NCT06138743

Brief 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.

Trial Health

80
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
78

participants targeted

Target at P75+ for phase_1

Timeline
7mo left

Started Mar 2024

Typical duration for phase_1

Geographic Reach
4 countries

11 active sites

Status
recruiting

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

Study Progress79%
Mar 2024Dec 2026

First Submitted

Initial submission to the registry

November 14, 2023

Completed
4 days until next milestone

First Posted

Study publicly available on registry

November 18, 2023

Completed
4 months until next milestone

Study Start

First participant enrolled

March 4, 2024

Completed
2.7 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

December 1, 2026

Expected
Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

December 1, 2026

Last Updated

November 10, 2025

Status Verified

November 1, 2025

Enrollment Period

2.7 years

First QC Date

November 14, 2023

Last Update Submit

November 6, 2025

Conditions

Myotonic Dystrophy 1

Outcome Measures

Primary Outcomes (1)

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

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

Secondary Outcomes (11)

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

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

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

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

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

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

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

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

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

    (Part 2): Baseline, Day 120

  • +6 more secondary outcomes

Study Arms (4)

ARO-DM1 Intravenous (IV) Infusion

EXPERIMENTAL

Single or multiple doses of ARO-DM1 by IV infusion

Drug: ARO-DM1 Intravenous (IV) Infusion

Placebo by IV Infusion

PLACEBO COMPARATOR

Single or multiple doses of placebo by IV infusion

Drug: Placebo Intravenous (IV) Infusion

ARO-DM1 Subcutaneous SC Injection

EXPERIMENTAL

Single or multiple doses of ARO-DM1 by sc injection

Drug: ARO-DM1 subcutaneous (SC) injection

Placebo by SC Injection

PLACEBO COMPARATOR

Single or multiple doses of placebo by sc injection

Drug: Placebo Subcutaneous (SC) Injection

Interventions

ARO-DM1 Intravenous (IV) InfusionDRUG

ARO-DM1 by intravenous (IV) infusion

ARO-DM1 Intravenous (IV) Infusion
Placebo Intravenous (IV) InfusionDRUG

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

Placebo by IV Infusion
ARO-DM1 subcutaneous (SC) injectionDRUG

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

ARO-DM1 Subcutaneous SC Injection
Placebo Subcutaneous (SC) InjectionDRUG

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

Placebo by SC Injection

Eligibility Criteria

Age18 Years - 65 Years
Sexall
Healthy VolunteersNo
Age GroupsAdult (18-64), Older Adult (65+)

You may qualify if:

  • 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.

You may not qualify if:

  • 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)

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (11)

Research Site

Liverpool, New South Wales, 2170, Australia

RECRUITING

Research Site

Birtinya, Queensland, 4575, Australia

RECRUITING

Research Site

Herston, Queensland, 4029, Australia

RECRUITING

Research Site

Melbourne, Victoria, 3004, Australia

RECRUITING

Research Site

Christchurch, 8011, New Zealand

RECRUITING

Research Site

Taichung, 40447, Taiwan

RECRUITING

Research Site

Taipei, 10041, Taiwan

RECRUITING

Research Site

Taipei, 11217, Taiwan

RECRUITING

Research Site

Bangkok, Bangkok, 10700, Thailand

RECRUITING

Research Site

Hat Yai, Changwat Songkhla, 90110, Thailand

RECRUITING

Research Site

Lampang, 52000, Thailand

RECRUITING

MeSH Terms

Conditions

Myotonic Dystrophy

Interventions

Injections

Condition Hierarchy (Ancestors)

Muscular DystrophiesMuscular Disorders, AtrophicMuscular DiseasesMusculoskeletal DiseasesMyotonic DisordersHeredodegenerative Disorders, Nervous SystemNeurodegenerative DiseasesNervous System DiseasesNeuromuscular DiseasesGenetic Diseases, InbornCongenital, Hereditary, and Neonatal Diseases and Abnormalities

Intervention Hierarchy (Ancestors)

Drug Administration RoutesDrug TherapyTherapeutics

Central Study Contacts

Medical Monitor

CONTACT

626-304-3400ARO-DM1@arrowheadpharma.com

Study Design

Study Type
interventional
Phase
phase 1
Allocation
RANDOMIZED
Masking
QUADRUPLE
Who Masked
PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
Purpose
TREATMENT
Intervention Model
PARALLEL
Sponsor Type
INDUSTRY
Responsible Party
SPONSOR

Study Record Dates

First Submitted

November 14, 2023

First Posted

November 18, 2023

Study Start

March 4, 2024

Primary Completion (Estimated)

December 1, 2026

Study Completion (Estimated)

December 1, 2026

Last Updated

November 10, 2025

Record last verified: 2025-11

Data Sharing

IPD Sharing
Will not share

Locations

NZ(1)TH(3)TW(3)AU(4)