Leniolisib for immune dysregulation in CVID
Research type
Research Study
Full title
A study to assess safety and tolerability, and explore efficacy of leniolisib for immune dysregulation in common variable immunodeficiency
IRAS ID
1011384
Contact name
Miranda Boshuizen
Contact email
Sponsor organisation
Pharming Technologies B.V.
Clinicaltrials.gov Identifier
Research summary
The purpose of this study is to find out about the safety, tolerability and efficacy of leniolisib for the treatment of immune dysregulation in common variable immunodeficiency (CVID). Leniolisib is an experimental drug which is not approved for the treatment of CVID by health authorities. However, leniolisib is approved in the United States, Israel and United Kingdom for the treatment of a related disease, activated phosphoinositide 3-kinase delta syndrome (APDS) in patients 12 years of age and older.
CVID is a disease that can cause the immune system to not function properly. This can lead to an increased chance of infections, and/or problems of immune dysregulation like developing an enlarged spleen, enlarged lymph nodes, low blood cell numbers, and autoimmune and inflammatory problems of the lungs, gastrointestinal tract, or other organs. The Sponsor of this study, Pharming Technologies, B.V., is researching leniolisib as a potential new treatment for the immune dysregulation problems associated with CVID.
Approximately 20 patients, age 12-75 years with CVID, will take part in this study at several different locations in the UK, Spain and USA.
Patients participating in this study will be required to attend between 12 and 14 study visits over a period of up to 35 weeks. The visits will vary in duration depending on the procedures performed, with 4 of these visits conducted as telephone calls with associated local/at home blood draws.REC name
East Midlands - Leicester South Research Ethics Committee
REC reference
25/EM/0049
Date of REC Opinion
2 Apr 2025
REC opinion
Further Information Favourable Opinion