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.

Study Status:

Not recruiting


Amyotrophic Lateral Sclerosis (ALS)

Study Type:


Type of Intervention:


Intervention Name:




Phase 2

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:


Neals Affiliated?


Coordinating Center Contact Information

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:


Estimated Enrollment:


Estimated Study Start Date:

11 / 02 / 2016

Estimated Study Completion Date:

01 / 01 / 2007

Posting Last Modified Date:

02 / 09 / 2012

Date Study Added to neals.org:

10 / 26 / 2005

Minimum Age:

18 Years

Maximum Age:


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

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