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NCT05747924 (AOC 1020 in Facioscapulohumeral Muscular Dystrophy): BVCT ex-ante prediction vs. reported outcome

On 30 Nov 2023, BioinvestGPT's BVCT predicted technical partial success and commercial failure for this trial. The 12 Jun 2024 readout is classified on the dashboard as True Negative (TN), a correct prediction. Recorded clinical-benefit conclusion: statistically significant and commercially insufficient (weak functional efficacy).

Clinical Trial

NCTID
NCT05747924
Acronym
FORTITUDE
Drug Name
AOC 1020
Drug MoA
anti-DUX4 siRNA oligonucleotide
Drug Modality
small interfering RNA (siRNA) antisense oligonucleotide
Drug Class
First-In-Class
Therapeutic Area
Musculoskeletal Diseases
Indication
Facioscapulohumeral Muscular Dystrophy (FSHD)
Human Patients
72
Sponsor
Avidity Biosciences
Phase
Phase 1 Phase 2

Prediction of , index 1001

Prospective Prediction
Prediction Index
1001
Prediction Date
Commercial Prediction
FAILURE (commercially insufficient clinical benefit)
Technical Prediction
partial SUCCESS (statistically maybe significant yet weak clinical benefit)

Readout of

Prediction Validation
Readout Date
Prediction To Readout
195 days in advance
Readout Data Interpretation
50% mean reduction in DUX4-targeted genes; yet with statistically insignificant trends of functional improvement.
Press Release
Prediction Result
Prediction Accuracy
Correct Prediction
Clinical Benefit Conclusion
statistically significant and commercially insufficient (weak functional 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, Phase 1/2 Study to Evaluate the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics, and Exploratory Efficacy of AOC 1020 Administered Intravenously to Participants With Facioscapulohumeral Muscular Dystrophy (FSHD)
Phase
Phase 1, Phase 2
Status
Completed
Enrollment
90
Lead sponsor
Avidity Biosciences, Inc.
Conditions
FSHD; FSHD1; FSHD2; FMD; FMD2; Fascioscapulohumeral Muscular Dystrophy; Fascioscapulohumeral Muscular Dystrophy Type 1; Fascioscapulohumeral Muscular Dystrophy Type 2; Dystrophies, Facioscapulohumeral Muscular; Dystrophy, Facioscapulohumeral Muscular; Facioscapulohumeral Muscular Dystrophy 1; Facioscapulohumeral Muscular Dystrophy 2; Facio-Scapulo-Humeral Dystrophy; Atrophy, Facioscapulohumeral; Atrophies, Facioscapulohumeral; Facioscapulohumeral Atrophy; Muscular Dystrophies; Muscular Dystrophy, Facioscapulohumeral; FSH Muscular Dystrophy; Landouzy Dejerine Dystrophy; Landouzy-Dejerine Muscular Dystrophy; Dystrophies, Landouzy-Dejerine; Dystrophy, Landouzy-Dejerine; Landouzy-Dejerine Syndrome; Muscular Dystrophy, Landouzy Dejerine; Progressive Muscular Dystrophy; FSH
Interventions
DRUG: AOC 1020; DRUG: Placebo
Design
Randomized · Sequential · Quadruple
Primary purpose
Treatment
Start
2023-04-04
Primary completion
2026-03-18
Completion
2026-03-20
Primary outcomes
  • Incidence of treatment-emergent adverse events (Cohorts A & B) (Through study completion, up to Day 365)
  • Change in plasma KHDC1L (Part C) (Across months 3 to 12)

NCT05747924 on ClinicalTrials.gov

Return to the dashboardClinicalTrials.gov

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