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 2, Open-label, Fixed-dose Study to Assess the Efficacy of Sodium Phenylbutyrate (ACER-001) in Treating Pediatric and Adult Patients with Medium Chain Acyl-CoA Dehydrogenase (MCAD) Deficiency Resulting from the Prevalent ACADM C.985 A>G (K304E) Mutation

Next Previous
X
Search
Trial Profile

A Phase 2, Open-label, Fixed-dose Study to Assess the Efficacy of Sodium Phenylbutyrate (ACER-001) in Treating Pediatric and Adult Patients with Medium Chain Acyl-CoA Dehydrogenase (MCAD) Deficiency Resulting from the Prevalent ACADM C.985 A>G (K304E) Mutation

Status: Recruiting
Phase of Trial: Phase II

Latest Information Update: 13 Mar 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 Sodium phenylbutyrate (Primary)
  • Indications Medium chain acyl CoA dehydrogenase deficiency
  • Focus Adverse reactions

    • Most Recent Events

    • 05 Mar 2025 Planned End Date changed from 1 Jul 2026 to 1 Jan 2027.
    • 05 Mar 2025 Planned primary completion date changed from 1 Apr 2026 to 1 Sep 2026.
    • 05 Mar 2025 Planned initiation date changed from 1 Feb 2025 to 1 Apr 2025.

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

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
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