has been completed and the infection has resolved, in the opinion of the investigator. 14. Known active hepatitis A, B, or C or human immunodeficiency virus (HIV)-positive. 15. Positive test indicative of active malaria infection at screening (Malaria test to be performed only if there is suspicion of active malaria infection). 16. Any condition affecting drug absorption, such as major surgery involving the stomach or small intestine (prior cholecystectomy is acceptable) 17. History of malignancy within the past 2 years prior to treatment Day 1 requiring chemotherapy and/or radiation (with the exception of local therapy for non-melanoma skin malignancy) 18. Received an investigational drug within 30 days or 5 half-lives, whichever is longer, of the parent/legal guardian signing the ICF 19. Parent or legal guardian/participants are unlikely to comply with the study procedures 20. Other medical, psychological, or addictive condition that, in the opinion of the Investigator, would: confound or interfere with evaluation of safety, efficacy, and/or PK of the investigational drug; prevent compliance with the study protocol; preclude informed consent; or, render the participant, parent, or caretaker unable/unlikely to comply with the study procedures 21. Use of herbal medications (eg, St. John’s wort) or sensitive CYP3A4 substrates with a narrow therapeutic index 22. Active symptomatic coronavirus disease of 2019 (COVID-19) infection 23. Known hypersensitivity to voxelotor or any other components of the study drug
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: The primary objective is to evaluate the effect of voxelotor compared to placebo on the TCD time-averaged mean of the maximum velocity (TAMMV) arterial cerebral blood flow at 24 weeks in SCD participants = 2 to < 15 years of age with conditional (170 to < 200 cm/sec) TCD flow velocity.;Secondary Objective: The secondary objectives are to evaluate the effects of voxelotor compared to placebo on: - Change in TCD flow velocity at Week 48 and Week 96 - Conversion of TCD category from conditional to abnormal - Reversion of TCD category from conditional to normal - Proportion of participants with TCD response - Change in Hb over time - Change in clinical measures of hemolysis;Primary end point(s): The primary efficacy endpoint is the change from Baseline at 24 weeks in TAMMV arterial cerebral blood flow, as measured by TCD.;Timepoint(s) of evaluation of this end point: Change from Baseline at 24 weeks in TAMMV arterial cerebral blood flow, as measured by TCD. | — |
Secondary
| Measure | Time frame |
|---|---|
| Secondary end point(s): - Change from Baseline in TCD flow velocity at Week 48 and Week 96 - Incidence of conversion to abnormal TCD flow velocity (= 200 cm/sec) over 24, 48, and 96 weeks - Incidence of reversion to normal TCD flow velocity (<170 cm/sec) over 24, 48, and 96 weeks - Proportion of participants with TCD flow velocity reduction = 15 cm/sec at Week 24, Week 48, and Week 96 - Change in Hb from Baseline at Week 24, Week 48, and Week 96 - Change in clinical measures of hemolysis (unconjugated bilirubin, % reticulocyte, absolute reticulocyte, and lactate dehydrogenase [LDH]) from Baseline at Week 24, Week 48, and Week 96;Timepoint(s) of evaluation of this end point: Measured at Weeks: 24, 48 and 96 | — |
Countries
Egypt, France, Ghana, Italy, Kenya, Nigeria, Oman, Saudi Arabia, United Kingdom, United States
Contacts
Public ContactVictor Ramiro, Senior Manager RA
Global Blood Therapeutics, Inc.
Outcome results
None listed