Clinical Trial Information

SMA Therapeutic Trials at Stanford University Medical Center

Dr. Ching H. Wang and his colleagues at Stanford University would like to announce two therapeutic trials using hydroxyurea as a treatment for pediatric spinal muscular atrophy (SMA). The first study is targeted at children with Type I SMA and less than 2 years of age. We are looking to enroll 18 patients over the next three years for this study. The second study is targeted at patients 2 to 8 years of age with SMA of intermediate severity (those diagnosed with type II and early type III SMA). We are looking to enroll 15 patients over the next year for this study. Patients in both studies should have genetic testing confirming homozygous deletion of the SMN1 gene.  

The treatment period for each participating patient will be 6 months. There will be a one month observation period prior to the treatment. During this time, a monthly visit to Stanford University Medical Center is required. Several clinical and laboratory observations will be obtained during the entire study period.

For more information for the SMA therapeutic trials at Stanford, please contact our study coordinator, Tony Trela, by phone at 650-498-7658 or by e-mail at sma@stanfordmed.org. Our website is http://sma.stanford.edu.  

Our Research Team

If after reading this pamphlet you would like to learn more about our research, please feel free to contact us using the information below.

Tony Trela, Research Coordinator

or

Ching Wang, Principle Investigator

or

visit our website at http://sma.stanford.edu

SMA Clinical Trials Stanford University

Introduction

This pamphlet is intended strictly as an informational resource for patients interested in participating in clinical trials relevant to Spinal Muscular Atrophy (SMA).  Reading this pamphlet or asking further questions about our research does not in any way oblige you to participate in the trials.

What is a clinical trial?

A clinical trial takes place after years of laboratory research investigating the cause and treatment of a disease. 

After identifying the cause of a disease, researchers and physicians can design potential treatments.  These treatments can be new drugs, or apply old drugs in new ways.

Clinical trials test treatments in human volunteers in order to check their safety and effectiveness before they can be approved for use in treating the general population. 

What is the purpose of these SMA clinical trials at Stanford?

These clinical trials are aimed at obtaining data on the safety and effectiveness of some medications that may help the symptoms of SMA patients.

In addition, these trials hope to identify some reliable tools to measure the clinical effects of these treatments.

Who is eligible for the trial?

Two groups of patients with SMA will be recruited for these studies.

1. Type I SMA patients less than two years of age.

2.  Type II SMA and severe Type III SMA patients between 2 and 8 years of age (those never achieve independent walking and those who were able to walk but need some form of assisted devise before the age of 6 years).

What are my child’s requirements for participation?

Full participation in either study will involve a time commitment of at least one month of baseline observation and six months of treatment.

Both studies will require periodic physical exams and blood draws from your child.  These will be used to monitor possible side effects of the treatment as well as for analysis in our research lab.

There will also be tests performed to measure your child’s muscle strength and breathing capacity.

There will be no cost for the family to participate in these studies. The investigators will pay for all procedures related to the trials (blood tests, physical therapy, etc.).  However, you will be responsible for costs of routine care for your child.

Who is funding these projects?

This project is partially funded by 

    1.  The Spinal Muscular Atrophy  

         Foundation (SMAF)

    2.  The Muscular Dystrophy 

         Association (MDA), USA