Title: Safety and Dose Escalating Study of Oral Sodium Phenylbutyrate in Subjects with Amyotrophic Lateral Sclerosis
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
Study start: April 2005
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. 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). Non-Randomized, Open Label, Uncontrolled, Single Group Assignment, Safety Study.
Inclusion Criteria:
- Diagnosed with ALS
- At least 18 years of age
- Women, who can become pregnant, must actively use effective birth control measures.
- Only veterans with ALS may apply for inclusion to trials located at VA medical centers.
Exclusion Criteria:
Must not have any other neurological (nervous system) disease
Location and Contact Information:
Patricia Loya, 781-687-2884, Patricia.Loya@med.va.gov
Sally MacDonald, RN 781-687-2963, Sally.MacDonald@med.va.gov
Iowa - No Longer Recruiting
Veteran’s Administration Hospital (Iowa City)
Iowa City, Iowa, 52242-1009, United States
Teresa Benda, RN 319-353-7105 teresa-benda@uiowa.edu
Praful Kelkar, MD, Principal Investigator
Kentucky - No Longer Recruiting
Lexington VA Medical Center, Lexington, Kentucky, 40502, United States;
Deborah Taylor, MS 859-257-5560 DTAYL2@email.uky.edu
Edward Kasarkis, MD, Principal Investigator
Maryland - No Longer Recruiting
Johns Hopkins University, Baltimore, Maryland, 21287, United States;
Lora Clawson, MSN, CRNP 410-955-8511 lclawso@jhmi.edu
Nicholas Maragakis, MD, Principal Investigator
Massachusetts
Edith Nourse Rogers Memorial Veterans Hospital, Bedford, Massachusetts, 01730, United States; No Longer Recruiting
Patricia Loya, BA 781-687-2884 Patricia.Loya@med.va.gov
Sally MacDonald, RNC 781-687-2963 Sally.MacDonald@med.va.gov
Merit E. Cudkowicz, MD, MSc, Principal Investigator
Massachusetts General Hospital, Boston, Massachusetts, 02129, United States; No longer recruiting
New York - No Longer Recruiting
Syracuse VA Medical Center, Syracuse, New York, 13210, United States;
Linda Schad 315-425-3473 Linda.Schad@med.va.gov
Francine J. Vriesendorp, MD, Principal Investigator
North Carolina - No Longer Recruiting
Durham VA Medical Center, Durham, North Carolina, 27705, United States;
Yvonne Golightly, MS, PT 919-286-0411 ext. 7484 golig003@mc.duke.edu
Richard Bedlack, MD, PhD, Principal Investigator
Ohio - No Longer Recruiting
Cincinnati VAMC
3200 Vine Street
Cincinnati, OH 45220
Laura Sams, MD
Laura.Sams@med.va.gov
Cincinnati VAMC(111C2)
3200 Vine Street
Cincinnati, OH 45220
Margaret Story, RN
513 475-6542 or 6540
Margaret.Story@med.va.gov
Pennsylvania - No Longer Recruiting
VA Pittsburgh Healthcare System
Neurology Section
MDP: 11N-U
University Drive C
Pittsburgh, PA 15240
Sasa Zivkovic, MD
zivkovics@upmc.edu
Vivian Brice, coordinator
412-688-6185
Vivian.Brice@med.va.gov
Texas - No Longer Recruiting
Michael E. DeBakey VA Medical Center, Houston, Texas, 77030, United States;
Carol Echols, PA 713-794-7140 echols.carolw@med.va.gov
Dennis Mosier, MD, PhD, Principal Investigator
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
Posted on October 17, 2008
