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Clinical Trial Summary

This is a first in human dose escalation trial to determine the safety of administering GPC2 CAR T cells in patients with advanced neuroblastoma.


Clinical Trial Description

Despite the use of intensive multimodal chemoradiotherapy, surgery, autologous stem cell transplant and disialoganglioside antigen (GD2)-targeted immunotherapy for the treatment of patients with high-risk neuroblastoma, approximately 60% of children still die from this disease and survivors suffer lifelong treatment related comorbidities. For patients who suffer a relapse after receiving therapy with standard of care multimodality treatment, there are no known curative options. Glypican 2 (GPC2) is highly expressed on the plasma membrane of most high-risk neuroblastomas, is further enriched in the tumor stem cell compartment, but is not expressed at significant levels on normal tissues, making it an ideal target for immune directed therapies. To therapeutically leverage GPC2's differential expression, a GPC2-directed CAR T cell therapy that potently inhibits the growth of neuroblastoma patient-derived xenografts has been developed. This investigation will be a single institution, open-label first in human, dose escalation and expansion study designed to assess the safety, tolerability, and manufacturing feasibility of GPC2 CAR T cells. ;


Study Design


Related Conditions & MeSH terms


NCT number NCT05650749
Study type Interventional
Source Children's Hospital of Philadelphia
Contact Melissa Varghese
Phone 8455535358
Email Varghesem@chop.edu
Status Recruiting
Phase Phase 1
Start date May 23, 2023
Completion date January 30, 2026

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