A Study to Evaluate the Safety and Efficacy of PTC518 in Participants With Huntington's Disease (HD) (NCT05358717) | Clinical Trial Compass
CompletedPhase 2
A Study to Evaluate the Safety and Efficacy of PTC518 in Participants With Huntington's Disease (HD)
United States, Australia159 participantsStarted 2022-06-03
Plain-language summary
The primary goal of this study is to evaluate the safety and pharmacodynamic effects of PTC518 compared with placebo in participants with HD.
Who can participate
Age range25 Years
SexALL
See this in plain English?
AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.
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-specifie…
What they're measuring
1
Number of Participants With Treatment-emergent Adverse Events (TEAEs)
Timeframe: Baseline up to Month 18
2
Percent Change From Baseline in Geometric Mean Blood tHTT Protein at Month 3