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Trial details imported from ClinicalTrials.gov
For full trial details, please see the original record at
https://clinicaltrials.gov/study/NCT06185764
Registration number
NCT06185764
Ethics application status
Date submitted
15/12/2023
Date registered
29/12/2023
Date last updated
18/06/2024
Titles & IDs
Public title
A Phase 1/2 Study of VX-670 in Adult Participants With Myotonic Dystrophy 1 (DM1)
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Scientific 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
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Secondary ID [1]
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2023-506028-10-00
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Secondary ID [2]
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VX23-670-001
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Universal Trial Number (UTN)
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Trial acronym
Galileo
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Linked study record
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Health condition
Health condition(s) or problem(s) studied:
Myotonic Dystrophy Type 1 (DM1)
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Condition category
Condition code
Musculoskeletal
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Other muscular and skeletal disorders
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Intervention/exposure
Study type
Interventional
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Description of intervention(s) / exposure
Treatment: Drugs - VX-670
Treatment: Drugs - Placebo
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.
Treatment: Drugs: VX-670
Solution for intravenous administration.
Treatment: Drugs: Placebo
Solution for intravenous administration.
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Intervention code [1]
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Treatment: Drugs
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Comparator / control treatment
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Control group
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Outcomes
Primary outcome [1]
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Parts A and B: Safety and Tolerability as Assessed by Number of Participants With Adverse Events (AEs)
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Assessment method [1]
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Timepoint [1]
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Part A: From Baseline up to Day 42; Part B: From Baseline and up to Day 168
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Secondary outcome [1]
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Part A: Maximum Observed Concentration (Cmax) of VX-670 and its Active Component in Plasma
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Assessment method [1]
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Timepoint [1]
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From Day 1 up to Day 42
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Secondary outcome [2]
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Part A: Area Under the Concentration Versus Time Curve (AUC) of VX-670 and its Active Component in Plasma
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Assessment method [2]
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Timepoint [2]
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From Day 1 up to Day 42
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Secondary outcome [3]
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Part B: Maximum Observed Concentration (Cmax) of VX-670 and its Active Component in Plasma After Each Dose
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Assessment method [3]
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Timepoint [3]
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From Day 1 up to Day 168
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Secondary outcome [4]
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Part B: Area Under the Concentration Versus Time Curve (AUC) of VX-670 and its Active Component in Plasma After Each Dose
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Assessment method [4]
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Timepoint [4]
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From Day 1 up to Day 168
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Secondary outcome [5]
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Part B: Concentration of VX-670 and its Active Component in Muscle
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Assessment method [5]
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Timepoint [5]
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Baseline and at Day 15
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Secondary outcome [6]
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Part B: Change in Splicing Index in Muscle Biopsy
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Assessment method [6]
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Timepoint [6]
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Baseline and at Day 15
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Eligibility
Key inclusion criteria
Key
- 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
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Minimum age
18
Years
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Maximum age
64
Years
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Sex
Both males and females
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Can healthy volunteers participate?
No
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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.
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Study design
Purpose of the study
Treatment
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Allocation to intervention
Randomised controlled trial
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Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
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Methods used to generate the sequence in which subjects will be randomised (sequence generation)
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Masking / blinding
Blinded (masking used)
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Who is / are masked / blinded?
The people receiving the treatment/s
The people administering the treatment/s
The people assessing the outcomes
The people analysing the results/data
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Intervention assignment
Other
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Other design features
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Phase
Phase 1
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Type of endpoint/s
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Statistical methods / analysis
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Recruitment
Recruitment status
Recruiting
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Data analysis
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Reason for early stopping/withdrawal
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Other reasons
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Date of first participant enrolment
Anticipated
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Actual
20/02/2024
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Date of last participant enrolment
Anticipated
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Actual
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Date of last data collection
Anticipated
1/12/2026
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Actual
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Sample size
Target
36
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Accrual to date
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Final
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Recruitment in Australia
Recruitment state(s)
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Recruitment hospital [1]
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Wesley Research Institute - Auchenflower
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Recruitment hospital [2]
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Neuroscience Clinical Trials Unit, Alfred Brain - Melbourne
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Recruitment postcode(s) [1]
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- Auchenflower
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Recruitment postcode(s) [2]
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- Melbourne
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Recruitment outside Australia
Country [1]
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Canada
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State/province [1]
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Chicoutimi
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Country [2]
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Canada
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State/province [2]
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Mont-Royal
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Country [3]
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Canada
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State/province [3]
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Montreal
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Country [4]
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Canada
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State/province [4]
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Ottawa
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Country [5]
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Canada
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State/province [5]
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Quebec
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Country [6]
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United Kingdom
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State/province [6]
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London
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Funding & Sponsors
Primary sponsor type
Commercial sector/industry
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Name
Vertex Pharmaceuticals Incorporated
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Address
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Country
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Ethics approval
Ethics application status
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Summary
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.
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Trial website
https://clinicaltrials.gov/study/NCT06185764
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Trial related presentations / publications
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Public notes
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Contacts
Principal investigator
Name
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Address
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Country
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Phone
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Fax
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Email
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Contact person for public queries
Name
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Medical Information
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Address
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Country
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Phone
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617-341-6777
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Fax
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Email
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[email protected]
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Contact person for scientific queries
Summary Results
For IPD and results data, please see
https://clinicaltrials.gov/study/NCT06185764
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