A Phase 1, Safety, Tolerability, PK & PD study of SC and IV ALXN1210
Research type
Research Study
Full title
A PHASE 1 STUDY TO EVALUATE THE SAFETY, TOLERABILITY, PHARMACOKINETICS, PHARMACODYNAMICS, AND IMMUNOGENICITY OF A SINGLE DOSE OF ALXN1210 ADMINISTERED SUBCUTANEOUSLY COMPARED TO INTRAVENOUSLY IN HEALTHY SUBJECTS
IRAS ID
209652
Contact name
Ulrike Lorch
Contact email
Sponsor organisation
Alexion Pharmaceuticals, Inc.
Eudract number
2016-001617-24
Duration of Study in the UK
0 years, 8 months, 30 days
Research summary
We are conducting a clinical study in healthy volunteers with an investigational drug known as ALXN1210. This is being developed by Alexion Pharmaceuticals, Inc. for the treatment of rare diseases related to the immune system such as paroxysmal nocturnal hemoglobinuria (PNH) and atypical hemolytic uremic syndrome (aHUS). These diseases result in the breakdown of red blood cells and platelets (hemolysis) which can be life-threatening. Eculizumab is the only approved drug for treatment of PNH and aHUS and is also manufactured by Alexion. ALXN1210 works in the same way as eculizumab but may be given less frequently and subcutaneously, which may provide a more convenient treatment for patients.\n\nALXN1210 has been given intravenously (iv) to healthy non-Japanese and Japanese volunteers as single and multiple ascending (increasing) doses (i.e. SAD and MAD) in previous and ongoing studies. No safety concerns have thus far been identified in these Phase 1 studies, which were randomized, blinded, and placebo controlled.\nThe aim of this study is to compare the safety, tolerability, pharmacokinetics (PK), pharmacodynamics (PD), and immunogenicity of a single dose of ALXN1210 administered subcutaneously compared to a single dose of ALXN1210 administered intravenously in healthy subjects.\n\nIt is anticipated that 42 volunteers will participate in the study, in three dose groups, each lasting up to 220 days, including a screening period of up to 70 days, and a treatment period of treatment period of 150 days. The first two dose groups will receive a single, subcutaneous dose of ALXN1210, the third will receive the same dose but intravenously for the purposes of pharmacokinetic comparison. \n\nWe will assess safety parameters including physical examination, vital sign measurements, laboratory evaluations, electrocardiogram (ECG) and monitoring of adverse events (AEs). Volunteers will be vaccinated against meningitis and will be administered antibiotics as explained in section A6.2.\n
REC name
South Central - Berkshire B Research Ethics Committee
REC reference
16/SC/0329
Date of REC Opinion
10 Aug 2016
REC opinion
Further Information Favourable Opinion