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

Conditions

• Autoimmune Lymphoproliferative Syndrome

Eligibility Criteria

Sex

ALL

Age

16 Years - 120 Years

Healthy Volunteers

Not accepted

Interventions

• Soquelitinib — DRUG
  Soquelitinib is an ITK inhibitor in clinical development for treating relapsed/refractory T-cell lymphoma.

Study Details

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 Dates

Start date

Mar 10, 2025

Status verified

Jun 2026

Primary completion

Dec 1, 2026

Completion

Dec 1, 2026

Study Design

Enrollment

15 participants (estimated)

Allocation

NA

Intervention model

SINGLE_GROUP

Primary purpose

TREATMENT

Arms

• Experimental: Interventional
  The dosage for this study is 200 mg twice daily for up to 360 days. If the participant repeats they study they will receive the same dosage for up to an additional 360 days.

Primary Outcome Measure

Reduction of spleen volume or target lymph node volume by 25% from baseline to day 90, assessed by CT or PET/CT scan. [ Time Frame: Day 90 ]

Central Contacts

• Alanvin D Orpia, R.N.
  (240) 550-3663
  [email protected]
• V. Koneti Rao, M.D.
  (301) 496-6502
  [email protected]

Locations (3)

Find similar trials in Bethesda, MD