Trial Profile

A Phase IIa Trial to Evaluate the Safety, Tolerability, and Biological Activity of LAM-002A (Apilimod Dimesylate Capsules) in C9ORF72-Associated Amyotrophic Lateral Sclerosis

Status: Completed

Phase of Trial: Phase II

Latest Information Update: 27 Jun 2025

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At a glance

Drugs Apilimod dimesylate (Primary)
Indications Amyotrophic lateral sclerosis
Focus Adverse reactions; Pharmacokinetics
Sponsors OrphAI Therapeutics

Most Recent Events

03 Sep 2024 Results published in the Brain.
20 Aug 2024 Planned End Date changed from 10 May 2024 to 10 Oct 2024.
24 Jan 2024 According to an OrphAI Therapeutics media release, Status changed from active, no longer recruiting to completed.

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