Clinical trial NCT06185764: A Phase 1/2 Study of VX-670 in Adult Participants With Myotonic Dystrophy 1 (DM1)
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
Study type: INTERVENTIONAL
Overall status: RECRUITING
Phase: PHASE1
PHASE2
Conditions: Myotonic Dystrophy Type 1 (DM1)
Keywords: DM1
DM2
Myotonic Dystrophy 1
Myotonic Dystrophy 2
Myotonic Dystrophy Type 1 (DM1)
Myotonic Dystrophy
DM
Myotonia
Dystrophy Myotonic
Myotonic Disorders
Steinert Disease
Vertex
Entrada
VX-670
VX670
PMO
ASO
Myotonic Muscular Dystrophy
Muscular Disorders, Atrophic
Muscular Diseases
Musculoskeletal Diseases
Neuromuscular Diseases
Nervous System Diseases
Genetic Diseases, Inborn
Heredodegenerative Disorders, Nervous System
Neurodegenerative Diseases
Muscular Dystrophies
Lead sponsor: Vertex Pharmaceuticals Incorporated
Sponsor class: INDUSTRY
First posted: 2023-12-29
Enrollment: 36
Sex: ALL
Minimum age (years): 18
Maximum age (years): 64
Standard ages: ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: SEQUENTIAL
Primary purpose: TREATMENT

Brief summary: 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.


Eligibility criteria: Key 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

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.

Primary outcomes:
measure: Parts A and B: Safety and Tolerability as Assessed by Number of Participants With Adverse Events (AEs)
timeFrame: Part A: From Baseline up to Day 42; Part B: From Baseline and up to Day 168
Secondary outcomes:
measure: Part A: Maximum Observed Concentration (Cmax) of VX-670 and its Active Component in Plasma
timeFrame: From Day 1 up to Day 42
measure: Part A: Area Under the Concentration Versus Time Curve (AUC) of VX-670 and its Active Component in Plasma
timeFrame: From Day 1 up to Day 42
measure: Part B: Maximum Observed Concentration (Cmax) of VX-670 and its Active Component in Plasma After Each Dose
timeFrame: From Day 1 up to Day 168
measure: Part B: Area Under the Concentration Versus Time Curve (AUC) of VX-670 and its Active Component in Plasma After Each Dose
timeFrame: From Day 1 up to Day 168
measure: Part B: Concentration of VX-670 and its Active Component in Muscle
timeFrame: Baseline and at Day 15
measure: Part B: Change in Splicing Index in Muscle Biopsy
timeFrame: Baseline and at Day 15
Other outcomes:
Locations:
city: San Carlos
country: United States
facility: Stanford Neuromuscular Research
state: California
city: Gainesville
country: United States
facility: University of Florida Clinical Research Center
state: Florida
city: Fairway
country: United States
facility: University of Kansas Medical Center
state: Kansas
city: Boston
country: United States
facility: Boston Children's Hospital
state: Massachusetts
city: Saint Louis
country: United States
facility: Washington University School of Medicine / St. Louis Children's Hospital
state: Missouri
city: Winston-Salem
country: United States
facility: Wake Forest Baptist Health
state: North Carolina
city: Philadelphia
country: United States
facility: University of Pennsylvania
state: Pennsylvania
city: Richmond
country: United States
facility: Virginia Commonwealth University (Sanger Hall)
state: Virginia
city: Auchenflower
country: Australia
facility: Wesley Research Institute
city: Melbourne
country: Australia
facility: Neuroscience Clinical Trials Unit, Alfred Brain
city: Leuven
country: Belgium
facility: Universitaire Ziekenhuizen Leuven - Campus Gasthuisberg
city: Chicoutimi
country: Canada
facility: Hopital de Chicoutimi
city: Montreal
country: Canada
facility: Altasciences Montreal
city: Montreal
country: Canada
facility: McGIll University
city: Ottawa
country: Canada
facility: University of Ottawa
city: Quebec
country: Canada
facility: CHU Research Centre of Quebec
city: Paris
country: France
facility: Neuromuscular Reference Center Institute of Myology
city: Muenchen
country: Germany
facility: Ludwig Maximilians Universitaet Muenchen
city: Milan
country: Italy
facility: Centro Clinico NeMO
city: Maastricht
country: Netherlands
facility: Maastricht University Medical Center
city: Valencia
country: Spain
facility: Hospital Universitario y Politécnico La Fe
city: Glasgow
country: United Kingdom
facility: Clinical Research Facility, Queen Elizabeth University Hospital
city: London
country: United Kingdom
facility: Leonard Wolfson Experimental Neurology Centre CRF
city: London
country: United Kingdom
facility: St. George's University Hospital
city: Salford
country: United Kingdom
facility: Salford Royal Hospital
city: Sheffield
country: United Kingdom
facility: Royal Hallamshire Hospital
Study officials:
References: