Study to assess long-term safety and efficacy of CDZ173 in patients with APDS/PASLI

Phase 1

Conditions: APDS/PASLI (Activated phosphoinositide 3-kinase delta syndrome/p110d-activating mutation causing senescent T cells, lymphadenopathy and immunodeficiency)
MedDRA version: 20.0Level: PTClassification code 10064859Term: Primary immunodeficiency syndromeSystem Organ Class: 10010331 - Congenital, familial and genetic disorders
Therapeutic area: Diseases [C] - Immune System Diseases [C20]

Registration Number: EUCTR2016-000468-41-IT

Lead Sponsor: OVARTIS PHARMA SERVICES AG

Brief Summary: Not available

Detailed Description: Not available

Recruitment & Eligibility

Status: Authorised-recruitment may be ongoing or finished

Sex: All

Target Recruitment: 42

Inclusion Criteria

- Patients must have participated in 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. Patients with mutations in either PIK3CD or PIK3R1 can be included.
Are the trial subjects under 18? yes
Number of subjects for this age range:
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 26
F.1.3 Elderly (>=65 years) yes
F.1.3.1 Number of subjects for this age range 1

Exclusion Criteria

- Patients who withdrew consent from the study CCDZ173X2201.
- Use of other investigational drugs, except CDZ173, within 5 half-lives of enrollment, or within 30 days, whichever is longer.
- Previous or concurrent use of immunosuppressive medication
- Administration of any live vaccines (including any attenuated live vaccines) starting from 6 weeks before study entry, during the study and up to 7 days
after the last dose of CDZ173 should be excluded.
- Pregnant or nursing (lactating) women, where pregnancy is defined as the state of a female after conception and until the termination of gestation.
- Women of child-bearing potential, defined as all women physiologically capable of becoming pregnant, unless they are using highly effective
methods of contraception during dosing and for 2 days after last dose ofstudy medication.
- Uncontrolled chronic or recurrent infectious disease (with the exception of those that are considered to be characteristic of APDS/PASLI).

Study & Design

Study Type: Interventional clinical trial of medicinal product

Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
Main Objective: To evaluate the long term safety and tolerability of CDZ173 in patients with APDS/PASLI.;Secondary Objective: - To evaluate the long term efficacy of CDZ173 to modify healthrelated quality of life in patients with APDS/PASLI.<br>- 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.<br>- To characterize the pharmacokinetics (trough concentrations) of CDZ173 in patients with APDS/PASLI.<br>- To evaluate the pharmacokinetics and relative bioavailability of CDZ173 film-coated tablets compared to CDZ173 hard-gelatin capsules.;Primary end point(s): All safety parameters (including AEs, physical exam, vital signs, ECG, safety laboratory (hematology, blood chemistry, urinalysis));Timepoint(s) of evaluation of this end point: 5 years and 3 months

Secondary Outcome Measures

Name	Time	Method
Secondary end point(s): - 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<br>- High sensitivity C-reactive protein (CRP), lactate dehydrogenase (LDH), frequencies of infections and other disease complications.<br>- Steady-state trough concentration of CDZ173.<br>- PK parameters (including but not limited to AUC0-12,ss and Cmax,ss);Timepoint(s) of evaluation of this end point: 5 years and 3 months

Related Trials