Studies Underway

There are currently 18 studies underway that include TVCC clients, their families, and/or TVCC staff:

• Canadian Neuromuscular Disease Registry
  Jan. 2010-Dec. 2021
• Myoblast transplantation in Duchenne and Becker muscular dystrophy patient
  May 2010-Nov. 2021
• Capturing the experience of sports-related concussions
  Mar. 2014-Nov. 2021
• A phase 3, randomized, double blind, sham-procedure controlled study to assess the clinical efficacy and safety of ISIS 396443 administered intrathecally in patients with later-onset spinal muscular atrophy
  Dec. 2014-Ongoing
• A two-part seamless, multi-center randomized, placebo-controlled, double-blind study to investigate the safety, tolerability, pharmacokinetics, pharmacodynamics and efficacy of Ro7034067 in type2 and 3 spinal muscular atrophy patients
  Dec. 2016-May 2022
• Trial readiness and endpoints assessment in congenital myotonic dystrophy
  Dec. 2016-Sept. 2022
• A double-blind, placebo-controlled, multicenter study with an open-label extension to evaluate the efficacy and safety of SRP-4045 and SRP-4053 in patients with Duchenne Muscular Dystrophy
  Dec. 2016-Jan. 2022
• Engagement in the pediatric rehabilitation intervention process: Its nature, measurement, and role in the determination of outcomes
  Oct. 2017-July 2022
• Children and teens in charge of their health: A feasibility study of solution-focused coaching to foster healthy lifestyles in children and young people with physical disabilities
  Dec. 2017-Feb. 2022
• A randomized, double-blind study to evaluate the efficacy and safety to Tideglusib versus placebo for the treatment of children and adolescents with congenital myotonic dystrophy (AMO study)
  Feb. 2018-Mar. 2022
• Health concerns of adolescents and adults with childhood onset physical disability
  June 2018-Mar. 2022
• Increasing our knowledge of social and motor skills in children with Autism Spectrum Disorder to help better understand participation in social activities 
  Oct. 2018-May 2022
• An open-label extension study for patients with Duchenne Muscular Dystrophy who participated in studies of SRP-5051
  Oct. 2018-Jan. 2022
• A phase2, two-part multiple-ascending dose study of SRP-5051 for dose determination, then dose expansion, in patients with Duchenne Muscular Dystrophy amenable to Exon 51-skipping treatment
  Jun. 2019-Sept. 2022
• A randomized, double-blind, dose finding and comparison study of the safety and efficacy of a high dose of Eteplirsen, preceded by an open-label dose escalation in patients with Duchenne muscular dystrophy with deletion mutations amenable to Exon 51 skipping
  Apr. 2020-Nov. 2021
• Escalating dose and randomized controlled study of Nusinersen (BIIB058) in participating with spinal muscular atrophy
  Apr. 2020-Sept. 2022
• Phase 3, multicenter, randomized, double-blind, placebo-controlled study to evaluate the safety and efficacy of PF-06939926 for the treatment of Duchenne muscular dystrophy
  Oct. 2020-Dec. 2021
• Fit & Function: Family perspective on fit between education interventions and functional needs for young children with neurodevelopmental disorders
  Jun. 2021-Apr. 2022