STRENGTH Study: Zolgensma for Children Aged 2 to 12 With Spinal Muscular Atrophy
In October 2021, Minister Dubé announced that Zolgensma, a drug produced by the Novartis pharmaceutical company, was added to the list of drugs offered in Quebec health facilities to treat children under the age of 2 with spinal muscular atrophy.
Spinal muscular atrophy is a rare genetic neuromuscular disease. It is the leading genetic cause of death in young children and is characterized by a progressive and permanent loss of the cells that control muscles. In the most severe cases, children experience increasing muscle weakness and paralysis, as well as difficulty swallowing and breathing.
As this drug is not currently available for children over 2 years of age, Novartis is using this new phase III study to explore the efficacy of gene therapy with a single dose of Zolgensma for children aged 2 to 12 years. This is an ultra-competitive study being conducted in 15 hospitals in 10 countries, with a recruitment target of only 28 pediatric patients worldwide.
As mentioned in our December 2022 newsletter, Dr. Maryam Oskoui, this study’s head researcher, highlighted the important role CATALIS’ FAST TRACK Evaluation Service plays in positioning Quebec at the forefront of clinical research – an advantage that has allows the province’s littlest patients to participate in this new study.
The Research Institute of the McGill University Health Centre (RI-MUCH) authorized the STRENGTH study in 12 weeks, putting it in first place among activated sites worldwide. The study is now open for recruitment and should allow the enrolment of 3 to 4 Quebec patients.
“This study is important because it represents the hope for a less invasive and more effective treatment than the ones our young patients are currently receiving. It goes without saying that by speeding up approval times for clinical studies, Quebec is becoming very competitive on the international stage, which increases our chances of conducting innovative studies like the STRENGTH study.” – Dr. Maryam Oskoui, Scientist at the RI-MUHC
“The FAST TRACK Evaluation Service has allowed us to begin clinical research projects more rapidly while still following the rigorous regulatory and ethical process which ensures patient safety. We are extremely proud of being the first site in the world to be activated for this study, which will allow Dr. Oskoui to offer this new treatment faster to her young patients.” Dr. Rhian Touyz, Executive Director and Chief Scientific Officer, RI-MUHC
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.