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Project Cure Spinal Muscular Atrophy

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. The primary goal of Project Cure SMA is to develop safe and well-tolerated clinical protocols to help identify effective therapies for SMA.


 

 

 

Project Cure SMA Clinical Trials

April 2008
Carni-Val Type I - a new clinical trial designed to evaluate the combination of Valproic acid (VPA) and L-Carnitine for the treatment of SMA in infants with Type I SMA

Primary Objective
This trial will assess the safety of VPA and L-Carnitine in infants and develop improved methods to assess the strength and motor abilities of severely affected infants.

Inclusion and Exclusion Criteria - please see Clinical Trials Criteria

Contact Information
If you are interested in participating and meet the above criteria, please contact the SLC Project Cure Site (see Locations.) You may also call the Project Cure Clinical Trial Manager, Dr. Sandra Reyna at 801-581-3551 or sreyna@projectcuresma.org


June 2007
Clinical Trials Criteria for Prospective Controlled Trial of Valproic Acid in Ambulant Adults with Spinal Muscular Atrophy (VALIANT SMA STUDY)

Primary Objective
The primary objective of this Phase II trial is to assess the efficacy of oral valproic acid (VPA) in adults with SMA.

Inclusion and Exclusion Criteria - please see Clinical Trials Criteria

Contact Information
John T. Kissel, MD, Professor of Neurology and Director, Division of Neuromuscular Disease at The Ohio State University Medical Center. If you are interested in participating and meet the above criteria, please contact Sharon Chelnick, Study Coordinator, at (614) 293-4973 or sharon.chelnick@osumc.edu.


May 2005
SMA CARNI-VAL TRIAL Multi-center Phase II Trial of Valproic Acid and Carnitine in Patients with Spinal Muscular Atrophy

Primary Objective
The primary objective of this Phase II trial is to assess the efficacy of oral valproic acid (VPA) and carnitine in SMA children 2-17 years of age.

Inclusion and Exclusion Criteria - please see Clinical Trials Criteria

Contact Information
If you are interested in participating and meet the above criteria, please contact the SLC Project Cure Site (see Locations.) You may also call the Project Cure Clinical Trial Manager, Dr. Sandra Reyna at 801-581-3551 or sreyna@projectcuresma.org


April 22, 2008