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 range12 Years – 75 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

✕. 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

Timeframe: Between baseline until Day 85

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 range12 Years – 75 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

✕. 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

Timeframe: Between baseline until Day 85