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.

Condition	Treatment or Intervention	Phase
Amyotrophic Lateral Sclerosis	Drug: sodium phenylbutyrate	Phase I Phase II

Further Study Details: 

Primary Outcomes: The ability to complete the dosage increase and maintenance phase at 21 grams per day.
Secondary Outcomes: 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.
Expected Total Enrollment:  40

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).

Ages Eligible for Study:  18 Years and above,  Genders Eligible for Study:  Both

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

Patricia Loya      781-687-2884    Patricia.Loya@med.va.gov
Sally MacDonald, RN      781-687-2963    Sally.MacDonald@med.va.gov

Iowa
      Veteran’s Administration Hospital (Iowa City), Iowa City,  Iowa,  52242-1009,  United States; Not yet recruiting
Kentucky
      Lexington VA Medical Center, Lexington,  Kentucky,  40502,  United States; Not yet recruiting
Maryland
      Johns Hopkins University, Baltimore,  Maryland,  21287,  United States; Not yet recruiting
Massachusetts
      Edith Nourse Rogers Memorial Veterans Hospital, Bedford,  Massachusetts,  01730,  United States; Recruiting
      Massachusetts General Hospital, Boston,  Massachusetts,  02129,  United States; No longer recruiting
New York
      Syracuse VA Medical Center, Syracuse,  New York,  13210,  United States; Recruiting
North Carolina
      Durham VA Medical Center, Durham,  North Carolina,  27705,  United States; Not yet recruiting
Texas
      Michael E. DeBakey VA Medical Center, Houston,  Texas,  77030,  United States; Not yet recruiting

Study chairs or principal investigators

Merit E. Cudkowicz, MD, MSc,  Principal Investigator,  Massachusetts General Hospital and Edith Nourse Rogers Memorial Veterans Hospital   
Robert J. Ferrante, PhD,  Principal Investigator,  Edith Nourse Rogers Memorial Veterans Hospital   
Robert H. Brown, Jr., MD, DPhil.,  Principal Investigator,  Massachusetts General Hospital   

Study ID Numbers:  0015
Record last reviewed:  April 2005
Last Updated:  April 7, 2005
Record first received:  April 7, 2005
ClinicalTrials.gov Identifier:  NCT00107770
Health Authority: United States: Food and Drug Administration
ClinicalTrials.gov processed this record on 2005-04-08