EUCTR2014-003876-22-CZ进行中（未招募）1 期
An open-label, non-randomized, within-patient dose-finding study followed by a randomized, subject, investigator and sponsor-blinded placebo controlled study to assess the efficacy and safety of CDZ173 (Leniolisib) in patients with APDS/PASLI (Activated phosphoinositide 3-kinase delta syndrome/ p110d-activating mutation causing senescent T cells, lymphadenopathy and immunodeficiency) - Study of efficacy of CDZ173 in patients with APDS/PASLI
适应症
试验速览
- 阶段
- 1 期
- 状态
- 进行中（未招募）
- 入组人数
- 36
研究概览
简要总结
暂无简介。
研究设计
- 研究类型
- Interventional clinical trial of medicinal product
入排标准
- 性别
- All
入选标准
- •Male and female patients 12 to 75 years of age (inclusive), who have a documented APDS/PASLI-associated genetic PI3K delta mutation. Patients with mutations in either PIK3CD or PIK3R1 can be included.
- •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-sino-pulmonary infections and/or organ dysfunction (e.g., lung, liver). Additionally, in part II, patients must have at least one measurable nodal lesion on a CT or MRI scan.
- •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, 90-139 mm Hg
- •-Diastolic blood pressure, 50-89 mm Hg
- •-Pulse rate, 50 - 100 bpm; up to 110 bpm in adolescents
- •Are the trial subjects under 18? yes
- •Number of subjects for this age range: 6
- •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
排除标准
- •Previous or concurrent use of immunosuppressive medication such as:
- •use of an mTOR inhibitor (e.g., sirolimus, 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 25 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.
- •Current use of medication known to be strong inhibitor or moderate or
- •strong inducers of isoenzyme CYP3A, if treatment cannot be
- •discontinued or switched to a different medication prior to starting study
- •Current use of medications 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 during dosing of study medication and for 2
- •days after stopping study treatment.
研究者
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