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

Recruiting now · Phase 2

Conditions studied: Autoimmune Lymphoproliferative Syndrome

In brief

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....

Key facts

Study ID

NCT06730126

Run by

National Institute of Allergy and Infectious Diseases (NIAID)

People needed

15

Starts

2025-03-10

Expected to finish

2026-12-01

Last updated by the study team

2026-08-07

Who can join

Age: 16 and older, up to 120. Sex: any. Healthy volunteers: not accepted.

You may not qualify if…

• An individual who meets any of the following criteria will be excluded from participation or re-enrollment in this study:
• Profound grade 3 or 4 cytopenias that cannot be improved with immunomodulatory treatments prior to enrolling in the clinical trial and starting the study drug. Per investigator discretion, individuals on a single agent may be included if the dose is stable for 12 weeks at screening and is not expected to confer additive toxicity.
• Renal impairment, defined as serum creatinine >1.5 mg/dL (or 133 micromol/L) or estimated glomerular filtration rate <60 mL/min/1.73 m\^2.
• Liver impairment, defined as bilirubin, alanine aminotransferase, or aspartate aminotransferase greater than 2.5 times the upper limit of normal.
• History of EBV-associated lymphoma.
• Active EBV infection with EBV load >300 copies/mL.
• Tuberculosis infection (active or latent) or undergoing tuberculosis treatment.
• Infection with HIV or hepatitis B or C.
• Current other invasive or systemic fungal, bacterial, or viral infection requiring therapy.
• History of opportunistic infection within the previous 180 days.
• History of invasive malignancy that required systemic therapy within the last 3 years.
• Current use of moderate or strong cytochrome P450 isozyme (CYP)3A inhibitors or inducers that cannot be stopped before day 0.
• Current use of P-glycoprotein (P-gp) inhibitors that cannot be stopped before day 0.
• Known hypersensitivity or contraindication to CT contrast agent.
• Pregnant or breastfeeding.
• Any condition that, in the opinion of the study team contraindicates participation in this study.

Where it is running

• National Institutes of Health Clinical Center — Bethesda, Maryland, United States (enrolling)
• The Children's Hospital of Philadelphia — Philadelphia, Pennsylvania, United States (enrolling)
• Texas Children's Hospital — Houston, Texas, United States (enrolling)

Full record on ClinicalTrials.gov

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