Safety, Tolerability, Pharmacokinetic, and Pharmacodynamic Study of ALXN2230 in Healthy Participants
Research type
Research Study
Full title
A Phase 1, Randomized, Double-Blind, Placebo-Controlled, Single Ascending Dose Study to Assess Safety, Tolerability, Pharmacokinetics, Pharmacodynamics, and Immunogenicity of ALXN2230 in Healthy Adult Participants
IRAS ID
1013011
Contact name
Rajahbala Veerasingham
Contact email
Sponsor organisation
Alexion Pharmaceuticals, Inc.
Clinicaltrials.gov Identifier
Research summary
The trial medicine (ALXN2230) is an experimental treatment being developed to treat autoimmune diseases, such as Berger’s disease (caused by build-up of faulty antibodies in the kidney), or idiopathic inflammatory myopathies (autoimmune diseases which cause muscle weakness and inflammation). In these diseases, the body makes antibodies that wrongly target your own organs, causing damage and inflammation. These are called pathogenic antibodies.
ALXN2230 is a type of drug called a ‘biologic’ which works by blocking multiple pathways to stop the production of antibodies, including pathogenic antibodies, and reduce inflammation. Most other medicines can only block 1 pathway involved in making pathogenic antibodies, so they often don’t fully stop them, or treatments can take several months to be effective. We hope that by targeting multiple pathways, the pathogenic antibodies can be stopped more quickly and effectively.
We’re monitoring for side effects, and collecting blood and urine samples to measure levels of ALXN2230 and responses to the study medicine. We also want to find out the effect of ALXN2230 on proteins that are linked to both immune responses (such as antibodies) and check if the body makes any antibodies against ALXN2230.
If this and future studies are successful, the study medicine would be a useful treatment for patients in many countries.
We’ll give up to 48 healthy participants (including 1 group of 8 Chinese participants, so that we can compare the results with those from other ethnic groups), aged 18-65 years, a single dose of ALXN2230 or placebo as an injection or infusion under the skin. We’ll start with a low dose and increase it as the study progresses.
Participants will take about 16 weeks to finish the study. They’ll be hospitalised for 9 days and 8 nights in a row, followed by 9 outpatient visits.
A pharmaceutical company Alexion Pharmaceuticals, Inc. is funding the trial.
The trial will take place at 1 centre in London.REC name
London - Harrow Research Ethics Committee
REC reference
25/LO/0690
Date of REC Opinion
19 Feb 2026
REC opinion
Further Information Favourable Opinion