Progress is being made every day
Now more than ever there are clinical trials taking place to find better treatments and improve quality of life for people affected by neuromuscular disorders. And, Canadian clinician investigators are playing an active role in this research effort.
Studies currently recruiting participants:
- A Phase 2 Study to Evaluate the Safety, Efficacy, Pharmacokinetics (PK) and Pharmacodynamics (PD) of PF-06252616 in Duchenne Muscular Dystrophy
Canadian sites: Montreal (Quebec), London(Ontario)
PF-06252616 is an experimental anti-myostatin monoclonal antibody. Myostatin acts in the body to help regulate muscle growth by inhibiting (blocking) muscle differentiation and growth. Blocking the activity of myostatin may have therapeutic application in treating muscle wasting diseases, such as Duchenne Muscular Dystrophy. Based on the proposed mechanism of action of PF-06252616, there is the potential to increase muscle mass and function in boys with Duchenne who have evidence of reduced muscle mass. This is a Phase 2 randomized, 2-period, double-blind, placebo-controlled, multiple ascending dose study to evaluate the safety, efficacy, PK and PD of PF-06252616 administered to ambulatory boys diagnosed with Duchenne Muscular Dystrophy.
- Study of BMS-986089 in Ambulatory Boys With DMD
Canadian sites: Montreal (Quebec), London (Ontario)
BMS-986089 is an investigational protein that is designed to bind to myostatin. Myostatin is a protein produced primarily in skeletal muscle cells that prevents muscle cell growth and differentiation. The purpose of this study is to determine the safety and tolerability of BMS-986089 in boys with Duchenne Muscular Dystrophy.
- Safety, Tolerability, Pharmacokinetics and Efficacy of CFZ533 in Moderate to Severe Myasthenia Gravis.
The purpose of this study is to evaluate safety, tolerability, pharmacokinetics/pharmacodynamics and efficacy of CFZ533 as an add-on therapy to standard of care in patients with moderate to severe myasthenia gravis (MG). The CFZ533 is a biologic which is an antibody that binds to a protein called CD40 which exists in some blood cells. The Montreal Neurological Institute and Hospital will be one of the locations.
- Transplantation of Myoblasts to Duchenne Muscular Dystrophy
Canadian sites: Quebec, London
Principal investigators: Drs Jack Puymirat and Dr. Craig Campbell
Sponsor id: Dr. Jacques P. Tremblay
This Phase I/II of the clinical trial is to investigate whether the transplantation of normal myoblasts throughout one muscle (the extensor carpi radialis) of the patients will increase the strength of that muscle. During this Phase I/II, the patients will be transplanted with myoblasts grown from the muscle biopsy of a donor and kept frozen in liquid nitrogen. Thirty million myoblasts will be injected per cm cube in a progressively higher surface of the radialis (i.e., 3, 6 and 9 cm2). The contralateral muscle will be injected with saline to serve as a control. The strength of both muscles will be measured at 3 months post transplantation to verify whether the myoblast transplantation increased the strength of the muscle. If there is no significant strength increase the protocol will be terminated immediately for that patient. If there is a significant strength increase, the patient will be maintained under immunosuppression until 6 months post transplant and his strength will be re-evaluated.
- Finding the Optimum Regimen for Duchenne Muscular Dystrophy (FOR-DMD)
Canadian Sites: London, Calgary, Vancouver, Winnipeg and Ottawa
Principal Investigator: Robert C. Griggs, MD University of Rochester
Principal Investigator: Kate Bushby, MD Newcastle University
- A Study to Assess the Efficacy and Safety of ISIS-SMN Rx in Infants With Spinal Muscular Atrophy
Canadian sites: Montreal, Toronto, Vancouver
- Stacking Exercises Aid the Decline in FVC and Sick Time (STEADFAST)
Canadian sites: Calgary, Edmonton, Halifax, Hamilton, London, Montreal, Ottawa, Quebec City, Toronto, Winnipeg, Vancouver
Principal Investigator: Sherri Katz, MD Childrens Hospital of Eastern Ontario
Breathing complications are the main reason for sickness and death in Duchenne muscular dystrophy. A technique called “lung volume recruitment” (LVR) is performed using a device consisting of a face mask or mouthpiece and self-inflating bag that inflates the lungs. This helps to clear the airways of secretions by increasing the forcefulness of a cough, and it may reduce areas of lung collapse and prevent pneumonia. Our aim is to conduct a multi-centre randomized controlled trial of LVR used twice daily in addition to conventional treatment, compared to conventional treatment. The impact of LVR therapy on lung function, respiratory infections, hospitalizations and quality of life will be evaluated. If effective, this simple treatment will eventually become part of standard of care worldwide for individuals with Duchenne muscular dystrophy, improving their lung function and quality of life.
- A comparison of three methods for improving expiratory coughflow and lung volume in children with neuromuscular diseases
Principal Investigator, Mike Seear, MD, British Columbia Children’s Hospital
This study will compare three different methods for improving expiratory cough flow and lung volume. The outcome of this project will provide data to help improve the management of lung complications in children with neuromuscular disorders. Read more
Ongoing studies with a Canadian team (not currently recruiting participants):
To find additional information, please contact the site investigator. Contacts at each location can be found by clicking on the hyperlinked title of the study that you are interested in. You will be directed to ClinicalTrials.gov – a searchable online database of clinical studies of human participants conducted around the world.
Muscular Dystrophy Canada does not fund clinical studies or participate in the selection process, unless noted otherwise.
Presenter: Deborah Chiabai
Description: Debra discusses her son, who is affected by Duchenne muscular dystrophy, and her perspective on having him participate in clinical trials.
An article from Quest magazine