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| Treatment of Fabry patients greater than 18 years with enzyme supplementation therapy: comparison of efficacy and toxicity of low dose (0.2 mg/kg) Fabrazyme® (agalsidase beta) or Replagal® (agalsidase alfa) |
| ISRCTN | ISRCTN45178534 |
| ClinicalTrials.gov identifier | |
| Public title | Treatment of Fabry patients greater than 18 years with enzyme supplementation therapy: comparison of efficacy and toxicity of low dose (0.2 mg/kg) Fabrazyme® (agalsidase beta) or Replagal® (agalsidase alfa) |
| Scientific title | |
| Acronym | N/A |
| Serial number at source | NTR216 |
| Study hypothesis | Evaluation of efficacy and safety of two different formulas of alfa-Galactosidase A, agalsidase beta (Fabrazyme®) and agalsidase alpha (Replagal®) in an equal dose of 0.2 mg/kg in order to detect any differences between these two drugs. |
| Lay summary | |
| Ethics approval | Received from the local medical ethics committee |
| Study design | Multicentre, randomised, active controlled, factorial trial |
| Countries of recruitment | Netherlands |
| Disease/condition/study domain | Fabry disease |
| Participants - inclusion criteria |
1. The patient must have given written informed consent
2. Patients must be 18 years or older 3. Patient must have a current diagnosis of Fabry disease 4. Patients must have a decreased alpha-Gal activity or proven alfa-Gal A mutation 5. Female patients must have a negative pregnancy test, and must use a medically accepted method of contraception 6. Patients must be willing to comply to the evaluation program 7. Patients must have a clinical presentation consistent with either typical or atypical Fabry disease Patients must have at least one major or two minor objective criteria: Major: 1. Severe acroparesthesias, that cannot satisfactorily be controlled with Carbamazepine 2. Decreased glomerular filtration rate (GFR) less than 80 ml/min 3. Proteinuria greater than 300 mg/ml 4. Documented cerebrovascular accident (CVA) 5. Cardiac infarction 6. Hypertrophic non-obstructive cardiomyopathy resulting in decreased exercise tolerance 7. Rhythm disturbances necessitating a pacemaker 8. Multiple lacunar infarctions on magnetic resonance imaging (MRI) Minor: 1. Documented transient ischaemic attack (TIA) 2. Cardiac hypertrophy on echo or MRI 3. Atrial fibrillation 4. Intraventricular conduction abnormality 5. Sensoric hearing loss as shown on a hearing test 6. Severe vertigo 7. Micro-albuminuria greater than 50 mg/L 8. Mild to moderate acroparesthesias 9. Gastro-intestinal complaints that can not be explained by other medical conditions than Fabry disease |
| Participants - exclusion criteria |
1. Patient is pregnant or lactating
2. Patient is unwilling to comply to the evaluation program |
| Anticipated start date | 29/05/2001 |
| Anticipated end date | 31/12/2005 |
| Status of trial | Completed |
| Patient information material | |
| Target number of participants | At least 18 (9 in each group). 24 recruited as of Jan'06 |
| Interventions | Patients will receive 0.2 mg/kg Fabrazyme® (agalsidase beta) or 0.2 mg/kg Replagal®(agalsidase alpha), every two weeks for a minumum of 12 months. If there is treatment failure (progression of renal disease, cardiac disease and/or a new cerebral stroke or TIA) during or after this period, patients will be advised to switch to Fabrazyme 1.0 mg/kg/2 weeks. |
| Primary outcome measure(s) | Wall-thickness (septum and left and right ventricle wall)/end-diastolic volume) on echocardiography. |
| Secondary outcome measure(s) |
1. Improvement of renal function as measured by GFR
2. Reduction of glycolipid accumulation in skin tissue (LM and biochemistry) 3. Reduction in pain as measured by the BPI 4. Reduction in glycosphingolipid in plasma and 24-hr urine 5. Quality of life scores (36-item Short Form Health Survey [SF-36]) |
| Sources of funding | The Dutch Health Care Insurance Board (CVZ) (The Netherlands) |
| Trial website | |
| Publications | |
| Contact name | Dr A C Vedder |
| Address |
Academic Medical Centre
Department of Internal Medicine, F4-247 PO Box 22660 |
| City/town | Amsterdam |
| Zip/Postcode | 1105 AZ |
| Country | Netherlands |
| Tel | +31 (0)20 566 4558 |
| Fax | +31 (0)20 691 9743 |
| a.c.vedder@amc.uva.nl | |
| Sponsor | Academic Medical Centre (AMC) (The Netherlands) |
| Address |
Department of Internal Medicine
Meibergdreef 9 |
| City/town | Amsterdam |
| Zip/Postcode | 1105 AZ |
| Country | Netherlands |
| Sponsor website: | http://www.amc.uva.nl |
| Date applied | 20/12/2005 |
| Last edited | 18/11/2008 |
| Date ISRCTN assigned | 20/12/2005 |
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