Activated PI3Kdelta Syndrome (APDS); PASLI Disease Clinical Trial
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)
| Verified date | March 2024 |
| Source | Pharming Technologies B.V. |
| Contact | n/a |
| Is FDA regulated | No |
| Health authority | |
| Study type | Interventional |
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.
| Status | Active, not recruiting |
| Enrollment | 37 |
| Est. completion date | January 21, 2027 |
| Est. primary completion date | January 21, 2027 |
| Accepts healthy volunteers | No |
| Gender | All |
| Age group | 12 Years to 75 Years |
| Eligibility | 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 PI3Kd inhibitors other than CDZ173. - Patients who are deemed by the Investigator to benefit from PI3Kd 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. |
| Country | Name | City | State |
|---|---|---|---|
| Belarus | Pharming Investigative Site | Minsk | |
| Czechia | Pharming Investigative Site | Prague 5 | CZE |
| Germany | Pharming Investigative Site | Dresden | |
| Italy | Pharming Investigative Site | Brescia | BS |
| Italy | Pharming Investigative Site | Palermo | PA |
| Netherlands | Pharming Investigative Site | Rotterdam | |
| Russian Federation | Pharming Investigative Site | Moscow | |
| United States | Pharming Investigative Site | Bethesda | Maryland |
| Lead Sponsor | Collaborator |
|---|---|
| Pharming Technologies B.V. |
United States, Belarus, Czechia, Germany, Italy, Netherlands, Russian Federation,
| Type | Measure | Description | Time frame | Safety issue |
|---|---|---|---|---|
| Primary | 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)) | 6 years 3 months | |
| Secondary | 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 | 6 years | |
| Secondary | 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 | 8 months | |
| Secondary | To characterize the pharmacokinetics (trough concentration) of CDZ173 in patients with APDS/PASLI | Steady-state trough concentration of CDZ173 | 9 months | |
| Secondary | • 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) | up to 6 months |