A Study of Selpercatinib (LY3527723) in Participants With RET-Mutant Medullary Thyroid Cancer (LIBRETTO-531)

A Multicenter, Randomized, Open-label, Phase 3 Trial Comparing Selpercatinib to Physicians Choice of Cabozantinib or Vandetanib in Patients With Progressive, Advanced, Kinase Inhibitor Naïve, RET-Mutant Medullary Thyroid Cancer (LIBRETTO-531)

The reason for this study is to see if the study drug selpercatinib is safe and more effective compared to a standard treatment in participants with rearranged during transfection (RET)-mutant medullary thyroid cancer (MTC) that cannot be removed by surgery or has spread to other parts of the body. Participants who are assigned to the standard treatment and discontinue due to progressive disease have the option to potentially crossover to selpercatinib.

Study Overview

• Status: Active, not recruiting
• Conditions: Medullary Thyroid Cancer
• Intervention / Treatment: Drug: Selpercatinib; Drug: Cabozantinib; Drug: Vandetanib

Detailed Description

Adaptive sample size re-estimation will be performed at interim analysis. The sample size could be increased from approximately 250 to 400 depending on the results of interim analysis.

• Study Type: Interventional
• Enrollment (Actual): 291
• Phase: Phase 3

Expanded Access

Available outside the clinical trial. See expanded access record.

Contacts and Locations

Study Locations

• Australia
  • New South Wales
    • Camperdown, New South Wales, Australia, 2050
      • Chris O'Brien Lifehouse
    • St Leonards, New South Wales, Australia, 2065
      • Royal North Shore Hospital
  • Victoria
    • Melbourne, Victoria, Australia, 3004
      • The Alfred Hospital
    • Melbourne, Victoria, Australia, 3050
      • Peter MacCallum Cancer Centre
  • Western Australia
    • Perth, Western Australia, Australia, 6009
      • Sir Charles Gairdner Hospital
• Belgium
  • Liège, Belgium, 4000
    • Centre Hospitalier Universitaire de Liège - Domaine Universitaire du Sart Tilman
  • Antwerpen
    • Edegem, Antwerpen, Belgium, 2650
      • Antwerp University Hospital
• Brazil
  • Rio de Janeiro, Brazil, 20230-130
    • Instituto Nacional de Câncer - INCA
  • Rio de Janeiro, Brazil, 22250-905
    • Grupo Oncoclínicas Botafogo
  • Rio de Janeiro, Brazil, 22775-001
    • Grupo COI - Clínicas Oncológicas Integradas
  • São Paulo, Brazil, 01246-000
    • Icesp - Instituto Do Câncer Do Estado de São Paulo
  • São Paulo, Brazil, 01452-000
    • Centro Paulista de Oncologia Clínica
  • Minas Gerais
    • Belo Horizonte, Minas Gerais, Brazil, 30380-680
      • OncoCentro
  • Paraná
    • Londrina, Paraná, Brazil, 86015-520
      • Hospital de Cancer de Londrina
  • Rio Grande Do Sul
    • Porto Alegre, Rio Grande Do Sul, Brazil, 90035-903
      • Hospital de Clínicas de Porto Alegre
  • São Paulo
    • Barretos, São Paulo, Brazil, 14784400
      • Fundação Pio XII - Hospital de Câncer de Barretos
    • Campinas, São Paulo, Brazil, 13060-904
      • Centro de Pesquisa Sao Lucas
    • Ribeirão Preto, São Paulo, Brazil, 14051-140
      • Hospital de Clínicas de Ribeirão Preto
    • Sao Paulo, São Paulo, Brazil, 01308-060
      • Hospital Sirio Libanes
    • Sao Paulo, São Paulo, Brazil, 04543-000
      • Instituto D'Or de Pesquisa e Ensino (IDOR)
• Canada
  • Ontario
    • Toronto, Ontario, Canada, M5G 2M9
      • Princess Margaret Cancer Centre
• China
  • Anhui
    • Hefei, Anhui, China, 230071
      • Anhui Provincial Hospital
  • Beijing
    • Beijing, Beijing, China, 100730
      • Beijing Tongren Hospital affiliated to Capital Medical University
  • Chongqing
    • Chongqing, Chongqing, China, 400030
      • Chongqing University Cancer Hospital
  • Fujian
    • Fuzhou, Fujian, China, 350005
      • The First Affiliated Hospital Of Fujian Medical University
  • Gansu
    • Lanzhou, Gansu, China, 730050
      • Gansu Cancer Hospital
  • Guangdong
    • Guangzhou, Guangdong, China, 510060
      • Sun Yat-Sen University Cancer Centre
  • Heilongjiang
    • Harbin, Heilongjiang, China, 150081
      • Harbin Medical University Cancer Hospital
  • Henan
    • Zhengzhou, Henan, China, 450008
      • Henan Cancer Hospital
  • Hunan
    • Changsha, Hunan, China, 410013
      • Hunan Cancer Hospital
  • Jiangsu
    • Nanjing, Jiangsu, China, 210000
      • Nanjing Drum Tower Hospital The Affiliated Hospital of Nanjing University Medical School
  • Jilin
    • Changchun, Jilin, China, 132000
      • Jilin Cancer Hospital
  • Shandong
    • Jinan, Shandong, China, 250013
      • Jinan Central Hospital
  • Shanghai
    • Shanghai, Shanghai, China, 200032
      • Fudan University Shanghai Cancer Center
  • Sichuan
    • Cheng Du, Sichuan, China, 610041
      • West China Hospital, Sichuan University
  • Tianjin
    • Tianjin, Tianjin, China, 300060
      • Tianjin Medical University Cancer Institute and Hospital
  • Yunnan
    • Kunming, Yunnan, China, 650032
      • First Affiliated Hospital of Kunming Medical University
  • Zhejiang
    • Hangzhou, Zhejiang, China, 310022
      • Zhejiang Cancer Hospital
    • Hangzhou, Zhejiang, China, 310014
      • Zhejiang Provincial people's Hospital
    • Hangzhou, Zhejiang, China, 310016
      • Sir Run Run Shaw Hospital
• Czechia
  • Olomouc, Czechia, 779 00
    • Fakultni nemocnice Olomouc
  • Brno-město
    • Brno, Brno-město, Czechia, 625 00
      • Fakultní nemocnice Brno Bohunice
  • Praha 5
    • Praha, Praha 5, Czechia, 150 06
      • Fakultni nemocnice Motol
• France
  • Alsace
    • Strasbourg, Alsace, France, 67065
      • Centre Paul Strauss
  • Aquitaine
    • Bordeaux, Aquitaine, France, 33076
      • Institut Bergonié - Centre Régional de Lutte Contre Le Cancer de Bordeaux et Sud Ouest
  • Bouches-du-Rhône
    • Marseille, Bouches-du-Rhône, France, 13915
      • Assistance Publique Hôpitaux de Marseille - Hôpital Nord
  • Calvados
    • Caen, Calvados, France, 14076
      • Centre Francois Baclesse
  • Côte-d'Or
    • Dijon, Côte-d'Or, France, 21079
      • Centre Georges Francois Leclerc
  • Haute-Garonne
    • Toulouse, Haute-Garonne, France, 31059
      • Institut Claudius Regaud
  • Maine-et-Loire
    • Angers, Maine-et-Loire, France, 49933
      • Centre Hospitalier Universitaire d'Angers
  • Nord
    • Lille, Nord, France, 59037
      • Hopital Claude Huriez - CHU de Lille
  • Orne
    • Paris, Orne, France, 75013
      • Pitie Salpetriere University Hospital
  • Puy-de-Dôme
    • Clermont-Ferrand, Puy-de-Dôme, France, 63011
      • Centre Jean Perrin - Centre Régional de Lutte contre le Cancer d'Auvergne
  • Rhône-Alpes
    • Lyon, Rhône-Alpes, France, 69008
      • Centre Leon Berard
  • Val-de-Marne
    • Villejuif, Val-de-Marne, France, 94800
      • Gustave Roussy
• Germany
  • Berlin, Germany, 10117
    • Charité Universitaetsmedizin Berlin - Campus Mitte
  • Hamburg, Germany, 20251
    • Hämato-Onkologie Hamburg, Prof. Laack und Partner
  • Bayern
    • München, Bayern, Germany, 81337
      • Klinikum der Universität München Grosshadern
    • Würzburg, Bayern, Germany, 97080
      • Klinikum der Universität München Grosshadern
  • Niedersachsen
    • Hannover, Niedersachsen, Germany, 30625
      • Medizinische Hochschule Hannover
  • Nordrhein-Westfalen
    • Essen, Nordrhein-Westfalen, Germany, 45122
      • Universitaetsklinikum Essen
  • Rheinland-Pfalz
    • Mainz, Rheinland-Pfalz, Germany, 55131
      • Universitätsmedizin Johannes Gutenberg Universität Mainz
  • Sachsen-Anhalt
    • Magdeburg, Sachsen-Anhalt, Germany, 39120
      • Otto-von-Guericke-Universitat Magdeburg
• Greece
  • Attikí
    • Athina, Attikí, Greece, 115 28
      • Alexandra General Hospital Of Athens
  • Irakleío
    • Heraklion, Irakleío, Greece, 711 10
      • University General Hospital of Heraklion
  • Thessaloniki
    • Thessaloníki, Thessaloniki, Greece, 570 01
      • European Interbalkan Medical Center
• India
  • Chandigarh, India, 160012
    • Post Graduate Institute of Medical Education & Research (PGIMER)
  • Kerala
    • Thiruvananthapuram, Kerala, India, 695011
      • Regional Cancer Centre - Thiruvananthapuram
  • Maharashtra
    • Nashik, Maharashtra, India, 422001
      • HCG Manavata Cancer Centre
    • Pune, Maharashtra, India, 411004
      • Deenanath Mangeshkar Hospital & Research Centre
    • Pune, Maharashtra, India, 411001
      • Grant Medical Foundation - Ruby Hall Clinic
  • West Bengal
    • Kolkata, West Bengal, India, 700054
      • Apollo Gleneagles Hospitals Kolkata
• Israel
  • HaMerkaz
    • Petah-Tikva, HaMerkaz, Israel, 4941492
      • Rabin Medical Center
    • Ramat Gan, HaMerkaz, Israel, 5265601
      • Sheba Medical Center
  • Yerushalayim
    • Jerusalem, Yerushalayim, Israel, 9112001
      • Hadassah Medical Center
• Italy
  • Catania, Italy, 95124
    • Azienda Ospedaliera Garibaldi
  • Campania
    • Napoli, Campania, Italy, 80131
      • University of Naples Federico II
  • Lazio
    • Roma, Lazio, Italy, 00161
      • Policlinico Umberto I
  • Lombardia
    • Milan, Lombardia, Italy, 20133
      • Fondazione IRCCS Istituto Nazionale dei Tumori
  • Milano
    • Milan, Milano, Italy, 20122
      • Istituto Auxologico Italiano
  • Piemonte
    • Torino, Piemonte, Italy, 10126
      • Azienda Ospedaliero-Universitaria Città della Salute e della Scienza di Torino
  • Roma
    • Rome, Roma, Italy, 00144
      • Istituto Nazionale Tumori Regina Elena
  • Toscana
    • Pisa, Toscana, Italy, 56124
      • Azienda Ospedaliera Universitaria Pisana
    • Siena, Toscana, Italy, 53100
      • Ospedale Le Scotte
  • Veneto
    • Padova, Veneto, Italy, 35128
      • Istituto Oncologico Veneto IRCCS
• Japan
  • Fukuoka, Japan, 810-8563
    • National Hospital Organization Kyushu Medical Center
  • Aichi
    • Nagoya, Aichi, Japan, 464-8681
      • Aichi Cancer Center Hospital
  • Chiba
    • Kashiwa, Chiba, Japan, 277-8577
      • National Cancer Center Hospital East
  • Hokkaido
    • Sapporo, Hokkaido, Japan, 060-8648
      • Hokkaido University Hospital
  • Hyogo
    • Kobe, Hyogo, Japan, 650-0017
      • Kobe University Hospital
  • Kanagawa
    • Yokohama, Kanagawa, Japan, 236-0004
      • Yokohama City University Hospital
  • Osaka
    • Suita, Osaka, Japan, 565-0871
      • Osaka University Hospital
  • Tokyo
    • Koto, Tokyo, Japan, 135-8550
      • Japanese Foundation for Cancer Research
• Korea, Republic of
  • Chungcheongbuk-do [Chungbuk]
    • Chungbuk, Chungcheongbuk-do [Chungbuk], Korea, Republic of, 28644
      • Chungbuk National University Hospital
  • Korea
    • Seoul, Korea, Korea, Republic of, 05505
      • Asan Medical Center
  • Kyǒnggi-do
    • Goyang-si, Kyǒnggi-do, Korea, Republic of, 10408
      • National Cancer Center
    • Seongnam, Kyǒnggi-do, Korea, Republic of, 13620
      • Seoul National University Bundang Hospital
  • Seoul-teukbyeolsi [Seoul]
    • Seoul, Seoul-teukbyeolsi [Seoul], Korea, Republic of, 3080
      • Seoul National University Hospital
    • Seoul, Seoul-teukbyeolsi [Seoul], Korea, Republic of, 6351
      • Samsung Medical Center
    • Seoul, Seoul-teukbyeolsi [Seoul], Korea, Republic of, 3722
      • Severance Hospital, Yonsei University Health System
• Netherlands
  • Groningen, Netherlands, 9713 GR
    • University Medical Center Groningen
  • Limburg
    • Maastricht, Limburg, Netherlands, 6229 HX
      • Maastricht UMC+
  • Noord-Holland
    • Amsterdam, Noord-Holland, Netherlands, 1066 CX
      • Nederlands Kanker Instituut - Antoni van Leeuwenhoek (NKI-AVL)
  • Zuid-Holland
    • Leiden, Zuid-Holland, Netherlands, 2333 ZA
      • Leids Universitair Medisch Centrum
• Poland
  • Śląskie
    • Gliwice, Śląskie, Poland, 44-101
      • Narodowy Instytut Onkologii - Oddzial w Gliwicach
  • Świętokrzyskie
    • Kielce, Świętokrzyskie, Poland, 25-734
      • Swietokrzyskie Centrum Onkologii, Samodzielny Publiczny Zaklad Opieki Zdrowotnej
• Russian Federation
  • Chelyabinskaya Oblast'
    • Chelyabinsk, Chelyabinskaya Oblast', Russian Federation, 454048
      • Clinic Evimed
  • Kalužskaja Oblast'
    • Obninsk, Kalužskaja Oblast', Russian Federation, 249036
      • A. Tsyb Medical Radiological Research Center - branch of the National Medical Research Radiological
  • Moskva
    • Moscow, Moskva, Russian Federation, 115478
      • Fed State Budgetary Inst "N.N. Blokhin Med Center of Oncology" MHRF
    • Moscow, Moskva, Russian Federation, 117292
      • Endocrinology Research Center of Rosmedtechnologies
  • Sankt-Peterburg
    • Saint Petersburg, Sankt-Peterburg, Russian Federation, 190020
      • Saint Petersburg State University
    • Saint Petersburg, Sankt-Peterburg, Russian Federation, 198255
      • Saint-Petersburg City Clinical Oncology Dispensary
• Spain
  • Madrid, Spain, 28040
    • Hospital Clinico San Carlos
  • Madrid, Spain, 28009
    • Hospital General Universitario Gregorio Marañon
  • Zaragoza, Spain, 50009
    • Hospital Universitario Miguel Servet
  • Barcelona [Barcelona]
    • Barcelona, Barcelona [Barcelona], Spain, 8035
      • Hospital Universitari Vall d'Hebron
  • Catalunya [Cataluña]
    • L'Hospitalet de Llobregat, Catalunya [Cataluña], Spain, 8907
      • Instituto Catalan de Oncologia - Hospital Duran i Reynals
  • Girona [Gerona]
    • Girona, Girona [Gerona], Spain, 17007
      • Institut Català d'Oncologia (ICO) - Girona
  • Madrid, Comunidad De
    • Madrid, Madrid, Comunidad De, Spain, 28034
      • Hospital Universitario Ramon Y Cajal
    • Madrid, Madrid, Comunidad De, Spain, 28046
      • Hospital Universitario La Paz
    • Madrid, Madrid, Comunidad De, Spain, 28027
      • Clinica Universidad de Navarra
    • Madrid, Madrid, Comunidad De, Spain, 28041
      • Hospital Universitario 12 de Octubre
  • Málaga
    • Malaga, Málaga, Spain, 29010
      • Hospital Universitario Virgen De La Victoria
  • Navarra
    • Pamplona, Navarra, Spain, 31008
      • Clinica Universidad de Navarra
• Taiwan
  • Taichung, Taiwan, 40447
    • China Medical University Hospital
  • Tainan, Taiwan, 704
    • National Cheng-Kung Uni. Hosp.
  • Taipei, Taiwan, 10002
    • National Taiwan University Hospital
• United Kingdom
  • Cardiff [Caerdydd Gb-crd]
    • Cardiff, Cardiff [Caerdydd Gb-crd], United Kingdom, CF14 2TL
      • Velindre Cancer Centre
  • England
    • Sheffield, England, United Kingdom, S10 2SJ
      • Weston Park Hospital
  • Glasgow City
    • Glasgow, Glasgow City, United Kingdom, G12OYN
      • Gartnavel General Hospital
  • Kensington And Chelsea
    • London, Kensington And Chelsea, United Kingdom, SW3 6JJ
      • Royal Marsden Hospital (Chelsea)
  • London, City Of
    • London, London, City Of, United Kingdom, NW1 2PG
      • University College London Hospital
  • Surrey
    • Sutton, Surrey, United Kingdom, SM2 5PT
      • Royal Marsden NHS Trust
  • Sutton
    • London, Sutton, United Kingdom, SM2 5PT
      • Royal Marsden Hospital (Sutton)
• United States
  • Alabama
    • Birmingham, Alabama, United States, 35249
      • University of Alabama at Birmingham
  • Arizona
    • Phoenix, Arizona, United States, 85054
      • Mayo Clinic in Arizona - Phoenix
  • California
    • Duarte, California, United States, 91010-0269
      • City of Hope National Medical Center
    • La Jolla, California, United States, 92037
      • University of California San Diego
    • Los Angeles, California, United States, 90095
      • UCLA Hematology/Oncology - Westwood (Building 100)
    • Sacramento, California, United States, 95817
      • University of California Davis (UC Davis) Comprehensive Cancer Center
    • San Francisco, California, United States, 94158
      • UCSF Medical Center at Mission Bay
    • Torrance, California, United States, 90502
      • Lundquist Institute for Biomedical Innovation at Harbor-UCLA Medical Center
  • Florida
    • Jacksonville, Florida, United States, 32224
      • Mayo Clinic in Florida
  • Georgia
    • Atlanta, Georgia, United States, 30322
      • Emory University
  • Illinois
    • Chicago, Illinois, United States, 60637
      • University of Chicago Medical Center
  • Massachusetts
    • Boston, Massachusetts, United States, 02114
      • Massachusetts General Hospital
  • Michigan
    • Ann Arbor, Michigan, United States, 48109
      • University of Michigan
    • Detroit, Michigan, United States, 48201
      • Karmanos Cancer Institute
  • Minnesota
    • Rochester, Minnesota, United States, 55905
      • Mayo Clinic in Rochester, Minnesota
  • Missouri
    • Saint Louis, Missouri, United States, 63110
      • Washington University
  • New York
    • New York, New York, United States, 10017
      • Memorial Sloan Kettering Cancer Center
  • Ohio
    • Cincinnati, Ohio, United States, 45219
      • University of Cincinnati Medical Center
    • Columbus, Ohio, United States, 43210
      • The James Cancer Hospital and Solove Research Institute at The Ohio State University Comprehensive Cancer Center
  • Oregon
    • Portland, Oregon, United States, 97213
      • Providence Cancer Center Oncology Hematology Care
  • Pennsylvania
    • Philadelphia, Pennsylvania, United States, 19114
      • Thomas Jefferson University
    • Philadelphia, Pennsylvania, United States, 19104
      • University of Pennsylvania Hospital
    • Pittsburgh, Pennsylvania, United States, 15232-1305
      • UPMC Hillman Cancer Center
  • Texas
    • Houston, Texas, United States, 77030
      • University of Texas MD Anderson Cancer Center
  • Wisconsin
    • Madison, Wisconsin, United States, 53792
      • University of Wisconsin Hospitals and Clinics

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 12 years and older (Child, Adult, Older Adult)
• Accepts Healthy Volunteers: No

Description

• At least 18 years of age (participants as young as 12 years of age will be allowed if permitted by local regulatory authorities).
• Histologically or cytologically confirmed, unresectable, locally advanced and/or metastatic MTC and no prior history of treatment with kinase inhibitors for advanced/metastatic disease.
• Radiographic progressive disease per Response Evaluation Criteria in Solid Tumors (RECIST) 1.1 at screening compared with a previous image taken within the prior 14 months as assessed by the BICR. Participants with measurable or non-measurable but evaluable disease are eligible; however, participants with non-measurable disease may not have disease limited to bone sites only.

• A defined/acceptable RET gene alteration identified in a tumor, germline deoxyribonucleic acid (DNA) or blood sample.

  • Tumor tissue in sufficient quantity to allow for retrospective central analysis of RET mutation status
• Eastern Cooperative Oncology Group performance status score of 0 to 2.
• Adequate hematologic, hepatic, and renal function and electrolytes.
• Men and women of childbearing potential must agree to use a highly effective contraceptive method during treatment with study drug and for 4 months following the last dose of study drug.
• Ability to swallow capsules.

Exclusion Criteria:

• An additional validated oncogenic driver in MTC if known that could cause resistance to selpercatinib treatment. Examples include, but are not limited to RAS or BRAF gene mutations and NTRK gene fusions.
• Symptomatic central nervous system (CNS) metastases, leptomeningeal carcinomatosis, or untreated spinal cord compression.
• Clinically significant active cardiovascular disease or history of myocardial infarction within 6 months, history of Torsades de pointes, or prolongation of the QTcF >470 milliseconds on more than one electrocardiogram (ECG) during screening. Participants who are intended to receive vandetanib if randomized to the control arm are ineligible if QTcF is >450 milliseconds.
• Active uncontrolled systemic bacterial, viral, or fungal infection or serious ongoing uncontrolled intercurrent illness.
• Active hemorrhage or at significant risk for hemorrhage.
• Other malignancy unless nonmelanoma skin cancer, carcinoma in situ or malignancy diagnosed ≥2 years previously and not currently active. Participants with multiple endocrine neoplasia type 2 (MEN2) associated pheochromocytoma may be eligible.

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: Randomized
• Interventional Model: Parallel Assignment
• Masking: None (Open Label)

Number of Arms

2

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: Selpercatinib; Selpercatinib given orally.	Drug: Selpercatinib; Administered orally; Other Names: LOXO-292; LY3527723
Active Comparator: Cabozantinib or Vandetanib; Cabozantinib or vandetanib given orally.	Drug: Cabozantinib; Administered orally; Drug: Vandetanib; Administered orally

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Progression Free Survival (PFS) by BICR	PFS by BICR	Baseline to Progressive Disease or Death from Any Cause (Estimated at up to 30 Months)

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Treatment Failure-Free Survival (TFFS) by Blinded Independent Committee Review (BICR)	TFFS by BICR	Baseline to Progressive Disease, Unacceptable Toxicity or Death from Any Cause (Estimated at up to 30 Months)
Overall Response Rate (ORR): Percentage of Participants with Complete Response (CR) or Partial Response (PR) by BICR	ORR: Percentage of Participants with CR or PR by BICR	Baseline through Disease Progression or Death (Estimated at up to 30 Months)
Duration of Response (DoR) by BICR	DoR by BICR	Date of CR or PR to Date of Disease Progression or Death Due to Any Cause (Estimated at up to 30 Months)
Overall Survival (OS)	OS	Baseline to Date of Death from Any Cause (Estimated at up to 60 Months)
PFS2 by Investigator	PFS2 by Investigator	Baseline to Second Disease Progression or Death from Any Cause (Estimated at up to 48 Months)
Comparative Tolerability: Percentage of Time with High Side Effect Bother Based on the Functional Assessment of Cancer Therapy-Side Effects (FACT-GP5)	FACT-G is a validated instrument used to measure quality of life (QOL) in participants with cancer. The single FACT-G item, GP5, "I am bothered by side effects of treatment," is a summary measure of the overall impact of treatment toxicity, based upon its association with the number and degree of adverse events in clinical trials. It uses a 5-point rating scale (0="not at all" and 4=equals "very much"). Higher GP5 scores indicates more bother from side effects	Baseline to Progressive Disease, Unacceptable Toxicity or Death from Any Cause (30 months)
The Concordance of the Local Lab and the Central Lab RET Results: Percentage of Participants with RET-Positive Specimens as Called by the Central Lab, which is also RET-Positive as Called by a Local Lab (Positive Percent Agreement)	The Concordance of the Local Lab and the Central Lab RET Results: Percentage of Participants with RET-Positive Specimens as Called by the Central Lab, which is also RET-Positive as Called by a Local Lab (Positive Percent Agreement)	Baseline

Collaborators and Investigators

• Sponsor: Loxo Oncology, Inc.
• Collaborators: Eli Lilly and Company
• Investigators: Study Director: Call 1-877-CTLILLY (1-877-285-4559) or 1-317-615-4559 Mon - Fri 9 AM - 5 PM, Eli Lilly and Company

Publications and helpful links

General Publications

• Wirth LJ, Brose MS, Elisei R, Capdevila J, Hoff AO, Hu MI, Tahara M, Robinson B, Gao M, Xia M, Maeda P, Sherman E. LIBRETTO-531: a phase III study of selpercatinib in multikinase inhibitor-naive RET-mutant medullary thyroid cancer. Future Oncol. 2022 Sep;18(28):3143-3150. doi: 10.2217/fon-2022-0657. Epub 2022 Aug 15.
• Jaber T, Dadu R, Hu MI. Medullary thyroid carcinoma. Curr Opin Endocrinol Diabetes Obes. 2021 Oct 1;28(5):540-546. doi: 10.1097/MED.0000000000000662.

Helpful Links

• A Study of Selpercatinib (LY3527723) in Participants With RET-Mutant Medullary Thyroid Cancer

Study record dates

Study Major Dates

• Study Start (Actual): February 11, 2020
• Primary Completion (Actual): May 22, 2023
• Study Completion (Estimated): February 28, 2026

Study Registration Dates

• First Submitted: December 24, 2019
• First Submitted That Met QC Criteria: December 24, 2019
• First Posted (Actual): December 26, 2019

Study Record Updates

• Last Update Posted (Estimated): January 18, 2024
• Last Update Submitted That Met QC Criteria: January 17, 2024
• Last Verified: January 1, 2024

More Information

Terms related to this study

• Keywords: targeted therapy; medullary thyroid carcinoma
• Additional Relevant MeSH Terms: Neoplasms by Histologic Type; Neoplasms; Neoplasms by Site; Adenocarcinoma; Carcinoma; Neoplasms, Glandular and Epithelial; Endocrine System Diseases; Endocrine Gland Neoplasms; Head and Neck Neoplasms; Neuroectodermal Tumors; Neoplasms, Germ Cell and Embryonal; Neoplasms, Nerve Tissue; Neuroendocrine Tumors; Thyroid Diseases; Thyroid Neoplasms; Carcinoma, Neuroendocrine
• Other Study ID Numbers: 17478; J2G-MC-JZJB (Other Identifier: Eli Lilly and Company); 2019-001978-28 (EudraCT Number)

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: YES
• IPD Plan Description: Anonymized individual patient level data will be provided in a secure access environment upon approval of a research proposal and a signed data sharing agreement.
• IPD Sharing Time Frame: Data are available 6 months after the primary publication and approval of the indication studied in the US and EU, whichever is later. Data will be indefinitely available for requesting.
• IPD Sharing Access Criteria: A research proposal must be approved by an independent review panel and researchers must sign a data sharing agreement.
• IPD Sharing Supporting Information Type: STUDY_PROTOCOL; SAP; CSR

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: Yes
• Studies a U.S. FDA-regulated device product: No