Families of Spinal Muscular Atrophy Announces New Clinical Trial Site for SMA at Duke University in North Carolina.
April 9, 2009.

Families of Spinal Muscular Atrophy is pleased to announce that Duke University in Durham, North Carolina has been added as a new site to the clinical trial network Project Cure SMA.

Dr. Priya Kishnani, Professor of Pediatrics, Chief Division of Medical Genetics, will serve as the site director for the study.  Dr. Edward C. Smith, Medical Instructor in the Department of Pediatrics, Division of Neurology, will be assisting at the site.  Dr. Kishnani has critical experience conducting clinical trials in infants with neuromuscular disease, having been the lead principle investigator during the clinical trials of the Genzyme Corporation drug Myozyme® for Pompe’s Disease. 

The Duke site will be part of the ongoing multi-center trial of Valproic Acid and Carnitine in infants with Type I Spinal Muscular Atrophy that is now recruiting at seven centers across North America.

The addition of the Duke University site is critical to this trial and to the future success of clinical studies in the SMA patient population.  Because Type I infants have a difficult time traveling, multiple regional trial sites will be key to conducting clinical trials for SMA.  Establishing a Project Cure SMA site in the Southeastern United States is another step in our long-term goal of growing the clinical network to encompass regional coverage across the country in preparation for pivotal trials of novel SMA drugs.

Carni-Val Type I Trial Update:
This clinical trial is designed to evaluate the combination of Valproic Acid (VPA) and L-Carnitine for the treatment of SMA in infants with Type I SMA and is being conducted by the Project Cure SMA Clinical Trial Group.  This trial, which is fully funded by Families of SMA for all US sites and for all support functions, will assess the safety of VPA and L-Carnitine in Type I  infants and will develop improved methods to assess the strength and motor abilities of severely affected infants.

The following seven North American sites are currently enrolling infants into the trial.
-Salt Lake City, Utah.
-Detroit, Michigan.
-Baltimore, Maryland.
-Montreal, Canada.
-Columbus, Ohio.
-Madison, Wisconsin.
-Durham, North Carolina.

A German site,  Klinikum der Universität zu Köln, will begin enrollment in June 2009.

At this time we are also announcing an update for the inclusion criteria for the trial. The age limit for study participation has now been increased to 12 months.  Thirty-six infants with SMA Type I, ages 2 weeks to 12 months at the time of enrollment, who have clinical features of SMA confirmed by genetic testing, will be enrolled in the trial. Because travel for infants with severe SMA is often difficult, study site investigators will be looking to enroll subjects who live close to the individual centers.

As of April 2009, 18 infants have been enrolled in the study, and all sites continue to enroll. 

If you are interested in participating in this study, please contact the clinical coordinator at a particular site closest to you. More details regarding eligibility criteria and potential enrollment can be found at www.clinicaltrials.gov and www.projectcuresma.org.  In addition, we recommend that families who are interested in being contacted for this trial or future clinical trials should register with the International SMA Patient Registry at Indiana University.

About Project Cure SMA:
Project Cure SMA is a collaborative initiative between Families of SMA and clinical investigators designed to help facilitate the rapid translation of promising new therapies to individuals with SMA.  A primary goal of Project Cure SMA is to develop safe and well tolerated clinical protocols to help identify truly effective therapies.  Funding for all support staff and for all sites in the United States is being provided by Families of SMA.  Funding for the site in Cologne, Germany is being provided by the “Initiative Forschung und Therapie fur SMA”.  Funding for the site in Montréal, Canada is being provided by Families of SMA Canada.