ISRCTN45178534已完成未知
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)
Academic Medical Centre (AMC) (The Netherlands)0 个研究点目标入组 34 人开始时间: 2005年12月20日最近更新:
适应症
试验速览
- 阶段
- 未知
- 状态
- 已完成
- 发起方
- 入组人数
- 34
研究概览
简要总结
2007 results in: https://www.ncbi.nlm.nih.gov/pubmed/17622343 (added 04/07/2019)
研究设计
- 研究类型
- Interventional
入排标准
- 性别
- All
入选标准
- •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:
- •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)
- •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
排除标准
- •1. Patient is pregnant or lactating
- •2. Patient is unwilling to comply to the evaluation program
研究者
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