Everolimus Plus Best Supportive Care vs Placebo Plus Best Supportive Care in the Treatment of Patients With Advanced Neuroendocrine Tumors (GI or Lung Origin)

NCT ID: NCT01524783

Last Updated: 2021-08-05

Study Results

Results available

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Basic Information

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Recruitment Status

COMPLETED

Clinical Phase

PHASE3

Total Enrollment

302 participants

Study Classification

INTERVENTIONAL

Study Start Date

2012-03-30

Study Completion Date

2020-08-07

Brief Summary

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The purpose of this study is to compare the antitumor activity of everolimus plus best supportive care versus placebo plus best supportive care in patients with progressive nonfunctional neuroendocrine tumor (NET) of gastrointestinal (GI) or lung origin without a history of, or current symptoms of carcinoid syndrome.

Detailed Description

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This was a prospective, multi-center, randomized, double-blind, parallel-group, placebo-controlled, two-arm Phase III study comparing the efficacy and safety of everolimus 10 mg daily to placebo in patients with advanced NET of GI or lung origin without a history of, or current symptoms of carcinoid syndrome.

After assessment of eligibility, participants qualifying for the study were randomized in a 2:1 ratio to receive either everolimus or matching placebo. Participants received daily oral doses of 10 mg everolimus or matching placebo as study drug. In both arms, the study drug was combined with best supportive care and treatment cycles were defined as 28 days. Participants were treated until disease progression as per Response Evaluation Criteria In Solid Tumors (RECIST) 1.0, intolerable toxicity, death, lost to follow-up or consent withdrawal. Regardless of the reason for study drug discontinuation, participants had a safety follow-up visit scheduled 30 days after the last dose of the study drug.

Per data monitoring committee recommendation, all participants on treatment with placebo were allowed to crossover to open-label treatment with everolimus. This change was implemented through protocol amendment 3 (issued on 06-May-2016) after which remaining participants entered into open-label phase of the study.

Conditions

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Advanced NET of GI Origin Advanced NET of Lung Origin Neuroendocrine Tumors

Study Design

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Allocation Method

RANDOMIZED

Intervention Model

PARALLEL

Primary Study Purpose

TREATMENT

Blinding Strategy

QUADRUPLE

Participants Caregivers Investigators Outcome Assessors

Study Groups

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Everolimus + BSC

Participants received everolimus 10 mg once daily plus best supportive care (BSC) throughout the study

Group Type EXPERIMENTAL

Everolimus

Intervention Type DRUG

Participants were treated with everolimus 10 mg (two 5 mg tablets) once daily orally taken

Best suportive care (BSC)

Intervention Type OTHER

Best supportive care includes all care provided to participants deemed necessary by the treating physician, such as but not restricted to anti-diarrheals and analgesics. The optimal care of the patient is based on the treating physician's best medical judgment.

Placebo + BSC

Participants received matching placebo once daily plus best supportive care (BSC) during the blinded period. Participants were allowed to crossover to treatment with everolimus 10mg once daily plus BSC during the open-label period.

Group Type PLACEBO_COMPARATOR

Placebo

Intervention Type DRUG

Participants were treated with two tablets of matching placebo once daily orally taken.

Best suportive care (BSC)

Intervention Type OTHER

Best supportive care includes all care provided to participants deemed necessary by the treating physician, such as but not restricted to anti-diarrheals and analgesics. The optimal care of the patient is based on the treating physician's best medical judgment.

Interventions

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Everolimus

Participants were treated with everolimus 10 mg (two 5 mg tablets) once daily orally taken

Intervention Type DRUG

Placebo

Participants were treated with two tablets of matching placebo once daily orally taken.

Intervention Type DRUG

Best suportive care (BSC)

Best supportive care includes all care provided to participants deemed necessary by the treating physician, such as but not restricted to anti-diarrheals and analgesics. The optimal care of the patient is based on the treating physician's best medical judgment.

Intervention Type OTHER

Other Intervention Names

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RAD001

Eligibility Criteria

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Inclusion Criteria

* Pathologically confirmed, well differentiated (G1 or G2), advanced (unresectable or metastatic), neuroendocrine tumor of GI or lung origin
* No history of and no active symptoms related to carcinoid syndrome
* In addition to treatment-naive patients, patients previously treated with SSA, Interferon (IFN), one prior line of chemotherapy, and/or PRRT were allowed into the study. Pretreated patients had to have progressed on or after the last treatment
* Radiological documented disease progression within 6 months prior to randomization
* Measurable disease
* WHO performance status ≤1
* Adequate bone marrow, liver and renal function

Exclusion Criteria

* Patients with poorly differentiated neuroendocrine carcinoma, high-grade neuroendocrine carcinoma, adenocarcinoid, pancreatic islet cell carcinoma, insulinoma, glucagonoma, gastrinoma, goblet cell carcinoid, large cell neuroendocrine carcinoma and small cell carcinoma
* Patients with pancreatic NET or NET of origins other than GI or Lung
* Patients with history of or active symptoms of carcinoid syndrome (e.g. flushing, diarrhea)
* Patients with more than one line of prior chemotherapy
* Prior targeted therapy
* Hepatic intra-arterial embolization within the last 6 months. Cryoablation or radiofrequency ablation of hepatic metastases within 2 months of randomization
* Prior therapy with mTOR inhibitors (e.g. sirolimus, temsirolimus, deforolimus)
* Known intolerance or hypersensitivity to everolimus or other rapamycin analogs (e.g. sirolimus, temsirolimus)
* Known impairment of gastrointestinal (GI) function or GI disease that may significantly alter the absorption of oral everolimus
* Uncontrolled diabetes mellitus as defined by HbA1c \>8% despite adequate therapy
* Patients who had any severe and/or uncontrolled medical conditions such as:

* unstable angina pectoris, symptomatic congestive heart failure, myocardial infarction ≤6 months prior to randomization, serious uncontrolled cardiac arrhythmia
* active or uncontrolled severe infection
* liver disease such as cirrhosis, decompensated liver disease, and chronic hepatitis (i.e. quantifiable HBV-DNA and/or positive HbsAg, quantifiable HCV-RNA)
* Chronic treatment with corticosteroids or other immunosuppressive agents
* Known history of HIV seropositivity
* Pregnant or nursing (lactating) women
Minimum Eligible Age

18 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Novartis Pharmaceuticals

INDUSTRY

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Principal Investigators

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Novartis Pharmaceuticals

Role: STUDY_DIRECTOR

Novartis Pharmaceuticals

Locations

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University of California San Diego - Moores Cancer Center Regulatory

La Jolla, California, United States

Site Status

Scripps Clinic Regulatory

La Jolla, California, United States

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Cedars Sinai Medical Center SC

Los Angeles, California, United States

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University of Colorado Cancer Centre SC

Aurora, Colorado, United States

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H Lee Moffitt Cancer Center and Research Institute HLM

Tampa, Florida, United States

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University of Chicago UC SC

Chicago, Illinois, United States

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Goshen Center for Cancer Care IU Health SC

Indianapolis, Indiana, United States

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Dana Farber Cancer Institute SC

Boston, Massachusetts, United States

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Memorial Sloan Kettering MSkCC SC

New York, New York, United States

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Montefiore Medical Center MMC

The Bronx, New York, United States

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Oregon Health and Science University OH&SU

Portland, Oregon, United States

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Vanderbilt University Medical Center Vanderbilt Med Ctr

Nashville, Tennessee, United States

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Texas Oncology P A Texas Oncology Amarillo

Dallas, Texas, United States

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Texas Oncology P A TX Oncology Baylor

Dallas, Texas, United States

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University of Texas Southwestern Medical Center

Dallas, Texas, United States

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University of Texas MD Anderson Cancer Center UT MD Anderson Cancer Ctr

Houston, Texas, United States

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University of Wisconsin / Paul P. Carbone Comp Cancer Center Univ Wisc

Madison, Wisconsin, United States

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Novartis Investigative Site

Innsbruck, Tyrol, Austria

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Vienna, , Austria

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Edegem, Antwerpen, Belgium

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Brussels, , Belgium

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Ghent, , Belgium

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Leuven, , Belgium

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Calgary, Alberta, Canada

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Vancouver, British Columbia, Canada

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Halifax, Nova Scotia, Canada

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London, Ontario, Canada

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Ottawa, Ontario, Canada

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Toronto, Ontario, Canada

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Montreal, Quebec, Canada

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Beijing, Beijing Municipality, China

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Beijing, , China

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Beijing, , China

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Beijing, , China

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Beijing, , China

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Bogotá, Cundinamarca, Colombia

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Brno, , Czechia

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Olomouc, , Czechia

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Prague, , Czechia

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Bad Berka, , Germany

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Berlin, , Germany

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Essen, , Germany

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Frankfurt, , Germany

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Hanover, , Germany

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Magdeburg, , Germany

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Mainz, , Germany

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Athens, , Greece

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Budapest, , Hungary

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Budapest, , Hungary

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Bologna, BO, Italy

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Brescia, BS, Italy

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Viagrande, CT, Italy

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Florence, FI, Italy

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Milan, MI, Italy

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Milan, MI, Italy

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Rozzano, MI, Italy

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Modena, MO, Italy

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Roma, RM, Italy

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Roma, RM, Italy

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Verona, VR, Italy

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Napoli, , Italy

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Napoli, , Italy

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Fukuoka, Fukuoka, Japan

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Osaka, Osaka, Japan

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Chuo Ku, Tokyo, Japan

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Beirut, , Lebanon

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El Achrafiyé, , Lebanon

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Amsterdam, , Netherlands

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Gliwice, Silesian Voivodeship, Poland

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Poznan, , Poland

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Rostov-on-Don, , Russia

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Riyadh, , Saudi Arabia

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Bratislava, Slovak Republic, Slovakia

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Parktown, , South Africa

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Seoul, Korea, South Korea

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Seoul, Seocho Gu, South Korea

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Seoul, , South Korea

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Seoul, , South Korea

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Seoul, , South Korea

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Seville, Andalusia, Spain

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Barcelona, Catalonia, Spain

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L'Hospitalet de Llobregat, Catalonia, Spain

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Taipei, Taiwan, ROC, Taiwan

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Kuei-Shan Chiang, Taoyuan/ Taiwan ROC, Taiwan

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Kaohsiung City, , Taiwan

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Taichung, , Taiwan

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Taipei, , Taiwan

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Bangkok, THA, Thailand

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Chiang Mai, , Thailand

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Gaziantep, , Turkey (Türkiye)

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Istanbul, , Turkey (Türkiye)

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Glasgow, Scotland, United Kingdom

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Cambridge, , United Kingdom

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London, , United Kingdom

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London, , United Kingdom

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Manchester, , United Kingdom

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Southampton, , United Kingdom

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Countries

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United States Austria Belgium Canada China Colombia Czechia Germany Greece Hungary Italy Japan Lebanon Netherlands Poland Russia Saudi Arabia Slovakia South Africa South Korea Spain Taiwan Thailand Turkey (Türkiye) United Kingdom

References

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Chan DL, Yao JC, Carnaghi C, Buzzoni R, Herbst F, Ridolfi A, Strosberg J, Kulke MH, Pavel M, Singh S. Markers of Systemic Inflammation in Neuroendocrine Tumors: A Pooled Analysis of the RADIANT-3 and RADIANT-4 Studies. Pancreas. 2021 Feb 1;50(2):130-137. doi: 10.1097/MPA.0000000000001745.

Reference Type DERIVED
PMID: 33560090 (View on PubMed)

Buzzoni R, Carnaghi C, Strosberg J, Fazio N, Singh S, Herbst F, Ridolfi A, Pavel ME, Wolin EM, Valle JW, Oh DY, Yao JC, Pommier R. Impact of prior therapies on everolimus activity: an exploratory analysis of RADIANT-4. Onco Targets Ther. 2017 Oct 16;10:5013-5030. doi: 10.2147/OTT.S142087. eCollection 2017.

Reference Type DERIVED
PMID: 29081664 (View on PubMed)

Fazio N, Buzzoni R, Delle Fave G, Tesselaar ME, Wolin E, Van Cutsem E, Tomassetti P, Strosberg J, Voi M, Bubuteishvili-Pacaud L, Ridolfi A, Herbst F, Tomasek J, Singh S, Pavel M, Kulke MH, Valle JW, Yao JC. Everolimus in advanced, progressive, well-differentiated, non-functional neuroendocrine tumors: RADIANT-4 lung subgroup analysis. Cancer Sci. 2018 Jan;109(1):174-181. doi: 10.1111/cas.13427. Epub 2017 Nov 9.

Reference Type DERIVED
PMID: 29055056 (View on PubMed)

Pavel ME, Singh S, Strosberg JR, Bubuteishvili-Pacaud L, Degtyarev E, Neary MP, Carnaghi C, Tomasek J, Wolin E, Raderer M, Lahner H, Valle JW, Pommier R, Van Cutsem E, Tesselaar MET, Fave GD, Buzzoni R, Hunger M, Eriksson J, Cella D, Ricci JF, Fazio N, Kulke MH, Yao JC. Health-related quality of life for everolimus versus placebo in patients with advanced, non-functional, well-differentiated gastrointestinal or lung neuroendocrine tumours (RADIANT-4): a multicentre, randomised, double-blind, placebo-controlled, phase 3 trial. Lancet Oncol. 2017 Oct;18(10):1411-1422. doi: 10.1016/S1470-2045(17)30471-0. Epub 2017 Aug 30.

Reference Type DERIVED
PMID: 28838862 (View on PubMed)

Yao JC, Fazio N, Singh S, Buzzoni R, Carnaghi C, Wolin E, Tomasek J, Raderer M, Lahner H, Voi M, Pacaud LB, Rouyrre N, Sachs C, Valle JW, Fave GD, Van Cutsem E, Tesselaar M, Shimada Y, Oh DY, Strosberg J, Kulke MH, Pavel ME; RAD001 in Advanced Neuroendocrine Tumours, Fourth Trial (RADIANT-4) Study Group. Everolimus for the treatment of advanced, non-functional neuroendocrine tumours of the lung or gastrointestinal tract (RADIANT-4): a randomised, placebo-controlled, phase 3 study. Lancet. 2016 Mar 5;387(10022):968-977. doi: 10.1016/S0140-6736(15)00817-X. Epub 2015 Dec 17.

Reference Type DERIVED
PMID: 26703889 (View on PubMed)

Other Identifiers

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2011-002887-26

Identifier Type: REGISTRY

Identifier Source: secondary_id

CRAD001T2302

Identifier Type: -

Identifier Source: org_study_id

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