Recruiting
Phase 2

Soquelitinib

Sponsor:

National Institute of Allergy and Infectious Diseases (NIAID)

Code:

NCT06730126

Conditions

Autoimmune Lymphoproliferative Syndrome

Eligibility Criteria

Sex: All

Age: 16 - 70+

Healthy Volunteers: Not accepted

Interventions

Soquelitinib

Study Details

Brief summary:

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....

Conditions

Autoimmune Lymphoproliferative Syndrome

Study ID

NCT06730126

Start date

Mar 10, 2025

Status verified date

Jun 9, 2026

Completion date

Dec 1, 2026

Anticipated

Primary completion date

Dec 1, 2026

Anticipated

Eligibility Criteria

Eligibility Criteria

Sex: All

Age: 16 - 70+

Healthy Volunteers: Not accepted

  • 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 their behalf (for ages <18 years).
3. Has a documented diagnosis of ALPS-FAS.
4. Has clinical evidence of active disease, defined as at least one enlarged lymph node and/or enlarged spleen.
5. If currently on corticosteroid therapy, then dose is less than 20 mg/day (prednisone equivalent) and has been stable for at least 4 weeks.
6. For participants to be seen at the NIH CC, co-enrolled on NIH protocol 93-I-0063.
7. 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 result in pregnancy, beginning 28 days before baseline until 3 months after the last dose. One method must be a barrier (eg, internal or external condom, cervical cap, or diaphragm). The second method may be any of the following:

7a. Oral contraceptive pill or hormonal patch or ring.

7b. Parenteral hormonal contraceptive implant.

7c. Intrauterine device.

EXCLUSION CRITERIA:

An individual who meets any of the following criteria will be excluded from participation or re-enrollment in this study:

1. 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 may be included if the dose is stable for 12 weeks at screening and is not expected to confer additive toxicity.
2. 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.
3. Liver impairment, defined as bilirubin, alanine aminotransferase, or aspartate aminotransferase greater than 2.5 times the upper limit of normal.
4. History of EBV-associated lymphoma.
5. Active EBV infection with EBV load >300 copies/mL.
6. Tuberculosis infection (active or latent) or undergoing tuberculosis treatment.
7. Infection with HIV or hepatitis B or C.
8. Current other invasive or systemic fungal, bacterial, or viral infection requiring therapy.
9. History of opportunistic infection within the previous 180 days.
10. History of invasive malignancy that required systemic therapy within the last 3 years.
11. Current use of moderate or strong cytochrome P450 isozyme (CYP)3A inhibitors or inducers that cannot be stopped before day 0.
12. Current use of P-glycoprotein (P-gp) inhibitors that cannot be stopped before day 0.
13. Known hypersensitivity or contraindication to CT contrast agent.
14. Pregnant or breastfeeding.
15. Any condition that, in the opinion of the study team contraindicates participation in this study.

Study Design

Enrollment

15 participants

Anticipated

Intervention Model

Single group

Primary purpose

Treatment

Interventions and Outcome Measures

Arms

experimental: Interventional

The dosage for this study is 200 mg twice daily for up to 360 days. If the participant repeats they study they will receive the same dosage for up to an additional 360 days.

Interventions

Soquelitinib

Soquelitinib is an ITK inhibitor in clinical development for treating relapsed/refractory T-cell lymphoma.

Primary outcome measure

  • Reduction of spleen volume or target lymph node volume by 25% from baseline to day 90, assessed by CT or PET/CT scan. [ Time Frame: Day 90 ]

Central Contacts and Locations

Central contacts

Locations

National Institutes of Health Clinical Center

Recruiting

Bethesda, Maryland, United States, 20892

Contacts

The Children's Hospital of Philadelphia

Recruiting

Philadelphia, Pennsylvania, United States, 19104

Contacts

Texas Children's Hospital

Recruiting

Houston, Texas, United States, 77030

Contacts

More Information

Sponsor

National Institute of Allergy and Infectious Diseases (NIAID)

Last update posted

Sep 1, 2026

Last verified

Jun 9, 2026

Keywords

  • Autoimmune Lymphoproliferative Syndrome
  • Fas
  • Hypersplenism
  • Lymphoproliferation
  • Interleukin-2 Inducible T-cell Kinase
  • Soquelitinib
  • Targeted Treatment

Trial information was received from ClinicalTrials.gov and was last updated on 2026-09-09. This information was provided to ClinicalTrials.gov by National Institute of Allergy and Infectious Diseases (NIAID) on 2026-09-01.