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

Primary Purpose

Activated PI3Kdelta Syndrome (APDS); PASLI Disease

Status
Active
Phase
Phase 2
Locations
International
Study Type
Interventional
Intervention
CDZ173
Sponsored by
Pharming Technologies B.V.
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Activated PI3Kdelta Syndrome (APDS); PASLI Disease focused on measuring APDS; PASLI; PI3Kdelta; p110delta-activating mutation causing senescent T cells, lymphadenopathy and immunodeficiency; Activated PI3Kdelta Syndrome;

Eligibility Criteria

12 Years - 75 Years (Child, Adult, Older Adult)All SexesDoes not accept healthy volunteers

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.

Sites / Locations

  • Novartis Investigative Site
  • Novartis Investigative Site
  • Novartis Investigative Site
  • Novartis Investigative Site
  • Novartis Investigative Site
  • Novartis Investigative Site
  • Novartis Investigative Site
  • Novartis Investigative Site

Arms of the Study

Arm 1

Arm Type

Experimental

Arm Label

CDZ173

Arm Description

140mg/day

Outcomes

Primary Outcome Measures

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))

Secondary Outcome Measures

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

Full Information

First Posted
August 4, 2016
Last Updated
October 27, 2022
Sponsor
Pharming Technologies B.V.
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1. Study Identification

Unique Protocol Identification Number
NCT02859727
Brief Title
Extension to the Study of Efficacy of CDZ173 in Patients With APDS/PASLI
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)
Study Type
Interventional

2. Study Status

Record Verification Date
October 2022
Overall Recruitment Status
Active, not recruiting
Study Start Date
September 8, 2016 (Actual)
Primary Completion Date
January 21, 2027 (Anticipated)
Study Completion Date
January 21, 2027 (Anticipated)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Pharming Technologies B.V.

4. Oversight

Studies a U.S. FDA-regulated Drug Product
Yes
Studies a U.S. FDA-regulated Device Product
No
Data Monitoring Committee
Yes

5. Study Description

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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Activated PI3Kdelta Syndrome (APDS); PASLI Disease
Keywords
APDS; PASLI; PI3Kdelta; p110delta-activating mutation causing senescent T cells, lymphadenopathy and immunodeficiency; Activated PI3Kdelta Syndrome;

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 2, Phase 3
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
37 (Actual)

8. Arms, Groups, and Interventions

Arm Title
CDZ173
Arm Type
Experimental
Arm Description
140mg/day
Intervention Type
Drug
Intervention Name(s)
CDZ173
Intervention Description
140 mg/day
Primary Outcome Measure Information:
Title
To evaluate the long term safety and tolerability of CDZ173 in patients with APDS/PASLI
Description
All safety parameters (including AEs, physical exam, vital signs, ECG, safety laboratory (hematology, blood chemistry, urinalysis))
Time Frame
6 years 3 months
Secondary Outcome Measure Information:
Title
To evaluate the long term efficacy of CDZ173 to modify health-related quality of life in patients with APDS/PASLI
Description
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
Title
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
Description
High sensitivity C-reactive protein (CRP), lactate dehydrogenase (LDH), frequencies of infections and other disease complication
Time Frame
8 months
Title
To characterize the pharmacokinetics (trough concentration) of CDZ173 in patients with APDS/PASLI
Description
Steady-state trough concentration of CDZ173
Time Frame
9 months
Title
• To evaluate the pharmacokinetics and relative bioavailability of CDZ173 film-coated tablets compared to CDZ173 hard-gelatin capsules
Description
PK parameters (including but not limited to AUC0-12,ss and Cmax,ss)
Time Frame
up to 6 months

10. Eligibility

Sex
All
Minimum Age & Unit of Time
12 Years
Maximum Age & Unit of Time
75 Years
Accepts Healthy Volunteers
No
Eligibility 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.
Facility Information:
Facility Name
Novartis Investigative Site
City
Bethesda
State/Province
Maryland
ZIP/Postal Code
20892
Country
United States
Facility Name
Novartis Investigative Site
City
Minsk
ZIP/Postal Code
223053
Country
Belarus
Facility Name
Novartis Investigative Site
City
Prague 5
State/Province
CZE
ZIP/Postal Code
15006
Country
Czechia
Facility Name
Novartis Investigative Site
City
Dresden
ZIP/Postal Code
01307
Country
Germany
Facility Name
Novartis Investigative Site
City
Brescia
State/Province
BS
ZIP/Postal Code
25123
Country
Italy
Facility Name
Novartis Investigative Site
City
Palermo
State/Province
PA
ZIP/Postal Code
90127
Country
Italy
Facility Name
Novartis Investigative Site
City
Rotterdam
ZIP/Postal Code
3000 CA
Country
Netherlands
Facility Name
Novartis Investigative Site
City
Moscow
ZIP/Postal Code
117198
Country
Russian Federation

12. IPD Sharing Statement

Plan to Share IPD
Undecided
IPD Sharing 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

Learn more about this trial

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

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