An Open-label, Single-arm Extension Study to Evaluate the Long-term Safety, Tolerability, and Efficacy of Leniolisib for Immune Dysregulation in Primary Immunodeficiency Disorders
Pharming Technologies B.V.
Summary
This is an open-label extension (OLE) study to extend treatment to patients with primary immunodeficiency (PID) disorders linked to phosphoinositide 3-kinase delta signaling who participated in a prior study of leniolisib, LE 7201. The primary objective is to assess long-term safety and tolerability of leniolisib. Secondary and exploratory objectives include various efficacy and immunophenotyping measures for leniolisib.
Eligibility
- Age range
- 12–75 years
- Sex
- All
- Healthy volunteers
- No
Inclusion Criteria: 1. Subject must have participated in LE 7201. 2. Subject is deemed by the Investigator to benefit from continued leniolisib therapy. 3. Subject or their legal representatives (for a patient under the age of 18 years) must be able to communicate with the Investigator and understand and comply with the requirements of the study, including an ability to provide written informed consent before any assessment is performed. Exclusion Criteria: 1. Subject has had a successful allogeneic hematopoietic stem cell transplant. 2. Previous or concurrent use of immunosuppressive medic…