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New treatment option for Autoimmune Lymphoproliferative Syndrome
Official title Study of the ITK Inhibitor Soquelitinib to Reduce Lymphoproliferation and Improve Cytopenias in Autoimmune Lymphoproliferative Syndrome (ALPS)-FAS Patients
ClinicalTrials.gov ID: NCT06730126
What this study is testing
What is Soquelitinib?
Soquelitinib is an investigational medicine, given as an once-daily pill taken by mouth, being studied as a potential treatment for autoimmune lymphoproliferative syndrome.
Plain-language explanation of the investigational treatment - it is being studied and is not an approved or proven therapy. The study team can confirm the details.
- What it's testing
- 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.
- Phase 2: a mid-size study of how well it works
A plain-language read of the study's public ClinicalTrials.gov listing. The study team confirms the details.
Who can take part
Ages 16 to 120
You may be able to join if
- To be eligible to participate in this study and to re-enroll, an individual must meet all the following criteria:
- 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...
- 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.
You likely can't join if
- An individual who meets any of the following criteria will be excluded from participation or re-enrollment in this study:
- Profound grade 3 or 4 cytopenias that cannot be improved with immunomodulatory treatments prior to enrolling in the clinical trial and starting the...
- 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.
See the full eligibility criteria
- To be eligible to participate in this study and to re-enroll, an individual must meet all the following criteria:
- 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.
- 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.
- Participants who can become pregnant or who can impregnate their partner must agree to either remain sexually abstinent or use two highly effective methods of contraception when engaging in sexual activities that can...
- An individual who meets any of the following criteria will be excluded from participation or re-enrollment in this study:
- 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...
- 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.
- Current other invasive or systemic fungal, bacterial, or viral infection requiring therapy.
- History of opportunistic infection within the previous 180 days.
- History of invasive malignancy that required systemic therapy within the last 3 years.
- Current use of moderate or strong cytochrome P450 isozyme (CYP)3A inhibitors or inducers that cannot be stopped before day 0.
- Current use of P-glycoprotein (P-gp) inhibitors that cannot be stopped before day 0.
- Known hypersensitivity or contraindication to CT contrast agent.
- Pregnant or breastfeeding.
- Any condition that, in the opinion of the study team contraindicates participation in this study.
The study team makes the final eligibility decision.
Where it's taking place
- Bethesda, Maryland, United States
- Philadelphia, Pennsylvania, United States
- Houston, Texas, United States
Questions & answers
Do participants get paid in this trial?
This listing doesn't specify compensation. Many trials still reimburse travel or offer a stipend, so it's worth asking the study team when you connect.
Is it free to join, and do I need insurance?
Searching and applying through BridgeMD is free. In clinical trials the study-related treatment and visits are generally provided at no cost to you. You usually don't need insurance to take part - confirm specifics with the study team.
How long does this study last?
The listing doesn't state an exact length. The study team walks you through the schedule and number of visits before you decide to enroll.
Who can join this trial?
This study is enrolling all sexes, 16 years to 120 years. The study team makes the final eligibility decision.
Where is this trial taking place?
Study sites include Bethesda, Maryland, United States; Philadelphia, Pennsylvania, United States; Houston, Texas, United States. Enter your location above to see the nearest site and check your eligibility.
Explore other conditions
BridgeMD is an information and trial-matching tool - not medical advice, and not the study sponsor. Details come from ClinicalTrials.gov; the study team decides eligibility.