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

Vertex Pharmaceuticals

Status and phase: Enrolling; Phase 2; Phase 1

Conditions: Myotonic Dystrophy Type 1 (DM1)

Treatments: Drug: Placebo; Drug: VX-670

Study type: Interventional

Funder types: Industry

Identifiers

NCT06185764

VX23-670-001

2023-506028-10-00 (Other Identifier)

Details and patient eligibility

About

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.

Enrollment: 36 estimated patients

Sex: All

Ages: 18 to 64 years old

Volunteers: No Healthy Volunteers

Inclusion and exclusion criteria

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.

Trial design

Primary purpose: Treatment

Allocation: Randomized

Interventional model: Sequential Assignment

Masking: Quadruple Blind

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36 participants in 4 patient groups, including a placebo group

Part A: Single Ascending Dose

Experimental group

Description:

Participants will be randomized to receive a single dose of different dose levels of VX-670.

Treatment:

Drug: VX-670

Part A: Placebo

Placebo Comparator group

Description:

Participants will be randomized to receive single dose of placebo matched to VX-670.

Treatment:

Drug: Placebo

Part B: Single and Multiple Ascending Dose

Experimental group

Description:

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.

Treatment:

Drug: VX-670

Part B: Placebo

Placebo Comparator group

Description:

Participants will be randomized to receive single or multiple doses of placebo matched to VX-670.

Treatment:

Drug: Placebo