Leniolisib OLE for Immune Dysregulation in PID

• Research type

  Research Study

• Full title

  An open-label, single-arm extension study to evaluate the long-term safety, tolerability, and efficacy of leniolisib for immune dysregulation in patients with primary immunodeficiency

• IRAS ID

  1012828

• Contact name

  Miranda Boshuizen

• Contact email

  m.boshuizen@pharming.com

• Sponsor organisation

  Pharming Technologies B.V.

• Eudract number

  2025-522444-40

• Clinicaltrials.gov Identifier

  NCT06990529

• Research summary

  This is an open-label extension (OLE) study to extend treatment to primary immunodeficiency (PID) patients with immune dysregulation who participated in prior clinical study of leniolisib, LE 7201 and LE 8201. The purpose of this study is to find out about the safety and tolerability of leniolisib when it is taken for a long time. 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, Australia and the 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.The number of subjects in this OLE study will be based on the rollover of subjects previously participating in LE 7201 and LE 8201 studies, which is approximately 12 and 20 subjects, respectively. The study will take place at several different locations in the UK, Spain and USA. Patients participating in this study will be required to attend between 7 and 10 study visits over a period of approximately 3 years. The visits will vary in duration depending on the procedures performed.

• REC name

  West Midlands - Edgbaston Research Ethics Committee

• REC reference

  26/WM/0016

• Date of REC Opinion

  18 Feb 2026

• REC opinion

  Further Information Favourable Opinion