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.
Study Type
INTERVENTIONAL
Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
DOUBLE
Enrollment
88
Synthetic small molecule supplied as 500 mg tablets, administered Orally
Placebo
Percentage of Participants Who Achieved Resolution of the Target Ulcers by Week 12 in Randomized Treatment Period
An ulcer qualified as a target ulcer, if met the following criteria: a) duration of ulcer was greater than equal to (\>=) 2 weeks and less than (\<) 6 months at screening; b) healed by \<25 percent (%) during the SOC run-in phase prior to randomization; c) size was greater than (\>2) square centimeters (cm\^2) at any visit, prior to randomization. Resolution of the target ulcer was defined as skin re-epithelialization confirmed at 2 consecutive study visits, 2 weeks apart during the 12-week randomized treatment period. For participants with more than one target ulcer, all target ulcers must be confirmed resolved in order for the participant to be a responder.
Time frame: Through Week 12 of randomized treatment period
Time to Resolution of Target Ulcers up to Week 12 in Randomized Treatment Period
An ulcer qualified as a target ulcer, if met the following criteria: a) duration of ulcer was \>=2 weeks and \<6 months at screening; b) healed by \<25% during the SOC run-in phase prior to randomization; c) size was \>2 cm\^2 at any visit, prior to randomization. Resolution of the target ulcer was defined as skin re-epithelialization confirmed at 2 consecutive study visits, 2 weeks apart during the 12-week randomized treatment period. Time to resolution of target ulcers was defined as the first time of the observed ulcer was resolved. For participants with more than one target ulcer, time to resolution of target ulcers was defined as time to the last target ulcer confirmed resolved. For participants who did not have the last target ulcer confirmed resolved by Week 12 or who were discontinued from study, the time to resolution was censored. Median time to event was evaluated using Kaplan-Meier method.
Time frame: From Day 1 to censoring date before Week 12 or maximum up to Week 12 of randomized treatment period, whichever occurred first
Change From Baseline in Total Surface Area of Target Ulcers at Week 12 in Randomized Treatment Period
An ulcer qualified as a target ulcer, if met the following criteria: a) duration of ulcer was \>=2 weeks and \<6 months at screening; b) healed by \<25% during the SOC run-in phase prior to randomization; c) size was \>2 cm\^2 at any visit, prior to randomization.
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Complexo Hospitalar Universitário Professor Edgard Santos- Universidade Federal Da Bahia
Salvador, Estado de Bahia, Brazil
Universidade Federal Da Bahia Hospital Universitário Professor Edgard Santos
Salvador, Estado de Bahia, Brazil
Hospital São Rafael
Salvador, Estado de Bahia, Brazil
Hospital das Clinicas da Universidade Federal de Minas Gerais
Belo Horizonte, Minas Gerais, Brazil
Multihemo Servicos Medicos S/A
Recife, Pernambuco, Brazil
Hospital das Clínicas da Faculdade de Medicina da Universidade de Sao Paulo
Cerqueira Cesar - Sao Paulo, São Paulo, Brazil
Hospital das Clinicas da Faculdade de Medicina de Ribeirão Preto da Universidade de Sao Paulo
Ribeirão Preto, São Paulo, Brazil
Esho Empresa De Servicos Hospitalares S.A/ Hospital Samaritano Higienopolis
São Paulo, Brazil
Hospital das Clínicas da Faculdade de Medicina da Universidade de Sao Paulo
São Paulo, Brazil
Hospital das Clínicas da Faculdade de Medicina da Universidade de Sao Paulo
São Paulo, Brazil
...and 13 more locations
Time frame: Baseline, Week 12 of randomized treatment period
Percentage of Participants With New Ulcers by Week 12 in Randomized Treatment Period
New ulcers were defined as ulcer(s) that were not captured at visit 1/screening. In this outcome measure, participants with occurrence of new ulcers during 12 weeks of treatment were considered.
Time frame: Through Week 12 of randomized treatment period