Clinical trial

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

Name

VX23-670-001

Description

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.

Trial arms

Trial start

2024-02-20

Estimated PCD

2026-12-01

Trial end

2026-12-01

Status

Recruiting

Phase

Early phase I

Treatment

VX-670

Solution for intravenous administration.

Arms:

Part A: Single Ascending Dose, Part B: Single and Multiple Ascending Dose

Placebo

Solution for intravenous administration.

Arms:

Part A: Placebo, Part B: Placebo

Size

Primary endpoint

Parts A and B: Safety and Tolerability as Assessed by Number of Participants With Adverse Events (AEs)

Part A: From Baseline up to Day 42; Part B: From Baseline and up to Day 168

Eligibility 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.

Protocol

{'studyType': 'INTERVENTIONAL', 'phases': ['PHASE1', 'PHASE2'], 'designInfo': {'allocation': 'RANDOMIZED', 'interventionModel': 'SEQUENTIAL', 'primaryPurpose': 'TREATMENT', 'maskingInfo': {'masking': 'QUADRUPLE', 'whoMasked': ['PARTICIPANT', 'CARE_PROVIDER', 'INVESTIGATOR', 'OUTCOMES_ASSESSOR']}}, 'enrollmentInfo': {'count': 36, 'type': 'ESTIMATED'}}

Updated at

2024-04-11

1 organization

2 products

1 indication

Organization

Vertex Pharmaceuticals

Product

Placebo

Indication

Myotonic Dystrophy Type 1 (DM1)

Product

VX-670