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New drug aims to cut sickle cell pain crises in half

NCT ID NCT05031780

Disease control Sponsor: Agios Pharmaceuticals, 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 Results may not be published yet.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This status has not been confirmed recently, so it may be out of date.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 study tests a drug called mitapivat in 286 people with sickle cell disease, ages 16 and older. The goal is to see if mitapivat can raise hemoglobin levels and reduce painful sickle cell crises compared to a placebo. The trial has two phases: first to find the best dose, then to confirm its safety and effectiveness, with an option for long-term follow-up.

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

286 people

The number who actually took part.

Started

Feb 2022

Expected to finish

Feb 2030

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

16 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: * Age 16 years or older (18 years or older \[France and Germany\]); participants age 16 or 17 years must physically have completed puberty; * Documented diagnosis of sickle cell disease (SCD) (HbSS, HbSC \[combined heterozygosity for hemoglobins S and C\], HbS/beta 0- thalassemia, HbS/ beta plus thalassemia, or other sickle cell syndrome variants); * At least 2 SCPCs and no more than 10 SCPCs in the past 12 months; * Hemoglobin at least 5.5 and 10.5 gram per deciliter (g/dL) at the most. Hemoglobin concentration must be based on an average of at least 2 Hb concentration measurements (separated by ≥7 days) collected during the Screening Period; * If taking hydroxyurea, the hydroxyurea dose must be stable for at least 90 days before starting study drug. Discontinuation of hydroxyurea requires a 90-day washout prior to informed assent/consent; * Women capable of becoming pregnant must agree to use 2 forms of contraception. Exclusion Criteria: * Pregnant, breastfeeding, or parturient; * Receiving regularly scheduled transfusions; * Hepatobiliary disorders including but not limited to significant liver disease or gallbladder disease; * Severe kidney disease; * Prior exposure to gene therapy or prior bone marrow or stem cell transplantation; * Currently receiving treatment with a disease-modifying therapy for SCD (eg, voxelotor, crizanlizumab, L-glutamine), with the exception of hydroxyurea. The last dose of voxelotor, crizanlizumab, and L-glutamine must have been administered at least 90 days before randomization; * Currently receiving treatment with hematopoietic stimulating agents; the last dose must have been administered at least 90 days before starting study drug; * Received treatment on another investigational trial within 90 days prior to start of study drug or plans to participate in another investigational drug trial; * Taking medications that are strong inhibitors of CYP3A4/5 or strong inducers of CYP3A4 that cannot be stopped in an acceptable timeframe before starting study drug (timeframe will be discussed with your doctor).

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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

Contacts and locations

Locations

  • A.O.R.N. "A. Cardarelli"

    Naples, Campania, 80131, Italy

  • AOU dell'Università degli Studi della Campania Luigi Vanvitelli

    Naples, Campania, 80138, Italy

  • Acibadem Adana Hospital

    Seyhan, Adana, 01130, Turkey (Türkiye)

  • American University of Beirut Medical Center

    Beirut, Beirut, 11-0236, Lebanon

  • American University of Beirut Medical Center

    Beirut, 4407-2020, Lebanon

  • Azienda Ospedaliera Ospedali Riuniti Villa Sofia-Cervello

    Palermo, Sicily, 90146, Italy

  • Azienda Ospedaliero Universitaria Di Modena Policlinico

    Modena, Emilia-Romagna, 41100, Italy

  • Boston Children's Hospital

    Boston, Massachusetts, 02115-5724, United States

  • Boston Medical Center & Boston University School of Medicine

    Boston, Massachusetts, 02118, United States

  • CHR de la Citadelle

    Liège, 4000, Belgium

  • CHU Guadeloupe

    Pointe à Pitre, Guadeloupe, 97139, France

  • CHU Hôpital Henri Mondor

    Créteil, Val-de-Marne, 94000, France

  • CHU Montreal

    Montreal, Quebec, H2X 3E4, Canada

  • Cambridge University Hospitals NHS Foundation Trust

    Cambridge, CB2 0QQ, United Kingdom

  • Children's Hospital Oakland

    Oakland, California, 94609, United States

  • Children's Hospital of Michigan

    Detroit, Michigan, 48201, United States

  • Children's National Hospital

    Washington D.C., District of Columbia, 20010-2916, United States

  • Clinique CHC MontLégia

    Liège, 4000, Belgium

  • Cure 4 The Kids Foundation, A Division of Roseman University of Health Sciences

    Las Vegas, Nevada, 89106, United States

  • East Carolina University - Brody School of Medicine

    Greenville, North Carolina, 27834, United States

  • Ente Ospedaliero Ospedali Galliera

    Genoa, Liguria, 16128, Italy

  • Erasmus MC

    Rotterdam, South Holland, 3015 GD, Netherlands

  • Evelina Children's Hospital

    London, City of London, SE1 7EH, United Kingdom

  • Gertrude's Children's Hospital

    Nairobi, 42325- 00100, Kenya

  • Guy's and St Thomas' NHS Foundation Trust

    London, SE1 7EH, United Kingdom

  • HEMORIO Instituto Nacional de Hematologia

    Rio de Janeiro, 20211-030, Brazil

  • HaEmek Medical Center

    Afula, 18101, Israel

  • Hacettepe Universitesi Tip Fakultesi Hastanesi

    Ankara, Adana, 06200, Turkey (Türkiye)

  • Hacettepe University

    Ankara, Adana, Turkey (Türkiye)

  • Hammersmith Hospital

    London, W12 0HS, United Kingdom

  • Hammoud Hospital University Medical Center

    Sidon, H96G+247, Lebanon

  • Hopitaux de La Timone

    Marseille, Bouches-du-Rhône, 13005, France

  • Hospital Das Clínicas da Faculdade de Medicina de Ribeirão Preto - USP

    Ribeirão Preto, São Paulo, 14051-140, Brazil

  • Hospital Sao Lucas Da Pontificia Universidade Catolica Do Rio Grande Do Sul (PUCRS)

    Porto Alegre, Rio Grande do Sul, 90619-900, Brazil

  • Hospital das Clínicas da Faculdade de Medicina da Universidad de São Paulo

    São Paulo, 05403-010, Brazil

  • Hospital de Clinicas de Porto Alegre (HCPA) - PPDS

    Porto Alegre, Rio Grande do Sul, 90035-903, Brazil

  • Hospital de Clínicas da Unicamp

    Campinas, São Paulo, 13083-878, Brazil

  • Hôpital Erasme

    Anderlecht, Brussels Capital, 1070, Belgium

  • Hôpital Européen Georges Pompidou

    Paris, Île-de-France Region, 75015, France

  • Hôpital Pellegrin, CHU de Bordeaux

    Bordeaux, Gironde, 33000, France

  • IRCCS Ospedale Pediatrico Bambino Gesù - INCIPIT - PIN

    Rome, Lazio, 00165, Italy

  • Institut Universitaire du Cancer de Toulouse - Oncopole

    Toulouse, Haute-Garonne, 31059, France

  • KEMRI CRDR Clinical Research Clinic Nairobi

    Nairobi, 00100, Kenya

  • KEMRI/CRDR Siaya Clinical Research Annex

    Nairobi, 00200, Kenya

  • Kaiser Permanente - Largo Medical Center

    Largo, Maryland, 20774-5374, United States

  • Kemri Usamru

    Kisumu, Western, 40100, Kenya

  • King Abdullah International Medical Research Center

    Riyadh, 1515 (KAIMRC), Saudi Arabia

  • King Khalid University Hospital

    Riyadh, Ar Riya, 11472, Saudi Arabia

  • King's College Hospital NHS Foundation Trust

    London, SE5 9RS, United Kingdom

  • Kondele Children's Hospital

    Kisumu, 40100, Kenya

  • LSU Health Sciences Center - Shreveport

    Shreveport, Louisiana, 71103-4228, United States

  • Lagos University Teaching Hospital

    Surulere, Lagos, 101014, Nigeria

  • Lifespan at Rhode Island Hospital

    Providence, Rhode Island, 02903, United States

  • Manchester Royal Infirmary, Manchester University NHS Foundation Trust

    Manchester, M13 9WL, United Kingdom

  • Massachusetts General Hospital

    Boston, Massachusetts, 02114-2621, United States

  • McGill University Health Center

    Montreal, Quebec, H4A 3J1, Canada

  • McMaster University - St. Joseph's Healthcare Hamilton

    Hamilton, Ontario, L8N 3Z5, Canada

  • MedStar Washington Hospital Center

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

  • Mississippi Center for Advanced Medicine

    Madison, Mississippi, 39110-6115, United States

  • Multihemo Servicos Medicos S/A

    Recife, Pernambuco, 50070-460, Brazil

  • National Heart Lung and Blood Institute

    Bethesda, Maryland, 20814, United States

  • National Hospital Abuja

    Abuja, Federal Capital Territory, 900271, Nigeria

  • Nini Hospital

    Tarablus, North Lebanon, 1434, Lebanon

  • Penn Medicine - University of Pennsylvania Health System

    Philadelphia, Pennsylvania, 19104-5127, United States

  • Praxis Pesquisa Medica

    Santo André, São Paulo, Brazil

  • Rambam Medical Center

    Haifa, Ḥeifā, 31096, Israel

  • Riley Hospital For Children

    Indianapolis, Indiana, 46202-5109, United States

  • Seattle Cancer Care Alliance, University of Washington

    Seattle, Washington, 98195, United States

  • Southern Specialty Clinic

    Flowood, Mississippi, 39232, United States

  • St. Christopher's Hospital for Children

    Philadelphia, Pennsylvania, 19134-1011, United States

  • Strathmore University

    Nairobi, 00200, Kenya

  • Sultan Qaboos University Hospital, Hematology Department, COM&HS

    Muscat, Musqal, H5QC+36M, Oman

  • Sylvester Comprehensive Cancer Center-Miami

    Miami, Florida, 33101, United States

  • Texas Children's Hospital

    Houston, Texas, 77030, United States

  • UCLA Health

    Los Angeles, California, 90095-1678, United States

  • Universitair Medisch Centrum Utrecht

    Utrecht, 3584 CX, Netherlands

  • Universitair Ziekenhuis Antwerpen

    Edegem, Brussels Capital, 2650, Belgium

  • University College London Hospitals (UCLH)

    London, WC1E 6BT, United Kingdom

  • University Health Network

    Toronto, Ontario, M5G2C4, Canada

  • University of Abuja Teaching Hospital

    Abuja, Federal Capital Territory, 900271, Nigeria

  • University of California San Diego

    La Jolla, California, 92037-1337, United States

  • University of Chicago Medical Center

    Chicago, Illinois, 60637-1443, United States

  • University of Connecticut Health Center

    Farmington, Connecticut, 06030-0001, United States

  • University of Michigan

    Ann Arbor, Michigan, 48109-5000, United States

  • University of Texas Health Science Center of Houston

    Houston, Texas, 77030-1501, United States

  • Universitätsklinikum Essen

    Essen, 45147, Germany

  • Universitätsklinikum Regensburg

    Regensburg, 93053, Germany

  • Victoria Biomedical Research Institute (VIBRI)

    Kisumu, 40100, Kenya

  • Virginia Commonwealth University

    Richmond, Virginia, 23298-5058, United States

  • ZAS Cadix

    Antwerp, 2030, Belgium

  • Ziv Medical Center

    Safed, Ḥeifā, 13100, Israel

More trials for these conditions

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