**OPEN FOR ENROLLMENT**

Newly Enrolling NPTUNE Study

Columbia University is currently participating in a Phase I/IIB dose escalating trial for patients with Spinal Muscular Atrophy (SMA) Type I and Type II/III. This multi-center study, commonly referred to as NPTUNE [National Institute of Neurological Disorders and Stroke (NINDS) Pilot Therapeutics Network], is sponsored by NINDS and organized by Westat, a contract research organization (CRO) located in Rockville, MD. The purpose of this study is to evaluate multiple dosage levels of a drug called sodium phenylbutyrate (NaPB) to determine the highest dose (maximum tolerated dose – MTD) that can be administered safely to children with SMA. NaPB is an approved drug for use in infants and children with rare metabolic disorders, known as urea cycle disorders. It is not known whether this drug is helpful to children with SMA. Sodium phenylbutyrate increases the expression of the Survival Motor Neuron protein in laboratory experiments using cells of SMA patients. The Survival Motor Neuron expression is low in patients with SMA, and researchers hope that increasing it will benefit patients.

During each study visit, participants will undergo some testing, have blood drawn and study medication will also be given. The information gained from this clinical trial will help evaluate this medication as a potential treatment for SMA. This study will help find the right dose to be used in future studies. This is an important step for future larger studies testing the efficacy of Sodium phenylbutyrate in SMA.

Please contact the Study Coordinator, Jackie Montes, PT, at 212-342-5767 or jm598@columbia.edu for more information.

**OPEN FOR ENROLLMENT**

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Research in Spinal Muscular Atrophy: We are conducting a clinical research study for patients with spinal muscular atrophy. The goal of this study is to obtain information needed to plan a clinical trial in spinal muscular atrophy. This research is being done at three centers on the east coast - Columbia University (New York City), Children's Hospital (Boston), and Children's Hospital (Philadelphia). Participants are evaluated at one of these clinics regularly at no charge. During each visit, participants undergo several tests. The tests are safe and only a few of them cause mild discomfort. The information gained in this research is an important step in establishing a clinical trial for spinal muscular atrophy. Patient participation is an important part of furthering clinical research. Enrollment in this study will close in July but there is still an opportunity to participate. We encourage you to contact the study coordinator closest to you to find out if your child can participate in this important study.


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