Not provided
| ID | Type | Description | Link |
|---|---|---|---|
| 2024-517983-47-00 | Other Identifier | EU CT Number |
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
The purpose of the study is to evaluate the long-term safety and tolerability, efficacy and pharmacokinetics of VX-670 in participants with Myotonic Dystrophy Type I (DM1).
Not provided
Not provided
Not provided
Not provided
Not provided
| Label | Type | Description | Intervention Names |
|---|---|---|---|
| VX-670 | Experimental | Participants will receive multiple doses of VX-670. |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| VX-670 | Drug | Solution for intravenous administration. |
|
| Measure | Description | Time Frame |
|---|---|---|
| Safety and Tolerability as Assessed by Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs) | From Day 1 up to Week 108 |
| Measure | Description | Time Frame |
|---|---|---|
| Maximum Observed Concentration (Cmax) of VX-670 and its Active Component in Plasma | From Day 1 up to Week 96 | |
| Area Under the Concentration Versus Time Curve (AUC) of VX-670 and its Active Component in Plasma | From Day 1 up to Week 96 |
Not provided
Key Inclusion Criteria:
• Completed study drug treatment in parent study VX23-670-001 (NCT06185764)
Key Exclusion Criteria:
• History of any illness or any clinical condition as pre-specified in the protocol
Other protocol defined Inclusion/Exclusion criteria may apply.
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Wesley Research Institute | Auchenflower | Australia | ||||
| Neuroscience Clinical Trials Unit, Alfred Brain |
Details on Vertex data sharing criteria and process for requesting access can be found at: https://www.vrtx.com/our-science/clinical-trials-data-sharing/
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
| Melbourne |
| Australia |
| Universitaire Ziekenhuizen Leuven - Campus Gasthuisberg - Pulmonology | Leuven | Belgium |
| Altasciences Montreal | Montreal | Canada |
| Montreal Neurological Institute-Hospital | Montreal | Canada |
| University of Ottawa | Ottawa | Canada |
| Universite Laval - Neurology | Québec | Canada |
| Friedrich Baur Institute | München | Germany |
| Maastricht UMC | Maastricht | Netherlands |
| Hospital Universitario y Politecnico La Fe - Neurology | Valencia | Spain |
| Queen Elizabeth University Hospital - Neurology | Glasgow | United Kingdom |
| Leonard Wolfson Experimental Neurology Centre CRF | London | United Kingdom |
| Royal Hallamshire Hospital - Neurology | Sheffield | United Kingdom |
| ID | Term |
|---|---|
| D009223 | Myotonic Dystrophy |
| D009222 | Myotonia |
| D020967 | Myotonic Disorders |
| D020966 | Muscular Disorders, Atrophic |
| D009135 | Muscular Diseases |
| D009140 | Musculoskeletal Diseases |
| D009468 | Neuromuscular Diseases |
| D009422 | Nervous System Diseases |
| D030342 | Genetic Diseases, Inborn |
| D020271 | Heredodegenerative Disorders, Nervous System |
| D019636 | Neurodegenerative Diseases |
| D009136 | Muscular Dystrophies |
| ID | Term |
|---|---|
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
| D020879 | Neuromuscular Manifestations |
| D009461 | Neurologic Manifestations |
| D012816 | Signs and Symptoms |
| D013568 | Pathological Conditions, Signs and Symptoms |
Not provided
Not provided