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
Arrowhead Pharmaceuticals
78 participants
Mar 4, 2024
INTERVENTIONAL
Conditions
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.
Eligibility
Plain Language Summary
Simplified for easier understanding
This summary was AI-generated to explain the trial in plain language. It is not medical advice. Always discuss eligibility with your doctor before enrolling in a clinical trial.
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Interventions
ARO-DM1 by intravenous (IV) infusion
0.9% NaCl calculated volume to match active treatment by IV infusion
ARO-DM1 by subcutaneous (SC) injection(s)
0.9% NaCl calculated volume to match active treatment by SC injection(s)
Locations(11)
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NCT06138743