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NCT04770753 (Mitapivat in Non-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 3 Jan 2024 readout is classified on the dashboard as True Negative (TN), a correct prediction. Recorded clinical-benefit conclusion: statistically significant and commercially insufficient.

Clinical Trial

NCTID
NCT04770753
Acronym
ENERGIZE
Drug Name
Mitapivat
Drug MoA
small molecule PKR activator
Drug Modality
small molecule
Drug Class
First-In-Class
Therapeutic Area
Hematologic Diseases
Indication
Non-Transfusion-Dependent Alpha- or Beta-Thalassemia
Human Patients
194
Sponsor
Agios Pharmaceuticals
Phase
Phase 3

Prediction of , index 1110

Prospective Prediction
Prediction Index
1110
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
50 days in advance
Readout Data Interpretation
48.8% placebo-adjusted Hemoglobin response an increase of ≥1 g/dL in average hemoglobin concentrations from week 12 through week 24 compared with baseline for beta-thalassemia (vs 77.7% placebo-adjusted hemoglobin response achieved by the SoC in phase 2 beyond trial) 6MWT not met.
Press Release
Prediction Result
Prediction Accuracy
Correct Prediction
Clinical Benefit Conclusion
statistically significant and 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 Non-Transfusion-Dependent Alpha- or Beta-Thalassemia (ENERGIZE)
Phase
Phase 3
Status
Active not recruiting
Enrollment
194
Lead sponsor
Agios Pharmaceuticals, Inc.
Conditions
Non-Transfusion-dependent Alpha-Thalassemia; Non-Transfusion-dependent Beta-Thalassemia
Interventions
DRUG: Placebo Matching Mitapivat; DRUG: Mitapivat
Design
Randomized · Parallel · Quadruple
Primary purpose
Treatment
Start
2021-12-20
Primary completion
2023-11-13
Completion
2028-12
Results first posted
2025-01-24
Primary outcomes
  • Double-Blind Period: Percentage of Participants Who Achieved Hemoglobin (Hb) Response From Week 12 Through Week 24 Compared With Baseline (Double-Blind Period: Baseline up to Week 12 through Week 24)

NCT04770753 on ClinicalTrials.gov

Return to the dashboardClinicalTrials.gov

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