A Multicenter, Dose Ranging Safety and Pharmacokinetics Study of Arimoclomol in Amyotrophic Lateral Sclerosis (ALS)

Study Purpose:

The primary purpose of this study is to evaluate the safety and tolerability of arimoclomol in ALS patients following 90 days of dosing. In addition, the amount of arimoclomol in blood and cerebrospinal fluid will be measured.


Amyotrophic Lateral Sclerosis (ALS),  Familial ALS,  Sporadic ALS

Study Type:

Interventional Trial

Study Category:

Drug Trial

Study Status:

Not enrolling


Phase II

Study Chair(s)/Principal Investigator(s):

Merit Cudkowicz, MD (Massachusetts General Hospital)
Jeremy Shefner, MD (State University of New York - Upstate Medical University)

Clinicaltrials.gov ID (11 digit #):


Neals Affiliated?


Coordinating Center Contact Information

.(JavaScript must be enabled to view this email address)
.(JavaScript must be enabled to view this email address) United States

Full Study Summary:

Arimoclomol is a small molecule that upregulates "molecular chaperones" in cells under stress. Arimoclomol extends survival by five weeks when given both pre-symptomatically and at disease onset in a mutant superoxide dismutase (SOD1) transgenic mouse model of ALS. Furthermore, it has been demonstrated to have neuroprotective and neuroregenerative effects in other rat models of nerve damage. Molecular chaperone proteins are critical in the cellular response to stress and protein misfolding. Recent data suggest that the SOD1 mutation responsible for ALS in some patients with familial disease reduces the availability of a variety of molecular chaperones, and thus weakens their ability to respond to cellular stress. Protein misfolding and consequent aggregation may play a role in the pathogenesis of both the familial and sporadic forms of ALS. Therapeutic agents such as arimoclomol that improve cellular chaperone response to protein misfolding may be helpful in ALS.

Study Sponsor:


Participant Duration:

Estimated Enrollment:


Estimated Study Start Date:


Estimated Study Completion Date:


Posting Last Modified Date:


Date Study Added to alsconsortium.org:

  • Eligibility Criteria


    Female, Male

    Minimum Age:


    Maximum Age:


    Min Vital Capacity (% predicted normal):


    Time since Symptom Onset:


    Time since Diagnosis:


    Can participants use Riluzole?

    Inclusion Criteria:

    •Familial or sporadic ALS
    •Vital capacity equal to or more than 60% predicted value for gender, height and age at the screening visit
    •First ALS symptoms occurred no more than five years prior to screening
    •Must be able to take oral medication

    Exclusion Criteria:

    •Dependence on mechanical ventilation

  • Site Contact Information

    University of California, Irvine Medical Center
    Irvine, California 92868
    United States

    University of Miami School of Medicine
    Miami, Florida 33136
    United States

    University of Kansas Medical Center
    Kansas City, Kansas 66160
    United States

    Massachusetts General Hospital
    Boston, Massachusetts 02129
    United States

    Hennepin Faculty Associates/Berman Center
    Minneapolis, Minnesota 55404
    United States

    SUNY Upstate Medical University
    Syracuse, New York 13210
    United States

    Duke University
    Durham, North Carolina 27705
    United States

    Penn State Milton S. Hershey Medical Center
    Hershey, Pennsylvania 17033
    United States

    Drexel University College of Medicine
    Philadelphia, Pennsylvania 19107
    United States

    University of Texas Health Science Center at San Antonio
    San Antonio, Texas 78229
    United States

  • Study Results