Six ALS Clinical Trials: Arimoclomol

by Amy Madsen on Wed, 2013-05-01 09:23

Name: Arimoclomol

How it works: Arimoclomol, developed by CytRx Corporation, is believed to work by increasing production of the so-called "chaperone" molecules heat-shock protein 70 and heat-shock protein 90. These molecules aid in cell repair by helping newly synthesized proteins fold correctly, or by detecting proteins that are misfolded — and therefore potentially toxic — and refolding them into their correct shapes.

Skeletal formula of arimoclomol
(courtesy of ChemSpider).

Purpose of trial: To see if arimoclomol has a beneficial effect on functional ability, disease progression and longevity in people with ALS.

Type of trial: Phase 2-3 trial. The trial is a project of the Northeast ALS Consortium (NEALS), an ALS research network.

Trial sites:

  1. University of Miami Miller School of Medicine, Miami, Fla.
  2. Massachusetts General Hospital, Boston

Outcome measures:

  1. The primary outcome measure is the rate of functional decline in people taking the drug, compared to those taking a placebo.
  2. Secondary measures are disease progression, longevity, the need for tracheostomy or permanent assisted ventilation, and the safety and tolerability of the drug.

Currently recruiting? Active, currently recruiting.

Background info: Arimoclomol has been shown to extend life in an animal model of ALS. Read Treatment with Arimoclomol, a Coinducer of Heat Shock Proteins, Delays Disease Progression in ALS Mice. View PubMed results for scientific papers about arimoclomol and ALS.

Additional info: For trial information and enrollment criteria, see Phase 2/3 Randomized, Placebo-Controlled Trial of Arimoclomol in SOD1 Positive Familial Amyotrophic Lateral Sclerosis.

To read about the other ALS clinical trials:

Note: The effect of these drugs on people with ALS is still unknown. Individuals are strongly advised not to use these drugs to treat ALS until they have been proven safe and effective, and have been approved for use in ALS. Always consult with your physician before adding any medications to your treatment plan. 

About Clinical Trials

About Clinical Trials

A clinical trial is a test, in humans, of an experimental treatment. Although it's possible that benefit may be derived from participating in a clinical trial, it's also possible that no benefit, or even harm, may occur.

MDA has no ability to influence who is chosen to participate in a clinical trial.

To learn more, see Learn About Clinical Studies and Being a Co-Adventurer, which is about neuromuscular disease clinical trials. To see a continuously updated database of clinical trials, go to

Amy Madsen
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