Trial Profile

A Phase 3, Randomised, Placebo-Controlled Trial of Arimoclomol in Amyotropic Lateral Sclerosis

Status: Completed

Phase of Trial: Phase III

Latest Information Update: 01 Sep 2026

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 Arimoclomol (Primary)
Indications Amyotrophic lateral sclerosis
Focus Registrational; Therapeutic Use
Acronyms ORARIALS-01
Sponsors Orphazyme; Zevra Therapeutics

Most Recent Events

25 Aug 2026 According to a Zevra Therapeutics media release, data from this trial to be presented in posters featuring long-term real-world data on MIPLYFFA (arimoclomol) for the treatment of Niemann-Pick disease type C (NPC) at the Society for the Study of Inborn Errors of Metabolism (SSIEM) Annual Symposium.
01 Jul 2024 Results evaluating the safety and efficacy of arimoclomol in patients with amyotrophic lateral sclerosis, published in the Lancet Neurology
07 May 2021 Results published in the Orphazyme media release.

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

If your organization or you do not have a subscription, try one of the following:

Contacting your organization’s admin about adding this content to your AdisInsight subscription
Buying a PDF version of any individual profile
Request a free trial (opens in a new window)

If your organization has a subscription, there are several access options, even while working remotely:

Working within your organization’s network
Login with username/password or try to access via your institution
Persisted access using your organization’s identifier stored in your user browser for 90 days

For assistance, contact us at asktheexpert.adisinsight@springer.com