New pill aims to ease sickle cell pain and boost hemoglobin

NCT ID NCT05431088

Disease control Sponsor: Pfizer Source: ClinicalTrials.gov ↗

First seen Jun 27, 2026 · Last updated Aug 19, 2026 · Updated 2 times

Summary

This study tests an oral drug called osivelotor in about 389 adults and adolescents with sickle cell disease. The goal is to see if it can raise hemoglobin levels and reduce the number of painful vaso-occlusive crises. Participants will take the pill daily, and researchers will monitor safety and effectiveness over several months.

What this could mean

Our plain-language read of the trial. This is informational only, not medical advice or a prediction.

Active substance: osivelotor (a drug taken by mouth to help red blood cells carry oxygen better)
What this could lead to: If successful, this could provide a new daily pill to reduce painful crises and improve anemia for people with sickle cell disease.
What could go wrong: This is an early-to-mid-stage trial, so the drug may not prove effective or safe enough. Side effects are still being studied, and results may not apply to all patients.

This is an AI summary of the original study and may miss details. Read our disclaimer.

Study facts

What this study's own registry entry says, in plain language.

Phase

Phase 2/3

Runs two stages together: whether the treatment works, then large-scale confirmation.

Participants

About 389 people

The number the study aims to enrol. It can still change while the study runs.

Started

Sep 2022

Expected to finish

Dec 2032

An estimate. End dates often move.

Lead sponsor

A company

The lead sponsor is a pharmaceutical, biotech, or medical-device company.

Who can take part

This study's own entry requirements. Only the study team can say for certain whether you qualify.

Ages

12 years and older

Sex

Anyone

Healthy volunteers

Not accepted

This study is not open to healthy volunteers. The entry requirements below say who it is open to.

Show the full entry requirements

Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.

Inclusion Criteria: Part A and Part B: * Male or female with SCD, HbSS and HbSB-zero * Participants with Hemoglobin ≥ 5.5 and ≤ 10.5 g/dL during Screening and considered stable 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 years and older, inclusive at screening. * Participants with more than or equal to 2 and ≤ 10 VOCs within 12 months of Screening. OLE: \- Participants who have completed the Part B successfully will be eligible. Exclusion Criteria: Part A and Part B: * 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 or anytime during the screening period. * Participants in Sub-Saharan Africa or who have relocated from Sub-Saharan Africa within 6 months. OLE: * Have any unresolved clinically significant adverse event, laboratory abnormality, or safety finding from Part B that, in the opinion of the Investigator, increases the risk of study drug administration. * Met permanent treatment discontinuation criteria during Part B. * Have withdrawn consent or are unable to comply with study procedure

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Conditions

The condition(s) this trial relates to.

sickle cell disease

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

Sickle cell disease

How to take part

Only the study team decides who joins. These are the ways to reach them.

The places running it

60 sites in 5 countries. The list below names each one and where it is.
The official record

ClinicalTrials.gov lists the study team's own contact details, including names and phone numbers. We don't republish those.

Open the record ↗
A doctor treating you

A doctor who knows your case can contact a study site on your behalf, and can tell you whether this study is worth pursuing at all.

Contacts and locations

Locations

AIIMS Delhi
NOT_YET_RECRUITING

New Delhi, National Capital Territory of Delhi, 110029, India
AIIMS Raipur
NOT_YET_RECRUITING

Raipur, Chhattisgarh, 492099, India
AIIMS, Bhubaneswar
NOT_YET_RECRUITING

Bhubaneswar, Odisha, 751019, India
AMR Clinical
RECRUITING

Mobile, Alabama, 36608, United States
Alpha Clinical Research Georgia
ACTIVE_NOT_RECRUITING

Dunwoody, Georgia, 30350, United States
Aminu Kano Teaching Hospital
COMPLETED

Kano, 700233, Nigeria
BP - A Beneficência Portuguesa de São Paulo
NOT_YET_RECRUITING

São Paulo, São Paulo, 01323-900, Brazil
BP A Beneficência Portuguesa de São Paulo
NOT_YET_RECRUITING

São Paulo, São Paulo, 01321 001, Brazil
Bristol Royal Infirmary
RECRUITING

Bristol, BS2 8EX, United Kingdom
CPHosp Medicina, Ensino e Pesquisa Ltda
NOT_YET_RECRUITING

São, São Paulo, 01228-000, Brazil
Casa de Saude Santa Marcelina
NOT_YET_RECRUITING

São Paulo, São Paulo, 08270-070, Brazil
Casa de Saude Santa Marcelina
NOT_YET_RECRUITING

São Paulo, São Paulo, 08270-120, Brazil
Center for Research In Therapeutic Sciences (CREATES), Strathmore University Medical Centre
COMPLETED

Nairobi, 00200, Kenya
Children's Center for Advanced Pediatrics
RECRUITING

Atlanta, Georgia, 30329, United States
Children's Hospital of Michigan
NOT_YET_RECRUITING

Detroit, Michigan, 48201, United States
Chopda Medicare & Research Centre Pvt. Ltd: Magnum Heart Institute
RECRUITING

Nashik, Maharashtra, 422005, India
Clinical & Translational Research Center (CTRC)
NOT_YET_RECRUITING

Chapel Hill, North Carolina, 27514, United States
Connecticut Children's Medical Center
NOT_YET_RECRUITING

Hartford, Connecticut, 06106, United States
Duke University IDS Pharmacy
RECRUITING

Durham, North Carolina, 27710, United States
Duke University Medical Center
RECRUITING

Durham, North Carolina, 27710, United States
ECU Physicians, Brody Outpatient Clinic
RECRUITING

Greenville, North Carolina, 27834, United States
Edward Jenner Research Group LLC.
RECRUITING

Plantation, Florida, 33317, United States
Esho Empresa de Servicos Hospitalares S A
NOT_YET_RECRUITING

São Paulo, SÃO Paulo- Brasil, 01232-011, Brazil
Esho Empresa de Servicos Hospitalares S A
NOT_YET_RECRUITING

São Paulo, 01232-010, Brazil
Fundacao Faculdade Regional de Medicina de Sao Jose do Rio Preto
RECRUITING

São José do Rio Preto, São Paulo, 15090-000, Brazil
Gertrude's Children's Hospital
COMPLETED

Nairobi, 00100, Kenya
Great Lakes Research Institute, Flourish Detroit
NOT_YET_RECRUITING

Southfield, Michigan, 48075, United States
Guy's & St Thomas' NHS Foundation Trust
RECRUITING

London, SE1 9RT, United Kingdom
Haemato Oncology Care Centre
RECRUITING

Vadodara, Gujarat, 390020, India
Hammersmith Hospital
RECRUITING

London, W12 0HS, United Kingdom
Hammersmith Hospital, Imperial College Healthcare NHS Trust
RECRUITING

London, W12 0HS, United Kingdom
Hospital Das Clinicas da Faculdade de Medicina de Ribeirão Preto - USP
NOT_YET_RECRUITING

Ribeirão Preto, São Paulo, 14051-140, Brazil
Hospital das Clinicas da Faculdade de Medicina da Universidade de Sao Paulo
ACTIVE_NOT_RECRUITING

São Paulo, 05403-000, Brazil
Hospital das Clinicas da Faculdade de Medicina da Universidade de Sao Paulo
ACTIVE_NOT_RECRUITING

São Paulo, 05403-010, Brazil
Icahn School of Medicine at Mount Sinai
RECRUITING

New York, New York, 10029, United States
Inova Schar Cancer Institute
RECRUITING

Fairfax, Virginia, 22031, United States
Instituto Estadual de Hematologia Arthur Siqueira Cavalcanti - HEMORIO
NOT_YET_RECRUITING

Rio de Janeiro, 20211-030, Brazil
Jack & Sheryl Morris Cancer Center
RECRUITING

New Brunswick, New Jersey, 08901, United States
Jacobi Medical Center
RECRUITING

The Bronx, New York, 10461, United States
KEMRI/CRDR, Siaya, KEMRI Clinical Research Annex
COMPLETED

Kisumu, Siaya County, 40600, Kenya
Kenya Medical Research Institute - Centre for Respiratory Disease Research
COMPLETED

Nairobi, 00100, Kenya
King Khalid University Hospital
NOT_YET_RECRUITING

Riyadh, Riyadh Region, 11472, Saudi Arabia
King's College Hospital NHS Foundation Trust
RECRUITING

London, SE5 9RS, United Kingdom
LSU Health Baton Rouge-North Clinic
ACTIVE_NOT_RECRUITING

Baton Rouge, Louisiana, 70805, United States
Lagos University Teaching Hospital
COMPLETED

Lagos, 100254, Nigeria
McGovern Medical School at UTHealth
ACTIVE_NOT_RECRUITING

Houston, Texas, 77030, United States
Memorial Hermann Hospital, Texas Medical Center - Clinical Research Unit (CRU)
ACTIVE_NOT_RECRUITING

Houston, Texas, 77030, United States
Mississippi Center for Advanced Medicine
ACTIVE_NOT_RECRUITING

Madison, Mississippi, 39110, United States
Multihemo Servicos Medicos S/A
NOT_YET_RECRUITING

Recife, Pernambuco, 50070-460, Brazil
Nirmal Hospital Pvt Ltd
RECRUITING

Surat, Gujarat, 395002, India
Our Lady of the Lake Hospital, Inc.
ACTIVE_NOT_RECRUITING

Baton Rouge, Louisiana, 70808, United States
Our lady of the Lake Hospital
ACTIVE_NOT_RECRUITING

Baton Rouge, Louisiana, 70808, United States
Pediatric Hematology / Oncology a division of Kidz Medical services
RECRUITING

West Palm Beach, Florida, 33407, United States
Prisma Health - Upstate
RECRUITING

Greenville, South Carolina, 29605, United States
Prisma Health Greenville Memorial Hospital
RECRUITING

Greenville, South Carolina, 29605, United States
Prisma Health Greer Memorial Hospital
RECRUITING

Greer, South Carolina, 29650, United States
Prisma Health Hillcrest Hospital
RECRUITING

Simpsonville, South Carolina, 29681, United States
Prisma Health Laurens County Hospital
RECRUITING

Clinton, South Carolina, 29325, United States
Prisma Health North Greenville Hospital
RECRUITING

Travelers Rest, South Carolina, 29690, United States
Prisma Health Outpatient Radiology - Greenville
RECRUITING

Greenville, South Carolina, 29605, United States
Prisma Health Patewood Hospital
RECRUITING

Greenville, South Carolina, 29615, United States
Robert Wood Johnson University Hospital
RECRUITING

New Brunswick, New Jersey, 08901, United States
Rutgers Cancer Institute of New Jersey
RECRUITING

New Brunswick, New Jersey, 08901, United States
Smilow Cancer Hospital
NOT_YET_RECRUITING

New Haven, Connecticut, 06511, United States
Sonar Clinical Research
ACTIVE_NOT_RECRUITING

Riverdale, Georgia, 30274, United States
Southern Specialty Research
RECRUITING

Flowood, Mississippi, 39232, United States
St. Mary's Medical Center
RECRUITING

West Palm Beach, Florida, 33407, United States
The University of Texas Health Science Center at Houston
ACTIVE_NOT_RECRUITING

Houston, Texas, 77030, United States
UNC Eastowne Medical Office Building - Consent Only
NOT_YET_RECRUITING

Chapel Hill, North Carolina, 27517, United States
UNC Health
NOT_YET_RECRUITING

Chapel Hill, North Carolina, 27514, United States
UNC IDS
NOT_YET_RECRUITING

Morrisville, North Carolina, 27560, United States
UT Physicians Comprehensive Sickle Cell Clinic
ACTIVE_NOT_RECRUITING

Houston, Texas, 77030, United States
University College Hospital Ibadan
COMPLETED

Ibadan, Oyo/ibadan North, 200212, Nigeria
University College London Hospital
RECRUITING

London, NW1 2PG, United Kingdom
University Health
RECRUITING

Kansas City, Missouri, 64108, United States
University Hospitals Bristol and Weston NHS Foundation Trust
RECRUITING

Bristol, BS2 8ED, United Kingdom
University Medical Center New Orleans
ACTIVE_NOT_RECRUITING

New Orleans, Louisiana, 70112, United States
University of Illinois Hospital and Health Sciences System
RECRUITING

Chicago, Illinois, 60612, United States
University of Illinois Hospital and Health Sciences System - Investigational Drug Services (IDS)
RECRUITING

Chicago, Illinois, 60612, United States
University of Illinois at Chicago Clinical Research Center
RECRUITING

Chicago, Illinois, 60612, United States

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