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

Phase 2Active Not RecruitingNCT06185764

Vertex Pharmaceuticals Incorporated47 enrolled

Overview

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 Type

INTERVENTIONAL

Allocation

RANDOMIZED

Purpose

TREATMENT

Masking

QUADRUPLE

Enrollment

Conditions

Myotonic Dystrophy Type 1 (DM1)

Interventions

VX-670DRUG

Solution for intravenous administration.

PlaceboDRUG

Solution for intravenous administration.

Eligibility

Sex: ALLMin age: 18 YearsMax age: 64 Years

Medical Language ↔ Plain English

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.

Locations (26)

Stanford Neuromuscular Research

San Carlos, California, United States

Outcomes

Primary Outcomes

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

Secondary Outcomes

Part A: Maximum Observed Concentration (Cmax) of VX-670 and its Active Component in Plasma

Time frame: 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

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

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

Part B: Concentration of VX-670 and its Active Component in Muscle

Time frame: Baseline, Day 15 and Day 120

Part B: Change in Splicing Index in Muscle Biopsy

Time frame: Baseline, Day 15 and Day 120

Data from ClinicalTrials.gov

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