Clinical trial NCT05561140: Resolution of Sickle Cell Leg Ulcers With Voxelotor
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
Study type: INTERVENTIONAL
Overall status: TERMINATED
Phase: PHASE3
Conditions: Sickle Cell Disease
Leg Ulcers
Keywords: Sickle Cell Disease, SCD
Lead sponsor: Pfizer
Sponsor class: INDUSTRY
First posted: 2022-09-30
Enrollment: 88
Sex: ALL
Minimum age (years): 12
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

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.


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

Primary outcomes:
measure: The effect of voxelotor and standard of care compared to placebo and SOC on leg ulcer healing
timeFrame: From pre-dose to Week 12
Secondary outcomes:
measure: Time to resolution of target ulcer(s)
timeFrame: From pre-dose to Week 12
measure: Change in total surface area(s) of target ulcer(s)
timeFrame: From pre-dose to Week 12
measure: Incidence of new ulcers
timeFrame: From pre-dose to Week 12
Other outcomes:
Locations:
city: Salvador
country: Brazil
facility: Complexo Hospitalar Universitário Professor Edgard Santos- Universidade Federal Da Bahia
state: Bahia
city: Salvador
country: Brazil
facility: Universidade Federal Da Bahia Hospital Universitário Professor Edgard Santos
state: Bahia
city: Salvador
country: Brazil
facility: Hospital São Rafael
state: Bahia
city: Belo Horizonte
country: Brazil
facility: Hospital das Clinicas da Universidade Federal de Minas Gerais
state: MG
city: Belo Horizonte
country: Brazil
facility: Hospital das Clinicas da Universidade Federal de Minas Gerais
state: Minas Gerais
city: Recife
country: Brazil
facility: Multihemo Servicos Medicos S/A
state: Pernambuco
city: Cerqueira Cesar - Sao Paulo
country: Brazil
facility: Hospital das Clínicas da Faculdade de Medicina da Universidade de Sao Paulo
state: SP
city: Ribeirão Preto
country: Brazil
facility: Hospital das Clinicas da Faculdade de Medicina de Ribeirão Preto da Universidade de Sao Paulo
state: SÃO Paulo
city: Sao Paulo
country: Brazil
facility: Esho Empresa De Servicos Hospitalares S.A/ Hospital Samaritano Higienopolis
city: Sao Paulo
country: Brazil
facility: Hospital das Clínicas da Faculdade de Medicina da Universidade de Sao Paulo
city: São Paulo
country: Brazil
facility: Hospital das Clínicas da Faculdade de Medicina da Universidade de Sao Paulo
city: Butere
country: Kenya
facility: KEMRI Centre for Clinical Research Butere County Hospital
state: Kakamega County
city: Kisumu
country: Kenya
facility: KEMRI Kondele Children's Hospital within Jaramogi Oginga Odinga Teaching and Referral Hospital.
city: Nairobi
country: Kenya
facility: Gertrude's Children's Hospital
city: Nairobi
country: Kenya
facility: KEMRI-Centre for Respiratory Diseases Research-Nairobi
city: Nairobi
country: Kenya
facility: Strathmore University Medical Centre
city: Siaya
country: Kenya
facility: KEMRI-CRDR, KEMRI Clinical Research Annex
city: Gwagwalada
country: Nigeria
facility: SYNLAB
state: Abuja
city: Calabar
country: Nigeria
facility: University of Calabar Teaching Hospital
state: Cross River
city: Abuja
country: Nigeria
facility: SYNLAB
state: FCT
city: Gwagwalada
country: Nigeria
facility: University of Abuja Teaching Hospital
state: FCT
city: Kaduna
country: Nigeria
facility: Barau Dikko Teaching Hospital/Kaduna State University
city: Kano
country: Nigeria
facility: Aminu kano Teaching Hospital
city: Lagos
country: Nigeria
facility: Lagos University Teaching Hospital
Study officials:
affiliation: Pfizer
name: Pfizer CT.gov Call Center
role: STUDY_DIRECTOR
References: