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PI3K Inhibitor

Leniolisib for Primary Immunodeficiency

Phase 2

Recruiting

Led By Gulbu Uzel, M.D.

Research Sponsored by Pharming Technologies B.V.

Eligibility Criteria Checklist

Specific guidelines that determine who can or cannot participate in a clinical trial

Must have

Subjects must be 12 to 75 years of age

Diagnosed with a primary immunodeficiency disorder (PID) due to disease-causing pathogenic or likely pathogenic variant(s) in specific genes or diagnosed with RAS associated leukoproliferative disorder (excluding juvenile myelomonocytic leukemia) due to somatic variants in NRAS or KRAS

Must not have

Subject has had a successful hematopoietic stem-cell transplant (HSCT)

Previous or concurrent use of specific immunosuppressive medications

Timeline

Screening 3 weeks

Treatment Varies

Follow Up from baseline to the end of 20 weeks of treatment

Awards & highlights

No Placebo-Only Group

Summary

This trial is testing the safety and tolerability of a drug called leniolisib. They will be giving different doses of the drug to the same patient to see how well they tolerate it. They

Who is the study for?

This trial is for people aged 12-75 with Primary Immunodeficiency (PID) who have certain blood issues, enlarged spleen or lymph nodes, specific blood pressure and heart rate ranges, lung disease related to PID, and genetic mutations linked to PID. It's not suitable for those with conditions that could explain their symptoms like iron-deficiency.

What is being tested?

The study tests Leniolisib at three different doses in patients with immune dysregulation due to PIDs. It's an open-label trial where everyone knows what treatment they're getting. The main goal is safety; other goals include how the body processes the drug and its effectiveness.

What are the potential side effects?

Since this is a safety-focused study of Leniolisib for immune dysregulation in PIDs, potential side effects are being closely monitored but may include reactions typical of new medications affecting the immune system.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below

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I am between 12 and 75 years old.

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I have a genetic disorder affecting my immune system or a specific type of blood disorder not including juvenile myelomonocytic leukemia.

Exclusion Criteria

You may be eligible for the trial if you check “No” for criteria below:

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I have successfully undergone a stem-cell transplant.

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I have used certain medications that suppress my immune system.

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I am not pregnant, nursing, or I use effective birth control if of child-bearing age.

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I am taking medication that affects the CYP3A enzyme.

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I am currently taking medication that affects specific body processes.

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I have a long-term infection that isn't under control.

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I have an uncontrolled EBV-related condition after a transplant.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ from baseline to the end of 20 weeks of treatment

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~from baseline to the end of 20 weeks of treatment

This trial's timeline: 3 weeks for screening, Varies for treatment, and from baseline to the end of 20 weeks of treatment for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.

Primary study objectives

Number of Participants with Treatment-emergent adverse events (TEAEs), Serious Adverse Events (SAEs) , and Adverse Events (AEs)

Secondary study objectives

To assess the PK of leniolisib in PID disorders linked to PI3K

To assess the clinical efficacy of leniolisib on PFTs in PID disorders linked to PI3K

To assess the clinical efficacy of leniolisib on PID-related ILD in PID disorders linked to PI3K

+9 more

Awards & Highlights

No Placebo-Only Group

All patients enrolled in this study will receive some form of active treatment.

Trial Design

1Treatment groups

Experimental Treatment

Group I: LeniolisibExperimental Treatment1 Intervention

All subjects participating will receive Leniolisib film-coated tablets (FCTs) at a starting dose of 10 mg BID for 4 weeks, followed by 30 mg BID for 4 weeks, and then 70 mg BID for 12 weeks.

0 / 9Eligibility criteria met