A Multicenter, Double-Blind Study to Investigate the Safety and Efficacy of Arimoclomol in Volunteers With ALS
Withdrawn before enrolling · Phase 2 · Has a placebo group
Conditions studied: Amyotrophic Lateral Sclerosis
In brief
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.
Key facts
- Study ID
- NCT00561366
- Run by
- CytRx
- People needed
- 0
- Last updated by the study team
- 2012-02-09
Who can join
Age: 18 and older. Sex: any. Healthy volunteers: not accepted.
You may qualify if…
- Familial or sporadic ALS.
- Diagnosed with laboratory-supported probable, probable or definite ALS according to the World Federation of Neurology El Escorial criteria for less than or equal to 36 months' duration prior to the Screening Visit.
- Vital capacity (VC) equal to or greater than 70% predicted value for gender, height and age at the Screening Visit.
- Geographic accessibility to the study site.
- Ability to take oral medication at the Screening Visit, based on verbal report.
- Fluency in English, Spanish or Canadian French.
You may not qualify if…
- History of known sensitivity or intolerability to arimoclomol or to any other related compound.
- Prior exposure to arimoclomol through a clinical trial or physician-sponsored IND.
- Exposure to any investigational agent within 30 days of the Screening Visit.
- Presence of any of the following clinical conditions:
- Substance abuse within the past year
- Unstable cardiac, pulmonary, renal, hepatic, endocrine, hematologic, or active malignancy or infectious disease
- AIDS or AIDS-related complex
- Unstable psychiatric illness defined as psychosis (hallucinations or delusions), untreated major depression within 90 days of the Screening Visit.
- Laboratory values: Screening serum creatinine greater than or equal to 1.5 mg/dL, creatinine clearance less than 70 cc/min, alanine aminotransferase (ALT) greater than 3.0 times the upper limit of normal, total bilirubin greater than 1.5 times the upper limit of normal, white blood cell (WBC) count less than 3,500/mm3, platelet concentration of <100,000/ul, hematocrit level of less than 33 % for female or less than 35 % for male, or coagulation tests (PT, PTT) greater than or equal to 1.5 times upper limit of normal.
- Female volunteers who are breast-feeding.
Where it is running
- University of California Los Angeles - Tier 2 Site — Pacific Palisades, California, United States
- University of California - San Francisco - Tier 2 Site — San Francisco, California, United States
- University of Colorado Health Sciences Center - Tier 2 Site — Denver, Colorado, United States
- University of Miami - Tier 2 Site — Miami, Florida, United States
- Emory University - Tier 2 site — Atlanta, Georgia, United States
- Northwestern University, Dept. of Neurology - Tier 2 Site — Chicago, Illinois, United States
- University of Kansas Medical Center - Tier 2 site — Kansas City, Kansas, United States
- John Hopkins University - Tier 2 Site — Baltimore, Maryland, United States
- Massachusetts General Hospital - Tier 1 Site — Boston, Massachusetts, United States
- Baystate Medical Center - Tier 2 Site — Springfield, Massachusetts, United States
- Saint Louis University, Neuromuscular Div. - Tier 2 Site — St Louis, Missouri, United States
- Washington University - Tier 2 Site — St Louis, Missouri, United States
- BryanLGH Medical Center - Tier 2 Site — Lincoln, Nebraska, United States
- Upstate Clinical Research, LLC - Tier 2 Site — Albany, New York, United States
- Mount Sinai School of Medicine - Tier 2 Site — New York, New York, United States
- Columbia University Medical Center - Tier 2 site — New York, New York, United States
- SUNY Downstate Medical Center - Tier 1 Site — Syracuse, New York, United States
- Duke University Medical Center - Tier 1 Site — Durham, North Carolina, United States
- Wake Forest University School of Medicine -Tier 2 Site — Winston-Salem, North Carolina, United States
- Cleveland Clinic Foundation -Tier 2 site — Cleveland, Ohio, United States
- Providence ALS Center - Tier 2 Site — Portland, Oregon, United States
- Pennsylvania State University School of Medicine - Tier 2 Site — Hershey, Pennsylvania, United States
- Drexel University College of Medicine - Tier 1 Site — Philadelphia, Pennsylvania, United States
- University of Pittsburgh Medical Center - Tier 2 Site — Pittsburgh, Pennsylvania, United States
- Vanderbilt University Medical Center - Tier 2 — Nashville, Tennessee, United States
Full record on ClinicalTrials.gov
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