A Study to Evaluate the Safety and Efficacy of PTC518 in Participants With Huntington's Disease (HD)
NCT ID: NCT05358717
Last Updated: 2026-01-13
Study Results
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View full resultsBasic Information
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COMPLETED
PHASE2
159 participants
INTERVENTIONAL
2022-06-03
2025-07-31
Brief Summary
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Detailed Description
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Participants who complete this study and agree to participate in a long-term extension (LTE) study, will be enrolled in a separate LTE study PTC518-CNS-004-HD (NCT06254482).
Conditions
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Study Design
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RANDOMIZED
PARALLEL
TREATMENT
DOUBLE
Study Groups
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PTC518 5 mg
Participants will receive PTC518 5 milligrams (mg) tablets once daily orally for 12 months.
PTC518
PTC518 will be administered per dose and schedule specified in the arm.
PTC518 10 mg
Participants will receive PTC518 10 mg tablets once daily orally for 12 months.
PTC518
PTC518 will be administered per dose and schedule specified in the arm.
PTC518 20 mg
Participants will receive PTC518 20 mg tablets once daily orally for 12 months.
PTC518
PTC518 will be administered per dose and schedule specified in the arm.
Placebo
Participants will receive placebo matching to PTC518 tablets once daily orally for 12 months.
Placebo
Placebo matching to PTC518 will be administered per schedule specified in the arm.
Interventions
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PTC518
PTC518 will be administered per dose and schedule specified in the arm.
Placebo
Placebo matching to PTC518 will be administered per schedule specified in the arm.
Eligibility Criteria
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Inclusion Criteria
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
Exclusion Criteria
* 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
25 Years
ALL
No
Sponsors
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PTC Therapeutics
INDUSTRY
Responsible Party
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Locations
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University of California, San Diego
La Jolla, California, United States
Vanderbilt University Medical Center
Nashville, Tennessee, United States
The University of Texas Health Science Center at Houston; McGovern Medical School
Houston, Texas, United States
University of Washington Department of Neurology
Seattle, Washington, United States
Monash Health
Clayton, , Australia
Westmead Hospital
Sydney, , Australia
Medical University Innsbruck
Innsbruck, , Austria
The Ottawa Hospital, Parkinson's and Movement Disorders Clinic
Ottawa, , Canada
Centre Hospitalier Universitaire d'Angers
Angers, , France
Hôpital Universitaire de Marseille Hôpital de la Timone
Marseille, , France
Brain and Spine Institute Paris
Paris, , France
Charite University Medicine Berlin
Berlin, , Germany
Ruhr-Univ. Bochum St. Joseph-Hospital
Bochum, , Germany
George-Huntington-Institut
Münster, , Germany
Ulm University, UKU, Dep. of Neurology
Ulm, , Germany
Irccs Istituto Delle Scienze Neurologiche Di Bologna Uoc Clinica Neurologica
Bologna, , Italy
IRCCS Carlo Besta Neurological Institutte
Milan, , Italy
IRCCS Casa Sollievo della Sofferenza Research Hospital
San Giovanni Rotondo, , Italy
Leiden University Medical Center
Leiden, , Netherlands
University of Otago, New Zealand Brain Research Institute
Christchurch, , New Zealand
Hospital Universitario Cruces
Barakaldo, , Spain
Hospital de la Santa Creu I Sant Pau
Barcelona, , Spain
Hospital Universitario Burgos
Burgos, , Spain
Hospital Ramón y Cajal
Madrid, , Spain
The Barbery National Centre for Mental Health
Birmingham, , United Kingdom
Cardiff University Schools of Medicine and Biosciences
Cardiff, , United Kingdom
UCL Queen Square Institute of Neurology National Hospital for Neurology and Neurosurgery
London, , United Kingdom
Manchester University NHS Foundation Trust
Manchester, , United Kingdom
Countries
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References
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Estevez-Fraga C, Tabrizi SJ, Wild EJ. Huntington's Disease Clinical Trials Corner: March 2024. J Huntingtons Dis. 2024;13(1):1-14. doi: 10.3233/JHD-240017.
Estevez-Fraga C, Tabrizi SJ, Wild EJ. Huntington's Disease Clinical Trials Corner: November 2022. J Huntingtons Dis. 2022;11(4):351-367. doi: 10.3233/JHD-229006.
Provided Documents
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Document Type: Study Protocol
Document Type: Statistical Analysis Plan
Other Identifiers
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2021-003852-18
Identifier Type: EUDRACT_NUMBER
Identifier Source: secondary_id
PTC518-CNS-002-HD
Identifier Type: -
Identifier Source: org_study_id
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