Title

Phase II/III Randomized, Placebo-Controlled Trial of Arimoclomol in SOD1 Positive Familial ALS

Short name

Arimoclomol

Principal Investigators

Michael Benatar, MD, PhD (University of Miami)
Merit Cudkowicz, MD, MSc (MGH)

Site-PI

Nazem Atassi, MD (MGH), Tahseen Mozaffar, MD (UC Irvine)

Summary

This is a seamless, adaptive design, phase II/III trial that aims to determine the safety and efficacy of Arimoclomol in patients with rapidly progressive SOD1 positive familial ALS. Patients from anywhere in the United States and Canada are eligible to participate and the study will cover the costs of travel to a study center. After screening procedures and confirmation of SOD1 mutation status, eligible participants travel to a study center (University of Miami or MGH) for Baseline study procedures and randomization. Study evaluations then occur monthly for 6 months and every other month for a further 6 months. The Month-2 visit again requires travel to a study center, but all other study procedures are performed via telephone and/or in participants’ homes.

Eligibility criteria

• Age ≥ 18 years
  
• Familial ALS
  
• Demonstrable mutation in the SOD1 gene that is reported to be associated with rapid disease progression
  
• Diagnosis within 9 months of the Baseline visit to study center
  
• Either on or off Riluzole

Funding agency

Amyotrophic Lateral Sclerosis Association
Food and Drug Administration
CytRx Corporation (provider of study drug)

Collaborators

Nazem Atassi, MD (Massachusetts General Hospital, Harvard Medical School)
Jack Barber, PhD (CytRx Corporation)
Merit Cudkowicz, MD, MSc (Massachusetts General Hospital, Harvard Medical School)
Carlayne Jackson, MD, FAAN (University of Texas at San Antonio)
Robert Lawson (Massachusetts General Hospital Neurology Clinical Trials Unit)
Carl Leventhal, MD (Consultant)
Michael McDermott, PhD (University of Rochester Medical Center)
David Schoenfeld, PhD (Harvard University)
Jeremy Shefner, MD, PhD (SUNY Upstate Medical University)
Kevin Boylan, MD (Mayo Jacksonville)
Scott Wieland, PhD (CytRx Corporation)
NEALS Investigators http://www.alsconsortium.org/

Project manager

Maggy De Tullio, BS, MBA

Research Coordinator

Natasha Garcia, B.A.

Contact information

Email: sod1@med.miami.edu Phone: 305-243-0108

Link(s)

http://clinicaltrials.gov/ct2/show/NCT00706147?term=benatar&rank=2

Publications

Benatar M, Kurent J, Moore DH. Treatment for familial amyotrophic lateral sclerosis/motor neuron disease. Cochrane Database Syst Rev. 2009 Jan 21;(1):CD006153. Review.

Benatar M. Lost in translation: treatment trials in the SOD1 mouse and in human ALS. Neurobiol Dis. 2007 Apr;26(1):1-13.

Presentations and Lectures

Gronka S, Walker M, Wuu J, Stanislaw C, Fanos J, Atassi N, Jaffa M, Andersen P, Cudkowicz M, Benatar M. Telemedicine to facilitate ALS research. Platform presentation at the 21st International Symposium on ALS/MND, Orlando, FL, Dec 11-13, 2010

Walker ML, Gronka S, Usher SU, Atassi N, Wuu J, Watson ML, Shefner J, Cudkowicz M, Benatar M. Utility of Forced Expiratory Volume in 6 Seconds for Assessment of Respiratory Function in Patients with Amyotrophic Lateral Sclerosis. Accepted for Poster Presentation at the 62nd Annual Meeting of the American Academy of Neurology, Toronto, April 2010

Tackling the Challenges of ALS: A Familial Perspective.
    21st International ALS/MND Meeting, Orlando, Florida, December 2010
    Neurology Grand Rounds, University of Miami. Miami, March, 2010
    Annual William L Halseth ALS Lecture, University of Colorado Denver. Aurora, February, 2010
    Neurology Grand Rounds, University of South Florida. Tampa, December, 2009
    20th International ALS/MND Meeting. Berlin, December 2009
     Neurology Grand Rounds, University of Virginia. Charlottesville, June 2009
    Human Genetics Grand Rounds, Emory University. Atlanta, February 2009

Phase II/III Randomized Placebo-Controlled Trial of Arimoclomol in SOD1 Positive Familial ALS.
    ALS Association Drug Discovery Symposium. Seattle, May 2009
    Packard Center 9th Annual Meeting. Baltimore, April 2009
    7th Annual Northeast ALS Consortium Meeting. Boston, October 2008
    6th Annual Northeast ALS Consortium Meeting. Boston, September 2007

Benatar M. A Bird’s Eye View of the SOD1 Mouse: Using meta-analysis to guide clinical trials in familial ALS. 131st Annual American Academy of Neurology meeting, Chicago, October 2006