Current Concierge-Supported Trials

Pediatric Trials Supported by the Clinical Trial Concierge

Open for Recruitment

Vamorolone NCT05185622

A Phase II Open-Label, Multiple Dose Study to Assess the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics, and Exploratory Efficacy of Vamorolone in Boys Ages 2 to <4 Years and 7 to <18 Years With Duchenne Muscular Dystrophy (DMD)- Sponsor: ReveraGen BioPharma, Inc.

Sponsor: ReveraGen BioPharma, Inc.

Canadian Sites

232SM203 DEVOTE NCT02196467

Escalating dose and randomized, controlled study of nusinersen (BIIB058) in participants with SMA
The objective of this study is to examine the clinical efficacy of nusinersen administered intrathecally at higher doses to participants with SMABiogen.

Sponsor: Biogen

Canadian Site:

  • Children’s Hospital at London Health Sciences Centre (Dr. Craig Campbell).
232SM302 ONWARD NCT04729907

A Long-Term Extension Study of Nusinersen (BIIB058) Administered at Higher Doses in Participants with Spinal Muscular Atrophy who Previously Participated in an Investigational Study with Nusinersen.

Sponsor: Biogen

Canadian Site:

  • Children’s Hospital at London Health Sciences Centre (Dr. Craig Campbell).
NCT03692312

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) (also have the open-label extension to this study, Efficacy and Safety of Tideglusib in Congenital Myotonic Dystrophy”).

Sponsor: AMO

Canadian Site:

NCT04281485

A Phase 3, multicentre, randomized, double-blind, placebo-controlled study to evaluate the safety and efficacy of PF-06939926 for the treatment of Duchenne Muscular Dystrophy.

Sponsor: Pfizer

Canadian Sites:

  • Children's Hospital of Eastern Ontario (Dr. Hanns Lochmüller).
  • Alberta Children's Hospital (Dr. Jean Mah) [CLOSED].
WVE-N531-001 NCT04906460

An open-label phase 1b/2a study of WVE-N531 in patients with DMD
The objective of this study is to evaluate the safety, tolerability, PK, PD, and clinical effects of intravenous WVE-N531 in patients with DMD amenable to exon 53 skipping intervention.

Sponsor: Wave

Canadian Sites:

  • Children’s Hospital at London Health Sciences Centre (Dr. Craig Campbell).
NCT02500381

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.

Sponsor: Sarepta

Canadian Sites:

SAREPTA 5051-201 NCT04004065

A Phase 2, 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.

Sponsor: Sarepta

Canadian Sites:

  • Children’s Hospital at London Health Sciences Centre (Dr. Craig Campbell)
NCT03783923

A Multi-Center, Longitudinal Study of the Natural History of Subjects with Limb Girdle Muscular Dystrophy (LGMD) Type 2E (LGMD2E/R4), Type 2D (LGMD2D/R3), and Type 2C
(LGMD2C/R5)

Sponsor: Sarepta

Canadian Sites:

  • Children’s Hospital at London Health Sciences Centre (Dr. Craig Campbell).
NCT04632940 Pamrevlumab 094

A Phase 3, Randomized, Double-Blind, Trial of Pamrevlumab (FG-3019) or Placebo in Combination with Systemic Corticosteroids in Subjects with Ambulatory Duchenne Muscular Dystrophy (DMD) (093/094).

Sponsor: FibroGen

Canadian Site:

  • Children’s Hospital at London Health Sciences Centre (Dr. Craig Campbell).

Forecasted

NCT05524883 DYNE-251

A Randomized, Double-Blind, Placebo-Controlled, Multiple Ascending Dose Study Assessing Safety, Tolerability, Pharmacodynamics, Efficacy, and Pharmacokinetics of DYNE-251 Administered to Participants with Duchenne Muscular Dystrophy Amenable to Exon 51 Skipping.

Sponsor: Dyne Therapeutics

Canadian Site:

  • Children’s Hospital at London Health Sciences Centre (Dr. Craig Campbell). [PENDING]

Closed for Recruitment

396443-CS11 SHINE Study NCT02594124

An open-label extension study for patients with SMA who previously participated in investigational studies of ISIS 396443
The objective of this study is to gather additional information on the long-term safety, tolerability, and efficacy of repeated 12-mg doses of ISIS 396443 (nusinersen) administered as intrathecal injections by lumbar puncture in subjects with SMA who previously participated in investigational studies of ISIS 396443Biogen.

Sponsor: Biogen

Canadian Sites:

  • Children’s Hospital at London Health Sciences Centre (Dr. Craig Campbell)
NCT02908685 BP39055 SUNFISH

A Study to Investigate the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics and Efficacy of Risdiplam (RO7034067) in Type 2 and 3 Spinal Muscular Atrophy (SMA) Participants (SUNFISH).

Sponsor: Roche

Canadian Site:

  • Children’s Hospital at London Health Sciences Centre (Dr. Craig Campbell).
NCT03179631

A Phase 3, Randomized, Double-blind, Placebo-controlled Efficacy and Safety Study of Ataluren in Patients With Nonsense Mutation Duchenne Muscular Dystrophy and Open-Label Extension -( also have the open label extension to this study).

Sponsor: PTC Therapeutics

Canadian Sites:

124-GD-016-DMD NCT01247207

An open-label safety study for ataluren (PTC 124) patients with nonsense mutation dystrophinopathy
The objective of this study is to assess the safety and tolerability of 10, 10, 20 mg/kg ataluren in subjects with nmDBMD who had prior exposure to ataluren in a PTC-sponsored clinical trial or treatment plan, and siblings of those patients (provided those patients have completed the placebo-controlled portion of the trial).

Sponsor: PTC Therapeutics

Canadian Site:

  • Children’s Hospital at London Health Sciences Centre (Dr. Craig Campbell).

Adult Trials Supported by the Clinical Trial Concierge

Open for Recruitment

NCT04886518 HBS-101-CL-005

DM1, Harmony Biosciences study. Safety and Efficacy of Pitolisant on Excessive Daytime Sleepiness and Other Non-Muscular Symptoms in Patients With Myotonic Dystrophy Type 1 Followed by an Open-Label Extension.

Sponsor: Harmony

Canadian Sites:

  • The Ottawa Hospital (Dr. Hanns Lochmüller)
  • Montreal Neurological Institute and Hospital (Dr. Erin O'Ferrall)
  • Hôpital de Chicoutimi (Dr. Cynthia Gagnon)
NCT04990388

Safety, Tolerability, and Pharmacokinetics of UX053 in Patients With Glycogen Storage Disease Type III (GSD III)

Sponsor: Ultragenyx Pharmaceutical Inc.

Canadian Sites:

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.