SAFETY, TOLERABILITY, PD AND PK OF IV ALXN1210 IN HEALTHY SUBJECTS
Research type
Research Study
Full title
A PHASE 1, RANDOMIZED, BLINDED, PLACEBO-CONTROLLED, MULTIPLE ASCENDING DOSE STUDY TO EVALUATE THE SAFETY, TOLERABILITY, PHARMACOKINETICS, AND PHARMACODYNAMICS OF ALXN1210 ADMINISTERED INTRAVENOUSLY TO HEALTHY SUBJECTS
IRAS ID
173050
Contact name
Ulrike Lorch
Contact email
Sponsor organisation
Alexion Pharmaceuticals, Inc.
Eudract number
2014-005495-29
Duration of Study in the UK
1 years, 3 months, 14 days
Research summary
We are conducting a clinical study with an investigational drug known as ALXN1210 at Richmond Pharmacology Ltd clinical research unit. ALXN1210 is being developed by Alexion Pharmaceuticals, Inc. for the treatment of rare diseases related to the immune system. The complement system is an important part of the natural immune system which helps our bodies to fight diseases. Abnormalities in the complement system may cause rare diseases such as paroxysmal nocturnal hemoglobinuria (PNH) and atypical hemolytic uremic syndrome (aHUS) which is caused by a genetic mutation. These diseases result in the breakdown of red blood cells and platelets (hemolysis) which can be life-threatening. \n\nThere is currently one approved treatment for complement-mediated diseases known as eculizumab, which prevents hemolysis, and is currently used to treat patients with PNH and aHUS. Eculizumab is also manufactured by Alexion (the same sponsor as for this study). ALXN1210 works in the same way as eculizumab but may be given less frequently than eculizumab which may provide an alternative treatment for patients.\n\nALXN1210 has been given intravenously (iv) to healthy volunteers in single ascending doses (i.e. SAD) study; but this is the first time ALXN1210 will be given as multiple ascending doses (i.e. MAD). The main aim of this study is to establish the doses and dosing intervals to be used in future patient studies. It is anticipated that up to 40 healthy volunteers will participate in the study. Each volunteer will participate in the study for just over a year including a 10 week screening period, a 4-5 month treatment period and a 6-7 month follow up period.\n\nWe will assess safety parameters including tuberculosis testing, physical examination, vital sign measurements, immunogenicity testing, laboratory evaluations, electrocardiogram (ECG), monitoring of adverse events (AEs) and pregnancy tests. Volunteers will be vaccinated against meningitis as explained in section A6.2.
REC name
London - London Bridge Research Ethics Committee
REC reference
15/LO/0257
Date of REC Opinion
24 Apr 2015
REC opinion
Further Information Favourable Opinion