RESOLVE: Resolution of Sickle Cell Leg Ulcers With Voxelotor

Sponsor
Global Blood Therapeutics (Industry)
Overall Status
Recruiting
CT.gov ID
NCT05561140
Collaborator
(none)
80
12
2
18.7
6.7
0.4

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
  • Drug: Voxelotor Oral Tablet
  • Other: Placebo
Phase 3

Study Design

Study Type:
Interventional
Anticipated Enrollment :
80 participants
Allocation:
Randomized
Intervention Model:
Parallel Assignment
Masking:
Double (Participant, Investigator)
Primary Purpose:
Treatment
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
Actual Study Start Date :
Jun 30, 2022
Anticipated Primary Completion Date :
Dec 19, 2023
Anticipated Study Completion Date :
Jan 19, 2024

Arms and Interventions

Arm Intervention/Treatment
Experimental: Voxeletor + SOC (Standard of Care)

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

Placebo Comparator: Placebo + SOC (Standard of Care)

Other: Placebo
Placebo

Outcome Measures

Primary Outcome Measures

  1. 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

  1. Time to resolution of target ulcer(s) [From pre-dose to Week 12]

  2. Change in total surface area(s) of target ulcer(s) [From pre-dose to Week 12]

  3. Incidence of new ulcers [From pre-dose to Week 12]

Eligibility Criteria

Criteria

Ages Eligible for Study:
12 Years and Older
Sexes Eligible for Study:
All
Accepts Healthy Volunteers:
No
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

  1. 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

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.
Responsible Party:
Global Blood Therapeutics
ClinicalTrials.gov Identifier:
NCT05561140
Other Study ID Numbers:
  • GBT440-042
First Posted:
Sep 30, 2022
Last Update Posted:
Sep 30, 2022
Last Verified:
May 1, 2022
Individual Participant Data (IPD) Sharing Statement:
No
Plan to Share IPD:
No
Studies a U.S. FDA-regulated Drug Product:
Yes
Studies a U.S. FDA-regulated Device Product:
No
Keywords provided by Global Blood Therapeutics
Additional relevant MeSH terms:

Study Results

No Results Posted as of Sep 30, 2022