Active — Not RecruitingPhase 3

Pediatric Patients Aged 1 to 6 Years With APDS

United States, Japan, Portugal16 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 range

1 Year – 6 Years

Sex

ALL

See this in plain English?

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

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

Questions worth asking your doctor

Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.

1Based on my diagnosis and history, is this trial worth exploring for me — or is there a standard treatment we should try first?
2What does this trial's phase tell us about how much is already known about its safety and benefit?
3What would taking part actually involve for me — visits, tests, time, and travel?
4What are the known and possible risks or side effects I should weigh, and how would they be monitored?
5If this trial isn't the right fit, what other options or trials would you suggest I look into?

Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.

Questions for the trial coordinator

The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.

1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?

A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.

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 IDNCT05693129

SponsorPharming Technologies B.V.

Sponsor typeINDUSTRY

Study typeINTERVENTIONAL

Primary completion2028-07-09

View on ClinicalTrials.gov More APDS trials

Plain-language summary

Who can participate

Age range

1 Year – 6 Years

Sex

ALL

See this in plain English?

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

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

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