Resolution of Sickle Cell Leg Ulcers With Voxelotor

NCT ID: NCT05561140

Last Updated: 2025-07-09

Basic Information

• Recruitment Status: TERMINATED
• Clinical Phase: PHASE3
• Total Enrollment: 88 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2022-05-30
• Study Completion Date: 2024-10-22

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.

Conditions

Sickle Cell Disease; Leg Ulcers

Study Design

• Allocation Method: RANDOMIZED
• Intervention Model: PARALLEL
• Primary Study Purpose: TREATMENT
• Blinding Strategy: DOUBLE

Study Groups

Voxeletor + SOC (Standard of Care)

Group Type: EXPERIMENTAL

Voxelotor Oral Tablet

Intervention Type: DRUG

Synthetic small molecule supplied as 500 mg tablets, administered Orally

Placebo + SOC (Standard of Care)

Group Type: PLACEBO_COMPARATOR

Placebo

Intervention Type: OTHER

Placebo

Interventions

Voxelotor Oral Tablet

Synthetic small molecule supplied as 500 mg tablets, administered Orally

Intervention Type: DRUG

Placebo

Placebo

Intervention Type: OTHER

Eligibility Criteria

Inclusion Criteria

1. Male or female participants with documented diagnosis of SCD (HbSS, HbS/β0 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

• Minimum Eligible Age: 12 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Pfizer

INDUSTRY

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Principal Investigators

Pfizer CT.gov Call Center

Role: STUDY_DIRECTOR

Pfizer

Locations

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

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, , Kenya

SYNLAB

Gwagwalada, Abuja, Nigeria

University of Calabar Teaching Hospital

Calabar, Cross River State, Nigeria

SYNLAB

Abuja, Federal Capital Territory, Nigeria

University of Abuja Teaching Hospital

Gwagwalada, Federal Capital Territory, Nigeria

Barau Dikko Teaching Hospital/Kaduna State University

Kaduna, , Nigeria

Aminu kano Teaching Hospital

Kano, , Nigeria

Lagos University Teaching Hospital

Lagos, , Nigeria

Countries

Brazil; Kenya; Nigeria

Provided Documents

Document Type: Study Protocol

View Document

Document Type: Statistical Analysis Plan

View Document

Related Links

https://pmiform.com/clinical-trial-info-request?StudyID=GBT440-042

To obtain contact information for a study center near you, click here.

Other Identifiers

C5341026

Identifier Type: OTHER

Identifier Source: secondary_id

GBT440-042

Identifier Type: -

Identifier Source: org_study_id