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PI3Kδ inhibitor

CDZ173 for Activated PI3Kdelta Syndrome

Phase 2 & 3
Waitlist Available
Research Sponsored by Novartis Pharmaceuticals
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial
Must have
Patients must have participated in the study CCDZ173X2201 or were treated previously with PI3Kδ inhibitors other than CDZ173
Documented APDS/PASLI-associated genetic PI3K delta mutation
Timeline
Screening 3 weeks
Treatment Varies
Follow Up 6 years
Awards & highlights

Summary

This trial tests CDZ173, a drug that blocks a protein to calm the immune system, in patients with a genetic condition causing an overactive immune system (APDS/PASLI).

Who is the study for?
This trial is for patients with APDS/PASLI, a genetic condition causing enlarged lymph nodes and immune system issues. Participants must have been in the CCDZ173X2201 study or treated with other PI3Kδ inhibitors, understand the study requirements, and provide informed consent.
What is being tested?
The trial tests long-term use of CDZ173, a drug targeting a specific part of cells (PI3Kδ) that's overactive in APDS/PASLI. It's an open-label study to assess safety, tolerability, effectiveness, and how the body processes the drug over time.
What are the potential side effects?
As CDZ173 targets immune cell function, potential side effects may include increased risk of infections, possible allergic reactions to the medication itself or its components. Specific side effect details are not provided but will be monitored throughout the trial.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below
Select...
I have been part of the CCDZ173X2201 study or treated with PI3Kδ inhibitors.
Select...
My condition is linked to a specific genetic mutation in PI3K delta.

Timeline

Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~6 years
This trial's timeline: 3 weeks for screening, Varies for treatment, and 6 years for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.
Primary study objectives
To evaluate the long term safety and tolerability of CDZ173 in patients with APDS/PASLI
Secondary study objectives
To characterize the pharmacokinetics (trough concentration) of CDZ173 in patients with APDS/PASLI
To evaluate the long term efficacy of CDZ173 by means of biomarkers reflecting the efficacy of CDZ173 to reduce systemic inflammatory components of the disease in patients with APDS/PASLI
To evaluate the long term efficacy of CDZ173 to modify health-related quality of life in patients with APDS/PASLI
+1 more

Side effects data

From 2021 Phase 2 & 3 trial • 37 Patients • NCT02435173
17%
Amylase increased
17%
Blood creatine phosphokinase increased
17%
Cough
17%
Lipase increased
100%
80%
60%
40%
20%
0%
Study treatment Arm
Part I: CDZ173 10 mg
Part I: CDZ173 30 mg
Part I: CDZ173 70 mg
Part II: Placebo
Part II: CDZ173 70 mg
Part I: Total

Trial Design

1Treatment groups
Experimental Treatment
Group I: CDZ173Experimental Treatment1 Intervention
140mg/day
Treatment
First Studied
Drug Approval Stage
How many patients have taken this drug
CDZ173
2015
Completed Phase 3
~70

Research Highlights

Information in this section is not a recommendation. We encourage patients to speak with their healthcare team when evaluating any treatment decision.
Mechanism Of Action
Side Effect Profile
Prior Approvals
Other Research
Selective PI3Kδ inhibitors, such as CDZ173, work by specifically targeting and inhibiting the delta isoform of the phosphoinositide 3-kinase (PI3K) enzyme. This inhibition is crucial for patients with Activated PI3Kdelta Syndrome (APDS) or PASLI Disease because the condition is characterized by hyperactivation of the PI3Kδ pathway due to genetic mutations. By blocking this pathway, PI3Kδ inhibitors help to reduce abnormal immune cell proliferation and function, thereby alleviating symptoms and preventing disease progression. This targeted approach is essential for managing APDS/PASLI effectively, as it directly addresses the underlying molecular dysfunction.
Dual Targeting by Inhibition of Phosphoinositide-3-Kinase and Mammalian Target of Rapamycin Attenuates the Neuroinflammatory Responses in Murine Hippocampal Cells and Seizures in C57BL/6 Mice.A systematic review of the safety and efficacy of currently used treatment modalities in the treatment of patients with PIK3CA-related overgrowth spectrum.SHORT syndrome due to a novel de novo mutation in PRKCE (Protein Kinase Cɛ) impairing TORC2-dependent AKT activation.

Find a Location

Who is running the clinical trial?

Novartis PharmaceuticalsLead Sponsor
2,893 Previous Clinical Trials
4,200,914 Total Patients Enrolled
Pharming Technologies B.V.Lead Sponsor
21 Previous Clinical Trials
1,363 Total Patients Enrolled

Media Library

CDZ173 (PI3Kδ inhibitor) Clinical Trial Eligibility Overview. Trial Name: NCT02859727 — Phase 2 & 3
Activated PI3Kdelta Syndrome or PASLI Disease Research Study Groups: CDZ173
Activated PI3Kdelta Syndrome or PASLI Disease Clinical Trial 2023: CDZ173 Highlights & Side Effects. Trial Name: NCT02859727 — Phase 2 & 3
CDZ173 (PI3Kδ inhibitor) 2023 Treatment Timeline for Medical Study. Trial Name: NCT02859727 — Phase 2 & 3
~8 spots leftby Jan 2027