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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
Approval Probability 71%
TA Base Rate26%
Adjusted LOA41%
ML RiskLOW_RISK
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Market & Valuation
rNPV $3.2B
Market Size $9.4B
Revenue Basis $1.6B
Competitors 6
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Trial Design
CONTROLLEDDMCBiomarker
Total Trials1
Total Enrollment45
FDA Designations
No designations recorded
Clinical Trials (1)
NCT IDTitlePhaseStatusEnrollmentVelocityDesignStartCompletionLast UpdatedSitesCountries
NCT05650749GPC2 CAR T Cells for Relapsed or Refractory Neuroblastoma and Metastatic RetinoblastomaPHASE1 RECRUITING 45May 23, 2023Jan 30, 2030Dec 29, 20251 United States
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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 — 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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Recent Changes (Last 90 Days)
LOWMay 24, 2026NCT05650749studyFirstPostDate: changed