Soquelitinib for Autoimmune Lymphoproliferative Syndrome
What You Need to Know Before You Apply
What is the purpose of this trial?
This trial tests a new treatment called soquelitinib for individuals with Autoimmune Lymphoproliferative Syndrome (ALPS), a rare condition characterized by excessive production of immune cells, leading to symptoms such as swollen lymph nodes and an enlarged spleen. The trial aims to determine if soquelitinib can manage ALPS more effectively and with fewer side effects than current treatments. Participants should have ALPS with active symptoms like enlarged lymph nodes or spleen and must not have certain severe health conditions or infections. As a Phase 2 trial, this research measures how well soquelitinib works in an initial, smaller group of people, offering participants a chance to potentially benefit from a promising new treatment.
Will I have to stop taking my current medications?
The trial does not specify if you need to stop all current medications, but you cannot use certain drugs that affect liver enzymes (CYP3A inhibitors or inducers) or P-glycoprotein inhibitors unless they can be stopped before the trial starts.
Is there any evidence suggesting that soquelitinib is likely to be safe for humans?
Research has shown that soquelitinib is generally well-tolerated. In earlier studies with patients who have T cell disorders, soquelitinib was found to be safe. Some side effects were reported, but they were mostly mild or moderate. No serious side effects directly linked to the drug were noted in these studies.
Since this trial is in an early stage, researchers are closely monitoring its safety and how well participants tolerate it. This study will continue to assess the safety of soquelitinib for people with autoimmune lymphoproliferative syndrome (ALPS). The aim is to ensure any side effects are manageable and do not outweigh the possible benefits. Participants will have regular check-ups to monitor any symptoms or changes.12345Why do researchers think this study treatment might be promising?
Soquelitinib is unique because it targets autoimmune lymphoproliferative syndrome (ALPS) with a fresh approach. Unlike traditional treatments like immunosuppressants or corticosteroids, which broadly dampen the immune system, soquelitinib offers a more targeted action potentially reducing unwanted immune activity while sparing healthy cells. Researchers are excited about its mechanism that may provide a more effective and safer alternative by specifically addressing the root cause of the disorder. This precision could lead to fewer side effects and improved outcomes for patients with ALPS.
What evidence suggests that soquelitinib might be an effective treatment for ALPS?
Research has shown that soquelitinib might help people with Autoimmune Lymphoproliferative Syndrome (ALPS) by reducing the number of excess white blood cells. This reduction is crucial because too many of these cells can cause issues like swollen spleens and lymph nodes. Early studies suggest that soquelitinib blocks certain immune system actions, potentially helping to manage ALPS symptoms. Although more research is needed, the initial results are promising enough to warrant further study of soquelitinib in humans. Participants in this trial will receive varying dosages of soquelitinib to further evaluate its effectiveness and safety.13567
Who Is on the Research Team?
V. Koneti Rao, M.D.
Principal Investigator
National Institute of Allergy and Infectious Diseases (NIAID)
Are You a Good Fit for This Trial?
This trial is for individuals aged 16 and older diagnosed with Autoimmune Lymphoproliferative Syndrome (ALPS), a condition where the immune system stores too many lymphocytes, leading to enlarged spleen and lymph nodes. Participants must be able to attend clinic visits and perform self-monitoring.Inclusion Criteria
Timeline for a Trial Participant
Screening
Participants are screened for eligibility to participate in the trial
Treatment Stage 1
Participants receive 200 mg of soquelitinib twice daily for up to 360 days with monthly visits
Interim Analysis
Safety and futility interim analysis conducted after 90 days
Treatment Stage 2
If stage 1 is successful, 6 new participants receive the same dosage for up to 360 days
Follow-up
Participants are monitored for safety and effectiveness after treatment
What Are the Treatments Tested in This Trial?
Interventions
- Soquelitinib
Find a Clinic Near You
Who Is Running the Clinical Trial?
National Institute of Allergy and Infectious Diseases (NIAID)
Lead Sponsor