Actively Recruiting
Immunotherapy for Malignant Pediatric Brain Tumors Using Adoptive Cellular Therapy (IMPACT) Phase 1 Study
Led by Children's National Research Institute · Updated on 2026-03-10
12
Participants Needed
1
Research Sites
104 weeks
Total Duration
On this page
AI-Summary
What this Trial Is About
Researchers are evaluating the safety and feasibility of a new immunotherapy approach using personalized tumor-specific cytotoxic T lymphocytes (TSA-T) in young patients with embryonal brain tumors and recurrent ependymoma. This open-label phase 1 study focuses on children under 5 years with newly diagnosed embryonal brain tumors and patients up to 30 years with recurrent ependymoma. The treatment targets personalized tumor antigens derived from each patient's tumor tissue to address the challenges of relapse after intensive chemotherapy and surgery. Participants will receive TSA-T infusions after completing standard treatment, which includes surgery, chemotherapy, stem cell rescue, and possible radiation for newly diagnosed patients, and surgery followed by re-irradiation and optional bridging chemotherapy for recurrent cases. TSA-T doses will escalate through planned dose levels, administered intracerebroventricularly via a Rickham reservoir. Patients may receive up to eight infusions spaced weeks apart, with dose adjustments depending on availability and response. During the study, participants will have regular evaluations including neurological exams, imaging, and laboratory tests to monitor safety, tumor response, and immune effects. Safety assessments focus on infusion-related adverse events over 42 days after the first infusion. Longer-term outcomes such as progression-free and overall survival will also be tracked for up to five years. Treatment adherence and clinical stability will be closely monitored throughout the participation period, which may last several months or longer depending on response and eligibility for continued infusions.
CONDITIONS
Brief Title
Immunotherapy for Malignant Pediatric Brain Tumors Employing Adoptive Cellular Therapy (IMPACT)
Who Can Participate
Eligibility Criteria
You may qualify if you...
- New diagnosis of CNS embryonal tumors including medulloblastoma, embryonal tumor with multilayered rosettes, pineoblastoma, atypical teratoid/rhabdoid tumor, or embryonal brain tumor NOS (Group A)
- Radiographic evidence of recurrent ependymoma with planned or recent re-resection (Group B)
- Age less than 5 years for Group A or between 1 and 30 years for Group B
- Availability or expectation of sufficient fresh or frozen tumor tissue
- Karnofsky or Lansky performance score of at least 60%
- Adequate organ function including ANC ≥750/μL, ALC >500/μL, platelets ≥75K, bilirubin ≤3x ULN, AST and ALT <5x ULN, serum creatinine ≤1.0 mg/dL or 1.5x ULN for age, and pulse oximetry >90% on room air
- For Group B females of childbearing potential, negative pregnancy test and agreement to use contraception through 6 months post-treatment
- Capability of providing informed consent (patient or guardian)
- Suitable size for mononuclear cell apheresis and stem cell rescue as applicable
- Medically eligible for surgical placement of a Rickham reservoir for intracerebroventricular therapy administration
- Stable neurologic exam with stable or decreasing steroid dose prior to treatment
- For patients with programmable shunts, able to tolerate shunt closure for at least 4 hours
You will not qualify if you...
- Uncontrolled infections
- Known HIV infection
- Medulloblastoma of the SHH subtype (Group A only)
- Fever above 38.0°C at procurement
- Prior investigational immunotherapy within 28 days before procurement
- Unable to tolerate apheresis or line placement
- Overly bulky tumors with herniation, significant midline shift, or brainstem involvement
- Progressive disease before subsequent infusions
- Recent use of immunosuppressive T cell antibodies within 28 days
- Current use of steroids at doses >0.05 mg/kg/day
- Presence of non-programmable ventriculoperitoneal shunts
- Recovery not complete from prior surgery or treatments as specified
- Pregnancy or inability to use contraception if applicable
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Your Study Journey
Duration - 2 to 4 weeks
Participants are screened for eligibility to participate in the trial.
1 visit (in-person)
Duration - Varies depending on treatment regimen
Participants receive standard treatment including surgery, chemotherapy, and/or radiation based on their group assignment before starting the experimental infusion therapy.
Multiple visits depending on treatment cycles and procedures
Duration - Up to several months depending on number of infusions
Participants receive multi-tumor antigen specific cytotoxic T lymphocyte infusions through a Rickham reservoir, with up to 8 infusions spaced at least 28 to 42 days apart depending on response and clinical status.
Up to 8 infusion visits spaced at least 28 to 42 days apart
Duration - Up to 5 years
Participants are monitored for safety, tumor response, and long-term outcomes after completion of the infusion treatment.
Regular visits as scheduled for ongoing assessments
Trial Site Locations
Total: 1 location
1
Children's National Hospital
Washington D.C., District of Columbia, United States, 20010
Actively Recruiting
Research Team
B
Brian Rood, MD
F
Fahmida Hoq, MBBS
How is the study designed?
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
NON_RANDOMIZED
Model
SINGLE_GROUP
Primary Purpose
TREATMENT
Number of Arms
2
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