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

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ClinicalTrials.gov Identifier: NCT02859727
Recruitment Status : Recruiting
First Posted : August 9, 2016
Last Update Posted : October 16, 2019
Sponsor:
Information provided by (Responsible Party):
Novartis ( Novartis Pharmaceuticals )

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.

Condition or disease Intervention/treatment Phase
Activated PI3Kdelta Syndrome (APDS); PASLI Disease Drug: CDZ173 Phase 2 Phase 3

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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 42 participants
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: 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)
Actual Study Start Date : September 8, 2016
Estimated Primary Completion Date : September 2, 2025
Estimated Study Completion Date : September 2, 2025

Resource links provided by the National Library of Medicine


Arm Intervention/treatment
Experimental: CDZ173
140mg/day
Drug: CDZ173
140 mg/day




Primary Outcome Measures :
  1. 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))


Secondary Outcome Measures :
  1. 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

  2. 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

  3. To characterize the pharmacokinetics (trough concentration) of CDZ173 in patients with APDS/PASLI [ Time Frame: 9 months ]
    Steady-state trough concentration of CDZ173

  4. • 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)



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Ages Eligible for Study:   12 Years to 75 Years   (Child, Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

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.


Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02859727


Contacts
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Contact: Novartis Pharmaceuticals 1-888-669-6682 novartis.email@novartis.com
Contact: Novartis Pharmaceuticals +41613241111

Locations
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United States, Maryland
Novartis Investigative Site Recruiting
Bethesda, Maryland, United States, 20892
Czechia
Novartis Investigative Site Recruiting
Prague 5, CZE, Czechia, 15006
Ireland
Novartis Investigative Site Recruiting
Dublin, Ireland
Italy
Novartis Investigative Site Recruiting
Palermo, PA, Italy, 90127
Netherlands
Novartis Investigative Site Recruiting
Rotterdam, Netherlands, 3000 CA
Sponsors and Collaborators
Novartis Pharmaceuticals

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Responsible Party: Novartis Pharmaceuticals
ClinicalTrials.gov Identifier: NCT02859727     History of Changes
Other Study ID Numbers: CCDZ173X2201E1
2016-000468-41 ( EudraCT Number )
First Posted: August 9, 2016    Key Record Dates
Last Update Posted: October 16, 2019
Last Verified: October 2019
Individual Participant Data (IPD) Sharing Statement:
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


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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Keywords provided by Novartis ( Novartis Pharmaceuticals ):
APDS; PASLI; PI3Kdelta; p110delta-activating mutation causing senescent T cells, lymphadenopathy and immunodeficiency; Activated PI3Kdelta Syndrome;
Additional relevant MeSH terms:
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Lymphadenopathy
Syndrome
Disease
Pathologic Processes
Lymphatic Diseases