Medical Information Only. Always consult your healthcare provider before enrolling in any clinical trial.
NCT04770779 · ClinicalTrials.gov registry record · Phase 3
A Study Evaluating the Efficacy and Safety of Mitapivat in Participants With Transfusion-Dependent Alpha- or Beta-Thalassemia (α- or β-TDT)
A Phase 3 study of Transfusion-dependent Beta-Thalassemia and Transfusion-dependent Alpha-Thalassemia, sponsored by Agios Pharmaceuticals.
- Active
- Registry status
- Phase 3
- Development phase
- 258
- Enrollment target
- 20
- Study locations
NCT04770779: Active Phase 3 study of Transfusion-dependent Beta-Thalassemia and Transfusion-dependent Alpha-Thalassemia, sponsored by Agios Pharmaceuticals.
NCT04770779 is a Phase 3 study of Transfusion-dependent Beta-Thalassemia and Transfusion-dependent Alpha-Thalassemia that is active but no longer recruiting, run by Agios Pharmaceuticals. The registered enrollment target is 258 participants, above the 27-participant average among 3 other Transfusion-dependent Beta-Thalassemia trials with a reported enrollment target (856% higher). The trial reports 20 study locations across 11 states. According to ClinicalTrials.gov, the official US trial registry.
The verdict
NCT04770779, a Phase 3 study of Transfusion-dependent Beta-Thalassemia and Transfusion-dependent Alpha-Thalassemia, is active but no longer recruiting, sponsored by Agios Pharmaceuticals.
- ACTIVE NOT RECRUITING
- Registry status
- Phase 3
- Development phase
- 258 participants
- Enrollment target
- 20
- Study locations
Study 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.
Primary Outcome
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
Conditions Studied
Interventions
- DRUG Mitapivat
- DRUG Placebo Matching Mitapivat
Study Locations (20)
Other
- Hospital Das Clínicas da Faculdade de Medicina de Ribeirão Preto - USP - Ribeirão Preto
- GSH Banco de Sangue de São Paulo - São Paulo
- MHAT "Dr. Nikola Vasiliev" AD - Kyustendil
- UMHAT "Dr. Georgi Stranski" Pleven - Pleven
- UMHAT "Sveti Georgi" EAD - Plovdiv
- SHATHD Sofia - Sofia
- UMHAT "Prof. Dr. Stoyan Kirkovich" - Stara Zagora
- Rigshospitalet - Hovedstaden
California
- San Diego Hospital, UC San Diego Health - La Jolla
- Children's Hospital Oakland - Oakland
- Stanford Medicine - Palo Alto
Arizona
- Phoenix Children's Hospital - Phoenix
Massachusetts
- Boston Children's Hospital - Boston
Michigan
- Children's Hospital of Michigan - Detroit
New York
- Weill Cornell Medical Center - New York
North Carolina
- Duke University Medical Center - Durham
Pennsylvania
- Penn Medicine - University of Pennsylvania Health System - Philadelphia
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 258 participants |
| Start Date | 2021-11-30 |
| Est. Completion | 2029-06 |
| Phase | Phase 3 |
What the registry record for NCT04770779 still lists
NCT04770779 is an interventional study that assigns participants to a tested intervention. The registered 258 participants enrollment target is mid-sized for trials with a published cap, above the 27-participant average among 3 other Transfusion-dependent Beta-Thalassemia trials with a reported enrollment target (856% higher).
The record links to 2 conditions, with Transfusion-dependent Beta-Thalassemia appearing as the primary indexed condition, and to 2 interventions - of which Mitapivat is the first listed.
NCT04770779 names 20 study sites across 11 states, led by Other, California, Arizona.
Frequently Asked Questions
What is clinical trial NCT04770779 about?
NCT04770779 is a clinical study titled "A Study Evaluating the Efficacy and Safety of Mitapivat in Participants With Transfusion-Dependent Alpha- or Beta-Thalassemia (α- or β-TDT)". 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.
What is the current status of trial NCT04770779?
This trial is currently active not recruiting. It is a Phase 3 study. The enrollment target is 258 participants. The study started on 2021-11-30. Estimated completion is 2029-06.
What conditions does trial NCT04770779 study?
This clinical trial studies the following conditions: Transfusion-dependent Beta-Thalassemia, Transfusion-dependent Alpha-Thalassemia.
What interventions are being tested in trial NCT04770779?
The interventions under investigation include: Mitapivat (DRUG), Placebo Matching Mitapivat (DRUG).
Who is sponsoring clinical trial NCT04770779?
This trial is sponsored by Agios Pharmaceuticals, which has 44 total clinical trials registered on ClinicalTrials.gov.
Where is trial NCT04770779 being conducted?
This trial has 20 study locations across Arizona, California, Massachusetts, Michigan, New York. Contact the study sites directly through ClinicalTrials.gov for enrollment availability.
Learn More About Clinical Trials
Similar trials for Transfusion-dependent Beta-Thalassemia
Matched on the same primary condition, ranked to surface studies in the same phase first, then by recruiting status, no relevance scoring or editorial curation.
-
Long-term Follow-up of Subjects With Transfusion-Dependent β-Thalassemia (TDT) Treated With Ex Vivo Gene Therapy
ACTIVE NOT RECRUITING
-
EDIT-301 for Autologous Hematopoietic Stem Cell Transplant (HSCT) in Participants With Transfusion-Dependent Beta Thalassemia (TDT)
ACTIVE NOT RECRUITING · Phase 1
-
A Study to Assess the Safety, Tolerability, and Efficacy of ST-400 for Treatment of Transfusion-Dependent Beta-thalassemia (TDT)
COMPLETED · Phase 1
Nationwide trials with similar profiles
Cross-condition peers matched on enrollment target and registry start date, not the same-condition list above.
Similar enrollment target
- NCT03785288 · 258 participants · Phase 3
Vaginal Cuff Brachytherapy Fractionation Study
- NCT04643002 · 258 participants · Phase 1
Isatuximab in Combination With Novel Agents in RRMM - Master Protocol
- NCT04924322 · 258 participants · Phase 2
Catheter-Related Early Thromboprophylaxis With Enoxaparin Studies
- NCT05043012 · 258 participants · NA
Diagnostic Performance Of Non-Endorectal Coil Multiparametric Magnetic Resonance Imaging Of Prostate With Flexible AIR Coil (DoNEMAC Study)
Similar registry start date
- NCT05962346 · started 2026-12 · NA
Fetal Endoscopic Tracheal Occlusion for Congenital Diaphragmatic Hernia
- NCT07125183 · started 2026-12 · Phase 2
Study on Efficacy and Tolerability of Weekly Doxorubicin in Elderly Patients With Advanced or Metastatic Leiomyosarcoma
- NCT07292298 · started 2026-11 · Phase 2
Phase 2 Single-Arm Rectal Cancer Brachytherapy for Patients With Low-Lying Residual Adenocarcinoma After Total Neoadjuvant Therapy to Improve Organ Preservation Rates
- NCT04263285 · started 2026-10 · NA
Treatment of Depression Post-SCI