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

US Department of Veterans Affairs (VA)

Status and phase: Completed; Phase 2; Phase 1

Conditions: Amyotrophic Lateral Sclerosis

Treatments: Drug: sodium phenylbutyrate

Study type: Interventional

Funder types: Other; Other U.S. Federal agency

Identifiers

NCT00107770

0015

Details and patient eligibility

About

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.

Full 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).

Enrollment: 40 patients

Sex: All

Ages: 18+ years old

Volunteers: No Healthy Volunteers

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.

Trial design

Primary purpose: Treatment

Allocation: Non-Randomized

Interventional model: Single Group Assignment

Masking: None (Open label)

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40 participants in 1 patient group

1

Other group

Description:

ALS patient

Treatment:

Drug: sodium phenylbutyrate