Trial Review

The ANZCTR website will be unavailable from 1pm until 3pm (AEDT) on Wednesday the 30th of October for website maintenance. Please be sure to log out of the system in order to avoid any loss of data.

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been endorsed by the ANZCTR. Before participating in a study, talk to your health care provider and refer to this information for consumers
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
Country [2] 0 0
United States of America
State/province [2] 0 0
California
Country [3] 0 0
United States of America
State/province [3] 0 0
Colorado
Country [4] 0 0
United States of America
State/province [4] 0 0
District of Columbia
Country [5] 0 0
United States of America
State/province [5] 0 0
New York
Country [6] 0 0
United States of America
State/province [6] 0 0
Tennessee
Country [7] 0 0
United States of America
State/province [7] 0 0
Texas
Country [8] 0 0
United States of America
State/province [8] 0 0
Washington
Country [9] 0 0
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
Country [12] 0 0
Canada
State/province [12] 0 0
Vancouver
Country [13] 0 0
France
State/province [13] 0 0
Angers
Country [14] 0 0
France
State/province [14] 0 0
Marseille
Country [15] 0 0
France
State/province [15] 0 0
Paris
Country [16] 0 0
Germany
State/province [16] 0 0
Bochum
Country [17] 0 0
Germany
State/province [17] 0 0
Münster
Country [18] 0 0
Germany
State/province [18] 0 0
Ulm
Country [19] 0 0
Italy
State/province [19] 0 0
Bologna
Country [20] 0 0
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
Country [23] 0 0
New Zealand
State/province [23] 0 0
Christchurch
Country [24] 0 0
Spain
State/province [24] 0 0
Barakaldo
Country [25] 0 0
Spain
State/province [25] 0 0
Barcelona
Country [26] 0 0
Spain
State/province [26] 0 0
Burgos
Country [27] 0 0
Spain
State/province [27] 0 0
Madrid
Country [28] 0 0
United Kingdom
State/province [28] 0 0
Birmingham
Country [29] 0 0
United Kingdom
State/province [29] 0 0
Cardiff
Country [30] 0 0
United Kingdom
State/province [30] 0 0
Leeds
Country [31] 0 0
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