Protocollised follow-up of Pompe patients receiving enzyme replacement therapy on a compassionate use basis.

Expanded access use of recombinant human Acid Alpha-Glucosidase in patients with Infantile-onset/ Late-onset Pompe Disease.

Status

Recruiting

Phases

Unknown

Study type

Interventional

Source

NL-OMON

Registry ID

NL-OMON21598

Enrollment

Registered

2005-09-12

Start date

1999-01-01

Completion date

Unknown

Last updated

2024-02-28

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

Conditions

Pompe Disease

Interventions

Enzyme replacement therapy.

Eligibility

Inclusion criteria

Inclusion criteria: Confirmed diagnosis of Pompe Disease infantile-onset: age less than 1 year, delayed motor milestones and/or hypertrophic cardiomyopathy. late-onset 1.: 24 hour/day artificial ventilation, wheelchair bound or previously enrolled in AGLU 1202 study.

Exclusion criteria

Exclusion criteria: Infantile-onset: congenital abnormalities, allergy to food and/or proteins, ventilator dependency; Late-onset: developmental delays not explained by Pompe's Disease, allergies and severe co-morbidity.

Design outcomes

Primary

Measure	Time frame
Infantile: Survival; Late-onset: Improvement and/or stabilisation of muscle function.	—

Secondary

Measure	Time frame
Infantile: improvement of cardiac hypertrophy and function, achievement of motor milestones; Late-onset: improvement and/or stabilisation of pulmonary function, improvement of quality of life.	—

Contacts

Public ContactA. Ploeg, van der

Erasmus Medical Center, Sophia Children's Hospital, P.O. Box 2060

a.vanderploeg@erasmusmc.nl+31 (0)10 4637044

Outcome results

None listed

Source: NL-OMON (via WHO ICTRP)