A Pilot Study of the Safety and Preliminary Efficacy of Recombinant Human Arginase 1 (rhArg1) in Patients With Relapsed or Refractory Leukemia or Lymphoma
This is a pilot, open-label study of PEG-BCT-100 in patients with relapsed/refractory
leukemia or lymphoma who have satisfied all inclusion/exclusion criteria.
Approximately 15 subjects will be enrolled or when 10 evaluable subjects are included in the
study. Evaluable subjects are defined as subjects who have received 4 consecutive doses of
PEG-BCT-100 1600U/kg within 6 weeks from the first 1600U/kg dose and completed the first
disease response assessment.
After the initiation of trial treatment, safety parameters will be evaluated throughout the
study. Adverse event (AE) will be graded according to the National Cancer Institute Common
Toxicity Criteria for Adverse Events version 4.0 (NCI CTC AE v4). AE and serious AE (SAE)
will be detected and recorded on the Case Report Forms (CRFs) until 15-28 days after the
last dose of PEG-BCT-100.
Patients who achieve complete remission (CR)/complete remission with incomplete blood count
recovery (CRi) following the 4 consecutive 1600U/kg doses may continue PEG-BCT-100 at the
discretion of the investigator.
Patients who achieve partial remission (PR) or stable disease (SD) following the 4
consecutive 1600U/kg doses can continue treatment up to disease progression. Further
continuation will be determined by the clinical judgment of the Investigator. Patients who
have disease progression will be discontinued PEG-BCT-100.
Blood samples for PK and PD analysis will be collected and analyzed.
Endpoint Classification: Safety/Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment
safety of PEG-BCT-100 in patients with relapsed or refractory leukemia or lymphoma receiving PEG-BCT-100
The primary endpoint is defined as the adverse event and serious adverse event related to the trial treatment. The adverse event includes unexpected clinical findings which could be related to the metabolic consequences of acute or prolong arginine depletion. The following measures will be used to evaluate the safety of the trial treatment: vital signs laboratory tests of safety i.e. hematology, blood biochemistry, urinalysis Adverse events (NCI CTC AE, version 4.0)
Alan K Chiang, Dr.
Department of Paediatrics and Adolescent Medicine, The University of Hong Kong
Hong Kong: Department of Health