ISRCTN17503205进行中（未招募）2 期
Multicentre, open label, prospective, single arm study of the safety and impact of eculizumab withdrawal in patients with atypical haemolytic uraemic syndrome
适应症
试验速览
- 阶段
- 2 期
- 状态
- 进行中（未招募）
- 入组人数
- 39
研究概览
简要总结
2022 Protocol article in https://pubmed.ncbi.nlm.nih.gov/36123058/ (added 20/09/2022)
研究设计
- 研究类型
- Interventional
入排标准
- 性别
- All
入选标准
- •All patients must fulfil the following criteria in order to be eligible for the trial:
- •1. Age =2+ years of age
- •2. On Eculizumab treatment for at least 6 months
- •3. In remission with no evidence of ongoing microangiopathic haemolytic anaemia (MAHA) activity at screening defined by:
- •3.1. Platelet count > lower limit of normal as determined by local reference range
- •3.2. Lactate Dehydrogenase (LDH) 4. Normal renal function or Chronic Kidney Disease (CKD) stages 1-3
- •5. Absence of decline of renal function confirmed by review of available assessments of renal function for the preceding 6 months by the Chief Investigator and clinical members of the Trial Management Group (TMG)
- •6. Willing to attend for safety monitoring assessments
- •7. Willing to travel only to countries that can supply Eculizumab (to be confirmed with co-ordinating centre prior to travel).
- •The following criteria must be met by those only wishing to be enrolled in the withdrawal component of the trial:
- •8. Able to perform or parent/guardian to perform and record self-monitoring urinalysis
- •9. Sexually active female patients must have a negative pregnancy test at screening and be using an effective contraception for the duration of the study (implant, injectable [combined hormone], intrauterine device, intrauterine system, male sterilisation,
- •injectable (single hormone), combined oral, progesterone only)
- •10. Fulfil one of the following criteria:
- •10.1. Be postmenopausal
- •10.2. Have undergone surgical sterilisation
排除标准
- •1. Severe non-renal disease manifestations at initial presentation with aHUS, which in the opinion of the Chief Investigator and/or the clinical members of the TMG makes the risk of treatment withdrawal unacceptable
- •2. Loss of a previous transplant kidney to recurrent aHUS
- •3. Transplant recipient with a pathogenic mutation in C3, CFH or CFB
- •4. Current or planned pregnancy
- •5. Unable to give informed consent or assent, or unable to obtain parent/guardian consent if under 16 years of age
- •6. Unable to comply with monitoring protocol
- •7. Current participation in another clinical trial
- •8. Haematuria rating of 3+
- •9. Severe, uncontrolled hypertension (systolic blood pressure > 160 mmHg) that is likely to induce at TMA
研究者
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