New treatment using engineered T cells for pediatric solid tumors
Interleukin-15 Armored Glypican-3-specific Chimeric Antigen Receptor Expressing Autologous T Cells as Immunotherapy for Children With Solid Tumors
This study is testing a new treatment using specially modified immune cells to see if it can help children and young adults with certain hard-to-treat tumors.
Quick facts
| Phase | Phase 1 |
|---|---|
| Study type | Interventional |
| Enrollment | 24 (estimated) |
| Ages | 1 Year to 21 Years |
| Sex | All |
| Sponsor | Baylor College of Medicine Academic / other |
| Drugs / interventions | CAR T, chemotherapy, prednisone, chimeric antigen receptor, cyclophosphamide, fludarabine, Cytoxan |
| Locations | 1 site (Houston, Texas) |
| Trial ID | NCT04377932 on ClinicalTrials.gov |
What this trial studies
This clinical trial investigates a novel approach to treating pediatric solid tumors by using genetically engineered T cells that target Glypican 3 (GPC3), a protein found on certain cancer cells. Participants will undergo lymphodepletion chemotherapy to prepare their immune system before receiving the engineered T cells. The study aims to evaluate the safety and effectiveness of this treatment in children and young adults with GPC3-positive tumors that have not responded to standard therapies. Approximately 15-24 subjects will be enrolled to assess the potential benefits of this innovative therapy.
Who should consider this trial
Good fit: Ideal candidates are children and young adults aged 1 to 21 with GPC3-positive solid tumors.
Not a fit: Patients with a history of hypersensitivity to murine proteins or active infections may not benefit from this study.
Why it matters
Potential benefit: If successful, this treatment could provide a new option for children with difficult-to-treat solid tumors.
How similar studies have performed: Previous studies using CAR T cell therapies have shown promise, indicating potential for success with this novel approach.
Eligibility criteria
Show full inclusion / exclusion criteria
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 * Life expectancy of ≥ 16 weeks * Lansky or Karnofsky score ≥60% * 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 * serum AST\< 5 times ULN * total bilirubin \< 3 times ULN for age * INR ≤1.7 (for patients with hepatocellular carcinoma only) * absolute neutrophil count \> 500/microliter * platelet count \> 25,000/microliter (can be transfused but must be at that level prior to treatment) * Hgb ≥7.0 g/dl (can be transfused but must be at that level prior to treatment) * pulse oximetry \>90% on room air * Refractory or relapsed disease after treatment with up- front therapy and at least one salvage treatment cycle * Recovered from acute toxic effects of all prior chemotherapy and investigational agents before entering this study * Sexually active patients must be willing to utilize one of the more effective birth control methods for 3 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 or Hepatitis C virus infections) * 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 this trial is running
Houston, Texas
- Texas Children's Hospital — Houston, Texas, United States (Recruiting)
Study contacts
- Principal investigator: Andras Heczey, MD — Baylor College of Medicine
- Study coordinator: Andras Heczey
- Email: axheczey@txch.org
- Phone: (832) 824-4233
How to participate
- Review the eligibility criteria above with your treating physician.
- Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
- Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.