Overview

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

Status:
Recruiting
Trial end date:
2026-10-01
Target enrollment:
0
Participant gender:
All
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.
Phase:
Phase 1
Accepts Healthy Volunteers?
No
Details
Lead Sponsor:
Arrowhead Pharmaceuticals
Criteria
Inclusion Criteria:

- Genetically confirmed diagnosis of DM1

- Clinician-assessed signed of DM1 including clinically apparent myotonia

- Onset of DM1 symptoms occurred after the age of 12 years

- Walk for at least 10 meters independently at Screening

- Subjects of childbearing potential must agree to use highly effective contraception in
addition to a condom during the study and for at least 90 days following the end of
study or last dose of study drug, whichever is later. Subjects must not donate sperm
or eggs during the study and for at least 90 days following the end of study or last
dose of study drug whichever is later.

Exclusion Criteria:

- Inadequately controlled diabetes

- Confirmed diagnosis of congenital DM1

- Uncontrolled hypertension

- History of Tibialis Anterior (TA) biopsy within 3 months of Day 1 or planning to
undergo TA biopsies during the study period

- Clinically significant cardiac, liver or renal disease

- HIV infection (seropositive) at Screening

- Seropositive for hepatitis B (HBV) or hepatitis C (HCV) at screening

- Untreated or poorly controlled epilepsy

- Treatment with anti-myotonia medication within a period of 5 half-lives of the
medication prior to Screening.

Note: Additional inclusion/exclusion criteria may apply per protocol