Project Cure SMA
The Project Cure SMA Clinical Trial Network 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. Please click here for a recent newsletter describing their work.
1. Open-Label Testing of Tolerability and Safety of Sodium Phenyl Butyrate in Type II / III Children with SMA
2. Open-Label Trial for Tolerability and Safety of Valproic Acid in Type II / III Children with SMA (ClinicalTrials.gov identifier:
NCT00374075)
3. CARNI-VAL: Valproic Acid and Carnitine in Patients With Spinal Muscular Atrophy (ClinicalTrials.gov identifier:
NCT00227266)
4. Valproic Acid in Ambulant Adults With Spinal Muscular Atrophy (VALIANT SMA; ClinicalTrials.gov identifier:
NCT00481013 )
5. CARNIVAL Type I: Valproic Acid and Carnitine in Infants With Spinal Muscular Atrophy (SMA) Type I (ClinicalTrials.gov identifier:
NCT00661453)
They have also published 10 papers on SMA clinical trials:
1. SMA VALIANT Trial: A prospective, double-blind, placebo controlled trial of valproic acid in ambulatory adults with spinal muscular atrophy. Muscle Nerve. 2013 May 16. doi: 10.1002/mus.23904. Please click here for more details.
2. Reliability and validity of the TIMPSI for infants with spinal muscular atrophy type I. Pediatr Phys Ther. 2013 Summer: 25:140-8: doi: 10.1097/PEP.0b013e31828a205f. Please click here for more details.
3. SMA CARNI-VAL TRIAL PART II: A prospective, single-armed trial of l-carnitine and valproic acid in ambulatory children with spinal muscular atrophy. PLoS One. July 2011. 6(7): e21296. Please click here for more details.
4. Reliability of the modified hammersmith functional motor scale in young children with spinal muscular atrophy. Muscle and Nerve. Epub 2011 January. Please click here for more details.
5. SMA CARNI-VAL Trial Part I: double-blind, randomized, placebo-controlled trial of L-carnitine and valproic acid in spinal muscular atrophy. PLoS One. 2010 19;5(8):e12140. Please click here for more details.
6. Compound muscle action potential and motor function in children with spinal muscular atrophy. Muscle and Nerve. 2010 Nov;42(5):703-8. Please click here for more details.
7. Phase II open label study of valproic acid in spinal muscular atrophy. PLoS One. 2009;4(5):e5268. Please click here for more details.
8. Perspectives on clinical trials in spinal muscular atrophy. Journal of Child Neurology. 2007 Aug;22(8):957-66.
9. A modified Hammersmith functional motor scale for use in multi-center research on spinal muscular atrophy. Neuromuscular Disorders. 2006 Jul;16(7):417-26.
10. Natural history of denervation in SMA: relation to age, SMN2 copy number, and function. Annals of Neurology. 2005 May;57(5):704-12.
There are 7 North American locations for Project Cure SMA.
1) Salt Lake City, Utah
PI: Dr. Kathryn Swoboda
University of Utah
Department of Neurology
30 North 1900 East #3R210
Salt Lake City, Utah 84132
(801) 585-9717
2) Hartford, Connecticut
PI: Dr. Gyula Acsadi
Chief of Pediatric Neurology
Connecticut Children's Medical Center
282 Washington Street
Hartford, CT, 06106
3) Baltimore, Maryland
PI: Dr. Thomas Crawford
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, fax: (410) 614 - 2297
4) Montreal, Canada
PI: Dr. Guy D'Anjou
Hopital Sainte-Justine
Neurologie Pediatrique, 5e bloc 4
3175, Cote-Sainte-Catherine Road
Montreal, Quebec, Canada H3T 1C5
(514) 374-1710 ext. 8278
5) Columbus, Ohio
PI: Dr. John Kissel
Ohio State University
Department of Neurology
445 Means Hall 1654 Upham Drive
Columbus, Ohio 43210-1228
(614) 293-4973
6) Madison, Wisconsin
PI: Dr. Mary Schroth
University of Wisconsin Children’s Hospital
Pediatric Pulmonary & Cystic Fibrosis Center
600 Highland Avenue
Madison, WI 53792-9988
(608) 262-4982
7) Durham, North Carolina
PI: Dr. Priya Kishnani
Co PI: Dr. Edward Smith
Duke University Medical Center
(919)-681-1945
