OMS906 PNH Long-term Extension Study
Research type
Research Study
Full title
An Open-Label Study to Evaluate the Long-Term Safety, Tolerability and Efficacy of OMS906 in Patients with Paroxysmal Nocturnal Hemoglobinuria (PNH)
IRAS ID
1008856
Contact name
Catherine Melfi
Contact email
Sponsor organisation
Omeros Corporation
ISRCTN Number
ISRCTN73211658
Research summary
Paroxysmal nocturnal haemoglobinuria (PNH) is a rare blood disease that causes red blood cells to break apart, releasing the haemoglobin inside. This happens because the surface of a person’s blood cells is missing a protein that protects them from the body's immune system. The release of haemoglobin causes many of the PNH symptoms like dark urine, anaemia, tiredness, difficulty swallowing and stomach pain. If left untreated, PNH can lead to serious medical complications.
This is a study to determine whether participants who have received the investigational medicine called OMS906 in two previous PNH studies and responded well to it, continue to find it safe, tolerate it well and maintain a good response to it.
OMS906 is an antibody. Antibodies work by binding to specific proteins in the body so that the harmful effect of that protein (or pathogens such as viruses) are removed. OMS906 binds to proteins involved in a specific part of the immune system. As a result, it is hoped the red blood cells in PNH patients which are missing a surface protein will not be attacked by the immune system and not break apart, thereby improving PNH symptoms, or by stopping PNH symptoms from getting worse.
The study will include about 25 participants who are over 18 years old and have participated in one of two previous OMS906 studies; one for PNH patients who had received ravulizumab previously but with only a partial response to it, and one for PNH patients who had not received previous treatment. The study will include one evaluation visit and up to 14 treatment visits (at 8 weekly intervals) over a 2-year period.
The participants PNH symptoms will be closely monitored during the study and results from the study may be used to develop future treatments for PNH.
The study is organised and funded by Omeros Corporation a biopharmaceutical company developing PNH treatments and is being conducted at St. James's University Hospital in the UK and at 4 other centres in Europe.REC name
London - Chelsea Research Ethics Committee
REC reference
23/LO/0944
Date of REC Opinion
13 Dec 2023
REC opinion
Further Information Favourable Opinion