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NCT05031780 (Mitapivat in Sickle Cell Disease): BVCT ex-ante prediction vs. reported outcome

On 28 Jul 2025, BioinvestGPT's BVCT predicted technical partial success and commercial failure for this trial. The 19 Nov 2025 readout is classified on the dashboard as True Negative (TN), a correct prediction. Recorded clinical-benefit conclusion: statistically insignificant and commercially insufficient.

Clinical Trial

NCTID
NCT05031780
Acronym
RISE UP
Drug Name
Mitapivat (AG-348)
Drug MoA
pyruvate kinase activator
Drug Modality
small molecule
Drug Class
First-In-Class
Therapeutic Area
Hemic and Lymphatic Diseases
Indication
Sickle Cell Disease
Human Patients
286
Sponsor
Agios Pharmaceuticals
Phase
Phase 2 Phase 3

Prediction of , index 1109

Prospective Prediction
Prediction Index
1109
Prediction Date
Commercial Prediction
FAILURE (commercially insufficient clinical benefit in Hb response rate/SCPCs/SAEs slightly inferior to the SoC hydroxyurea)
Technical Prediction
partial SUCCESS (statistically maybe significant yet moderate clinical benefit in Hb response rate and SCPCs compared with placebo yet featuring a negative dose-response relationship and moderate toxicity)

Readout of

Prediction Validation
Readout Date
Prediction To Readout
114 days in advance
Readout Data Interpretation
mitapivat failed to meet the key primary efficacy endpoint of annualized rate of SCPCs (pain crises) or the key secondary efficacy endpoint PROMIS Fatigue
Press Release
Prediction Result
Prediction Accuracy
Correct Prediction
Clinical Benefit Conclusion
statistically insignificant 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 2/3, Double-Blind, Randomized, Placebo-Controlled, Multicenter Study to Evaluate the Efficacy and Safety of Mitapivat in Subjects With Sickle Cell Disease
Phase
Phase 2, Phase 3
Status
Active not recruiting
Enrollment
286
Lead sponsor
Agios Pharmaceuticals, Inc.
Conditions
Sickle Cell Disease
Interventions
DRUG: Mitapivat; OTHER: Mitapivat-matching placebo; OTHER: Mitapivat-matching placebo
Design
Randomized · Sequential · Quadruple
Primary purpose
Treatment
Start
2022-02-11
Primary completion
2025-10-30
Completion
2030-02
Primary outcomes
  • Phase 2: Percentage of Participants With Hemoglobin (Hb) Response (Week 12)
  • Phase 2: Percentage of Participants With Treatment-Emergent Adverse Events (AEs) and Treatment-Emergent Serious AEs (SAEs) (Up to Week 12)
  • Phase 3: Percentage of Participants With Hb Response (Week 52)
  • Phase 3: Annualized Rate of Sickle Cell Pain Crises (SCPCs) (Up to Week 52)

NCT05031780 on ClinicalTrials.gov

Return to the dashboardClinicalTrials.gov

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