BioinvestGPT

BioinvestGPT ApS · Research use only

NCT05358717 (PTC518 in Huntington's Disease): BVCT ex-ante prediction vs. reported outcome

On 5 Mar 2024, BioinvestGPT's BVCT predicted technical success and commercial failure for this trial. 2 readouts followed: 20 Jun 2024, True Negative (TN); 5 May 2025, True Negative (TN).

Clinical Trial

NCTID
NCT05358717
Acronym
PIVOT-HD
Drug Name
PTC518
Drug MoA
HTT mRNA degradation
Drug Modality
small molecule
Drug Class
First-In-Class
Therapeutic Area
Nervous System Diseases
Indication
Huntington's Disease
Human Patients
252
Sponsor
PTC Therapeutics
Phase
Phase 2

Prediction of , index 557

Prospective Prediction
Prediction Index
557
Prediction Date
Commercial Prediction
FAILURE (commercially insufficient clinical benefit in UHDRS-TFC score)
Technical Prediction
SUCCESS (statistically significant clinical benefit in lowering tHTT level)

Readout of

Prediction Validation
Readout Date
Prediction To Readout
107 days in advance
Readout Data Interpretation
43% mHTT lowering for 10mg; TMS (2.0 points worsening for 5mg and 1.3 points worsening for 10mg vs. 4.9 points worsening for placebo).
Press Release
Prediction Result
Prediction Accuracy
Correct Prediction
Clinical Benefit Conclusion
statistically significant and commercially insufficient (TMS and UHDRS scores are both functional endpoints; pending for UHDRSdata)
Prediction Classification
True Negative (TN)

Readout of

Prediction Validation
Readout Date
Prediction To Readout
426 days in advance
Readout Data Interpretation
PTC518 failed to deliver statistically significant improvement in the efficacy endpoint cUHDRS at month 12
Press Release
Prediction Result
Prediction Accuracy
Correct Prediction
Clinical Benefit Conclusion
statistically significant (mHTT) and commercially insufficient (UHDRS)
Prediction Classification
True Negative (TN)

ClinicalTrials.gov record

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

Official title
A Phase 2A, Randomized, Placebo-Controlled, Dose-Ranging Study to Evaluate the Safety and Efficacy of PTC518 in Subjects With Huntington's Disease
Phase
Phase 2
Status
Completed
Enrollment
159
Lead sponsor
Novartis Pharmaceuticals
Conditions
Huntington Disease
Interventions
DRUG: PTC518; DRUG: Placebo
Design
Randomized · Parallel · Double
Primary purpose
Treatment
Start
2022-06-03
Primary completion
2025-02-07
Completion
2025-07-31
Results first posted
2026-01-13
Primary outcomes
  • Number of Participants With Treatment-emergent Adverse Events (TEAEs) (Baseline up to Month 18)
  • Percent Change From Baseline in Geometric Mean Blood tHTT Protein at Month 3 (Baseline, Month 3)

NCT05358717 on ClinicalTrials.gov

Return to the dashboardClinicalTrials.gov

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