Stopping Eculizumab Treatment Safely in atypical Haemolytic Uraemic Syndrome (SETS aHUS)

Phase 1

• Conditions: Atypical Haemolytic Uraemic Syndrome (aHUS); Therapeutic area: Diseases [C] - Immune System Diseases [C20]

• Registration Number: EUCTR2017-003916-37-GB
• Lead Sponsor: ewcastle Upon Tyne Hospitals NHS Foundation Trust

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Results First Posted: 1900-01-01
• Study Start: 2018-02-19
• Last Update Posted: 30 June 2019

Recruitment & Eligibility

• Status: Authorised-recruitment may be ongoing or finished
• Sex: Not specified
• Target Recruitment: 50

Inclusion Criteria

•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

Exclusion Criteria

•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)

Study & Design

• Study Type: Interventional clinical trial of medicinal product
• Study Design: Not specified