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NCT05524883 (DYNE-251 in Duchenne Muscular Dystrophy): BVCT ex-ante prediction vs. reported outcome

On 21 Nov 2023, BioinvestGPT's BVCT predicted technical partial success and commercial failure for this trial. The 3 Sep 2024 readout is classified on the dashboard as True Negative (TN), a correct prediction. Recorded clinical-benefit conclusion: statistically partially significant (dystrophin expression) and partially insignificant (functional endpoints) and commercially probably insufficient (weak efficacy).

Clinical Trial

NCTID
NCT05524883
Acronym
DELIVER
Drug Name
DYNE-251
Drug MoA
dystrophin exon 51 skipping Fab
Drug Modality
siRNA
Drug Class
First-In-Class
Therapeutic Area
Musculoskeletal Diseases
Indication
Duchenne Muscular Dystrophy (DMD)
Human Patients
88
Sponsor
Dyne Therapeutics
Phase
Phase 1 Phase 2

Prediction of , index 1253

Prospective Prediction
Prediction Index
1253
Prediction Date
Commercial Prediction
FAILURE (commercially insufficient clinical benefit in 6MWD)
Technical Prediction
partial SUCCESS (statistically significant clinical benefit in dystrophin protein level); partial FAILURE (statistically insignificant clinical benefit in NSAA/10MRW/FVC)

Readout of

Prediction Validation
Readout Date
Prediction To Readout
287 days in advance
Readout Data Interpretation
a mean absolute dystrophin expression of 3.71% of normal; yet weak functional efficacy(change from baseline in SV95C only meet EMA-defined minimal clinically important difference (MCID); whereas other functional efficacy endpoints NSAA/10MWR probably didn’t reach MCID).
Press Release
Prediction Result
Prediction Accuracy
Correct Prediction
Clinical Benefit Conclusion
statistically partially significant (dystrophin expression) and partially insignificant (functional endpoints) and commercially probably insufficient (weak efficacy)
Prediction Classification
True Negative (TN)

ClinicalTrials.gov record

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

Official title
A Randomized, Double-Blind, Placebo-Controlled, Multiple Ascending Dose Study Assessing Safety, Tolerability, Pharmacodynamics, Efficacy, and Pharmacokinetics of DYNE-251 Administered to Participants With Duchenne Muscular Dystrophy Amenable to Exon 51 Skipping
Phase
Phase 1, Phase 2
Status
Active not recruiting
Enrollment
86
Lead sponsor
Dyne Therapeutics
Conditions
Duchenne Muscular Dystrophy (DMD)
Interventions
DRUG: DYNE-251; DRUG: Placebo
Design
Randomized · Sequential · Quadruple
Primary purpose
Treatment
Start
2022-08-12
Primary completion
2031-09
Completion
2031-09
Primary outcomes
  • Number of Participants With Treatment-Emergent Adverse Events (TEAEs) (Through study completion, up to Week 337)
  • Change From Baseline in Dystrophin Protein Levels in Muscle Tissue at Week 25 (Baseline, Week 25)

NCT05524883 on ClinicalTrials.gov

Return to the dashboardClinicalTrials.gov

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