Den här översättningen är inte klar ännu. Den här sidan är just nu på engelska.

Gå till den engelska sidan

Cure My Disease

Don't miss your cure!

 Log in / Sign up

New pill could slash sickle cell pain crises

NCT ID NCT04624659

Disease control Sponsor: Forma Therapeutics, Inc. Source: ClinicalTrials.gov ↗

What the study statuses mean

This study's is highlighted.

Recruitment status, easiest to join first

Recruiting now
This trial is taking on new participants right now.
Not yet recruiting
Registered, but not yet taking participants.
By invitation only
Not open to general applications. Only people the study team invites can take part.
Paused
Paused for now. It may or may not start again.
Ongoing This study
Running, but no longer taking on new participants.
Completed
The trial has finished. Results may not be published yet.
Stopped early
Stopped early, before it reached the end. That can be for many reasons, including safety.
Cancelled
Cancelled before anyone took part.

Expanded access (not trials)

Expanded access
Not a trial. This treatment can be requested outside a study, case by case, for people who qualify.
Expanded access (paused)
Not a trial. The treatment can normally be requested outside a study, but is unavailable right now.
Expanded access (ended)
Not a trial. The treatment could once be requested outside a study, but no longer can.
Approved
The treatment has been approved, so it is available normally rather than through this programme.

When the status isn't known

Details not published
The full record has not been published yet, so there is little to show here.
Status unknown
This status has not been confirmed recently, so it may be out of date.

First seen Jun 27, 2026 · Last updated Jun 27, 2026

Summary

This large Phase 3 trial is testing an oral drug called etavopivat in 450 people with sickle cell disease. The goal is to see if taking a pill once daily can raise hemoglobin levels and reduce the number of painful vaso-occlusive crises compared to a placebo. Participants must have had at least two crises in the past year.

What this could mean

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

Active substance
Etavopivat (a pyruvate kinase activator taken as a pill once daily)
What this could lead to
If it works, this could provide a new daily pill to help people with sickle cell disease have fewer painful blockages and higher hemoglobin levels.
What could go wrong
This is still being tested in a large Phase 3 trial, so it may not prove effective or safe for everyone. Side effects are possible, and it requires ongoing daily use.

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 3

Large-scale testing in a bigger group. Usually the last step before a treatment can be approved.

Participants

About 450 people

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

Started

Jan 2021

Expected to finish

Mar 2027

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 to 65 years

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.

Key Inclusion Criteria: * Provision of consent * Patient has a confirmed diagnosis of sickle cell disease * At least 2 episodes of vaso-occlusive crises in the past 12 months * Hemoglobin ≥ 5.5 and ≤ 10.5 g/dL (≥ 55 and ≤ 105 g/L) during screening * Patients taking hydroxyurea, must demonstrate a stable dose for at least 90 days prior to start of study treatment * Patients on crizanlizumab or L-glutamine treatment at the time of consent must be on a stable dose for ≥ 12 months and must be ≥ 80% compliant with the planned regimen at the time of consent and meet the VOC eligibility criteria * Female patients of childbearing potential must use highly effective methods of contraception, male patients are willing to use barrier methods of contraception Key Exclusion Criteria: * More than 15 vaso-occlusive crises within the past 12 months * Female who is breastfeeding or pregnant * Hepatic dysfunction characterized by: * Alanine aminotransferase (ALT) \> 4.0 × upper limit of normal (ULN) * Direct bilirubin \> 3.0 × ULN * Known HIV positivity * Active hepatitis B or hepatitis C infection * Severe renal dysfunction or on chronic dialysis * History of unstable or deteriorating cardiac or pulmonary disease within 6 months prior to consent including but not limited to the following: * Unstable angina pectoris or myocardial infarction or elective coronary intervention * Congestive heart failure requiring hospitalization * Uncontrolled clinically significant arrhythmias * Symptomatic pulmonary hypertension * History of overt clinical stroke within previous 2 years or any history of an intracranial hemorrhage * History of deep venous thrombosis requiring systemic anti-coagulation therapy for ≥ 6 weeks, occurring within 6 months prior to Day 1 of study treatment. Prior/Concomitant Therapy * Patients receiving regularly scheduled blood (RBC) transfusion therapy (also termed chronic, prophylactic, or preventive transfusion) * Receiving or use of concomitant medications that are strong inducers of CYP3A4/5 within 2 weeks of starting study treatment or anticipated need for such agents during the study * Use of voxelotor within 28 days prior to starting study treatment or anticipated need for this agent during the study * Use of an experimental selectin antagonist (eg, monoclonal antibody or small molecule) within 28 days of starting study treatment or anticipated need for such agents during the study * Use of erythropoietin or other hematopoietic growth factor treatment within 28 days of starting study treatment or anticipated need for such agents during the study * Receipt of prior cellular-based therapy (eg, hematopoietic cell transplant, gene modification therapy)

Get updates

Get notified about this study

Sign up to get updates when this study changes or when new studies for Sickle cell disease are added.

Vår säkerhetsrekommendation!

Genom att skicka in godkänner du våra Användarvillkor

Conditions

The condition(s) this trial relates to.

anemia hematologic disorder Hemolysis hemolytic anemia hereditary disease inherited hemoglobinopathy sickle cell disease Sickle Cell Trait Vaso-Occlusive Crises

As listed by the trial registrant

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

Sickle cell disease

Contacts and locations

Locations

  • 'Ippokrateio' General Hospital of Thessaloniki

    Thessaloniki, 54642, Greece

  • ARNAS Ospedali Civico Di Cristina Benfratelli - Presidio Ospedaliero "Civio e Benfratelli"

    Palermo, 90127, Italy

  • Adana City Training and Research Hospital

    Adana, 4522, Turkey (Türkiye)

  • Advanced Pharma CR

    Miami, Florida, 33147, United States

  • Ain Shams University Hospital

    Cairo, 1181, Egypt

  • Al Kasr AL Aini Hospital

    Cairo, 11956, Egypt

  • All India Institute of Medical Sciences (AIIMS)

    New Delhi, 110029, India

  • American University of Beirut Medical center

    Beirut, 11-0236, Lebanon

  • Aminu Kano Teaching Hospital (AKTH)

    Kano, 3452, Nigeria

  • Ap-Hp-Hopital Henri Mondor

    Créteil, 94010, France

  • Ap-Hp-Hopital Robert Debre

    Paris, 75019, France

  • Arnold Palmer Children's Hospital

    Orlando, Florida, 32806, United States

  • Azienda Ospedale Universita Padova

    Padova, 35128, Italy

  • Azienda Ospedaliera Universitaria San Luigi Gonzaga - S.C.D.O. Microcitemie e malattie rare ematologiche

    Orbassano, 10043, Italy

  • Azienda Ospedaliero Universitaria Policlinico G. Rodolico - San Marco

    Catania, 95123, Italy

  • Barau Dikko Teaching Hospital, Kaduna

    Kaduna, 800212, Nigeria

  • Başkent Üniversitesi Adana Dr. Turgut Noyan Uygulama ve Araştırma Merkezi

    Adana, 1240, Turkey (Türkiye)

  • Boston Medical Center

    Boston, Massachusetts, 02118, United States

  • CHU Sainte-Justine Mother and Child University Hospital

    Montreal, Quebec, H3T 1C5, Canada

  • Cairo University

    Cairo, 111111, Egypt

  • Center for Blood Disorders Augusta University

    Augusta, Georgia, 30912, United States

  • Centre Hospitalier Universitaire de Montpellier-Hopital Saint-Eloi

    Montpellier, 34295, France

  • Centro per lo Studio e la Cura delle Amiloidosi Sistemiche Fondazione IRCCS Policlinico San Matteo

    Pavia, PV, 27100, Italy

  • Charité - Campus Virchow-Klinikum - Klinik für Pädiatrie mit Schwerpunkt Onkologie und Hämatologie

    Berlin, 13353, Germany

  • Children's Healthcare of Atlanta Investigational Medication Pharmacy

    Atlanta, Georgia, 30342, United States

  • Children's Hosp-New Orleans

    New Orleans, Louisiana, 70118, United States

  • Children's Hosptl Philadelphia

    Philadelphia, Pennsylvania, 19104, United States

  • Children's National Health Hospital

    Washington D.C., District of Columbia, 20010, United States

  • Churchill Hospital

    Oxford, OX3 7LE, United Kingdom

  • Columbia University Medical Center_New York

    New York, New York, 10032, United States

  • Complejo Hospitalario Universitario A Coruña

    A Coruña, 15006, Spain

  • Cornerstone Research Institute

    Altamonte Springs, Florida, 32701, United States

  • Duke University Medical Center

    Durham, North Carolina, 27710, United States

  • East Carolina University

    Greenville, North Carolina, 27858, United States

  • Ege Üniversitesi Tip Fakultesi Hastanesi - Nefroloji

    Izmir, 35040, Turkey (Türkiye)

  • Emory University School of Medicine

    Atlanta, Georgia, 30303, United States

  • Foundation for Sickle Cell Disease Research

    Hollywood, Florida, 33023, United States

  • GHR Mulhouse Sud-Alsace

    Mulhouse, 68100, France

  • Gen Hosp Athens Ippokrateio,B' Uni Clinic Internal Medicine

    Athens, 115 27, Greece

  • General Hospital Of Larissa Koutlibaneio And Triantafylleio - Thalassemia and SCD Unit

    Larissa, 41221, Greece

  • General University Hospital of Patras

    Pátrai, Larissa, 265 04, Greece

  • Gertrude's Children's Hospital

    Nairobi, 100, Kenya

  • Ghana Institute of Clinical Genetics, Korle Bu Teaching Hospital (KBTH)

    Accra, 0, Ghana

  • Guys and St Thomas NHS Foundation Trust / Evelina Childrens Hospital

    London, SE18 3RA, United Kingdom

  • Hacettepe Üniversitesi Hastanesi- Hematoloji

    Ankara, 6230, Turkey (Türkiye)

  • Hammersmith Hospital - London

    London, W2 1NY, United Kingdom

  • Hospices Civils de Lyon-Hopital Edouard Herriot

    Lyon, 69437, France

  • Hospital Clínico San Carlos

    Madrid, 28040, Spain

  • Hospital Germans Trias i Pujol

    Badalona, 8916, Spain

  • Hospital Gregorio Marañón

    Madrid, 28009, Spain

  • Hospital Nini

    Tripoli, 113-6044, Lebanon

  • Hospital Ramón y Cajal

    Madrid, 28034, Spain

  • Hospital Sant Joan de Déu

    Barcelona, 8950, Spain

  • Hospital Universitario La Paz

    Madrid, 28046, Spain

  • Hospital Universitario Virgen del Rocío

    Seville, 41013, Spain

  • Hospital Universitario de Cruces

    Barakaldo, 48903, Spain

  • Hospital Vall d'Hebron

    Barcelona, 08035, Spain

  • Howard University

    Washington D.C., District of Columbia, 20060, United States

  • Jacobi Medical Center

    The Bronx, New York, 10461, United States

  • Jimmy Everest Cancer Hematology & Oncology

    Oklahoma City, Oklahoma, 73104, United States

  • K.J Somaiya Hospital and Research Centre

    Mumbai, Maharashtra, 400022, India

  • KEMRI CRDR Siaya Clinical Research Annex, Country Referral Hospital

    Kisumu, 40600, Kenya

  • KEMRI Kondele Children Hospital, Kisumu

    Kisumu, 40100, Kenya

  • Karmanos Cancer Institute

    Detroit, Michigan, 48201, United States

  • King Fahad Specialist Hospital-Dammam

    Dammam, 32253, Saudi Arabia

  • King Khaled University Hospital,King Saud Univ. Med. City

    Riyadh, 12372 6864, Saudi Arabia

  • Kings College Hospital - Haematology

    London, SE5 9RS, United Kingdom

  • Kintampo Health Research Centre (KHRC)

    Kintampo, Bono East, 27WF+98M, Ghana

  • Kombewa Clinical Research Centre

    Kisumu, 1932, Kenya

  • Lagos University Teaching Hospital, Lagos

    Lagos, 102215, Nigeria

  • Leicester Royal Infirmary - Haemostasis & Thrombosis Unit

    Leicester, LE1 5WW, United Kingdom

  • Levine Cancer Institute

    Charlotte, North Carolina, 28204, United States

  • Lynn Institute of Tulsa

    Tulsa, Oklahoma, 74135, United States

  • Mary Bridge Children's Health

    Tacoma, Washington, 98405, United States

  • Massachusetts General Hospital_Boston

    Boston, Massachusetts, 02114, United States

  • Master Centre for Lebanon

    Amman, 11844, Lebanon

  • Medical University of South Carolina

    Charleston, South Carolina, 29425, United States

  • Mersin Üniversitesi Tip Fakültesi Hastanesi- Çiftlikköy Yerleşkesi- Hematoloji

    Mersin, 33110, Turkey (Türkiye)

  • Methodist University Hospital

    Memphis, Tennessee, 38104, United States

  • Montefiore Medical Center

    The Bronx, New York, 10467, United States

  • NYC Health+Hospitals

    Brooklyn, New York, 11203, United States

  • Neuro-Behavioral Clinical Research

    North Canton, Ohio, 44720, United States

  • North Shore Long Island Jewish Medical Center

    New Hyde Park, New York, 11040, United States

  • Ospedale Pediatrico Bambino Ges

    Roma, 00165, Italy

  • Pacific Research Partners

    Oakland, California, 94607, United States

  • Phoenix Children's Hsptl

    Phoenix, Arizona, 85016, United States

  • Prince Mohammad Bin Naser Hospital

    Jizan, 82943, Saudi Arabia

  • Prisma Health-Upstate

    Greenville, South Carolina, 29605, United States

  • Providence Hematolgy

    Vancouver, British Columbia, V6E 1M7, Canada

  • Queens Hospital Center

    Jamaica, New York, 11432-1121, United States

  • Rhode Island Hospital

    Providence, Rhode Island, 02903, United States

  • Royal Hallamshire Hospital

    Sheffield, S11 8RN, United Kingdom

  • Saint-Denis Hospital Center

    Saint-Denis, 93200, France

  • Sonar Clinical Research

    Atlanta, Georgia, 30315, United States

  • Srirama Chandra Bhanj Medical College & Hospital (S.C.B Medical College)

    Cutack, 753007, India

  • Sultan Qaboos University Hospital

    Muscat, Sultanet of Oman/Muscat/Al Khoud, 123, Oman

  • Sydnawy University Hospital

    Zagazig, 44519, Egypt

  • The Hospital for Sick Children

    Toronto, Ontario, M5G 1X8, Canada

  • The Ohio State University Medical Center

    Columbus, Ohio, 43203, United States

  • Thomas Jefferson University Hospital

    Philadelphia, Pennsylvania, 19107, United States

  • UC Davis Medical Center

    Sacramento, California, 95817, United States

  • UCSF Oakland Benioff ChildHosp

    Oakland, California, 94609, United States

  • UT Health University of Texas

    Houston, Texas, 77030, United States

  • Uniklinik Heidelberg - Klinik für Pädiatrische Onkologie, Hämatologie, Immunologie und Pneumologie

    Heidelberg, 69120, Germany

  • Univ of Alabama Birmingham

    Birmingham, Alabama, 35233, United States

  • University Health Network - Toronto General Hospital

    Toronto, Ontario, M5G 2C4, Canada

  • University Hospital Lewisham, Lewisham and Greenwich NHS Trust

    London, SE13 6LH, United Kingdom

  • University Of California Irvine

    Orange, California, 92868, United States

  • University Of Maryland School of Medicine

    Baltimore, Maryland, 21201, United States

  • University Of Miami

    Miami, Florida, 33136, United States

  • University of Abuja Teaching Hospital, Gwagwalada, Abuja

    Abuja, 228, Nigeria

  • University of Cincinnati

    Cincinnati, Ohio, 45219, United States

  • University of Connecticut

    Farmington, Connecticut, 06030, United States

  • University of Florida

    Gainesville, Florida, 32610, United States

  • University of Illinois at Chicago

    Chicago, Illinois, 60612, United States

  • University of Nebraska Medical Center

    Omaha, Nebraska, 68198-2168, United States

  • University of Nigeria Teaching Hospital (UNTH)

    Lagos, 102215, Nigeria

  • University of Pittsburgh Medical Center

    Pittsburgh, Pennsylvania, 15213-4306, United States

  • Universitätsklinikum Frankfurt

    Frankfurt am Main, 60596, Germany

  • Universitätsklinikum Freiburg-Zentrum für Kinder- und Jugendmedizin

    Freiburg im Breisgau, 79106, Germany

  • Universitätsklinikum Hamburg-Eppendorf - University Medical Center Hamburg-Eppendorf

    Hamburg, 20246, Germany

  • Universitätsklinikum Regensburg

    Regensburg, 93053, Germany

  • VM Medical Park Mersin Hastanesi- Kardiyoloji

    Mersin, 33200, Turkey (Türkiye)

  • Versiti Blood Center of Wisconsin

    Milwaukee, Wisconsin, 53226, United States

  • Victoria Hospital, Bangalore

    Bangalore, 560002, India

  • Virginia Commonwealth University

    Richmond, Virginia, 23298, United States

  • Wake Forest Baptist Health

    Winston-Salem, North Carolina, 27157, United States

  • Washington University School of Medicine_St. Louis

    St Louis, Missouri, 63110, United States

  • Weill Cornell Med Coll-NYPH

    New York, New York, 10065, United States

  • Yale University

    New Haven, Connecticut, 06519, United States

  • Yashoda Hospital

    Hyderabad, Telangana, 500084, India

  • [Legal] Alexandria Clinical Research LLC

    Alexandria, 0, Egypt

  • [Legal] Alexandria Clinical Research LLC

    Alexandria, 21131, Egypt

  • [Legal] Baylor College of Medicine

    Houston, Texas, 77030, United States

  • [Legal] Children's Hospital Medical Center dba Cincinnati Children's

    Cincinnati, Ohio, 45229, United States

  • [Legal] Collaborative Neuroscience Network, LLC

    Long Beach, California, 90806, United States

  • [Legal] Dr. Vince Clinical Research, LLC and Dr. Vince Clinical Research, P.A.

    Detroit, Michigan, 48201, United States

  • [Legal] St. Jude Children's Research Hospital

    Memphis, Tennessee, 38105, United States

  • [Legal] The University of North Carolina at Chapel Hill

    Chapel Hill, North Carolina, 27514, United States

  • [Legal] Woodland International Research Group, LLC

    Little Rock, Arkansas, 72211, United States

  • iResearch Atlanta, LLC

    Decatur, Georgia, 30030, United States

  • Çukurova Üniversitesi Tıp Fakültesi Balcalı Hastanesi- Hematoloji

    Adana, 1130, Turkey (Türkiye)

More trials for these conditions

Other studies related to the condition(s) this trial covers.