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

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

TERMINATED

Clinical Phase

PHASE2/PHASE3

Total Enrollment

37 participants

Study Classification

INTERVENTIONAL

Study Start Date

2016-09-08

Study Completion Date

2025-01-30

Brief Summary

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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.

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Detailed Description

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Conditions

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Activated PI3Kdelta Syndrome (APDS); PASLI Disease

Study Design

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Allocation Method

NA

Intervention Model

SINGLE_GROUP

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

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CDZ173

140mg/day

Group Type EXPERIMENTAL

CDZ173

Intervention Type DRUG

140 mg/day

Interventions

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CDZ173

140 mg/day

Intervention Type DRUG

Eligibility Criteria

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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.