Engineered t cells take on childhood cancer in early trial
NCT ID NCT01822652
First seen Jun 27, 2026 · Last updated Jun 27, 2026
Summary
This early-phase trial tests a new type of immune therapy for children with neuroblastoma that has come back or not responded to treatment. Researchers take a patient's own T cells, add new genes to help them recognize and kill neuroblastoma cells and survive longer, then infuse them back. The study also uses chemotherapy and an additional drug (pembrolizumab) to boost the cells' effectiveness. The main goal is to find the safest dose and check for side effects.
What this could mean
Our plain-language read of the trial. This is informational only, not medical advice or a prediction.
- Active substance
- genetically modified T cells (iC9-GD2 T cells) plus chemotherapy (cyclophosphamide and fludarabine) and pembrolizumab
- What this could lead to
- If successful, this could point toward a more effective treatment for children with hard-to-treat neuroblastoma by helping immune cells survive longer and kill cancer cells.
- What could go wrong
- This is a very early, small phase 1 trial with only 11 participants, so results may not apply broadly. The modified cells could cause serious side effects, and the treatment may not shrink tumors.
This is an AI summary of the original study and may miss details. Read our disclaimer.
Study facts
What this study's own registry entry says, in plain language.
- Phase
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Phase 1
The first testing in people. Mainly checks safety and dose, usually in a small group.
- Participants
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11 people
The number who actually took part.
- Start date
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Aug 2013
- Expected to finish
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Oct 2030
An estimate. End dates often move.
- Lead sponsor
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Other sponsor
The registry's catch-all category, for sponsors it does not file as a company, a government agency, or a research network.
Who can take part
This study's own entry requirements. Only the study team can say for certain whether you qualify.
- Ages
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Children (under 18), adults (18 to 64) and older adults (65 and over)
- Sex
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Anyone
- Healthy volunteers
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Not accepted
This study is not open to healthy volunteers. The entry requirements below say who it is open to.
Show the full entry requirements Hide the full entry requirements
Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.
Inclusion Criteria: PROCUREMENT * High risk neuroblastoma with persistent or relapsed disease * Life expectancy of at least 12 weeks * Karnofsky/Lansky score of 60% or greater * Absence of HAMA prior to enrollment (only in patients that have been previously treated with murine antibodies) * Informed consent and assent (as applicable) obtained from parent/guardian and child TREATMENT: * High risk neuroblastoma with persistent or relapsed disease * Life expectancy of at least 12 weeks * Karnofsky/Lansky score of 60% or greater * Patients must have an ANC greater than or equal to 500, platelet count greater than or equal to 20,000 * Pulse Ox greater than or equal to 90% on room air * AST and ALT less than 5 times the upper limit of normal * Total bilirubin less than 3 times the upper limit of normal * Serum creatinine less than 3 times upper limit of normal. Creatinine clearance is needed for patients with creatinine greater than 1.5 times upper limit of normal * TSH normal for age. Patients using thyroid medication to facilitate a euthyroid state must be on a stable dose for at least 1 month prior to planned infusion * Recovered from acute effects of all prior chemotherapy. If some effects of therapy have become chronic (i.e., treatment associated thrombocytopenia), the patient must be clinically stable and meet all other eligibility criteria * Absence of human anti-mouse antibodies (HAMA) prior to enrollment for patients who have received prior therapy with murine antibodies * Patients must have autologous transduced activated T-cells with greater than or equal to 20% expression of GD2 * Pembrolizumab available for infusion * Informed consent and assent (as applicable) obtained from parent/guardian and child Exclusion Criteria: PROCUREMENT: * Rapidly progressive disease * History of hypersensitivity to murine protein containing products TREATMENT: * Rapidly progressive disease * Currently receiving other investigational drugs * History of hypersensitivity to murine protein containing products * History of cardiomegaly or bilateral pulmonary infiltrates on chest radiograph or CT. However, patients with cardiomegaly on imaging may be enrolled if they have an assessment of cardiac function (i.e., ECHO or MUGA) within 3 weeks of starting protocol therapy that is within normal limits. Additionally, patients with bilateral pulmonary infiltrates on imaging may be enrolled if the lesions are not consistent with active neuroblastoma (i.e., negative on functional imaging with PET or MIBG, or by pathologic assessment). * Evidence of tumor potentially causing airway obstruction * Patients who are pregnant, lactating, or unwilling to use birth control * Patients currently receiving immunosuppressive drugs such as corticosteroids, tacrolimus or cyclosporine * Patients previously experienced severe toxicity from cyclophosphamide or fludarabine * Severe previous toxicity from pembrolizumab or other PD-1 targeted antibody
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Conditions
The condition(s) this trial relates to.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.
Contacts and locations
Locations
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Houston Methodist Hospital
Houston, Texas, 77030, United States
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Texas Children's Hospital
Houston, Texas, 77030, United States
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Other studies related to the condition(s) this trial covers.
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