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.