Using engineered T cells to treat pediatric solid tumors

Is this trial a fit for you?

A plain-English reading of the study's rules. Only the study team can confirm whether you qualify.

What this trial studies

This clinical trial investigates a novel treatment for pediatric patients with GPC3-positive solid tumors, including liver cancer and rhabdomyosarcoma, by utilizing genetically modified T cells known as CAR T cells. The study involves collecting blood from participants to grow these T cells, which are then engineered to target and kill cancer cells. Prior to the infusion of these CAR T cells, participants will undergo lymphodepletion chemotherapy to enhance the effectiveness of the treatment. The trial aims to assess the safety and efficacy of this approach in a small group of patients.

Why it matters

Who can join: the full rules

Procurement Eligibility

Inclusion Criteria:

* Diagnosis of GPC3-positive\* solid tumors (as determined by immunohistochemistry with an extent score of \>=Grade 2 \[\>25% positive tumor cells\] and an intensity score of \>= 2 \[scale 0-4\]).
* Age ≥1 year and ≤ 21 years
* Lansky or Karnofsky score ≥60%
* Life expectancy ≥16 weeks
* Barcelona Clinic Liver Cancer Stage A, B or C (for patients with hepatocellular carcinoma only)
* Child-Pugh-Turcotte score \<7 (for patients with hepatocellular carcinoma only)
* Informed consent explained to, understood by and signed by patient/guardian. Patient/guardian given copy of informed consent

Exclusion Criteria:

* History of hypersensitivity reactions to murine protein-containing products OR presence of human anti-mouse antibody (HAMA) prior to enrollment (only patients who have received prior therapy with murine antibodies).
* History of organ transplantation
* Known HIV positivity
* Active bacterial, fungal or viral infection (except Hepatitis B or Hepatitis C virus infections)

Treatment Eligibility

Inclusion Criteria:

* Age ≥ 1 year and ≤ 21 years
* Barcelona Clinic Liver Cancer Stage A, B or C (for patients with hepatocellular carcinoma only)
* Lansky or Karnofsky score ≥ 60%
* Child-Pugh-Turcotte score \< 7 (for patients with hepatocellular carcinoma only)
* Adequate organ function:
* Creatinine clearance as estimated by Cockcroft Gault or Schwartz ≥ 60 ml/min
* Total bilirubin \< 3 times ULN for age
* INR ≤1.7 (for patients with hepatocellular carcinoma only)
* Absolute neutrophil count \> 750/µl
* Platelet count \> 75,000/µl (Needs to be confirmed prior to treatment whether with or without transfusion)
* Hgb ≥ 8.0 g/dl (Needs to be confirmed prior to treatment whether with or without transfusion)
* Pulse oximetry ≥ 92% on room air
* Incurable disease after treatment with up- front therapy (Patients who have relapsed disease despite a standard of care salvage therapy)
* Wash out period, such that patient has recovered from acute toxic effects of all prior chemotherapy and investigational agents before entering this study, and returned to their clinical baseline, as determined by history and physical exam.
* Sexually active patients must be willing to utilize one of the more effective birth control methods for 6 months after the T-cell infusion.
* Informed consent explained to, understood by and signed by patient/guardian. Patient/guardian given copy of informed consent

Exclusion Criteria:

* Pregnancy or lactation
* Uncontrolled infection
* Systemic steroid treatment (greater than or equal to 0.5 mg prednisone equivalent/kg/day, dose adjustment or discontinuation of medication must occur at least 24 hours prior to CAR T cell infusion)
* Known HIV positivity
* Active bacterial, fungal or viral infection \[except Hepatitis B (HBV patients with active disease who meet the criteria for anti-HBV therapy should be on a suppressive antiviral therapy prior to initiation of cancer therapy) or Hepatitis C virus infections (should have completed curative antiviral treatment with HCV viral load below the limit of quantification\]
* Congestive heart failure (as defined by New York Heart Association Functional Classification III or IV), unstable angina, serious uncontrolled cardiac arrhythmia, a myocardial infarction within 6 months prior to study entry or a history of myocarditis
* Active autoimmune or inflammatory disorder
* Live vaccines within 30 days prior to enrollment
* History of organ transplantation
* History of hypersensitivity reactions to murine protein-containing products OR presence of human anti-mouse antibody (HAMA) prior to enrollment (only patients who have received prior therapy with murine antibodies)

Where it's running

• Texas Children's Hospital
  Houston, Texas, United States Recruiting

Study contacts

• Principal investigator: David Steffin, MD — Baylor College of Medicine
• Study coordinator: David Steffin
• Email: dhsteffi@texaschildrens.org
• Phone: (832) 824-4233

Similar studies

Other studies for the same condition or treatment. Recruiting studies are listed first.

• GPC3-targeted CAR T cells armored with IL-15 and IL-21 for pediatric non‑CNS solid tumors

  This treatment will try personalized CAR T cells that target GPC3 and are engineered to make IL‑15 and IL‑21 in children and young adults with relapsed or refractory non‑CNS solid tumors that express GPC3.

  Recruiting Phase 1 Seattle Children's Hospital · Seattle, Washington · NCT07148050

• IL-15 and IL-21–armored GPC3-CAR T cell therapy for adults with GPC3-positive solid tumors

  This trial tests whether CAR T cells engineered to target GPC3 and fortified with IL-15 and IL-21 can treat adults with GPC3-positive liver and other solid tumors.

  Recruiting Phase 1 Baylor College of Medicine · Houston, Texas · NCT06198296

• Becotatug Vedotin for EGFR-positive relapsed, refractory, or metastatic pediatric solid tumors

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  Recruiting Phase 1 Sun Yat-sen University · Guangzhou, Guangdong · NCT07584499

• Personalized B7-H3 CAR T cells combined with PRAME antigen-specific T cells for relapsed embryonal tumors

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  Recruiting Phase 1 Children's National Research Institute · Washington D.C., District of Columbia and 1 other locations · NCT07172958

• FGFR4-directed CAR T cell therapy for children and young adults with recurrent or refractory rhabdomyosarcoma

  This trial will try a personalized CAR T cell treatment that targets the FGFR4 protein for people aged 3–39 whose rhabdomyosarcoma has returned or not responded after at least two prior treatments.

  Recruiting Phase 1 National Institutes of Health Clinical Center (CC) · Bethesda, Maryland · NCT06865664

• Alpha/beta T‑Cell and B‑Cell Depletion plus Zoledronic Acid for Pediatric and Young Adult Solid Tumors

  This study will try removing alpha/beta T cells and B cells from donor stem cell grafts and giving zoledronic acid to see if it helps children and young adults (6 months to 25 years) with relapsed or high‑risk solid tumors tolerate allogeneic transplant and reduce relapse.

  Recruiting Phase 1/2 University of Florida · Gainesville, Florida · NCT06625190

How to take part

1. Review the eligibility criteria above with your treating physician.
2. Check the official listing on ClinicalTrials.gov for the latest contact details and recruitment status.
3. Contact the study coordinator or principal investigator to ask for pre-screening. Pre-screening is free and never obligates you to enroll.