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An Open-label, Non-randomized, Within-patient Dose-finding Study Followed by a Randomized, Subject, Investigator and Sponsor Blinded Placebo Controlled Study to Assess the Efficacy and Safety of CDZ173 (Leniolisib) in Patients With APDS/PASLI (Activated Phosphoinositide 3-kinase Delta Syndrome/ p110δ-activating Mutation Causing Senescent T Cells, Lymphadenopathy and Immunodeficiency)

Trial Profile

An Open-label, Non-randomized, Within-patient Dose-finding Study Followed by a Randomized, Subject, Investigator and Sponsor Blinded Placebo Controlled Study to Assess the Efficacy and Safety of CDZ173 (Leniolisib) in Patients With APDS/PASLI (Activated Phosphoinositide 3-kinase Delta Syndrome/ p110δ-activating Mutation Causing Senescent T Cells, Lymphadenopathy and Immunodeficiency)

Status: Completed
Phase of Trial: Phase II/III

Latest Information Update: 13 Mar 2024

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At a glance

  • Drugs Leniolisib (Primary)
  • Indications Common variable immunodeficiency; Immunodeficiency disorders; Lymphadenopathy
  • Focus Adverse reactions; Pharmacodynamics; Pharmacokinetics; Registrational; Therapeutic Use
  • Sponsors Novartis; Novartis Pharmaceuticals
  • Most Recent Events

    • 03 Oct 2023 Interim results of an open-label extension study (between September 2016 and August 2021) assessing safety of Leniolisib for patients with Activated phosphoinositide 3-kinase delta syndrome, published in the Journal of Allergy and Clinical Immunology.
    • 24 Mar 2023 According to Pharming Healthcare Media Release, the US FDA has approved Joenja (leniolisib) for the treatment of activated phosphoinositide 3-kinase delta syndrome (APDS) in adult and pediatric patients 12 years of age and older, based on findings from this trial, which evaluated efficacy and safety in 31 patients diagnosed with APDS aged 12 years and older and also submitted as part of the application were data from a long-term, open-label extension part of trial.
    • 16 Mar 2023 According to Pharming Healthcare Media Release, European marketing authorisation is expected approximately two months later after the CHMP which is expected in the 2H 2023.
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