CDZ173 for Activated PI3Kdelta Syndrome

No longer recruiting at 26 trial locations

Overseen By Novartis Pharmaceuticals

Age: Any Age

Sex: Any

Trial Phase: Phase 2 & 3

Sponsor: Novartis Pharmaceuticals

Must be taking: PI3Kδ inhibitors

Disqualifiers: Medically significant unrelated disease, others

No Placebo Group All trial participants will receive the active study treatment (no placebo)

Prior Safety Data This treatment has passed at least one previous human trial

Compensation: Varies

Number of Visits: Unknown

Duration: 6 years

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial explores the long-term effects of CDZ173, a PI3Kdelta inhibitor, for individuals with activated PI3Kdelta syndrome (APDS). The goal is to assess the safety and effectiveness of CDZ173 over time for those who participated in a previous related study or have used similar treatments. This trial suits patients with a specific genetic mutation linked to APDS who have faced frequent infections or other immune system issues. Participants should have been part of a previous study or have tried other PI3Kdelta inhibitors. As a Phase 2, Phase 3 trial, this research measures the treatment's effectiveness in an initial, smaller group and represents the final step before FDA approval, offering participants a chance to contribute to the development of a potentially groundbreaking therapy.

Do I have to stop taking my current medications for the trial?

The trial information does not specify if you need to stop taking your current medications. It's best to discuss this with the trial team or your doctor.

Is there any evidence suggesting that CDZ173 is likely to be safe for humans?

Research has shown that CDZ173, a treatment for activated PI3Kδ syndrome (APDS), is generally well-tolerated by patients. In studies, individuals with APDS who took CDZ173 experienced fewer side effects compared to those who did not. Reports indicate that CDZ173 is unlikely to cause serious side effects. While some patients experienced mild issues, these were not severe enough to discontinue treatment. This suggests that CDZ173 is relatively safe for long-term use in individuals with this genetic condition. ^{1 2 3 4 5}

Why are researchers excited about this possible treatment for activated PI3Kdelta syndrome?

Unlike standard treatments for Activated PI3Kdelta Syndrome, which often include broad immunosuppressants, CDZ173 specifically targets the PI3Kdelta pathway. This precision targeting helps to directly address the underlying cause of the syndrome rather than just managing symptoms. Researchers are excited about CDZ173 because it offers a more focused approach with the potential for fewer side effects and improved efficacy. Additionally, its once-daily oral administration makes it convenient for patients compared to some existing therapies that might require more complex dosing regimens.

What evidence suggests that CDZ173 might be an effective treatment for APDS?

Research has shown that CDZ173, also known as leniolisib, may help treat Activated PI3Kdelta Syndrome (APDS). Studies have found that it can lower the high rates of infections often seen in people with this condition. Additionally, CDZ173 reduces lymphadenopathy, decreasing swelling in the lymph nodes. Those who used this treatment experienced fewer respiratory infections and changes in their blood's immune markers. Overall, leniolisib appears well tolerated and offers important benefits for individuals with APDS. ^{2 3 4 6 7}

Are You a Good Fit for This Trial?

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.

Inclusion Criteria

I have been part of the CCDZ173X2201 study or treated with PI3Kδ inhibitors.

My doctor thinks a PI3Kδ inhibitor treatment could help me.

I (or my legal guardian) can communicate well and follow the study's requirements.

Exclusion Criteria

I have a serious health condition not related to APDS/PASLI.

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive long-term CDZ173 treatment, a selective PI3Kδ inhibitor, to evaluate safety, tolerability, efficacy, and pharmacokinetics

6 years

Follow-up

Participants are monitored for safety and effectiveness after treatment

6 months

Open-label extension

Participants continue to receive CDZ173 treatment to establish long-term safety and efficacy

Long-term

What Are the Treatments Tested in This Trial?

Interventions

• CDZ173

Trial Overview 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.

How Is the Trial Designed?

Group I: CDZ173 Experimental Treatment 1 Intervention

140mg/day

Who Is Running the Clinical Trial?

Novartis Pharmaceuticals

Lead Sponsor

Trials

2,963

Recruited

4,275,000+

Founded

1996

Headquarters

Basel, Switzerland

Known For

Precision medicine

Top Products

Gleevec, Cosentyx, Entresto, Kisqali

Dr. Vas Narasimhan

Novartis Pharmaceuticals

Chief Executive Officer since 2018

MD from Harvard Medical School

Dr. Shreeram Aradhye

Novartis Pharmaceuticals

Chief Medical Officer since 2021

MD

Pharming Technologies B.V.

Lead Sponsor

Trials

23

Recruited

1,400+

Citations

1. pubmed.ncbi.nlm.nih.gov pubmed.ncbi.nlm.nih.gov/39673396

Comparative efficacy of leniolisib (CDZ173) versus ... This externally controlled study compared the long-term effects of leniolisib on annual rate of respiratory tract infections and change in serum immunoglobulin ...

2. clinicaltrials.gov clinicaltrials.gov/study/NCT02859727?cond=%22Activated%20PI3K-delta%20syndrome%22&rank=4

Extension to the Study of Efficacy of CDZ173 in Patients ... 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 ...

3. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC11754865/

Comparative efficacy of leniolisib (CDZ173) versus ... The results demonstrate the potential for leniolisib to offer important benefits for people with APDS by reducing the high rates of infections in this ...

4. novctrd.com novctrd.com/ctrdweb/patientsummary/patientsummaries?patientSummaryId=1058

The effects and safety of CDZ173 for adults and young ... The purpose was to learn about the safety and effects of CDZ173 in people with APDS. APDS is a genetic condition that leads to a weakened immune system. In APDS ...

5. ashpublications.org ashpublications.org/blood/article/141/9/971/493284/A-randomized-placebo-controlled-phase-3-trial-of

A randomized, placebo-controlled phase 3 trial of the PI3Kδ ... Overall, leniolisib was well tolerated and significant improvement over placebo was notable in the coprimary endpoints, reducing lymphadenopathy ...

6. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC5601375/

Discovery of CDZ173 (Leniolisib), Representing a ... Therefore, CDZ173 could differentiate by a more favorable safety profile. ... In patients with activated PI3Kδ syndrome (APDS), mutations in the PIK3CD ...

7. trial.medpath.com trial.medpath.com/clinical-trial/cb0c0596ce982e01/euctr2014-003876-22-gb-cdz173-efficacy-apds-pasli

Study to assess the safety and efficacy of CDZ173 in patients ... Male and female patients age 12 to 75 years of age (inclusive), who have a documented APDS/PASLI-associated genetic PI3K delta mutation