BioinvestGPT

BioinvestGPT ApS · Research use only

NCT04004065 (SRP-5051 in Duchenne Muscular Dystrophy): BVCT ex-ante prediction vs. reported outcome

On 11 Aug 2023, BioinvestGPT's BVCT predicted technical partial success and commercial failure for this trial. The 6 Nov 2024 readout is classified on the dashboard as True Negative (TN), a correct prediction. Recorded clinical-benefit conclusion: statistically partially significant (dystrophin expression) and commercially insufficient (weak efficacy).

Clinical Trial

NCTID
NCT04004065
Acronym
MOMENTUM
Drug Name
SRP-5051
Drug MoA
Menin-MLL1 inhibitor
Drug Modality
small molecule
Drug Class
First-In-Class
Therapeutic Area
Musculoskeletal Diseases
Indication
Duchenne Muscular Dystrophy (DMD)
Human Patients
62
Sponsor
Sarepta Therapeutics
Phase
Phase 2

Prediction of , index 1217

Prospective Prediction
Prediction Index
1217
Prediction Date
Commercial Prediction
FAILURE (commercially insufficient clinical benefit in 6MWD)
Technical Prediction
partial SUCCESS (statistically significant clinical benefit in dystrophin level)

Readout of

Prediction Validation
Readout Date
Prediction To Readout
453 days in advance
Readout Data Interpretation
SRP5051 resulted in a mean absolute dystrophin expression of 5.17% at week 28; yet its further development has been discontinued due to unremarkable benefit-risk data
Press Release
Additional Readout Data
Prediction Result
Prediction Accuracy
Correct Prediction
Clinical Benefit Conclusion
statistically partially significant (dystrophin expression) and commercially insufficient (weak efficacy)
Prediction Classification
True Negative (TN)

ClinicalTrials.gov record

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

Official title
A Phase 2, Two-Part, Multiple-Ascending-Dose Study of SRP-5051 for Dose Determination, Then Dose Expansion, in Patients With Duchenne Muscular Dystrophy Amenable to Exon 51-Skipping Treatment
Phase
Phase 2
Status
Terminated
Enrollment
62
Lead sponsor
Sarepta Therapeutics, Inc.
Conditions
Duchenne Muscular Dystrophy
Interventions
DRUG: Vesleteplirsen
Design
Randomized · Sequential · None
Primary purpose
Treatment
Start
2019-06-26
Primary completion
2023-10-30
Completion
2025-02-07
Primary outcomes
  • Part A: Incidence of Adverse Events (AEs) (Part A: Baseline up to 75 weeks)
  • Part B: Change From Baseline in Dystrophin Protein Level at Week 28 (Part B: Baseline, Week 28)

NCT04004065 on ClinicalTrials.gov

Return to the dashboardClinicalTrials.gov

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