Without research, there are no new treatments

Duchenne muscular dystrophy (DMD) is a disease that almost exclusively affects boys and whose incidence is 1 in 3,500 – 5,000. It is extremely rare that Duchenne muscular dystrophy (DMD) will affect girls.

It is a degenerative disease of the muscles caused by a genetic mutation. The Duchenne muscular dystrophy (DMD) – for which no treatment is currently available – directly affects skeletal muscles. Without treatment, the consequences of the disease are dire for those afflicted and their families.

Clinical trials provide early access to treatments, contribute to medical knowledge about a condition, help guide future research, and have the potential to impact how people with the same condition are treated in the future.

ECS can help find a clinical trial that matches your condition. Their mission is to help every Canadian patient find a clinical trial that matches his/her medical condition. These services are free. You can fill out the form here.

In collaboration with Simplified Clinical Trials, here are the clinical trials currently taking place in Canada and Quebec. For more information on clinical trials being recruited in Canada for DMD, go to clinicaltrialssimplified.com.

Quebec

There are currently eight clinical trials underway for Duchenne muscular dystrophy in Canada, two of which have sites in Quebec: myoblast transplantation and the trial of Vamorolone.

Transplanting myoblasts

Consists of transplanting healthy muscle cells, called myoblasts, into forearm muscle in Duchenne patients and measuring muscle strength at 3 and six months posttransplantation. This trial is for patients aged 16 and over.

 

Vamorolone

The second trial aims to investigate the efficacy and safety of a drug called Vamorolone, a glucocorticoid, in boys with Duchenne aged 4-7 years.

 

Canada

Here are details of the six trials that are taking place elsewhere in Canada and that do not have a site in Quebec at this time: RO7239361, Givinostat, Edasalonexent, ESSENCE study (SRP-4045 and SRP 4053), Ataluren and Exondys 51.

 

RO7239361

Active, not recruiting

In this trial, a drug called RO7239361 is tested in boys with Duchenne aged 6 to 11 years. RO7239361 is a medicine that acts on myostatin, a protein that decreases muscle growth.

  • The RO7239361 binds to a protein called myostatin and limits its function. Myostatin is a naturally occurring protein that is produced by the body to stop muscles growing too large. Limiting myostatin has been shown in some studies to increase muscle mass. The hope is that stopping myostatin could increase muscle growth in children with Duchenne.
  • Clinical Trials, RocheClinical trials simplified

 

Givinostat

This trial involves a drug called Givinostat in boys with Duchenne aged 6 to 17 years. Givinostat is tested for its ability to increase the ability of muscles to regenerate.

 

Edasalonexent

Active, not recruiting

This trial tests a drug called Edasalonexent in boys with DMD aged 4 to 7 years. Edasalonexent acts on NF-kB, a protein that contributes to muscle degeneration in DMD.

 

SRP-4045 et le SRP-4053

This trial tests SRP-4045 and SRP-4053 in boys with Duchenne aged 7 to 13 years. SRP-4045 and SRP-4053 make it possible to skip the damaged portion of the dystrophin gene to allow the production of a shorter form of this protein.

  • Mutations in the dystrophin gene are one cause of DMD. Most commonly, one or more exons (a portion of a gene) are missing, and the remaining exons don’t fit together correctly. (Think of a zipper that doesn’t work properly because teeth are missing.) Because of this error, cells cannot make the dystrophin protein that muscles need to work properly. Without it, muscle cells become damaged and, over time, are replaced with scar tissue and fat. To fix the broken genetic machinery, scientists are developing drugs that skip over parts that contain missing or defective exons. In this way, the machine can produce a less imperfect dystrophin protein, which may improve muscle function in children with exon mutations. Sarepta investigational therapies in the ESSENCE study use a technique referred to as exon skipping. Skipping a specific exon next to the mutation is intended to allow the body to make a shortened form of the dystrophin protein.
  • Clinical Trials, Sarepta, ESSENCE StudyClinical trials simplified
  • A Double-Blind, Placebo-Controlled, Multi-Center Study With an Open-Label Extension to Evaluate the Efficacy and Safety of SRP-4045 and SRP-4053 in Patients With DMD

 

Ataluren

This trial tests Ataluren in male patients with DMD aged five years and older.

 

Exondys 51, SRP-5051

This trial tests SRP-5051 in male patients with DMD.

 

For more information on clinical trials currently recruiting in Canada for DMD, visit clinicaltrialssimplified.com/Muscular dystrophy

To participate in a clinical trial on DMD, please complete the registration form on clinicaltrialssimplified.com. For more information, contact CTS at info@clinicaltrialssimplified.com or 1-888-982-2782.