No registrations found.
ID
Source
Brief title
Health condition
Pompe Disease
Sponsors and support
Intervention
Outcome measures
Primary outcome
Infantile: Survival;
Late-onset: Improvement and/or stabilisation of muscle function.
Secondary outcome
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.
Background summary
Protocollised follow-up of cardio-pulmonary function and musculo-skeletal functio in Pompe patients receiving enzyme replacement therapy on a compessionate use basis.
Study objective
Enzyme therapy with recombinant human alpha glucosidase results in prolonged survival; improvement or stabilisation of cardiac hypertrophy and function, improvement or stabilisation of pulmonary function and improvement or stabilisation of muscle function and strength.
Intervention
Enzyme replacement therapy.
P.O. Box 2060
A. Ploeg, van der
Dr. Molewaterplein 60
Rotterdam 3000 CB
The Netherlands
+31 (0)10 4637044
a.vanderploeg@erasmusmc.nl
P.O. Box 2060
A. Ploeg, van der
Dr. Molewaterplein 60
Rotterdam 3000 CB
The Netherlands
+31 (0)10 4637044
a.vanderploeg@erasmusmc.nl
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
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
Recruitment
Followed up by the following (possibly more current) registration
No registrations found.
Other (possibly less up-to-date) registrations in this register
No registrations found.
In other registers
| Register | ID |
|---|---|
| NTR-new | NL334 |
| NTR-old | NTR372 |
| Other | : N/A |
| ISRCTN | ISRCTN72578000 |
Summary results
Enzyme therapy for pompe's disease with recombinant human alpha-glucosidase from rabbit milk (J. Inherit. Metab. Dis. 2001) <br>
Long term IV treatment of pompe's disease with recombinant human alpha-glucosidase from milk (pediatrics 2004)
Enzyme replacement therapy in late-onset Pompe's disease: a three year follow-up (Ann. Neurology 2004)<br>
Morphological changes in muscle tissue of patients with Infantile Pompe's disease receiving enzym replacement therapy (Muscle Nerv 2003).