drugset / Trial / NCT05358717
A Study to Evaluate the Safety and Efficacy of PTC518 in Participants With Huntington's Disease (HD)
RandomizedParallel-groupDouble-blindTreatment
Summary
The primary goal of this study is to evaluate the safety and pharmacodynamic effects of PTC518 compared with placebo in participants with HD.
Timeline
- Start
- 2022-06-03
- Primary completion
- 2025-02-07
- Completion
- 2025-07-31
Outcome
Met primary endpoint
registry analysis (superiority test); Placebo vs PTC518 5 mg; p < 0.0001; LS Mean Difference -15.97 (95% CI -23.59 to -8.34); Mixed Models Analysis NCT05358717 ↗
release “The Phase II PIVOT-HD study of votoplam in patients with Stage 2 and Stage 3 Huntington’s disease met its primary endpoint” novartis.com ↗
Publications
- Estevez-Fraga C, Tabrizi SJ, Wild EJ. Huntington's Disease Clinical Trials Corner: March 2024. J Huntingtons Dis. 2024;13(1):1-14. doi: 10.3233/JHD-240017.
- Estevez-Fraga C, Tabrizi SJ, Wild EJ. Huntington's Disease Clinical Trials Corner: November 2022. J Huntingtons Dis. 2022;11(4):351-367. doi: 10.3233/JHD-229006.
Drugs
| Evaluation | Drug | Modality | Dose | Route |
|---|---|---|---|---|
| Subject | Votoplam | Small molecule | 5 mg | Oral |
| Subject | Votoplam | Small molecule | 10 mg | Oral |
| Subject | Votoplam | Small molecule | 20 mg | Oral |