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

Phase 3TerminatedNCT02859727

Pharming Technologies B.V.37 enrolled

Overview

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 Type

INTERVENTIONAL

Allocation

Purpose

TREATMENT

Masking

NONE

Enrollment

Conditions

Activated PI3Kdelta Syndrome (APDS); PASLI Disease

Interventions

CDZ173DRUG

140 mg/day

Eligibility

Sex: ALLMin age: 12 YearsMax age: 75 Years

Medical Language ↔ Plain English

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.

Locations (8)

Pharming Investigative Site

Bethesda, Maryland, United States

Pharming Investigative Site

Minsk, Belarus

Pharming Investigative Site

Prague, CZE, Czechia

Pharming Investigative Site

Dresden, Germany

Outcomes

Primary Outcomes

To evaluate the long term safety and tolerability of CDZ173 in patients with APDS/PASLI

All safety parameters (including AEs, physical exam, vital signs, ECG, safety laboratory (hematology, blood chemistry, urinalysis))

Time frame: 6 years 3 months

Secondary Outcomes

To evaluate the long term efficacy of CDZ173 to modify health-related quality of life in patients with APDS/PASLI

SF-36 (Short Form 36) Survey and WPAI-CIQ (Work Productivity Activity Impairment plus Classroom Impairment Questionnaire), Visual analogue scales for Physician's Global Assessment (PGA) and Patient's Global Assessment (PtGA), patient narratives by Investigator

Time frame: 6 years

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

High sensitivity C-reactive protein (CRP), lactate dehydrogenase (LDH), frequencies of infections and other disease complication

Time frame: 8 months

To characterize the pharmacokinetics (trough concentration) of CDZ173 in patients with APDS/PASLI

Steady-state trough concentration of CDZ173

Time frame: 9 months

• To evaluate the pharmacokinetics and relative bioavailability of CDZ173 film-coated tablets compared to CDZ173 hard-gelatin capsules

PK parameters (including but not limited to AUC0-12,ss and Cmax,ss)

Time frame: up to 6 months

Data from ClinicalTrials.gov

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