Study to assess the safety and efficacy of CDZ173 in patients with APDS/PASLI

Recruitment & Eligibility

Status: ot Recruiting

Sex: All

Target Recruitment: 37

Inclusion Criteria

• Male and female patients age 12 to 75 years of age (inclusive), who have a documented APDS/PASLI-associated genetic PI3K delta mutation
• In part I and part II, patients must have nodal and/or extranodal lymphoproliferation, and clinical findings and manifestations compatible with APDS/PASLI such as a history of repeated oto-sinopulmonary infections and/or organ dysfunction (e.g., lung, liver). In part II, patients must have, additionally, at least one nodal lesion on the CT or MRI scan at baseline described by the Cheson criteria.
• At screening, vital signs (systolic and diastolic blood pressure and pulse rate) will be assessed in the sitting position after the patient has
rested for at least three minutes. Sitting vital signs should be within the following ranges:
¿ Systolic blood pressure, 90-160 mm Hg
¿ Diastolic blood pressure, 50-95 mm Hg
¿ Pulse rate, 40 - 100 bpm
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 24
F.1.3 Elderly (>=65 years) yes
F.1.3.1 Number of subjects for this age range 1

Exclusion Criteria

• Use of unstable i.v. Ig / s.c. Ig in the last 6 months before screening. Stable maintenance immunoglobulin regimen, as per local practice, such as regular injections with a consistent dosing interval (e.g., monthly) injections is acceptable
• Previous or concurrent use of immuno-suppressive medication such as:
- use of an mTOR inhibitor (e.g., rapamycin, everolimus) or a PI3Kd inhibitor (selective or non-selective PI3K inhibitors) within 6 weeks prior to first dosing, however short-term use for up to a total of 5 days is allowed but only up to 1 month prior to enrollment in the study.
- B cell depleters (e.g., rituximab) within 6 months prior to first dosing of study medication; if patients have received prior treatment with a B cell depleter, absolute B lymphocyte counts in the blood must have regained normal values.
- Belimumab or cyclophosphamide within 6 months prior to first dosing of study medication.
- Cyclosporine A, mycophenolate, 6-mercaptopurine, azathioprine or methotrexate within 3 months prior to first dosing of study medication.
- Glucocorticoids above 10 mg prednisone or equivalent per day within 2 weeks prior to first dosing of study medication.
- Other immunosuppressive medication where effects are expected to persist at start of dosing of study medication.
• Patients currently being treated with medication known to be strong inhibitors or moderate or strong inducers of isoenzyme CYP3A, if treatment cannot be discontinued or switched to a different medication prior to starting study treatment.
• Patients currently being treated with drugs that are metabolized by isoenzyme CYP1A2 and have a narrow therapeutic index (drugs whose exposure-response indicates that increases in their exposure levels by the concomitant use of potent inhibitors may lead to serious safety concerns (e.g., Torsades de Pointes)).
• Administration of live vaccines (this includes 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
• Pregnant or nursing (lactating) women, where pregnancy is defined as the state of a female after conception and until the termination of gestation, confirmed by a positive hCG laboratory test.
• Women of child-bearing potential, defined as all women physiologically capable of becoming pregnant, unless they are using highly effective
methods of contraception (excluding oral, patch, subcutaneous implant or injected hormonal contraceptives) during dosing of study medication
and for 2 days after stopping study treatment.

Study & Design

Study Type: Interventional clinical trial of medicinal product

Study Design: Not specified

Primary Outcome Measures

Name	Time	Method

Secondary Outcome Measures

Name	Time	Method

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