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NCT05481879 (DYNE-101 in Myotonic Dystrophy Type 1): BVCT ex-ante prediction vs. reported outcome

On 21 Nov 2023, BioinvestGPT's BVCT predicted technical success and commercial failure for this trial. The 3 Jan 2024 readout is classified on the dashboard as True Positive (TP), a correct prediction. Recorded clinical-benefit conclusion: statistically significant and commercially TBD.

Clinical Trial

NCTID
NCT05481879
Acronym
ACHIEVE
Drug Name
DYNE-101
Drug MoA
anti-DMPK siRNA
Drug Modality
small interfering RNA (siRNA) antisense oligonucleotide
Drug Class
First-In-Class
Therapeutic Area
Musculoskeletal Diseases
Indication
Myotonic Dystrophy Type 1
Human Patients
72
Sponsor
Dyne Therapeutics
Phase
Phase 1 Phase 2

Prediction of , index 1254

Prospective Prediction
Prediction Index
1254
Prediction Date
Commercial Prediction
FAILURE (commercially insufficient clinical benefit in 10MWR)
Technical Prediction
SUCCESS (statistically significant clinical benefit in toxicity)

Readout of

Prediction Validation
Readout Date
Prediction To Readout
43 days in advance
Readout Data Interpretation
40% DMPK knowddown and 3.8 second benefit in myotonia at 6 months; no report of key 10MWR functional endpoints.
Press Release
Prediction Result
Prediction Accuracy
Correct Prediction
Clinical Benefit Conclusion
statistically significant and commercially TBD
Prediction Classification
True Positive (TP)

ClinicalTrials.gov record

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

Official title
A Randomized, Placebo-Controlled, Multiple Ascending Dose Study Assessing Safety, Tolerability, Pharmacodynamics, Efficacy, and Pharmacokinetics of DYNE-101 Administered to Participants With Myotonic Dystrophy Type 1
Phase
Phase 1, Phase 2
Status
Active not recruiting
Enrollment
127
Lead sponsor
Dyne Therapeutics
Conditions
Myotonic Dystrophy Type 1 (DM1)
Interventions
DRUG: DYNE-101; DRUG: Placebo
Design
Randomized · Sequential · Quadruple
Primary purpose
Treatment
Start
2022-09-05
Primary completion
2029-07
Completion
2029-07
Primary outcomes
  • MAD Cohorts: Number of Participants With Treatment-Emergent Adverse Events (TEAEs) (Through study completion, up to Week 257)
  • Dose Expansion Cohorts: Change From Baseline in Myotonia as Measured by Video Hand Opening Time (vHOT) (Baseline up to Week 25)

NCT05481879 on ClinicalTrials.gov

Return to the dashboardClinicalTrials.gov

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