EUCTR2016-000378-38-NL进行中（未招募）1 期
A Randomized, Double blind, Active Control Study of the Safety and Efficacy of PRX-102 compared to Agalsidase Beta on Renal Function in Patients with Fabry Disease Previously Treated With Agalsidase Beta
相关药物
试验速览
- 阶段
- 1 期
- 状态
- 进行中（未招募）
- 入组人数
- 78
研究概览
简要总结
暂无简介。
研究设计
- 研究类型
- Interventional clinical trial of medicinal product
入排标准
入选标准
- •Eligible subjects must fulfill the following inclusion criteria:
- •1. Symptomatic adult Fabry disease patients, age 18-60 years
- •Plasma and/or leucocyte alpha galactosidase activity (by activity assay) less than 30% mean normal levels and one or more of the described characteristic features of Fabry disease:
- •i. neuropathic pain,
- •ii. cornea verticillata,
- •iii. clustered angiokeratoma
- •3. Females:
- •a.historical genetic test results consistent with Fabry pathogenic mutation One or more of the described characteristic features of Fabry disease:
- •i. neuropathic pain,
- •ii. cornea verticillata,
- •iii. clustered angiokeratoma
- •b. or in the case of novel mutations a first degree male family member with Fabry disease with the same mutation, and one or more of the characteristic features of Fabry disease
- •I. neuropathic pain,
- •ii. cornea verticillata,
- •iii. clustered angiokeratoma
- •4. Screening eGFR by CKD-EPI equation 40 to 120 mL/min/1.73 m2
- •5. Linear negative slope of eGFR of = 2 mL/min/1.73 m2 based on at least 3 serum creatinine values over approximately 1 year (range of 9 to 18 months, including the value obtained at the screening visit)
- •6. Treatment with a dose of 1 mg/kg agalsidase beta per infusion every 2 weeks for at least one year and at least 80% of 13 (10.4) mg/kg total dose over the last 6 months
- •7. Female patients and male patients whose co-partners are of child-bearing potential agree to use a medically accepted method of contraception, not including the rhythm method.
- •Are the trial subjects under 18? no
- •Number of subjects for this age range:
- •F.1.2 Adults (18-64 years) yes
- •F.1.2.1 Number of subjects for this age range 78
- •F.1.3 Elderly (>=65 years) no
- •F.1.3.1 Number of subjects for this age range
排除标准
- •The presence of any of the following excludes a subject from study enrollment:
- •1. History of anaphylaxis or Type 1 hypersensitivity reaction to agalsidase beta
- •2. Known non-pathogenic Fabry mutations
- •3. History of renal dialysis or transplantation
- •4. History of acute kidney injury in the 12 months prior to screening, including specific kidney diseases (e.g., acute interstitial nephritis, acute glomerular and vasculitic renal diseases); non-specific conditions (e.g, ischemia, toxic injury); as well as extrarenal pathology (e.g., prerenal azotemia, and acute postrenal obstructive nephropathy)
- •5. Patient with a screening eGFR value between 91-120 mL/min/1.73m2, havingan historical eGFR value higher than 120ml/min/1.73m2 (during 9 to18 months before screening)
- •6. Angiotensin converting enzyme (ACE) inhibitor or angiotensin receptor blocker (ARB) therapy initiated or dose changed in the 4 weeks prior to screening
- •7. Urine protein to creatinine ratio (UPCR) > 0.5 g/g (0.5 mg/mg or 500 mg/g) and not treated with an ACE inhibitor or ARB
- •8. Cardiovascular event (myocardial infarction, unstable angina) in the 6 month period before randomization
- •9. Congestive heart failure NYHA Class IV
- •10. Cerebrovascular event (stroke, transient ischemic attack) in the 6 month period before randomization
- •11. Known history of hypersensitivity to Gadolinium contrast agent that is not managed by the use of premedication
- •12. Female subjects who are pregnant, planning to become pregnant during the study, or are breastfeeding
- •13. Presence of any medical, emotional, behavioral or psychological condition that, in the judgment of the Investigator and/or Medical Director, would interfere with the patient’s compliance with the requirements of the study
研究者
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