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
Status: Recruiting
Location: See all (26) locations...
Intervention Type: Drug
Study Type: Interventional
Study Phase: Phase 1/Phase 2
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
Participation Requirements
Sex: All
Minimum Age: 18
Maximum Age: 64
Healthy Volunteers: f
View:
• \- 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
Locations
United States
California
Stanford Neuromuscular Research
RECRUITING
San Carlos
Florida
University of Florida Clinical Research Center
RECRUITING
Gainesville
Kansas
University of Kansas Medical Center
RECRUITING
Fairway
Massachusetts
Boston Children's Hospital
RECRUITING
Boston
Missouri
Washington University School of Medicine / St. Louis Children's Hospital
RECRUITING
St Louis
North Carolina
Wake Forest Baptist Health
RECRUITING
Winston-salem
Pennsylvania
University of Pennsylvania
RECRUITING
Philadelphia
Virginia
Virginia Commonwealth University (Sanger Hall)
RECRUITING
Richmond
Other Locations
Australia
Wesley Research Institute
RECRUITING
Auchenflower
Neuroscience Clinical Trials Unit, Alfred Brain
RECRUITING
Melbourne
Belgium
Universitaire Ziekenhuizen Leuven - Campus Gasthuisberg
RECRUITING
Leuven
Canada
Hopital de Chicoutimi
RECRUITING
Chicoutimi
Altasciences Montreal
RECRUITING
Montreal
McGIll University
RECRUITING
Montreal
University of Ottawa
RECRUITING
Ottawa
CHU Research Centre of Quebec
RECRUITING
Québec
France
Neuromuscular Reference Center Institute of Myology
RECRUITING
Paris
Germany
Ludwig Maximilians Universitaet Muenchen
RECRUITING
München
Italy
Centro Clinico NeMO
RECRUITING
Milan
Netherlands
Maastricht University Medical Center
RECRUITING
Maastricht
Spain
Hospital Universitario y Politécnico La Fe
RECRUITING
Valencia
United Kingdom
Clinical Research Facility, Queen Elizabeth University Hospital
RECRUITING
Glasgow
Leonard Wolfson Experimental Neurology Centre CRF
RECRUITING
London
St. George's University Hospital
RECRUITING
London
Salford Royal Hospital
RECRUITING
Salford
Royal Hallamshire Hospital
RECRUITING
Sheffield
Contact Information
Primary
Medical Information
medicalinfo@vrtx.com
617-341-6777
Time Frame
Start Date: 2024-02-20
Estimated Completion Date: 2026-12-31
Participants
Target number of participants: 36
Treatments
Experimental: Part A: Single Ascending Dose
Participants will be randomized to receive a single dose of different dose levels of VX-670.
Placebo_comparator: Part A: Placebo
Participants will be randomized to receive single dose of placebo matched to VX-670.
Experimental: Part B: Single and Multiple Ascending Dose
Participants will be randomized to receive single and multiple doses of different dose levels of VX-670. The dose levels will be determined based on the data from Part A.
Placebo_comparator: Part B: Placebo
Participants will be randomized to receive single or multiple doses of placebo matched to VX-670.
Related Therapeutic Areas
Sponsors
Leads: Vertex Pharmaceuticals Incorporated