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NCT04770779 (Mitapivat in Transfusion-Dependent Alpha- or Beta-Thalassemia): BVCT ex-ante prediction vs. reported outcome

On 14 Nov 2023, BioinvestGPT's BVCT predicted technical failure and commercial failure for this trial. The 8 Dec 2024 readout is classified on the dashboard as True Negative (TN), a correct prediction. Recorded clinical-benefit conclusion: statistically significant yet commercially insufficient.

Clinical Trial

NCTID
NCT04770779
Acronym
ENERGIZE-T
Drug Name
Mitapivat
Drug MoA
small molecule PKR activator
Drug Modality
small molecule
Drug Class
First-In-Class
Therapeutic Area
Hemic and Lymphatic Diseases
Indication
Transfusion-Dependent Alpha- or Beta-Thalassemia (α- or β-TDT)
Human Patients
258
Sponsor
Agios Pharmaceuticals
Phase
Phase 3

Prediction of , index 1111

Prospective Prediction
Prediction Index
1111
Prediction Date
Commercial Prediction
FAILURE (commercially insufficient clinical benefit)
Technical Prediction
FAILURE (statistically insignificant clinical benefit)

Readout of

Prediction Validation
Readout Date
Prediction To Readout
390 days in advance
Readout Data Interpretation
all endpoints met with 30.4% of patients achieved a transfusion reduction response (50% reduction during any 12-week period); compared to 12.6% of patients in the placebo arm with 2-sided p= 0.0003 (vs 50% achieved by Reblozyl in phase 3 BELIEVE trial NCT02604433) hepatocellular toxicity identified as a potential risk
Press Release
Prediction Result
Prediction Accuracy
Correct Prediction
Clinical Benefit Conclusion
statistically significant yet commercially insufficient
Prediction Classification
True Negative (TN)

ClinicalTrials.gov record

ClinicalTrials.gov record, retrieved 2026-09-24. Third-party data.

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)
Phase
Phase 3
Status
Active not recruiting
Enrollment
258
Lead sponsor
Agios Pharmaceuticals, Inc.
Conditions
Transfusion-dependent Alpha-Thalassemia; Transfusion-dependent Beta-Thalassemia
Interventions
DRUG: Placebo Matching Mitapivat; DRUG: Mitapivat
Design
Randomized · Parallel · Quadruple
Primary purpose
Treatment
Start
2021-11-30
Primary completion
2024-04-11
Completion
2029-06
Results first posted
2025-05-25
Primary outcomes
  • Double-blind Period: Percentage of Participants Who Achieved Transfusion Reduction Response (TRR) (Double-blind Period: Baseline through Week 48)

NCT04770779 on ClinicalTrials.gov

Return to the dashboardClinicalTrials.gov

How to read these records: evidence · methodology · all trials