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| [ Print-friendly version ] |
| Protocolised follow-up of Pompe patients receiving enzyme replacement therapy on a compassionate use basis |
| ISRCTN | ISRCTN72578000 |
| ClinicalTrials.gov identifier | |
| Public title | Protocolised follow-up of Pompe patients receiving enzyme replacement therapy on a compassionate use basis |
| Scientific title | |
| Acronym | N/A |
| Serial number at source | N/A |
| Study hypothesis |
Enzyme therapy with recombinant human alpha glucosidase results in:
1. Prolonged survival 2. Improvement or stabilisation of cardiac hypertrophy and function 3. Improvement or stabilisation of pulmonary function 4. Improvement or stabilisation of muscle function and strength |
| Ethics approval | Ethics approval received from the local medical ethics committee |
| Study design | Protocolised follow up of parallel group trial |
| Countries of recruitment | The Netherlands |
| Disease/condition/study domain | Pompe Disease |
| Participants - inclusion criteria |
1. Confirmed diagnosis of Pompe Disease
2. Infantile-onset: a. age less than one year b. delayed motor milestones, and/or c. hypertrophic cardiomyopathy 3. Late-onset: a. 24 hour/day artificial ventilation b. wheelchair bound c. previously enrolled in AGLU 1202 study |
| Participants - exclusion criteria |
1. Infantile-onset:
a. congenital abnormalities b. allergy to food and/or proteins c. ventilator dependency 2. Late-onset: a. developmental delays not explained by Pompe's Disease b. allergies c. severe co-morbidity |
| Anticipated start date | 01/01/1999 |
| Anticipated end date | 01/01/2050 |
| Status of trial | Ongoing |
| Patient information material | |
| Target number of participants | 12 |
| Interventions | Enzyme replacement therapy |
| Primary outcome measure(s) |
1. Infantile: Survival
2. Late-onset: Improvement and/or stabilisation of muscle function |
| Secondary outcome measure(s) |
1. Infantile:
a. improvement of cardiac hypertrophy and function b. achievement of motor milestones 2. Late-onset: a. improvement and/or stabilisation of pulmonary function b. improvement of quality of life |
| Sources of funding | Genzyme Corporation (The Netherlands) |
| Trial website | |
| Publications | |
| Contact name | Dr A van der Ploeg |
| Address |
Erasmus Medical Centre
Sophia Children's Hospital P.O. Box 2060 |
| City/town | Rotterdam |
| Zip/Postcode | 3000 CB |
| Country | Netherlands |
| Tel | +31 (0)10 463 7044 |
| a.vanderploeg@erasmusmc.nl | |
| Sponsor | Erasmus Medical Centre (The Netherlands) |
| Address |
Sophia Children's Hospital
Dr. Molewaterplein 60 |
| City/town | Rotterdam |
| Zip/Postcode | 3015 GJ |
| Country | Netherlands |
| Sponsor website: | http://www.erasmusmc.nl/ |
| Date applied | 23/02/2007 |
| Last edited | 23/02/2007 |
| Date ISRCTN assigned | 23/02/2007 |
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