Contact information
Type
Scientific
Primary contact
Dr A C Vedder
ORCID ID
Contact details
Academic Medical Centre
Department of Internal Medicine
F4-247
PO Box 22660
Amsterdam
1105 AZ
Netherlands
+31 (0)20 566 4558
a.c.vedder@amc.uva.nl
Additional identifiers
EudraCT number
ClinicalTrials.gov number
Protocol/serial number
NTR216
Study information
Scientific title
Acronym
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.
Ethics approval
Received from the local medical ethics committee
Study design
Multicentre, randomised, active controlled, factorial trial
Primary study design
Interventional
Secondary study design
Randomised controlled trial
Trial setting
Hospitals
Trial type
Treatment
Patient information sheet
Condition
Fabry disease
Intervention
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.
Intervention type
Drug
Phase
Not Specified
Drug names
Agalsidase beta (Fabrazyme®), agalsidase alpha (Replagal®)
Primary outcome measures
Wall-thickness (septum and left and right ventricle wall)/end-diastolic volume) on echocardiography.
Secondary outcome measures
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])
Overall trial start date
29/05/2001
Overall trial end date
31/12/2005
Reason abandoned
Eligibility
Participant 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
Participant type
Patient
Age group
Adult
Gender
Both
Target number of participants
At least 18 (9 in each group). 24 recruited as of Jan'06
Participant exclusion criteria
1. Patient is pregnant or lactating
2. Patient is unwilling to comply to the evaluation program
Recruitment start date
29/05/2001
Recruitment end date
31/12/2005
Locations
Countries of recruitment
Netherlands
Trial participating centre
Academic Medical Centre
Amsterdam
1105 AZ
Netherlands
Sponsor information
Organisation
Academic Medical Centre (AMC) (The Netherlands)
Sponsor details
Department of Internal Medicine
Meibergdreef 9
Amsterdam
1105 AZ
Netherlands
Sponsor type
Hospital/treatment centre
Website
Funders
Funder type
Government
Funder name
The Dutch Health Care Insurance Board (CVZ) (The Netherlands)
Alternative name(s)
Funding Body Type
Funding Body Subtype
Location
Results and Publications
Publication and dissemination plan
Not provided at time of registration
Intention to publish date
Participant level data
Not provided at time of registration
Results - basic reporting
Publication summary