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A Multicenter, Double-Blind Study to Investigate the Safety and Efficacy of Arimoclomol in Volunteers With ALS
A Multicenter, Double-Blind, Placebo-Controlled, Phase 2b Study to Investigate the Safety and Efficacy of Arimoclomol in Volunteers With Amyotrophic Lateral Sclerosis (ALS)
1 other identifier
interventional
N/A
2 countries
35
Brief 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.
Trial Health
Trial Health Score
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35 active sites
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Trial Relationships
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Study Timeline
Key milestones and dates
First Submitted
Initial submission to the registry
November 16, 2007
CompletedFirst Posted
Study publicly available on registry
November 20, 2007
CompletedFebruary 9, 2012
February 1, 2012
November 16, 2007
February 8, 2012
Conditions
Keywords
Outcome Measures
Primary Outcomes (1)
ALSFRS-R
9 months
Secondary Outcomes (6)
ALSFRS-R
18 months
Survival
18 months
Muscle strength
9 and 18 months
Pulmonary function
9 and 18 months
MUNE
9 and 18 months
- +1 more secondary outcomes
Study Arms (2)
1
PLACEBO COMPARATOR2
EXPERIMENTALInterventions
Eligibility Criteria
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.
Contact the study team to confirm eligibility.
Sponsors & Collaborators
- CytRxlead
Study Sites (35)
University of California Los Angeles - Tier 2 Site
Pacific Palisades, California, 90272, United States
University of California - San Francisco - Tier 2 Site
San Francisco, California, 94117, United States
University of Colorado Health Sciences Center - Tier 2 Site
Denver, Colorado, 80262, United States
University of Miami - Tier 2 Site
Miami, Florida, 33136, United States
Emory University - Tier 2 site
Atlanta, Georgia, 30322, United States
Northwestern University, Dept. of Neurology - Tier 2 Site
Chicago, Illinois, 60611, United States
University of Kansas Medical Center - Tier 2 site
Kansas City, Kansas, 66160, United States
John Hopkins University - Tier 2 Site
Baltimore, Maryland, 21287, United States
Massachusetts General Hospital - Tier 1 Site
Boston, Massachusetts, 02114, United States
Baystate Medical Center - Tier 2 Site
Springfield, Massachusetts, 01199, United States
Saint Louis University, Neuromuscular Div. - Tier 2 Site
St Louis, Missouri, 63110, United States
Washington University - Tier 2 Site
St Louis, Missouri, 63110, United States
BryanLGH Medical Center - Tier 2 Site
Lincoln, Nebraska, 68506, United States
Upstate Clinical Research, LLC - Tier 2 Site
Albany, New York, 12205, United States
Mount Sinai School of Medicine - Tier 2 Site
New York, New York, 10029, United States
Columbia University Medical Center - Tier 2 site
New York, New York, 10032, United States
SUNY Downstate Medical Center - Tier 1 Site
Syracuse, New York, 13210, United States
Duke University Medical Center - Tier 1 Site
Durham, North Carolina, 27705, United States
Wake Forest University School of Medicine -Tier 2 Site
Winston-Salem, North Carolina, 27157, United States
Cleveland Clinic Foundation -Tier 2 site
Cleveland, Ohio, 44195, United States
Providence ALS Center - Tier 2 Site
Portland, Oregon, 97213, United States
Pennsylvania State University School of Medicine - Tier 2 Site
Hershey, Pennsylvania, 17033, United States
Drexel University College of Medicine - Tier 1 Site
Philadelphia, Pennsylvania, 19107, United States
University of Pittsburgh Medical Center - Tier 2 Site
Pittsburgh, Pennsylvania, 15213, United States
Vanderbilt University Medical Center - Tier 2
Nashville, Tennessee, 37232-2551, United States
Texas Neurology, PA - Tier 2 Site
Dallas, Texas, 75214, United States
University of Texas Health Science Center - Tier 2 Site
San Antonio, Texas, 78229-3900, United States
University of Vermont, College of Medicine - Tier 2
Burlington, Vermont, 05405, United States
University of Virginia - Tier 2 Sites
Charlottesville, Virginia, 22908, United States
Virginia Mason Clinic - Tier 2 Site
Seattle, Washington, 98101, United States
Medical College of Wisconsin - Tier 2 Site
Milwaukee, Wisconsin, 53226, United States
University of British Columbia, Gordon and Leslie Diamond Health Care Centre - Tier 2 Site
Vancouver, British Columbia, V5Z 2G9, Canada
London Health Science Center - Tier 2 Site
London, Ontario, N6A 5A5, Canada
University of Toronto, Sunnybrook Health Sciences Centre - Tier 2 Site
Toronto, Ontario, M4N 3M5, Canada
Montreal Neurological Institute - Tier 2
Montreal, Quebec, H3A 2B4, Canada
MeSH Terms
Conditions
Interventions
Condition Hierarchy (Ancestors)
Study Officials
- PRINCIPAL INVESTIGATOR
Merit Cudkowicz, MD, MSc
Massachusetts General Hospital
- PRINCIPAL INVESTIGATOR
Jeremy Shefner, MD, PhD
State University of New York - Upstate Medical University
Study Design
- Study Type
- interventional
- Phase
- phase 2
- Allocation
- RANDOMIZED
- Masking
- QUADRUPLE
- Who Masked
- PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
- Purpose
- TREATMENT
- Intervention Model
- PARALLEL
- Sponsor Type
- INDUSTRY
- Responsible Party
- SPONSOR
Study Record Dates
First Submitted
November 16, 2007
First Posted
November 20, 2007
Last Updated
February 9, 2012
Record last verified: 2012-02