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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/study/NCT05358717




Registration number
NCT05358717
Ethics application status
Date submitted
27/04/2022
Date registered
3/05/2022
Date last updated
15/03/2024

Titles & IDs
Public title
A Study to Evaluate the Safety and Efficacy of PTC518 in Participants With Huntington's Disease (HD)
Scientific title
A Phase 2A, Randomized, Placebo-Controlled, Dose-Ranging Study to Evaluate the Safety and Efficacy of PTC518 in Subjects With Huntington's Disease
Secondary ID [1] 0 0
2021-003852-18
Secondary ID [2] 0 0
PTC518-CNS-002-HD
Universal Trial Number (UTN)
Trial acronym
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Huntington Disease 0 0
Condition category
Condition code

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
Treatment: Drugs - PTC518
Treatment: Drugs - Placebo

Experimental: PTC518 5 mg - Participants will receive PTC518 5 milligrams (mg) tablets once daily orally for 12 months.

Experimental: PTC518 10 mg - Participants will receive PTC518 10 mg tablets once daily orally for 12 months.

Experimental: PTC518 20 mg - Participants will receive PTC518 20 mg tablets once daily orally for 12 months.

Placebo comparator: Placebo - Participants will receive placebo matching to PTC518 tablets once daily orally for 12 months.


Treatment: Drugs: PTC518
PTC518 will be administered per dose and schedule specified in the arm.

Treatment: Drugs: Placebo
Placebo matching to PTC518 will be administered per schedule specified in the arm.
Intervention code [1] 0 0
Treatment: Drugs
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Number of Participants With Adverse Events (AEs)
Timepoint [1] 0 0
Baseline up to Month 18
Primary outcome [2] 0 0
Change From Baseline in Blood Total Huntingtin Protein (tHTT) at Month 3
Timepoint [2] 0 0
Baseline, Month 3
Secondary outcome [1] 0 0
Change From Baseline in Caudate Volume as Assessed Via Volumetric Magnetic Resonance Imaging (vMRI) at Month 12
Timepoint [1] 0 0
Baseline, Month 12
Secondary outcome [2] 0 0
Change From Baseline in Composite Unified Huntington's Disease Rating Scale (cUHDRS) Scores at Month 12
Timepoint [2] 0 0
Baseline, Month 12
Secondary outcome [3] 0 0
Change From Baseline in Blood tHTT Protein at Month 12
Timepoint [3] 0 0
Baseline, Month 12
Secondary outcome [4] 0 0
Change From Baseline in Cerebrospinal Fluid (CSF) Mutant Huntingtin Protein (mHTT) at Month 12
Timepoint [4] 0 0
Baseline, Month 12
Secondary outcome [5] 0 0
Change From Baseline in Blood mHTT Protein at Month 12
Timepoint [5] 0 0
Baseline, Month 12

Eligibility
Key inclusion criteria
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
Minimum age
25 Years
Maximum age
No limit
Sex
Both males and females
Can healthy volunteers participate?
No
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.

Study design
Purpose of the study
Treatment
Allocation to intervention
Randomised controlled trial
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Masking / blinding
Blinded (masking used)
Who is / are masked / blinded?
The people receiving the treatment/s


The people analysing the results/data
Intervention assignment
Parallel
Other design features
Phase
Phase 2
Type of endpoint/s
Statistical methods / analysis

Recruitment
Recruitment status
Recruiting
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
25/04/2022
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
31/07/2024
Actual
Sample size
Target
252
Accrual to date
Final
Recruitment in Australia
Recruitment state(s)
Recruitment hospital [1] 0 0
Monash Health - Clayton
Recruitment hospital [2] 0 0
Westmead Hospital - Sydney
Recruitment postcode(s) [1] 0 0
3168 - Clayton
Recruitment postcode(s) [2] 0 0
2145 - Sydney
Recruitment outside Australia
Country [1] 0 0
United States of America
State/province [1] 0 0
Alabama
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United States of America
State/province [2] 0 0
California
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United States of America
State/province [3] 0 0
Colorado
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United States of America
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District of Columbia
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United States of America
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New York
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United States of America
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Tennessee
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United States of America
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Texas
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United States of America
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Washington
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Austria
State/province [9] 0 0
Innsbruck
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Canada
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Montreal
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Canada
State/province [11] 0 0
Ottawa
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Canada
State/province [12] 0 0
Vancouver
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France
State/province [13] 0 0
Angers
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France
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Marseille
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France
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Paris
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Germany
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Bochum
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Germany
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Münster
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Germany
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Ulm
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Italy
State/province [19] 0 0
Bologna
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Italy
State/province [20] 0 0
Milan
Country [21] 0 0
Italy
State/province [21] 0 0
San Giovanni Rotondo
Country [22] 0 0
Netherlands
State/province [22] 0 0
Leiden
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New Zealand
State/province [23] 0 0
Christchurch
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Spain
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Barakaldo
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Spain
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Barcelona
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Spain
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Burgos
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Spain
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Madrid
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United Kingdom
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Birmingham
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United Kingdom
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Cardiff
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United Kingdom
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Leeds
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United Kingdom
State/province [31] 0 0
London
Country [32] 0 0
United Kingdom
State/province [32] 0 0
Manchester

Funding & Sponsors
Primary sponsor type
Commercial sector/industry
Name
PTC Therapeutics
Address
Country

Ethics approval
Ethics application status

Summary
Brief summary
Trial website
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 0 0
Address 0 0
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for public queries
Name 0 0
Patient Advocacy
Address 0 0
Country 0 0
Phone 0 0
1-866-562-4620
Fax 0 0
Email 0 0
[email protected]
Contact person for scientific queries

No information has been provided regarding IPD availability


What supporting documents are/will be available?

No Supporting Document Provided



Results publications and other study-related documents

No documents have been uploaded by study researchers.

Results not provided in https://clinicaltrials.gov/study/NCT05358717