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

Status

Recruiting

Phases

Phase 3

Study type

Interventional

Source

EU CTIS

Registry ID

CTIS2022-501095-25-01

Acronym

ATB200-08

Enrollment

Registered

2023-10-19

Start date

2024-11-04

Completion date

Unknown

Last updated

2026-01-26

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Pediatric Subjects with Infantile-onset Pompe Disease Aged 0 to < 18 Years

Brief summary

The primary endpoint is the proportion of subjects with infusion-associated reactions (IARs).

Detailed description

incidence of treatment-emergent adverse events (TEAEs), including treatment-emergent serious adverse events (TESAEs), hypersensitivity/anaphylactic reactions, and TEAEs leading to discontinuation of study drug, changes in clinical laboratory test results, changes in vital signs, changes in 12-lead ECG results, changes in echocardiogram parameters (other than LVMI)

Interventions

DRUGMiglustat Oral Solution 40 mg/mL

DRUGAT2221

DRUGATB200

Eligibility

Sex/Gender

All

Age

0 Years to 17 Years

Design outcomes

Primary

Measure	Time frame
The primary endpoint is the proportion of subjects with infusion-associated reactions (IARs).	—

Secondary

Measure	Time frame
incidence of treatment-emergent adverse events (TEAEs), including treatment-emergent serious adverse events (TESAEs), hypersensitivity/anaphylactic reactions, and TEAEs leading to discontinuation of study drug, changes in clinical laboratory test results, changes in vital signs, changes in 12-lead ECG results, changes in echocardiogram parameters (other than LVMI)	—

Countries

France, Germany, Italy, Netherlands

Outcome results

None listed

Source: EU CTIS · Data processed: Feb 4, 2026