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

Recruiting Phase 2 Interventional Study
Autoimmune Lymphoproliferative Syndrome
No Placebo Group Every participant receives an active treatment — no one gets a placebo. Prior Safety Data This treatment has already been tested in at least one earlier human trial.
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At a Glance
Age
16 – 120
Sex
Any
Trial phase
Phase 2
Study type
Interventional
Purpose
Treatment
Participants needed
15 (estimated)
Sponsor
National Institute of Allergy and Infectious Diseases (NIAID) · NIH
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About This Trial
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 (so…
Trial Locations
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Eligibility Criteria
* INCLUSION CRITERIA: To be eligible to participate in this study and to re-enroll, an individual must meet all the following criteria: 1. Aged \>= 16 years. 2. Able to provide informed consent (for ages \>= 18 years) or has a parent(s) or guardian(s) who can provide permission to participate on t…
Contacts

Alanvin D Orpia, R.N.

(240) 550-3663

alanvin.orpia@nih.gov

CONTACT

V. Koneti Rao, M.D.

(301) 496-6502

kr191c@nih.gov

CONTACT