COMET
Research type
Research Study
Full title
A phase 3 randomized, multicenter, multinational, double-blinded study comparing the efficacy and safety of repeated biweekly infusions of neoGAA (GZ402666) and alglucosidase alfa in treatment-naïve patients with late-onset Pompe disease
IRAS ID
201847
Contact name
Volker Straub
Contact email
Sponsor organisation
Genzyme Corporation
Eudract number
2016-000942-77
Duration of Study in the UK
4 years, 2 months, 30 days
Research summary
Summary of Research
This is a randomised, multicentre, multinational double-blinded study looking at comparing the efficacy and safety of neoGAA compared to algucosidase alfa given every 2 weeks in treatment-naïve patients with late-onset Pompe disease.
The objectives of this study are to determine efficacy and safety of neoGAA on skeletal muscle compared to algucosidase alfa.
Pompe is a rare, genetic disease caused by a deficiency of the enzyme acid alphaglucosidase (GAA) which breaks down glycogen. The glycogen that accumulates in body tissues, especially cardiac and respiratory muscles as well as other major skeletal muscles, leads to muscle weakness, loss of respiratory function and often premature death.
Alglucosidase Alfa (AA), a longterm enzyme replacement therapy (ERT), is a licensed product used worldwide in patients with Pompe disease. NeoGAA is a second generation ERT for Pompe disease, that in preclinical studies, is more potent than the existing standard of care. NeoGAA differs from GAA by having additional bisM6P residues on the surface which is intended to increase the uptake of GAA into the cells, particularly skeletal muscle cells, thus helping in the clearance of glycogen.
Summary of Results
This summary is for the study titled "A Phase 3 randomized, multicenter, multinational, double blinded study comparing the efficacy and safety of repeated biweekly infusions of avalglucosidase alfa and alglucosidase alfa in treatment naïve patients with late onset Pompe disease".
The research was carried out by Sanofi Research and Development. The study was conducted at 70 sites in 25 countries worldwide.
The study took place from November 2016 to May 2023.
This research was needed to compare a new treatment called avalglucosidase alfa to the existing treatment alglucosidase alfa in patients with late-onset Pompe disease who had not been treated before. Pompe disease is a rare genetic disorder that causes muscle weakness and breathing problems.
The main questions studied were:
• How well does avalglucosidase alfa work compared to alglucosidase alfa in improving breathing function?
• Is avalglucosidase alfa safe to use?101 patients with late-onset Pompe disease participated in the study. Patients were randomly assigned to receive either avalglucosidase alfa or alglucosidase alfa as an intravenous infusion every two weeks for about a year. After that, all patients received avalglucosidase alfa for up to 3 more years.
The main results showed:
• Avalglucosidase alfa showed substantial improvement in breathing function as compared to alglucosidase alfa after 1 year of treatment
• Avalglucosidase alfa also improved walking distance and muscle strength
• The safety of avalglucosidase alfa was similar to alglucosidase alfa, with possibly fewer side effects
• Benefits of avalglucosidase alfa were maintained for up to 3 years of treatmentThis study has helped patients by showing avalglucosidase alfa may be a more effective treatment option for late-onset Pompe disease. It has helped researchers better understand the long-term effects of this new therapy.
Further research is ongoing to study avalglucosidase alfa in other types of Pompe disease.
To learn more about this study, you can visit https://gbr01.safelinks.protection.outlook.com/?url=http%3A%2F%2Fwww.clinicaltrials.gov%2F&data=05%7C02%7Cnewcastlenorthtyneside1.rec%40hra.nhs.uk%7C2ec23a2f026e44c945a608dd5714482e%7C8e1f0acad87d4f20939e36243d574267%7C0%7C0%7C638762466795345233%7CUnknown%7CTWFpbGZsb3d8eyJFbXB0eU1hcGkiOnRydWUsIlYiOiIwLjAuMDAwMCIsIlAiOiJXaW4zMiIsIkFOIjoiTWFpbCIsIldUIjoyfQ%3D%3D%7C0%7C%7C%7C&sdata=re1o1iv2vdi1X4ynKH4A1Cu528LzM3HSYPlJBwPwtjc%3D&reserved=0 and search for NCT02782741 or https://gbr01.safelinks.protection.outlook.com/?url=https%3A%2F%2Fclick.pstmrk.it%2F3ts%2Fwww.clinicaltrialsregister.eu%252F%2FNBTI%2FsKi7AQ%2FAQ%2F1dde8dcc-c105-4a4f-b90b-f7a6fdd0def9%2F2%2FsPtHAfcOhm&data=05%7C02%7Cnewcastlenorthtyneside1.rec%40hra.nhs.uk%7C2ec23a2f026e44c945a608dd5714482e%7C8e1f0acad87d4f20939e36243d574267%7C0%7C0%7C638762466795358293%7CUnknown%7CTWFpbGZsb3d8eyJFbXB0eU1hcGkiOnRydWUsIlYiOiIwLjAuMDAwMCIsIlAiOiJXaW4zMiIsIkFOIjoiTWFpbCIsIldUIjoyfQ%3D%3D%7C0%7C%7C%7C&sdata=Jpj%2Fh3ki%2FTr6WsFJVtVZTHhnMb3W3%2FMLgcyVJmsMEf4%3D&reserved=0 and search for 2016-000942-77
REC name
North East - Newcastle & North Tyneside 1 Research Ethics Committee
REC reference
16/NE/0190
Date of REC Opinion
15 Jul 2016
REC opinion
Further Information Favourable Opinion