Extension to the Study of Efficacy of CDZ173 in Patients With APDS/PASLI

March 9, 2026 updated by: Pharming Technologies B.V.

An Open-label, Non-randomized Extension Study to Evaluate the Long Term Safety, Tolerability, Efficacy and Pharmacokinetics of CDZ173 in Patients With APDS/PASLI (Activated Phosphoinositide 3-kinase Delta Syndrome/p110δ-activating Mutation Causing Senescent T Cells, Lymphadenopathy and Immunodeficiency)

This study is designed to provide long-term CDZ173 treatment, a selective PI3Kδ inhibitor, to the patients with genetically activated PI3Kδ, i.e., patients with APDS/PASLI who participated in the CCDZ173X2201 study or who were treated previously with PI3Kδ inhibitors other than CDZ173. The study is open-label designed to establish the long-term safety, tolerability, efficay and pharmacokinetics of CDZ173 in the target population.

Study Overview

Status

Terminated

Conditions

Intervention / Treatment

Study Type

Interventional

Enrollment (Actual)

37

Phase

  • Phase 2
  • Phase 3

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • Belarus
      • Minsk, Belarus, 223053
        • Pharming Investigative Site
  • Czechia
    • CZE
      • Prague, CZE, Czechia, 15006
        • Pharming Investigative Site
  • Germany
      • Dresden, Germany, 01307
        • Pharming Investigative Site
  • Italy
    • BS
      • Brescia, BS, Italy, 25123
        • Pharming Investigative Site
    • PA
      • Palermo, PA, Italy, 90127
        • Pharming Investigative Site
  • Netherlands
      • Rotterdam, Netherlands, 3000 CA
        • Pharming Investigative Site
  • Russia
      • Moscow, Russia, 117198
        • Pharming Investigative Site
  • United States
    • Maryland
      • Bethesda, Maryland, United States, 20892
        • Pharming Investigative Site

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

12 years to 75 years (Child, Adult, Older Adult)

Accepts Healthy Volunteers

No

Description

Inclusion Criteria:

  • Written informed consent must be obtained before any assessment is performed.
  • Paients must have participated in the study CCDZ173X2201 or were treated previously with PI3Kδ inhibitors other than CDZ173.
  • Patients who are deemed by the Investigator to benefit from PI3Kδ 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.

Exclusion Criteria:

- Any medically significant disease or condition that is unrelated to APDS/PASLI

Other protocol-defined inclusion/exclusion criteria may apply.

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: N/A
  • Interventional Model: Single Group Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Experimental: CDZ173
140mg/day
Drug: CDZ173
140 mg/day

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
To Evaluate the Number of Participants With (S)AEs During Treatment With CDZ173
Time Frame: 6 years 3 months
Number of participants with adverse events reported, including serious adverse events
6 years 3 months

Secondary Outcome Measures

Outcome Measure
Measure Description
Time Frame
To Evaluate the Long Term Efficacy of CDZ173 Using SF-36 General Health Score
Time Frame: At baseline, after 1 year, after 3 years, after 4 years and after 5 years of study participation
SF-36 (Short Form 36) Survey general health score per participant reported for duration of particpation in study. Scores are reported on a scale of 0 - 100. All items are scored so that a high score defines a more favorable health state.
At baseline, after 1 year, after 3 years, after 4 years and after 5 years of study participation

Other Outcome Measures

Outcome Measure
Measure Description
Time Frame
Responder Analysis Soluble Protein Biomarkers - naïve B Cells
Time Frame: up to 252 days
Responder analysis soluble protein biomarkers - Change in naïve b cells from baseline up to Day 252
up to 252 days
Sum of Product of Diameters (SPD) of Index Lesions
Time Frame: Baseline and D252
Participants were scanned through MRI or CT imaging
Baseline and D252
Spleen Organ Evaluation - Volume in mm3
Time Frame: Baseline and D252
Participants were scanned through MRI or CT
Baseline and D252

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Pharming Technologies B.V.

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

General Publications

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

September 8, 2016

Primary Completion (Actual)

January 30, 2025

Study Completion (Actual)

January 30, 2025

Study Registration Dates

First Submitted

August 4, 2016

First Submitted That Met QC Criteria

August 4, 2016

First Posted (Estimated)

August 9, 2016

Study Record Updates

Last Update Posted (Actual)

March 30, 2026

Last Update Submitted That Met QC Criteria

March 9, 2026

Last Verified

March 1, 2026

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • CCDZ173X2201E1
  • 2016-000468-41 (EudraCT Number)

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

UNDECIDED

IPD Plan Description

Novartis is committed to sharing with qualified external researchers, access to patient-level data and supporting clinical documents from eligible studies. These requests are reviewed and approved by an independent review panel on the basis of scientific merit. All data provided is anonymized to respect the privacy of patients who have participated in the trial in line with applicable laws and regulations.

This trial data availability is according to the criteria and process described on www.clinicalstudydatarequest.com

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

Yes

Studies a U.S. FDA-regulated device product

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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