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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

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Trial Profile

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

Status: Active, no longer recruiting
Phase of Trial: Phase III

Latest Information Update: 03 Dec 2025

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At a glance

  • Drugs Leniolisib (Primary)
  • Indications Immunodeficiency disorders
  • Focus Adverse reactions; Registrational; Therapeutic Use
  • Sponsors Pharming Group NV

Most Recent Events

  • 06 Nov 2025 According to Pharming Group NV media release, company expect to report results from this study in the coming months and if the data are supportive we plan to seek regulatory approval for this younger pediatric population.
  • 28 Oct 2025 Planned End Date changed from 28 Feb 2026 to 28 Oct 2026.
  • 28 Oct 2025 Planned primary completion date changed from 30 Sep 2025 to 9 Jul 2026.

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