ALXN1210 IN PATIENTS WITH ATYPICAL HEMOLYTIC UREMIC SYNDROME (aHUS)
Research type
Research Study
Full title
SINGLE ARM, STUDY OF ALXN1210 IN COMPLEMENT INHIBITOR TREATMENT-NAÏVE ADULT AND ADOLESCENT PATIENTS WITH ATYPICAL HEMOLYTIC UREMIC SYNDROME (aHUS)
IRAS ID
213166
Contact name
David Kavanagh
Contact email
Sponsor organisation
Alexion Pharmaceuticals Inc
Eudract number
2016-002027-29
Clinicaltrials.gov Identifier
128367, IND number
Duration of Study in the UK
3 years, 2 months, 13 days
Research summary
The purpose of this study is to determine the ability of a new drug, ALXN1210, to treat the disease atypical Hemolytic Uremic Syndrome (aHUS) in adolescent and adult patients. aHUS is a rare, life-threatening, genetic disease that can damage vital organs such as the kidneys, heart and brain. A drug similar to ALXN1210, called eculizumab (brand name Soliris), is currently the standard of care for aHUS patients.
ALXN1210 is being developed for the treatment of disorders involving the complement system, a branch of the body’s immune system that fights against infection. In aHUS, the complement system is not controlled properly and this can result in damage to the blood vessels, known as thrombotic microangiopathy (TMA). In TMA, platelets (which are required for normal blood clotting) become overactive and form clots in blood vessels throughout the body. These clots can block blood flow, create inflammation, and travel to other organs, causing further damage.
ALXN1210 is classified as an immunosuppressant drug as it works by suppressing the activity of a specific portion of the complement system. aHUS is an example of a complement disease that could be eventually treated with ALXN1210. Patients with aHUS who have not had any previous treatment with a complement inhibitor are eligible.
The dose of ALXN1210 received by patients will be based on body weight. ALXN1210 will be administered intravenously into a vein and blood samples will be collected throughout the study for various analyses.
There are 3 periods in this study: Screening Period (up to 7 days), a 26-week Initial Evaluation Period and an Extension Period (up to 2 years). The duration of participation in the study is estimated to be approximately 2 and a half years. It is expected that approximately 200 centres will be opened around the world in order to enrol 55 participants.
REC name
East Midlands - Leicester South Research Ethics Committee
REC reference
16/EM/0436
Date of REC Opinion
24 Nov 2016
REC opinion
Further Information Favourable Opinion