- ICH GCP
- US Clinical Trials Registry
- Clinical Trial NCT06185764
A Phase 1/2 Study of VX-670 in Adult Participants With Myotonic Dystrophy 1 (DM1) (Galileo)
April 10, 2024 updated by: Vertex Pharmaceuticals Incorporated
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
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.
Study Overview
Status
Recruiting
Conditions
Intervention / Treatment
Study Type
Interventional
Enrollment (Estimated)
36
Phase
- Phase 2
- Phase 1
Contacts and Locations
This section provides the contact details for those conducting the study, and information on where this study is being conducted.
Study Contact
- Name: Medical Information
- Phone Number: 617-341-6777
- Email: medicalinfo@vrtx.com
Study Locations
-
-
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Mont-Royal, Canada
- Recruiting
- Altasciences Montreal
-
Montreal, Canada
- Recruiting
- McGill University
-
Ottawa, Canada
- Recruiting
- University of Ottawa
-
-
Participation Criteria
Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.
Eligibility Criteria
Ages Eligible for Study
- Adult
Accepts Healthy Volunteers
No
Description
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.
Study Plan
This section provides details of the study plan, including how the study is designed and what the study is measuring.
How is the study designed?
Design Details
- Primary Purpose: Treatment
- Allocation: Randomized
- Interventional Model: Sequential Assignment
- Masking: Quadruple
Arms and Interventions
Participant Group / Arm |
Intervention / Treatment |
---|---|
Experimental: Part A: Single Ascending Dose
Participants will be randomized to receive a single dose of different dose levels of VX-670.
|
Solution for intravenous administration.
|
Placebo Comparator: Part A: Placebo
Participants will be randomized to receive single dose of placebo matched to VX-670.
|
Solution for intravenous administration.
|
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.
|
Solution for intravenous administration.
|
Placebo Comparator: Part B: Placebo
Participants will be randomized to receive single or multiple doses of placebo matched to VX-670.
|
Solution for intravenous administration.
|
What is the study measuring?
Primary Outcome Measures
Outcome Measure |
Time Frame |
---|---|
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
|
Part A: From Baseline up to Day 42; Part B: From Baseline and up to Day 168
|
Secondary Outcome Measures
Outcome Measure |
Time Frame |
---|---|
Part A: Maximum Observed Concentration (Cmax) of VX-670 and its Active Component in Plasma
Time Frame: From Day 1 up to Day 42
|
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
|
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
|
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
|
From Day 1 up to Day 168
|
Part B: Concentration of VX-670 and its Active Component in Muscle
Time Frame: Baseline and at Day 15
|
Baseline and at Day 15
|
Part B: Change in Splicing Index in Muscle Biopsy
Time Frame: Baseline and at Day 15
|
Baseline and at Day 15
|
Collaborators and Investigators
This is where you will find people and organizations involved with this study.
Study record dates
These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.
Study Major Dates
Study Start (Actual)
February 20, 2024
Primary Completion (Estimated)
December 1, 2026
Study Completion (Estimated)
December 1, 2026
Study Registration Dates
First Submitted
December 15, 2023
First Submitted That Met QC Criteria
December 15, 2023
First Posted (Actual)
December 29, 2023
Study Record Updates
Last Update Posted (Actual)
April 11, 2024
Last Update Submitted That Met QC Criteria
April 10, 2024
Last Verified
December 1, 2023
More Information
Terms related to this study
Keywords
- Nervous System Diseases
- Musculoskeletal Diseases
- Genetic Diseases, Inborn
- Neurodegenerative Diseases
- Neuromuscular Diseases
- Myotonic Dystrophy
- DM1
- Muscular Diseases
- Muscular Dystrophies
- ASO
- DM
- DM2
- Heredodegenerative Disorders, Nervous System
- Steinert Disease
- Myotonic Dystrophy Type 1 (DM1)
- Myotonic Dystrophy 1
- Myotonia
- Dystrophy Myotonic
- Myotonic Disorders
- Myotonic Muscular Dystrophy
- Muscular Disorders, Atrophic
- Myotonic Dystrophy 2
- Vertex
- Entrada
- VX-670
- VX670
- PMO
Additional Relevant MeSH Terms
Other Study ID Numbers
- VX23-670-001
- 2023-506028-10-00 (Other Identifier: EUCT Number)
Plan for Individual participant data (IPD)
Plan to Share Individual Participant Data (IPD)?
NO
IPD Plan Description
Details on Vertex data sharing criteria and process for requesting access can be found at: https://www.vrtx.com/independent-research/clinical-trial-data-sharing
Drug and device information, study documents
Studies a U.S. FDA-regulated drug product
Yes
Studies a U.S. FDA-regulated device product
No
This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.
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