NCT06254482

Brief Summary

The primary goal of this study is to evaluate the long-term safety and pharmacodynamic effects of votoplam in participants with HD.

Trial Health

82
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
144

participants targeted

Target at P75+ for phase_2

Timeline
39mo left

Started Aug 2023

Longer than P75 for phase_2

Geographic Reach
9 countries

22 active sites

Status
active not recruiting

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

Study Progress47%
Aug 2023Jul 2029

Study Start

First participant enrolled

August 25, 2023

Completed
5 months until next milestone

First Submitted

Initial submission to the registry

February 2, 2024

Completed
10 days until next milestone

First Posted

Study publicly available on registry

February 12, 2024

Completed
5.4 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

July 16, 2029

Expected
Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

July 16, 2029

Last Updated

November 21, 2025

Status Verified

November 1, 2025

Enrollment Period

5.9 years

First QC Date

February 2, 2024

Last Update Submit

November 18, 2025

Conditions

Huntington Disease

Keywords

Neurodegenerative disorderRare disease

Outcome Measures

Primary Outcomes (2)

  • Number of Participants With Treatment-emergent Adverse Events (TEAEs)

    Baseline up to Month 54

  • Blood Total Huntingtin Protein (tHTT) Levels

    Baseline up to Month 52

Secondary Outcomes (4)

  • Change From Baseline in Caudate Volume as Assessed Via Volumetric Magnetic Resonance Imaging (vMRI) at Month 48

    Baseline, Month 48

  • Change From Baseline in Composite Unified Huntington's Disease Rating Scale (cUHDRS) Scores at Month 48

    Baseline, Month 48

  • Cerebrospinal Fluid (CSF) Mutant Huntingtin Protein (mHTT) Levels

    Baseline up to Month 48

  • Blood mHTT Protein Levels

    Baseline up to Month 52

Study Arms (2)

Votoplam 5 mg

EXPERIMENTAL

Participants will receive Votoplam 5 mg tablets once daily orally for 48 months.

Drug: Votoplam

Votoplam 10 mg

EXPERIMENTAL

Participants will receive votoplam 10 mg tablets once daily orally for 48 months.

Drug: Votoplam

Interventions

VotoplamDRUG

Votoplam will be administered per dose and schedule specified in the arm.

Also known as: HTT227 / PTC518
Votoplam 10 mgVotoplam 5 mg

Eligibility Criteria

Age25 Years+
Sexall
Healthy VolunteersNo
Age GroupsAdult (18-64), Older Adult (65+)

You may qualify if:

  • Participant who has completed the Treatment Period in Study PTC518-CNS-002-HD.

You may not qualify if:

  • Participants who have not previously completed the Treatment Period in Study PTC518-CNS-002-HD.

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (24)

Novartis Investigative Site

Clayton, 3168, Australia

Location

Novartis Investigative Site

Westmead, 2145, Australia

Location

Novartis Investigative Site

Innsbruck, 6020, Austria

Location

Novartis Investigative Site

Ottawa, K1Y 4E9, Canada

Location

Novartis Investigative Site

Angers, 49100, France

Location

Novartis Investigative Site

Marseille, 13385, France

Location

Novartis Investigative Site

Paris, 75013, France

Location

Novartis Investigative Site

Berlin, 10117, Germany

Location

Novartis Investigative Site

Bochum, 44791, Germany

Location

Novartis Investigative Site

Münster, 48149, Germany

Location

Novartis Investigative Site

Ulm, 89081, Germany

Location

Novartis Investigative Site

Bologna, 40139, Italy

Location

Novartis Investigative Site

Milan, 20133, Italy

Location

Novartis Investigative Site

San Giovanni Rotondo, 71013, Italy

Location

Novartis Investigative Site

Leiden, 2333 ZA, Netherlands

Location

Novartis Investigative Site

Christchurch, 8011, New Zealand

Location

Novartis Investigative Site

Barakaldo, 48903, Spain

Location

Novartis Investigative Site

Barcelona, 8025, Spain

Location

Novartis Investigative Site

Burgos, 90550, Spain

Location

Novartis Investigative Site

Madrid, 28034, Spain

Location

Novartis Investigative Site

Birmingham, B15 2FG, United Kingdom

Location

Novartis Investigative Site

Cardiff, CF14 4XW, United Kingdom

Location

Novartis Investigative Site

London, WC1N 3BG, United Kingdom

Location

Novartis Investigative Site

Manchester, M13 9WL, United Kingdom

Location

MeSH Terms

Conditions

Huntington DiseaseNeurodegenerative DiseasesRare Diseases

Condition Hierarchy (Ancestors)

Basal Ganglia DiseasesBrain DiseasesCentral Nervous System DiseasesNervous System DiseasesDementiaChoreaDyskinesiasMovement DisordersHeredodegenerative Disorders, Nervous SystemGenetic Diseases, InbornCongenital, Hereditary, and Neonatal Diseases and AbnormalitiesCognition DisordersNeurocognitive DisordersMental DisordersDisease AttributesPathologic ProcessesPathological Conditions, Signs and Symptoms

Study Design

Study Type
interventional
Phase
phase 2
Allocation
RANDOMIZED
Masking
DOUBLE
Who Masked
PARTICIPANT, INVESTIGATOR
Purpose
TREATMENT
Intervention Model
PARALLEL
Sponsor Type
INDUSTRY
Responsible Party
SPONSOR

Study Record Dates

First Submitted

February 2, 2024

First Posted

February 12, 2024

Study Start

August 25, 2023

Primary Completion (Estimated)

July 16, 2029

Study Completion (Estimated)

July 16, 2029

Last Updated

November 21, 2025

Record last verified: 2025-11

Data Sharing

IPD Sharing
Will share

Novartis is committed to sharing with qualified external researchers, access to patient-level data and supporting clinical documents from eligible studies. These requests are reviewed and approved by an independent review panel on the basis of scientific merit. All data provided is anonymized to respect the privacy of patients who have participated in the trial in line with applicable laws and regulations. This trial data availability is according to the criteria and process described on www.clinicalstudydatarequest.com

Locations

AU(1)NZ(1)ES(4)DE(4)FR(3)CA(1)NL(1)GB(4)IT(3)