Resolution of Sickle Cell Leg Ulcers With Voxelotor

Sickle Cell Disease Clinical Trial

— RESOLVE  

Official 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

Clinical Trial Details — Status: Active, not recruiting

Administrative data

• NCT number: NCT05561140
• Other study ID #: GBT440-042
• Secondary ID: C5341026
• Status: Active, not recruiting
• Phase: Phase 3
• Start date: May 30, 2022
• Est. completion date: August 20, 2024

Study information

• Verified date: April 2024
• Source: Pfizer
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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.

Recruitment information / eligibility

• 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

Related Conditions & MeSH terms

• Anemia, Sickle Cell
• Leg Ulcer
• Leg Ulcers
• Sickle Cell Disease
• Ulcer

Intervention

Drug:
• Voxelotor Oral Tablet
Synthetic small molecule supplied as 500 mg tablets, administered Orally

Other:
• Placebo
Placebo

Locations

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

Sponsors (1)

Lead Sponsor	Collaborator
Pfizer

Countries where clinical trial is conducted

Brazil,  Kenya,  Nigeria, 

Outcome

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

External Links

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