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

Status

Not yet recruiting

Phases

Phase 1Phase 2

Study type

Interventional

Source

EU CTIS

Registry ID

CTIS2024-513579-42-00

Enrollment

Registered

2026-01-29

Start date

Unknown

Completion date

Unknown

Last updated

2026-01-30

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Type 1 Myotonic Dystrophy

Brief summary

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)

Detailed description

Plasma PK of ARO-DM1 in subjects with DM1.

Interventions

DRUGARO-DM1

DRUGPlacebo - Sodium Chloride 9 mg/ml (0.9%) - Solution for injection

Eligibility

Sex/Gender

All

Age

18 Years to 64 Years

Design outcomes

Primary

Measure	Time frame
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)	—

Secondary

Measure	Time frame
Plasma PK of ARO-DM1 in subjects with DM1.	—

Outcome results

None listed

Source: EU CTIS · Data processed: Feb 4, 2026