Safety and Pharmacokinetic Study of ALXN1720 in Healthy Adult Subjects
Research type
Research Study
Full title
A Phase 1, Randomized, Double-blind, Placebo-controlled, Single and Multiple Ascending Dose Study of Subcutaneous and Intravenous ALXN1720 With and Without rHuPH20 in Healthy Subjects
IRAS ID
263315
Contact name
Ulrike Lorch
Contact email
Sponsor organisation
Alexion Pharma GmbH
Eudract number
2018-004500-19
Duration of Study in the UK
0 years, 10 months, 29 days
Research summary
We are conducting a clinical trial for a new drug called ALXN1720, which has been developed for treatment of disorders of the immune system. One aspect of immune function is called the complement system, where the splitting of a protein called C5 into two proteins called C5a and C5b stimulates inflammation. In healthy individuals this is beneficial, but in people with certain diseases (complement-mediated disease) this process is abnormal.\nALXN1720 is a bispecific antibody (antibody binding two different targets). It works by binding C5 to prevent it splitting into C5a and C5b, therefore preventing inflammation harmful to people with complement-mediated disease, as well as binding to serum albumin (a protein found in the blood) which allows it to last longer in circulation. The C5-blocking function of ALXN1720 is similar to eculizumab, a monoclonal antibody licensed for treating complement-mediated disease by intravenous infusion. ALXN1720 has been designed to be given by subcutaneous infusion, to allow administration at home. \nALXN1720 is effective at stopping the splitting of C5 when tested in the laboratory and in animals. It has been well-tolerated, with the main safety concern being an increased infection risk, a side-effect shared with all C5-blocking medicines (see A6-2).\nThe purpose of this study is to test the safety of ALXN1720, measure how the body processes ALXN1720 and measure how effective ALXN1720 is at reducing free C5 levels.\nIn this study, we propose recruiting up to 72 healthy volunteers into nine cohorts (groups). In each cohort, volunteers are randomly assigned to receive ALXN1720 or placebo in a 6:2 ratio.\nCohorts 1-7 receive a single infusion under the skin (ALXN1720 SC) or intravenously (ALXN1720 IV). Cohorts 8 and 9 receive three doses, one week apart between each dose (ALXN1720 SC).\nIn cohort 7, hyaluronidase is co-administered (ALXN1720 SC/rHuPH20 SC), aiding ALXN1720’s absorption.
REC name
London - London Bridge Research Ethics Committee
REC reference
19/LO/0743
Date of REC Opinion
15 Jul 2019
REC opinion
Favourable Opinion