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Safety Study of Oral Sodium Phenylbutyrate in Subjects With ALS (Amyotrophic Lateral Sclerosis)

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
ClinicalTrials.gov Identifier: NCT00107770
Recruitment Status : Completed
First Posted : April 8, 2005
Last Update Posted : January 11, 2010
Sponsor:
Collaborator:
Muscular Dystrophy Association
Information provided by:
VA Office of Research and Development

Tracking Information
First Submitted Date  ICMJE April 7, 2005
First Posted Date  ICMJE April 8, 2005
Last Update Posted Date January 11, 2010
Study Start Date  ICMJE April 2005
Actual Primary Completion Date November 2006   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: October 10, 2008)
safety and tolerability [ Time Frame: 20 weeks ]
Original Primary Outcome Measures  ICMJE
 (submitted: June 23, 2005)
The ability to complete the dosage increase and maintenance phase at 21 grams per day
Change History Complete list of historical versions of study NCT00107770 on ClinicalTrials.gov Archive Site
Current Secondary Outcome Measures  ICMJE
 (submitted: June 23, 2005)
  • The number of side effects at each dosage, including abnormalities in vital signs, physical examination, blood tests and EKGs, change in vital capacity (breathing function) and ALS functional rating scale
  • Relationship between blood levels and sodium phenylbutyrate dosage
Original Secondary Outcome Measures  ICMJE Same as current
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title  ICMJE Safety Study of Oral Sodium Phenylbutyrate in Subjects With ALS (Amyotrophic Lateral Sclerosis)
Official Title  ICMJE Safety and Dose Escalating Study of Oral Sodium Phenylbutyrate in Subjects With Amyotrophic Lateral Sclerosis
Brief Summary The purpose of the study is to evaluate the safety of sodium phenylbutyrate (NaPB) treatment in subjects with amyotrophic lateral sclerosis (ALS) and the ability to take this medication without major side effects.
Detailed Description

Although it is known that nerve cells die in the brains and spinal cords of patients who have ALS, the cause of the cell death is unknown. There is evidence that this cell death may be caused by changes in DNA, the body's genetic material. Drugs such as sodium phenylbutyrate (NaPB) can increase the expression of genes, block how the motor nerve cells in ALS die, and may prove to be an effective therapy for ALS. NaPB has shown an improvement in survival in mice with conditions similar to ALS.

STUDY DESIGN:

All research participants will take sodium phenylbutyrate for a total of 20 weeks. The dose of medication will be increased every 2 to 4 weeks until a maximum, easily tolerated dose is achieved (study maximum is 21 g/day).

Study Type  ICMJE Interventional
Study Phase  ICMJE Phase 1
Phase 2
Study Design  ICMJE Allocation: Non-Randomized
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Condition  ICMJE Amyotrophic Lateral Sclerosis
Intervention  ICMJE Drug: sodium phenylbutyrate
histone deacteylase inhibitor
Study Arms  ICMJE 1
ALS patient
Intervention: Drug: sodium phenylbutyrate
Publications * Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Completed
Actual Enrollment  ICMJE
 (submitted: June 23, 2005)
40
Original Enrollment  ICMJE Same as current
Actual Study Completion Date  ICMJE September 2007
Actual Primary Completion Date November 2006   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

Inclusion Criteria:

  • Diagnosed with ALS
  • At least 18 years of age
  • Women, who can become pregnant, must actively use effective birth control measures

Exclusion Criteria:

  • Must not have any other neurological (nervous system) disease

Veterans only are eligible to participate at VA sites.

Sex/Gender  ICMJE
Sexes Eligible for Study: All
Ages  ICMJE 18 Years and older   (Adult, Older Adult)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE Contact information is only displayed when the study is recruiting subjects
Listed Location Countries  ICMJE United States
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT00107770
Other Study ID Numbers  ICMJE 0015
Has Data Monitoring Committee No
U.S. FDA-regulated Product Not Provided
IPD Sharing Statement  ICMJE Not Provided
Responsible Party Ferrante, Robert - Principal Investigator, Department of Veterans Affairs
Study Sponsor  ICMJE US Department of Veterans Affairs
Collaborators  ICMJE Muscular Dystrophy Association
Investigators  ICMJE
Principal Investigator: Robert Ferrante, PhD MSc Edith Nourse Rogers Memorial Veterans Hospital, Bedford
PRS Account VA Office of Research and Development
Verification Date October 2008

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP