An Open-label, Multicenter, Multinational, Ascending Dose Study of the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics, and Exploratory Efficacy of Repeated Biweekly Infusions of neoGAA in naive and Alglucosidase Alfa Treated Late-onset Pompe Disease Patients
Latest Information Update: 15 Dec 2023
At a glance
- Drugs Avalglucosidase alfa (Primary)
- Indications Glycogen storage disease type II
- Focus Adverse reactions; Biomarker
- Acronyms NEO1
- Sponsors Genzyme Corporation; Sanofi
- 01 Dec 2023 Population PK (PopPK) analysis was performed using nonlinear mixed effect modeling approach on pooled data from three clinical trials (NCT01898364, NCT02032524 and NCT02782741) with LOPD patients, and a phase 2 study (NCT03019406) with infantile-onset Pompe disease patients to propose alternative dosing regimens for pediatric LOPD patients based on a bodyweight cut-off,, published in the Journal of Pharmacokinetics and Pharmacodynamics.
- 26 May 2022 Results of an analysis of data up to 6.5 years experience with Avalglucosidase alfa during the NEO1 and NEO-EXT studies published in the Neurology
- 26 May 2020 Results presented at the 6th Congress of the European Academy of Neurology