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CAR T-cell Therapy

CAR T-Cell Therapy for Neuroblastoma and Osteosarcoma

Phase 1

Recruiting

Led By George Hucks, MD

Research Sponsored by UNC Lineberger Comprehensive Cancer Center

Eligibility Criteria Checklist

Specific guidelines that determine who can or cannot participate in a clinical trial

Must have

Adequate performance status as defined by Lansky or Karnofsky performance status of ≥ 60 (Lansky for <16 years of age)

First or greater relapse of neuroblastoma following completion of aggressive multi-drug frontline therapy

Must not have

Has a known additional malignancy that is active and/or progressive requiring treatment

Timeline

Screening 3 weeks

Treatment Varies

Follow Up 15 years

Awards & highlights

No Placebo-Only Group

Summary

This trial is testing a new cancer treatment that combines two existing ways of fighting disease: antibodies and T cells. Antibodies are molecules that fight infections and protect your body from diseases caused by bacteria and toxic substances. T cells are special infection-fighting blood cells that can kill other cells, including tumor cells or cells that are infected. The new treatment being researched is called autologous T lymphocyte chimeric antigen receptor cells (CAR) cells targeted against the disialoganglioside (GD2) antigen that express Interleukin (IL)-15, and the inducible caspase 9 safety switch (iC9

Who is the study for?

This trial is for people with specific cancers: relapsed/refractory neuroblastoma or osteosarcoma. Participants need a life expectancy of at least 12 weeks, have had previous aggressive treatment, and must not be pregnant or breastfeeding. They should not have hypersensitivity to the drugs used in the study or any other active malignancy requiring treatment.

What is being tested?

The trial tests a new therapy combining T cells (immune cells) and antibodies targeting GD2, a cancer cell marker, enhanced with IL-15 to boost effectiveness and an iCaspase9 safety switch. It's given after pre-treatment with Cyclophosphamide and Fludarabine to prepare the body.

What are the potential side effects?

Potential side effects include reactions related to immune response such as fever, fatigue, allergic reactions due to murine proteins in the CAR T-cells, and possible complications from preconditioning drugs like nausea or low blood counts.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below

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I can do most activities but may need help.

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My neuroblastoma has returned after completing initial intense treatment.

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My neuroblastoma worsened for the first time during initial intense treatment.

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I do not have any known brain or spinal cord diseases.

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My initial diagnosis was confirmed as neuroblastoma or ganglioneuroblastoma.

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I have high-risk neuroblastoma or osteosarcoma that's not responding to treatment.

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I do not need medication for seizures.

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My cancer's growth can be tracked with specific medical criteria.

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My neuroblastoma is high-risk and has not responded to treatment or has come back.

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My neuroblastoma hasn't fully responded after 4+ cycles of intense chemotherapy.

Exclusion Criteria

You may be eligible for the trial if you check “No” for criteria below:

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I have another cancer that is growing and needs treatment.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ 15 years

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~15 years

This trial's timeline: 3 weeks for screening, Varies for treatment, and 15 years for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.

Primary study objectives

Neuroblastoma

Secondary study objectives

Anti-tumor response rate to iC9.GD2.CAR.IL-15 t cell administration in pediatric subjects with relapsed or refractory neuroblastoma per Revised International Neuroblastoma Response Criteria (INCR) or relapsed/refractory osteosarcoma by RECIST v1.1

Expansion and persistence of iC9.GD2.CAR.IL-15 cells in vivo

Identify the maximum tolerated dose (MTD) of iC9.GD2.CAR.IL-15 T cells administered to pediatric subjects with relapsed or refractory neuroblastoma or relapsed/refractory osteosarcoma

+2 more

Awards & Highlights

No Placebo-Only Group

All patients enrolled in this study will receive some form of active treatment.

Trial Design

1Treatment groups

Experimental Treatment

Group I: iC9.GD2.CAR.IL-15 T-cellsExperimental Treatment3 Interventions

The continuous reassessment method (CRM) will be used to estimate the maximum-tolerated dose (MTD) of cells that to be given in dose escalation cohorts comprised of 2-6 subjects. The final MTD will be the dose with estimated probability of dose limiting toxicity (DLT) closest to the target toxicity rate of 20%. Three cell doses will be evaluated: 0.5 x 10\^6 cells/kg, 1.0 x 10\^6 cells/kg, 1.5 x 10\^6 cells/kg. Cohort enrollment will be staggered and each subject must complete at least 2 weeks of the cell treatment without incident of DLT before another subject can be enrolled at that dose level. A minimum of two subjects must complete the 4-week post-infusion DLT period before enrollment at the next higher dose level will be considered. If dose level 1 is determined to be above a tolerable dose, de-escalation would occur to dose level -1 where subjects would receive 0.25 x 10\^6 cells/kg.

Treatment

First Studied

Drug Approval Stage

How many patients have taken this drug

Cyclophosphamide

2010

Completed Phase 4

~2310

Fludarabine

2012

Completed Phase 4

~1860