FGFR4-CAR T Cells for Rhabdomyosarcoma

Recruiting in Palo Alto (17 mi)

Overseen bySrivandana Akshintala, M.D.

Age: < 65

Sex: Any

Travel: May Be Covered

Time Reimbursement: Varies

Trial Phase: Phase 1

Waitlist Available

Sponsor: National Cancer Institute (NCI)

No Placebo Group

Trial Summary

What is the purpose of this trial?Background: Rhabdomyosarcoma (RMS) is a cancer of soft tissues. It is the most common soft tissue sarcoma seen in children. RMS cancer cells have a protein called FGFR4 on their surface. Researchers want to try a new kind of treatment for RMS: They will collect a person s own T cells, a type of immune cell; then they will change the T cells so they are better able to target the FGFR4 protein and attack RMS tumor cells. The modified T cells are chimeric antigen receptor (CAR) T cells. The treatment in this study is called FGFR4-CAR T cells. Objective: To test FGFR4-CAR T cells in children and young adults with RMS. Eligibility: People aged 3 to 39 years with RMS. The RMS must have failed to respond or returned after at least 2 rounds of standard treatment. Design: Participants will be screened. They will have physical exam, imaging scans, blood tests, and tests of their heart. They may have a tissue sample taken from their tumor. They will undergo apheresis: Blood will be taken from the body through a catheter. The blood will pass through a machine that separates out the T cells, and the remaining blood will be returned to the body. The collected T cells will be taken to a lab to create FGFR4-CAR T cells. Once the FGFR4-CART cells are ready, participants can receive these T cells. For 4 days they will receive drugs to prepare their body for the FGFR4-CAR T cells. After this, the modified T cells will be infused into a vein. Participants will be then monitored closely to watch for any side effects from the CART cells and be followed to see what effect the CART cells have on their tumors. They will have follow-up visits for up to 5 years. Long-term follow-up will be another 10 years.

Eligibility Criteria

This trial is for children and young adults aged 3 to 39 with Rhabdomyosarcoma (RMS) that hasn't improved or has come back after at least two standard treatments. Participants must have completed necessary screenings, including imaging scans, heart and nervous system tests, and possibly a tumor tissue sample.

Inclusion Criteria

Participants must have measurable disease per RECIST 1.1 or non-measurable disease on imaging

Individuals of child-bearing potential must agree to use highly effective contraception

Participants must be willing to be enrolled into protocol 15C0028 'Follow-Up Evaluation for Gene-Therapy Related Delayed Adverse Events after Participation in Pediatric Oncology Branch Clinical Trials' after 5 years on this trial

+11 more

Exclusion Criteria

Seropositive for human immunodeficiency virus (HIV) antibody

Seropositive for hepatitis C (HCV) or positive for Hepatitis B (HBV) surface antigen (HbsAg)

Pregnancy confirmed with beta-HCG serum or urine pregnancy test performed in IOCBP at screening

+6 more

Participant Groups

The study is testing FGFR4-CAR T cells therapy on RMS patients. It involves taking the patient's own T cells through apheresis, modifying them in a lab to target RMS more effectively, then infusing these engineered T cells back into the patient after pre-treatment drugs.

2Treatment groups

Experimental Treatment

Group I: Arm 2Experimental Treatment4 Interventions

Lymphodepleting preparative regimen (fludarabine and cyclophosphamide) followed by infusion of FGFR4-CAR T cells at the MTD

Group II: Arm 1Experimental Treatment4 Interventions

Lymphodepleting preparative regimen (fludarabine and cyclophosphamide) followed by infusion of FGFR4-CAR T cells escalation/de-escalation dose levels