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

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ClinicalTrials.gov Identifier: NCT06138743
Recruitment Status : Recruiting
First Posted : November 18, 2023
Last Update Posted : April 19, 2024
Sponsor:
Information provided by (Responsible Party):
Arrowhead Pharmaceuticals

Study Description
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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.

Condition or disease Intervention/treatment Phase
Myotonic Dystrophy 1 Drug: ARO-DM1 for Injection Drug: Placebo Phase 1

Study Design
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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 48 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
Official 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
Actual Study Start Date : March 4, 2024
Estimated Primary Completion Date : October 2025
Estimated Study Completion Date : October 2026

Resource links provided by the National Library of Medicine


Arms and Interventions
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Arm Intervention/treatment
Experimental: ARO-DM1
ARO-DM1 for Injection
 Drug: ARO-DM1 for Injection
single or multiple doses of ARO-DM1 by intravenous (IV) infusion
Placebo Comparator: Placebo
(0.9% NaCl)
 Drug: Placebo
calculated volume to match active treatment by IV infusion


Outcome Measures
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Primary Outcome Measures :
  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; Multiple dose phase (Part 2): Up to Day 180 or Day 360 ]

Secondary Outcome Measures :
  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: Time to Maximum Observed Plasma Concentration (Tmax) [ 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 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 ]
  4. 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 ]
  5. 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 ]
  6. PK of ARO-DM1: Elimination half-life (t1/2) [ 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 ]
  7. PK of ARO-DM1: Apparent Systemic Clearance (CL/F) [ 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 ]
  8. PK of ARO-DM1:m Apparent Terminal-phase Volume of Distribution (Vz/F) [ 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 ]

Eligibility Criteria
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Ages Eligible for Study:   18 Years to 65 Years   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
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

Contacts and Locations
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Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT06138743


Contacts
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Contact: Medical Monitor 626-304-3400 ARO-DM1@arrowheadpharma.com

Locations
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Australia, Queensland
Research Site Recruiting
Herston, Queensland, Australia, 4006
New Zealand
Research Site Recruiting
Christchurch, New Zealand, 8011
Sponsors and Collaborators
Arrowhead Pharmaceuticals
More Information
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Responsible Party: Arrowhead Pharmaceuticals
ClinicalTrials.gov Identifier: NCT06138743    
Other Study ID Numbers: ARODM1-1001
First Posted: November 18, 2023    Key Record Dates
Last Update Posted: April 19, 2024
Last Verified: March 2024
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Product Manufactured in and Exported from the U.S.: Yes
Additional relevant MeSH terms:
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Myotonic Dystrophy
Muscular Dystrophies
Muscular Disorders, Atrophic
Muscular Diseases
Musculoskeletal Diseases
Myotonic Disorders
 Heredodegenerative Disorders, Nervous System
Neurodegenerative Diseases
Nervous System Diseases
Neuromuscular Diseases
Genetic Diseases, Inborn