The Mini-COMET study (NCT03019406) evaluated the efficacy and safety of avalglucosidase alfa (Nexviadyme) in patients with infantile-onset Pompe disease who had previously shown clinical deterioration or suboptimal response to alglucosidase alfa (Myozyme) :
- The study included 22 patients under the age of 18 years divided into three groups.
- Patients who showed clinical deterioration were assigned to groups 1 and 2 and received avalglucosidase alfa at doses of 20 and 40 mg/kg every other week, respectively, for 6 months.
- Patients who showed a suboptimal response to Myozyme were divided into groups 3a and 3b who received either avalglucosidase alfa 40 mg/kg or continued treatment with Myozyme every two weeks, respectively, for 6 months.
- Treatment was well tolerated and no serious treatment-related adverse events were observed.
- Participants receiving 40 mg/kg showed improvement or stabilization of mobility and motor skills.
The study is being conducted in 6 countries around the world, including France, for a planned duration of 7 years, and all participants will be treated with Nexviadyme.
