Clinical Trial: Extension to the Study of Efficacy of CDZ173 in Patients With APDS/PASLI

Study Status: Recruiting
Recruit Status: Recruiting
Study Type: Interventional

Official Title: An Open-label Extension Study to Evaluate the Long Term Safety, Tolerability, Efficacy and Pharmacokinetics of CDZ173 in Patients With APDS/PASLI (Activated Phosphoinositide 3-kinase Delta Syndrome/p1

Brief Summary: 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 that already participated to the CDZ173X2201 study. The study is open-label designed to establish the long-term safety, tolerability, efficay and pharmacokinetics of CDZ173 in the target population.

Detailed Summary:
Sponsor: Novartis Pharmaceuticals

Current Primary Outcome: To evaluate the long term safety and tolerability of CDZ173 in patients with APDS/PASLI who completed the study CCDZ173X2201. [ Time Frame: 10 months ]

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


Original Primary Outcome: Same as current

Current Secondary Outcome:

  • To evaluate the long term efficacy of CDZ173 by means of clinical assessments and biomarkers reflecting the reduction of systemic inflammatory components of the disease in patients with APDS/PASLI who completed study CCDZ173X2201. [ Time Frame: 10 months ]
    C-reactive protein (CRP), lactate dehydrogenase (LDH), Visual analogue scales for PtGA, WPAI-CIQ (Work Productivity Activity Impairment plus Classroom Impairment Questionnaire), frequencies of infection and other disease complications, narratives by Investigator
  • To characterize the pharmacokinetics of CDZ173 in patients with APDS/PASLI who completed study CCDZ173X2201. [ Time Frame: 10 months ]
    Steady-state pre-dose concentration of CDZ173


Original Secondary Outcome: Same as current

Information By: Novartis

Dates:
Date Received: August 4, 2016
Date Started: September 8, 2016
Date Completion: December 31, 2020
Last Updated: April 18, 2017
Last Verified: April 2017