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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/ct2/show/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
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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
State/province [8] 0 0
Washington
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Austria
State/province [9] 0 0
Innsbruck
Country [10] 0 0
Canada
State/province [10] 0 0
Montreal
Country [11] 0 0
Canada
State/province [11] 0 0
Ottawa
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Canada
State/province [12] 0 0
Vancouver
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France
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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
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Bologna
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Italy
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Milan
Country [21] 0 0
Italy
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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
State/province [24] 0 0
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
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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
The primary goal of this study is to evaluate the safety and pharmacodynamic effects of
PTC518 compared with placebo in participants with HD.
Trial website
https://clinicaltrials.gov/ct2/show/NCT05358717
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
medinfo@ptcbio.com
Contact person for scientific queries



Summary Results

For IPD and results data, please see https://clinicaltrials.gov/ct2/show/NCT05358717