- ICH GCP
- US Clinical Trials Registry
- Clinical Trial NCT05031780
A Study Evaluating the Efficacy and Safety of Mitapivat (AG-348) in Participants With Sickle Cell Disease (RISE UP)
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
Study Overview
Status
Conditions
Intervention / Treatment
Detailed Description
Study Type
Enrollment (Estimated)
Phase
- Phase 2
- Phase 3
Contacts and Locations
Study Contact
- Name: Agios Medical Affairs
- Phone Number: 833-228-8474
- Email: medinfo@agios.com
Study Locations
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Liège, Belgium, 4000
- Recruiting
- Chr de La Citadelle
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Liège, Belgium, 4000
- Recruiting
- Clinique CHC MontLégia
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Brussels
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Anderlecht, Brussels, Belgium, 1070
- Recruiting
- Hopital Erasme
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Antwerpen, Brussels, Belgium, 2060
- Recruiting
- ZNA Stuivenberg
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Edegem, Brussels, Belgium, 2650
- Recruiting
- Universitair Ziekenhuis Antwerpen
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Rio De Janeiro, Brazil, 20211-030
- Recruiting
- HEMORIO Instituto Nacional de Hematologia
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São Paulo, Brazil, 05403-010
- Recruiting
- Instituto do Cancer do Estado de São Paulo, Hospital das Clínicas da Faculdade de Medicina da Universidad de São Paulo
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Pernambuco
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Recife, Pernambuco, Brazil, 50070-460
- Not yet recruiting
- Multihemo Servicos Medicos S/A
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Rio Grande Do Sul
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Porto Alegre, Rio Grande Do Sul, Brazil, 90619-900
- Recruiting
- Hospital Sao Lucas Da Pontificia Universidade Catolica Do Rio Grande Do Sul (PUCRS)
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Porto Alegre, Rio Grande Do Sul, Brazil, 90035-903
- Not yet recruiting
- Hospital de Clinicas de Porto Alegre (HCPA) - PPDS
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São Paulo
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Campinas, São Paulo, Brazil, 13083-878
- Recruiting
- Hospital de Clinicas da UNICAMP
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Ribeirão Preto, São Paulo, Brazil, 14051-140
- Recruiting
- Hospital Das Clínicas da Faculdade de Medicina de Ribeirão Preto - USP
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Santo Andre, São Paulo, Brazil
- Active, not recruiting
- Praxis pesquisa médica
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Ontario
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Hamilton, Ontario, Canada, L8N 4A6
- Not yet recruiting
- McMaster University - St. Joseph's Healthcare Hamilton
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Toronto, Ontario, Canada, M5G2C4
- Recruiting
- University Health Network
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Quebec
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Montreal, Quebec, Canada, H3A 2B4
- Recruiting
- McGill University Health Center
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Montreal, Quebec, Canada, H2X 3E4
- Recruiting
- CHU Montreal
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Bouches-du-Rhône
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Marseille, Bouches-du-Rhône, France, 13005
- Recruiting
- Hopitaux de La Timone
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Gironde
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Bordeaux, Gironde, France, 33000
- Recruiting
- Hôpital Pellegrin, CHU de Bordeaux
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Guadeloupe
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Pointe-à-Pitre, Guadeloupe, France, 97159
- Recruiting
- CHU Guadeloupe
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Haute-Garonne
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Toulouse, Haute-Garonne, France, 31100
- Recruiting
- Institut Universitaire du Cancer de Toulouse - Oncopole
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Ile De France
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Paris, Ile De France, France, 75015
- Recruiting
- Hopital Europeen Georges Pompidou
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Val-de-Marne
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Créteil, Val-de-Marne, France, 94000
- Recruiting
- CHU Hôpital Henri Mondor
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Essen, Germany, 45147
- Recruiting
- Universitatsklinikum Essen
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Regensburg, Germany, 93053
- Recruiting
- Universitätsklinikum Regensburg
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Accra, Ghana
- Not yet recruiting
- Korle-Bu Teaching Hospital
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Kumasi, Ghana
- Not yet recruiting
- Komfo Anokye Teaching Hospital
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Afula, Israel, 1834111
- Active, not recruiting
- Haemek Medical Center
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Ḥeifā
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Haifa, Ḥeifā, Israel, 31096
- Recruiting
- Rambam Medical Center
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Safed, Ḥeifā, Israel, 13100
- Recruiting
- Ziv Medical Center
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Campania
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Napoli, Campania, Italy, 80131
- Active, not recruiting
- A.O.R.N. "A. Cardarelli"
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Napoli, Campania, Italy, 80138
- Active, not recruiting
- AOU dell'Università degli Studi della Campania Luigi Vanvitelli
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Napoli, Campania, Italy, 80131
- Not yet recruiting
- AORN A Cardarelli
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Emilia-Romagna
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Modena, Emilia-Romagna, Italy, 41100
- Recruiting
- Azienda Ospedaliero Universitaria Di Modena Policlinico
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Lazio
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Roma, Lazio, Italy, 00165
- Recruiting
- IRCCS Ospedale Pediatrico Bambino Gesù - INCIPIT - PIN
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Liguria
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Genova, Liguria, Italy, 16128
- Recruiting
- Ente Ospedaliero Ospedali Galliera
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Sicilia
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Palermo, Sicilia, Italy, 90146
- Not yet recruiting
- Azienda Ospedaliera Ospedali Riuniti Villa Sofia-Cervello
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Kisumu, Kenya, 40100
- Recruiting
- Kondele Children's Hospital
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Kisumu, Kenya, 40100
- Active, not recruiting
- Victoria Biomedical Research Institute (VIBRI)
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Nairobi, Kenya, 00200
- Recruiting
- Strathmore University
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Nairobi, Kenya, 00100
- Not yet recruiting
- KEMRI CRDR Clinical Research Clinic Nairobi
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Nairobi, Kenya, 00200
- Not yet recruiting
- KEMRI/CRDR Siaya Clinical Research Annex
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Nairobi, Kenya, 42325- 00100
- Not yet recruiting
- Gertrude's Children's Hospital
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Western
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Kisumu, Western, Kenya, 40100
- Not yet recruiting
- Kemri Usamru
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Sidon, Lebanon
- Recruiting
- Hammoud Hospital University Medical Center
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Beyrouth
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Beirut, Beyrouth, Lebanon
- Recruiting
- American University of Beirut Medical Center
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Liban Nord
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Tarablus, Liban Nord, Lebanon
- Recruiting
- Nini Hospital
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Utrecht, Netherlands, 3584 CX
- Recruiting
- Universitair Medisch Centrum Utrecht
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Zuid-Holland
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Rotterdam, Zuid-Holland, Netherlands, 3015 GD
- Recruiting
- Erasmus MC
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Calabar, Nigeria
- Not yet recruiting
- University of Calabar Teaching Hospital
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Enugu, Nigeria
- Not yet recruiting
- The University of Nigeria Teaching Hospiatal
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Kaduna, Nigeria
- Not yet recruiting
- Barau Dikko Teaching Hospital (BDTH), Kaduna
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Abuja Capital Territory
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Abuja, Abuja Capital Territory, Nigeria
- Active, not recruiting
- National Hospital Abuja
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Abuja, Abuja Capital Territory, Nigeria
- Active, not recruiting
- University of Abuja Teaching Hospital
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Lagos
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Suru-Lere, Lagos, Nigeria, 101014
- Active, not recruiting
- Lagos University Teaching Hospital
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Musqal
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Muscat, Musqal, Oman
- Recruiting
- Sultan Qaboos University Hospital, Hematology Department, COM&HS
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Riyadh, Saudi Arabia, 1515 (KAIMRC)
- Active, not recruiting
- King Abdullah International Medical Research Center
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Istanbul, Turkey, 34093
- Not yet recruiting
- Istanbul Universitesi Istanbul Tip Fakultesi Hastanesi
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Izmir, Turkey, 35040
- Not yet recruiting
- Ege Universitesi Tip Fakultesi Hastanesi
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Adana
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Ankara, Adana, Turkey
- Active, not recruiting
- Hacettepe University
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Seyhan, Adana, Turkey, 01130
- Active, not recruiting
- Acibadem Adana Hospital
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Içel
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Yenisehir, Içel, Turkey, 33110
- Not yet recruiting
- Mersin University Medical Faculty
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Cambridge, United Kingdom, CB2 0QQ
- Recruiting
- Cambridge University Hospitals NHS Foundation Trust
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London, United Kingdom, W12 0HS
- Recruiting
- Hammersmith Hospital
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London, United Kingdom, SE5 9RS
- Not yet recruiting
- King's College Hospital NHS Foundation Trust
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London, United Kingdom, SE1 7EH
- Not yet recruiting
- Guy's and St Thomas' NHS Foundation Trust
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London, United Kingdom, WC1E 6BT
- Not yet recruiting
- University College London Hospitals (UCLH)
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Manchester, United Kingdom, M13 9WL
- Recruiting
- Manchester Royal Infirmary, Manchester University NHS Foundation Trust
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City Of London
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London, City Of London, United Kingdom, SE1 7EH
- Recruiting
- Evelina Children's Hospital
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California
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La Jolla, California, United States, 92037-1337
- Recruiting
- University of California San Diego
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Los Angeles, California, United States, 90095-1678
- Recruiting
- UCLA Health
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Oakland, California, United States, 94609
- Not yet recruiting
- Children's Hospital and Research Center at Oakland
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Oakland, California, United States, 94609
- Active, not recruiting
- Children's Hospital Oakland
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Connecticut
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Farmington, Connecticut, United States, 06030-0001
- Not yet recruiting
- University of Connecticut Health Center
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New Haven, Connecticut, United States, 06520
- Not yet recruiting
- Yale University
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District of Columbia
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Washington, District of Columbia, United States, 20010
- Not yet recruiting
- MedStar Washington Hospital Center
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Washington, District of Columbia, United States, 20010-2916
- Recruiting
- Children's National Hospital
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Florida
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Miami, Florida, United States, 33101
- Not yet recruiting
- Sylvester Comprehensive Cancer Center-Miami
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Georgia
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Atlanta, Georgia, United States, 30342-3283
- Not yet recruiting
- Children's Healthcare of Atlanta
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Illinois
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Chicago, Illinois, United States, 60637-1443
- Not yet recruiting
- University of Chicago Medical Center
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Indiana
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Indianapolis, Indiana, United States, 46202-5109
- Not yet recruiting
- Riley Hospital for Children
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Louisiana
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Shreveport, Louisiana, United States, 71103-4228
- Not yet recruiting
- LSU Health Sciences Center - Shreveport
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Maryland
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Bethesda, Maryland, United States, 20814
- Recruiting
- National Heart Lung and Blood Institute
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Largo, Maryland, United States, 20774-5374
- Recruiting
- Kaiser Permanente - Largo Medical Center
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Massachusetts
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Boston, Massachusetts, United States, 02114-2621
- Active, not recruiting
- Massachusetts General Hospital
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Boston, Massachusetts, United States, 02118
- Recruiting
- Boston Medical Center & Boston University School of Medicine
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Boston, Massachusetts, United States, 02115-5724
- Not yet recruiting
- Boston Children's Hospital
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Michigan
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Ann Arbor, Michigan, United States, 48109-5000
- Recruiting
- University of Michigan
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Detroit, Michigan, United States, 48201
- Recruiting
- Children's Hospital of Michigan
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Mississippi
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Madison, Mississippi, United States, 39110-6115
- Recruiting
- Mississippi Center for Advanced Medicine
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Nevada
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Las Vegas, Nevada, United States, 89106
- Recruiting
- Cure 4 The Kids Foundation, A Division of Roseman University of Health Sciences
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North Carolina
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Greenville, North Carolina, United States, 27834
- Recruiting
- East Carolina University - Brody School of Medicine
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Ohio
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Cleveland, Ohio, United States, 44195-0001
- Not yet recruiting
- The Cleveland Clinic Foundation
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Pennsylvania
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Hershey, Pennsylvania, United States, 17033
- Active, not recruiting
- Penn State Health Milton S. Hershey Medical Center
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Philadelphia, Pennsylvania, United States, 19134-1011
- Recruiting
- St. Christopher's Hospital for Children
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Philadelphia, Pennsylvania, United States, 19104-5127
- Active, not recruiting
- Penn Medicine - University of Pennsylvania Health System
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Rhode Island
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Providence, Rhode Island, United States, 02903
- Not yet recruiting
- Lifespan at Rhode Island Hospital
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Texas
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Houston, Texas, United States, 77030
- Recruiting
- Texas Children's Hospital
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Houston, Texas, United States, 77030-1501
- Recruiting
- University of Texas Health Science Center of Houston
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Virginia
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Richmond, Virginia, United States, 23298-5058
- Recruiting
- Virginia Commonwealth University
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Washington
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Seattle, Washington, United States, 98195
- Recruiting
- Seattle Cancer Care Alliance, University of Washington
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Participation Criteria
Eligibility Criteria
Ages Eligible for Study
Accepts Healthy Volunteers
Description
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).
Study Plan
How is the study designed?
Design Details
- Primary Purpose: Treatment
- Allocation: Randomized
- Interventional Model: Sequential Assignment
- Masking: Quadruple
Arms and Interventions
Participant Group / Arm |
Intervention / Treatment |
---|---|
Experimental: Phase 2: Mitapivat 50 mg BID
Double-blind Period: Mitapivat 50 milligrams (mg) twice daily (BID) for 12 weeks.
|
Mitapivat tablets
Other Names:
|
Experimental: Phase 2: Mitapivat 100 mg BID
Double-blind Period: Mitapivat 100 mg BID for 12 weeks.
|
Mitapivat tablets
Other Names:
|
Placebo Comparator: Phase 2: Placebo
Double-blind Period: Mitapivat-matching placebo for 12 weeks.
|
Placebo to match 50 mg or 100 mg tablets
Placebo to match 100 mg tablets
|
Experimental: Phase 2: Open-Label Extension Period
Participants who received mitapivat 50mg BID in the double-blind period may choose to receive mitapivat 50mg BID for 216 weeks after. Participants who received mitapivat 100mg BID in the double-blind period may choose to receive mitapivat 100 mg BID for 216 weeks after. Participants who received mitapivat-matching placebo in the double-blind period, may be randomized to receive either mitapivat 50 mg or 100 mg BID for 216 weeks after. |
Mitapivat tablets
Other Names:
|
Experimental: Phase 3: Mitapivat 100 mg BID
Double-blind Period: Mitapivat 100 mg BID for 52 weeks.
|
Mitapivat tablets
Other Names:
|
Placebo Comparator: Phase 3: Placebo
Double-blind Period: Mitapivat-matching placebo for 52 weeks.
|
Placebo to match 50 mg or 100 mg tablets
Placebo to match 100 mg tablets
|
Experimental: Phase 3: Open-Label Extension Period
Participants may choose to receive mitapivat 100 mg BID for 216 weeks after the Double-blind Period. Participants who received mitapivat-matching placebo in the double-blind period, may choose to receive mitapivat 100 mg BID for 216 weeks after the Double-blind Period. |
Mitapivat tablets
Other Names:
|
What is the study measuring?
Primary Outcome Measures
Outcome Measure |
Time Frame |
---|---|
Phase 2: Percentage of Participants With Hemoglobin (Hb) Response
Time Frame: Week 12
|
Week 12
|
Phase 2: Percentage of Participants With Treatment-Emergent Adverse Events (AEs) and Treatment-Emergent Serious AEs (SAEs)
Time Frame: Up to Week 12
|
Up to Week 12
|
Phase 3: Percentage of Participants With Hb Response
Time Frame: Week 52
|
Week 52
|
Phase 3: Annualized Rate of Sickle Cell Pain Crises (SCPCs)
Time Frame: Up to Week 52
|
Up to Week 52
|
Secondary Outcome Measures
Outcome Measure |
Time Frame |
---|---|
Phase 2: Change From Baseline in Hb Concentration
Time Frame: Baseline, Week 10 up to Week 12
|
Baseline, Week 10 up to Week 12
|
Phase 2: Change From Baseline in Indirect Bilirubin
Time Frame: Baseline, Week 10 up to Week 12
|
Baseline, Week 10 up to Week 12
|
Phase 2: Change From Baseline in Lactate Dehydrogenase (LDH)
Time Frame: Baseline, Week 10 up to Week 12
|
Baseline, Week 10 up to Week 12
|
Phase 2: Change From Baseline in Absolute Reticulocytes Count
Time Frame: Baseline, Week 10 up to Week 12
|
Baseline, Week 10 up to Week 12
|
Phase 2: Change From Baseline in Percent Reticulocytes
Time Frame: Baseline, Week 10 up to Week 12
|
Baseline, Week 10 up to Week 12
|
Phase 2: Change From Baseline in Erythropoietin
Time Frame: Baseline, Week 10 up to Week 12
|
Baseline, Week 10 up to Week 12
|
Phase 2: Change From Baseline in Patient-Reported Outcomes Measurement Information System® (PROMIS®) Fatigue 13a Short Form (SF) Score
Time Frame: Baseline, Week 10 up to Week 12
|
Baseline, Week 10 up to Week 12
|
Phase 2: Annualized Rate of SCPCs
Time Frame: Up to Week 12
|
Up to Week 12
|
Phase 2: Pharmacokinetic/Pharmacodynamic Relationship: Evaluate the Exposure of Mitapivat to the Change in Adenosine Triphosphate (ATP) and 2,3-Diphosphoglycerate (2,3-DPG)
Time Frame: Day 1 up to Week 8
|
Day 1 up to Week 8
|
Phase 2: Mitapivat Concentration Over Time
Time Frame: Day 1 up to Week 8
|
Day 1 up to Week 8
|
Phase 2: Mitapivat Area Under the Concentration
Time Frame: Day 1 up to Week 8
|
Day 1 up to Week 8
|
Phase 2: Mitapivat Maximum (Peak) Concentration
Time Frame: Day 1 up to Week 8
|
Day 1 up to Week 8
|
Phase 3: Change From Baseline in Hb Concentration
Time Frame: Baseline, Week 24 up to Week 52
|
Baseline, Week 24 up to Week 52
|
Phase 3: Change From Baseline in Indirect Bilirubin
Time Frame: Baseline, Week 24 up to Week 52
|
Baseline, Week 24 up to Week 52
|
Phase 3: Change From Baseline in Percent Reticulocytes
Time Frame: Baseline, Week 24 up to Week 52
|
Baseline, Week 24 up to Week 52
|
Phase 3: Change From Baseline in PROMIS® Fatigue 13a SF Scores
Time Frame: Baseline, Week 24 up to Week 52
|
Baseline, Week 24 up to Week 52
|
Phase 3: Annualized Frequency of Hospitalizations for SCPC
Time Frame: Up to Week 52
|
Up to Week 52
|
Phase 3: Change From Baseline in LDH Concentration
Time Frame: Baseline, Week 24 up to Week 52
|
Baseline, Week 24 up to Week 52
|
Phase 3: Change From Baseline in Absolute Reticulocytes
Time Frame: Baseline, Week 24 up to Week 52
|
Baseline, Week 24 up to Week 52
|
Phase 3: Change From Baseline in Erythropoietin
Time Frame: Baseline, Week 24 up to Week 52
|
Baseline, Week 24 up to Week 52
|
Phase 3: Percentage of Participants With Improvement in the Patient Global Impression of Severity (PGIS) -Fatigue
Time Frame: Baseline, Weeks 24, 28, 40, and 52
|
Baseline, Weeks 24, 28, 40, and 52
|
Phase 3: Percentage of Participants With Improvement in the Patient Global Impression of Change (PGIC) -Fatigue
Time Frame: Baseline, Weeks 24, 28, 40, and 52
|
Baseline, Weeks 24, 28, 40, and 52
|
Phase 3: Time to First SCPC
Time Frame: Up to Week 52
|
Up to Week 52
|
Phase 3: Time to Second SCPC
Time Frame: Up to Week 52
|
Up to Week 52
|
Phase 3: Annualized Rate of Hospitalization Days for SCPC
Time Frame: Up to Week 52
|
Up to Week 52
|
Phase 3: Annualized Rate of Emergency Room Visits for SCPC
Time Frame: Up to Week 52
|
Up to Week 52
|
Phase 3: Change From Baseline in 6-Minute Walk Test (6MWT)
Time Frame: Baseline, Week 52
|
Baseline, Week 52
|
Phase 3: Change From Baseline in PROMIS Pain Intensity
Time Frame: Baseline, Week 24 and 52
|
Baseline, Week 24 and 52
|
Phase 3: Change From Baseline in Adult Sickle Cell Quality of Life Measurement Information System (ASCQ-Me) Pain Impact
Time Frame: Baseline, Week 24 and 52
|
Baseline, Week 24 and 52
|
Phase 3: PGIC of Pain
Time Frame: Baseline, Week 52
|
Baseline, Week 52
|
Phase 3: Change From Baseline in PGIS of Pain
Time Frame: Baseline, Week 52
|
Baseline, Week 52
|
Phase 3: Percentage of Participants With Treatment-Emergent Adverse Events (AEs) and Treatment-Emergent Serious AEs (SAEs)
Time Frame: Up to 56 weeks
|
Up to 56 weeks
|
Phase 3: Pharmacokinetic/Pharmacodynamic Relationship: Evaluate the Exposure of Mitapivat to the Change in ATP and 2,3-DPG Levels
Time Frame: Day 1 up to Week 40
|
Day 1 up to Week 40
|
Phase 3: Mitapivat Concentration Over Time
Time Frame: Day 1 up to Week 40
|
Day 1 up to Week 40
|
Phase 3: Mitapivat Area Under the Concentration Curve
Time Frame: Day 1 up to Week 40
|
Day 1 up to Week 40
|
Phase 3: Mitapivat Maximum (Peak) Concentration
Time Frame: Day 1 up to Week 40
|
Day 1 up to Week 40
|
Collaborators and Investigators
Sponsor
Study record dates
Study Major Dates
Study Start (Actual)
Primary Completion (Estimated)
Study Completion (Estimated)
Study Registration Dates
First Submitted
First Submitted That Met QC Criteria
First Posted (Actual)
Study Record Updates
Last Update Posted (Actual)
Last Update Submitted That Met QC Criteria
Last Verified
More Information
Terms related to this study
Additional Relevant MeSH Terms
Other Study ID Numbers
- AG348-C-020
- 2021-001674-34 (EudraCT Number)
Plan for Individual participant data (IPD)
Plan to Share Individual Participant Data (IPD)?
Drug and device information, study documents
Studies a U.S. FDA-regulated drug product
Studies a U.S. FDA-regulated device product
This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.
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Agios Pharmaceuticals, Inc.Active, not recruitingTransfusion-dependent Alpha-Thalassemia | Transfusion-dependent Beta-ThalassemiaSpain, Taiwan, Thailand, United States, France, Canada, Malaysia, Germany, Netherlands, Bulgaria, United Kingdom, Turkey, Italy, Greece, United Arab Emirates, Brazil, Denmark, Lebanon, Saudi Arabia
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Agios Pharmaceuticals, Inc.Active, not recruitingNon-Transfusion-dependent Alpha-Thalassemia | Non-Transfusion-dependent Beta-ThalassemiaSpain, Taiwan, Thailand, United Kingdom, Malaysia, United States, Netherlands, Bulgaria, Turkey, Italy, Canada, Brazil, France, United Arab Emirates, Denmark, Greece, Lebanon, Saudi Arabia
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Agios Pharmaceuticals, Inc.Active, not recruitingPediatric Pyruvate Kinase Deficiency | Pediatric Hemolytic AnemiaUnited States, Spain, Denmark, Turkey, Netherlands, Czechia, United Kingdom, Germany, Canada, Switzerland
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EuroBloodNet AssociationNot yet recruitingHereditary Red Blood Cell Disorder (Disorder)Denmark, Netherlands
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Agios Pharmaceuticals, Inc.CompletedHealthy VolunteersUnited States
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Agios Pharmaceuticals, Inc.RecruitingPediatric Pyruvate Kinase Deficiency | Pediatric Hemolytic AnemiaUnited States, Spain, Germany, Netherlands, Canada, France, Israel, Switzerland, Italy
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Agios Pharmaceuticals, Inc.Not yet recruitingSickle Cell Disease | Nephropathy
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Agios Pharmaceuticals, Inc.Enrolling by invitationAnemia, Hemolytic | Pyruvate Kinase DeficiencyJapan