UnknownPhase 3

An Open-Label Study to Assess the Safety & Efficacy of Leniolisib in Japanese Patients With APDS

Japan3 participantsStarted 2023-08-03

Plain-language summary

An Open-Label, Non-Randomized Study to Assess the Safety and Efficacy of Leniolisib in Japanese Patients With Activated Phosphoinositide 3-Kinase Delta Syndrome (APDS) Followed By an Open-Label Long-Term Extension. For the treatment of activated phosphoinositide 3-kinase delta (PI3Kδ) syndrome (APDS).

Who can participate

Age range

12 Years – 75 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.

Exclusion criteria

. A mammalian target of rapamycin inhibitor (e.g., sirolimus, rapamycin, or everolimus) or a PI3Kδ inhibitor (selective or non-selective phosphoinositide 3-kinase inhibitors) within 6 weeks prior to first dose.
. B-cell depleters (e.g., rituximab) within 6 months prior to first dose of study treatment.
. Belimumab or cyclophosphamide within 6 months prior to first dose of study treatment.
. Cyclosporine A, mycophenolate, 6-mercaptopurine, azathioprine, or methotrexate within 3 months prior to first dose of study treatment.
. Glucocorticoids above 25 mg prednisone or equivalent per day within 2 weeks prior to first dose of study treatment.
. Other immunosuppressive medications where effects are expected to persist at start of dosing of study treatment.

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: Incidence of treatment-emergent adverse events ((AEs), serious adverse events (SAEs), and AEs

Timeframe: Between baseline until Day 85

Part II: Long-term Incidence of treatment-emergent adverse events ((AEs), serious adverse events (SAEs), and AEs

Timeframe: At Day 252, through study completion, an average of 1 year

Part I: Change from baseline in clinical laboratory test results

Timeframe: Between baseline until Day 85

Part II: Long-term change from baseline in clinical laboratory test results

Timeframe: At Day 252, through study completion, an average of 1 year

Part I: Change from baseline in vital signs

Timeframe: Between baseline until Day 85

Part II: Long-term change from baseline in vital signs

Timeframe: At Day 252, through study completion, an average of 1 year

Part I: Change from baseline in physical examination findings

Trial details

NCT IDNCT06249997

SponsorPharming Technologies B.V.

Sponsor typeINDUSTRY

Study typeINTERVENTIONAL

Primary completion2025-01-31

Contact for this trial

Jason Bradt, MD

+31715247400 J.Bradt@pharming.com

View on ClinicalTrials.gov More APDS Gene Mutation trials

Plain-language summary

Who can participate

Age range

12 Years – 75 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.

Exclusion criteria

. A mammalian target of rapamycin inhibitor (e.g., sirolimus, rapamycin, or everolimus) or a PI3Kδ inhibitor (selective or non-selective phosphoinositide 3-kinase inhibitors) within 6 weeks prior to first dose.
. B-cell depleters (e.g., rituximab) within 6 months prior to first dose of study treatment.
. Belimumab or cyclophosphamide within 6 months prior to first dose of study treatment.
. Cyclosporine A, mycophenolate, 6-mercaptopurine, azathioprine, or methotrexate within 3 months prior to first dose of study treatment.
. Glucocorticoids above 25 mg prednisone or equivalent per day within 2 weeks prior to first dose of study treatment.
. Other immunosuppressive medications where effects are expected to persist at start of dosing of study treatment.

What they're measuring

Part I: Incidence of treatment-emergent adverse events ((AEs), serious adverse events (SAEs), and AEs

Timeframe: Between baseline until Day 85

Part II: Long-term Incidence of treatment-emergent adverse events ((AEs), serious adverse events (SAEs), and AEs

Timeframe: At Day 252, through study completion, an average of 1 year

Part I: Change from baseline in clinical laboratory test results

Timeframe: Between baseline until Day 85

Part II: Long-term change from baseline in clinical laboratory test results

Timeframe: At Day 252, through study completion, an average of 1 year

Part I: Change from baseline in vital signs

Timeframe: Between baseline until Day 85

Part II: Long-term change from baseline in vital signs

Timeframe: At Day 252, through study completion, an average of 1 year

Part I: Change from baseline in physical examination findings