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Clinical Trials in the EU / 2024-513579-42-00
Ongoing Phase I/II

A Phase 1/2a Dose-Escalating Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of ARO-DM1 in Subjects With Type 1 Myotonic Dystrophy Who are ≥18 to ≤65 Years

2024-513579-42-00 · tracked via the Priya Life Science EU tracker
Sponsor
Sarepta Therapeutics Inc.
Sponsor type
Pharmaceutical company
Therapeutic area
Nervous System Diseases
Decision date
29/01/2026
Enrollment target
58
Sites
Spain, Germany, Belgium, Italy

Condition studied

Type 1 Myotonic Dystrophy

Investigational medicinal product(s)

ARO-DM1SODIUM CHLORIDE

Eligibility

Age group
18-64 years
Sex
Female, Male

Primary endpoint

Incidence, frequency, and severity of treatment-emergent adverse events (TEAEs) and treatment-related AEs in subjects with DM1 over time through the end of study (EOS)

Endpoint detail

Plasma PK of ARO-DM1 in subjects with DM1.

Official registry record

This page summarises publicly available CTIS data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.

View 2024-513579-42-00 on CTIS ↗ ← All trials in the EU