CAR T Cells for Pediatric Solid Tumors

(IMPACT Trial)

Overseen By Andras Heczey, MD

Age: < 65

Sex: Any

Trial Phase: Phase 1

Sponsor: Seattle Children's Hospital

Must not be taking: Systemic steroids, Live vaccines

Disqualifiers: Organ transplant, HIV, Autoimmune, others

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

Compensation: Varies

Number of Visits: 1

Duration: 4 weeks

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial tests a new treatment called CAR T cells for children and young adults with difficult-to-treat solid tumors. The goal is to determine if these modified immune cells can safely and effectively combat tumors that have returned or are unresponsive to other treatments. Suitable candidates for this trial have a tumor that expresses a specific protein (GPC3) and have not succeeded with other therapies. This innovative treatment, specifically SC-CAR.GPC3xIL15.21 CAR T cells, involves creating a personalized therapy from a small blood sample to target cancer cells. As a Phase 1 trial, this research aims to understand how the treatment works in people, offering participants the chance to be among the first to receive this new therapy.

Do I need to stop my current medications for the trial?

The trial requires that you stop taking systemic steroids at least 24 hours before the CAR T cell infusion. Other medications are not specifically mentioned, so it's best to discuss your current medications with the trial team.

Is there any evidence suggesting that this treatment is likely to be safe for humans?

Research has shown that a new treatment, SC-CAR.GPC3xIL15.21 T cells, is being tested for certain solid tumors in children and young adults. These T cells are designed to target and attack cancer cells with a protein called GPC3 on their surface.

Studies are examining the types and frequency of side effects to assess safety. Researchers observe how often these side effects occur and their severity. As this is an early-stage trial, the primary focus is on understanding safety and treatment tolerance.

While detailed information on side effects for this specific treatment is not yet available, similar CAR T cell therapies have commonly shown side effects like fever, tiredness, and low blood cell counts. Clinical trials closely monitor these therapies to address any safety concerns promptly. ^{1 2 3 4 5}

Why do researchers think this study treatment might be promising?

Researchers are excited about SC-CAR.GPC3xIL15.21 CAR T cells because they use a novel approach to target pediatric solid tumors. Unlike traditional treatments like surgery, chemotherapy, or radiation, which can affect healthy cells too, this therapy specifically reprograms a patient’s own T cells to seek out and destroy cancer cells. This treatment leverages the power of a protein called GPC3, which is often found on the surface of these tumors, and combines it with an immune-boosting component, IL-15, to enhance the T cells' persistence and effectiveness. This targeted action not only promises greater precision in attacking cancer cells but also aims to reduce the side effects typically associated with conventional cancer therapies.

What evidence suggests that this treatment might be an effective treatment for pediatric solid tumors?

Research has shown that SC-CAR.GPC3xIL15.21 CAR T cells, the treatment under study in this trial, might be promising for treating solid tumors in children and young adults. These specially modified T cells target a protein called GPC3, found on the surface of some tumors. Early studies indicate that these cells can shrink tumors by attacking cancer cells more effectively. The added proteins, IL-15 and IL-21, help the T cells survive longer and fight cancer more efficiently. This approach also includes a safety feature called the iC9 suicide gene, which can be activated to destroy the T cells if necessary. Overall, this method aims to boost the body's ability to fight cancer while ensuring safety. ^{1 2 3 4 6}

Who Is on the Research Team?

Colleen Annesley, MD

Principal Investigator

Seattle Children's Hospital

Corinne Summers, MD

Principal Investigator

Seattle Children's Hospital

Are You a Good Fit for This Trial?

This trial is for pediatric and young adult patients with certain types of solid tumors that have not responded to previous treatments. These tumors must express a protein called glypican-3 (GPC3). Participants need to provide a blood sample for the creation of personalized CAR T cells.

Inclusion Criteria

Informed consent explained to, understood by and signed by patient/guardian

Adequate laboratory values

Sexually active patients must be willing to utilize one of the more effective birth control methods for 12 months after the T-cell infusion

Exclusion Criteria

I have not received any live vaccines in the last 30 days.

Active autoimmune or inflammatory disorder

I do not have any infections that aren't responding to treatment.

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

1 visit (in-person)

Treatment

Participants receive lymphodepleting chemotherapy followed by CAR T cell infusion

4 weeks

Multiple visits (in-person) for treatment and monitoring

Follow-up

Participants are monitored closely for side effects and response to treatment

4 weeks

Frequent visits (in-person) for monitoring

Long-term follow-up

Participants are followed for 15 years after CAR T cell infusion with decreasing visit frequency

What Are the Treatments Tested in This Trial?

Interventions

• SC-CAR.GPC3xIL15.21 CAR T cells

Trial Overview The study tests bioengineered T cells designed to target GPC3-positive tumors in children and young adults. These special T cells also produce proteins, IL-15 and IL-21, which may help them survive longer and fight cancer more effectively.

How Is the Trial Designed?

Group I: SC-CAR.GPC3xIL15.21 T cells Experimental Treatment 1 Intervention

Autologous SC-CAR.GPC3xIL15.21 T cell product will be infused as a single infusion.

Who Is Running the Clinical Trial?

Seattle Children's Hospital

Lead Sponsor

Trials

319

Recruited

5,232,000+

Citations

1. clinicaltrials.gov clinicaltrials.gov/study/NCT07148050

Study Details | NCT07148050 | Immunotherapy for Solid ... This research study will use these cells (named GPC3xIL15.21) to treat patients with solid tumors that express GPC3 on their surface. The ...

2. clinicaltrials.gov clinicaltrials.gov/study/NCT07224568

Cytokine Armored GPC3 Specific Chimeric Antigen ... This research study will use these cells (named GPC3xIL15.21) to treat patients with solid tumors that express GPC3 on their surface. The GPC3xIL15.21 CAR T ...

3. clin.larvol.com clin.larvol.com/trial-detail/NCT07148050

21 Armored Glypican-3-specific Chimeric Antigen Receptor ... To estimate anti-tumor responses by measuring changes in tumor burden using disease-specific evaluations following SC-CAR.GPC3xIL15.21 T cells. Standard ...

4. withpower.com withpower.com/trial/phase-1-hepatoblastoma-11-2025-2b2a7

CAR T Cells for Pediatric Solid Tumors (IMPACT Trial) These special T cells also produce proteins, IL-15 and IL-21, which may help them survive longer and fight cancer more effectively. Autologous SC-CAR. GPC3xIL ...

5. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC11030543/

Interleukin-15-armored GPC3-CAR T cells for patients with ... Interleukin-15 (IL15) promotes the survival of T lymphocytes and enhances the antitumor properties of CAR T cells in preclinical models of solid neoplasms ...

6. clinicaltrials.gov clinicaltrials.gov/study/NCT07224568?rank=5

Cytokine Armored GPC3 Specific Chimeric Antigen ... This research study will use these cells (named GPC3xIL15.21) to treat patients with solid tumors that express GPC3 on their surface. The ...