JPRN-jRCT2031220625招募中3 期
An Open-label, Single Arm Study of the Safety, Pharmacokinetics, Pharmacodynamics, and Efficacy of Leniolisib in Pediatric Patients (Aged 4 to 11 Years) with APDS (Activated Phosphoinositide 3-Kinase Delta Syndrome) Followed by an Open-label Long-term Extension
Relan Anurag0 个研究点目标入组 2 人开始时间: 2023年2月9日最近更新:
适应症
试验速览
- 阶段
- 3 期
- 状态
- 招募中
- 发起方
- 入组人数
- 2
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
- >= 4age old 至 <= 11age old（—）
- 性别
- All
入选标准
- •Patient is male or female and between the age of 4 to 11 years old at time of the first study procedure. Females should be of nonchildbearing potential at screening.
- •-Patient weighs >=13 kg and <45 kg at baseline.
- •-Patient has confirmed PI3Kdelta genetic mutation of either the PIK3CD (APDS1) or PIK3R1 (APDS2) gene.
- •- Patient has at least 1 measurable nodal lesion on magnetic resonance imaging (MRI)/low-dose computed tomography (CT).
- •-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).
- •-Patient has the ability to ingest unaltered study-related medications without difficulty.
排除标准
- •-Patient has previous or concurrent use of immunosuppressive medication such as:
- •a. An mTOR inhibitor (eg, sirolimus, rapamycin, everolimus) or a PI3Kdelta inhibitor (selective or non-selective PI3K inhibitors) within 6 weeks prior to first dose.
- •i. 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. B cell depleters (eg, rituximab) within 6 months prior to first dose of study medication.
- •i. If 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. Systemic glucocorticoids above a dose equivalent to either >=2 mg/kg of body weight or >=20 mg/day of prednisone/prednisolone or equivalent.
- •f. Other immunosuppressive medication where effects are expected to persist at start of dosing of study medication.
- •-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.
- •-Patient is currently using a medication known to be strong inhibitor or moderate or strong inducer of isoenzyme CYP3A (see Table 2), if treatment cannot be discontinued or switched to a different medication prior to starting study treatment.
- •-Patient is currently using 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 [eg, Torsades de Pointes]).
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