Recruiting
Study Start: February, 2019
Study Completion: December, 2026
NCT03903835

ProBio: A Biomarker Driven Study in Patients With Metastatic Prostate Cancer (ProBio)

Keywords:
Prostate cancer
Cancer

Brief Summary

ProBio is an international, outcome-adaptive, multi-arm, open-label, multiple assignment randomized biomarker driven platform trial in patients with metastatic prostate cancer. Patients will be randomized to control or experimental treatment arms. Patients in the control arm will receive standard of care following national guidelines. Patients in the experimental arm will be randomized to treatments based on a biomarker signature inferred from diagnostic tissue or liquid biopsy profiling. The predefined biomarker signatures are tumor properties or mutations in genes/pathways with previously demonstrated clinical validity (e.g. prognostic value or association with treatment response). The biomarker signatures are identified using a hybridisation capture gene panel specifically designed for prostate cancer.

Detailed Description

ProBio is an outcome-adaptive, multi-arm, open-label, multiple assignment randomised biomarker driven platform trial in patients with metastatic hormone-sensitive and castration-resistant prostate cancer.
Patients will be randomised to control or experimental treatment class arms. Patients in the control arm will receive standard of care following national guidelines and will remain within the control arm throughout the course of the trial. Patients in the experimental arm will be randomised to a treatment class (consisting of one or multiple drugs) based on a biomarker signature. The biomarker signatures are defined as tumour properties or mutations in certain genes/pathways identified in the scientific literature as important in prostate cancer treatment response. The biomarker signatures are identified using a gene panel specifically designed for advanced prostate cancer.
Alterations in the following genes/pathways or combinations thereof constitute the biomarker signatures:
  • Androgen receptor
  • DNA-repair deficiency
  • TP53
  • TMPRSS2-ERG gene fusion
  • PI3K pathway alterations
Patients in the experimental arm can be randomized to the following treatments classes:
for mHSPC
  • AR signalling inhibitors (Abiraterone acetate, Enzalutamide, Apalutamide)
  • Taxane-based chemotherapy in combination with ARSI (Docetaxel plus Abiraterone acetate, or Darolutamide)
  • PolyADP Ribose Polymerase Inhibitors (Niraparib plus Abiraterone Acetate)
for mCRPC
  • AR signalling inhibitors (Enzalutamide, Abiraterone acetate)
  • Poly ADP Ribose Polymerase Inhibitors (Niraparib plus Abiraterone acetate)
  • Selective AKT inhibitor (Capivasertib plus Docetaxel)
ProBio will use outcome-adaptive randomization, adapting the randomization based on the observed progression free survival (PFS) within biomarker signatures. Treatments will initially be assigned to patients based on the biomarker signatures for which that treatment is most likely to be effective. The trial will be analyzed within a Bayesian framework, which allows for calculations of the probability for each treatment that it is superior to standard of care within a given signature. Each experimental arm will be evaluated for efficacy relative to the control arm with the same biomarker signatures.
Participants and treating physicians will be blinded to ctDNA profile of each patient. The biomarker signatures will thus not influence treatment choice among controls (reflecting today's standard of care).
Further ProBio will use the sequential multiple assignments trial (SMART) concept, where each patient who progresses within the trial will re-enter the trial and be re-assigned to another treatment based on the patient's current ctDNA profile. Patients will be withdrawn after in total maximal three randomized consecutive treatments after inclusion into the study.
The randomization probabilities within the experimental arm are defined in proportion to the probability that each treatment is superior to standard of care within a given biomarker signature, and therefore change as data accumulates in the trial and knowledge accumulates for what biomarker signatures and specific treatments that are more probable to be effective.
Trial results will be evaluated regularly by an independent data and safety monitoring board (DSMB). The DSMB will evaluate treatment-signature combinations with respect to:
  • Graduation for superiority: A treatment-biomarker signature combination will be graduated from the trial if it has a Bayesian predictive probability of success in a future confirmatory phase III trial exceeding a pre-specified threshold (85%).
  • Termination for futility: Treatment-biomarker signature combinations will be dropped from the trial for futility when success probabilities drop sufficiently low (less than 10% using a minimum of 20 patients assigned to the specific treatment-biomarker signature combination).
  • Alternatively, if the maximum sample size of 300 and 150 patients (for mHSPC and mCRPC, respectively) assigned to a treatment biomarker signature is reached without graduation for superiority, assignments to that combination will end.
ProBio is a platform study. This means that new treatments and biomarker signatures can be added to the experimental arm in the future. This will be done after protocol amendments.

Intervention / Treatment

01
Enzalutamide Oral Capsule
Drug
02
Abiraterone Oral Tablet
Drug
03
Carboplatin
Drug
04
Cabazitaxel 60 mg Solution for Injection
Drug
05
Docetaxel Injectable Solution
Drug
06
Radium Chloride Ra-223
Drug
07
Niraparib plus Abiraterone acetate plus Prednisone
Drug
08
Capivasertib plus Docetaxel
Drug
09
Apalutamide
Drug
10
Darolutamide
Drug

Study Design

Primary Purpose

Treatment

Allocation

Randomized

Interventional Model

Sequential Assignment

Interventional Model Description

ProBio will use outcome-adaptive randomization, adapting the randomization based on the observed PFS within biomarker signatures. Treatments will initially be assigned to patients based on the biomarker signatures for which that treatment is most likely to be effective. The trial will be analysed within a Bayesian framework, which allows for calculations of the probability for each treatment that is superior to standard of care within a given signature. Each experimental arm will be evaluated for efficacy relative to the control arm with the same biomarker signatures.

Masking

None (Open Label)

Arm Groups

Active Comparator: Control: Standard Care

Randomization between assignment to the control arm or the biomarker driven arm will be stratified on biomarker signatures, previous treatment, and fraction of ctDNA and will therefore occur after the results from the ctDNA profiling is obtained. Patients in the control arm will receive standard of care following national guidelines.

Experimental: Treatment 1 in mHSPC: AR signalling inhibitors (ARSi)

Assignments to therapy in the biomarker driven arms will be done on the basis of the biomarker signature using the current information about the efficacy of the various regimens for that signature. Information from previous studies may be incorporated in the randomization at study onset if such reliable data exists. Specifically, patients with an intact androgen receptor (AR) and without TP53 mutations will have increased chance of being randomized to treatment with ARSi.

Experimental: Treatment 2 in mHSPC: taxane-based chemotherapy in combination with ARSi

Patients with TP53 mutations and TMPRSS2-ERG gene fusions will have an increased chance of being randomised to treatment with chemotherapy plus an ARSi.

Experimental: Treatment 3 in mHSPC: Poly ADP Ribose Polymerase (PARP) inhibitor

DNA-repair deficient patients will have an increased chance of receiving PARP inhibitors at study onset.

Experimental: Treatment 1 in mCRPC: AR signalling inhibitors (ARSi)

Specifically, patients with an intact androgen receptor (AR) and without TP53 mutations will have increased chance of being randomized to treatment with ARSi

Experimental: Treatment 2 in mCRPC: Poly ADP Ribose Polymerase (PARP) inhibitor

DNA-repair deficient patients will have an increased chance of receiving PARP inhibitors at study onset.

Experimental: Treatment 3 in mCRPC: selective AKT Inhibitor

Patients with alterations in the PI3K pathway will have an increased chance of receiving the combination treatment with Capivasertib plus Docetaxel.

Experimental: Treatment 4 in mCRPC: Carboplatin

Only patients with DNA-repair deficiency will be treated with Carboplatin as second line treatment in the castration-resistant phase of ProBio.

Eligibility Criteria

  • Man with histologically confirmed prostate adenocarcinoma, initiating systemic therapy for metastatic disease, encompassing newly diagnosed (i.e. de novo) hormone sensitive prostate cancer (mHSPC) or first-line castration resistant prostate cancer (mCRPC)
  • Distant metastatic disease documented by positive bone scan or metastatic lesions on CT or MRI
  • Adequate health as assessed by the investigator to receive all available treatments in the trial
  • ECOG/WHO (Eastern Cooperative Oncology Group / World Health Organization) performance score 0-2
  • Adequate organ and bone marrow function
  • Albumin greater than or equal to 28 g/L
  • Able to understand the patient information and sign written informed consent

  • Other malignancies within 5 years except non-melanoma skin cancer
  • Within 6 months of randomization: myocardial infarction, unstable angina, angioplasty, bypass surgery, stroke, TIA (transient ischemic attack), or congestive heart failure NYHA (New York Heart Association) class III or IV
  • Uncontrolled hypertension
  • Uncontrolled hypotension
  • Received systemic therapy (with the exception of standard ADT) prior to study inclusion, for the CRPC indication
  • Any severe acute or chronic medical condition that places the patient at increased risk of serious toxicity or interferes with the interpretation of study results
  • Unable to comply with study procedures
  • Current participation in another clinical trial that will be in conflict with the present study, administration of an investigational therapeutic or invasive surgical procedure within 28 days prior to study enrolment
  • Patients who are unlikely to comply with the protocol
  • Any condition or situation which, in the opinion of the investigator, would put the subject at risk, may confound study results, or interfere with the subject's participation in this study
  • Any medical condition that would make use of the study treatments contraindicated, according to the SmPC, e.g. significant heart or liver disease.

Outcome Measures

Primary Outcome Measures

Progression free survival (PFS) in mCRPC

Progression will be evaluated by the established international standards of the Prostate Cancer Working Group version 3 (PCWG3) and for soft tissue metastases (e.g. lung, liver and lymph nodes) according to the Response Evaluation Criteria in Solid Tumors (RECIST v. 1.1).

Until progressive disease or 60 months from start of treatment, whatever occurs first.

Progression free survival (PFS) in mHSPC

Time to development of castration-resistance, as defined by EAU guidelines (biochemical progression or radiologic progression).

From date of treatment start until the date of first documentation of progression, assessed up to 60 months

Secondary Outcome Measures

Treatment response rate in mCRPC

Treatment response is evaluated according to PCWG3 and RECIST 1.1.

4 months after treatment start

Overall survival (OS)

OS is defined as time to death from any cause (overall and prostate cancer specific)

From enrolment to completion of study (60 months)

Patient Reported Outcome Measures (PROM)

QoL will be assessed using the EORTC QLQ-C30 instrument

From enrolment to completion of study (60 months)

Cost-effectiveness

Cost effectiveness will be assessed by using the EQ-5D-5L instrument to estimate health utilities. Treatment costs will be based on drug costs and reimbursement data.

From enrolment to completion of study (60 months)

Number of Participants With Adverse Events as a Measure of Safety and Tolerability

Common Terminology Criteria for Adverse Events (CTCAE) developed and maintained by the US National Cancer Institute will be used to record adverse events

From enrolment to completion of study (60 months)

Treatment response rate in mHSPC

Response rates at 6 months on therapy will be evaluated by the established standards of EAU Guidelines.

6 months after treatment start

Where is this study conducted?

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