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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

Titles & IDs
Public title
A Phase 1/2 Study of VX-670 in Adult Participants With Myotonic Dystrophy 1 (DM1)
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
Secondary ID [1] 0 0
2023-506028-10-00
Secondary ID [2] 0 0
VX23-670-001
Universal Trial Number (UTN)
Trial acronym
Galileo
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Myotonic Dystrophy Type 1 (DM1) 0 0
Condition category
Condition code
Musculoskeletal 0 0 0 0
Other muscular and skeletal disorders

Intervention/exposure
Study type
Interventional
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.
Intervention code [1] 0 0
Treatment: Drugs
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Parts A and B: Safety and Tolerability as Assessed by Number of Participants With Adverse Events (AEs)
Timepoint [1] 0 0
Part A: From Baseline up to Day 42; Part B: From Baseline and up to Day 168
Secondary outcome [1] 0 0
Part A: Maximum Observed Concentration (Cmax) of VX-670 and its Active Component in Plasma
Timepoint [1] 0 0
From Day 1 up to Day 42
Secondary outcome [2] 0 0
Part A: Area Under the Concentration Versus Time Curve (AUC) of VX-670 and its Active Component in Plasma
Timepoint [2] 0 0
From Day 1 up to Day 42
Secondary outcome [3] 0 0
Part B: Maximum Observed Concentration (Cmax) of VX-670 and its Active Component in Plasma After Each Dose
Timepoint [3] 0 0
From Day 1 up to Day 168
Secondary outcome [4] 0 0
Part B: Area Under the Concentration Versus Time Curve (AUC) of VX-670 and its Active Component in Plasma After Each Dose
Timepoint [4] 0 0
From Day 1 up to Day 168
Secondary outcome [5] 0 0
Part B: Concentration of VX-670 and its Active Component in Muscle
Timepoint [5] 0 0
Baseline and at Day 15
Secondary outcome [6] 0 0
Part B: Change in Splicing Index in Muscle Biopsy
Timepoint [6] 0 0
Baseline and at Day 15

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

Key
Minimum age
18 Years
Maximum age
64 Years
Sex
Both males and females
Can healthy volunteers participate?
No
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 design
Purpose of the study
Treatment
Allocation to intervention
Randomised controlled trial
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Masking / blinding
Blinded (masking used)
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
Intervention assignment
Other
Other design features
Phase
Phase 1
Type of endpoint/s
Statistical methods / analysis

Recruitment
Recruitment status
Recruiting
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
20/02/2024
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
1/12/2026
Actual
Sample size
Target
36
Accrual to date
Final
Recruitment in Australia
Recruitment state(s)
Recruitment hospital [1] 0 0
Wesley Research Institute - Auchenflower
Recruitment hospital [2] 0 0
Neuroscience Clinical Trials Unit, Alfred Brain - Melbourne
Recruitment postcode(s) [1] 0 0
- Auchenflower
Recruitment postcode(s) [2] 0 0
- Melbourne
Recruitment outside Australia
Country [1] 0 0
Canada
State/province [1] 0 0
Chicoutimi
Country [2] 0 0
Canada
State/province [2] 0 0
Mont-Royal
Country [3] 0 0
Canada
State/province [3] 0 0
Montreal
Country [4] 0 0
Canada
State/province [4] 0 0
Ottawa
Country [5] 0 0
Canada
State/province [5] 0 0
Quebec
Country [6] 0 0
United Kingdom
State/province [6] 0 0
Glasgow
Country [7] 0 0
United Kingdom
State/province [7] 0 0
London
Country [8] 0 0
United Kingdom
State/province [8] 0 0
Salford

Funding & Sponsors
Primary sponsor type
Commercial sector/industry
Name
Vertex Pharmaceuticals Incorporated
Address
Country

Ethics approval
Ethics application status

Summary
Brief summary
Trial website
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 0 0
Address 0 0
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for public queries
Name 0 0
Medical Information
Address 0 0
Country 0 0
Phone 0 0
617-341-6777
Fax 0 0
Email 0 0
medicalinfo@vrtx.com
Contact person for scientific queries

Data sharing statement
Will individual participant data (IPD) for this trial be available (including data dictionaries)?
No
No/undecided IPD sharing reason/comment
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


What supporting documents are/will be available?

No Supporting Document Provided



Results publications and other study-related documents

No documents have been uploaded by study researchers.


Results not provided in https://clinicaltrials.gov/study/NCT06185764