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A Multicenter, Dose Ranging Safety and Pharmacokinetics Study of Arimoclomol in ALS

This study has been completed.
Sponsor:
Information provided by (Responsible Party):
CytRx
ClinicalTrials.gov Identifier:
NCT00244244
First received: October 25, 2005
Last updated: February 8, 2012
Last verified: February 2012
  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.


Condition Intervention Phase
Amyotrophic Lateral Sclerosis (ALS)
Drug: arimoclomol
Phase 2

Study Type: Interventional
Study Design: Allocation: Randomized
Endpoint Classification: Safety/Efficacy Study
Intervention Model: Single Group Assignment
Masking: Double-Blind
Primary Purpose: Treatment
Official Title: A Multicenter, Dose Ranging Safety and Pharmacokinetics Study of Arimoclomol in Amyotrophic Lateral Sclerosis (ALS)

Resource links provided by NLM:


Further study details as provided by CytRx:

Primary Outcome Measures:
  • Safety

Secondary Outcome Measures:
  • Pharmacokinetics
  • ALSFRS-R
  • Vital Capacity

Estimated Enrollment: 80
Study Start Date: October 2005
Study Completion Date: January 2007
Primary Completion Date: January 2007 (Final data collection date for primary outcome measure)
Detailed Description:

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.

  Eligibility

Ages Eligible for Study:   18 Years and older
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
Criteria

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
  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT00244244

Locations
United States, California
University of California, Irvine Medical Center
Irvine, California, United States, 92868
United States, Florida
University of Miami School of Medicine
Miami, Florida, United States, 33136
United States, Kansas
University of Kansas Medical Center
Kansas City, Kansas, United States, 66160
United States, Massachusetts
Massachusetts General Hospital
Boston, Massachusetts, United States, 02129
United States, Minnesota
Hennepin Faculty Associates/Berman Center
Minneapolis, Minnesota, United States, 55404
United States, New York
SUNY Upstate Medical University
Syracuse, New York, United States, 13210
United States, North Carolina
Duke University
Durham, North Carolina, United States, 27705
United States, Pennsylvania
Penn State Milton S. Hershey Medical Center
Hershey, Pennsylvania, United States, 17033
Drexel University College of Medicine
Philadelphia, Pennsylvania, United States, 19107
United States, Texas
University of Texas Health Science Center at San Antonio
San Antonio, Texas, United States, 78229
Sponsors and Collaborators
CytRx
Investigators
Principal Investigator: Merit Cudkowicz, MD Massachusetts General Hospital
Principal Investigator: Jeremy Shefner, MD State University of New York - Upstate Medical University
  More Information

Publications:
Responsible Party: CytRx
ClinicalTrials.gov Identifier: NCT00244244     History of Changes
Other Study ID Numbers: AALS-001
Study First Received: October 25, 2005
Last Updated: February 8, 2012
Health Authority: United States: Food and Drug Administration

Additional relevant MeSH terms:
Amyotrophic Lateral Sclerosis
Motor Neuron Disease
Sclerosis
Central Nervous System Diseases
Metabolic Diseases
Nervous System Diseases
Neurodegenerative Diseases
Neuromuscular Diseases
Pathologic Processes
Proteostasis Deficiencies
Spinal Cord Diseases
TDP-43 Proteinopathies

ClinicalTrials.gov processed this record on November 20, 2014