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

NCT ID: NCT03903835

Last Updated: 2025-04-09

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

RECRUITING

Clinical Phase

PHASE3

Total Enrollment

750 participants

Study Classification

INTERVENTIONAL

Study Start Date

2019-02-01

Study Completion Date

2026-12-31

Brief Summary

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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

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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.

Conditions

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Metastatic Castration-resistant Prostate Cancer (mCRPC) Metastatic Hormone-Sensitive Prostate Cancer (mHSPC)

Study Design

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Allocation Method

RANDOMIZED

Intervention Model

SEQUENTIAL

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.
Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

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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.

Group Type ACTIVE_COMPARATOR

Enzalutamide Oral Capsule

Intervention Type DRUG

Detailed conditions for the use of the study treatments including dose and dosages are described in accordance with the marketing authorization in the SmPC (Summary of Product Characteristics).

Abiraterone Oral Tablet

Intervention Type DRUG

Detailed conditions for the use of the study treatment including dose and dosages are described in accordance with the marketing authorisation in the SmPC.

Cabazitaxel 60 mg Solution for Injection

Intervention Type DRUG

Detailed conditions for the use of the study treatment including dose and dosages are described in accordance with the marketing authorisation in the SmPC.

Docetaxel Injectable Solution

Intervention Type DRUG

Detailed conditions for the use of the study treatment including dose and dosages are described in accordance with the marketing authorisation in the SmPC.

Radium Chloride Ra-223

Intervention Type DRUG

Detailed conditions for the use of the study treatment including dose and dosages are described in accordance with the marketing authorisation in the SmPC.

Apalutamide

Intervention Type DRUG

Detailed conditions for the use of the study treatments including dose and dosages are described in accordance with the marketing authorization in the SmPC (Summary of Product Characteristics).

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.

Group Type EXPERIMENTAL

Enzalutamide Oral Capsule

Intervention Type DRUG

Detailed conditions for the use of the study treatments including dose and dosages are described in accordance with the marketing authorization in the SmPC (Summary of Product Characteristics).

Abiraterone Oral Tablet

Intervention Type DRUG

Detailed conditions for the use of the study treatment including dose and dosages are described in accordance with the marketing authorisation in the SmPC.

Apalutamide

Intervention Type DRUG

Detailed conditions for the use of the study treatments including dose and dosages are described in accordance with the marketing authorization in the SmPC (Summary of Product Characteristics).

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.

Group Type EXPERIMENTAL

Abiraterone Oral Tablet

Intervention Type DRUG

Detailed conditions for the use of the study treatment including dose and dosages are described in accordance with the marketing authorisation in the SmPC.

Docetaxel Injectable Solution

Intervention Type DRUG

Detailed conditions for the use of the study treatment including dose and dosages are described in accordance with the marketing authorisation in the SmPC.

Darolutamide

Intervention Type DRUG

Detailed conditions for the use of the study treatments including dose and dosages are described in accordance with the marketing authorization in the SmPC (Summary of Product Characteristics).

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.

Group Type EXPERIMENTAL

Niraparib plus Abiraterone acetate plus Prednisone

Intervention Type DRUG

Niraparib and Abiraterone acetate will be provided by Janssen and will be provided either as a fixed dose combination or as single agents. Detailed use of the study treatment including dose and dosages are described in the Investigator's brochures and SmPC.

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.

Group Type EXPERIMENTAL

Enzalutamide Oral Capsule

Intervention Type DRUG

Detailed conditions for the use of the study treatments including dose and dosages are described in accordance with the marketing authorization in the SmPC (Summary of Product Characteristics).

Abiraterone Oral Tablet

Intervention Type DRUG

Detailed conditions for the use of the study treatment including dose and dosages are described in accordance with the marketing authorisation in the SmPC.

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.

Group Type EXPERIMENTAL

Niraparib plus Abiraterone acetate plus Prednisone

Intervention Type DRUG

Niraparib and Abiraterone acetate will be provided by Janssen and will be provided either as a fixed dose combination or as single agents. Detailed use of the study treatment including dose and dosages are described in the Investigator's brochures and SmPC.

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.

Group Type EXPERIMENTAL

Capivasertib plus Docetaxel

Intervention Type DRUG

Capivasertib is provided by AstraZeneca and will be given in combination with Docetaxel. All subjects will be given up to ten 21-day docetaxel cycles. All subjects will receive Capivasertib, which will be administered as tablets taken twice a day orally, on a 4 days on/3 days off continuous schedule, commencing cycle one, day 2, until disease progression.

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.

Group Type EXPERIMENTAL

Carboplatin

Intervention Type DRUG

Carboplatin will be administered every 3rd week with an AUC (area under curve) = 5 with a dose calculated according to the Carboplatin AUC Dose calculation (Calvert formula):Dose (mg) = TargetAUC (mg/ml x min) x \[GFR ml/min + 25\].

Interventions

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Enzalutamide Oral Capsule

Detailed conditions for the use of the study treatments including dose and dosages are described in accordance with the marketing authorization in the SmPC (Summary of Product Characteristics).

Intervention Type DRUG

Abiraterone Oral Tablet

Detailed conditions for the use of the study treatment including dose and dosages are described in accordance with the marketing authorisation in the SmPC.

Intervention Type DRUG

Carboplatin

Carboplatin will be administered every 3rd week with an AUC (area under curve) = 5 with a dose calculated according to the Carboplatin AUC Dose calculation (Calvert formula):Dose (mg) = TargetAUC (mg/ml x min) x \[GFR ml/min + 25\].

Intervention Type DRUG

Cabazitaxel 60 mg Solution for Injection

Detailed conditions for the use of the study treatment including dose and dosages are described in accordance with the marketing authorisation in the SmPC.

Intervention Type DRUG

Docetaxel Injectable Solution

Detailed conditions for the use of the study treatment including dose and dosages are described in accordance with the marketing authorisation in the SmPC.

Intervention Type DRUG

Radium Chloride Ra-223

Detailed conditions for the use of the study treatment including dose and dosages are described in accordance with the marketing authorisation in the SmPC.

Intervention Type DRUG

Niraparib plus Abiraterone acetate plus Prednisone

Niraparib and Abiraterone acetate will be provided by Janssen and will be provided either as a fixed dose combination or as single agents. Detailed use of the study treatment including dose and dosages are described in the Investigator's brochures and SmPC.

Intervention Type DRUG

Capivasertib plus Docetaxel

Capivasertib is provided by AstraZeneca and will be given in combination with Docetaxel. All subjects will be given up to ten 21-day docetaxel cycles. All subjects will receive Capivasertib, which will be administered as tablets taken twice a day orally, on a 4 days on/3 days off continuous schedule, commencing cycle one, day 2, until disease progression.

Intervention Type DRUG

Apalutamide

Detailed conditions for the use of the study treatments including dose and dosages are described in accordance with the marketing authorization in the SmPC (Summary of Product Characteristics).

Intervention Type DRUG

Darolutamide

Detailed conditions for the use of the study treatments including dose and dosages are described in accordance with the marketing authorization in the SmPC (Summary of Product Characteristics).

Intervention Type DRUG

Other Intervention Names

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Xtandi Zytiga Xofigo Akeega Erleada Nubeqa

Eligibility Criteria

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Inclusion 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

Exclusion Criteria

* 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 subjects 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.
Minimum Eligible Age

18 Years

Eligible Sex

MALE

Accepts Healthy Volunteers

No

Sponsors

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The Swedish Research Council

OTHER_GOV

Sponsor Role collaborator

Kom Op Tegen Kanker

OTHER

Sponsor Role collaborator

Janssen Pharmaceutica N.V., Belgium

INDUSTRY

Sponsor Role collaborator

AstraZeneca

INDUSTRY

Sponsor Role collaborator

Cancerfonden

UNKNOWN

Sponsor Role collaborator

Karolinska Institutet

OTHER

Sponsor Role lead

Responsible Party

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Henrik Grönberg

Professor of Cancer Epidemiology

Responsibility Role PRINCIPAL_INVESTIGATOR

Principal Investigators

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Henrik Grönberg, Professor

Role: PRINCIPAL_INVESTIGATOR

Karolinska Institutet

Martin Eklund, Professor

Role: STUDY_DIRECTOR

Karolinska Institutet

Johan Lindberg, PhD

Role: STUDY_DIRECTOR

Karolinska Institutet

Piet Ost, Professor

Role: PRINCIPAL_INVESTIGATOR

University Hospital Ghent, Belgium

Jan Oldenburg, Professor

Role: PRINCIPAL_INVESTIGATOR

University Hospital, Akershus

Ashkan Mortezavi, MD, PhD

Role: PRINCIPAL_INVESTIGATOR

University Hospital, Basel, Switzerland

Locations

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OLV Ziekenhuis Aalst

Aalst, , Belgium

Site Status RECRUITING

GZA Sint-Augustinus

Antwerp, , Belgium

Site Status RECRUITING

AZ Sint-Jan AV

Bruges, , Belgium

Site Status RECRUITING

AZ Sint-Lucas

Bruges, , Belgium

Site Status RECRUITING

Ziekenhuis Oost-Limburg

Genk, , Belgium

Site Status RECRUITING

University Hospital Ghent

Ghent, , Belgium

Site Status RECRUITING

AZ Jan Palfijn Ziekenhuis

Ghent, , Belgium

Site Status NOT_YET_RECRUITING

Jessa ziekenhuis

Hasselt, , Belgium

Site Status RECRUITING

AZ Groeninge

Kortrijk, , Belgium

Site Status RECRUITING

University Hospital Luik

Liège, , Belgium

Site Status RECRUITING

AZ Damiaan

Ostend, , Belgium

Site Status RECRUITING

VITAZ

Sint-Niklaas, , Belgium

Site Status RECRUITING

Ålesund Sjukehus

Ålesund, , Norway

Site Status NOT_YET_RECRUITING

Kreftsenter Kristiansand

Kristiansand, , Norway

Site Status RECRUITING

Akershus Universitetssykehus

Lørenskog, , Norway

Site Status RECRUITING

Stavanger Universitetssjukehus

Stavanger, , Norway

Site Status RECRUITING

Universitetssykehuset Nord-Norge Tromsö

Tromsø, , Norway

Site Status NOT_YET_RECRUITING

Falu lasarett

Falun, Region Dalarna, Sweden

Site Status RECRUITING

Södra Alvsborgs sjukhus

Borås, , Sweden

Site Status RECRUITING

Länssjukhuset Ryhov - Onkologiska kliniken

Jönköping, , Sweden

Site Status RECRUITING

Länssjukhuset

Kalmar, , Sweden

Site Status RECRUITING

Centralsjukhuset Region Värmland

Karlstad, , Sweden

Site Status RECRUITING

Universitetssjukhuset Örebro

Örebro, , Sweden

Site Status RECRUITING

Karolinska University Hospital

Stockholm, , Sweden

Site Status RECRUITING

Capio St.Görans Hospital

Stockholm, , Sweden

Site Status RECRUITING

Länssjukhuset Sundsvall Härnösand

Sundsvall, , Sweden

Site Status RECRUITING

Norrlands Universitetssjukhus

Umeå, , Sweden

Site Status RECRUITING

Akademiska sjukhuset

Uppsala, , Sweden

Site Status RECRUITING

Hallands sjukhus Varberg

Varberg, , Sweden

Site Status RECRUITING

Centrallasarettet Onkologkliniken

Vaxjo, , Sweden

Site Status RECRUITING

St. Claraspital

Basel, , Switzerland

Site Status RECRUITING

Universitätsspital Basel

Basel, , Switzerland

Site Status RECRUITING

Countries

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Belgium Norway Sweden Switzerland

Central Contacts

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Berit Larsson, MSc

Role: CONTACT

+46 8 52482576

Henrik Grönberg, Professor

Role: CONTACT

+46 70 3411356

Facility Contacts

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Bram De Laere, PhD

Role: primary

Bram De Laere, PhD

Role: primary

Bram De Laere, PhD

Role: primary

Bram De Laere, PhD

Role: primary

Bram De Laere, PhD

Role: primary

Bram De Laere, PhD

Role: primary

Bram De Laere, PhD

Role: primary

Bram De Laere, PhD

Role: primary

Bram De Laere, PhD

Role: primary

Bram De Laere, PhD

Role: primary

Bram De Laere, PhD

Role: primary

Bram De Laere, PhD

Role: primary

Berit Larsson, MSc

Role: primary

Maria Persson, RN

Role: primary

Berit Larsson, MSc

Role: primary

Berit Larsson, MSc

Role: primary

Berit Larsson, MSc

Role: primary

Berit Larsson, MSc

Role: primary

Role: backup

Susanne Johansson, B.Sc.

Role: primary

Berit Larsson, MSc

Role: primary

Berit Larsson, MSc

Role: primary

Berit Larsson, MSc

Role: primary

Susanne Johansson, B.Sc.

Role: primary

Berit Larsson, MSc

Role: primary

Henrik Grönberg, Professor

Role: primary

Berit Larsson, MSc

Role: backup

Berit Larsson, MSc

Role: primary

Berit Larsson, MSc

Role: primary

Berit Larsson, MSc

Role: primary

Susanne Johansson, B.Sc.

Role: primary

Berit Larsson, MSc

Role: primary

Maria Persson, RN

Role: primary

Maria Persson, RN

Role: primary

References

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Crippa A, Laere B, Discacciati A, Larsson B, Persson M, Johansson S, D'hondt S, Hjalm-Eriksson M, Pettersson L, Enblad G, Ullen A, Lumen N, Karlsson CT, Sandzen J, Janes E, Ghysel C, Olsson M, Sautois B, Schatteman P, Roock W, Bruwaene SV, Verbiene I, Darras J, Everaert E, Maeseneer D, Anden M, Strijbos M, Luyten D, Mortezavi A, Oldenburg J, Ost P, Lindberg J, Gronberg H, Eklund M; ProBio Investigators. Prognostic Value of the Circulating Tumor DNA Fraction in Metastatic Castration-resistant Prostate Cancer: Results from the ProBio Platform Trial. Eur Urol Oncol. 2025 Apr 21:S2588-9311(25)00037-9. doi: 10.1016/j.euo.2025.02.002. Online ahead of print.

Reference Type DERIVED
PMID: 40263079 (View on PubMed)

De Laere B, Crippa A, Discacciati A, Larsson B, Persson M, Johansson S, D'hondt S, Bergstrom R, Chellappa V, Mayrhofer M, Banijamali M, Kotsalaynen A, Schelstraete C, Vanwelkenhuyzen JP, Hjalm-Eriksson M, Pettersson L, Ullen A, Lumen N, Enblad G, Thellenberg Karlsson C, Janes E, Sandzen J, Schatteman P, Nyre Vigmostad M, Olsson M, Ghysel C, Sautois B, De Roock W, Van Bruwaene S, Anden M, Verbiene I, De Maeseneer D, Everaert E, Darras J, Aksnessether BY, Luyten D, Strijbos M, Mortezavi A, Oldenburg J, Ost P, Eklund M, Gronberg H, Lindberg J. Androgen receptor pathway inhibitors and taxanes in metastatic prostate cancer: an outcome-adaptive randomized platform trial. Nat Med. 2024 Nov;30(11):3291-3302. doi: 10.1038/s41591-024-03204-2. Epub 2024 Aug 20.

Reference Type DERIVED
PMID: 39164518 (View on PubMed)

De Laere B, Crippa A, Discacciati A, Larsson B, Oldenburg J, Mortezavi A, Ost P, Eklund M, Lindberg J, Gronberg H; ProBio Investigators. Clinical Trial Protocol for ProBio: An Outcome-adaptive and Randomised Multiarm Biomarker-driven Study in Patients with Metastatic Prostate Cancer. Eur Urol Focus. 2022 Nov;8(6):1617-1621. doi: 10.1016/j.euf.2022.03.005. Epub 2022 Mar 19.

Reference Type DERIVED
PMID: 35317973 (View on PubMed)

Crippa A, De Laere B, Discacciati A, Larsson B, Connor JT, Gabriel EE, Thellenberg C, Janes E, Enblad G, Ullen A, Hjalm-Eriksson M, Oldenburg J, Ost P, Lindberg J, Eklund M, Gronberg H. The ProBio trial: molecular biomarkers for advancing personalized treatment decision in patients with metastatic castration-resistant prostate cancer. Trials. 2020 Jun 26;21(1):579. doi: 10.1186/s13063-020-04515-8.

Reference Type DERIVED
PMID: 32586393 (View on PubMed)

Other Identifiers

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EudraCT No 2018-002350-78

Identifier Type: -

Identifier Source: org_study_id

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