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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

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Trial Profile

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

Status: Recruiting
Phase of Trial: Phase I

Latest Information Update: 06 Mar 2025

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At a glance

  • Drugs SRP 1003 (Primary)
  • Indications Myotonic dystrophy
  • Focus Adverse reactions
  • Sponsors Arrowhead Pharmaceuticals

    • Most Recent Events

    • 26 Feb 2025 According to a Sarepta Therapeutics media release, this trial is one of the clinical-stage programs covered under the collaboration agreement with Arrowhead Pharmaceuticals, under which Sarepta has obtained exclusive global rights from Arrowhead to four clinical-stage and three preclinical-stage programs.
    • 10 Feb 2025 According to Arrowhead Pharmaceuticals media release,the company has the potential to receive $300 million in near-term payments associated with the continued enrollment of certain cohorts of a Phase 1/2 study of ARO-DM1, which Arrowhead is on track to achieve during the next 12 months.
    • 01 Oct 2024 Planned number of patients changed from 48 to 36.

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