Recruiting
Phase 1

GD2-C7R T Cells

Sponsor:

Baylor College of Medicine

Code:

NCT04099797

Conditions

Diffuse Intrinsic Pontine Glioma

High Grade Glioma

Embryonal Tumor

Ependymal Tumor

Eligibility Criteria

Sex: All

Age: 1 - 25

Healthy Volunteers: Not accepted

Interventions

C7R-GD2.CART cells (IV and ICV infusion)

C7R-GD2.CART cells (IV and ICV infusion)

Study Details

Brief summary:

In this study, there are two treatment groups called Cohort 1 and Cohort 2. Cohort 1 is for patients with diffuse midline glioma, diffuse intrinsic pontine glioma, medulloblastoma, or another rare high-grade glioma that expresses GD2. Cohort 2 is for patients with a type of cancer called progressive diffuse intrinsic pontine glioma that expresses GD2.

Because there is no standard treatment at this time, patients are asked to volunteer in a gene transfer research study using special immune cells called T cells. T cells are a type of white blood cell that help the body fight infection.

This research study combines two different ways of fighting cancer: antibodies and T cells. Both antibodies and T cells have been used to treat cancer patients. They have shown promise but have not been strong enough to cure most patients.

Researchers have found from previous research that they can put a new antibody gene into T cells that will make them recognize cancer cells and kill them. GD2 is a protein found on several different cancers. Researchers testing brain cancer cells found that many of these cancers also have GD2 on their surface.

In a study for neuroblastoma in children, a gene called a chimeric antigen receptor (CAR) was made from an antibody that recognizes GD2. This gene was put into the patients own T cells and given back to 11 patients. The cells did grow for a while but started to disappear from the blood after 2 weeks. The researchers think that if T cells are able to last longer they may have a better chance of killing tumor cells.

In this study, a new gene will be added to the GD2 T cells that can potentially cause the cells to live longer. T cells need substances called cytokines to survive. The gene C7R has been added that gives the cells a constant supply of cytokine and helps them to survive for a longer period of time.

In other studies using T cells researchers found that giving chemotherapy before the T cell infusion can improve the amount of time the T cells stay in the body and therefore the effect the T cells can have. This is called lymphodepletion and it will allow the T cells to expand and stay longer in the body and potentially kill cancer cells more effectively.

After treating 11 patients, the largest safe dose of GD2-CAR T cells given in the vein (IV) was determined. We are now combining an IV infusion with an infusion directly into the brain through the Ommaya reservoir or programmable VP shunt. The goal is to find the largest safe dose of GD2-C7R T cells that can be administered in this way.

Patients will now be assigned to Cohort 1 and 2 based on their tumor type.

The GD2.C7R T cells are an investigational product not approved by the FDA.

The purpose of this study is to combine infusions into the vein in the first treatment cycle with infusions directly into the cerebrospinal fluid (CSF) in the brain (intracerebroventricularly) through the ommaya reservoir or programmable VP shunt for infusions cycles 2-24. The goal is to find the largest safe dose of GD2-C7R T cells that can be administered in this way, and additionally to evaluate how long they can be detected in the blood and CSF and what affect they have on brain cancer.

Conditions

Diffuse Intrinsic Pontine Glioma

High Grade Glioma

Embryonal Tumor

Ependymal Tumor

Study ID

NCT04099797

Start date

Feb 3, 2020

Status verified date

Jul, 2026

Completion date

Feb, 2041

Anticipated

Primary completion date

Feb, 2027

Anticipated

Eligibility Criteria

Eligibility Criteria

Sex: All

Age: 1 - 25

Healthy Volunteers: Not accepted

Procurement Inclusion Criteria:

Cohort 1:

1. Histologically confirmed, GD2-expressing newly diagnosed DMG/HGG (including pontine) or confirmation of H3K27 alteration if sufficient tissue for GD2 staining by IHC is not available. Newly diagnosed is defined as prior to radiographic progression or recurrence.

OR

Histologically confirmed, GD2-expressing recurrent, refractory, or progressive DMG/HGG (except pontine) or confirmation of positive H3K27 alteration if sufficient tissue for GD2 staining by IHC is not available.

OR

Recurrent, refractory, or progressive high-grade CNS tumor with confirmed GD2-expression. Examples include: medulloblastoma "CNS embryonal tumors, AT/RT, ependymal tumors, diffuse gliomas or glioneuronal tumors.

Cohort 2:

Recurrent, refractory, or progressive pontine HGG with confirmed GD2-expression or H3K27-altered DMG
2. Tumors less than 5 cm in maximum dimension at enrollment

1. Tumors with ≤25% increase in size (on any dimension) on MRI 4-8 weeks post-radiotherapy remain eligible for study
2. Tumors with >25% increase in size on post-radiation imaging may be reassessed with repeat MRI in 4-6 weeks, and are eligible if tumor size is subsequently ≤ 25% increased compared with pre-irradiation MRI.
3. Tumors with sizes between 5 and 5.5 cm are eligible if the tumor was surgically debulked
3. Measurable disease on at least 2 dimensions on MRI
4. Age 12 months to 25 years
5. Functional score (Karnofsky/Lansky) ≥ 50 expected at infusion (≥60 for cohort 2)

Procurement Exclusion Criteria:

1. Patients who are pregnant or breast feeding
2. Any patient with other risk factors for whom administration of investigational agent is deemed not in the patient's best interest, in the opinion of the investigator.

Treatment Inclusion Criteria

Cohort 1:

1. Histologically confirmed, GD2-expressing newly diagnosed DMG/HGG (including pontine) or confirmation of H3K27 alteration if sufficient tissue for GD2 staining by IHC is not available. Newly diagnosed is defined as prior to radiographic progression or recurrence.

OR

Histologically confirmed, GD2-expressing recurrent, refractory, or progressive DMG/HGG (except pontine) or confirmation of positive H3K27 alteration if sufficient tissue for GD2 staining by IHC is not available.

OR

Recurrent, refractory, or progressive high -grade CNS tumor with confirmed GD2-expression. Examples include: medulloblastoma, CNS embryonal tumors, AT/RT, ependymal tumors, diffuse gliomas, or glioneuronal tumors.

Cohort 2:

Recurrent, refractory, or progressive pontine H3K27-altered for DMG or HGG with confirmed GD2-expression.
2. Tumors less than 5 cm in maximum dimension at enrollment

1. Tumors with ≤25% increase in size (on any dimension) on MRI 4-8 weeks post-radiotherapy remain eligible for study
2. Tumors with >25% increase in size on post-radiation imaging may be reassessed with repeat MRI in 4-6 weeks, and are eligible if tumor size is subsequently ≤ 25% increased compared pre-irradiation MRI
3. Tumors with sizes between 5 and 5.5 cm are eligible if the tumor was surgically debulked
3. Measurable disease on at least 2 dimensions on MRI
4. Central line (PICC or other) and Ommaya reservoir or VP shunt in place or planned to be placed. Central line/PICC may be omitted for cycles that do not include lymphodepletion
5. Age 12 months to 25 years
6. Functional score (Karnofsky/Lansky) ≥ 50 (≥60 for cohort 2)
7. Patients must have completed standard of care radiation therapy at least 4 weeks prior to administration of investigational agent. If bevacizumab was administered for management of radiation necrosis, therapy must be completed at least 4 weeks prior to administration of investigational agent.
8. Stable neurologic exam for 7 days prior to enrollment
9. Stable or decreasing dose of steroids (max. allowable dose of dexamethasone is 0.1 mg/kg/day over the past 7 days prior to infusion of investigational therapy)
10. Organ function:

1. ANC > 1000 cells/ul
2. Platelet count > 100,000 cells/ul
3. Total bilirubin < 1.5x ULN
4. ALT and AST < 5x ULN
5. Serum creatinine or kidney within 2x ULN for age

Treatment Exclusion Criteria

1. Patients who received any other forms of immunotherapy ≤ 42 days before administration of investigational agent
2. Patients who received colony-stimulating factors within 14 days prior to administration of lymphodepletion
3. Patients receiving any concurrent anti-cancer therapy (treatment must occur at least three half-lives following prior anti-cancer therapy)
4. Patients who are pregnant or breast feeding
5. Any patient with other risk factors for whom administration of investigational agent is deemed not in the patient's best interest, in the opinion of the investigator.

Study Design

Enrollment

56 participants

Anticipated

Allocation

Non randomized

Intervention Model

Parallel Assignment

Primary purpose

Treatment

Interventions and Outcome Measures

Arms

experimental: C7R-GD2.CAR T cells (Cohort 1)

The dose level for autologous cell C7R-GD2.CAR T cells administered via intravenous (IV) infusion was determined in the initial phase of the protocol. The standard IV dose is 20 million cells/m2 with lymphodepletion chemotherapy.

In this subsequent phase of the study, the safe dosing levels for autologous cell C7R-GD2.CAR T cell immunotherapy administered intracerebroventricularly (ICV) via ommaya reservoir or programmable VP shunt will be determined.

experimental: C7R-GD2.CAR T cells (Cohort 2)

The dose level for autologous cell C7R-GD2.CAR T cells administered via intravenous (IV) infusion was determined in the initial phase of the protocol. The standard IV dose is 20 million cells/m2 with lymphodepletion chemotherapy.

In cycles 2-24, the safe dosing levels for autologous cell C7R-GD2.CAR T cell immunotherapy administered intracerebroventricularly (ICV) via ommaya reservoir or programmable VP shunt will be determined.

Interventions

C7R-GD2.CART cells (IV and ICV infusion)

Dose levels administered are by IV infusion followed by ICV infusion.

Cycle 1: 20 million cells/m2 delivered IV with lymphodepletion.

Cycle 2 (and subsequent cycles):

Dose level 1: 5 million cells ICV with lymphodepletion. Dose level 2: 10 million cells ICV with lymphodepletion. Dose level 3: 15 million cells ICV with lymphodepletion

C7R-GD2.CART cells (IV and ICV infusion)

Dose levels administered are by IV infusion followed by ICV infusion. Cycle 1: 20 million cells/m2 delivered IV with lymphodepletion.

Cycle 2 (and subsequent cycles):

Dose level 1: 5 million cells ICV with lymphodepletion. Dose level 2: 10 million cells ICV with lymphodepletion.

Dose level 3: 15 million cells ICV with lymphodepletion

Primary outcome measure

  • Dose limiting toxicity (DLT) rate [ Time Frame: 4 weeks post T cell infusion ]

Central Contacts and Locations

Central contacts

Locations

Texas Children's Hospital

Recruiting

Houston, Texas, United States, 77030

Contacts

More Information

Sponsor

Baylor College of Medicine

Last update posted

Aug 3, 2026

Last verified

Jul, 2026

Keywords

  • Gene Therapy
  • CAR T-cells
  • chimeric antigen receptor
  • Brain Cancer
  • Immunotherapy
  • Glioma
  • Brain tumor
  • DIPG
  • ETMRs
  • Medulloepithelioma
  • Atypical teratoid/rhabdoid tumors

Trial information was received from ClinicalTrials.gov and was last updated on 2026-09-10. This information was provided to ClinicalTrials.gov by Baylor College of Medicine on 2026-08-03.