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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/NCT06254482




Registration number
NCT06254482
Ethics application status
Date submitted
2/02/2024
Date registered
12/02/2024
Date last updated
30/05/2024

Titles & IDs
Public title
An Extension Study to Evaluate the Long-Term Safety and Efficacy of PTC518 in Participants With Huntington's Disease (HD)
Scientific title
A Phase 2b, Double-Blind, Randomized Extension Study to Evaluate the Long-Term Safety and Efficacy of PTC518 in Participants With Huntington's Disease
Secondary ID [1] 0 0
2023-504628-24-00
Secondary ID [2] 0 0
PTC518-CNS-004-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

Experimental: PTC518 5 mg - Participants will receive PTC518 5 mg tablets once daily orally for 24 months.

Experimental: PTC518 10 mg - Participants will receive PTC518 10 mg tablets once daily orally for 24 months.

Experimental: PTC518 20 mg - Participants will receive PTC518 20 mg tablets once daily orally for 24 months.


Treatment: Drugs: PTC518
PTC518 will be administered per dose and 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 Treatment-emergent Adverse Events (TEAEs)
Timepoint [1] 0 0
Baseline up to Month 30
Primary outcome [2] 0 0
Blood Total Huntingtin Protein (tHTT) Levels
Timepoint [2] 0 0
Baseline up to Month 28
Secondary outcome [1] 0 0
Change From Baseline in Caudate Volume as Assessed Via Volumetric Magnetic Resonance Imaging (vMRI) at Month 24
Timepoint [1] 0 0
Baseline, Month 24
Secondary outcome [2] 0 0
Change From Baseline in Composite Unified Huntington's Disease Rating Scale (cUHDRS) Scores at Month 24
Timepoint [2] 0 0
Baseline, Month 24
Secondary outcome [3] 0 0
Cerebrospinal Fluid (CSF) Mutant Huntingtin Protein (mHTT) Levels
Timepoint [3] 0 0
Baseline up to Month 24
Secondary outcome [4] 0 0
Blood mHTT Protein Levels
Timepoint [4] 0 0
Baseline up to Month 28

Eligibility
Key inclusion criteria
- Participant who has completed the Treatment Period in Study PTC518-CNS-002-HD.
Minimum age
25 Years
Maximum age
No limit
Sex
Both males and females
Can healthy volunteers participate?
No
Key exclusion criteria
- Participants who have not previously completed the Treatment Period in Study
PTC518-CNS-002-HD.

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
25/08/2023
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
20/07/2027
Actual
Sample size
Target
250
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 - Westmead
Recruitment postcode(s) [1] 0 0
3168 - Clayton
Recruitment postcode(s) [2] 0 0
2145 - Westmead
Recruitment outside Australia
Country [1] 0 0
Austria
State/province [1] 0 0
Innsbruck
Country [2] 0 0
Canada
State/province [2] 0 0
Ottawa
Country [3] 0 0
France
State/province [3] 0 0
Angers
Country [4] 0 0
France
State/province [4] 0 0
Marseille
Country [5] 0 0
France
State/province [5] 0 0
Paris
Country [6] 0 0
Germany
State/province [6] 0 0
Berlin
Country [7] 0 0
Germany
State/province [7] 0 0
Bochum
Country [8] 0 0
Germany
State/province [8] 0 0
Münster
Country [9] 0 0
Germany
State/province [9] 0 0
Ulm
Country [10] 0 0
Italy
State/province [10] 0 0
Bologna
Country [11] 0 0
Italy
State/province [11] 0 0
Milan
Country [12] 0 0
Italy
State/province [12] 0 0
San Giovanni Rotondo
Country [13] 0 0
Netherlands
State/province [13] 0 0
Leiden
Country [14] 0 0
New Zealand
State/province [14] 0 0
Christchurch
Country [15] 0 0
Spain
State/province [15] 0 0
Barakaldo
Country [16] 0 0
Spain
State/province [16] 0 0
Barcelona
Country [17] 0 0
Spain
State/province [17] 0 0
Burgos
Country [18] 0 0
Spain
State/province [18] 0 0
Madrid
Country [19] 0 0
United Kingdom
State/province [19] 0 0
Birmingham
Country [20] 0 0
United Kingdom
State/province [20] 0 0
Cardiff
Country [21] 0 0
United Kingdom
State/province [21] 0 0
London
Country [22] 0 0
United Kingdom
State/province [22] 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 long-term safety and pharmacodynamic
effects of PTC518 in participants with HD.
Trial website
https://clinicaltrials.gov/ct2/show/NCT06254482
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/NCT06254482