Multicentre, open label, prospective, single arm study of the safety and impact of eculizumab withdrawal in patients with atypical haemolytic uraemic syndrome - SETS aHUS: Stopping Eculizumab Treatment Safely in aHUS

ewcastle Upon Tyne Hospitals NHS Foundation Trust0 个研究点目标入组 50 人开始时间: 2018年2月19日最近更新: 2019年6月30日

适应症

试验速览

暂无简介。

•Age =2+ years of age
•On Eculizumab treatment for at least 6 months
•In remission with no evidence of ongoing microangiopathic haemolytic anaemia (MAHA) activity at screening defined by:
•-Platelet count > lower limit of normal as determined by local reference range
•-LDH •Normal renal function or CKD stages 1-3
•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 TMG
•Willing to attend for safety monitoring assessments
•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.
•Able to perform or parent/guardian to perform and record self-monitoring urinalysis
•Sexually active female patients who are of child bearing age must have a negative pregnancy test at screening and consent to use effective contraception for the duration of the study as listed in table 1 in the protocol
•OR fulfil one of the following criteria:
•-Be post-menopausal
•-Have undergone surgical sterilisation
•Are the trial subjects under 18? yes
•Number of subjects for this age range: 20
•F.1.2 Adults (18-64 years) yes
•F.1.2.1 Number of subjects for this age range 28
•F.1.3 Elderly (>=65 years) no
•F.1.3.1 Number of subjects for this age range 2

•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.
•Loss of a previous transplant kidney to recurrent aHUS
•Transplant recipient with a pathogenic mutation in C3, CFH or CFB
•Current or planned pregnancy
•Unable to give informed consent or assent, or unable to obtain parent/guardian consent if under 16 years of age
•Unable to comply with monitoring protocol
•Current participation in another clinical trial
•Haematuria rating of 3+
•Severe, uncontrolled hypertension (systolic blood pressure >160 mmHg) that is likely to induce at TMA.
•Current participation in another clinical trial (not including participation in ahus registries)

发起方

ewcastle Upon Tyne Hospitals NHS Foundation Trust