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

  • End date
    Dec 31, 2022
  • participants needed
  • sponsor
    PTC Therapeutics
Updated on 10 May 2022


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


Participants will first be randomized to Part A or Part B in a 1:1 randomization ratio, and then to active treatment (PTC518 5 mg in Part A or 10 mg in Part B) or matching placebo within each part in a 2:1 ratio of active treatment to placebo. A Drug Safety Monitoring Board (DSMB) Charter will undertake an unblinded review of safety data in Part A and Part B and provide a recommendation on when Part C (with a 20 mg active treatment arm) can be initiated. At that time, participants will be randomized to any study Part that is currently open for enrollment, and then to either active treatment or placebo (in a 2:1 ratio) within that Part.

Condition Huntington Disease
Treatment Placebo, PTC518
Clinical Study IdentifierNCT05358717
SponsorPTC Therapeutics
Last Modified on10 May 2022


Yes No Not Sure

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)

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
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Additional screening procedures may be conducted by the study team before you can be confirmed eligible to participate.

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If you are confirmed eligible after full screening, you will be required to understand and sign the informed consent if you decide to enroll in the study. Once enrolled you may be asked to make scheduled visits over a period of time.

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Complete your scheduled study participation activities and then you are done. You may receive summary of study results if provided by the sponsor.

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