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


- candidate number1581
- NTR NumberNTR372
- ISRCTNISRCTN72578000
- Date ISRCTN created23-feb-2007
- date ISRCTN requested28-okt-2005
- Date Registered NTR12-sep-2005
- Secondary IDsN/A 
- Public TitleProtocollised follow-up of Pompe patients receiving enzyme replacement therapy on a compassionate use basis.
- Scientific TitleExpanded access use of recombinant human Acid Alpha-Glucosidase in patients with Infantile-onset/ Late-onset Pompe Disease.
- ACRONYMN/A
- hypothesisEnzyme 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.
- Healt Condition(s) or Problem(s) studiedPompe disease
- Inclusion criteriaConfirmed 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 criteriaInfantile-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.
- mec approval receivedyes
- multicenter trialno
- randomisedno
- groupParallel
- Type-
- Studytypeintervention
- planned startdate 1-jan-1999
- planned closingdate1-jan-2050
- Target number of participants12
- InterventionsEnzyme replacement therapy.
- Primary outcomeInfantile: Survival;
Late-onset: Improvement and/or stabilisation of muscle function.
- Secondary outcomeInfantile: improvement of cardiac hypertrophy and function, achievement of motor milestones;
Late-onset: improvement and/or stabilisation of pulmonary function, improvement of quality of life.
- Timepoints
- Trial web siteN/A
- statusopen: patient inclusion
- CONTACT FOR PUBLIC QUERIESDr. A. Ploeg, van der
- CONTACT for SCIENTIFIC QUERIESDr. A. Ploeg, van der
- Sponsor/Initiator Erasmus Medical Center, Sophia Children's Hospital
- Funding
(Source(s) of Monetary or Material Support)
Genzyme Corporation
- PublicationsRecombinant human alpha-glucosidase from rabbit milk in Pompa patients (The Lancet 2000).
Enzyme therapy for pompe's disease with recombinant human alpha-glucosidase from rabbit milk (J. Inherit. Metab. Dis. 2001)
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)
Morphological changes in muscle tissue of patients with Infantile Pompe's disease receiving enzym replacement therapy (Muscle Nerv 2003).
- Brief summaryProtocollised follow-up of cardio-pulmonary function and musculo-skeletal functio in Pompe patients receiving enzyme replacement therapy on a compessionate use basis.
- Main changes (audit trail)
- RECORD12-sep-2005 - 23-feb-2007


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