PARC: a phase I/II study evaluating the safety and activity of pegylated recombinant human arginase BCT-100 in relapsed/refractory cancers of children and young adults

Autor: Nicola Fenwick, Rebekah Weston, Keith Wheatley, Jodie Hodgson, Lynley Marshall, Martin Elliott, Guy Makin, Antony Ng, Bernadette Brennan, Stephen Lowis, Jenny Adamski, John Paul Kilday, Rachel Cox, Mike Gattens, Andrew Moore, Toby Trahair, Milind Ronghe, Martin Campbell, Helen Campbell, Molly W. Williams, Maria Kirby, Natasha Van Eijkelenburg, Jennifer Keely, Ugo Scarpa, Victoria Stavrou, Livingstone Fultang, Sarah Booth, Paul Cheng, Carmela De Santo, Francis Mussai
Jazyk: angličtina
Rok vydání: 2024
Předmět:
Zdroj: Frontiers in Oncology, Vol 14 (2024)
Druh dokumentu: article
ISSN: 2234-943X
DOI: 10.3389/fonc.2024.1296576
Popis: BackgroundThe survival for many children with relapsed/refractory cancers remains poor despite advances in therapies. Arginine metabolism plays a key role in the pathophysiology of a number of pediatric cancers. We report the first in child study of a recombinant human arginase, BCT-100, in children with relapsed/refractory hematological, solid or CNS cancers.ProcedurePARC was a single arm, Phase I/II, international, open label study. BCT-100 was given intravenously over one hour at weekly intervals. The Phase I section utilized a modified 3 + 3 design where escalation/de-escalation was based on both the safety profile and the complete depletion of arginine (defined as adequate arginine depletion; AAD
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