Active — Not RecruitingPhase 3

Pediatric Patients Aged 4 to 11 Years With APDS

United States, France15 participantsStarted 2023-02-01

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 4 to 11 years) with activated phosphoinositide 3-kinase delta (PI3Kδ) syndrome (APDS).

Who can participate

Age range4 Years – 11 Years

SexALL

See this in plain English?

AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

Exclusion criteria

✕. Patient is male or female and between the age of 4 to 11 years old at the time of the first study procedure.
✕. Patient weighs ≥13 kg and \<45 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 magnetic resonance imaging/low-dose computed tomography 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 and/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 (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:
✕. 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.

What they're measuring

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

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

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

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

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

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

Trial details

NCT IDNCT05438407

SponsorPharming Technologies B.V.

Sponsor typeINDUSTRY

Study typeINTERVENTIONAL

Primary completion2026-11-30

View on ClinicalTrials.gov More APDS trials

Plain-language summary

Who can participate

Age range4 Years – 11 Years

SexALL

See this in plain English?

AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

Exclusion criteria

✕. Patient is male or female and between the age of 4 to 11 years old at the time of the first study procedure.
✕. Patient weighs ≥13 kg and \<45 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 magnetic resonance imaging/low-dose computed tomography 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 and/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 (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:
✕. 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.

What they're measuring

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

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

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

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

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

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