NCT06138743

Study of ARO-DM1 in Subjects With Type 1 Myotonic Dystrophy

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: Recruiting
• Phase: Phase 1 / Phase 2
• Study type: Interventional
• Enrollment: 78 (estimated)
• Sponsor: Arrowhead Pharmaceuticals · Industry
• Sex: All
• Age: 18 Years – 65 Years
• Healthy volunteers: Not accepted

Summary

This is a phase 1/2a double-blinded, placebo-controlled, dose-escalating study to evaluate the safety, tolerability, pharmacokinetics (PK) and pharmacodynamics (PD) of single and multiple ascending doses of ARO-DM1 compared to placebo in male and female subjects with type 1 myotonic dystrophy (DM1). Participants who have provided written informed consent and met all protocol eligibility requirements will be randomized to receive single (Part 1) or multiple (Part 2) doses of ARO-DM1 or placebo.

Conditions

• Myotonic Dystrophy 1

Interventions

Type	Name	Description
DRUG	ARO-DM1 Intravenous (IV) Infusion	ARO-DM1 by intravenous (IV) infusion
DRUG	Placebo Intravenous (IV) Infusion	0.9% NaCl calculated volume to match active treatment by IV infusion
DRUG	ARO-DM1 subcutaneous (SC) injection	ARO-DM1 by subcutaneous (SC) injection(s)
DRUG	Placebo Subcutaneous (SC) Injection	0.9% NaCl calculated volume to match active treatment by SC injection(s)

Timeline

• Start date: 2024-03-04
• Primary completion: 2026-12-01
• Completion: 2026-12-01
• First posted: 2023-11-18
• Last updated: 2025-11-10

Locations

11 sites across 4 countries: Australia, New Zealand, Taiwan, Thailand

Regulatory

• FDA-regulated drug study