Study of Efficacy of CDZ173 in Patients With APDS/PASLI

  • STATUS
    Recruiting
  • End date
    Jun 8, 2021
  • participants needed
    36
  • sponsor
    Novartis Pharmaceuticals
Updated on 3 March 2021
Investigator
Novartis Pharmaceuticals
Primary Contact
National Institute of Health NIH (9.6 mi away) Contact
+9 other location

Summary

This study is designed to explore CDZ173, a selective PI3K inhibitor, in patients with genetically activated PI3K, i.e., patients with APDS/PASLI. The study consists of two parts. Part I is the open label part designed to establish the safety and pharmacokinetics of CDZ173 in the target population, as well as to select the optimal dose to be tested in part II. Part II is designed to assess efficacy and safety of CDZ173 in this population.

Description

This study is designed to explore CDZ173, a selective PI3K inhibitor, in patients with genetically activated PI3K, i.e., patients with APDS/PASLI (Activated phosphoinositide 3-kinase delta syndrome/ p110-activating mutation causing senescent T cells, lymphadenopathy and immunodeficiency)I.

The study consists of two parts. Part I is the open label part designed to establish the safety and pharmacokinetics of CDZ173 in the target population, as well as to select the optimal dose to be tested in part II.

Part II is designed to assess efficacy and safety of CDZ173 in this population.

Details
Condition Common Variable Immunodeficiency (CVID) More Specifically Activated PI3Kdelta Syndrome APDS p110delta-activating Mutation Causing Senescent T Cells, Lymphadenopathy and Immunodeficiency PASLI, Common Variable Immunodeficiency (CVID) More Specifically Activated PI3Kdelta Syndrome APDS p110delta-activating Mutation Causing Senescent T Cells, Common Variable Immunodeficiency (CVID) More Specifically Activated PI3Kdelta Syndrome APDS p110delta-activating Mutation Causing Senescent T Cells, Common Variable Immunodeficiency (CVID) More Specifically Activated PI3Kdelta Syndrome APDS p110delta-activating Mutation Causing Senescent T Cells, Common Variable Immunodeficiency (CVID) More Specifically Activated PI3Kdelta Syndrome (APDS) p110delta-activating Mutation Causing Senescent T Cells, Lymphadenopathy and Immunodeficiency (PASLI), Common Variable Immunodeficiency (CVID) More Specifically Activated PI3Kdelta Syndrome (APDS) p110delta-activating Mutation Causing Senescent T Cells, Lymphadenopathy and Immunodeficiency (PASLI), Common Variable Immunodeficiency (CVID) More Specifically Activated PI3Kdelta Syndrome (APDS) p110delta-activating Mutation Causing Senescent T Cells, Lymphadenopathy and Immunodeficiency (PASLI), Common Variable Immunodeficiency (CVID) More Specifically Activated PI3Kdelta Syndrome (APDS) p110delta-activating Mutation Causing Senescent T Cells, Lymphadenopathy and Immunodeficiency (PASLI), Common Variable Immunodeficiency (CVID) More Specifically Activated PI3Kdelta Syndrome (APDS) p110delta-activating Mutation Causing Senescent T Cells, Lymphadenopathy and Immunodeficiency (PASLI), Common Variable Immunodeficiency (CVID) More Specifically Activated PI3Kdelta Syndrome (APDS) p110delta-activating Mutation Causing Senescent T Cells, Lymphadenopathy and Immunodeficiency (PASLI), Common Variable Immunodeficiency (CVID) More Specifically Activated PI3Kdelta Syndrome (APDS) p110delta-activating Mutation Causing Senescent T Cells, Lymphadenopathy and Immunodeficiency (PASLI), Common Variable Immunodeficiency (CVID) More Specifically Activated PI3Kdelta Syndrome (APDS) p110delta-activating Mutation Causing Senescent T Cells, Lymphadenopathy and Immunodeficiency (PASLI), Common Variable Immunodeficiency (CVID) More Specifically Activated PI3Kdelta Syndrome (APDS) p110delta-activating Mutation Causing Senescent T Cells, Lymphadenopathy and Immunodeficiency (PASLI), Common Variable Immunodeficiency (CVID) More Specifically Activated PI3Kdelta Syndrome (APDS) p110delta-activating Mutation Causing Senescent T Cells, Lymphadenopathy and Immunodeficiency (PASLI)
Treatment CDZ173
Clinical Study IdentifierNCT02435173
SponsorNovartis Pharmaceuticals
Last Modified on3 March 2021

Eligibility

Yes No Not Sure

Inclusion Criteria

Is your age between 12 yrs and 75 yrs?
Gender: Male or Female
Do you have any of these conditions: Common Variable Immunodeficiency (CVID) More Specifically Activated PI3Kdelta Syndrome (APDS) p110delta-activating Mutation Causing Senescent T Cells ...?
Do you have any of these conditions: Lymphadenopathy and Immunodeficiency PASLI or Lymphadenopathy and Immunodeficiency (PASLI) or Common Variable Immunodeficiency (CVID) More Specificall...?
patients who have a documented APDS/PASLI-associated genetic PI3K delta mutation
In Part I and Part II, patients must have nodal and/or extranodal lymphoproliferation, and clinical findings and manifestations compatible with APDS/PASLI such as a history of repeated oto-sino-pulmonary infections and/or organ dysfunction (e.g., lung, liver). Additionally, in part II, patients must have at least one measurable nodal lesion on a CT or MRI scan

Exclusion Criteria

Any medically significant disease or condition that is unrelated to APDS/PASLI
Other protocol-defined inclusion/exclusion criteria may apply
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