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Trial record 1 of 1 for:    ptc | Huntington Disease
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A Study to Evaluate the Safety and Efficacy of PTC518 in Participants With Huntington's Disease (HD)

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ClinicalTrials.gov Identifier: NCT05358717
Recruitment Status : Recruiting
First Posted : May 3, 2022
Last Update Posted : July 13, 2022
Sponsor:
Information provided by (Responsible Party):
PTC Therapeutics

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

Condition or disease Intervention/treatment Phase
Huntington Disease Drug: PTC518 Drug: Placebo Phase 2

Detailed Description:
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.

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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 162 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Double (Participant, Investigator)
Primary Purpose: Treatment
Official Title: A Phase 2a, Randomized, Placebo-Controlled, Dose-Ranging Study to Evaluate the Safety and Efficacy of PTC518 in Subjects With Huntington's Disease
Actual Study Start Date : April 25, 2022
Estimated Primary Completion Date : December 31, 2022
Estimated Study Completion Date : December 31, 2022

Resource links provided by the National Library of Medicine


Arm Intervention/treatment
Experimental: PTC518 5 mg
Participants will receive PTC518 5 milligrams (mg) tablets once daily orally for 12 weeks.
Drug: PTC518
PTC518 will be administered per dose and schedule specified in the arm.

Experimental: PTC518 10 mg
Participants will receive PTC518 10 mg tablets once daily orally for 12 weeks.
Drug: PTC518
PTC518 will be administered per dose and schedule specified in the arm.

Experimental: PTC518 20 mg
Participants will receive PTC518 20 mg tablets once daily orally for 12 weeks.
Drug: PTC518
PTC518 will be administered per dose and schedule specified in the arm.

Placebo Comparator: Placebo
Participants will receive placebo matching to PTC518 tablets once daily orally for 12 weeks.
Drug: Placebo
Placebo matching to PTC518 will be administered per schedule specified in the arm.




Primary Outcome Measures :
  1. Number of Participants With Adverse Events (AEs) [ Time Frame: Baseline up to Day 113 ]
  2. Change From Baseline in Blood Total Huntingtin Protein (tHTT) at Day 85 [ Time Frame: Baseline, Day 85 ]

Secondary Outcome Measures :
  1. Change From Baseline in Blood HTT Messenger Ribonucleic Acid (mRNA) at Days 29, 57, and 85 [ Time Frame: Baseline, Days 29, 57, and 85 ]
  2. Change From Baseline in Cerebrospinal Fluid (CSF) Mutant Huntingtin Protein (mHTT) at Day 85 [ Time Frame: Baseline, Day 85 ]
  3. Change From Baseline in Blood mHTT at Day 85 [ Time Frame: Baseline, Day 85 ]


Information from the National Library of Medicine

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Ages Eligible for Study:   25 Years and older   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
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.


Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT05358717


Contacts
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Contact: Patient Advocacy 1-866-562-4620 medinfo@ptcbio.com

Locations
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Sponsors and Collaborators
PTC Therapeutics
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Responsible Party: PTC Therapeutics
ClinicalTrials.gov Identifier: NCT05358717    
Other Study ID Numbers: PTC518-CNS-002-HD
2021-003852-18 ( EudraCT Number )
First Posted: May 3, 2022    Key Record Dates
Last Update Posted: July 13, 2022
Last Verified: July 2022

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Keywords provided by PTC Therapeutics:
Neurodegenerative disorder
Rare disease
Additional relevant MeSH terms:
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Huntington Disease
Basal Ganglia Diseases
Brain Diseases
Central Nervous System Diseases
Nervous System Diseases
Movement Disorders
Heredodegenerative Disorders, Nervous System
Neurodegenerative Diseases
Genetic Diseases, Inborn
Cognition Disorders
Neurocognitive Disorders
Mental Disorders
Dementia
Chorea
Dyskinesias