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 3 Randomized, Double-Blind, Placebo-Controlled, Global Study to Evaluate the Efficacy and Safety of Intravenous AOC 1001 for the Treatment of Myotonic Dystrophy Type 1

Next Previous
Trial Profile

A Phase 3 Randomized, Double-Blind, Placebo-Controlled, Global Study to Evaluate the Efficacy and Safety of Intravenous AOC 1001 for the Treatment of Myotonic Dystrophy Type 1

Status: Recruiting
Phase of Trial: Phase III

Latest Information Update: 28 May 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 Delpacibart etedesiran (Primary)
  • Indications Myotonic dystrophy
  • Focus Registrational; Therapeutic Use
  • Acronyms HARBOR
  • Sponsors Avidity Biosciences

Most Recent Events

  • 08 Apr 2025 According to an Avidity Biosciences media release, company expects to submit marketing applications starting 2026 in the U.S., European Union and Japan.
  • 08 Apr 2025 According to an Avidity Biosciences media release, company announced that the Japan Ministry of Health, Labour and Welfare (MHLW) has granted Orphan Drug designation (ODD) to delpacibart etedesiran (del-desiran) for the treatment of myotonic dystrophy type 1 (DM1).
  • 12 Mar 2025 According to an Avidity Biosciences media release, trial design from this study will be presented at at the 2025 MDA Clinical & Scientific Conference (MDA) in Dallas, Texas, being held March 16-19, 2025.

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