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)

Not Applicable

Completed

• Conditions: Fabry disease; Nutritional, Metabolic, Endocrine

• Registration Number: ISRCTN45178534
• Lead Sponsor: Academic Medical Centre (AMC) (The Netherlands)

Brief Summary

2007 results in: https://www.ncbi.nlm.nih.gov/pubmed/17622343 (added 04/07/2019)

Detailed Description

Not available

Study Timeline

• Results First Posted: 1900-01-01
• Study Start: 2005-12-20
• Study Completion: 2005-12-31
• Last Update Posted: 29 July 2019

Recruitment & Eligibility

• Status: Completed
• Sex: All
• Target Recruitment: 34

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

Exclusion Criteria

1. Patient is pregnant or lactating
2. Patient is unwilling to comply to the evaluation program

Study & Design

• Study Type: Interventional
• Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
Wall-thickness (septum and left and right ventricle wall)/end-diastolic volume) on echocardiography.

Secondary Outcome Measures

Name	Time	Method
<br> 1. Improvement of renal function as measured by GFR<br> 2. Reduction of glycolipid accumulation in skin tissue (LM and biochemistry)<br> 3. Reduction in pain as measured by the BPI<br> 4. Reduction in glycosphingolipid in plasma and 24-hr urine<br> 5. Quality of life scores (36-item Short Form Health Survey [SF-36])<br>