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.
Aged >= 16 years.
Able to provide informed consent (for ages >= 18 years) or has a parent(s) or guardian(s) who can provide permission to participate on their behalf (for ages <18 years).
Has a documented diagnosis of ALPS-FAS.
Has clinical evidence of active disease, defined as at least one enlarged lymph node and/or enlarged spleen.
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 m².
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.
Single group
Soquelitinib
DrugSoquelitinib is an ITK inhibitor in clinical development for treating relapsed/refractory T-cell lymphoma.
Reduction of spleen volume or target lymph node volume by 25% from baseline to day 90, assessed by CT or PET/CT scan.
To determine the efficacy of soquelitinib in reducing spleen volume or target lymph node volume in people with ALPS-FAS.
Shrinkage in spleen volume from baseline to day 90.
To determine the efficacy of soquelitinib in improvement in lymphoproliferation and autoimmune disease, including cytopenias, in people with ALPS-FAS.
Shrinkage in target lymph node volume from baseline to day 90.
To determine the efficacy of soquelitinib in improvement in lymphoproliferation and autoimmune disease, including cytopenias, in people with ALPS-FAS.
Reduction of cytopenias by one severity level from baseline to day 90
To determine the efficacy of soquelitinib in improvement in lymphoproliferation and autoimmune disease, including cytopenias, in people with ALPS-FAS and to determine the safety and tolerability of soquelitinib in people with ALPS-FAS.
Reduction in prednisone dosage.
To determine the efficacy of soquelitinib in improvement in lymphoproliferation and autoimmune disease, including cytopenias, in people with ALPS-FAS
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