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…
Bron
Verkorte titel
Aandoening
Pompe Disease
Ondersteuning
Onderzoeksproduct en/of interventie
Uitkomstmaten
Primaire uitkomstmaten
Infantile: Survival;<br>
Late-onset: Improvement and/or stabilisation of muscle function.
Achtergrond van het onderzoek
Protocollised follow-up of cardio-pulmonary function and musculo-skeletal functio in Pompe patients receiving enzyme replacement therapy on a compessionate use basis.
Doel van het onderzoek
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.
Onderzoeksproduct en/of interventie
Enzyme replacement therapy.
Publiek
P.O. Box 2060
A. Ploeg, van der
Dr. Molewaterplein 60
Rotterdam 3000 CB
The Netherlands
+31 (0)10 4637044
a.vanderploeg@erasmusmc.nl
Wetenschappelijk
P.O. Box 2060
A. Ploeg, van der
Dr. Molewaterplein 60
Rotterdam 3000 CB
The Netherlands
+31 (0)10 4637044
a.vanderploeg@erasmusmc.nl
Belangrijkste voorwaarden om deel te mogen nemen (Inclusiecriteria)
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.
Belangrijkste redenen om niet deel te kunnen nemen (Exclusiecriteria)
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.
Opzet
Deelname
Opgevolgd door onderstaande (mogelijk meer actuele) registratie
Geen registraties gevonden.
Andere (mogelijk minder actuele) registraties in dit register
Geen registraties gevonden.
In overige registers
Register | ID |
---|---|
NTR-new | NL334 |
NTR-old | NTR372 |
Ander register | : N/A |
ISRCTN | ISRCTN72578000 |
Samenvatting resultaten
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).