Extension to the Study of Efficacy of CDZ173 in Patients With APDS/PASLI
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ClinicalTrials.gov Identifier: NCT02859727 |
Recruitment Status :
Active, not recruiting
First Posted : August 9, 2016
Last Update Posted : March 22, 2024
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Condition or disease | Intervention/treatment | Phase |
---|---|---|
Activated PI3Kdelta Syndrome (APDS); PASLI Disease | Drug: CDZ173 | Phase 2 Phase 3 |
Study Type : | Interventional (Clinical Trial) |
Actual Enrollment : | 37 participants |
Allocation: | N/A |
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 : | January 21, 2027 |
Estimated Study Completion Date : | January 21, 2027 |
Arm | Intervention/treatment |
---|---|
Experimental: CDZ173
140mg/day
|
Drug: CDZ173
140 mg/day |
- To evaluate the long term safety and tolerability of CDZ173 in patients with APDS/PASLI [ Time Frame: 6 years 3 months ]All safety parameters (including AEs, physical exam, vital signs, ECG, safety laboratory (hematology, blood chemistry, urinalysis))
- To evaluate the long term efficacy of CDZ173 to modify health-related quality of life in patients with APDS/PASLI [ Time Frame: 6 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: 8 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)
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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 |
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.
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
United States, Maryland | |
Pharming Investigative Site | |
Bethesda, Maryland, United States, 20892 | |
Belarus | |
Pharming Investigative Site | |
Minsk, Belarus, 223053 | |
Czechia | |
Pharming Investigative Site | |
Prague 5, CZE, Czechia, 15006 | |
Germany | |
Pharming Investigative Site | |
Dresden, Germany, 01307 | |
Italy | |
Pharming Investigative Site | |
Brescia, BS, Italy, 25123 | |
Pharming Investigative Site | |
Palermo, PA, Italy, 90127 | |
Netherlands | |
Pharming Investigative Site | |
Rotterdam, Netherlands, 3000 CA | |
Russian Federation | |
Pharming Investigative Site | |
Moscow, Russian Federation, 117198 |
Responsible Party: | Pharming Technologies B.V. |
ClinicalTrials.gov Identifier: | NCT02859727 |
Other Study ID Numbers: |
CCDZ173X2201E1 2016-000468-41 ( EudraCT Number ) |
First Posted: | August 9, 2016 Key Record Dates |
Last Update Posted: | March 22, 2024 |
Last Verified: | March 2024 |
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 |
Studies a U.S. FDA-regulated Drug Product: | Yes |
Studies a U.S. FDA-regulated Device Product: | No |
APDS; PASLI; PI3Kdelta; p110delta-activating mutation causing senescent T cells, lymphadenopathy and immunodeficiency; Activated PI3Kdelta Syndrome; |
Lymphadenopathy Syndrome Disease Pathologic Processes Lymphatic Diseases |