A Phase 1/2 Study of VX-670 in Adult Participants With Myotonic Dystrophy 1 (DM1)
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, Placebo.
- Who it may be relevant to
- Registry conditions: Myotonic Dystrophy Type 1 (DM1). Basic parameters: 18 years — 64 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
- United States, Australia, Belgium, Canada, France +5
- 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
A Phase 1/2, Randomized, Double-blind, Placebo-controlled Single- and Multiple-dose Escalation Study Evaluating the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of VX-670 in Adult Subjects With Myotonic Dystrophy Type 1
Overview
The purpose of the study is to evaluate the safety, tolerability, pharmacokinetics, and pharmacodynamics of VX-670 at different single and multiple doses in participants with DM1.
Interventions
- Drug VX-670
Solution for intravenous administration. - Drug Placebo
Solution for intravenous administration.
Primary outcome measures
- Parts A and B: Safety and Tolerability as Assessed by Number of Participants With Adverse Events (AEs) [Time frame: Part A: From Baseline up to Day 42; Part B: From Baseline and up to Day 168]
Secondary outcome measures (6)
- Part A: Maximum Observed Concentration (Cmax) of VX-670 and its Active Component in Plasma [Time frame: From Day 1 up to Day 42]
- Part A: Area Under the Concentration Versus Time Curve (AUC) of VX-670 and its Active Component in Plasma [Time frame: From Day 1 up to Day 42]
- Part B: Maximum Observed Concentration (Cmax) of VX-670 and its Active Component in Plasma After Each Dose [Time frame: From Day 1 up to Day 168]
- Part B: Area Under the Concentration Versus Time Curve (AUC) of VX-670 and its Active Component in Plasma After Each Dose [Time frame: From Day 1 up to Day 168]
- Part B: Concentration of VX-670 and its Active Component in Muscle [Time frame: Baseline, Day 15 and Day 120]
- Part B: Change in Splicing Index in Muscle Biopsy [Time frame: Baseline, Day 15 and Day 120]
Eligibility criteria
Inclusion criteria
\- Documented clinical diagnosis of DM1 with age of onset greater than (>) 1 year of age and documented positive genetic test for DM1 in the subject with cytosine thymine guanine (CTG) repeat of at least 100
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
- Randomized
- Model
- Sequential
- Masking
- Quadruple blind
- Primary purpose
- Treatment
Study locations
United States · 8 centers
- Stanford Neuromuscular Research — San Carlos
- University of Florida Clinical Research Center — Gainesville
- University of Kansas Medical Center — Fairway
- Boston Children's Hospital — Boston
- Washington University School of Medicine / St. Louis Children's Hospital — St Louis
- Wake Forest Baptist Health — Winston-Salem
- University of Pennsylvania — Philadelphia
- Virginia Commonwealth University (Sanger Hall) — Richmond
Canada · 5 centers
- Hopital de Chicoutimi — Chicoutimi
- Altasciences Montreal — Montreal
- McGIll University — Montreal
- University of Ottawa — Ottawa
- CHU Research Centre of Quebec — Québec
United Kingdom · 5 centers
- Clinical Research Facility, Queen Elizabeth University Hospital — Glasgow
- Leonard Wolfson Experimental Neurology Centre CRF — London
- St. George's University Hospital — London
- Salford Royal Hospital — Salford
- Royal Hallamshire Hospital — Sheffield
Australia · 2 centers
- Wesley Research Institute — Auchenflower
- Neuroscience Clinical Trials Unit, Alfred Brain — Melbourne
Belgium · 1 center
- Universitaire Ziekenhuizen Leuven - Campus Gasthuisberg — Leuven
France · 1 center
- Neuromuscular Reference Center Institute of Myology — Paris
Germany · 1 center
- Ludwig Maximilians Universitaet Muenchen — München
Italy · 1 center
- Centro Clinico NeMO — Milan
Netherlands · 1 center
- Maastricht University Medical Center — Maastricht
Spain · 1 center
- Hospital Universitario y Politécnico La Fe — Valencia
Identifiers
NCT: NCT06185764 · VX23-670-001 · 2023-506028-10-00