CDZ173 for Activated PI3Kdelta Syndrome

Recruiting at 24 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

37 Participants Needed

Trial Summary

What is the purpose of this trial?

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).

Eligibility Criteria

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

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

Treatment Details

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.

Participant Groups

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+