ALXN1210-PNH-201: ALXN1210 Phase 2 study in patients with PNH
Research type
Research Study
Full title
A Phase 2, Open-Label, Multiple Ascending Dose Study to Evaluate the Efficacy, Safety, Tolerability, Immunogenicity, Pharmacokinetics, and Pharmacodynamics of ALXN1210 Administered Intravenously to Patients with Paroxysmal Nocturnal Hemoglobinuria
IRAS ID
182410
Contact name
Anita Hill
Contact email
Sponsor organisation
Alexion Pharmaceuticals, Inc.
Eudract number
2015-002674-20
Duration of Study in the UK
3 years, 0 months, 0 days
Research summary
Summary of ResearchSummary o
The purpose of this study is to determine if an investigational product called ALXN1210, is safe and effective for the treatment of Paroxysmal Nocturnal Haemoglobinuria (PNH)a condition that causes destruction of red blood cells and release into the urine. A drug similar to ALXN1210, called eculizumab, is approved for the treatment of PNH in 46 countries under the brand name Soliris®.
ALXN1210 is being developed for the treatment of disorders involving
the complement system. The complement system is a part of the complex immune system (system that fights against infections). ALXN1210 works by suppressing the activity of a specific portion of the complement system; it is therefore classified as an immunosuppressant drug. PNH is an example of a complement disease that could be eventually treated with ALXN1210.It is expected that approximately 30 centres will be opened around the world in order to enrol 18 participants for evaluation.
This study is divided into three separate treatment groups, called cohorts. All treatment groups will receive the study drug. All treatment groups will receive different treatment doses. The study drug will be administered intravenously into a vein.
There will be 3 periods in this study: Screening Period, Study Period and Extension Period. The duration of participation in the study is approximately 2.8 years.Summary of Results
Plain language summary is still being drafted per EU CTR requirements and as per HRA website.
Plain language summary can be used for UK as well.
This is expected November 2022.REC name
North West - Greater Manchester Central Research Ethics Committee
REC reference
15/NW/0779
Date of REC Opinion
27 Oct 2015
REC opinion
Further Information Favourable Opinion