An open-label, non-randomized extension study to evaluate the long term safety, tolerability, efficacy and pharmacokinetics of CDZ173 (leniolisib) in patients with APDS/PASLI (activated phosphoinositide 3-kinase delta syndrome/p110δ-activating mutation causing senescent T cells, lymphadenopathy and immunodeficiency)

Phase 2

Recruiting

• Conditions: Activated phosphoinositide 3-kinase delta syndrome/ p110δ -activating mutation causing senescent T cells; lymphadenopathy and immunodeficiency; 10021460

• Registration Number: NL-OMON54847
• Lead Sponsor: Pharming Group

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Last Update Posted: 23 April 2024

Recruitment & Eligibility

• Status: Recruiting
• Sex: Not specified
• Target Recruitment: 5

Inclusion Criteria

• Patients must have completed the study CCDZ173X2201 EOT/EOS visit, or were
treated previously with PI3Kδ inhibitors other than CDZ173.
• Patients who are deemed by the Investigator to benefit from PI3K inhibitor
therapy.
• Patients or their legal representatives (for patients under the age of 18
years) must be able to communicate well with the Investigator, to understand
and comply with the requirements of the study.
• Documented APDS/PASLI-associated genetic PI3K delta mutation.
Patients with mutations in either PIK3CD or PIK3R1 can be included.
Other protocol-defined inclusion criteria may apply

Exclusion Criteria

• Patients who withdrew consent from the study CCDZ173X2201
• Use of other investigational drugs, except CDZ173, within 5 half -lives of
enrollment, or within 30 days, whichever is longer.
• Concurrent use of immunosuppressive medication
• Administration of any live vaccines (including any attenuated live vaccines)
starting from 6 weeks before study entry, during the study and up to 7 days
after the last dose of CDZ173 should be excluded.
• Pregnant or nursing (lactating) women, where pregnancy is defined as the
state of a female after conception and until the termination of gestation.
• Women of child-bearing potential, defined as all women physiologically
capable of becoming pregnant, unless they are using highly effective methods of
contraception during dosing and for 2 days after last dose of study medication.
• Uncontrolled chronic or recurrent infectious disease (with the exception of
those that are considered to be characteristic of APDS/PASLI). , For patients
who did not participate in study CCDZ173X2201 but were treated previously with
PI3Kδ inhibitors other than CDZ173, the following additional exclusion criteria
apply:
• Vital signs (systolic and diastolic blood pressure and pulse rate) will be
assessed in the sitting position after the patient has rested for at least
three minutes.
• Patient must have a minimum body weight of 45 kg
• Evidence of tuberculosis infection as defined by a positive QuantiFERON TB
test (or comparable test) at screening. If presence of latent tuberculosis is
established then treatment according to local
country guidelines must have been completed before patients can be considered
for enrollment.
• Use of unstable dosing regimen with i.v. Ig / s.c. Ig in the last 6 months
before screening. Stable maintenance immunoglobulin regimen, as per local
practice, such as regular injections with a consistent dosing interval (e.g.,
monthly) is acceptable
• History of acquired immunodeficiency diseases, or a positive HIV (ELISA and
Western blot) test result at screening.
• A positive Hepatitis B surface antigen or Hepatitis C test (by PCR) result at
screening.
Other protocol-defined exclusion criteria may apply

Study & Design

• Study Type: Interventional
• Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
<p>To evaluate the long term safety and tolerability of CDZ173 in patients with<br /><br>APDS/PASLI </p><br>

Secondary Outcome Measures

Name	Time	Method
<p>- To evaluate the long term efficacy of CDZ173 to modify healthrelated quality<br /><br>of life in patients with APDS/PASLI.<br /><br>- To evaluate the long term efficacy of CDZ173 by means of biomarkers<br /><br>reflecting the efficacy of CDZ173 to reduce systemic inflammatory components of<br /><br>the disease in patients with APDS/PASLI.<br /><br>- To characterize the pharmacokinetics (trough concentrations) of CDZ173 in<br /><br>patients with APDS/PASLI.<br /><br>- To evaluate the pharmacokinetics and relative bioavailability of CDZ173<br /><br>film-coated tablets compared to CDZ173 hard-gelatin capsules</p><br>