Myotonic Dystrophies

Myotonic dystrophies are inherited neuromuscular disorders characterized by progressive muscular wasting (dystrophy) and muscular stiffness (myotonia). It affects both muscles and others organs such as heart, eye, breathing appartus, endocrine system, digestive tract or nervous system, these are multisystem diseases.

Clinical trials on MD ongoing at the Institute:

  • DYNE101-DM1-201: A Randomized, Placebo-Controlled, Multiple Ascending Dose Study Assessing Safety, Tolerability, Pharmacodynamics, Efficacy, and Pharmacokinetics of DYNE-101 Administered to Participants with Myotonic Dystrophy Type 1
  • PIP7 (MEX-NM-303): Open-label Extension Study to Evaluate the Long-term Safety and Efficacy of Mexiletine in Paediatric Patients with Myotonic Disorders Who Have Completed MEX-NM-301 Study
  • ArthemiR CT-ATX-01-DM1-1.1: A Phase 1/2a Double-Blind, Placebo-controlled, Single- and Multiple Ascending Dose Study to Assess the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics and Preliminary Clinical Efficacy of Intravenous Administration of ATX-01 In Male and Female Participants aged 18 to 64 with Classic Myotonic Dystrophy Type 1 (DM1)
  • Vertex VX23-670-001: Phase 1/2, Randomized, Double-blind, Placebo‑controlled Single- and Multiple‑dose, Escalation Study Evaluating the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics ofVX‑670 in Adult Subjects with Myotonic Dystrophy Type 1
  • Avidity AOC 1001-CS3: A Phase 3 Randomized, Double-Blind, Placebo-Controlled, Global Study to Evaluate the Efficacy and Safety of Intravenous AOC 1001 for the Treatment of Myotonic Dystrophy Type 1

 

Acronym

DYNE101-DM1-201

PIP7 (MEX-NM-303)

Intervention DYNE 101 Namuscla ™ (Mexiletine)
Pathology DM1 Myotonic Disorders
Principal investigator
Guillaume Bassez Arnaud Isapof
Sponsor DYNE Therapeutics Lupin
Study status Active Ongoing
Recruitment status Ongoing Closed
Population Adult Child
+ infos on clinicaltrilas.gov + infos on clinicaltrials.gov

 

Acronym

ArthemiR CT-ATX-01-DM1-1.1

Vertex VX23-670-001

Avidity AOC 1001-CS3

Intervention ATX-01 VX-670 AOC 1001
Pathology DM1 DM1 DM1
Principal investigator
Guillaume Bassez Guillaume Bassez Guillaume Bassez
Sponsor ARTHEx Biotech S. L. Vertex Avidity Biosciences
Study status Ongoing Ongoing Ongoing
Recruitment status Recruiting Not started Recruiting
Population Adult Adult Adult
+ infos on clinicaltrilas.gov + infos on clinicaltrials.gov + infos on clinicaltrials.gov

Contact : essais-adultes@institut-myologie.org