MyOpportunITy 3
Research type
Research Study
Full title
An Open-Label Extension Study to Investigate the Long-Term Safety, Tolerability, and Efficacy of Rozanolixizumab in Study Participants With Persistent or Chronic Primary Immune Thrombocytopenia (ITP)
IRAS ID
1003022
Contact name
Nichola Cooper
Contact email
Sponsor organisation
UCB Biopharma SRL
Eudract number
2019-000883-40
Research summary
Immune thrombocytopenia (ITP) is a clinical disorder which causes a decrease number
of platelets (a type of cell that helps blood to clot) in the blood. People who have ITP
tend to bleed often, develop rashes of purple spots (purpura) on the skin due to blood
accumulation under the skin (or develop small red/purple spots (petechiae) caused by
bleeding from small blood vessels into the skin.
Most often, ITP is caused when people develop autoantibodies against proteins on the
surface of the platelet (referred to as antigens). Autoantibody formation mean that their
bodies produce a certain protein normally involved in the immune response against
foreign particles such as bacteria and viruses, but in ITP this protein is targeted towards
the body’s own cells, in this case, platelets. The antibodies against platelets are called
pathogenic IgG autoantibodies. The presence of these autoantibody reduces the number
of platelets in the blood and can lead to ITP.
Treatments aimed at reducing the quantity of these autoantibodies, are being used to
treat autoimmune diseases including ITP. The primary goal for treatment of ITP is to
achieve a platelet count that prevents major bleeding.
Rozanolixizumab is being developed to reduce the amount of pathogenic IgG
autoantibodies and is targeted to a specific step in the pathway that recycles and
maintains pathogenic IgG autoantibodies. Rozanolixizumab was designed to block this
step, leading to less pathogenic IgG autoantibodies resulting in increased platelet
numbers.
This study is designed to assess whether repeat treatment over a period of 52 weeks with
rozanolixizumab subcutaneous infusion is safe and well tolerated and will result in
platelet increases as a measure of efficacy.REC name
South West - Central Bristol Research Ethics Committee
REC reference
20/SW/0139
Date of REC Opinion
29 Oct 2020
REC opinion
Further Information Favourable Opinion