Sickle Cell Disease Clinical Trial
— RESOLVEOfficial title:
A Phase 3, Multicenter, Randomized, Double-Blind, Placebo-Controlled Trial to Evaluate the Efficacy of Voxelotor for the Treatment of Leg Ulcers in Patients With Sickle Cell Disease
Verified date | April 2024 |
Source | Pfizer |
Contact | n/a |
Is FDA regulated | No |
Health authority | |
Study type | Interventional |
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.
Status | Active, not recruiting |
Enrollment | 87 |
Est. completion date | August 20, 2024 |
Est. primary completion date | June 26, 2024 |
Accepts healthy volunteers | No |
Gender | All |
Age group | 12 Years and older |
Eligibility | Inclusion Criteria: 1. Male or female participants with documented diagnosis of SCD (HbSS, HbS/ß0 thalassemia) 2. Age 12 years and older 3. At least 1 cutaneous ulcer(s) on the lower extremity (leg, ankle, or dorsum of foot) that meets the following criteria: - Duration: = 2 weeks and < 6 months at Screening, and - Size: > 2 cm2 prior to randomization 4. 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: 1. Target ulcer(s) healed by = 25% during the standard of care run-in period prior to randomization 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 < 2.0 g/dL 6. RBC transfusion within 60 days of initiation of study drug 7. Receiving regularly scheduled RBC transfusion therapy (also termed chronic, prophylactic, or preventive transfusion) during the study 8. Planned elective surgery within the next 6 months 9. Anemia due to bone marrow failure (eg, myelodysplasia) 10. Absolute reticulocyte count < 100 × 109/L 11. Screening alanine aminotransferase (ALT) > 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 Other protocol-defined Eligibility Criteria that apply |
Country | Name | City | State |
---|---|---|---|
Brazil | Hospital das Clinicas da Universidade Federal de Minas Gerais | Belo Horizonte | MG |
Brazil | Hospital das Clinicas da Universidade Federal de Minas Gerais | Belo Horizonte | Minas Gerais |
Brazil | Hospital das Clínicas da Faculdade de Medicina da Universidade de Sao Paulo | Cerqueira Cesar - Sao Paulo | SP |
Brazil | Multihemo Servicos Medicos S/A | Recife | Pernambuco |
Brazil | Hospital das Clinicas da Faculdade de Medicina de Ribeirão Preto da Universidade de Sao Paulo | Ribeirão Preto | SÃO Paulo |
Brazil | Complexo Hospitalar Universitário Professor Edgard Santos- Universidade Federal Da Bahia | Salvador | Bahia |
Brazil | Hospital São Rafael | Salvador | Bahia |
Brazil | Universidade Federal Da Bahia Hospital Universitário Professor Edgard Santos | Salvador | Bahia |
Brazil | Esho Empresa De Servicos Hospitalares S.A/ Hospital Samaritano Higienopolis | Sao Paulo | |
Brazil | Hospital das Clínicas da Faculdade de Medicina da Universidade de Sao Paulo | Sao Paulo | |
Brazil | Hospital das Clínicas da Faculdade de Medicina da Universidade de Sao Paulo | São Paulo | |
Kenya | KEMRI Centre for Clinical Research Butere County Hospital | Butere | Kakamega County |
Kenya | KEMRI Kondele Children's Hospital within Jaramogi Oginga Odinga Teaching and Referral Hospital. | Kisumu | |
Kenya | Gertrude's Children's Hospital | Nairobi | |
Kenya | KEMRI-Centre for Respiratory Diseases Research-Nairobi | Nairobi | |
Kenya | Strathmore University Medical Centre | Nairobi | |
Kenya | KEMRI-CRDR, KEMRI Clinical Research Annex | Siaya | |
Nigeria | University of Calabar Teaching Hospital | Calabar | Cross River |
Nigeria | SYNLAB | Gwagwalada | Abuja |
Nigeria | Barau Dikko Teaching Hospital/Kaduna State University | Kaduna | |
Nigeria | Aminu kano Teaching Hospital | Kano | |
Nigeria | Lagos University Teaching Hospital | Lagos |
Lead Sponsor | Collaborator |
---|---|
Pfizer |
Brazil, Kenya, Nigeria,
Type | Measure | Description | Time frame | Safety issue |
---|---|---|---|---|
Primary | The effect of voxelotor and standard of care compared to placebo and SOC on leg ulcer healing | 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 | From pre-dose to Week 12 | |
Secondary | Time to resolution of target ulcer(s) | From pre-dose to Week 12 | ||
Secondary | Change in total surface area(s) of target ulcer(s) | From pre-dose to Week 12 | ||
Secondary | Incidence of new ulcers | From pre-dose to Week 12 |
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