NeoGAA, a new generation of enzymotherapy developed by Sanofi Genzyme, will be tested in 96 patients with type 2 glycogenosis (Pompe disease). Recruitment of this trial is underway.
This international trial will be conducted in more than 20 different countries. In France, Reference Centres for “Neuromuscular Diseases” in Nice, Paris, Lille, Lyon, Marseilles, Bordeaux and Angers, as well as the “Hereditary Diseases of Metabolism” reference centre.
The aim is to compare the safety and efficacy of neoGAA with that of Myozyme®, the only treatment currently available for Pompe disease. A total of 96 people will be treated over 1 year, either with neoGAA or Myozyme®. This trial began in October 2016 and is expected to be completed by 2020.
A new generation recombinant enzyme
NeoGAAA and Myozyme® are recombinant forms (created by genetic engineering) of the human acid alpha-glucosidase (GAA), the defective enzyme in Pompe disease. Myozyme® has been used since 2006 to treat Pompe disease.
NeoGAA is a second-generation drug candidate developed from Myozyme® to increase its efficacy. It has already been tested in humans in a phase 1/2 trial (NEO1) conducted over 6 months in 24 patients with Pompe disease. Based on preliminary results from this trial, NeoGAA was qualified as an orphan drug by the European Medicines Agency. This designation applies to drug candidates (which have not yet proved their efficacy) in rare diseases, to facilitate the different stages of their development.
To participate in the trial
The trial is being conducted in more than 75 different centres around the world, including 8 in France (Nice, Paris, Lille, Lyon, Marseille, Bordeaux, Angers).
Individuals wishing to participate in this trial must meet certain criteria and obligations related to the trial protocol (to ensure valid and reliable conclusions). Main eligibility criteria include having Pompe disease, being at least 3 years old and having never received a Myozyme® infusion.
> More information about the trial protocol is available on clinicaltrials.gov.
> For further information or to participate in this trial as a patient, you can contact your consultant or the Reference Centre for Neuromuscular Diseases in Nice.
Contact: Pr Sabrina SACCONI- sacconi.s@chu-nice.fr; tel: 04 92 03 57 53.
