Soquelitinib for Autoimmune Lymphoproliferative Syndrome

Recruiting at 2 trial locations

Overseen By V. Koneti Rao, M.D.

Age: Any Age

Sex: Any

Trial Phase: Phase 2

Sponsor: National Institute of Allergy and Infectious Diseases (NIAID)

Must not be taking: CYP3A inhibitors, P-gp inhibitors

Disqualifiers: Renal impairment, Liver impairment, HIV, others

No Placebo Group All trial participants will receive the active study treatment (no placebo)

Prior Safety Data This treatment has passed at least one previous human trial

Compensation: Varies

Number of Visits: 10

15 Participants Needed

Trial Summary

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.

What is the purpose of 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 (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.

Research Team

V. Koneti Rao, M.D.

Principal Investigator

National Institute of Allergy and Infectious Diseases (NIAID)

Eligibility Criteria

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

* INCLUSION CRITERIA

1. Aged \>= 16 years.

3. Has a documented diagnosis of ALPS-FAS.

Timeline

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

1 visit (in-person)

Treatment Stage 1

Participants receive 200 mg of soquelitinib twice daily for up to 360 days with monthly visits

52 weeks

8 visits (in-person), 6 visits (remote)

Interim Analysis

Safety and futility interim analysis conducted after 90 days

3 months

1 visit (in-person)

Treatment Stage 2

If stage 1 is successful, 6 new participants receive the same dosage for up to 360 days

52 weeks

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

Treatment Details

Interventions

• Soquelitinib

Trial Overview The study is testing Soquelitinib, an ITK inhibitor taken orally twice daily, aiming to reduce lymphocyte numbers and improve low blood cell counts in ALPS patients. The trial includes physical exams, blood tests, remote check-ins, and imaging scans over one year with a possible extension.

Participant Groups

Group I: Interventional Experimental Treatment 1 Intervention

The initial dosage for stage 1 of this study is 200 mg twice daily for up to 360 days. If stage 1 is repeated because there are no successes at this dosage, then it will be increased to 400 mg twice daily for up to 360 days. Alternatively, if there are safety concerns at either dosage, then it will be decreased to 100 mg twice daily for up to 360 days.

Who Is Running the Clinical Trial?

National Institute of Allergy and Infectious Diseases (NIAID)

Lead Sponsor

Trials

3,361

Recruited

5,516,000+