NCT00244244

Brief 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.

Trial Health

87
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
80

participants targeted

Target at P50-P75 for phase_2

Timeline
Completed

Started Oct 2005

Shorter than P25 for phase_2

Geographic Reach
1 country

10 active sites

Status
completed

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

Study Start

First participant enrolled

October 1, 2005

Completed
24 days until next milestone

First Submitted

Initial submission to the registry

October 25, 2005

Completed
1 day until next milestone

First Posted

Study publicly available on registry

October 26, 2005

Completed
1.2 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

January 1, 2007

Completed
Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

January 1, 2007

Completed
Last Updated

February 9, 2012

Status Verified

February 1, 2012

Enrollment Period

1.3 years

First QC Date

October 25, 2005

Last Update Submit

February 8, 2012

Conditions

Amyotrophic Lateral Sclerosis (ALS)

Outcome Measures

Primary Outcomes (1)

  • Safety

Secondary Outcomes (3)

  • Pharmacokinetics

  • ALSFRS-R

  • Vital Capacity

Interventions

arimoclomolDRUG

Eligibility Criteria

Age18 Years+
Sexall
Healthy VolunteersNo
Age GroupsAdult (18-64), Older Adult (65+)

You may qualify if:

  • 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

You may not qualify if:

  • Dependence on mechanical ventilation

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (10)

University of California, Irvine Medical Center

Irvine, California, 92868, United States

Location

University of Miami School of Medicine

Miami, Florida, 33136, United States

Location

University of Kansas Medical Center

Kansas City, Kansas, 66160, United States

Location

Massachusetts General Hospital

Boston, Massachusetts, 02129, United States

Location

Hennepin Faculty Associates/Berman Center

Minneapolis, Minnesota, 55404, United States

Location

SUNY Upstate Medical University

Syracuse, New York, 13210, United States

Location

Duke University

Durham, North Carolina, 27705, United States

Location

Penn State Milton S. Hershey Medical Center

Hershey, Pennsylvania, 17033, United States

Location

Drexel University College of Medicine

Philadelphia, Pennsylvania, 19107, United States

Location

University of Texas Health Science Center at San Antonio

San Antonio, Texas, 78229, United States

Location

Related Publications (6)

  • 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. doi: 10.1038/nm1021. Epub 2004 Mar 21.

    PMID: 15034571BACKGROUND

  • Benn SC, Brown RH Jr. Putting the heat on ALS. Nat Med. 2004 Apr;10(4):345-7. doi: 10.1038/nm0404-345. No abstract available.

    PMID: 15057226BACKGROUND

  • Kurthy M, Mogyorosi T, Nagy K, Kukorelli T, Jednakovits A, Talosi L, Biro 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. doi: 10.1111/j.1749-6632.2002.tb04306.x.

    PMID: 12079878BACKGROUND

  • Kalmar B, Burnstock G, Vrbova 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. doi: 10.1006/exnr.2002.7945.

    PMID: 12093085BACKGROUND

  • Muchowski PJ, Wacker JL. Modulation of neurodegeneration by molecular chaperones. Nat Rev Neurosci. 2005 Jan;6(1):11-22. doi: 10.1038/nrn1587.

    PMID: 15611723BACKGROUND

  • 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. doi: 10.1016/S0014-4886(03)00343-1.

    PMID: 14769355BACKGROUND

MeSH Terms

Conditions

Amyotrophic Lateral Sclerosis

Interventions

arimoclomol

Condition Hierarchy (Ancestors)

Spinal Cord DiseasesCentral Nervous System DiseasesNervous System DiseasesMotor Neuron DiseaseNeurodegenerative DiseasesTDP-43 ProteinopathiesNeuromuscular DiseasesProteostasis DeficienciesMetabolic DiseasesNutritional and Metabolic Diseases

Study Officials

  • Merit Cudkowicz, MD

    Massachusetts General Hospital

    PRINCIPAL INVESTIGATOR

  • Jeremy Shefner, MD

    State University of New York - Upstate Medical University

    PRINCIPAL INVESTIGATOR

Study Design

Study Type
interventional
Phase
phase 2
Allocation
RANDOMIZED
Masking
DOUBLE
Purpose
TREATMENT
Intervention Model
SINGLE GROUP
Sponsor Type
INDUSTRY
Responsible Party
SPONSOR

Study Record Dates

First Submitted

October 25, 2005

First Posted

October 26, 2005

Study Start

October 1, 2005

Primary Completion

January 1, 2007

Study Completion

January 1, 2007

Last Updated

February 9, 2012

Record last verified: 2012-02

Locations

US(10)