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CancerPhase 1/2PARCNCT03455140

A Study Evaluating the Safety and Activity of Pegylated Recombinant Human Arginase (BCT-100)

A Phase 1/2 study testing PEG- BCT-100 in people with Cancer and Pediatric Solid Tumor, run by University of Birmingham at 14 sites in 3 countries.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Official record
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Trial summary

PARC is an international phase I/II trial evaluating the safety and activity of pegylated recombinant human arginase (BCT-100) in children and young people with relapsed/refractory leukaemia, neuroblastoma, sarcoma and high grade gliomas (brain cancers). Read more about this study

Who is it for?
Ages 1 year to 25 years
Conditions the study is for
Cancer, Pediatric Solid Tumor, Pediatric AML
What is being tested?
PEG- BCT-100
Type of treatment
Injection or infusion
Could I receive a placebo?
No, everyone receives an active treatment
How many people
49 participants
Where
14 sitesin 3 countries
Study dates
28 Aug 2018to 22 Jul 2022
Phase
Phase 1/2
Are there results?
None posted in the registry

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

  • Ages 1 year to 25 years
  • All sexes
  • No healthy volunteers
  • Child, Adult

Who may be able to join

  • 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).
Show all 11Show fewer
  • 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

Who cannot join

  • 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

Trial locations and contacts

14 sites in 3 countries.

14 sites in 3 countries

United Kingdom7 sites

  • Birmingham Children's Hospital

    Birmingham

  • Bristol Royal Hospital for Children

    Bristol

  • Addenbrookes Hospital

    Cambridge

  • Royal Hospital for Children

    Glasgow

  • Leeds Children's Hospital

    Leeds

  • Royal Manchester Children's Hospital

    Manchester

  • Royal Marsden Hospital

    Sutton

Other trials in United Kingdom

Australia6 sites

  • Women's & Children's Hospital

    Adelaide

  • Queensland Children's Hospital

    Brisbane

  • Royal Children's Hospital Melbourne

    Melbourne

  • Perth Children's Hospital

    Perth

  • Children's Hospital Westmead

    Sydney

  • Sydney Children's Hospital

    Sydney

Other trials in Australia

Netherlands1 site

  • Princes Maxima Centrum

    Utrecht

Other trials in Netherlands

About this study

Official 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

PARC is an international phase I/II trial evaluating the safety and activity of pegylated recombinant human arginase (BCT-100) in children and young people with relapsed/refractory leukaemia, neuroblastoma, sarcoma and high grade gliomas (brain cancers).

Currently the outcomes for these patients are poor and the therapeutic options are limited with a significant toxicity burden. Therefore new treatments which work in different ways to standard chemotherapy are urgently needed. Research has shown that arginine (a nutrient) is important in the survival of cancer cells. BCT-100 is a drug which can deplete arginine levels and starve cancer cells - a completely new approach. BCT-100 has been tested in adults and shown to be active with almost no side-effects. This trial will test whether this dose of BCT-100 is also safe and active in children with relapsed/refractory leukaemia, neuroblastoma, sarcoma and high grade glioma. The trial will also study how BCT-100 is broken down in the body and look for new biological markers of treatment response. Up to 64 children with relapsed cancers will be recruited over 2 years.

Being tested
PEG- BCT-100
Keywords
Arginine, Arginase, Children, Young Adult
Medical subject headings
Neoplasms, Precursor Cell Lymphoblastic Leukemia-Lymphoma

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • PEG- BCT-100Drug

    Also called rhArg1peg5000

    PEGylated recombinant human arginase 1

Study groups in NCT03455140
Study groupRoleWhat they receive
Group 1 - LeukaemiaExperimentalPEG- BCT-100 in patients with Leukaemia Starting dose 1600U/Kg IV infusion weekly
Receives: Drug: PEG- BCT-100
Group 2 - NeuroblastomaExperimentalPEG- BCT-100 in patients with Neuroblastoma Starting dose 1600U/Kg IV infusion weekly
Receives: Drug: PEG- BCT-100
Group 3 - SarcomasExperimentalPEG- BCT-100 in patients with Sarcomas Starting dose 1600U/Kg IV infusion weekly
Receives: Drug: PEG- BCT-100
Group 4 - High Grade GliomaExperimentalPEG- BCT-100 in patients with High Grade Gliomas Starting dose 1600U/Kg IV infusion weekly
Receives: Drug: PEG- BCT-100

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. 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

    Safety profile as measured by the occurrence/non-occurrence of DLT within 28 days of treatment with BCT-100.

    • Optimal dose as measured by the complete depletion of arginine. This is defined as AAD <8μM arginine in the blood after 3 doses of BCT-100.

    Measured: 28 days

  2. 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.

    Disease response (Complete Response (CR) or Partial Response (PR)) after 8 weeks of treatment with BCT-100

    Measured: After 8 weeks

Secondary outcomes (14)

  1. The incidence and severity of Adverse Events (AEs) as Assessed by CTCAE v4

    Incidence and severity of Adverse Events (AEs) defined by National Cancer Institute Common Terminology Criteria for Adverse Events (CTCAE) v4

    Measured: 28 days after treatment completion

  2. Disease response - Leukaemia

    Disease response ( CR / PR) according to Cheson criteria

    Measured: Within 1 year

  3. Disease response - Sarcoma

    Disease response ( CR / PR) according to RECIST criteria

    Measured: Within 1 year

  4. Disease response - High Grade Glioma

    Disease response ( CR / PR) according to RANO criteria

    Measured: Within 1 year

  5. Disease response - Neuroblastoma

    Disease response ( CR / PR) according to INCR criteria

    Measured: Within 1 year

  6. Progression free survival (PFS)

    Measured: Up to three years after registration

  7. Overall survival (OS).

    Measured: Up to three years after registration

  8. Maximum Plasma Concentration [Cmax], of BCT-100 in the paediatric population.

    Measured: Up to 24 weeks

  9. Time to maximum Plasma Concentration [Tmax], of BCT-100 in the paediatric population.

    Measured: Up to 24 weeks

  10. Minimum Plasma Concentration [Cmin], of BCT-100 in the paediatric population.

    Measured: Up to 24 weeks

  11. Area Under the Curve [AUC], of BCT-100 in the paediatric population.

    Measured: Up to 24 weeks

  12. Duration of adequate arginine depletion in blood.

    BCT-100 concentration in blood

    Measured: Up to 24 weeks

  13. Duration of adequate arginine depletion in bone marrow .

    BCT-100 concentration in bone marrow

    Measured: Up to 24 weeks

  14. Duration of adequate arginine depletion in cerebrospinal fluid.

    BCT-100 concentration in cerebrospinal fluid

    Measured: Up to 24 weeks

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted6 Mar 2018
  2. Study start28 Aug 2018
  3. Primary completion22 Jul 2022
  4. Study completion22 Jul 2022

Resources

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design
Study type
Interventional
Phase
Phase 1/2
Primary purpose
Treatment
Allocation
Non-randomised
Intervention model
Parallel groups
Masking
None (open label)
Enrolment
49 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT03455140
Sponsor's protocol ID
RG_16-040
Acronym
PARC
Status last verified
Sep 2022
First submitted
1 Feb 2018
Last updated
2 Sep 2022
Results posted
No
Data monitoring committee
Yes
FDA-regulated drug
No
FDA-regulated device
No
Individual data sharing
No

Publications

  • Fenwick N, Weston R, Wheatley K, Hodgson J, Marshall L, Elliott M, Makin G, Ng A, Brennan B, Lowis S, Adamski J, Kilday JP, Cox R, Gattens M, Moore A, Trahair T, Ronghe M, Campbell M, Campbell H, Williams MW, Kirby M, Van Eijkelenburg N, Keely J, Scarpa U, Stavrou V, Fultang L, Booth S, Cheng P, De Santo C, Mussai F. 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. Front Oncol. 2024 Jan 31;14:1296576. doi: 10.3389/fonc.2024.1296576. eCollection 2024.

    PubMed 38357205

Questions about this trial

What is this clinical trial studying?

A Phase 1/2 study testing PEG- BCT-100 in people with Cancer and Pediatric Solid Tumor, run by University of Birmingham at 14 sites in 3 countries.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 11 inclusion and 4 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: finished, as of the sponsor's last update on 2 Sep 2022. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 14 sites in United Kingdom, Australia, Netherlands. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this combined phase mean?

Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.

Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

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 (time frame: 28 days). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT03455140, which also lists 1 linked publication.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 2 Sep 2022.

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