Clinical Trial of Sodium Phenylbutyrate in Children With Spinal Muscular Atrophy Type I

PHASE1/PHASE2TerminatedINTERVENTIONAL
Enrollment

5

Participants

Timeline

Start Date

January 31, 2008

Primary Completion Date

May 31, 2009

Study Completion Date

May 31, 2009

Conditions
Spinal Muscular Atrophy Type I
Interventions
DRUG

sodium phenylbutyrate

500 mg/kg/day, depending upon tolerability subsequent dosages may increase to 675, 900, or 1200 mg/kg/day to identify maximum tolerated dose (MTD) and then an additional 6 participants will enroll at the MTD.

Trial Locations (5)

10032

Columbia University, 180 Fort Washington Avenue, 5th Floor, New York

75207

University of Texas Southwestern Medical Center at Dallas, Division of Pediatric Neurology, Children's Medical Center of Dallas, Ambulatory Care Pavilion, 2350 Stemmons Freeway, Suite #5074, Dallas

94305-5235

Stanford University Medical Center, 300 Pasteur Drive, Room A343, Stanford

02115

Children's Hospital, Boston, 300 Longwood Avenue, Fegan 11, Boston

19104-4399

The Children's Hospital of Philadelphia, Clinical Trials Office, A-230, 34th St. and Civic Center Boulevard, Philadelphia

Sponsors

Lead Sponsor

All Listed Sponsors
collaborator

National Institute of Neurological Disorders and Stroke (NINDS)

NIH

lead

Westat

OTHER