2025-11-18
notable
Enrolling by Invitation→Active, Not Recruiting Study Identification, Study Status, Sponsor/Collaborators, Study Description, Study Design, Arms and Interventions, Contacts/Locations, IPDSharing v11
Trial statusEnrolling by Invitation→Active, Not Recruiting
Primary completion date2027-06-30→2029-07-16
Completion date2027-07-20→2029-07-16
Enrollment targetestimated 250→confirmed 144
Study arms3→2
Lead sponsorPTC Therapeutics→Novartis Pharmaceuticals
InterventionsPTC518 (Drug)→Votoplam (Drug)
Study description-255 characters
[...] 54 additional months.
All participants will receive active PTC518votoplam in this extension study.
Participants who received PTC518votoplam in the parent Study PTC518-CNS-002-HD will continue at the [...] evel they received in that study in a blinded fashion (5, 10, or 20 milligrams [mg]).
Participants who received placebo in the parent Study PTC518-CNS-002-HD will be allocated to a PTC518votoplam dose level according to the same dosing group in which they were previously randomized (5, 10, or 20 mg).
The 20 mg dosing arm will be open for randomization only if and when the Data Safety and Monitorin [...]
Study sites24 entries, revised
MonashNovartis HealthInvestigative Site
WestmeadNovartis HospitalInvestigative Site
MedicalNovartis UniversityInvestigative InnsbruckSite
TheNovartis OttawaInvestigative Hospital,Site
Novartis Parkinson'sInvestigative andSite
Novartis MovementInvestigative DisordersSite
Novartis ClinicInvestigative Site
CentreNovartis HospitalierInvestigative UniversitaireSite
Novartis d'AngersInvestigative Site
HôpitalNovartis UniversitaireInvestigative deSite
Novartis MarseilleInvestigative HôpitalSite
Novartis deInvestigative laSite
Novartis TimoneInvestigative Site
BrainNovartis andInvestigative SpineSite
[...] [...]
Study title+2 characters
An Extension Study to Evaluate the Long-Term Safety and Efficacy of PTC518Votoplam in Participants With Huntington's Disease (HD)
2025-07-09
minor
Study Status, Study Description, Arms and Interventions, Outcome Measures v10
Secondary endpoints4 entries, revised
Change From Baseline in Caudate Volume as Assessed Via Volumetric Magnetic Resonance Imaging (vMRI) at Month 2448
Change From Baseline in Composite Unified Huntington's Disease Rating Scale (cUHDRS) Scores at Month 2448
Study descriptionrevised
[...] study will undergo baseline evaluations and be assessed for 3054 additional months.
All participants will receive active PTC [...]
2024-08-04
notable
Recruiting→Enrolling by Invitation Study Status, Contacts/Locations v4
Trial statusRecruiting→Enrolling by Invitation
Study sitesdetails revised at 24 of 24 sites