RESOLVE: Resolution of Sickle Cell Leg Ulcers With Voxelotor
Study Details
Study Description
Brief Summary
This study is a Phase 3, multicenter, randomized, placebo-controlled study to evaluate the efficacy of voxelotor and standard of care for the treatment of leg ulcers in participants with sickle cell disease. The study is divided into a 5 study periods: Screening, Run-in, Randomized Treatment, Open-label Treatment, and Follow-up/End of Study (EOS).
The study will be conducted in approximately 80 eligible participants at approximately 20 global clinical trial sites.
Condition or Disease | Intervention/Treatment | Phase |
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Phase 3 |
Study Design
Arms and Interventions
Arm | Intervention/Treatment |
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Experimental: Voxeletor + SOC (Standard of Care)
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Drug: Voxelotor Oral Tablet
Synthetic small molecule supplied as 500 mg tablets, administered Orally
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Placebo Comparator: Placebo + SOC (Standard of Care)
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Other: Placebo
Placebo
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Outcome Measures
Primary Outcome Measures
- The effect of voxelotor and standard of care compared to placebo and SOC on leg ulcer healing [From pre-dose to Week 12]
To assess the effect of voxelotor and standard of care compared to placebo and SOC on leg ulcer healing in participants ≥ 12 years of age with SCD, as measured by the proportion of participants with complete resolution of target ulcer(s) in each treatment group by Week 12
Secondary Outcome Measures
- Time to resolution of target ulcer(s) [From pre-dose to Week 12]
- Change in total surface area(s) of target ulcer(s) [From pre-dose to Week 12]
- Incidence of new ulcers [From pre-dose to Week 12]
Eligibility Criteria
Criteria
Inclusion Criteria:
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Male or female participants with documented diagnosis of SCD (HbSS, HbS/β0 thalassemia)
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Age 12 years and older
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At least 1 cutaneous ulcer(s) on the lower extremity (leg, ankle, or dorsum of foot) that meets the following criteria:
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Duration: ≥ 2 weeks and < 6 months at Screening, and
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Size: > 2 cm2 prior to randomization
- Written informed consent (≥ 18 years) or parental/guardian consent and participant assent (≥ 12-17 years) per IEC policy and requirements, consistent with ICH guidelines
Exclusion Criteria:
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Target ulcer(s) healed by ≥ 25% during the standard of care run-in period prior to randomization
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Active infection/purulence at ulcer site, or exposed tendon or bone at the ulcer site, based on Investigator's clinical judgment
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Current osteomyelitis at or near the ulcer site
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Known vascular abnormalities that would preclude healing in the opinion of the Investigator (eg, pre-existing severe arterial insufficiency in the affected limb)
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Serum albumin < 2.0 g/dL
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RBC transfusion within 60 days of initiation of study drug
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Receiving regularly scheduled RBC transfusion therapy (also termed chronic, prophylactic, or preventive transfusion) during the study
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Planned elective surgery within the next 6 months
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Anemia due to bone marrow failure (eg, myelodysplasia)
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Absolute reticulocyte count < 100 × 109/L
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Screening alanine aminotransferase (ALT) > 4 × upper limit of normal (ULN)
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Severe renal dysfunction (estimated glomerular filtration rate [eGFR] < 30 mL/min/1.73 m2 by Schwartz formula) or is on chronic dialysis
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Clinically significant bacterial, fungal, parasitic, or viral infection that requires therapy
Other protocol-defined Eligibility Criteria that apply
Contacts and Locations
Locations
Site | City | State | Country | Postal Code | |
---|---|---|---|---|---|
1 | Kenya Medical Research Institute - Kondele | Kisumu | Western | Kenya | 40100 |
2 | KEMRI/CRDR Siaya Clinical Research Annex | Siaya | Western | Kenya | 00144 |
3 | KEMRI CCR - Butere | Butere | Kenya | 50101 | |
4 | Gertrude's Children's Hospital | Nairobi | Kenya | 00100 | |
5 | Strathmore University | Nairobi | Kenya | 00200 | |
6 | KEMRI CRDR Clinical Research Clinic Nairobi | Nairobi | Kenya | ||
7 | University of Abuja Teaching Hospital | Abuja | Nigeria | 900105 | |
8 | University of Calabar Teaching Hospital | Calabar | Nigeria | 540242 | |
9 | University of Nigeria Teaching Hospital | Enugu | Nigeria | 402109 | |
10 | Barau Dikko Teaching Hospital/Kaduna State University | Kaduna | Nigeria | 9727 | |
11 | Aminu Kano Teaching Hospital | Kano | Nigeria | 3452 | |
12 | Lagos University Teaching Hospital Haematology | Suru Lere | Nigeria | 102215 |
Sponsors and Collaborators
- Global Blood Therapeutics
Investigators
None specified.Study Documents (Full-Text)
None provided.More Information
Publications
None provided.- GBT440-042