Genetic Diseases Paediatrics
Conditions
Interventions
Placebo
Sponsors
Global Blood Therapeutics Inc
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: Participants who meet all the following criteria will be eligible for enrollment in this study: 1. Male or female participants with documented diagnosis of SCD one sickle gene with 2 sickle cell genes [HbSS], sickle hemoglobin and one beta thalassemia gene [HbS/ß0] thalassemia 2. Age 12 years and older 3. At least 1 cutaneous ulcer on the lower extremity (leg, ankle, or dorsum of foot) that meets the following criteria: • Duration: = 2 weeks and 2 square centimeters (cm2) prior to randomization • Note: Participants with multiple ulcers are eligible for the study. For each participant, target ulcer(s) for evaluation in this study will be identified and tracked. To qualify as a target ulcer, the ulcer must meet the criteria above. 4. Participants, who if female and of child-bearing potential, agree to use highly effective methods of contraception from study start to 30 days after the last dose of study drug and who if male, agree to use barrier methods of contraception from study start to 30 days after the last dose of study drug. 5. Females of child-bearing potential are required to have a negative pregnancy test before the administration of study drug. 6. Written informed consent (= 18 years) or parental/guardian consent and participant assent (= 12–17 years) per International Ethics Committee (IEC) policy and requirements, consistent with International Council for Harmonisation (ICH) guidelines
Exclusion criteria
Exclusion criteria: Participants meeting any of the following exclusion criteria will not be eligible for study enrollment: 1. Target ulcer(s) healed by = 25% during the SOC Run-in prior to randomization Note: For participants with multiple ulcers, the participant is not eligible for the study if all target ulcers have healed by = 25% during the SOC Run-in. 2. Active infection/purulence at ulcer site, or exposed tendon or bone at the ulcer site, based on Investigator’s clinical judgment 3. Current osteomyelitis at or near the ulcer site 4. Known vascular abnormalities that would preclude healing in the opinion of the Investigator (eg, pre-existing severe arterial insufficiency in the affected limb) 5. Serum albumin 4 × upper limit of normal (ULN) 12. Severe renal dysfunction (estimated glomerular filtration rate [eGFR] < 30 mL/min/1.73 m2 by Schwartz formula) or is on chronic dialysis 13. Clinically significant bacterial, fungal, parasitic, or viral infection that requires therapy: • Participants with acute bacterial infection requiring systemic antibiotic use should delay Screening/enrollment until the course of antibiotic therapy has been completed • Participants with known active hepatitis A, B, or C or who are known to be human immunodeficiency virus (HIV) positive • Participants with active SARS-CoV-2 infection (COVID-19) 14. Females who are breast-feeding or pregnant 15. History of hematopoietic stem cell transplant or gene therapy 16. Participated in another clinical trial of an investigational product (or medical device) within 30 days or 5 half-live
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| The primary objective of this study is to assess the effect of voxelotor and SOC compared to placebo and SOC on leg ulcer healing in participants = 12 years of age with SCD, as measured by the proportion of participants achieving resolution of target ulcer(s) in each treatment group by Week 12 | — |
Secondary
| Measure | Time frame |
|---|---|
| The secondary objectives of this study are to evaluate the effect of voxelotor and SOC compared to placebo and SOC on: • Time to resolution of target ulcer(s) • Change in total surface area of target ulcer(s) • Incidence of new ulcers | — |
Countries
Kenya, Nigeria
Contacts
Public ContactTheresa Thuener
Clinical Operations Study Oversight
Outcome results
None listed