Cipaglucosidase alfa

BIOLOGICAL4 trials

Sponsors

Amicus Therapeutics

Conditions

Glycogen Storage Disease Type II Infantile OnsetPompe DiseasePompe Disease (Late-onset)

Phase 3

A Study Comparing ATB200/AT2221 With Alglucosidase Alfa/Placebo in Adult Subjects With Late-onset Pompe Disease

CompletedNCT03729362

Amicus TherapeuticsPompe Disease (Late-onset)

Start: 2018-12-04End: 2021-01-15Updated: 2025-09-11

ZIP Study-OL Study of Safety, PK, Efficacy, PD, Immunogenicity of ATB200/AT2221 in Pediatrics Aged 0 to < 18 y.o. w/LOPD

Active, not recruitingNCT03911505

Amicus TherapeuticsPompe Disease (Late-onset)

Start: 2020-02-13End: 2026-06-30Updated: 2025-10-27

A Study to Evaluate the Safety, Efficacy, PK, PD and Immunogenicity of Cipaglucosidase Alfa/Miglustat in IOPD Subjects Aged 0 to <18

RecruitingNCT04808505

Amicus TherapeuticsGlycogen Storage Disease Type II Infantile Onset

Start: 2023-07-18End: 2027-07-01Target: 36Updated: 2026-01-22

Unknown Phase

A Global Prospective Observational Registry of Patients With Pompe Disease

RecruitingNCT06121011

Amicus TherapeuticsPompe Disease

Start: 2024-02-16End: 2034-12-20Target: 500Updated: 2026-03-10

Related Papers

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

Switching Enzyme Replacement Therapy for Late‐Onset Pompe Disease From Alglucosidase Alfa to Cipaglucosidase Alfa Plus Miglustat: Post Hoc Effect Size Analysis of <scp>PROPEL</scp>

Muscle & Nerve2025-05-072 citations

Challenges in multinational rare disease clinical studies during COVID-19: regulatory assessment of cipaglucosidase alfa plus miglustat in adults with late-onset Pompe disease

Journal of Neurology2025-01-012 citations

Switching treatment to cipaglucosidase alfa plus miglustat positively affects patient-reported outcome measures in patients with late-onset Pompe disease.

2024-11-134 citations

Health-Related Quality-of-Life Utility Values in Adults With Late-Onset Pompe Disease: Analyses of EQ-5D Data From the PROPEL Clinical Trial

Journal of health economics and outcomes research2024-09-181 citations

Health-Related Quality-of-Life Utility Values in Adults With Late-Onset Pompe Disease: Analyses of EQ-5D Data From the PROPEL Clinical Trial

Journal of health economics and outcomes research2024-09-18

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

Validation of the Patient-Reported Outcomes Measurement Information System (PROMIS<sup>®</sup>) physical function questionnaire in late-onset Pompe disease using PROPEL phase 3 data.

2024-01-314 citations

Quality of Life with Late-Onset Pompe Disease: Qualitative Interviews and General Public Utility Estimation in the United Kingdom

Journal of health economics and outcomes research2023-03-037 citations

Quality of Life with Late-Onset Pompe Disease: Qualitative Interviews and General Public Utility Estimation in the United Kingdom

Journal of health economics and outcomes research2023-01-017 citations

Safety and efficacy of cipaglucosidase alfa plus miglustat versus alglucosidase alfa plus placebo in late-onset Pompe disease (PROPEL): an international, randomised, double-blind, parallel-group, phase 3 trial.

2021-12-0185 citations