Clinical trial NCT00107770: Safety Study of Oral Sodium Phenylbutyrate in Subjects With ALS (Amyotrophic Lateral Sclerosis)
Official title: Safety and Dose Escalating Study of Oral Sodium Phenylbutyrate in Subjects With Amyotrophic Lateral Sclerosis
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE1
PHASE2
Conditions: Amyotrophic Lateral Sclerosis
Keywords: ALS
histone deacetylase inhibition
open label
safety study
sodium phenylbutyrate
Lead sponsor: VA Office of Research and Development
Sponsor class: FED
Collaborators: name: Muscular Dystrophy Association
First posted: 2005-04-08
Enrollment: 40
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NON_RANDOMIZED
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

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).

Eligibility criteria: 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.

Primary outcomes:
measure: safety and tolerability
timeFrame: 20 weeks
Secondary outcomes:
measure: 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
measure: Relationship between blood levels and sodium phenylbutyrate dosage
Other outcomes:
Locations:
city: Iowa City
country: United States
facility: VA Medical Center, Iowa City
state: Iowa
city: Lexington
country: United States
facility: VA Medical Center, Lexington
state: Kentucky
city: Baltimore
country: United States
facility: VA Maryland Health Care System, Baltimore
state: Maryland
city: Bedford
country: United States
facility: Edith Nourse Rogers Memorial Veterans Hospital, Bedford
state: Massachusetts
city: Boston
country: United States
facility: VA Medical Center, Jamaica Plain Campus
state: Massachusetts
city: Syracuse
country: United States
facility: VA Medical Center, Syracuse
state: New York
city: Durham
country: United States
facility: VA Medical Center, Durham
state: North Carolina
city: Cincinnati
country: United States
facility: VA Medical Center, Cincinnati
state: Ohio
city: Pittsburgh
country: United States
facility: VA Pittsburgh Health Care System
state: Pennsylvania
city: Houston
country: United States
facility: Michael E. DeBakey VA Medical Center (152)
state: Texas
Study officials:
affiliation: Edith Nourse Rogers Memorial Veterans Hospital, Bedford
name: Robert Ferrante, PhD MSc
role: PRINCIPAL_INVESTIGATOR
References: