LE3302

• Research type

  Research Study

• Full title

  An Open-label, Single Arm Study of the Safety, Pharmacokinetics, Pharmacodynamics, and Efficacy of Leniolisib in Pediatric Patients (Aged 1 to 6 Years) with APDS (Activated Phosphoinositide 3-Kinase Delta Syndrome) Followed by an Open-label Long-term Extension

• IRAS ID

  1007759

• Contact name

  Leisa Waynick

• Contact email

  L.Waynick@pharming.com

• Sponsor organisation

  Pharming Technologies B.V.

• Clinicaltrials.gov Identifier

  NCT05693129

• Research summary

  The purpose of this study is to find out about the safety and efficacy of leniolisib for the treatment of APDS (Activated Phosphoinositide 3-Kinase Delta Syndrome).
  Activated phosphoinositide 3-kinase delta (PI3Kδ) syndrome (APDS) is an ultra-rare, genetic, life-threatening disorder of the immune system classified within a group of disorders called primary immune deficiencies (PIDs). APDS is caused by genetic variations in either of two genes known as PIK3CD or PIK3R1. Both of these genes are vital to the development and function of immune cells in the body.

  As with other PIDs, the majority of patients with APDS show symptoms and manifestations before the age of 18 years, although, there are some manifestations that are recognized when patients are adults. Although the age of clinical onset can vary widely, most patients present with symptoms early in childhood. Currently there are no approved therapies for APDS

  Leniolisib is an experimental drug that has shown some acceptability and effectiveness in young children with the target disease area which will be used as the study drug in this study.
  Approximately 15 children aged 1 to 6 years will take part in this study at several different locations internationally.

  This is a 2-part study. The study has a screening period (up to 7 weeks), a treatment period of Part I (up to 12 weeks), a treatment period of Part II (up to 52 weeks), and a follow-up period (up to 4 weeks).

  Study drug will be administered orally 2 times a day. Dosage will be determined according to the children’s body weight.

  Study procedures will include medical history, mutation testing, blood tests, MRI/CT scans, stool testing and questionnaires.

• REC name

  West Midlands - Edgbaston Research Ethics Committee

• REC reference

  23/WM/0154

• Date of REC Opinion

  12 Sep 2023

• REC opinion

  Further Information Favourable Opinion