Now Available for Public Comment: Notice of Proposed Rulemaking (NPRM) for FDAAA 801 and NIH Draft Reporting Policy for NIH-Funded Trials

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

October 25, 2005
February 8, 2012
October 2005
January 2007   (final data collection date for primary outcome measure)
Safety
Same as current
Complete list of historical versions of study NCT00244244 on ClinicalTrials.gov Archive Site
  • Pharmacokinetics
  • ALSFRS-R
  • Vital Capacity
Same as current
Not Provided
Not Provided
 
A Multicenter, Dose Ranging Safety and Pharmacokinetics Study of Arimoclomol in ALS
A Multicenter, Dose Ranging Safety and Pharmacokinetics Study of Arimoclomol in Amyotrophic Lateral Sclerosis (ALS)

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.

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.

Interventional
Phase 2
Allocation: Randomized
Endpoint Classification: Safety/Efficacy Study
Intervention Model: Single Group Assignment
Masking: Double-Blind
Primary Purpose: Treatment
Amyotrophic Lateral Sclerosis (ALS)
Drug: arimoclomol
Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Completed
80
January 2007
January 2007   (final data collection date for primary outcome measure)

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
Both
18 Years and older
No
Contact information is only displayed when the study is recruiting subjects
United States
 
NCT00244244
AALS-001
Not Provided
CytRx
CytRx
Not Provided
Principal Investigator: Merit Cudkowicz, MD Massachusetts General Hospital
Principal Investigator: Jeremy Shefner, MD State University of New York - Upstate Medical University
CytRx
February 2012

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP