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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Past Trials

  • TRIAL TITLE: Safety and Efficacy of Pitolisant on Excessive Daytime Sleepiness and Other Non-Muscular Symptoms in Patients With Myotonic Dystrophy Type 1

    DESCRIPTION: The primary objective of this study is to evaluate the safety and efficacy of pitolisant compared with placebo in treating excessive daytime sleepiness (EDS) in patients with Myotonic Dystrophy Type 1 ages 18 to 65 years. The secondary objectives of this study are to assess the impact of pitolisant on fatigue, cognitive function and the burden of disease along with assessing the long-term safety and effectiveness of pitolisant in patients with Myotonic Dystrophy Type 1 ages 18 to 65 years.

    SPONSOR: Harmony Biosciences, LLC

    INDICATION: Myotonic Dystrophy 1, Excessive Daytime Sleepiness

    STUDY PROTOCOL: HBS-101-CL-005

    PHASE: 2

    STATUS: Complete

    RECRUITING PATIENTS: No

  • TRIAL TITLE: A Study to Assess Video Hand Opening Time and Ambulation in Healthy Volunteers and Individuals with Myotonic Dystrophy Type 1.

    DESCRIPTION: This is a multi-center, non-interventional study in individuals with DM1 who are currently ambulant and healthy volunteers. The study will evaluate vHOT in clinic and remote settings as a measurement of myotonia and will evaluate 10MWT, 10MWR, and a wearable device to assess ambulation.

    SPONSOR: Vertex Pharmaceuticals Incorporated

    INDICATION: Myotonic Dystrophy Type 1

    STUDY PROTOCOL: VX22-MDO-801

    PHASE: Non-Interventional

    STATUS: Inactive

    RECRUITING PATIENTS: No