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NCT05243017 (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
NCT05243017
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
15
Sponsor
uniQure
Phase
Phase 1 Phase 2

Prediction of , index 908

Prospective Prediction
Prediction Index
908
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 Ib/II Randomized, Double-Blind 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
14
Lead sponsor
UniQure Biopharma B.V.
Conditions
Huntington Disease
Interventions
GENETIC: intra-striatal rAAV5-miHTT
Design
Randomized · Sequential · None
Primary purpose
Treatment
Start
2021-10-07
Primary completion
2029-03
Completion
2029-10-07
Primary outcomes
  • Evaluate the safety and tolerability of bilateral striatal delivery of AMT-130 as a total HTT gene lowering therapy in adult subjects with early manifest HD assessed by Adverse Events (6 months)
  • Evaluate the safety and tolerability of bilateral striatal delivery of AMT-130 as a total HTT gene lowering therapy in adult subjects with early manifest HD assessed by Vital Signs - Blood Pressure (6 months)
  • Evaluate the safety and tolerability of bilateral striatal delivery of AMT-130 as a total HTT gene lowering therapy in adult subjects with early manifest HD assessed by Vital Signs - Respiratory Rate (6 months)
  • Evaluate the safety and tolerability of bilateral striatal delivery of AMT-130 as a total HTT gene lowering therapy in adult subjects with early manifest HD assessed by Vital Signs - Heart Rate (6 months)
  • Evaluate the safety and tolerability of bilateral striatal delivery of AMT-130 as a total HTT gene lowering therapy in adult subjects with early manifest HD assessed by Electrocardiograms (6 months)

NCT05243017 on ClinicalTrials.gov

Return to the dashboardClinicalTrials.gov

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