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| ID | Type | Description | Link |
|---|---|---|---|
| 2023-504628-24-00 | Other Identifier | EU CT Number | |
| CHTT227A12201 | Other Identifier | Novartis |
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The primary goal of this study is to evaluate the long-term safety and pharmacodynamic effects of votoplam in participants with HD.
Participants who completed the Treatment Period in the parent Phase 2a Study PTC518-CNS-002-HD (NCT05358717), fulfilled the enrollment criteria, and chose to enroll in this extension study will undergo baseline evaluations and be assessed for 54 additional months.
All participants will receive active votoplam in this extension study. Participants who received votoplam in the parent Study PTC518-CNS-002-HD will continue at the same dose level they received in that study in a blinded fashion (5, 10 milligrams [mg]). Participants who received placebo in the parent Study PTC518-CNS-002-HD will be allocated to a votoplam dose level according to the same dosing group in which they were previously randomized (5, 10 mg).
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Votoplam 5 mg | Experimental | Participants will receive Votoplam 5 mg tablets once daily orally for 48 months. |
|
| Votoplam 10 mg | Experimental | Participants will receive votoplam 10 mg tablets once daily orally for 48 months. |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Votoplam | Drug | Votoplam will be administered per dose and schedule specified in the arm. |
|
| Measure | Description | Time Frame |
|---|---|---|
| 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 |
| Measure | Description | Time Frame |
|---|---|---|
| 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 |
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Inclusion Criteria:
Exclusion Criteria:
Note: Other inclusion and exclusion criteria may apply.
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| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Novartis Investigative Site | Clayton | 3168 | Australia | |||
| Novartis Investigative Site |
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
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| Cerebrospinal Fluid (CSF) Mutant Huntingtin Protein (mHTT) Levels | Baseline up to Month 48 |
| Blood mHTT Protein Levels | Baseline up to Month 52 |
| Westmead |
| 2145 |
| Australia |
| Novartis Investigative Site | Innsbruck | 6020 | Austria |
| Novartis Investigative Site | Ottawa | K1Y 4E9 | Canada |
| Novartis Investigative Site | Angers | 49100 | France |
| Novartis Investigative Site | Marseille | 13385 | France |
| Novartis Investigative Site | Paris | 75013 | France |
| Novartis Investigative Site | Berlin | 10117 | Germany |
| Novartis Investigative Site | Bochum | 44791 | Germany |
| Novartis Investigative Site | Münster | 48149 | Germany |
| Novartis Investigative Site | Ulm | 89081 | Germany |
| Novartis Investigative Site | Bologna | 40139 | Italy |
| Novartis Investigative Site | Milan | 20133 | Italy |
| Novartis Investigative Site | San Giovanni Rotondo | 71013 | Italy |
| Novartis Investigative Site | Leiden | 2333 ZA | Netherlands |
| Novartis Investigative Site | Christchurch | 8011 | New Zealand |
| Novartis Investigative Site | Barakaldo | 48903 | Spain |
| Novartis Investigative Site | Barcelona | 8025 | Spain |
| Novartis Investigative Site | Burgos | 90550 | Spain |
| Novartis Investigative Site | Madrid | 28034 | Spain |
| Novartis Investigative Site | Birmingham | B15 2FG | United Kingdom |
| Novartis Investigative Site | Cardiff | CF14 4XW | United Kingdom |
| Novartis Investigative Site | London | WC1N 3BG | United Kingdom |
| Novartis Investigative Site | Manchester | M13 9WL | United Kingdom |
| ID | Term |
|---|---|
| D006816 | Huntington Disease |
| D019636 | Neurodegenerative Diseases |
| D035583 | Rare Diseases |
| ID | Term |
|---|---|
| D001480 | Basal Ganglia Diseases |
| D001927 | Brain Diseases |
| D002493 | Central Nervous System Diseases |
| D009422 | Nervous System Diseases |
| D003704 | Dementia |
| D002819 | Chorea |
| D020820 | Dyskinesias |
| D009069 | Movement Disorders |
| D020271 | Heredodegenerative Disorders, Nervous System |
| D030342 | Genetic Diseases, Inborn |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
| D003072 | Cognition Disorders |
| D019965 | Neurocognitive Disorders |
| D001523 | Mental Disorders |
| D020969 | Disease Attributes |
| D010335 | Pathologic Processes |
| D013568 | Pathological Conditions, Signs and Symptoms |
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