Discovery of Osivelotor (GBT021601): A Potent, Next-Generation Sickle Hemoglobin Polymerization Inhibitor.
Zhe Li, Carsten Alt, Kobina Dufu...
https://pubmed.ncbi.nlm.nih.gov/40832524Actively Recruiting
Phase 2
Phase 3
Age: 12Years +
All Genders
ID05431088
A Phase 2/3 Randomized, Multicenter Study of Osivelotor Administered Orally to Adult and Adolescent Participants With Sickle Cell Disease
Led by Pfizer · Updated on 2026-03-20
389
Participants Needed
49
Research Sites
104 weeks
Total Duration
On this page
AI-Summary
What this Trial Is About
Researchers are evaluating the safety, tolerability, effectiveness, and how the body processes and responds to osivelotor in people with sickle cell disease (SCD). This multicenter, Phase 2/3 study focuses on both adults and adolescents with SCD, aiming to determine the best dose and assess the drug's effects over time. The study has three parts. Part A tests safety, tolerability, and dose-finding in adults with SCD, starting with randomization to different daily doses of osivelotor, ranging from 100 mg to potentially 200 mg, over 12 weeks. Part B compares osivelotor to placebo in adults and adolescents over 48 weeks, with adults receiving an initial 300 mg daily dose for 7 days followed by 150 mg daily, while adolescent dosing will be defined later. The Open Label Extension (OLE) offers long-term open-label osivelotor treatment for up to two years after Part B. Participants will be monitored throughout the study with regular visits to assess safety, blood responses, and how well they tolerate the medication. The main results will be reviewed through 12 weeks in Part A, 48 weeks in Part B, and approximately 24 months in the OLE. The study includes blood tests, monitoring of vaso-occlusive crises, and other health evaluations to understand osivelotor's effects and safety over time.
CONDITIONS
Brief Title
A Phase 2/3 Study in Adult and Adolescent Participants With SCD
Who Can Participate
Age: 12Years +
All Genders
Eligibility Criteria
You may qualify if you...
- Male or female with sickle cell disease
- Participants with stable hemoglobin value as judged by the Investigator
- Stable dose of hydroxyurea and/or L-glutamine for at least 90 days prior to consent, with no planned dose changes
- Age between 12 and 65 years inclusive (for Part B)
- Experienced 2 to 10 vaso-occlusive crises within 12 months of screening
- Completed Part B to be eligible for Open Label Extension
You will not qualify if you...
- More than 10 vaso-occlusive crises within 12 months of screening
- Pregnant or breastfeeding female participants
- Regular or recent (within 90 days) red blood cell transfusion therapy
- Hospitalization for sickle cell crisis or vaso-occlusive event within 14 days before consent or during screening
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
1
2
3
Your Study Journey
Screening
Duration - 2 to 4 weeks
Participants are screened for eligibility to participate in the trial.
Part A: Treatment
Duration - Up to 12 weeks
Participants receive daily doses of osivelotor to evaluate safety, tolerability, and efficacy and determine the optimal dose.
Part B: Treatment
Duration - 48 weeks
Participants receive osivelotor or placebo to evaluate efficacy over a 48-week period.
Open Label Extension
Duration - Up to approximately 24 months
Participants receive open-label osivelotor to evaluate long-term safety and hematologic responses.
Trial Site Locations
Total: 49 locations
1
Smilow Cancer Hospital
New Haven, Connecticut, United States, 06511
Not Yet Recruiting
2
Edward Jenner Research Group Center LLC
Plantation, Florida, United States, 33317
Actively Recruiting
3
Pediatric Hematology / Oncology a division of Kidz Medical services
West Palm Beach, Florida, United States, 33407
Not Yet Recruiting
4
St. Mary's Medical Center
West Palm Beach, Florida, United States, 33407
Not Yet Recruiting
5
Alpha Clinical Research Georgia
Dunwoody, Georgia, United States, 30350
Active, Not Recruiting
6
Sonar Clinical Research
Riverdale, Georgia, United States, 30274
Active, Not Recruiting
7
University of Illinois at Chicago Clinical Research Center
Chicago, Illinois, United States, 60612
Not Yet Recruiting
8
University of Illinois Hospital and Health Sciences System - Investigational Drug Services (IDS)
Chicago, Illinois, United States, 60612
Not Yet Recruiting
9
University of Illinois Hospital and Health Sciences System
Chicago, Illinois, United States, 60612
Not Yet Recruiting
10
LSU Health Baton Rouge-North Clinic
Baton Rouge, Louisiana, United States, 70805
Active, Not Recruiting
11
Our Lady of the Lake Hospital, Inc.
Baton Rouge, Louisiana, United States, 70808
Active, Not Recruiting
12
Our lady of the Lake Hospital
Baton Rouge, Louisiana, United States, 70808
Active, Not Recruiting
13
University Medical Center New Orleans
New Orleans, Louisiana, United States, 70112
Active, Not Recruiting
14
Mississippi Center for Advanced Medicine
Madison, Mississippi, United States, 39110
Active, Not Recruiting
15
University Health
Kansas City, Missouri, United States, 64108
Not Yet Recruiting
16
Clinical & Translational Research Center (CTRC)
Chapel Hill, North Carolina, United States, 27514
Not Yet Recruiting
17
UNC Health
Chapel Hill, North Carolina, United States, 27514
Not Yet Recruiting
18
UNC Eastowne Medical Office Building - Consent Only
Chapel Hill, North Carolina, United States, 27517
Not Yet Recruiting
19
UNC IDS
Morrisville, North Carolina, United States, 27560
Not Yet Recruiting
20
McGovern Medical School at UTHealth
Houston, Texas, United States, 77030
Active, Not Recruiting
21
Memorial Hermann Hospital, Texas Medical Center - Clinical Research Unit (CRU)
Houston, Texas, United States, 77030
Active, Not Recruiting
22
The University of Texas Health Science Center at Houston
Houston, Texas, United States, 77030
Active, Not Recruiting
23
UT Physicians Comprehensive Sickle Cell Clinic
Houston, Texas, United States, 77030
Active, Not Recruiting
24
Inova Schar Cancer Institute
Fairfax, Virginia, United States, 22031
Actively Recruiting
25
Hospital Universitario Professor Edgar Santos
Salvador, Estado de Bahia, Brazil, 40110-060
Not Yet Recruiting
26
Multihemo Servicos Medicos S/A
Recife, Pernambuco, Brazil, 50070-460
Not Yet Recruiting
27
Hospital Das Clinicas da Faculdade de Medicina de Ribeirão Preto - USP
Ribeirão Preto, São Paulo, Brazil, 14051-140
Not Yet Recruiting
28
Fundacao Faculdade Regional de Medicina de Sao Jose do Rio Preto
São José do Rio Preto, São Paulo, Brazil, 15090-000
Not Yet Recruiting
29
BP A Beneficência Portuguesa de São Paulo
São Paulo, São Paulo, Brazil, 01321 001
Not Yet Recruiting
30
BP - A Beneficência Portuguesa de São Paulo
São Paulo, São Paulo, Brazil, 01323-900
Not Yet Recruiting
31
Casa de Saude Santa Marcelina
São Paulo, São Paulo, Brazil, 08270-070
Not Yet Recruiting
32
Casa de Saude Santa Marcelina
São Paulo, São Paulo, Brazil, 08270-120
Not Yet Recruiting
33
Esho Empresa de Servicos Hospitalares S A
São Paulo, SÃO Paulo- Brasil, Brazil, 01232-011
Not Yet Recruiting
34
Instituto Estadual de Hematologia Arthur Siqueira Cavalcanti - HEMORIO
Rio de Janeiro, Brazil, 20211-030
Not Yet Recruiting
35
Esho Empresa de Servicos Hospitalares S A
São Paulo, Brazil, 01232-010
Not Yet Recruiting
36
Real e Benemerita Associacao Portuguesa de Sao Paulo
São Paulo, Brazil, 01321-001
Not Yet Recruiting
37
Hospital das Clinicas da Faculdade de Medicina da Universidade de Sao Paulo
São Paulo, Brazil, 05403-000
Active, Not Recruiting
38
Hospital das Clinicas da Faculdade de Medicina da Universidade de Sao Paulo
São Paulo, Brazil, 05403-010
Active, Not Recruiting
39
Nirmal Hospital Pvt Ltd
Surat, Gujarat, India, 395002
Active, Not Recruiting
40
Chopda Medicare & Research Centre Pvt. Ltd: Magnum Heart Institute
Nashik, Maharashtra, India, 422005
Active, Not Recruiting
41
KEMRI/CRDR, Siaya, KEMRI Clinical Research Annex
Kisumu, Siaya County, Kenya, 40600
Not Yet Recruiting
42
Gertrude's Children's Hospital
Nairobi, Kenya, 00100
Not Yet Recruiting
43
Kenya Medical Research Institute - Centre for Respiratory Disease Research
Nairobi, Kenya, 00100
Not Yet Recruiting
44
Center for Research In Therapeutic Sciences (CREATES), Strathmore University Medical Centre
Nairobi, Kenya, 00200
Not Yet Recruiting
45
University College Hospital Ibadan
Ibadan, Oyo/ibadan North, Nigeria, 200212
Active, Not Recruiting
46
Aminu Kano Teaching Hospital
Kano, Nigeria, 700233
Active, Not Recruiting
47
Lagos University Teaching Hospital
Lagos, Nigeria, 100254
Active, Not Recruiting
48
University Hospitals Bristol and Weston NHS Foundation Trust
Bristol, United Kingdom, BS2 8ED
Not Yet Recruiting
49
Bristol Royal Infirmary
Bristol, United Kingdom, BS2 8EX
Not Yet Recruiting
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Research Team
P
Pfizer Pfizer CT.gov Call Center
How is the study designed?
Study Type
INTERVENTIONAL
Masking
QUADRUPLE
Allocation
RANDOMIZED
Model
PARALLEL
Primary Purpose
TREATMENT
Number of Arms
3
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Mofiyin Obadina, Sam Wilson, Vimal K Derebail...
https://pubmed.ncbi.nlm.nih.gov/37254256