NCT01193257 is a Phase 3 clinical trial of Orteronel in Prostate Cancer, sponsored by Millennium Pharmaceuticals, Inc..

Who is sponsoring NCT01193257?

NCT01193257 is sponsored by Millennium Pharmaceuticals, Inc..

What drugs are being tested in NCT01193257?

Interventions in NCT01193257: Orteronel, Prednisone, Orteronel Placebo.

What condition does NCT01193257 study?

NCT01193257 studies Prostate Cancer.

Is NCT01193257 still recruiting?

NCT01193257 is currently completed. Last updated 19 December 2018.

Are results published for NCT01193257?

Yes — primary outcome results have been posted to ClinicalTrials.gov. See the outcomes section above for details.

Last reviewed 2018-12-19 · How we verify

NCT01193257

Name: Drug Landscape Pharmaceutical Database
Creator: Drug Landscape
Published: 2026-01-01
License: https://creativecommons.org/licenses/by/4.0/

Study Comparing Orteronel Plus Prednisone in Participants With Metastatic Castration-Resistant Prostate Cancer.

Completed Phase 3 Results posted Last updated 19 December 2018

What this trial tests

Phase 3 trial testing Orteronel in Prostate Cancer in 1,099 participants. Completed in 29 February 2016.

Timeline

15 November 2010

Primary endpoint
16 May 2013

29 February 2016

Quick facts

Lead sponsor	Millennium Pharmaceuticals, Inc.
Phase	Phase 3
Status	Completed
Study type	INTERVENTIONAL
Allocation	randomized
Design	parallel
Masking	double
Primary purpose	treatment
Enrollment	1,099
Start date	15 November 2010
Primary completion	16 May 2013
Estimated completion	29 February 2016
Sites	78 locations across France, Italy, Finland, Netherlands, Greece, Belgium, Estonia, Hungary

Drugs / interventions tested

Orteronel — full drug profile →
Prednisone (prednisone) — full drug profile →
Orteronel Placebo — full drug profile →

Conditions studied

Prostate Cancer — all drugs for Prostate Cancer →

Sponsor

Millennium Pharmaceuticals, Inc. — full company profile →

Who can join

18 and older, male only, with Prostate Cancer. Patients with the condition only — healthy volunteers not accepted.

Results — posted to ClinicalTrials.gov

Per-arm endpoint measurements with 95% confidence intervals where reported. Source: trial results section.

Overall Survival Primary · Baseline until death (approximately up to 4.5 years)

Overall survival was calculated from the date of participant randomization to the date of participant death due to any cause. Participants without documentation of death at time of the analysis were censored as of the date the participant was last known to be alive, or the data cutoff date, whichever was earlier.

Group	Value	95% CI
Placebo + Prednisone	15.3	13.48 – 16.86
Orteronel + Prednisone	17.1	15.45 – 18.67

Radiographic Progression-free Survival (rPFS) Secondary · Baseline until disease progression or death, whichever occurred first (approximately up to 4.5 years)

rPFS was defined as the time from randomization until radiographic disease progression or death due to any cause, whichever occurred first. Radiographic disease progression was defined as the occurrence of 1 or more of the following: The appearance of 2 or more new lesions on radionuclide bone scan as defined by prostate cancer working group (PCWG)2; Should 2 or more new bone lesions be evident at the first assessment (8-week assessment) on treatment, 2 or more additional new lesions must have been evident on a confirmatory assessment at least 6 weeks later; One or more new soft tissue/viscera

Group	Value	95% CI
Placebo + Prednisone	5.7	5.46 – 6.97
Orteronel + Prednisone	8.3	7.76 – 8.48

Percentage of Participants Achieving 50 Percent Reduction From Baseline in Prostate Specific Antigen (PSA50 Response) at Week 12 Secondary · Week 12

The PSA50 was defined as the percentage of participants who had a PSA decline of at least 50 percent (%) from baseline.

Group	Value	95% CI
Placebo + Prednisone	9.9
Orteronel + Prednisone	24.9

Percentage of Participants With Pain Response at Week 12 Secondary · Week 12

Pain response was defined as the occurrence of 1 of the following and confirmed by an additional assessment, at least 3 weeks but not more than 5 weeks later: A greater than or equal to (\>=) 2 point reduction from baseline in BPI-SF worst pain score without an increase in analgesic use; or a 25 percent (%) or more reduction in analgesic use from baseline without an increase in worst pain score from baseline.

Group	Value	95% CI
Placebo + Prednisone	9.0
Orteronel + Prednisone	12.1

Number of Participants Reporting One or More Treatment-emergent Adverse Events (TEAEs) Secondary · Baseline up to 30 days after last dose of study drug (Cycle 59 Day 58)

Group	Value	95% CI
Placebo + Prednisone	345
Orteronel + Prednisone	719

Number of Participants With Abnormal Physical Examination Findings Secondary · Baseline up to 30 days after last dose of study drug (Cycle 59 Day 58)

Group	Value	95% CI
Placebo + Prednisone	0
Orteronel + Prednisone	1

Number of Participants With TEAEs Related to Vital Signs Secondary · Baseline up to 30 days after last dose of study drug (Cycle 59 Day 58)

Hypertension

Group	Value	95% CI
Placebo + Prednisone	21
Orteronel + Prednisone	83

Hypotension

Group	Value	95% CI
Placebo + Prednisone	8
Orteronel + Prednisone	31

Pyrexia

Group	Value	95% CI
Placebo + Prednisone	18
Orteronel + Prednisone	51

Number of Participants With TEAEs Related to Weight Secondary · Baseline up to 30 days after last dose of study drug (Cycle 59 Day 58)

Weight decreased

Group	Value	95% CI
Placebo + Prednisone	32
Orteronel + Prednisone	107

Weight increased

Group	Value	95% CI
Placebo + Prednisone	7
Orteronel + Prednisone	6

Number of Participants With Worst Change From Baseline in Eastern Co-operative Oncology Group (ECOG) Performance Status Secondary · Baseline up to End-of-treatment (EOT) (Cycle 59 Day 58)

ECOG assessed participant's performance status on 5 point scale: 0=Fully active/able to carry on all pre-disease activities without restriction; 1=restricted in physically strenuous activity, ambulatory/able to carry out light or sedentary work; 2=ambulatory (\>50% of waking hours \[hrs\]), capable of all self care, unable to carry out any work activities; 3=capable of only limited self care, confined to bed/chair \>50% of waking hrs; 4=completely disabled, cannot carry on any self care, totally confined to bed/chair; 5=dead. Worst change was defined as the worst overall change that occurred i

Baseline: 0; Overall: 0

Group	Value	95% CI
Placebo + Prednisone	56
Orteronel + Prednisone	112

Baseline: 0; Overall: 1

Group	Value	95% CI
Placebo + Prednisone	70
Orteronel + Prednisone	121

Baseline: 0; Overall: 2

Group	Value	95% CI
Placebo + Prednisone	13
Orteronel + Prednisone	47

Baseline: 0; Overall: 3

Group	Value	95% CI
Placebo + Prednisone	5
Orteronel + Prednisone	18

Baseline: 0; Overall: 4

Group	Value	95% CI
Placebo + Prednisone	1
Orteronel + Prednisone	3

Baseline: 1; Overall: 0

Group	Value	95% CI
Placebo + Prednisone	3
Orteronel + Prednisone	10

Baseline: 1; Overall: 1

Group	Value	95% CI
Placebo + Prednisone	103
Orteronel + Prednisone	179

Baseline: 1; Overall: 2

Group	Value	95% CI
Placebo + Prednisone	53
Orteronel + Prednisone	113

Number of Participants With Abnormal Clinically Significant Electrocardiogram (ECG) Findings Secondary · Cycle 59 Day 58

Group	Value	95% CI
Placebo + Prednisone	1
Orteronel + Prednisone	3

Number of Participants With TEAEs Categorized Into Investigations Related to Chemistry, Hematology or Steroid Hormone Panel Secondary · Baseline up to 30 days after last dose of study drug (Cycle 59 Day 58)

Digestive enzymes

Group	Value	95% CI
Placebo + Prednisone	9
Orteronel + Prednisone	140

Renal function analyses

Group	Value	95% CI
Placebo + Prednisone	13
Orteronel + Prednisone	41

Liver function analyses

Group	Value	95% CI
Placebo + Prednisone	14
Orteronel + Prednisone	38

Tissue enzyme analyses NEC

Group	Value	95% CI
Placebo + Prednisone	16
Orteronel + Prednisone	30

Coagulation and bleeding analyses

Group	Value	95% CI
Placebo + Prednisone	1
Orteronel + Prednisone	17

Mineral and electrolyte analyses

Group	Value	95% CI
Placebo + Prednisone	5
Orteronel + Prednisone	9

White blood cell analyses

Group	Value	95% CI
Placebo + Prednisone	4
Orteronel + Prednisone	8

Carbohydrate tolerance analyses-including diabetes

Group	Value	95% CI
Placebo + Prednisone	0
Orteronel + Prednisone	8

Percentage of Participants Achieving PSA50 Response at Any Time During the Study Secondary · Cycle: 4, 7, 10, 13, 16, 19, 22, and 25

The PSA50 was defined as the percentage of participants who had a PSA decline of at least 50% from baseline.

Cycle 4 (n= 283; 559)

Group	Value	95% CI
Placebo + Prednisone	12.72
Orteronel + Prednisone	32.74

Cycle 7 (n= 163; 403)

Group	Value	95% CI
Placebo + Prednisone	18.40
Orteronel + Prednisone	38.21

Cycle 10 (n= 102; 267)

Group	Value	95% CI
Placebo + Prednisone	22.55
Orteronel + Prednisone	36.70

Cycle 13 (n= 55; 171)

Group	Value	95% CI
Placebo + Prednisone	23.64
Orteronel + Prednisone	40.94

Cycle 16 (n= 34; 107)

Group	Value	95% CI
Placebo + Prednisone	23.53
Orteronel + Prednisone	44.86

Cycle 19 (n= 24; 68)

Group	Value	95% CI
Placebo + Prednisone	20.83
Orteronel + Prednisone	42.65

Cycle 22 (n= 14; 36)

Group	Value	95% CI
Placebo + Prednisone	28.57
Orteronel + Prednisone	52.78

Cycle 25 (n= 8; 16)

Group	Value	95% CI
Placebo + Prednisone	25.00
Orteronel + Prednisone	62.50

Adverse events — posted to ClinicalTrials.gov

Time frame: Treatment-emergent adverse events are adverse events that started after the first dose of double-blind study drug and no more than 30 days (Cycle 59 Day 58) after the last dose of study drug.. Reporting threshold: 5%. Adverse-event reports describe events observed during the trial — not all are caused by the drug.

Placebo + Prednisone

Serious: 148/363 (41%)

Deaths: —

Orteronel + Prednisone

Serious: 384/732 (52%)

Deaths: —

Serious adverse events (337 terms)

Reaction	System	Placebo + Prednisone	Orteronel + Prednisone
Prostate cancer	Neoplasms benign, malignant and unspecified (incl cysts and polyps)	—	—
Anaemia	Blood and lymphatic system disorders	—	—
Urinary tract infection	Infections and infestations	—	—
Pulmonary embolism	Respiratory, thoracic and mediastinal disorders	—	—
Pneumonia	Infections and infestations	—	—
General physical health deterioration	General disorders	—	—
Vomiting	Gastrointestinal disorders	—	—
Spinal cord compression	Nervous system disorders	—	—
Dehydration	Metabolism and nutrition disorders	—	—
Sepsis	Infections and infestations	—	—
Urinary retention	Renal and urinary disorders	—	—
Bone pain	Musculoskeletal and connective tissue disorders	—	—
Nausea	Gastrointestinal disorders	—	—
Urosepsis	Infections and infestations	—	—
Acute kidney injury	Renal and urinary disorders	—	—
Lipase increased	Investigations	—	—
Renal failure	Renal and urinary disorders	—	—
Abdominal pain	Gastrointestinal disorders	—	—
Fatigue	General disorders	—	—
Asthenia	General disorders	—	—
Back pain	Musculoskeletal and connective tissue disorders	—	—
Hyperkalaemia	Metabolism and nutrition disorders	—	—
Hyperglycaemia	Metabolism and nutrition disorders	—	—
Metastatic pain	Neoplasms benign, malignant and unspecified (incl cysts and polyps)	—	—
Haematuria	Renal and urinary disorders	—	—

Other adverse events (34 terms — click to expand)

Reaction	System	Placebo + Prednisone	Orteronel + Prednisone
Nausea	Gastrointestinal disorders	—	—
Vomiting	Gastrointestinal disorders	—	—
Fatigue	General disorders	—	—
Constipation	Gastrointestinal disorders	—	—
Decreased appetite	Metabolism and nutrition disorders	—	—
Diarrhoea	Gastrointestinal disorders	—	—
Back pain	Musculoskeletal and connective tissue disorders	—	—
Weight decreased	Investigations	—	—
Lipase increased	Investigations	—	—
Arthralgia	Musculoskeletal and connective tissue disorders	—	—
Muscle spasms	Musculoskeletal and connective tissue disorders	—	—
Anaemia	Blood and lymphatic system disorders	—	—
Asthenia	General disorders	—	—
Amylase increased	Investigations	—	—
Bone pain	Musculoskeletal and connective tissue disorders	—	—
Pain in extremity	Musculoskeletal and connective tissue disorders	—	—
Hypertension	Vascular disorders	—	—
Dizziness	Nervous system disorders	—	—
Oedema peripheral	General disorders	—	—
Headache	Nervous system disorders	—	—
Insomnia	Psychiatric disorders	—	—
Dyspnoea	Respiratory, thoracic and mediastinal disorders	—	—
Cough	Respiratory, thoracic and mediastinal disorders	—	—
Hot flush	Vascular disorders	—	—
Urinary tract infection	Infections and infestations	—	—
Musculoskeletal pain	Musculoskeletal and connective tissue disorders	—	—
Pyrexia	General disorders	—	—
Abdominal pain	Gastrointestinal disorders	—	—
Dyspepsia	Gastrointestinal disorders	—	—
Hypokalaemia	Metabolism and nutrition disorders	—	—
Myalgia	Musculoskeletal and connective tissue disorders	—	—
Musculoskeletal chest pain	Musculoskeletal and connective tissue disorders	—	—
Blood creatinine increased	Investigations	—	—
Muscular weakness	Musculoskeletal and connective tissue disorders	—	—

Most-reported serious reactions: Prostate cancer, Anaemia, Urinary tract infection, Pulmonary embolism, Pneumonia, General physical health deterioration, Vomiting, Spinal cord compression.

Data from ClinicalTrials.gov NCT01193257 adverse events section.

Sponsor's own description

This is a randomized, double-blind, multicenter, phase 3 study evaluating orteronel plus prednisone compared with placebo plus prednisone in men with metastatic, castration-resistant prostate cancer (mCRPC) that has progressed following Docetaxel-based therapy

Publications & conference data

8 peer-reviewed publications reference this trial (live from Europe PMC):

Targeting signaling pathways in prostate cancer: mechanisms and clinical trials.
He Y, Xu W, Xiao YT, Huang H, et al · · 2022 · cited 192× · PMID 35750683 · DOI 10.1038/s41392-022-01042-7
Circulating tumor cells as biomarkers in prostate cancer.
Danila DC, Fleisher M, Scher HI. · · 2011 · cited 178× · PMID 21680546 · DOI 10.1158/1078-0432.ccr-10-2650
Circulating Tumor Cell Number as a Response Measure of Prolonged Survival for Metastatic Castration-Resistant Prostate Cancer: A Comparison With Prostate-Specific Antigen Across Five Randomized Phase III Clinical Trials.
Heller G, McCormack R, Kheoh T, Molina A, et al · · 2018 · cited 175× · PMID 29272162 · DOI 10.1200/jco.2017.75.2998
Targeting the androgen receptor pathway in castration-resistant prostate cancer: progresses and prospects.
Ferraldeschi R, Welti J, Luo J, Attard G, et al · · 2015 · cited 151× · PMID 24837363 · DOI 10.1038/onc.2014.115
End points and outcomes in castration-resistant prostate cancer: from clinical trials to clinical practice.
Scher HI, Morris MJ, Basch E, Heller G. · · 2011 · cited 150× · PMID 21859988 · DOI 10.1200/jco.2011.35.8648
Androgen action and metabolism in prostate cancer.
Green SM, Mostaghel EA, Nelson PS. · · 2012 · cited 143× · PMID 22453214 · DOI 10.1016/j.mce.2011.09.046
New strategies in metastatic prostate cancer: targeting the androgen receptor signaling pathway.
Attard G, Richards J, de Bono JS. · · 2011 · cited 141× · PMID 21372223 · DOI 10.1158/1078-0432.ccr-10-0567
Biomarkers in the management and treatment of men with metastatic castration-resistant prostate cancer.
Armstrong AJ, Eisenberger MA, Halabi S, Oudard S, et al · · 2012 · cited 134× · PMID 22099611 · DOI 10.1016/j.eururo.2011.11.009

Verify or expand the search:

Other trials of Orteronel

Trials testing the same drug.

NCT02053311 — Orteronel Maintenance Therapy in Patients With mCRPC Previously Treated With Novel Hormonal Agents · Phase 3 · withdrawn
NCT01666314 — Study in Japan and Ex-Japan to Characterize the Pharmacokinetic and Pharmacodynamic Response to Orteronel (TAK-700) in C · Phase 1, PHASE2 · completed
NCT01193244 — Study Comparing Orteronel Plus Prednisone in Participants With Chemotherapy-Naive Metastatic Castration-Resistant Prosta · Phase 3 · completed

Other recruiting trials for Prostate Cancer

Currently open trials in the same condition.

NCT06960798 — Characterizing the Genomic Landscape of Prostate Cancer in Native American Populations (NAT-Geno) · recruiting
NCT07237269 — Abi/Pred + ADT vs ADT in PSMA-Positive, Conventionally Node-Negative Prostate Cancer · Phase 2 · recruiting
NCT07234981 — PSMA-PET Guided De-escalation of Salvage Radiation Treatment in Recurrent Prostate Cancer · Phase 2 · recruiting
NCT07027124 — Neoadjuvant ADT + Darolutamide With Pembrolizumab, Followed by Adjuvant Pembrolizumab in Molecularly Stratified High-Ris · Phase 2 · recruiting
NCT07426094 — PRO-BOOST-N: Prostate-First Versus Combined Prostate and Nodal Dose Escalation in PSMA PET-Staged Node-Positive Prostate · Phase 2, PHASE3 · recruiting

Other Millennium Pharmaceuticals, Inc. trials

Trials by the same sponsor.

NCT03888534 — Intravenous Ixazomib in Pediatric Participants With Relapsed or Refractory Acute Lymphoblastic Leukemia (ALL) or Lymphob · Phase 1 · withdrawn
NCT04056468 — A Study to Evaluate Pharmacokinetics (PK) and Safety of Oral Mobocertinib in Participants With Moderate or Severe Hepati · Phase 1 · completed
NCT04454918 — Study to Assess Absolute Bioavailability (ABA) of TAK-906 and to Characterize Mass Balance, Pharmacokinetics (PK), Metab · Phase 1 · completed
NCT04056455 — A Study of Mobocertinib Capsules in People With Severe Kidney Problems and People With Healthy Kidneys · Phase 1 · completed
NCT04091438 — A Study of a Single Intravenous Infusion Dose of TAK-925 in Participants With Idiopathic Hypersomnia · Phase 1 · completed

Verify against primary sources

ClinicalTrials.gov — authoritative US registry record
WHO ICTRP — international registry index
EU Clinical Trials Register
Sponsor press releases (Google)

Data sources for this page

Trial protocol + status: ClinicalTrials.gov NCT01193257 (US National Library of Medicine, public domain)
Publications: Europe PMC API search by NCT ID, retrieved 10 June 2026
Drug + disease cross-links: matched in real time against Drug Landscape's normalised drug + company + condition tables
Sponsor: as reported to ClinicalTrials.gov by Millennium Pharmaceuticals, Inc.
Last refreshed: 19 December 2018

Drug Landscape aggregates and links these public records for informational use only. Always verify against the primary source before clinical or regulatory decisions. Canonical URL: https://druglandscape.com/trial/NCT01193257.

Primary sources · FDA · ClinicalTrials.gov · EMA · SEC EDGAR · ChEMBL · Wikidata · full sourcing