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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.

The study group is looking for SMA patients ages 18 to 60 at the time of study enrollment who are able to walk at least 30 feet without assistance (no canes, walkers, etc.). Participants must have a confirmed diagnosis of 5q SMA through standard genetic testing.

Exclusions for participation include (but are not limited) to the following: non-ambulatory type 3 adults and all type 2 adults; patients with co-existing medical conditions that preclude travel, testing or study medications; patients who have participated in a treatment trial for SMA in the 3 months prior to this trial, or plan on enrolling in any other treatment trial during the duration of this trial; the need for BiPAP support for > 12 hours/day; use of medications or supplements which interfere with VPA metabolism and increase the potential risks of the medications, or are hypothesized to have a beneficial effect in SMA animal models or human neuromuscular disorders within 3 months of study enrollment; and women who are pregnant or who intend to become pregnant while participating in the research study or who are breastfeeding.

Contact Information : 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.

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".