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Trial record 1 of 1 for:    NCT02859727
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Extension to the Study of Efficacy of CDZ173 in Patients With APDS/PASLI

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT02859727
Recruitment Status : Recruiting
First Posted : August 9, 2016
Last Update Posted : April 28, 2020
Sponsor:
Information provided by (Responsible Party):
Novartis ( Novartis Pharmaceuticals )

Tracking Information
First Submitted Date  ICMJE August 4, 2016
First Posted Date  ICMJE August 9, 2016
Last Update Posted Date April 28, 2020
Actual Study Start Date  ICMJE September 8, 2016
Estimated Primary Completion Date September 1, 2026   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: June 5, 2018)
To evaluate the long term safety and tolerability of CDZ173 in patients with APDS/PASLI [ Time Frame: 3 years 3 months ]
All safety parameters (including AEs, physical exam, vital signs, ECG, safety laboratory (hematology, blood chemistry, urinalysis))
Original Primary Outcome Measures  ICMJE
 (submitted: August 4, 2016)
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))
Change History
Current Secondary Outcome Measures  ICMJE
 (submitted: August 21, 2018)
  • To evaluate the long term efficacy of CDZ173 to modify health-related quality of life in patients with APDS/PASLI [ Time Frame: 3 years ]
    • 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
  • 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 [ Time Frame: 3 years 3 months ]
    • High sensitivity C-reactive protein (CRP), lactate dehydrogenase (LDH), frequencies of infections and other disease complication
  • To characterize the pharmacokinetics (trough concentration) of CDZ173 in patients with APDS/PASLI [ Time Frame: 9 months ]
    Steady-state trough concentration of CDZ173
  • • To evaluate the pharmacokinetics and relative bioavailability of CDZ173 film-coated tablets compared to CDZ173 hard-gelatin capsules [ Time Frame: up to 6 months ]
    • PK parameters (including but not limited to AUC0-12,ss and Cmax,ss)
Original Secondary Outcome Measures  ICMJE
 (submitted: August 4, 2016)
  • 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
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title  ICMJE Extension to the Study of Efficacy of CDZ173 in Patients With APDS/PASLI
Official Title  ICMJE An Open-label, Non-randomized 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/p110δ-activating Mutation Causing Senescent T Cells, Lymphadenopathy and Immunodeficiency)
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 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.
Detailed Description Not Provided
Study Type  ICMJE Interventional
Study Phase  ICMJE Phase 2
Phase 3
Study Design  ICMJE Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Condition  ICMJE Activated PI3Kdelta Syndrome (APDS); PASLI Disease
Intervention  ICMJE Drug: CDZ173
140 mg/day
Study Arms  ICMJE Experimental: CDZ173
140mg/day
Intervention: Drug: CDZ173
Publications * Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Recruiting
Estimated Enrollment  ICMJE
 (submitted: June 5, 2018)
42
Original Estimated Enrollment  ICMJE
 (submitted: August 4, 2016)
36
Estimated Study Completion Date  ICMJE September 1, 2026
Estimated Primary Completion Date September 1, 2026   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

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.

Sex/Gender  ICMJE
Sexes Eligible for Study: All
Ages  ICMJE 12 Years to 75 Years   (Child, Adult, Older Adult)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE
Contact: Novartis Pharmaceuticals 1-888-669-6682 novartis.email@novartis.com
Contact: Novartis Pharmaceuticals +41613241111
Listed Location Countries  ICMJE Czechia,   Ireland,   Italy,   Netherlands,   United States
Removed Location Countries Czech Republic
 
Administrative Information
NCT Number  ICMJE NCT02859727
Other Study ID Numbers  ICMJE CCDZ173X2201E1
2016-000468-41 ( EudraCT Number )
Has Data Monitoring Committee Yes
U.S. FDA-regulated Product
Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
IPD Sharing Statement  ICMJE
Plan to Share IPD: Undecided
Plan Description:

Novartis is committed to sharing with qualified external researchers, access to patient-level data and supporting clinical documents from eligible studies. These requests are reviewed and approved by an independent review panel on the basis of scientific merit. All data provided is anonymized to respect the privacy of patients who have participated in the trial in line with applicable laws and regulations.

This trial data availability is according to the criteria and process described on www.clinicalstudydatarequest.com

Responsible Party Novartis ( Novartis Pharmaceuticals )
Study Sponsor  ICMJE Novartis Pharmaceuticals
Collaborators  ICMJE Not Provided
Investigators  ICMJE Not Provided
PRS Account Novartis
Verification Date April 2020

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP