NCT02285101

Brief Summary

The goal of this clinical research study is to evaluate the safety of PEG-BCT- 100 given as an infusion to treat patients who bear advanced solid tumors that are dependent on arginine (melanoma, renal cell carcinoma, prostate cancer and hepatocellular carcinoma), and who have progressed after receiving approved or established therapies. This is a Phase 1 study; PEG-BCT-100 is an enzyme that degrades arginine and is an investigational drug.

Trial Health

87
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
23

participants targeted

Target at P25-P50 for phase_1

Timeline
Completed

Started Nov 2014

Longer than P75 for phase_1

Geographic Reach
1 country

2 active sites

Status
completed

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

First Submitted

Initial submission to the registry

October 23, 2014

Completed
9 days until next milestone

Study Start

First participant enrolled

November 1, 2014

Completed
5 days until next milestone

First Posted

Study publicly available on registry

November 6, 2014

Completed
4.2 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

February 1, 2019

Completed
Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

February 1, 2019

Completed
Last Updated

April 24, 2020

Status Verified

April 1, 2020

Enrollment Period

4.3 years

First QC Date

October 23, 2014

Last Update Submit

April 22, 2020

Conditions

Keywords

arginine auxotrophic tumorsPEG-BCT-100arginase

Outcome Measures

Primary Outcomes (2)

  • Number of patients undergoing adverse events (AEs) or serious adverse events (SAEs)

    at least 13 weeks

  • Optimal Biological Dose

    The optimal biological dose (OBD) of PEG-BCT-100 will be calculated based on the pharmacodynamics (PD) endpoint of plasma arginine depletion relative to plasma pharmacokinetics (PK) of PEG-BCT-100

    13 weeks

Secondary Outcomes (8)

  • Maximum Tolerated Dose and Dosing Schedule

    4 weeks of treatment

  • Overall response

    13 weeks

  • Pharmacokinetics (PK)-PEG-BCT-100 concentration

    13 weeks

  • Pharmacodynamics (PD)

    13 weeks

  • PK-PEG-BCT-100 plasma clearance

    13 weeks

  • +3 more secondary outcomes

Study Arms (6)

Cohort 1

EXPERIMENTAL

PEG-BCT-100 at 0.5 mg/kg administered as a single dose on day 1 (week 1); if not DLTs are observed, PEG-BCT- 100 will be administered at 0.5 mg/kg on days 22 (week 4) and 29 (week 5). Patients responding to treatment or with stable disease may receive 8 additional weekly administrations of PEG-BCT-100 at 0.5 mg/kg. Beyond week 13, patients responding to treatment or with stable disease may continue to receive weekly PEG-BCT-100 at 0.5 mg/kg until disease progression at the discretion of the investigator.

Biological: PEG-BCT-100

Cohort 2

EXPERIMENTAL

PEG-BCT-100 at 1.0 mg/kg administered as a single dose on day 1 (week 1); if not DLTs are observed, PEG-BCT- 100 will be administered at 1.0 mg/kg on days 22 (week4) and 29 (week 5). Patients responding to treatment or with stable disease may receive 8 additional weekly administrations of PEG-BCT-100 at 1.0 mg/kg. Beyond week 13, patients responding to treatment or with stable disease may continue to receive weekly PEG-BCT-100 at 1.0 mg/kg until disease progression at the discretion of the investigator.

Biological: PEG-BCT-100

Cohort 3

EXPERIMENTAL

PEG-BCT-100 at 1.7 mg/kg administered as a single dose on day 1 (week 1); if not DLTs are observed, PEG-BCT- 100 will be administered at 1.7 mg/kg on days 22 (week 4) and 29 (week 5). Patients responding to treatment or with stable disease may receive 8 additional weekly administrations of PEG-BCT-100 at 1.7 mg/kg. Beyond week 13, patients responding to treatment or with stable disease may continue to receive weekly PEG-BCT-100 at 1.7 mg/kg until disease progression at the discretion of the investigator.

Biological: PEG-BCT-100

Cohort 4

EXPERIMENTAL

PEG-BCT-100 at 1.7 mg/kg administered as a single dose on day 1 (week 1); if not DLTs are observed, PEG-BCT- 100 will be administered at 2.7 mg/kg on days 22 (week 4) and 29 (week 5). Patients responding to treatment or with stable disease may receive 8 additional weekly administrations of PEG-BCT-100 at 2.7 mg/kg. Beyond week 13, patients responding to treatment or with stable disease may continue to receive weekly PEG-BCT-100 at 2.7 mg/kg until disease progression at the discretion of the investigator.

Biological: PEG-BCT-100

Cohort 5

EXPERIMENTAL

PEG-BCT-100 at 1.7 mg/kg administered as a single dose on day 1 (week 1); if not DLTs are observed, PEG-BCT- 100 will be administered at 4.0 mg/kg on days 22 (week 4) and 29 (week 5). Patients responding to treatment or with stable disease may receive 8 additional weekly administrations of PEG-BCT-100 at 4.0 mg/kg. Beyond week 13, patients responding to treatment or with stable disease may continue to receive weekly PEG-BCT-100 at 4.0 mg/kg until disease progression at the discretion of the investigator.

Biological: PEG-BCT-100

Cohort 6

EXPERIMENTAL

PEG-BCT-100 at a dose to be determined administered as a single dose on day 1 (week 1); if not DLTs are observed, PEG-BCT-100 will be administered at at a dose to be determined on days 22 (week 4) and 29 (week 5). Patients responding to treatment or with stable disease may receive 8 additional weekly administrations of PEG-BCT-100 at at a dose to be determined. Beyond week 13, patients responding to treatment or with stable disease may continue to receive weekly PEG-BCT-100 at at a dose to be determined until disease progression at the discretion of the investigator.

Biological: PEG-BCT-100

Interventions

PEG-BCT-100BIOLOGICAL
Also known as: pegylated recombinant human arginase
Cohort 1Cohort 2Cohort 3Cohort 4Cohort 5Cohort 6

Eligibility Criteria

Age18 Years+
Sexall
Healthy VolunteersNo
Age GroupsAdult (18-64), Older Adult (65+)

You may qualify if:

  • Confirmed diagnosis of Stage IIIb/IV malignant melanoma or castration resistant adenocarcinoma of prostate (CRPC).
  • Advanced cancer not candidate for treatment with modality or agents that are approved or have established efficacy. Candidates who cannot tolerate standard treatment or whose cancers have progressed on current standard of care.
  • Males or females 18 years-old and above.
  • Ability to understand and willingness to provide written informed consent;
  • Karnofsky performance status (see Appendix 13.3) of 80% or above and expected survival of more than 12 weeks.
  • Negative urine pregnancy test, if female, and willingness to use an effective method of contraception during the entire study period whether the patient is male or female.

You may not qualify if:

  • Has received cancer treatment, e.g. chemotherapy, targeted biologic or enzymes, either approved or investigational, within 4 weeks prior to the start of the PEG-BCT-100;
  • Advancing liver failure indicated by uncontrolled ascites, pleural effusions, or encephalopathy.
  • Child-Pugh score of B and C (see Appendix 13.4).
  • Significant hepatic, renal or bone marrow dysfunction indicated by: total bilirubin \>2.0 mg/dL, evidence of bile duct obstruction, serum albumin \<2.5 g/dL, serum ALT or AST \>2.5 x upper limit of normal, serum creatinine ≥1.5 mg/dL, ANC ≥1.5 x 109/L, platelets \<100 x 109/L, or INR \>2.0.
  • Significant cardiac or pulmonary disease defined by New York Heart Association (NYHA) Class III or IV (see Appendix 13.5), left ventricular ejection fraction (LVEF) lower than institutional normal limits by echo or MUGA, history of myocardial infarction within the past 6 months, significant unstable arrhythmia or evidence of ischemia on ECG.
  • Pregnant or nursing women. NOTE: Women of child-bearing potential and men must agree to use adequate contraception (hormonal or barrier method of birth control or abstinence) prior to study entry and for the duration of study participation. Should a woman become pregnant or suspect she is pregnant while participating in this study, she should inform her treating physician immediately.
  • Significant active infection including HIV requiring oral or parenteral anti-infective therapies.
  • Use of investigational drug(s) within 4 weeks of enrollment.
  • Prior treatment with arginine depleting agent.

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (2)

California Cancer Associates for Research and Excellence, cCARE

San Diego, California, 92111, United States

Location

John Wayne Cancer Institute

Santa Monica, California, 90404, United States

Location

Related Publications (1)

  • De Santo C, Cheng P, Beggs A, Egan S, Bessudo A, Mussai F. Metabolic therapy with PEG-arginase induces a sustained complete remission in immunotherapy-resistant melanoma. J Hematol Oncol. 2018 May 18;11(1):68. doi: 10.1186/s13045-018-0612-6.

MeSH Terms

Conditions

Melanoma

Interventions

BCT-100

Condition Hierarchy (Ancestors)

Neuroendocrine TumorsNeuroectodermal TumorsNeoplasms, Germ Cell and EmbryonalNeoplasms by Histologic TypeNeoplasmsNeoplasms, Nerve TissueNevi and MelanomasSkin NeoplasmsNeoplasms by SiteSkin DiseasesSkin and Connective Tissue Diseases

Study Officials

  • Alberto Bessudo, MD

    California Cancer Associates For Research and Excellence, cCARE

    PRINCIPAL INVESTIGATOR
  • Steven O'Day, MD

    Saint John's Cancer Institute

    PRINCIPAL INVESTIGATOR

Study Design

Study Type
interventional
Phase
phase 1
Allocation
NON RANDOMIZED
Masking
NONE
Purpose
TREATMENT
Intervention Model
PARALLEL
Sponsor Type
INDUSTRY
Responsible Party
SPONSOR

Study Record Dates

First Submitted

October 23, 2014

First Posted

November 6, 2014

Study Start

November 1, 2014

Primary Completion

February 1, 2019

Study Completion

February 1, 2019

Last Updated

April 24, 2020

Record last verified: 2020-04

Locations