Condition category
Nutritional, Metabolic, Endocrine
Date applied
20/12/2005
Date assigned
20/12/2005
Last edited
18/11/2008
Prospective/Retrospective
Retrospectively registered
Overall trial status
Completed
Recruitment status
No longer recruiting

Plain English Summary

Not provided at time of registration

Trial website

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

http://www.amc.uva.nl

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

Publication citations

Additional files

Editorial Notes