CTIS2022-502180-38-00进行中（未招募）1 期
An Open-label, Single Arm Study of the Safety, Pharmacokinetics, Pharmacodynamics, and Efficacy of Leniolisib in Pediatric Patients (Aged 1 to 6 Years) with APDS (Activated Phosphoinositide 3-Kinase Delta Syndrome) Followed by an Open-label Long-term Extension - LE 3302
适应症
试验速览
- 阶段
- 1 期
- 状态
- 进行中（未招募）
- 入组人数
- 15
研究概览
简要总结
暂无简介。
研究设计
- 研究类型
- Interventional
入排标准
- 年龄范围
- 0 至 17（—）
- 性别
- All
入选标准
- •1. Patient is male or female and between the age of 1 to 6 years old at time of the first study procedure., 10.Patient parent or legal guardian agrees patient will not participate in any other interventional study while enrolled in this study., 2.Patient weighs =8 and =37 kg at baseline., 3.Patient has a confirmed PI3Kd genetic mutation of either the PIK3CD (APDS1) or PIK3R1 (APDS2) gene., 4.Patient has at least 1 measurable nodal lesion on magnetic resonance imaging or low-dose computed tomography within 6 months of screening., 5.Patient has nodal or extranodal lymphoproliferation and clinical findings consistent with APDS (eg, a history of repeated oto-sino-pulmonary infections and/or organ dysfunction consistent with APDS)., 6.Patient has the ability to ingest unaltered study-related medications without difficulty in the investigator's opinion., 7.At screening, vital signs (body temperature, systolic blood pressure [BP], diastolic BP, and pulse rate [PR]) will be assessed in the sitting position (infants may be assessed lying down) after the patient has been at rest for at least 3 minutes. Patient’s sitting vital signs should be within the following ranges: a.Systolic BP: Less than the 95th percentile adjusted for sex, age, and height percentile. b.Diastolic BP: Less than the 95th percentile adjusted for sex, age, and height percentile. c.Pulse rate: -Age <2 years: 100 to 190 beats per minute (bpm) -Age 2 to 6 years: 60 to 140 bpm, 8.Institutional review board- or independent ethics committee (IEC)-approved written informed consent or assent and privacy language as per national and local regulations must be obtained from the patient and/or parent or legal guardian prior to any study-related procedures., 9.Patient parent or legal guardian is willing and able to complete the informed consent or assent process and comply with study procedures and visit schedule.
排除标准
- •1.Patient has previous or concurrent use of immunosuppressive medication such as: a.an mTOR inhibitor (eg, sirolimus, rapamycin, everolimus) or a PI3Kd inhibitor (selective or non-selective PI3K inhibitors) within 6 weeks prior to first dose. oShort-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.B cell depleters (eg, rituximab) within 6 months prior to first dose of study medication. oIf patient has received prior treatment with a B cell depleter, absolute B lymphocyte counts in the blood must have regained normal values. c.Belimumab or cyclophosphamide within 6 months prior to first dose of study medication. d.Cyclosporine A, mycophenolate, 6-mercaptopurine, azathioprine, or methotrexate within 3 months prior to first dose of study medication. e.Glucocorticoids above a dose equivalent to either =2 mg/kg of body weight for body weights less than 10 kg or =20 mg/day for body weights = 10 kg of prednisone or prednisolone or equivalent within 2 weeks prior to first dose of study medication. f.Other immunosuppressive medication where effects are expected to persist at start of dosing of study medication., 18.Patient has uncontrolled chronic or recurrent infectious disease (with the exception of those that are considered to be characteristic of APDS) or evidence of tuberculosis infection as defined by a positive Mantoux tuberculin skin test or a positive QuantiFERON-TB Gold skin test at screening. If presence of latent tuberculosis is established, then treatment according to local country guidelines must have been completed before patients can be considered for enrollment., 10.Patient is receiving concurrent treatment with another investigational therapy or use of another investigational therapy less than 4 weeks from the first study procedure., 2.Patient has a history or current diagnosis of electrocardiogram (ECG) abnormalities indicating significant risk of safety for patients participating in the study such as: a.History of familial long QT syndrome or known family history of Torsades de Pointes. b.Concomitant clinically significant cardiac arrhythmias, eg, sustained ventricular tachycardia, and clinically significant second or third degree atrioventricular block without a pacemaker. c.Resting QTc (Fridericia preferred, but Bazett acceptable) >460 msec if the measurement is confirmed with an additional ECG repeated as soon as possible. d.Concomitant use of agents known to prolong the QT interval unless it can be permanently discontinued for the duration of the study., 3.Patient is currently using a medication known to be strong inhibitor or moderate or strong inducer of isoenzyme cytochrome P450 (CYP)3A, if treatment cannot be discontinued or switched to a different medication prior to starting study treatment., 4.Patient is currently using medications that are metabolized by isoenzyme CYP1A2 and have a narrow therapeutic index (NTI) (drugs whose exposure response indicates that increases in their exposure levels by the concomitant use of potent inhibitors may lead to serious safety concerns [eg, Torsades de Pointes])., 5.Patient is currently using medications known to be organic anion transporter protein (OATP)1B1, OATP1B3, and breast cancer resistance protein (BCRP) substrates., 6.Patient had been administered live vaccines (this includes any attenuated live vaccines) starting from 6 weeks before the anticipated first study drug administration, during the s
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