Trial Details
Trial status:
Active (recruiting)
Recruitment start date:
Chief Investigator:
Dr Karin Straathof
Recruitment target:
EudraCT number:
Contact details:
Lay summary:
Multi-modular Chimeric Antigen Receptor targeting GD2 in Neuroblastoma

A single-centre, non-randomised, open label Phase I clinical trial of an Advanced Therapy Investigational Medicinal Product (ATIMP) in children and teenagers aged 1-16 years with relapsed or refractory neuroblastoma. The study will assess the feasibility of generating the ATIMP (GD2 CAR T cells) and the safety of administering the ATIMP in participants with relapsed or refractory neuroblastoma. A total of 12 participants will be treated at 1 participating site.


Participants will undergo an unstimulated leucapheresis for the generation of the ATIMP which will take approximately 15 days to generate. Participants will receive lymphodepleting (LD) chemotherapy with fludarabine administered over 4 days(Day -6 to Day -3) and cyclophosphamide administered over 2 days (Day -4 and Day-3). This will be followed by CAR T cells infusion administered on Day 0. Participants will be followed up regularly until 1-year post-ATIMP infusion.  After 1 year, participants will continue to be followed up annually until 15 years post ATIMP infusion.

Key inclusion/exclusion criteria:

Key inclusion criteria:

  • Age between 1 and 16 years;
  • Tissue diagnosis of neuroblastoma;
  • Disease which has relapsed after or is refractory to at least one line of salvage combination chemotherapy;
  • Measurable/evaluable disease by imaging;
  • Absolute lymphocyte count ≥ 0.25 x 109/L;
  • Creatinine needs to be less than 1.5 of upper limit of normality for age (if higher, an estimated (calculated) creatinine clearance must be ≥ 60 ml/min/1.73 m2).
Key exclusion criteria:
  • Patients with only bone marrow detectable disease (in the absence of measurable disease by imaging);
  • Active, inoperative CNS disease including leptomeningeal disease;
  • Active HepB, HepC or HIV infection 

Duration of recruitment: Anticipated duration of recruitment will be 2 years.

The aim of the study is to evaluate the safety, efficacy and optimal dose of GD2 CAR T cells in participants with neuroblastoma.

Trial protocols
Trial protocols must not be applied to patients treated outside trials. UCL CTC can only ensure that approved trial investigators are provided with amendments to protocols.
No protocols have been currently made available for download
Trial documents
No documents have been currently made available for download
Contact Us
Cancer Research UK & UCL Cancer Trials Centre
University College London
90 Tottenham Court Road

View map
+44 (0)20 7679 9898 (General CTC Enquiries)
020 7679 9899
University College London, Gower Street, London, WC1E 6BT +44 (0)20 7679 2000

Copyright © 2024 UCL | Disclaimer | Freedom of Information | Accessibility | Privacy | Cookies | Contact Us