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NCT Number: NCT00107770

Safety Study of Oral Sodium Phenylbutyrate in Subjects With ALS (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.

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Key information

Conditions

Amyotrophic Lateral Sclerosis Central Nervous System Diseases Metabolic Diseases Motor Neuron Disease Nervous System Diseases Neurodegenerative Diseases Neuromuscular Diseases Nutritional and Metabolic Diseases Proteostasis Deficiencies Spinal Cord Diseases TDP-43 Proteinopathies

Age range

18 year and older

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 1 / Phase 2

Primary location

VA Medical Center, Iowa City, Iowa City, Iowa, United States

Loading trial locations.

About this study

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).

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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.

Treatment and study plan

Sodium Phenylbutyrate

Drug

histone deacteylase inhibitor

Primary outcomes

  1. safety and tolerability

    Time frame: 20 weeks

Secondary outcomes

  1. 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

  2. Relationship between blood levels and sodium phenylbutyrate dosage

Sponsors and collaborators

Lead sponsor

US Department of Veterans Affairs

Fed

Collaborators

  • Muscular Dystrophy Association

Registry information

Official study title

Safety and Dose Escalating Study of Oral Sodium Phenylbutyrate in Subjects With Amyotrophic Lateral Sclerosis

Important dates

Study start
2005
Primary completion
2006
Study completion
2007
First posted
Apr 8, 2005
Registry last updated
Jan 11, 2010

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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