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

• Status: Recruiting
• Trial end date: 2022-12-31
• Target enrollment: 0
• Participant gender: All

Summary

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

• Phase: Phase 2
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: PTC Therapeutics

Criteria

Key Inclusion Criteria:

• Genetically confirmed HD diagnosis with a cytosine-adenine-guanine (CAG) repeat length from 42 to 50, inclusive

• 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)

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.