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Clinical Trial Details — Status: Completed

Administrative data

NCT number NCT00244244
Other study ID # AALS-001
Secondary ID
Status Completed
Phase Phase 2
First received October 25, 2005
Last updated February 8, 2012
Start date October 2005
Est. completion date January 2007

Study information

Verified date February 2012
Source CytRx
Contact n/a
Is FDA regulated No
Health authority United States: Food and Drug Administration
Study type Interventional

Clinical Trial Summary

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.


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.


Recruitment information / eligibility

Status Completed
Enrollment 80
Est. completion date January 2007
Est. primary completion date January 2007
Accepts healthy volunteers No
Gender Both
Age group 18 Years and older
Eligibility 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

Study Design

Allocation: Randomized, Endpoint Classification: Safety/Efficacy Study, Intervention Model: Single Group Assignment, Masking: Double-Blind, Primary Purpose: Treatment


Related Conditions & MeSH terms


Intervention

Drug:
arimoclomol


Locations

Country Name City State
United States Massachusetts General Hospital Boston Massachusetts
United States Duke University Durham North Carolina
United States Penn State Milton S. Hershey Medical Center Hershey Pennsylvania
United States University of California, Irvine Medical Center Irvine California
United States University of Kansas Medical Center Kansas City Kansas
United States University of Miami School of Medicine Miami Florida
United States Hennepin Faculty Associates/Berman Center Minneapolis Minnesota
United States Drexel University College of Medicine Philadelphia Pennsylvania
United States University of Texas Health Science Center at San Antonio San Antonio Texas
United States SUNY Upstate Medical University Syracuse New York

Sponsors (1)

Lead Sponsor Collaborator
CytRx

Country where clinical trial is conducted

United States, 

References & Publications (6)

Benn SC, Brown RH Jr. Putting the heat on ALS. Nat Med. 2004 Apr;10(4):345-7. — View Citation

Kalmar B, Burnstock G, Vrbová G, Urbanics R, Csermely P, Greensmith L. Upregulation of heat shock proteins rescues motoneurones from axotomy-induced cell death in neonatal rats. Exp Neurol. 2002 Jul;176(1):87-97. — View Citation

Kalmar B, Greensmith L, Malcangio M, McMahon SB, Csermely P, Burnstock G. The effect of treatment with BRX-220, a co-inducer of heat shock proteins, on sensory fibers of the rat following peripheral nerve injury. Exp Neurol. 2003 Dec;184(2):636-47. — View Citation

Kieran D, Kalmar B, Dick JR, Riddoch-Contreras J, Burnstock G, Greensmith L. Treatment with arimoclomol, a coinducer of heat shock proteins, delays disease progression in ALS mice. Nat Med. 2004 Apr;10(4):402-5. Epub 2004 Mar 21. — View Citation

Kürthy M, Mogyorósi T, Nagy K, Kukorelli T, Jednákovits A, Tálosi L, Bíró K. Effect of BRX-220 against peripheral neuropathy and insulin resistance in diabetic rat models. Ann N Y Acad Sci. 2002 Jun;967:482-9. — View Citation

Muchowski PJ, Wacker JL. Modulation of neurodegeneration by molecular chaperones. Nat Rev Neurosci. 2005 Jan;6(1):11-22. Review. — View Citation

Outcome

Type Measure Description Time frame Safety issue
Primary Safety
Secondary Pharmacokinetics
Secondary ALSFRS-R
Secondary Vital Capacity
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