New drug aims to calm overactive immune system in rare disorders

NCT ID NCT06549114

Ongoing Disease control Sponsor: Pharming Technologies B.V. Source: ClinicalTrials.gov ↗

First seen Jun 27, 2026 · Last updated Jun 27, 2026

Summary

This study tests a drug called leniolisib in 12 people aged 12 to 75 with certain primary immunodeficiency disorders. The goal is to see if the drug is safe and can help control immune system problems. Participants will receive three different doses of the drug, and researchers will monitor side effects and how well the drug works.

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Conditions

The condition(s) this trial relates to.

autoimmune lymphoproliferative syndrome due to CTLA4 haploinsufficiency autoimmune lymphoproliferative syndrome type 4 FAS-related autoimmune lymphoproliferative syndrome immunodeficiency, common variable, 12 inborn error of immunity Primary Immunodeficiency Diseases PTEN harmartoma tumor syndrome with immune disorder

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

PRIMARY IMMUNODEFICIENCY DISORDERS (PIDS)

Contacts and locations

Locations

National Institute of Health

Bethesda, Maryland, 20892, United States