Pediatric Patients Aged 1 to 6 Years With APDS (NCT05693129) | Clinical Trial Compass
Active — Not RecruitingPhase 3
Pediatric Patients Aged 1 to 6 Years With APDS
United States, Japan16 participantsStarted 2023-08-30
Plain-language summary
This is a 2-part, prospective, open-label, single arm, multicenter study to evaluate the safety, tolerability, pharmacokinetics (PK), pharmacodynamics (PDx), and efficacy of leniolisib in at least 15 pediatric patients (aged 1 to 6 years) with activated phosphoinositide 3-kinase delta (PI3Kδ) syndrome (APDS)
Who can participate
Age range1 Year – 6 Years
SexALL
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Inclusion criteria
✓. Patient is male or female and between the age of 1 to 6 years old at time of the first study procedure.
✓. Patient weighs ≥8 and ≤37 kg at baseline.
✓. Patient has a confirmed PI3Kδ genetic mutation of either the PIK3CD (APDS1) or PIK3R1 (APDS2) gene.
✓. Patient has at least 1 measurable nodal lesion on MRI or low-dose CT within 6 months of screening.
✓. Patient has nodal or extranodal lymphoproliferation and clinical findings consistent with APDS (eg, a history of repeated oto-sino-pulmonary infections or organ dysfunction consistent with APDS).
✓. Patient has the ability to ingest unaltered study-related medications without difficulty in the investigator's opinion.
✓. At screening, vital signs (body temperature, systolic BP, diastolic BP, and pulse rate \[PR\]) 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. See Section 10.5, Appendix 5 to determine BP percentiles adjusted for sex, age, and height percentile. (National High Blood Pressure Education Program Working Group on High Blood Pressure in Children and Adolescents, 2004). See Section 10.5, Appendix 5 to determine height percentiles.
Exclusion criteria
✕. Patient has previous or concurrent use of immunosuppressive medication such as:
What they're measuring
1
Part I & II: Number of Participants with Treatment-emergent adverse events (TEAEs), Serious Adverse Events (SAEs) , and Adverse Events (AEs)
Timeframe: From baseline to end of 12 weeks, & From baseline to through study completion, an average of 1 year, plus 30 days
2
Part I & II: Change from baseline in clinical laboratory test results
Timeframe: From baseline to end of 12 weeks, & From baseline to through study completion, an average of 1 year
3
Part I & II: Change from baseline in vital signs
Timeframe: From baseline to end of 12 weeks, & From baseline to through study completion, an average of 1 year
4
Part I & II: Change from baseline in physical examination findings
Timeframe: From baseline to end of 12 weeks, & From baseline to through study completion, an average of 1 year
5
Part I & II: Change from baseline in electrocardiograms (ECGs)
Timeframe: From baseline to end of 12 weeks, & From baseline to through study completion, an average of 1 year, plus 30 days
6
Part I & II: Change from baseline in growth and physical development
Timeframe: From baseline to end of 12 weeks, & From baseline to through study completion, an average of 1 year
. an mTOR inhibitor (eg, sirolimus, rapamycin, everolimus) or a PI3Kδ inhibitor (selective or non-selective PI3K inhibitors) within 6 weeks prior to first dose.
✕. B cell depleters (eg, rituximab) within 6 months prior to first dose of study medication.
✕. Belimumab or cyclophosphamide within 6 months prior to first dose of study medication.
✕. Cyclosporine A, mycophenolate, 6-mercaptopurine, azathioprine, or methotrexate within 3 months prior to first dose of study medication.
✕. Glucocorticoids above a dose equivalent to either ≥2 mg/kg of body weight for weights less than 10 kg or ≥20 mg/day for weights ≥ 10 kg of prednisone or prednisolone or equivalent within 2 weeks prior to first dose of study medication.
✕. Other immunosuppressive medication where effects are expected to persist at start of dosing of study medication.
✕. Patient has a history or current diagnosis of ECG abnormalities indicating significant risk of safety for patients participating in the study such as:
Part I & II: Reduction in lymphadenopathy as measured by MRI or low-dose CT
Timeframe: Part I: Baseline and Day 85 Part II: at Day 252, through study completion, an average of 1 year
8
Part I: A Percentage of Inhibition of Unstimulated and Stimulated pAkt Levels in B Cells