A Study of Long-term Safety and Efficacy of VX-670 in Participants With Myotonic Dystrophy Type I
For patients and families
In plain language
An automatic summary of structured registry data. It is an orientation aid, not a substitute for the official protocol or a physician assessment.
- What is being studied
- The protocol lists: VX-670.
- Who it may be relevant to
- Registry conditions: Myotonic Dystrophy Type 1 (DM1). Basic parameters: from 18 years · All.
- What needs checking
- Age, condition and sex are only basic indicators. Prior treatment, laboratory values and other mandatory requirements appear in the eligibility criteria below.
- Where it takes place
- Australia, Belgium, Canada, Germany, Netherlands +2
- Next step
- Save the trial, show it to the treating physician, and confirm current recruitment with the study center. Costs, documents and travel →
Unsure about the terms? Read our patient guide →
Official title
An Open-label Extension Study Evaluating the Long-term Safety, Tolerability, Efficacy, Pharmacokinetics, and Pharmacodynamics of VX-670 in Adult Subjects With Myotonic Dystrophy Type I
Overview
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).
Interventions
- Drug VX-670
Solution for intravenous administration.
Primary outcome measures
- Safety and Tolerability as Assessed by Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs) [Time frame: From Day 1 up to Week 108]
Secondary outcome measures (2)
- Maximum Observed Concentration (Cmax) of VX-670 and its Active Component in Plasma [Time frame: From Day 1 up to Week 96]
- Area Under the Concentration Versus Time Curve (AUC) of VX-670 and its Active Component in Plasma [Time frame: From Day 1 up to Week 96]
Eligibility criteria
Inclusion criteria
- Completed study drug treatment in parent study VX23-670-001 (NCT06185764)
Exclusion criteria
- History of any illness or any clinical condition as pre-specified in the protocol
Other protocol defined Inclusion/Exclusion criteria may apply.
Criteria are shown verbatim from the registry (in English). Final eligibility is always assessed by the study center.
Healthy volunteers: No
Study design
- Allocation
- N/A
- Model
- Single group
- Masking
- Open label
- Primary purpose
- Treatment
Study locations
Canada · 4 centers
- Altasciences Montreal — Montreal
- Montreal Neurological Institute-Hospital — Montreal
- University of Ottawa — Ottawa
- Universite Laval - Neurology — Québec
United Kingdom · 3 centers
- Queen Elizabeth University Hospital - Neurology — Glasgow
- Leonard Wolfson Experimental Neurology Centre CRF — London
- Royal Hallamshire Hospital - Neurology — Sheffield
Australia · 2 centers
- Wesley Research Institute — Auchenflower
- Neuroscience Clinical Trials Unit, Alfred Brain — Melbourne
Belgium · 1 center
- Universitaire Ziekenhuizen Leuven - Campus Gasthuisberg - Pulmonology — Leuven
Germany · 1 center
- Friedrich Baur Institute — München
Netherlands · 1 center
- Maastricht UMC — Maastricht
Spain · 1 center
- Hospital Universitario y Politecnico La Fe - Neurology — Valencia
Identifiers
NCT: NCT06926621 · VX24-670-101 · 2024-517983-47-00