Actively Recruiting
Study of the ITK Inhibitor Soquelitinib to Reduce Lymphoproliferation and Improve Cytopenias in Autoimmune Lymphoproliferative Syndrome (ALPS)-FAS Patients
Led by National Institute of Allergy and Infectious Diseases (NIAID) · Updated on 2026-05-05
15
Participants Needed
3
Research Sites
90 weeks
Total Duration
On this page
AI-Summary
What this Trial Is About
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.
CONDITIONS
Official Title
Study of the ITK Inhibitor Soquelitinib to Reduce Lymphoproliferation and Improve Cytopenias in Autoimmune Lymphoproliferative Syndrome (ALPS)-FAS Patients
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Aged 16 years or older
- Able to provide informed consent or have a parent/guardian provide permission if under 18
- Documented diagnosis of ALPS-FAS
- Evidence of active disease with at least one enlarged lymph node or enlarged spleen
- If on corticosteroids, dose less than 20 mg/day prednisone equivalent and stable for 4 weeks
- For NIH CC participants, co-enrollment on NIH protocol 93-I-0063
- Agreement to use two effective contraception methods or remain abstinent if able to become pregnant or impregnate a partner, starting 28 days before baseline until 3 months after last dose
You will not qualify if you...
- Severe grade 3 or 4 cytopenias that cannot be improved before study
- Kidney impairment with serum creatinine over 1.5 mg/dL or low filtration rate
- Liver impairment with elevated bilirubin or liver enzymes over 2.5 times normal
- History of EBV-associated lymphoma
- Active EBV infection with high viral load
- Active or latent tuberculosis or tuberculosis treatment
- Infection with HIV or hepatitis B or C
- Current serious fungal, bacterial, or viral infection needing treatment
- History of opportunistic infection within 180 days
- Recent invasive cancer requiring systemic therapy within 3 years
- Current use of certain CYP3A or P-glycoprotein inhibitors that cannot be stopped
- Allergy or contraindication to CT contrast agent
- Pregnant or breastfeeding
- Any condition the study team feels makes participation unsafe
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Trial Site Locations
Total: 3 locations
1
National Institutes of Health Clinical Center
Bethesda, Maryland, United States, 20892
Actively Recruiting
2
The Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, United States, 19104
Actively Recruiting
3
Texas Children's Hospital
Houston, Texas, United States, 77030
Actively Recruiting
Research Team
A
Alanvin D Orpia, R.N.
CONTACT
V
V. Koneti Rao, M.D.
CONTACT
How is the study designed?
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
NA
Model
SINGLE_GROUP
Primary Purpose
TREATMENT
Number of Arms
1
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