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NCT06411288 (del-desiran in myotonic dystrophy type 1): BVCT ex-ante prediction vs. reported outcome

On 26 Jul 2026, BioinvestGPT's BVCT predicted technical failure and commercial failure for this trial. The 8 Sep 2026 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
NCT06411288
Acronym
HARBOR
Drug Name
del-desiran (AOC 1001)
Drug MoA
anti-TRF1 mAb conjugated with an anti-DMPK siRNA
Drug Modality
antibody-drug conjugate (ADC)
Drug Class
First-In-Class
Therapeutic Area
Musculoskeletal Diseases
Indication
myotonic dystrophy type 1
Human Patients
159
Sponsor
Novartis
Phase
Phase 3

Prediction of , index 2157

Prospective Prediction
Prediction Index
2157
Prediction Date
Commercial Prediction
FAILURE (commercially insufficient clinical benefit in vHOT and 10MWT slightly inferior to DYNE101 with RR > 1.2 and moderately inferior to PGN-EDODM1 with RR > 1.4)
Technical Prediction
FAILURE (statistically insignificant clinical benefit in vHOT and 10MWT compared with placebo)

Readout of

Prediction Validation
Readout Date
Prediction To Readout
44 days in advance
Readout Data Interpretation
Del-desiran did not meet its primary endpoint of vHOT
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 3 Randomized, Double-Blind, Placebo-Controlled, Global Study to Evaluate the Efficacy and Safety of Intravenous AOC 1001 for the Treatment of Myotonic Dystrophy Type 1
Phase
Phase 3
Status
Completed
Enrollment
159
Lead sponsor
Avidity Biosciences, Inc.
Conditions
DM1; Myotonic Dystrophy; Myotonic Dystrophy 1; Myotonia; Myotonic Dystrophy Type 1 (DM1); Dystrophy Myotonic; Myotonic Disorders; Steinert Disease; Steinert; Myotonic Muscular Dystrophy
Interventions
DRUG: AOC 1001 (del-desiran); DRUG: Placebo
Design
Randomized · Parallel · Quadruple
Primary purpose
Treatment
Start
2024-05-30
Primary completion
2026-07-23
Completion
2026-07-29
Primary outcomes
  • Hand function (Through Week 54)

NCT06411288 on ClinicalTrials.gov

Return to the dashboardClinicalTrials.gov

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