MASter-1 Study: Authorization in 8 Weeks to Explore a New Treatment for an Extremely Rare Disease
As mentioned in our December 2022 newsletter, CATALIS is proud to support the Quebec government’s Politique québécoise sur les maladies rares (Quebec policy on rare diseases – in French only), namely by allowing studies to be activated more quickly thanks to its FAST TRACK Evaluation Service.
Recently, 3 Novartis studies on rare diseases, were activated in record time. In addition to the STRENGTH study on Spinal Muscular Atrophy and the NEPTUNUS-2 study on Sjörgren’s syndrome, CATALIS’ FAST TRACK Evaluation Service also served to allow Novartis’ MASter-1 study to be authorized by the CHU Sainte-Justine in 8 weeks; the hospital’s site was the first to be activated in Canada, out of a total of 17 sites in 10 countries. Despite the number of sites in the process of being activated, this study seeks to recruit only 8 pediatric patients worldwide.
MASter-1 is a phase II study investigating a new treatment, MAS825, for an extremely rare infant autoimmune disease, NLRC4-GOF, which causes macrophage activation syndrome and necrotizing enterocolitis.
Currently, cortisone, which has limited effectiveness and inhibits the development in children, is the only treatment used to reduce inflammation caused by the disease. The Novartis study explores the efficacy of a new drug that could be less harmful to pediatric patients with the disease and, therefore, offer them a better quality of life.
Although the clinical trial is currently scheduled to recruit 2 to 3 pediatric patient in Quebec, Dr. De Bruycker, the study’s lead researcher, thinks that this study’s potential medical breakthroughs could also have a beneficial impact on many other children with autoimmune diseases who are looking for better treatments.
“As a doctor, you often feel powerless in the face of the devastation caused by rare diseases in children. Access to a potential new treatment through this clinical trial is an unparalleled source of hope for CHU Sainte-Justine patients and their families and could save lives.” – Dr. Jean-Jacques De Bruycker, lead researcher at the CHU Sainte-Justine
“In many cases, there is no therapeutic solution for patients with rare diseases. With CATALIS’ support, CHU Sainte-Justine is pleased to facilitate access to innovative clinical trials for patients with these diseases and to help identify promising treatments.” – Dr Jacques L. Michaud, Director, CHU Sainte-Justine Research Centre
”From an ethical standpoint, it is important for the patients and the people of Quebec, that clinical trials be conducted in Quebec. In this context, the FAST TRACK Evaluation Service is a positive example, in my opinion, of teamwork and of consolidating expertise to achieve this.” – Geneviève Cardinal, president of the CHU Sainte-Justine’s research ethics committee
The CATALIS’ FAST TRACK Evaluation Service has so far activated 11 clinical trials. Thanks to the commitment of its Network of partners, CATALIS is pleased to continue building on this momentum and reduce the approval times of clinical trials by almost 75% in order to accelerate the development of potential innovative treatments for patients.
If you are a pharmaceutical company and would like more information about the FAST TRACK Evaluation Service, please write to us at firstname.lastname@example.org.