Status:
ACTIVE_NOT_RECRUITING
A Study to Evaluate the Safety and Efficacy of PTC518 in Participants With Huntington's Disease (HD)
Lead Sponsor:
PTC Therapeutics
Conditions:
Huntington Disease
Eligibility:
All Genders
25+ years
Phase:
PHASE2
Brief Summary
The primary goal of this study is to evaluate the safety and pharmacodynamic effects of PTC518 compared with placebo in participants with HD.
Detailed Description
Participants will first be randomized to Part A or Part B or Parts D or E in a 1:1 randomization ratio, depending on their Huntington's disease Integrated Staging System (HD-ISS) staging criteria and ...
Eligibility Criteria
Inclusion
- Key
- 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
- 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.
Key Trial Info
Start Date :
April 25 2022
Trial Type :
INTERVENTIONAL
Allocation :
ACTUAL
End Date :
August 15 2025
Estimated Enrollment :
252 Patients enrolled
Trial Details
Trial ID
NCT05358717
Start Date
April 25 2022
End Date
August 15 2025
Last Update
February 20 2025
Active Locations (28)
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1
University of California, San Diego
La Jolla, California, United States, 92037
2
Vanderbilt University Medical Center
Nashville, Tennessee, United States, 37212
3
The University of Texas Health Science Center at Houston; McGovern Medical School
Houston, Texas, United States, 77030
4
University of Washington Department of Neurology
Seattle, Washington, United States, 98195