Evolution of the Innovative Therapies for Children With Cancer Consortium Trial Portfolio for Drug Development for Children With Cancer-Reference-Cited by-同舟云学术

Evolution of the Innovative Therapies for Children With Cancer Consortium Trial Portfolio for Drug Development for Children With Cancer

Published:2024-07-20 Issue:21 Volume:42 Page:2516-2526
ISSN:0732-183X
Container-title:Journal of Clinical Oncology
language:en
Short-container-title:JCO

Author:

Bautista Francisco¹²^ORCID,Verdú-Amorós Jaime¹³^ORCID,Geoerger Birgit⁴^ORCID,Rubio-San-Simón Alba¹^ORCID,Paoletti Xavier⁵^ORCID,Zwaan C. Michel²⁶^ORCID,Casanova Michela⁷^ORCID,Marshall Lynley V.⁸⁹,Carceller Fernando⁸⁹^ORCID,Doz Francois¹⁰^ORCID,Lecinse Carole¹¹,Vassal Gilles¹¹^ORCID,Pearson Andrew D.J.⁸^ORCID,Kearns Pamela¹²^ORCID,Moreno Lucas¹³^ORCID,

Affiliation:

1. Division of Pediatric Hematology and Oncology, Hospital Universitario Niño Jesús, Madrid, Spain

2. Princess Máxima Center, Utrecht, the Netherlands

3. Division of Pediatric Hematology and Oncology, Hospital Clínico Universitario de Valencia, Biomedical Research Institute, INCLIVA, Valencia, Spain

4. Pediatric and Adolescent Oncology Department, Gustave Roussy Cancer Campus, INSERM U1015, Université Paris-Saclay, Villejuif, France

5. Institut Curie & Université Versailles St Quentin & INSERM U900 STAMPM, Paris, France

6. Department of Pediatric Oncology, Erasmus MC-Sophia Children's Hospital, Rotterdam, the Netherlands

7. Pediatric Oncology Unit, Fondazione IRCCS Istituto Nazionale dei Tumori, Milan, Italy

8. Pediatric and Adolescent Oncology Drug Development, Children & Young People's Unit, The Royal Marsden NHS Foundation Trust, London, United Kingdom

9. Division of Clinical Studies and Cancer Therapeutics, The Institute of Cancer Research, London, United Kingdom

10. SIREDO Cancer Center (Care, Innovation and Research in Pediatric, Adolescents, and Young Adults Oncology), Curie Institute Paris, and University Paris Cité, Paris, France

11. Innovative Therapies for Children with Cancer, Gustave Roussy Cancer Campus, Villejuif, France

12. Institute of Cancer and Genomic Sciences, NIHR Birmingham Biomedical Research Centre, University of Birmingham, Birmingham, United Kingdom

13. Division of Pediatric Hematology and Oncology, Hospital Universitari Vall d'Hebron, Barcelona, Spain

Abstract

PURPOSE The aim of the Innovative Therapies for Children with Cancer (ITCC) consortium is to improve access to novel therapies for children and adolescents with cancer. The evolution of the ITCC clinical trial portfolio since 2003 was reviewed. METHODS All ITCC-labeled phase I/II trials opened between January 1, 2003 and February 3, 2018 were analyzed in two periods (2003-2010 and 2011-2018), and data were extracted from the ITCC database, regulatory agencies' registries, and publications. RESULTS Sixty-one trials (62% industry-sponsored) enrolled 3,198 patients. The number of trials in the second period increased by almost 300% (16 v 45). All biomarker-driven trials (n = 14) were conducted in the second period. The use of rolling six and model-based designs increased (1 of 9, 11% v 21 of 31, 68%), and that of 3 + 3 designs decreased (5 of 9, 55% v 5 of 31, 16%; P = .014). The proportion of studies evaluating chemotherapeutics only decreased (5 of 16, 31% v 4 of 45, 9%), the proportion of single-agent targeted therapies did not change (9 of 16, 56.2% v 24 of 45, 53.3%), the proportion of combination targeted therapies trials increased (2 of 16, 12%, v 17 of 45, 38%), the proportion of randomized phase II trials increased (1 of 7, 14% v 8 of 14, 57%). More trials were part of a pediatric investigation plan in the second period (4 of 16, 25% v 21 of 45, 46%). The median time for Ethics Committees' approvals was 1.7 times longer for academic compared with industry-sponsored trials. CONCLUSION This study reports a shift in the paradigm of early drug development for childhood cancers, with more biologically relevant targets evaluated in biomarker-driven trials or in combination with other therapies and with more model-based or randomized designs and a greater focus on fulfilling regulatory requirements. Improvement of trial setup and recruitment could increase the number of patients benefiting from novel agents.

Publisher

American Society of Clinical Oncology (ASCO)

Link

https://ascopubs.org/doi/pdfdirect/10.1200/JCO.23.01237

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