Study of the ITK Inhibitor Soquelitinib to Reduce Lymphoproliferation and Improve Cytopenias in Autoimmune Lymphoproliferative Syndrome (ALPS)-FAS Patients
Sponsor: National Institute of Allergy and Infectious Diseases (NIAID) • ClinicalTrials.gov ID: NCT06730126
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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.
Interventions
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Soquelitinib
(Drug)
Soquelitinib is an ITK inhibitor in clinical development for treating relapsed/refractory T-cell lymphoma.
Eligibility criteria
Conditions
Locations
- National Institutes of Health Clinical Center — Bethesda, Maryland, United States
- The Children's Hospital of Philadelphia — Philadelphia, Pennsylvania, United States
- Texas Children's Hospital — Houston, Texas, United States
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Data sourced from ClinicalTrials.gov, a service of the U.S. National Institutes of Health.
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