EUCTR2022-001624-14-NL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 4 to 11 Years) With APDS (Activated Phosphoinositide 3-Kinase Delta Syndrome) Followed By an Open-Label Long-Term Extension
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 clinical trial of medicinal product
入排标准
- 性别
- All
入选标准
- •1. Patient is male or female and between the age of 4 to 11 years old at the time of the first study procedure.
- •2. Patient weighs =13 kg and <45 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/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 (systolic blood pressure [BP], diastolic BP, and pulse rate) will be assessed in the sitting position after the patient has been at rest for at least 3 minutes. Patient’s sitting vital signs should be within the following ranges:
- •Systolic BP: Less than the 95th percentile adjusted for sex, age, and height percentile.
- •Diastolic BP: Less than the 95th percentile adjusted for sex, age, and height percentile.
- •Heart rate (HR):
- •i) Age 4 to <10 years: 60 to 140 bpm
- •ii) Age =10 years: 50 to 100 bpm
- •8. Institutional review board-/independent ethics committee-approved written informed consent/assent and privacy language as per national and local regulations must be obtained from the patient and parent/legal guardian prior to any study-related procedures.
- •9. Patient parent/legal guardian is willing and able to complete the informed consent/assent process and comply with study procedures and visit schedule.
- •10. Patient parent/legal guardian agrees patient will not participate in any other interventional study while enrolled in this study.
- •11. Female patients should be of non-childbearing potential at screening (should not have reached menarche). Male patients with partners of childbearing potential should be willing to use a highly effective method of contraception for at least 30 days after the last study procedure if at risk of pregnancy.
- •12. Female patient and parent/legal guardian must agree to the following if menses develops after screening, up to 30 days after the last study procedure:
- •True sexual abstinence defined as refraining from heterosexual activity during the entire period of the study through 6 months post-study or
- •Using a highly effective method of contraception for at least 30 days after the last study procedure if at risk of pregnancy.
- •Are the trial subjects under 18? yes
- •Number of subjects for this age range: 15
- •F.1.2 Adults (18-64 years) no
- •F.1.2.1 Number of subjects for this age range
- •F.1.3 Elderly (>=65 years) no
- •F.1.3.1 Number of subjects for this age range
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.
- •b. B cell depleters (eg, rituximab) within 6 months prior to first dose of study medication.
- •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.
- •2. Patient has a history or current diagnosis of electrocardiogram (ECG) abnormalities indicating significant risk of safety for patients participating in the study.
- •3. Patient is currently using a medication known to be a 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.
- •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 starting from 6 weeks before the anticipated first study drug administration, during the study, and up to 7 days after the last dose of leniolisib.
- •7. Patient has clinically significant abnormalities in hematology or clinical chemistry parameters as determined by the investigator or medical monitor.
- •8. Patient has liver disease or liver injury as indicated by clinically significant abnormal liver function tests (alanine aminotransferase and aspartate aminotransferase >2.5 times upper limit of normal), history of renal injury/renal disease (eg, renal trauma, glomerulonephritis, or one kidney only), or presence of impaired renal function as indicated by a serum creatinine level >1.5 mg/dL (133 µmol/L).
- •9. Patient has moderate or severe hepatic impairment (Child-Pugh Class B or C).
- •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.
- •11. Patient has active hepatitis B (eg, hepatitis B surface antigen reactive) or active hepatitis C (eg, hepatitis C virus RNA [qualitative] is detected) at screening.
- •12. Patient has human immunodeficiency virus (HIV) infection (HIV 1 or 2) at screening.
- •13. Patient has a positive coronavirus disease 19 result (polymerase chain reaction or antigen) within 1 week prior to first dose. The patient can be rescreened after a subsequent negative result.
- •14. Patient has a history of malignancy (except lymphoma) within 3 years before the first study procedure or has evidence of residual disease from a previously diagnosed malignancy.
- •15. Patient has a previous diagnosis of lymphoma that has been treated with chemotherapy, radiotherapy, or transplant within 1 year of the first study procedure or is anticipated to require lymphoma treatment within 6 months of the first study procedure.
- •16. Patient has a history of uncontrolled diabetes mellitus within 3 mo
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