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GPC2 CAR T cells

Phase 1

Refractory Neuroblastoma | Monoclonal antibody | Oncology |Gilead Sciences, Inc.|Last Updated: Dec 29, 2025

Success Probability

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Market & Valuation

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Trial Design

CONTROLLEDDMCBiomarker
Total Trials1
Total Enrollment45

FDA Designations

No designations recorded

Clinical trial landscape

GPC2 CAR T cells · 1 trial · 5 indications

Phase 1 1
NCT05650749GPC2 CAR T Cells for Relapsed or Refractory Neuroblastoma and Metastatic RetinoblastomaRefractory Neuroblastoma
RECRUITING45 Analytics
PHASE1RECRUITING
GPC2 CAR T Cells for Relapsed or Refractory Neuroblastoma and Metastatic Retinoblastoma
Refractory NeuroblastomaUnlock trial analytics

Study Endpoints

Primary Endpoints

Determine the Maximum Tolerated Dose of GPC2 CAR T cells
5 years

The Maximum Tolerated Dose of GPC2 CAR T cells will be determined by measuring the incidence of dose limiting toxicities following administration of the product.

Frequency of Adverse Events Following GPC2 CAR T cell administration
5 years

Assess the frequency and severity of treatment related adverse events following administration of GPC2 CAR T cells.

Secondary Endpoints

Manufacturing Feasibility of GPC2 CAR T cells
5 years
Persistence of GPC2 CAR T cells
5 years
Preliminarily define the clinical activity of GPC2 CAR T in patients with relapsed or refractory neuroblastoma or metastatic retinoblastoma
5 years
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Study Design & Arms

AllocationNON_RANDOMIZED
MaskingNONE
ModelSEQUENTIAL
PurposeTREATMENT

Treatment Arms

ArmTypeDescription
Dose Escalation ArmEXPERIMENTALThe dose escalation arm will determine the maximum tolerated dose of GPC2 CAR T cells using a standard 3+3 trial design.
Dose Expansion ArmEXPERIMENTALIf at least one dose from the dose expansion arm is determined to be safe, additional patients will be enrolled to the dose expansion arm to preliminarily evaluate the rate of response to GPC2 CAR T cells and further characterize the safety profile of GPC2 CAR T cells.

Interventions

NameTypeDescription
GPC2 CAR T cellsBIOLOGICALThe GPC2 CAR T investigational product is comprised of autologous human T cells that have been genetically modified to express a GPC2-targeting chimeric antigen receptor (CAR) transgene.
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Eligibility Criteria

Age Range1 Year to N/A
SexALL
Healthy VolunteersNo
Study Sites1

Neuroblastoma Inclusion Criteria: 1. Patients must be ≥ 1 year of age 2. Patients must have high-risk neuroblastoma according to COG risk classification at the time of study enrollment. Patients who were initially considered low- or intermediate-risk, but then reclassified as high-risk are also eli...

Countries:United States
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Frequently asked questions about GPC2 CAR T cells

What is GPC2 CAR T cells used for?

GPC2 CAR T cells is an investigational cell therapy being studied for the treatment of refractory neuroblastoma, a type of childhood cancer. It is also being evaluated in relapsed and high-risk neuroblastoma, as well as metastatic retinoblastoma. The therapy is currently in Phase 1 clinical development.

What does GPC2 CAR T cells target?

GPC2 CAR T cells targets the GPC2 protein, which is expressed on certain tumor cells. The therapy uses chimeric antigen receptor (CAR) T cells engineered to recognize and attack cells displaying GPC2. This targeted approach is designed to direct the immune system against cancer cells that carry this marker.

Who is developing GPC2 CAR T cells?

GPC2 CAR T cells is being developed by Gilead Sciences, Inc., a biopharmaceutical company traded on the NASDAQ under the ticker GILD. The therapy is currently in Phase 1 clinical trials for the treatment of refractory neuroblastoma and other related conditions.

What phase is GPC2 CAR T cells in?

GPC2 CAR T cells is in Phase 1 clinical development. It is an investigational therapy and has not yet been approved by regulatory authorities. The ongoing Phase 1 trial is actively recruiting participants to evaluate the safety and efficacy of this treatment in patients with refractory neuroblastoma.

What clinical trials is GPC2 CAR T cells in?

GPC2 CAR T cells is being studied in a Phase 1 clinical trial registered as NCT05650749. This trial is titled 'GPC2 CAR T Cells for Relapsed or Refractory Neuroblastoma and Metastatic Retinoblastoma' and is currently recruiting participants in the United States. The study aims to enroll 45 patients aged 1 year and older.

Is GPC2 CAR T cells the same as a monoclonal antibody?

GPC2 CAR T cells is a cell therapy, not a monoclonal antibody. It involves engineering a patient's T cells to express a chimeric antigen receptor that targets GPC2. This distinguishes it from antibody-based treatments, as it uses living cells to attack tumors rather than laboratory-made proteins.