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Families of SMA and Project Cure SMA are excited to announce 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, called Carni-Val Type I. 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. This study is being fully funded by Families of SMA. This is a multi-center trial; the centers now enrolling patients are listed on the Project Cure SMA website. Patients who can enroll in this study are infants with SMA Type I, ages 2 weeks to 9 months at time of enrollment, who have clinical features of SMA confirmed by genetic testing. Subjects will not be able to participate if there is evidence of other organ disease, medications intended to treat SMA are being used, medications that might interact with VPA are being used, or participation in other treatment studies of SMA within the previous 30 days has occurred. 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. If you are interested in participating in this study, please contact the clinical coordinator at a particular site closest to you. Contact information for coordinators and more details regarding eligibility criteria and potential enrollment can be found at both www.projectcuresma.org and www.fsma.org Note:
Funding for the site in Cologne in Germany is being provided by the "Initiative Forschung und Therapie für SMA".
Funding for the site in Montreal in Canada is being provided by "Families of SMA Canada".
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