|
|
|
| | New Multi-Center Trial of Valproic Acid and Carnitine in Infants with Type I Spinal Muscular Atrophy is Currently Recruiting at Six Centers Across North America.
In April Families of SMA announced 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. Specifically this trial, which is fully funded by Families of SMA, 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.
The following six North American sites are currently enrolling infants. A German site will begin enrollment in several months.
Salt Lake City, Utah
University of Utah
Department of Neurology
30 North 1900 East #3R210
Salt Lake City, Utah 84132
(801) 585-9717
Sofya Sadiq: saltlakecity@projectcuresma.org
Detroit, Michigan
Children's Hospital of Michigan
Department of Pediatrics and Neurology
Wayne State University, School of Medicine
Clinical Pharmacology
3901 Beaubien Blvd. Detroit, MI 48201
(313) 745-6615
Kristin Kennedy: detroit@projectcuresma.org
Baltimore, Maryland
Johns Hopkins Hospital
Department of Pediatric Neurology
Harriett Lane Children's Health Building
200 N. Wolfe Street, Suite 2158
Baltimore, MD 21287
phone: (443) 287 - 6294
Bessy Guevara: baltimore@projectcuresma.org
Montreal, Canada
CRME du CHU Ste-Justine
5200 Bélanger est
Montréal, Québec, Canada H1T 1C9
(514) 374-1710 ext. 8278
Monique Émond: montreal@projectcuresma.org
Columbus, Ohio
The Ohio State University Medical Center
Department of Neurology
1654 Upham Drive
445 Means Hall
Columbus, Ohio 43210-1228
(614)722-2654
Shelli.Farley: Shelli.Farley@NationwideChildrens.org
Madison, Wisconsin
University of Wisconsin SOM and PH
Department of Pediatrics
600 Highland Avenue
K4/938 CSC
Madison, WI 53792-9988
(608) 262-4982
Lisa Pharo: madison@projectcuresma.org
Thirty-six 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, 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.
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.
Note:
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 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.
|
|
|
