Study:
A Multicenter, Double-Blind, Placebo-Controlled, Phase 2b Study to Investigate the Safety and Efficacy of Arimoclomol in Volunteers With Amyotrophic Lateral Sclerosis (ALS)
Rationale:
n/a
Purpose:
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.
Study Status: Withdrawn
Recruiting:
n/a
| Condition |
Intervention |
Phase |
|
Amyotrophic Lateral Sclerosis |
Drug: Placebo
Drug: Arimoclomol |
Phase 2 |
Verified by
CytRx
February, 2012
Sponsored by: CytRx
Information provided by: CytRx
ClinicalTrials.gov identifier: NCT00561366
Study Type: Interventional
Study Design: Allocation: Randomized, Endpoint Classification: Safety/Efficacy Study, Intervention Model: Parallel Assignment, Masking: Double Blind (Subject, Caregiver, Investigator, Outcomes Assessor), Primary Purpose: Treatment
Cleveland Clinic Foundation -Tier 2 site
Cleveland, Ohio 44195
United States
Merit Cudkowicz, MD, MSc., Principal Investigator
Jeremy Shefner, MD, PhD., Principal Investigator