Phase 2: testing Soquelitinib for Autoimmune Lymphoproliferative Syndrome
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.
Official registry: https://clinicaltrials.gov/study/NCT06730126 · National Institute of Allergy and Infectious Diseases (NIAID) · enrollment listed as 15
What this phase means
Phase 2: researchers already know a roughly safe dose (from phase 1). This stage asks whether the treatment actually helps the condition, and watches side effects more closely.
Phase labels are FDA/ICH categories applied to the registry Phase field. They are not a finding that this study succeeded.
What the registry says happens
If you join, the listed intervention(s) are Soquelitinib. The registry lists allocation as na. Background: Autoimmune lymphoproliferative syndrome (ALPS) is a rare disorder of the immune system caused by a mutation in the FAS gene.
They plan to enroll about 15 people at 3 sites. Taking part typically means taking a study drug (or dummy pill) plus check-ins.
Official title (unrewritten)
A Phase 2a Study of the ITK Inhibitor Soquelitinib to Reduce Lymphoproliferation and Improve Cytopenias in Autoimmune Lymphoproliferative Syndrome (ALPS)-FAS Patients
Interventions: Soquelitinib
Primary outcome (registry wording): Reduction of spleen volume or target lymph node volume by 25% from baseline to day 90, assessed by CT or PET/CT scan.
Who it is for (from inclusion criteria)
- Ages 16 Years to 120 Years
- To be eligible to participate in this study and to re-enroll, an individual must meet all the following criteria:
- 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
- If currently on corticosteroid therapy, then dose is less than 20 mg/day (prednisone equivalent) and has been stable for at least 4 weeks
- For participants to be seen at the NIH CC, co-enrolled on NIH protocol 93-I-0063
- 7a. Oral contraceptive pill or hormonal patch or ring
- 7b. Parenteral hormonal contraceptive implant
Who the registry excludes
- An individual who meets any of the following criteria will be excluded from participation or re-enrollment in this study:
- 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
Public study contact (from the registry)
Alanvin D Orpia, R.N.
Published on ClinicalTrials.gov. Contact them as a possible participant. They decide eligibility.
Sites listed on ClinicalTrials.gov
3 listed- National Institutes of Health Clinical Center — Bethesda, Maryland (recruiting)
- The Children's Hospital of Philadelphia — Philadelphia, Pennsylvania (recruiting)
- Texas Children's Hospital — Houston, Texas (recruiting)
FAQ about this NCT record
Is NCT06730126 still recruiting?
The sponsor posted status “Recruiting” on ClinicalTrials.gov at last ingest. Sites can pause independently. Confirm with the official record and the listed contact.
What phase is NCT06730126?
Phase 2. Phase labels are FDA/ICH categories applied to the registry Phase field. They are not a finding that this study succeeded.
Can PlainTrials tell me if I qualify for NCT06730126?
No. The inclusion and exclusion lists on this page are readable extracts of the public eligibility block. Only the study team can screen you.
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