Either you have JavaScript disabled or your browser does not support Javascript . To work properly, this page requires JavaScript to be enabled.
How to enable JavaScript in your browser?

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

Next Previous
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: 14 Dec 2025

Price :
$35 *
Note:
  • Adis is an information provider.
  • Final gross price and currency may vary according to local VAT and billing address.
  • Your purchase entitles you to full access to the information contained in our trial profile at the time of purchase.
  • A link to download a PDF version of the trial profile will be included in your email receipt.

At a glance

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

Most Recent Events

  • 24 Nov 2025 According to an Arrowhead pharmaceuticals media release, having achieved the pre-specified patient enrollment target, a second milestone payment of USD dollar 200 million will be made to the company within 60 days. Patients are currently being dosed in cohort 4 (6 mg/kg), with plans to initiate dosing in the final cohort, cohort 5 (12 mg/kg), in early 2026.
  • 24 Nov 2025 According to an Arrowhead pharmaceuticals media release, the company today shared progress of phase 1/2 multiple ascending dose (MAD) clinical study of SRP-1003 for the treatment of type 1 myotonic dystrophy (DM1), reporting that cohorts 1 (1.5 mg/kg) and 2 (3 mg/kg) have been completed, cohort 3 (4.5 mg/kg) is fully enrolled and ongoing, and that, following a positive pre-specified drug safety committee review, the study is advancing to additional dose-escalation cohorts.
  • 24 Nov 2025 According to Arrowhead Pharmaceuticals media release, Accrual of patients in cohort 4 (6 mg/kg) of the multiple ascending dose (MAD) portion of the study is nearly complete and the company intends to initiate enrollment in cohort 5 (12 mg/kg) in the first quarter of 2026.

You need to be a logged in or subscribed to view this content

Request demo (opens in a new window)
If your organization or you do not have a subscription, try one of the following: If your organization has a subscription, there are several access options, even while working remotely:
  • Working within your organization’s network
  • with username/password or try to via your institution
  • Persisted access using your organization’s identifier stored in your user browser for 90 days
Back to top