Leniolisib for Immune Dysregulation in CVID (NCT06897358) | Clinical Trial Compass
Active — Not RecruitingPhase 2
Leniolisib for Immune Dysregulation in CVID
United States, Spain, United Kingdom20 participantsStarted 2025-02-12
Plain-language summary
In this study, common variable immunodeficiency (CVID) patients will all receive the study drug, leniolisib, for a treatment period of 6 months. Participants will start on a lower dose of leniolisib, followed by a mid and then a higher dose level. The primary goal is to assess the safety and tolerability of leniolisib, and secondary goal is to assess the potential for leniolisib to provide benefits for patients.
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.
Inclusion criteria
. Subject is 12 to 75 years of age (inclusive).
. Subject must have a minimum body weight of 45 kg
. Subject has a clinical diagnosis of CVID supported by all of the following (a thru c):
. A low IgG level compared to age-adjusted reference range \[OR if this cannot be documented, subject must have one of the following: i) absent isohemagglutinins and/or poor response to vaccines; or ii) Low class-switched memory B cells less than 2%\]
. Low IgA and/or IgM compared to age-adjusted reference range
. No identified secondary causes of hypogammaglobulinemia
. Inborn Errors of Immunity/ PID Panel testing:
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
1
Safety & Tolerability
Timeframe: From baseline to the end of 24 weeks of treatment
. Lacks an identified pathogenic/likely pathogenic genetic driver for their CVID primary immunodeficiency OR
Exclusion criteria
. Laboratory evidence of significant T cell deficiency including CD4+ T cells \<200/uL.
. Laboratory evidence of significant NK cell deficiency including NK cells \<1% of peripheral blood lymphocytes or less than 50/mcL.
. Clinical history of infections suggestive of clinically significant T cell or NK cell deficiency such as Pneumocystis jirovecii, atypical mycobacteria, severe warts, or unusually severe (as determined by the PI) infections with herpesviruses.
. Presence of uncontrolled chronic/recurrent infectious disease (except those considered to be characteristic of antibody deficiency).
. Positive blood polymerase chain reaction (PCR) for cytomegalovirus.
. Evidence of tuberculosis infection
. Positive blood cryptococcal antigen
. Previous or concurrent use of immunosuppressive medication, such as: