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ACTIVE NOT RECRUITING
NCT05031780
PHASE2/PHASE3

A Study Evaluating the Efficacy and Safety of Mitapivat (AG-348) in Participants With Sickle Cell Disease (RISE UP)

Sponsor: Agios Pharmaceuticals, Inc.

View on ClinicalTrials.gov

Summary

This clinical trial is a Phase 2/3 study that will determine the recommended dose of mitapivat and evaluate the efficacy and safety of mitapivat in sickle cell disease by testing how well mitapivat works compared to placebo to increase the amount of hemoglobin in the blood and to reduce or prevent the occurrence of sickle cell pain crises. In addition, the long-term effect of mitapivat on efficacy and safety will be explored in an open-label extension portion.

Official title: A Phase 2/3, Double-Blind, Randomized, Placebo-Controlled, Multicenter Study to Evaluate the Efficacy and Safety of Mitapivat in Subjects With Sickle Cell Disease

Key Details

Gender

All

Age Range

16 Years - Any

Study Type

INTERVENTIONAL

Enrollment

286

Start Date

2022-02-11

Completion Date

2030-02

Last Updated

2026-03-30

Healthy Volunteers

No

Interventions

DRUG

Mitapivat

Mitapivat tablets

OTHER

Mitapivat-matching placebo

Placebo to match 50 mg or 100 mg tablets

OTHER

Mitapivat-matching placebo

Placebo to match 100 mg tablets

Locations (91)

University of California San Diego

La Jolla, California, United States

UCLA Health

Los Angeles, California, United States

Children's Hospital Oakland

Oakland, California, United States

University of Connecticut Health Center

Farmington, Connecticut, United States

Children's National Hospital

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

MedStar Washington Hospital Center

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

Sylvester Comprehensive Cancer Center-Miami

Miami, Florida, United States

University of Chicago Medical Center

Chicago, Illinois, United States

Riley Hospital For Children

Indianapolis, Indiana, United States

LSU Health Sciences Center - Shreveport

Shreveport, Louisiana, United States

National Heart Lung and Blood Institute

Bethesda, Maryland, United States

Kaiser Permanente - Largo Medical Center

Largo, Maryland, United States

Massachusetts General Hospital

Boston, Massachusetts, United States

Boston Children's Hospital

Boston, Massachusetts, United States

Boston Medical Center & Boston University School of Medicine

Boston, Massachusetts, United States

University of Michigan

Ann Arbor, Michigan, United States

Children's Hospital of Michigan

Detroit, Michigan, United States

Southern Specialty Clinic

Flowood, Mississippi, United States

Mississippi Center for Advanced Medicine

Madison, Mississippi, United States

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

Las Vegas, Nevada, United States

East Carolina University - Brody School of Medicine

Greenville, North Carolina, United States

Penn Medicine - University of Pennsylvania Health System

Philadelphia, Pennsylvania, United States

St. Christopher's Hospital for Children

Philadelphia, Pennsylvania, United States

Lifespan at Rhode Island Hospital

Providence, Rhode Island, United States

University of Texas Health Science Center of Houston

Houston, Texas, United States

Texas Children's Hospital

Houston, Texas, United States

Virginia Commonwealth University

Richmond, Virginia, United States

Seattle Cancer Care Alliance, University of Washington

Seattle, Washington, United States

Hôpital Erasme

Anderlecht, Brussels Capital, Belgium

Universitair Ziekenhuis Antwerpen

Edegem, Brussels Capital, Belgium

ZAS Cadix

Antwerp, Belgium

CHR de la Citadelle

Liège, Belgium

Clinique CHC MontLégia

Liège, Belgium

Multihemo Servicos Medicos S/A

Recife, Pernambuco, Brazil

Hospital de Clinicas de Porto Alegre (HCPA) - PPDS

Porto Alegre, Rio Grande do Sul, Brazil

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

Porto Alegre, Rio Grande do Sul, Brazil

Hospital de Clínicas da Unicamp

Campinas, São Paulo, Brazil

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

Ribeirão Preto, São Paulo, Brazil

Praxis Pesquisa Medica

Santo André, São Paulo, Brazil

HEMORIO Instituto Nacional de Hematologia

Rio de Janeiro, Brazil

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

São Paulo, Brazil

McMaster University - St. Joseph's Healthcare Hamilton

Hamilton, Ontario, Canada

University Health Network

Toronto, Ontario, Canada

CHU Montreal

Montreal, Quebec, Canada

McGill University Health Center

Montreal, Quebec, Canada

Hopitaux de La Timone

Marseille, Bouches-du-Rhône, France

Hôpital Pellegrin, CHU de Bordeaux

Bordeaux, Gironde, France

CHU Guadeloupe

Pointe à Pitre, Guadeloupe, France

Institut Universitaire du Cancer de Toulouse - Oncopole

Toulouse, Haute-Garonne, France

CHU Hôpital Henri Mondor

Créteil, Val-de-Marne, France

Hôpital Européen Georges Pompidou

Paris, Île-de-France Region, France

Universitätsklinikum Essen

Essen, Germany

Universitätsklinikum Regensburg

Regensburg, Germany

HaEmek Medical Center

Afula, Israel

Rambam Medical Center

Haifa, Ḥeifā, Israel

Ziv Medical Center

Safed, Ḥeifā, Israel

A.O.R.N. "A. Cardarelli"

Naples, Campania, Italy

AOU dell'Università degli Studi della Campania Luigi Vanvitelli

Naples, Campania, Italy

Azienda Ospedaliero Universitaria Di Modena Policlinico

Modena, Emilia-Romagna, Italy

IRCCS Ospedale Pediatrico Bambino Gesù - INCIPIT - PIN

Rome, Lazio, Italy

Ente Ospedaliero Ospedali Galliera

Genoa, Liguria, Italy

Azienda Ospedaliera Ospedali Riuniti Villa Sofia-Cervello

Palermo, Sicily, Italy

Kemri Usamru

Kisumu, Western, Kenya

Kondele Children's Hospital

Kisumu, Kenya

Victoria Biomedical Research Institute (VIBRI)

Kisumu, Kenya

KEMRI CRDR Clinical Research Clinic Nairobi

Nairobi, Kenya

KEMRI/CRDR Siaya Clinical Research Annex

Nairobi, Kenya

Strathmore University

Nairobi, Kenya

Gertrude's Children's Hospital

Nairobi, Kenya

American University of Beirut Medical Center

Beirut, Beyrouth, Lebanon

Nini Hospital

Tarablus, Mohafazat Liban-Nord, Lebanon

American University of Beirut Medical Center

Beirut, Lebanon

Hammoud Hospital University Medical Center

Sidon, Lebanon

Erasmus MC

Rotterdam, South Holland, Netherlands

Universitair Medisch Centrum Utrecht

Utrecht, Netherlands

National Hospital Abuja

Abuja, Federal Capital Territory, Nigeria

University of Abuja Teaching Hospital

Abuja, Federal Capital Territory, Nigeria

Lagos University Teaching Hospital

Surulere, Lagos, Nigeria

Sultan Qaboos University Hospital, Hematology Department, COM&HS

Muscat, Musqal, Oman

King Khalid University Hospital

Riyadh, Ar Riya, Saudi Arabia

King Abdullah International Medical Research Center

Riyadh, Saudi Arabia

Hacettepe Universitesi Tip Fakultesi Hastanesi

Ankara, Adana, Turkey (Türkiye)

Hacettepe University

Ankara, Adana, Turkey (Türkiye)

Acibadem Adana Hospital

Seyhan, Adana, Turkey (Türkiye)

Evelina Children's Hospital

London, City of London, United Kingdom

Cambridge University Hospitals NHS Foundation Trust

Cambridge, United Kingdom

Guy's and St Thomas' NHS Foundation Trust

London, United Kingdom

King's College Hospital NHS Foundation Trust

London, United Kingdom

Hammersmith Hospital

London, United Kingdom

University College London Hospitals (UCLH)

London, United Kingdom

Manchester Royal Infirmary, Manchester University NHS Foundation Trust

Manchester, United Kingdom