Current Concierge-Supported Trials

The clinical trial (CT) Concierge is independently objective and serves as an impartial resource, not funded by industry and available for all neuromuscular stakeholders.

The order of trials listed here is at random and is not an indication of priority, nor does the inclusion of any trial listed here indicate bias towards any one company or site by the NMD4C or by extension the CT Concierge. NMD4C and its partnering organisations do not endorse participation in any particular trial, pharmaceutical company or product.

The trials listed here include those for which the Concierge is providing support to Canadian sites. This service is offered to all investigators from any region in Canada who wish to work with and receive support from the Concierge. You can contact the Concierge here for more information.

Recruiting

Open-label Phase 1b/2a Study of WVE-N531 in Patients With Duchenne Muscular Dystrophy - Wave Life Sciences

An Open-label Phase 1b/2a Study of WVE-N531 in Patients With Duchenne Muscular Dystrophy -Wave Life Sciences

Canadian Sites

  • Children's Hospital - London Health Sciences Centre  - London, ON

Inclusion Criteria:

  • Ambulatory or non-ambulatory male.
  • Age of ≥5 and ≤12 years at time of screening
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 - Pfizer

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 - Pfizer

Canadian Sites

  • Alberta Children's Hospital - Calgary, AB
  • BC Children’s Hospital - Vancouver, BC
  • Children's Hospital - London Health Sciences Centre - London, ON
  • Children's Hospital of Eastern Ottawa - Ottawa, ON

Inclusion Criteria

  • 4 Years to 7 Years  (Child)
  • Ambulatory
Phase 3, Randomized, Double-Blind, Trial of Pamrevlumab (FG-3019) or Placebo in Combination With Systemic Corticosteroids in Subjects With Non-ambulatory Duchenne Muscular Dystrophy (DMD)- Fibrogen

A Phase 3, Randomized, Double-Blind, Trial of Pamrevlumab (FG-3019) or Placebo in Combination With Systemic Corticosteroids in Subjects With Non-ambulatory Duchenne Muscular Dystrophy (DMD)- Fibrogen

Canadian Sites

  • BC Children’s Hospital - Vancouver, BC
  • Children's Hospital - London Health Sciences Centre - London, ON
  • Montreal Neurological Institute & Hospital - Montreal, QC

Inclusion Criteria:

  • Males
  • 12 years of age, non-ambulatory at screening initiation
Randomized, Double-Blind Study to Evaluate the Efficacy and Safety of Tideglusib Versus Placebo for the Treatment of Children and Adolescents with Congenital Myotonic Dystrophy (REACH CDM) - AMO Pharma Ltd

A Randomized, Double-Blind Study to Evaluate the Efficacy and Safety of Tideglusib Versus Placebo for the Treatment of Children and Adolescents with Congenital Myotonic Dystrophy (REACH CDM) -AMO Pharma Ltd

Canadian Sites

  • Children's Hospital - London Health Sciences Centre - London, ON
  • Children's Hospital of Eastern Ontario - Ottawa, ON

Inclusion Criteria:

  • Male or female children and adolescents aged ≥6 years and ≤16 years
  • Ambulatory
Study of SRP-4045 and SRP-4053 in DMD Patients (ESSENCE)- Sarepta Therapeutics, Inc

Study of SRP-4045 and SRP-4053 in DMD Patients (ESSENCE) - Sarepta Therapeutics, Inc

Canadian Sites

  • Alberta Children's Hospital - Calgary, AB
  • BC Children’s Hospital - Vancouver, BC
  • Children's Hospital - London Health Sciences Centre - London, ON
  • Children's Hospital of Eastern Ontario - Ottawa, ON

Inclusion Criteria:

  • Male
  • Between 7 and 13 years of age, inclusive, at randomization
Open-Label Study of Risdiplam in Infants With Genetically Diagnosed and Presymptomatic Spinal Muscular Atrophy - Hoffman-La Roche

An Open-Label Study of Risdiplam in Infants With Genetically Diagnosed and Presymptomatic Spinal Muscular Atrophy

Canadian Sites

  • Children's Hospital - London Health Sciences Centre - London, ON

Inclusion Criteria

  • Ages Eligible for Study: up to 6 Weeks (Child)
  • Sexes Eligible for Study: All
  • Accepts Healthy Volunteers: No
Phase 3 Randomized, Double-blind, Placebo-controlled, Multi-Jam A Phase 3 Randomized, Double-blind, Placebo-controlled, Multi-center Study to Assess the Efficacy and Safety of Viltolarsen in Ambulant Boys with Duchenne Muscular Dystrophy (DMD) - NS Pharma-065/NCNP-01-301

Inclusion Criteria

  • Male ≥ 4 years and < 8 years of age
  • Confirmed DMD mutation(s) in the dystrophin gene that is amenable to skipping of exon 53 to restore the dystrophin mRNA reading frame
  • Able to walk independently without assistive devices
  • TTSTAND < 10 seconds
  • Stable dose of glucocorticoid (GC) for at least 3 months prior to study entry and is expected to remain on stable dose of GC treatment for the duration of the study
  • Other inclusion criteria may apply