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NCT04120493 (AMT-130 in Early Manifest Huntington's Disease): BVCT ex-ante prediction vs. reported outcome

On 13 May 2024, BioinvestGPT's BVCT predicted technical partial success and commercial failure for this trial. The 9 Jul 2024 readout is classified on the dashboard as True Negative (TN), a correct prediction. Recorded clinical-benefit conclusion: still statistically maybe significant (not strictly placebo-controlled) and commercially insufficient (no motor cognitive function improvement).

Clinical Trial

NCTID
NCT04120493
Drug Name
AMT-130
Drug MoA
AAV gene therapy that delivers anti-HTT miRNA
Drug Modality
gene therapy
Drug Class
First-In-Class
Therapeutic Area
Nervous System Diseases
Indication
Early Manifest Huntington's Disease
Human Patients
36
Sponsor
uniQure
Phase
Phase 1 Phase 2

Prediction of , index 909

Prospective Prediction
Prediction Index
909
Prediction Date
Commercial Prediction
FAILURE (commercially insufficient clinical benefit in UHDRS)
Technical Prediction
partial SUCCESS (statistically maybe significant yet very weak clinical benefit in UHDRS)

Readout of

Prediction Validation
Readout Date
Prediction To Readout
57 days in advance
Readout Data Interpretation
externally statistically significant less cUHDRS (-0.2 vs -1 at Month 24 for high-dose only (p=0.007) but not low-dose (p=0.21); no statistical improvement in motor or cognitive function even for high-dose group.
Press Release
Prediction Result
Prediction Accuracy
Correct Prediction
Clinical Benefit Conclusion
still statistically maybe significant (not strictly placebo-controlled) and commercially insufficient (no motor cognitive function improvement)
Prediction Classification
True Negative (TN)

ClinicalTrials.gov record

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

Official title
A Phase 1/2, Randomized, Double-Blind, Sham Control and Open-Label Study to Explore Safety, Tolerability, and Efficacy Signals of Multiple Doses of Striatally-Administered rAAV5-miHTT Total Huntingtin Gene (HTT) Lowering Therapy (AMT-130) in Early Manifest Huntington's Disease
Phase
Phase 1, Phase 2
Status
Active not recruiting
Enrollment
43
Lead sponsor
UniQure Biopharma B.V.
Conditions
Huntington's Disease
Interventions
GENETIC: intra-striatal rAAV5-miHTT; OTHER: Imitation (sham) surgery
Design
Non randomized · Sequential · None
Primary purpose
Treatment
Start
2019-09-06
Primary completion
2029-06
Completion
2029-12
Primary outcomes
  • Number and type of Adverse Events (AE) (12 months (Cohorts 1 & 2) and 12 months (Cohort 3))

NCT04120493 on ClinicalTrials.gov

Return to the dashboardClinicalTrials.gov

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