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Engineered t cells take on childhood cancer in early trial

NCT ID NCT01822652

What the study statuses mean

This study's is highlighted.

Recruitment status, easiest to join first

Recruiting now
This trial is taking on new participants right now.
Not yet recruiting
Registered, but not yet taking participants.
By invitation only
Not open to general applications. Only people the study team invites can take part.
Paused
Paused for now. It may or may not start again.
Ongoing This study
Running, but no longer taking on new participants.
Completed
The trial has finished. Results may not be published yet.
Stopped early
Stopped early, before it reached the end. That can be for many reasons, including safety.
Cancelled
Cancelled before anyone took part.

Expanded access (not trials)

Expanded access
Not a trial. This treatment can be requested outside a study, case by case, for people who qualify.
Expanded access (paused)
Not a trial. The treatment can normally be requested outside a study, but is unavailable right now.
Expanded access (ended)
Not a trial. The treatment could once be requested outside a study, but no longer can.
Approved
The treatment has been approved, so it is available normally rather than through this programme.

When the status isn't known

Details not published
The full record has not been published yet, so there is little to show here.
Status unknown
This status has not been confirmed recently, so it may be out of date.

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

Phase 1

The first testing in people. Mainly checks safety and dose, usually in a small group.

Participants

11 people

The number who actually took part.

Start date

Aug 2013

Expected to finish

Oct 2030

An estimate. End dates often move.

Lead sponsor

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

Children (under 18), adults (18 to 64) and older adults (65 and over)

Sex

Anyone

Healthy volunteers

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

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.

neuroblastoma Recurrence

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

Contacts and locations

Locations

  • Houston Methodist Hospital

    Houston, Texas, 77030, United States

  • Texas Children's Hospital

    Houston, Texas, 77030, United States

More trials for these conditions

Other studies related to the condition(s) this trial covers.