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
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
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".