A Phase 1/2 Study of VX-670 in Adult Participants With Myotonic Dystrophy 1 (DM1)

Myotonic Dystrophy Type 1 (DM1) Clinical Trial

— Galileo  

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

Clinical Trial Details — Status: Recruiting

Administrative data

• NCT number: NCT06185764
• Other study ID #: VX23-670-001
• Secondary ID: 2023-506028-10-0
• Status: Recruiting
• Phase: Phase 1/Phase 2
• Start date: February 20, 2024
• Est. completion date: December 2026

Study information

• Verified date: December 2023
• Source: Vertex Pharmaceuticals Incorporated
• Contact: Medical Information
• Phone: 617-341-6777
• Email: medicalinfo[@]vrtx.com
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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.

Recruitment information / eligibility

• Status: Recruiting
• Enrollment: 36
• Est. completion date: December 2026
• Est. primary completion date: December 2026
• Accepts healthy volunteers: No
• Gender: All
• Age group: 18 Years to 64 Years

Eligibility

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.

Related Conditions & MeSH terms

• Myotonic Dystrophy
• Myotonic Dystrophy Type 1 (DM1)

Intervention

Drug:
• VX-670
Solution for intravenous administration.
• Placebo
Solution for intravenous administration.

Locations

Country	Name	City	State
Australia	Wesley Research Institute	Auchenflower
Australia	Neuroscience Clinical Trials Unit, Alfred Brain	Melbourne
Canada	Hopital de Chicoutimi	Chicoutimi
Canada	Altasciences Montreal	Mont-Royal
Canada	McGill University	Montreal
Canada	University of Ottawa	Ottawa
Canada	CHU Research Centre of Quebec	Quebec
United Kingdom	St. George's University Hospital	London
United Kingdom	University College London	London

Sponsors (1)

Lead Sponsor	Collaborator
Vertex Pharmaceuticals Incorporated

Countries where clinical trial is conducted

Australia,  Canada,  United Kingdom, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Parts A and B: Safety and Tolerability as Assessed by Number of Participants With Adverse Events (AEs)		Part A: From Baseline up to Day 42; Part B: From Baseline and up to Day 168
Secondary	Part A: Maximum Observed Concentration (Cmax) of VX-670 and its Active Component in Plasma		From Day 1 up to Day 42
Secondary	Part A: Area Under the Concentration Versus Time Curve (AUC) of VX-670 and its Active Component in Plasma		From Day 1 up to Day 42
Secondary	Part B: Maximum Observed Concentration (Cmax) of VX-670 and its Active Component in Plasma After Each Dose		From Day 1 up to Day 168
Secondary	Part B: Area Under the Concentration Versus Time Curve (AUC) of VX-670 and its Active Component in Plasma After Each Dose		From Day 1 up to Day 168
Secondary	Part B: Concentration of VX-670 and its Active Component in Muscle		Baseline and at Day 15
Secondary	Part B: Change in Splicing Index in Muscle Biopsy		Baseline and at Day 15