Trial Profile
An Open-Label, Fixed-Sequence, Ascending-Dose, First-in-Human Study to Assess the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics, and Efficacy of Intravenous Infusions of ATB200 Co-Administered With Oral AT2221 in Adult Subjects With Pompe Disease
Status:
Active, no longer recruiting
Phase of Trial:
Phase I/II
Latest Information Update: 10 Apr 2024
Price :
$35
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At a glance
- Drugs Cipaglucosidase alfa (Primary) ; Miglustat (Primary)
- Indications Glycogen storage disease type II
- Focus Adverse reactions; First in man; Pharmacokinetics; Registrational
- Sponsors Amicus Therapeutics
- 03 Apr 2024 Planned End Date changed from 1 Dec 2023 to 1 Jun 2024.
- 03 Apr 2024 Planned primary completion date changed from 1 Dec 2023 to 1 Jun 2024.
- 01 Apr 2024 Results evaluating long-term (up to 48 months) safety and efficacy of cipaglucosidase alfa plus miglustat in individuals living with Pompe disease published in the Journal of Neurology