Effects and health economic aspects of enzyme therapy in children and adults with Pompe disease; Long-term follow-up of patients receiving commercially available Myozyme

Status

Not yet recruiting

Phases

Phase 4

Study type

Interventional

Source

EU CTIS

Registry ID

CTIS2024-518269-92-00

Acronym

Pompe phys 02

Enrollment

156

Registered

2024-12-16

Start date

Unknown

Completion date

Unknown

Last updated

2024-12-16

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

Conditions

Pompe disease / glycogenosis type II / acid maltase deficiency

Brief summary

Survival, Muscle strength and function, Motor and mental outcome, Pulmonary function, Cardiac hypertrophy and function, Hearing loss, Disease specific symptoms, handicap, quality of life and fatigue, Muscle mass and regeneration, Costs

Interventions

DRUGMyozyme 50 mg powder for concentrate for solution for infusion

Eligibility

Sex/Gender

All

Age

0 Years to No maximum

Design outcomes

Primary

Measure	Time frame
Survival, Muscle strength and function, Motor and mental outcome, Pulmonary function, Cardiac hypertrophy and function, Hearing loss, Disease specific symptoms, handicap, quality of life and fatigue, Muscle mass and regeneration, Costs	—

Countries

Netherlands

Outcome results

None listed

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