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NCT Number: NCT06138743

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

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.

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Key information

Conditions

Myotonic Dystrophy 1 Congenital, Hereditary, and Neonatal Diseases and Abnormalities Genetic Diseases, Inborn Heredodegenerative Disorders, Nervous System Muscular Diseases Muscular Disorders, Atrophic Muscular Dystrophies Musculoskeletal Diseases Myotonic Disorders Myotonic Dystrophy Nervous System Diseases Neurodegenerative Diseases Neuromuscular Diseases

Age range

18 year–65 year

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 1 / Phase 2

Primary location

Research Site, Liverpool, New South Wales, Australia

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Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

Inclusion criteria

  • Genetically confirmed diagnosis of DM1
  • Clinician-assessed signs 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.
  • Abnormal coagulation parameters at Screening including platelet count, international normalized ratio (INR), prothrombin time, and activated partial thromboplastin time (APTT)

Note: Additional inclusion/exclusion criteria may apply per protocol

Treatment and study plan

ARO-DM1 Intravenous (IV) Infusion

Drug

ARO-DM1 by intravenous (IV) infusion

Placebo Intravenous (IV) Infusion

Drug

0.9% NaCl calculated volume to match active treatment by IV infusion

ARO-DM1 subcutaneous (SC) injection

Drug

ARO-DM1 by subcutaneous (SC) injection(s)

Placebo Subcutaneous (SC) Injection

Drug

0.9% NaCl calculated volume to match active treatment by SC injection(s)

Primary outcomes

  1. Number of Participants with Treatment -Emergent Adverse Events (TEAEs) Over Time Through End of Study (EOS)

    Time frame: Single-dose phase (Part 1): Up to Day 90(EOS); multiple-dose phase (Part 2): Up to Day 180(EOS)

Secondary outcomes

  1. Pharmacokinetics (PK) of ARO-DM1: Maximum Observed Plasma Concentration (Cmax)

    Time frame: Single-dose phase (Part 1): Up 24 hours post-dose; multiple-dose phase (Part 2): Through 24 hours post first and second dose

  2. PK of ARO-DM1: Area Under the Plasma Concentration Versus Time Curve from Zero to 24 Hours (AUC0-24)

    Time frame: Single-dose phase (Part 1): Up 24 hours post-dose; multiple-dose phase (Part 2): Through 24 hours post first and second dose

  3. PK of ARO-DM1: Area Under the Plasma Concentration Versus Time Curve from Zero to the Last Quantifiable Plasma Concentration (AUClast)

    Time frame: Single-dose phase (Part 1): Up 24 hours post-dose; multiple-dose phase (Part 2): Through 24 hours post first and second dose

  4. PK of ARO-DM1: Area Under the Plasma Concentration Versus Time Curve from Zero to Infinity (AUCinf)

    Time frame: Single-dose phase (Part 1): Up 24 hours post-dose; multiple-dose phase (Part 2): Through 24 hours post first and second dose

  5. Change from Baseline at Day 120 for Video Hand Opening Time (vHOT)

    Time frame: (Part 2): Baseline, Day 120

  6. Change from Baseline Over Time for the Timed Up and Go Test (TUG) Assessment

    Time frame: (Part 2): Baseline through EOS (up to 180 days)

  7. Change from Baseline Over Time for the 10-Meter Walk/Run Test (10MWT) Assessment

    Time frame: (Part 2): Baseline through EOS (up to 180 days)

  8. Change from Baseline Over Time for the Hand-held Quantitative Dynamometry Assessment

    Time frame: (Part 2): Baseline through EOS (up to 180 days)

  9. Change from Baseline Over Time for the Video Hand Opening Time (vHOT) Assessment

    Time frame: (Part 2): Baseline through EOS (up to 180 days)

  10. Change from Baseline Over Time for the Myotonic Dystrophy Type 1 Activity and Participation Scale (DM1-Activ-C) Assessment

    Time frame: (Part 2): Baseline through EOS (up to 180 days)

  11. Change from Baseline Over Time for the Myotonic Dystrophy Health Index (MDHI) Assessment

    Time frame: (Part 2): Baseline through EOS (up to 180 days)

Study contacts

Contact information is provided by the study sponsor or research team.

Medical Monitor

CONTACT

[email protected]

626-304-3400

Sponsors and collaborators

Lead sponsor

Arrowhead Pharmaceuticals

Industry

Registry information

Official study title

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

Important dates

Study start
2024
Primary completion
2026
Study completion
2026
First posted
Nov 18, 2023
Registry last updated
Nov 10, 2025

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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