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Engineered immune cells take aim at childhood cancer

NCT ID NCT02311621

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 Results may not be published yet.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This status has not been confirmed recently, so it may be out of date.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-stage trial tests a new approach for children and young adults with neuroblastoma that has come back or not responded to standard treatments. Doctors take a patient's own immune cells (T-cells), genetically modify them in the lab to recognize and attack a protein called CD171 found on neuroblastoma cells, and then infuse them back into the patient. The main goal is to find a safe dose and watch for side effects, while also checking if the treatment can shrink tumors.

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 (CAR T-cells) targeting CD171
What this could lead to
If successful, this could lead to a new treatment option for children with hard-to-treat neuroblastoma that has not responded to standard therapy.
What could go wrong
This is an early phase 1 trial focused on safety and dosing, so it is too soon to know if it will work. There are risks of serious side effects from the modified cells, 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

About 65 people

The number the study aims to enrol. It can still change while the study runs.

Started

Nov 2014

Expected to finish

Nov 2038

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

18 months to 26 years

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: * 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

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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

  • Seattle Children's Hospital

    Seattle, Washington, 98105, United States

More trials for these conditions

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