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Studies

Interventional Active not recruiting Phase 3

NCT04770779 on ClinicalTrials.gov

A Study Evaluating the Efficacy and Safety of Mitapivat in Participants With Transfusion-Dependent Alpha- or Beta-Thalassemia (α- or β-TDT)

Official title: A Phase 3, Double-Blind, Randomized, Placebo-Controlled, Multicenter Study Evaluating the Efficacy and Safety of Mitapivat in Subjects With Transfusion-Dependent Alpha- or Beta-Thalassemia (ENERGIZE-T)

  • Sponsor: Agios Pharmaceuticals (United States)
  • Enrollment: 258 (Actual)
  • Study start: 2021-11-30 (Actual)
  • Primary completion: 2024-04-11 (Actual)
  • Study completion: 2029-06-01 (Estimated)
  • Allocation: Randomized
  • InterventionModel: Parallel
  • Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
  • PrimaryPurpose: Treatment

Brief Summary

The primary objective of this study was to compare the effect of mitapivat versus placebo on transfusion burden in participants with α- or β-transfusion-dependent thalassemia.

Conditions

  • Transfusion-dependent Alpha-Thalassemia
  • Transfusion-dependent Beta-Thalassemia

Intervention / Treatment

  • Drug: Placebo Matching Mitapivat
  • Drug: Mitapivat
  • Drug: Mitapivat

Primary Outcome Measures

  • Double-blind Period: Percentage of Participants Who Achieved Transfusion Reduction Response (TRR) (Double-blind Period: Baseline through Week 48)
    TRR is defined as ≥50% reduction in transfused red blood cells (RBC) units with a reduction of ≥2 units of transfused RBCs in any consecutive 12-week period through Week 48 compared with baseline transfusion burden standardized to 12 weeks. Baseline transfusion burden standardized to 12 weeks= (12/24) × total number of RBC units transfused during 24-week period before the randomization date for participants randomized and not dosed or the start of study treatment for participants randomized and dosed. Participants withdrawn from the study before Week 12 (Day 85) were considered nonresponders.

Countries

Brazil, Bulgaria, Canada, Denmark, France, Germany, Greece, Italy, Lebanon, Malaysia, Netherlands, Saudi Arabia, Spain, Taiwan, Thailand, Turkey (Türkiye), United Arab Emirates, United Kingdom, United States

Locations and Investigators

  • Laiko General Hospital Of Athens, Athens, Greece
    • Maria, Dimopoulou
  • Nosokomeio Paidon I Agia Sofia, Athens, Greece
    • Antonis, Kattamis
  • Ippokratio General Hospital Of Thessaloniki, Thessaloniki, Greece
    • Efthymia, Vlachaki
  • General University Hospital Of Patras, Patras, Greece
  • Multiprofile Hospital For Active Treatment Dr Nikola Vasiliev AD, Kyustendil, Bulgaria
    • Desislava, Ilieva-Chiviyska
  • National Specialised Hospital For Active Treatment Of Haematological Diseases, Sofia, Bulgaria
    • Penka, Ganeva
  • University Multiprofile Hospital For Active Treatment Dr. Georgi Stranski EAD, Pleven, Bulgaria
    • Lachezar, Bogdanov
  • University Multiprofile Hospital For Active Treatment Saint Georgi EAD, Plovdiv, Bulgaria
    • Pencho, Georgiev
  • Umbal - Prof. D-R Stoyan Kirkovich AD, Stara Zagora, Bulgaria
    • Mariya, Todorova
  • Rigshospitalet, Copenhagen Oe, Denmark
    • Andreas, Glenthøj
  • Universitaet Leipzig, Leipzig, Germany
    • Carmen, Herling
  • Universitaetsklinikum Essen AöR, Essen, Germany
    • Ferras, Alashkar
  • Charite Universitaetsmedizin Berlin KöR, Berlin, Germany
    • Michaela, Schwarz
  • Hospices Civils De Lyon, Lyon Cedex 03, France
    • Giovanna, Cannas
  • Centre Hospitalier Regional De Marseille, Marseille, France
    • Jean, Mignard
  • Hopital Necker Enfants Malades, Paris, France
    • Laure, Joseph
  • Assistance Publique Hopitaux De Paris, Creteil, France
    • Pablo, Bartolucci
  • Universitair Medisch Centrum Utrecht, Utrecht, Netherlands
    • Eduard, van Beers
  • Academisch Medisch Centrum, Amsterdam, Netherlands
    • Barend Jacob, Biemond
  • Erasmus Universitair Medisch Centrum Rotterdam (Erasmus MC), Rotterdam, Netherlands
    • Anita, Rijneveld
  • Hospital Universitari Vall D Hebron, Barcelona, Spain
    • David, Beneitez Pastor
  • Hospital Universitario La Paz, Madrid, Spain
    • Ana, Mendoza Martinez
  • University Clinical Hospital Virgen De La Arrixaca, Murcia, Spain
    • Eduardo, Salido Fierrez
  • University Hospital Virgen Del Rocio S.L., Sevilla, Spain
    • Salvador, Payán-Pernía
  • Fondazione IRCCS Ca Granda Ospedale Maggiore Policlinico, Milan, Italy
    • Elena, Cassiniero
  • Azienda Socio Sanitaria Locale N. 8 Di Cagliari, Cagliari, Italy
    • Raffaella, Origa
  • Ente Ospedaliero Ospedali Galliera Di Genova, Genoa, Italy
    • Manuela, Balocco
  • Azienda Ospedaliero Universitaria Di Modena, Modena, Italy
    • Francesca, Ferrara
  • Azienda Sanitaria Locale Br, Brindisi, Italy
    • Annamaria, Pasanisi
  • Azienda Ospedaliera Universitaria Universita' Degli Studi Della Campania Luigi Vanvitelli, Naples, Italy
    • Silverio, Perrotta

Study Officials

  • Medical Affairs (Study Chair) — Agios Pharmaceuticals, Inc.