Study of the ITK Inhibitor Soquelitinib to Reduce Lymphoproliferation and Improve Cytopenias in Autoimmune Lymphoproliferative Syndrome (ALPS)-FAS Patients

• Status: RECRUITING
• Trial end date: 2026-12-01

Summary

Background:

Autoimmune lymphoproliferative syndrome (ALPS) is a rare disorder of the immune system caused by a mutation in the FAS gene. In ALPS, the body stores too many germ-fighting cells called lymphocytes. This can lead to an enlarged spleen and lymph nodes. Current treatments for ALPS can have many adverse effects. Better treatments for ALPS are needed.

Objective:

To test a study drug (soquelitinib) in people with ALPS.

Eligibility:

People aged 16 years and older with ALPS.

Design:

Participants will have 8 clinic visits and 6 remote visits within 1 year.

Participants will be screened. They will have a physical exam with blood and urine tests. Some may have tests of their lung function.

Soquelitinib is a tablet taken by mouth twice a day. Participants will record their doses and any symptoms on a paper or online form.

Blood tests and other procedures will be repeated during study visits. Three visits will include imaging scans. Participants will lie on a table that slides through a doughnut-shaped machine while X-rays capture pictures of the inside of their body.

Some participants may be able to remain in the study for a second year.

• Phase: PHASE2

Details

• Lead Sponsor: National Institute of Allergy and Infectious Diseases (NIAID)