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A Study to Evaluate the Safety and Efficacy of PTC518 in Participants With Huntington's Disease (HD)
A Study to Evaluate the Safety and Efficacy of PTC518 in Participants With Huntington's Disease (HD)

NCT05358717

CompletedPhase 2

Sponsor: Novartis Pharmaceuticals

Conditions: Huntington Disease

Interventions: PTC518, Placebo

Countries: United States, Australia, Austria, Canada, France, Germany, Italy, Netherlands

The primary goal of this study is to evaluate the safety and pharmacodynamic effects of PTC518 compared with placebo in participants with HD.

Eligibility overview

Sex: ALL

Age: 25 Years to —

Healthy volunteers: No

Study type: INTERVENTIONAL

Eligibility criteria
Key Inclusion Criteria:

* Genetically confirmed HD diagnosis with a cytosine-adenine-guanine (CAG) repeat length from 40 to 50, inclusive

Eligibility for HD-ISS Stage 2 Group (Parts A, B, and C):

* A Unified Huntington's Disease Rating Scale (UHDRS)-Independence Scale (IS) score of 100
* A UHDRS Total Functional Capacity (TFC) score of 13
* A score between 0.18 and 4.93 inclusive on the normed version of the HD prognostic index (PINHD)

Eligibility for HD-ISS Mild Stage 3 Group (Parts D, E, and F):

* A UHDRS Total Functional Capacity (TFC) score of 11 or 12, or a UHDRS TFC score of 13 with an UHDRS IS score of \<100

Key Exclusion Criteria:

* Receipt of an experimental agent within 90 days or 5 half-lives prior to Screening or anytime over the duration of this study, ribonucleic acid (RNA)- or deoxyribonucleic acid (DNA)-targeted HD-specific investigational agents such as antisense oligonucleotides, cell transplantation, or any other experimental brain surgery
* Any history of gene therapy exposure for the treatment of HD
* Participation in an investigational study or investigational paradigm (such as exercise/physical activity, cognitive therapy, brain stimulation, etc) within 90 days prior to Screening or anytime over the duration of this study
* Any medical history of brain or spinal disease that would interfere with the lumbar puncture process safety assessments
* Any medical history or condition that would interfere with the ability to complete the protocol-specified assessments (for example, implanted shunt, conditions precluding magnetic resonance imaging \[MRI\] scans)
* Pregnancy, planning on becoming pregnant during the course of the study or within 6 months of end of treatment, or currently breastfeeding

Note: Other inclusion and exclusion criteria may apply.
Locations (28)
  • La Jolla, California, United States
  • Nashville, Tennessee, United States
  • Houston, Texas, United States
  • Seattle, Washington, United States
  • Clayton, Australia
  • Sydney, Australia
  • Innsbruck, Austria
  • Ottawa, Canada
  • Angers, France
  • Marseille, France
  • Paris, France
  • Berlin, Germany
  • Bochum, Germany
  • Münster, Germany
  • Ulm, Germany
  • Bologna, Italy
  • Milan, Italy
  • San Giovanni Rotondo, Italy
  • Leiden, Netherlands
  • Christchurch, New Zealand
  • Barakaldo, Spain
  • Barcelona, Spain
  • Burgos, Spain
  • Madrid, Spain
  • Birmingham, United Kingdom
  • Cardiff, United Kingdom
  • London, United Kingdom
  • Manchester, United Kingdom