Myotonic Dystrophy Type 1

Condition Description

Myotonic dystrophy type 1 (DM1) is the chronic neuromuscular disease with the most prominent sleep disorders, including excessive daytime sleepiness (EDS), sleep apneas, periodic leg movements during sleep, and rapid eye movement sleep dysregulation.

Active Trials

  • TRIAL TITLE: A Phase 1 Placebo-Controlled Study to Assess the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of Single-Ascending Doses of PGN-EDODM1 in Adult Participants with Myotonic Dystrophy Type 1.

    DESCRIPTION: This is a Phase 1, multi-center, randomized, double-blind, placebo-controlled, single-ascending dose (SAD) study to evaluate safety, tolerability, PK, and PD of PGN-EDODM1 in adults with DM1. The study will be conducted in 3 cohorts consisting of 8 participants per cohort who will be randomized to receive a single dose of PGN-EDODM1 or placebo in a 3:1 ratio (6 active, 2 placebo).

    SPONSOR: PepGen Inc.

    INDICATION: Myotonic Dystrophy Type 1

    STUDY PROTOCOL: PGN-EDODM1-101

    PHASE: 1

    STATUS: Active

    RECRUITING PATIENTS: Yes

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Upcoming Trials

  • TRIAL 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

    DESCRIPTION: This 2-part, randomized, placebo-controlled study will evaluate the safety, tolerability, PK, and PD of single- and multiple ascending doses of VX-670 and PMO-0221a in male and female subjects with DM1. Approximately 36 subjects will be enrolled.

    SPONSOR: Vertex Pharmaceuticals Incorporated

    INDICATION: Myotonic Dystrophy Type 1

    STUDY PROTOCOL: VX23-670-001

    PHASE: 1/2

    STATUS: Pending site activation

    RECRUITING PATIENTS: Yes

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