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

This study has been completed.
Sponsor:
Collaborator:
Muscular Dystrophy Association
Information provided by:
Department of Veterans Affairs
ClinicalTrials.gov Identifier:
NCT00107770
First received: April 7, 2005
Last updated: January 8, 2010
Last verified: October 2008

April 7, 2005
January 8, 2010
April 2005
November 2006   (final data collection date for primary outcome measure)
safety and tolerability [ Time Frame: 20 weeks ] [ Designated as safety issue: No ]
The ability to complete the dosage increase and maintenance phase at 21 grams per day
Complete list of historical versions of study NCT00107770 on ClinicalTrials.gov Archive Site
  • 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 [ Designated as safety issue: No ]
  • Relationship between blood levels and sodium phenylbutyrate dosage [ Designated as safety issue: No ]
  • 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
Not Provided
Not Provided
 
Safety Study of Oral Sodium Phenylbutyrate in Subjects With ALS (Amyotrophic Lateral Sclerosis)
Safety and Dose Escalating Study of Oral Sodium Phenylbutyrate in Subjects With Amyotrophic Lateral Sclerosis

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.

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).

Interventional
Phase 1
Phase 2
Allocation: Non-Randomized
Endpoint Classification: Safety Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
Amyotrophic Lateral Sclerosis
Drug: sodium phenylbutyrate
histone deacteylase inhibitor
1
ALS patient
Intervention: Drug: sodium phenylbutyrate
Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Completed
40
September 2007
November 2006   (final data collection date for primary outcome measure)

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.

Both
18 Years and older
No
Contact information is only displayed when the study is recruiting subjects
United States
 
NCT00107770
0015
No
Ferrante, Robert - Principal Investigator, Department of Veterans Affairs
Department of Veterans Affairs
Muscular Dystrophy Association
Principal Investigator: Robert Ferrante, PhD MSc Edith Nourse Rogers Memorial Veterans Hospital, Bedford
Department of Veterans Affairs
October 2008

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