Clinical trial NCT05431088: A Phase 2/3 Study in Adult and Pediatric Participants With SCD
Official title: A Phase 2/3 Randomized, Multicenter Study of Osivelotor Administered Orally to Participants With Sickle Cell Disease and an Open-Label Pharmacokinetics Study in Pediatric Participants With Sickle Cell Disease
Study type: INTERVENTIONAL
Overall status: ACTIVE_NOT_RECRUITING
Phase: PHASE2
PHASE3
Conditions: Sickle Cell Disease
Lead sponsor: Pfizer
Sponsor class: INDUSTRY
First posted: 2022-06-24
Enrollment: 429
Sex: ALL
Minimum age (years): 6
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

Brief summary: The purpose of this study is to evaluate the safety, tolerability, efficacy, pharmacokinetics and pharmacodynamics of osivelotor.

Detailed description: This is a three-part, multicenter, Phase 2/3 study of orally administered osivelotor in participants with sickle cell disease (SCD).

Part A will evaluate the safety, tolerability, and efficacy of osivelotor in adult participants with SCD to determine an optimal dose.

Part B will evaluate the efficacy of osivelotor versus placebo in adult and adolescent participants with SCD for 48 weeks.

Part C will evaluate the pharmacokinetics (PK) and safety of single and multiple doses (MD) of open-label single arm osivelotor administered to pediatric participants.

Eligibility criteria: Inclusion Criteria:

Part A, Part B, and Part C:

* Male or female with SCD
* Participants with stable Hb value as judged by the Investigator
* For participants taking hydroxyurea and/or L-glutamine, the dose must be stable for at least 90 days prior to signing the ICF or assent and with no anticipated need for dose adjustments during the study in the opinion of the Investigator.

Part B:

* Participants with SCD ages 12 to 65 years, inclusive
* Participants with more than or equal to 2 and ≤ 10 VOCs within 12 months of Screening.

Exclusion Criteria:

Part A, Part B, and Part C:

* Participants who had more than 10 VOC within 12 months of screening
* Female participant who is breastfeeding or pregnant
* Participants who receive RBC transfusion therapy regularly or received an RBC transfusion ---for any reason within 90 days of Day 1
* Participants hospitalized for sickle cell crisis or other vaso-occlusive event within 14 days of signing the ICF

Primary outcomes:
measure: Part A
timeFrame: Through week 12
measure: Part B
timeFrame: Through week 48
measure: Part C
timeFrame: Through Week 6
measure: Part C
timeFrame: Through Week 2
Secondary outcomes:
Other outcomes:
Locations:
city: New Haven
country: United States
facility: Smilow Cancer Hospital
state: Connecticut
city: Washington
country: United States
facility: Children's National Hospital
state: District of Columbia
city: Plantation
country: United States
facility: Edward Jenner Research Group Center LLC
state: Florida
city: West Palm Beach
country: United States
facility: Pediatric Hematology / Oncology a division of Kidz Medical services
state: Florida
city: Atlanta
country: United States
facility: Sonar Clinical Research
state: Georgia
city: Dunwoody
country: United States
facility: Alpha Clinical Research Georgia
state: Georgia
city: Chicago
country: United States
facility: University of Illinois at Chicago Clinical Research Center
state: Illinois
city: Baton Rouge
country: United States
facility: LSU Health Baton Rouge-North Clinic
state: Louisiana
city: New Orleans
country: United States
facility: University Medical Center New Orleans
state: Louisiana
city: Madison
country: United States
facility: Mississippi Center for Advanced Medicine
state: Mississippi
city: Kansas City
country: United States
facility: University Health
state: Missouri
city: Chapel Hill
country: United States
facility: UNC Health
state: North Carolina
city: Houston
country: United States
facility: McGovern Medical School at UTHealth
state: Texas
city: Fairfax
country: United States
facility: Inova Schar Cancer Institute
state: Virginia
city: Salvador
country: Brazil
facility: Hospital Universitario Professor Edgar Santos
state: Bahia
city: Recife
country: Brazil
facility: Multihemo Servicos Medicos S/A
state: PE
city: Ribeirão Preto
country: Brazil
facility: Hospital Das Clinicas da Faculdade de Medicina de Ribeirão Preto - USP
state: SÃO Paulo
city: São José Do Rio Preto
country: Brazil
facility: Fundacao Faculdade Regional de Medicina de Sao Jose do Rio Preto
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: Real e Benemerita Associacao Portuguesa de Sao Paulo
city: São Paulo
country: Brazil
facility: Hospital das Clinicas da Faculdade de Medicina da Universidade de Sao Paulo
city: Kisumu
country: Kenya
facility: KEMRI/CRDR, Siaya, KEMRI Clinical Research Annex
state: Siaya
city: Nairobi
country: Kenya
facility: Center for Research In Therapeutic Sciences (CREATES), Strathmore University Medical Centre
city: Kano
country: Nigeria
facility: Aminu Kano Teaching Hospital
Study officials:
affiliation: Pfizer
name: Pfizer CT.gov Call Center
role: STUDY_DIRECTOR
References: