Clinical trial · Interventional
Engineered Neuroblastoma Cellular Immunotherapy (ENCIT)-01
A Phase 1 Feasibility and Safety Study of Cellular Immunotherapy for Recurrent/Refractory Neuroblastoma Using Autologous T-cells Lentivirally Transduced to Express CD171-specific Chimeric Antigen Receptors
- Source
- ClinicalTrials.gov
- Retrieved
- Sep 8, 2026
- Layer
- normalized (units and labels harmonized; values unchanged)
- Run
- ING-CLINICALTRIALS-20260908-000001
Summary
Brief summary (as posted)
Patients with recurrent or refractory neuroblastoma are resistance to conventional chemotherapy. For this reason, the investigators are attempting to use T cells obtained directly from the patient, which can be genetically modified to express a chimeric antigen receptor (CAR). The CAR enables the T cell to recognize and kill the neuroblastoma cell through the recognition of CD171, a protein expressed of the surface of the neuroblastoma cell in patients with neuroblastoma. This is a phase 1 study designed to determine the maximum tolerated dose of the CAR+ T cells.
Conditions
Conditions (2)
Free-text conditions as registered, with the CancerIndex entity they were reconciled to and the match type.
| Condition (as posted) | Mapped entity | Match | Confidence |
|---|---|---|---|
| Ganglioneuroblastoma | Ganglioneuroblastoma | ONTOLOGY_EXACT | 0.90 |
| Neuroblastoma | Neuroblastoma | ONTOLOGY_EXACT | 0.90 |
Interventions
Interventions (3)
| Intervention | Type | Mapped drug | Match |
|---|---|---|---|
| Patient Derived CD171 specific CAR T cells expressing EGFRt (2nd generation T cells) | Biological | — | UNRESOLVED |
| Patient Derived CD171 specific CAR T cells expressing EGFRt (3rd generation T cells) | Biological | — | UNRESOLVED |
| Patient Derived CD171 specific CAR T cells expressing EGFRt (long spacer 2nd generation T cells) | Biological | — | UNRESOLVED |
Design
Arms and outcomes
Arms (3)
- type
- EXPERIMENTAL
- label
- A: 2nd Generation CE7R CAR T Cells
- description
- Autologous CD4 and CD8 cells are lentivirally transduced to generate patient-derived CD171 specific CAR T cells also expressing an EGFRt. Patients will receive lymphodepletion chemotherapy prior to T cell infusion. CD171 specific CAR T cells will be administered approximately 2-3 days after lymphodepletion chemotherapy. Cells will be administered approximately 1:1 CD4 and CD8 cells with planned dose level evaluations of total T cell dose of 1x10\^6 cells/kg, 5x10\^6 cells/kg, 1x10\^7 cells/kg, 5x10\^7 cells/kg, and 1x10\^8 cells/kg will be evaluated.
- interventionNames
- Biological: Patient Derived CD171 specific CAR T cells expressing EGFRt (2nd generation T cells)
- type
- EXPERIMENTAL
- label
- B: 3rd Generation CE7R CAR T Cells
- description
- Autologous CD4 and CD8 cells are lentivirally transduced to generate patient-derived CD171 specific CAR T cells also expressing an EGFRt. Patients will receive lymphodepletion chemotherapy prior to T cell infusion. CD171 specific CAR T cells will be administered approximately 2-3 days after lymphodepletion chemotherapy. Cells will be administered approximately 1:1 CD4 and CD8 cells with planned dose level evaluations of total T cell dose of 1x10\^6 cells/kg, 5x10\^6 cells/kg, 1x10\^7 cells/kg, 5x10\^7 cells/kg, and 1x10\^8 cells/kg will be evaluated.
Eligibility
Eligibility (as posted)
- Sex
- All
- Minimum age
- 18 Months
- Maximum age
- 26 Years
Show eligibility criteria text
Inclusion Criteria: * Prior diagnosis of NB or ganglioneuroblastoma either by histologic verification and/or demonstration of tumor cells in the bone marrow with increased catecholamine levels. * Male or female subjects ≤ 26 years of age * Diagnosis of high risk NB at initial diagnosis or if non-high risk at time of initial diagnosis must have had evidence of metastatic progression when \> 18 months of age. * Measurable or evaluable disease * Lansky or Karnofsky performance status score of ≥ 50 * Life expectancy of ≥ 8 weeks. * Recovered from significant acute toxic effects of all prior chemotherapy, immunotherapy, or radiotherapy prior to enrollment onto this study. * ≥ 7 days since last chemotherapy or biologic therapy administration * No systemic corticosteroids (unless physiologic replacement dosing) within 7 days of enrollment. Topical Administration (e.g. inhaled or dermatologic) is allowed. * ≥ 3 half-lives or 30 days from time of last dose of anti-tumor directed antibody therapy, whichever is shorter from time of enrollment * ≥ 6 weeks from myeloablative therapy and autologous stem cell transplant (timed from stem cell infusion). Patients who received stem cell infusion following non-myelo-ablative therapy are eligible once they meet all other eligibility requirements. Patient must NOT have received a prior allogeneic hematopoietic stem cell transplant. * No prior genetically modified cell therapy that is still detectable. * Must not be receiving external beam radiation therapy at the time of study enrollment. ≥ 12 weeks from prior I131 MIBG therapy. * Adequate organ function * Adequate laboratory values * Negative HIV antigen and antibody, Hepatitis B surface antigen and Hepatitis C antibody within 3 months prior to enrollment. For patients with positive Hepatitis C Ab, negative PCR testing must be documented in order to be eligible. Exclusion Criteria: * History of relevant CNS pathology or current relevant CNS pathology (non-febrile seizure disorder requiring ongoing anti-epileptic medications, paresis, aphasia, cerebrovascular ischemia/hemorrhage, severe brain injuries, dementia, cerebellar disease, organic brain syndrome, psychosis, coordination or movement disorder). Patients may have CNS intracranial tumor. * Pregnant or breast-feeding * Unable to tolerate apheresis procedure including placement of temporary apheresis catheter if necessary * Presence of active malignancy other than NB * Presence of known intracranial metastatic neuroblastoma. Skull based disease with soft tissue extension is allowed. * Presence of active severe infection * Presence of any concurrent medical condition that, in the opinion of the protocol PI or designee, would prevent the patient from undergoing protocol-based therapy. * Presence of a primary immunodeficiency/bone marrow failure syndrome * Receiving any other anti-cancer agents or radiotherapy at the time of study entry * Unwilling or unable to provide consent/assent for participation in the study and 15-year follow-up
References
Publications (0)
Data not yet available