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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/study/NCT03455140


Additional trial details provided through ANZCTR are available at the end of this record.


Registration number
NCT03455140
Ethics application status
Date submitted
1/02/2018
Date registered
6/03/2018

Titles & IDs
Public title
A Study Evaluating the Safety and Activity of Pegylated Recombinant Human Arginase (BCT-100)
Scientific title
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
Secondary ID [1] 0 0
RG_16-040
Universal Trial Number (UTN)
Trial acronym
PARC
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Cancer 0 0
Pediatric Solid Tumor 0 0
Pediatric AML 0 0
Pediatric ALL 0 0
Condition category
Condition code

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
Treatment: Drugs - PEG- BCT-100

Experimental: Group 1 - Leukaemia - PEG- BCT-100 in patients with Leukaemia Starting dose 1600U/Kg IV infusion weekly

Experimental: Group 2 - Neuroblastoma - PEG- BCT-100 in patients with Neuroblastoma Starting dose 1600U/Kg IV infusion weekly

Experimental: Group 3 - Sarcomas - PEG- BCT-100 in patients with Sarcomas Starting dose 1600U/Kg IV infusion weekly

Experimental: Group 4 - High Grade Glioma - PEG- BCT-100 in patients with High Grade Gliomas Starting dose 1600U/Kg IV infusion weekly


Treatment: Drugs: PEG- BCT-100
PEGylated recombinant human arginase 1
Intervention code [1] 0 0
Treatment: Drugs
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Phase I: to establish the recommended phase II dose (RP2D) of BCT-100 in children and young adults as assessed by dose limiting toxicity (DLT) and complete arginine depletion
Timepoint [1] 0 0
28 days
Primary outcome [2] 0 0
Phase II: to determine the activity of single agent BCT-100 against relapsed/refractory leukaemia, neuroblastoma, sarcoma and high grade glioma in children and young adults as measured by disease response after 8 weeks.
Timepoint [2] 0 0
After 8 weeks
Secondary outcome [1] 0 0
The incidence and severity of Adverse Events (AEs) as Assessed by CTCAE v4
Timepoint [1] 0 0
28 days after treatment completion
Secondary outcome [2] 0 0
Disease response - Leukaemia
Timepoint [2] 0 0
Within 1 year
Secondary outcome [3] 0 0
Disease response - Sarcoma
Timepoint [3] 0 0
Within 1 year
Secondary outcome [4] 0 0
Disease response - High Grade Glioma
Timepoint [4] 0 0
Within 1 year
Secondary outcome [5] 0 0
Disease response - Neuroblastoma
Timepoint [5] 0 0
Within 1 year
Secondary outcome [6] 0 0
Progression free survival (PFS)
Timepoint [6] 0 0
Up to three years after registration
Secondary outcome [7] 0 0
Overall survival (OS).
Timepoint [7] 0 0
Up to three years after registration
Secondary outcome [8] 0 0
Maximum Plasma Concentration [Cmax], of BCT-100 in the paediatric population.
Timepoint [8] 0 0
Up to 24 weeks
Secondary outcome [9] 0 0
Time to maximum Plasma Concentration [Tmax], of BCT-100 in the paediatric population.
Timepoint [9] 0 0
Up to 24 weeks
Secondary outcome [10] 0 0
Minimum Plasma Concentration [Cmin], of BCT-100 in the paediatric population.
Timepoint [10] 0 0
Up to 24 weeks
Secondary outcome [11] 0 0
Area Under the Curve [AUC], of BCT-100 in the paediatric population.
Timepoint [11] 0 0
Up to 24 weeks
Secondary outcome [12] 0 0
Duration of adequate arginine depletion in blood.
Timepoint [12] 0 0
Up to 24 weeks
Secondary outcome [13] 0 0
Duration of adequate arginine depletion in bone marrow .
Timepoint [13] 0 0
Up to 24 weeks
Secondary outcome [14] 0 0
Duration of adequate arginine depletion in cerebrospinal fluid.
Timepoint [14] 0 0
Up to 24 weeks

Eligibility
Key inclusion criteria
* Aged 1- <25 years old at the time of study registration
* Histologically confirmed disease in one of the following four groups:

* Group 1 - Acute lymphoblastic leukaemia (ALL) and acute myeloid leukaemia (AML)
* Group 2 - Neuroblastoma Group 3 - Sarcoma
* Group 4 - High grade glioma (as defined by 2016 WHO CNS classification)
* Radiological or laboratory evidence of disease progression (during or after completion of first line treatment) or any subsequent recurrence (biopsy at relapse is not mandated).
* Measurable bone marrow disease (group 1) or at least one evaluable radiological site of disease (group 2, 3 and 4).
* Adequate liver function defined as a total bilirubin =1.5x the upper limit of normal for age and ALT = 3x the upper limit of normal for age
* Documented negative pregnancy test for female patients of childbearing potential within 7 days of trial entry
* Sexually active patients must agree to use adequate and appropriate contraception while on study drug and for 12 months following treatment discontinuation
* Written informed consent given by patient and/or parents/legal representative
Minimum age
1 Year
Maximum age
25 Years
Sex
Both males and females
Can healthy volunteers participate?
No
Key exclusion criteria
* Previous treatment with another therapeutic arginine depleting drug (bacterial or human) or arginase inhibitor
* Presence of any = CTCAE grade 3 clinically significant treatment-related toxicity from prior therapies
* Pregnant or lactating female
* Evidence of uncontrolled infection

Study design
Purpose of the study
Treatment
Allocation to intervention
Non-randomised trial
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Masking / blinding
Open (masking not used)
Who is / are masked / blinded?



Intervention assignment
Parallel
Other design features
Phase
Phase 1
Type of endpoint/s
Statistical methods / analysis

Recruitment
Recruitment status
Completed
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
28/08/2018
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
Actual
22/07/2022
Sample size
Target
Accrual to date
Final
49
Recruitment in Australia
Recruitment state(s)
Recruitment hospital [1] 0 0
Women's & Children's Hospital - Adelaide
Recruitment hospital [2] 0 0
Queensland Children's Hospital - Brisbane
Recruitment hospital [3] 0 0
Royal Children's Hospital Melbourne - Melbourne
Recruitment hospital [4] 0 0
Perth Children's Hospital - Perth
Recruitment hospital [5] 0 0
Children's Hospital Westmead - Sydney
Recruitment hospital [6] 0 0
Sydney Children's Hospital - Sydney
Recruitment postcode(s) [1] 0 0
- Adelaide
Recruitment postcode(s) [2] 0 0
- Brisbane
Recruitment postcode(s) [3] 0 0
- Melbourne
Recruitment postcode(s) [4] 0 0
- Perth
Recruitment postcode(s) [5] 0 0
- Sydney
Recruitment outside Australia
Country [1] 0 0
Netherlands
State/province [1] 0 0
Utrecht
Country [2] 0 0
United Kingdom
State/province [2] 0 0
Birmingham
Country [3] 0 0
United Kingdom
State/province [3] 0 0
Bristol
Country [4] 0 0
United Kingdom
State/province [4] 0 0
Cambridge
Country [5] 0 0
United Kingdom
State/province [5] 0 0
Glasgow
Country [6] 0 0
United Kingdom
State/province [6] 0 0
Leeds
Country [7] 0 0
United Kingdom
State/province [7] 0 0
Manchester
Country [8] 0 0
United Kingdom
State/province [8] 0 0
Sutton

Funding & Sponsors
Primary sponsor type
Other
Name
University of Birmingham
Address
Country

Ethics approval
Ethics application status

Summary
Brief summary
Trial website
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 0 0
Francis J Mussai, DPhil
Address 0 0
University of Birmingham
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for public queries
Name 0 0
Address 0 0
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for scientific queries

Data sharing statement
Will individual participant data (IPD) for this trial be available (including data dictionaries)?
No
No/undecided IPD sharing reason/comment


What supporting documents are/will be available?

No Supporting Document Provided



Results publications and other study-related documents

No documents have been uploaded by study researchers.


Results not provided in https://clinicaltrials.gov/study/NCT03455140

Additional trial details provided through ANZCTR
Accrual to date
Recruiting in Australia
Recruitment state(s)
SA
Funding & Sponsors
Primary sponsor
Primary sponsor name
Primary sponsor address
Primary sponsor country
Secondary sponsor category [1] 32
Other Collaborative groups
Name [1] 32
ANZCHOG
Address [1] 32
27-31 Wright Street, Clayton VIC 3168
Country [1] 32
Australia
Ethics approval
Ethics application status
 
Public notes
Women's and Children's Hospital

Contacts
Principal investigator
Title 157 0
Dr
Name 157 0
Maria Kirby
Address 157 0
72 King William Rd, North Adelaide SA 5006
Country 157 0
Australia
Phone 157 0
+618 8161 7411
Fax 157 0
Email 157 0
[email protected]
Contact person for public queries
Title 158 0
Mrs
Name 158 0
Robyn Strong
Address 158 0
27-31 Wright Street, Clayton VIC 3168
Country 158 0
Australia
Phone 158 0
+613 8572 2684
Fax 158 0
Email 158 0
[email protected]
Contact person for scientific queries
Title 159 0
Dr
Name 159 0
Maria Kirby
Address 159 0
72 King William Rd, North Adelaide SA 5006
Country 159 0
Australia
Phone 159 0
+618 8161 7411
Fax 159 0
Email 159 0
[email protected]