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CAR T-cell Therapy

GD2 CAR T Cells for Brain and Spinal Cord Gliomas

Phase 1

Recruiting

Led By Michelle Monje

Research Sponsored by Crystal Mackall, MD

Eligibility Criteria Checklist

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

Must have

Disease Status: Tissue diagnosis of H3K27M-mutated Diffuse Intrinsic Pontine Glioma (DIPG) with radiographically evident tumor restricted to the brainstem, OR Tissue diagnosis of H3K27M-mutated Diffuse Midline Glioma (DMG) of the spinal cord

Normal Organ and Marrow Function: Absolute neutrophil count (ANC) ≥ 1,000/uL, Platelet count ≥ 100,000/uL, Absolute lymphocyte count ≥ 150/uL, Hemoglobin ≥ 8 g/dL, Adequate renal, hepatic, pulmonary and cardiac function

Must not have

Prior GD2-antibody therapy

Tumor involvement of cerebellar vermis or hemispheres (pontocerebellar peduncle involvement is allowed), thalamic lesions, or supratentorial lesions

Timeline

Screening 3 weeks

Treatment Varies

Follow Up ime frame: day 28, 3 months, 6 months, 9 months and 12 months and 24 months post car t cell infusion

Awards & highlights

No Placebo-Only Group

Summary

This trial will test whether T cells can be successfully made from H3K27M-mutant DIPG or DMG patients, in order to better understand and treat these cancers.

Who is the study for?

This trial is for children and young adults aged 2 to 50 with H3K27M-mutant brainstem glioma (DIPG) or spinal cord glioma (DMG), post-radiation therapy, and without recent systemic treatments. US residents who can consent, practice birth control, have a negative pregnancy test if applicable, and have normal organ/marrow function are eligible. Exclusions include uncontrolled infections, certain tumor locations, ongoing corticosteroid use, significant medical conditions that could affect the study's outcome.

What is being tested?

The trial tests GD2-CAR T cells made from patients' immune cells against DIPG/DMG tumors in the brainstem or spinal cord. It checks if these CAR T cells can be created successfully and includes preparatory chemotherapy with Fludarabine and Cyclophosphamide before introducing the GD2-CAR T cells.

What are the potential side effects?

Potential side effects may include reactions related to immune cell infusion such as fever or chills; effects of chemotherapy like nausea or hair loss; increased risk of infection; fatigue; blood count changes leading to bruising or bleeding risks; and possible damage to organs due to an intense immune response.

Eligibility Criteria

Inclusion Criteria

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

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My cancer is a specific type that affects the brainstem or spinal cord and has a certain genetic mutation.

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My blood counts and organ functions are within normal ranges.

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I am over 16 and can care for myself but may not be able to do active work, or I am 16 or under and mostly active.

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I am between 2 and 50 years old.

Exclusion Criteria

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

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I have received GD2-antibody therapy before.

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My tumor affects specific brain areas but not the brain stem.

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I am currently on steroid medication.

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I have received CAR therapy before.

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I am not using any unapproved supplements, alternative therapies, or extreme diets.

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I have an autoimmune disease and haven't needed strong medication for it in the last 2 years.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ ime frame: day 28, 3 months, 6 months, 9 months and 12 months and 24 months post car t cell infusion

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~ime frame: day 28, 3 months, 6 months, 9 months and 12 months and 24 months post car t cell infusion

This trial's timeline: 3 weeks for screening, Varies for treatment, and ime frame: day 28, 3 months, 6 months, 9 months and 12 months and 24 months post car t cell infusion for reporting.

Treatment Details

Study Objectives

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

Primary study objectives

Maximum tolerated dose (MTD)/RP2D of GD2CART in subjects with H3K27M DIPG

Rate of successful manufacture of GD2CART using a retroviral vector in the Miltenyi CliniMACS Prodigy system

Safety of GD2CART in subjects with spinal H3 K27M-mutant DMG treated at the RP2D

Secondary study objectives

Measure resolution of toxicity

Overall Survival (OS)

Post-progression survival (PPS)

+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: GD2-CAR TExperimental Treatment3 Interventions

Rolling-6 dose escalation design will test GD2-CAR T cells in subjects with H3K27M-mutant DIPG. GD2CART will be administered in escalating doses on Day 0 in hospitalized subjects with either DIPG or spinal DMG Intravenously * Dose Level -1: 3x10\^5 transduced T cells/kg(± 20%) * Dose Level 1: 1x10\^6 transduced T cells/kg (± 20%) * Dose Level 2: 3x10\^6 transduced T cells/kg (± 20%) Intracerebroventricularly, without conditioning lymphodepletion chemotherapy * Dose Level -1: 10x10\^6 transduced T cells (±20%) * Dose Level 1: 30x10\^6 transduced T cells (±20%) * Dose Level 2: 50x10\^6 transduced T cells (±20%) * Dose Level 3: 100x10\^6 transduced T cells (±20%) Intracerebroventricularly after administration of conditioning lymphodepletion chemotherapy regimen with cyclophosphamide and fludarabine * Dose Level -1: 10x10\^6 transduced T cells (±20%) * Dose Level 1: 30x10\^6 transduced T cells (±20%) * Dose Level 2: 50x10\^6 transduced T cells (±20%)

Treatment

First Studied

Drug Approval Stage

How many patients have taken this drug

Fludarabine

2012

Completed Phase 4

~1860

Cyclophosphamide

2010

Completed Phase 4

~2310