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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/II

Latest Information Update: 29 Aug 2025

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At a glance

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

Most Recent Events

  • 12 Aug 2025 Planned number of patients changed from 36 to 90.
  • 12 Aug 2025 Planned End Date changed from 1 Jun 2025 to 1 Sep 2026.
  • 12 Aug 2025 Planned primary completion date changed from 1 Jun 2025 to 1 Sep 2026.

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