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

NCT06138743 · Status: RECRUITING · Phase: PHASE1/PHASE2 · Type: INTERVENTIONAL · Enrollment: 78

Last updated 2025-11-10

No results posted yet for this study

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

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)

Sponsors & Collaborators

  • Arrowhead Pharmaceuticals

    lead INDUSTRY

Study Design

Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
QUADRUPLE
Model
PARALLEL

Eligibility

Min Age
18 Years
Max Age
65 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2024-03-04
Primary Completion
2026-12-31
Completion
2026-12-31
FDA Drug
Yes

Countries

  • Australia
  • New Zealand
  • Taiwan
  • Thailand

Study Locations

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Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT06138743 on ClinicalTrials.gov