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AT2221

DRUG7 trials

Sponsors

Amicus Therapeutics Inc., Amicus Therapeutics

Conditions

Adult Subjects With Late Onset Pompe Disease (LOPD)Late-onset Pompe diseasePediatric Subjects with Infantile-onset Pompe Disease Aged 0 to < 18 YearsPompe DiseasePompe Disease (Late-onset)Pompe Disease Infantile-Onset

Phase 1

First-In-Human Study to Evaluate Safety, Tolerability, and PK of Intravenous ATB200 Alone and When Co-Administered With Oral AT2221
CompletedNCT02675465
Amicus TherapeuticsPompe Disease
Start: 2016-04-30End: 2024-08-22Updated: 2025-10-23

Phase 3

A Study to Assess the Long-term Safety and Efficacy of ATB200/AT2221 in Adult Subjects With Late-Onset Pompe Disease (LOPD)
CompletedNCT04138277
Amicus TherapeuticsPompe Disease (Late-onset)
Start: 2019-12-18End: 2024-12-31Updated: 2026-03-05
An Open-label Study to Evaluate the Safety, Efficacy, Pharmacokinetics, Pharmacodynamics, and Immunogenicity of Cipaglucosidase Alfa/Miglustat in Both ERT-experienced and ERT-naïve Pediatric Subjects with Infantile-onset Pompe Disease Aged 0 to <18 Years
RecruitingCTIS2022-501095-25-01
Amicus Therapeutics Inc.Pediatric Subjects with Infantile-onset Pompe Disease Aged 0 to < 18 Years
Start: 2024-11-04Target: 15Updated: 2026-01-26
An Open-label Study of the Safety, Pharmacokinetics, Efficacy, Pharmacodynamics, and Immunogenicity of Cipaglucosidase Alfa/Miglustat in Pediatric Subjects Aged 0 to < 18 Years with Late-onset Pompe Disease
RecruitingCTIS2022-502547-36-00
Amicus Therapeutics Inc.Late-onset Pompe disease
Start: 2024-03-21Target: 9Updated: 2025-12-29
A Phase 3 Open-label Extension Study to Assess the Long-term Safety and Efficacy of Intravenous ATB200 Co-administered With Oral AT2221 in Adult Subjects With Late-onset Pompe Disease
Active, not recruitingCTIS2023-505170-15-00
Amicus Therapeutics Inc.Adult Subjects With Late Onset Pompe Disease (LOPD)
Start: 2020-05-15Target: 34Updated: 2025-07-18

Unknown Phase

Expanded Access for ATB200/AT2221 for the Treatment of Pompe Disease
NCT03865836
Amicus TherapeuticsPompe Disease
Updated: 2025-09-15
Expanded Access for ATB200/AT2221 for the Treatment of IOPD
NCT04327973
Amicus TherapeuticsPompe Disease Infantile-Onset
Updated: 2025-09-10

Related Papers

A disease progression model comparing the long-term mobility and respiratory outcomes of adults with late-onset Pompe disease receiving cipaglucosidase alfa plus miglustat versus alglucosidase alfa.
2026-07-15
Safety of home administration of cipaglucosidase alfa plus miglustat in late-onset Pompe disease: results from multiple clinical trials
Therapeutic Advances in Rare Disease2026-01-01
An Indirect Treatment Comparison of Avalglucosidase Alfa versus Cipaglucosidase Alfa Plus Miglustat in Patients with Late-Onset Pompe Disease.
2025-09-08
104-week efficacy and safety of cipaglucosidase alfa plus miglustat in adults with late-onset Pompe disease: a phase III open-label extension study (ATB200-07).
2024-02-2811 citations
Long-term safety and efficacy of cipaglucosidase alfa plus miglustat in individuals living with Pompe disease: an open-label phase I/II study (ATB200-02).
2023-12-0611 citations
Long-term Follow-up of Cipaglucosidase Alfa/Miglustat in Ambulatory Patients with Pompe Disease: An Open-label Phase I/II Study (ATB200-02) (S48.007)
Neurology2023-04-251 citations
First-in-human Study of ATB200/AT2221 in Patients with Pompe Disease: Preliminary Results From the ATB200-02 Trial (S22.003)
Neurology2018-04-10