AzTrials
AzTrials

Medullary Thyroid CancerPhase 3LIBRETTO-531NCT04211337

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

A Phase 3 study testing Selpercatinib, Cabozantinib and Vandetanib in people with Medullary Thyroid Cancer, run by Loxo Oncology, Inc. at 143 sites in 20 countries.

Active, not recruiting

Under way, not looking for participants. Everyone needed has enrolled. The study is still running.

Official record
Search all trials

Trial summary

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. Read more about this study

Who is it for?
Ages 12 years and older
Conditions the study is for
Medullary Thyroid Cancer
What is being tested?
Selpercatinib, Cabozantinib and Vandetanib
Type of treatment
Pill or oral medicine
Could I receive a placebo?
No, everyone receives an active treatment
How many people
291 participants
Where
143 sitesin 20 countries
Study dates
11 Feb 2020to Nov 2027
Phase
Phase 3
Are there results?
Yes, posted in the registrySummary in the doctors' view

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

  • Ages 12 years and older
  • All sexes
  • No healthy volunteers
  • Child, Adult, Older adult

Who may be able to join

None listed.

Who cannot join

  • 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.

Trial locations and contacts

143 sites in 20 countries.

143 sites in 20 countries

China19 sites

  • Beijing Tongren Hospital affiliated to Capital Medical University

    Beijing, Beijing Municipality, 100730

  • Jilin Cancer Hospital

    Changchun, Jilin, 132000

  • Hunan Cancer Hospital

    Changsha, Hunan, 410013

  • West China Hospital, Sichuan University

    Chengdu, Sichuan, 610041

  • Chongqing University Cancer Hospital

    Chongqing, Chongqing Municipality, 400030

  • The First Affiliated Hospital Of Fujian Medical University

    Fuzhou, Fujian, 350005

  • Sun Yat-Sen University Cancer Centre

    Guangzhou, Guangdong, 510060

  • Zhejiang Provincial People's Hospital

    Hangzhou, Zhejiang, 310014

  • Sir Run Run Shaw Hospital

    Hangzhou, Zhejiang, 310016

  • Zhejiang Cancer Hospital

    Hangzhou, Zhejiang, 310022

  • Harbin Medical University Cancer Hospital

    Harbin, Heilongjiang, 150081

  • Anhui Provincial Hospital

    Hefei, Anhui, 230071

  • Jinan Central Hospital

    Jinan, Shandong, 250013

  • First Affiliated Hospital of Kunming Medical University

    Kunming, Yunnan, 650032

  • Gansu Cancer Hospital

    Lanzhou, Gansu, 730050

  • Nanjing Drum Tower Hospital The Affiliated Hospital of Nanjing University Medical School

    Nanjing, Jiangsu, 210000

  • Fudan University Shanghai Cancer Center

    Shanghai, Shanghai Municipality, 200032

  • Tianjin Medical University Cancer Institute and Hospital

    Tianjin, Tianjin Municipality, 300060

  • Henan Cancer Hospital

    Zhengzhou, Henan, 450008

Other trials in China

United States16 sites

  • University of Michigan

    Ann Arbor, Michigan, 48109

  • Emory University

    Atlanta, Georgia, 30322

  • University of Alabama at Birmingham

    Birmingham, Alabama, 35249

  • Massachusetts General Hospital

    Boston, Massachusetts, 02114

  • University of Chicago Medical Center

    Chicago, Illinois, 60637

  • University of Cincinnati Medical Center

    Cincinnati, Ohio, 45219

  • The James Cancer Hospital and Solove Research Institute at The Ohio State University Comprehensive Cancer Center

    Columbus, Ohio, 43210

  • City of Hope National Medical Center

    Duarte, California, 91010-0269

  • University of Texas MD Anderson Cancer Center

    Houston, Texas, 77030

  • UCLA Hematology/Oncology - Westwood (Building 100)

    Los Angeles, California, 90095

  • University of Wisconsin Hospitals and Clinics

    Madison, Wisconsin, 53792

  • Memorial Sloan Kettering Cancer Center

    New York, New York, 10017

  • University of Pennsylvania Hospital

    Philadelphia, Pennsylvania, 19104

  • Thomas Jefferson University

    Philadelphia, Pennsylvania, 19114

  • University of California Davis (UC Davis) Comprehensive Cancer Center

    Sacramento, California, 95817

  • Lundquist Institute for Biomedical Innovation at Harbor-UCLA Medical Center

    Torrance, California, 90502

Other trials in United States

Brazil13 sites

  • Fundação Pio XII - Hospital de Câncer de Barretos

    Barretos, São Paulo, 14784400

  • Oncocentro

    Belo Horizonte, Minas Gerais, 30380-680

  • Centro de Pesquisa Sao Lucas

    Campinas, São Paulo, 13060-904

  • Hospital de Cancer de Londrina

    Londrina, Paraná, 86015-520

  • Hospital de Clinicas de Porto Alegre

    Porto Alegre, Rio Grande do Sul, 90035-903

  • Hospital de Clínicas de Ribeirão Preto

    Ribeirão Preto, São Paulo, 14051-140

  • Instituto Nacional de Câncer - INCA

    Rio de Janeiro, 20230-130

  • Grupo Oncoclínicas Botafogo

    Rio de Janeiro, 22250-905

  • Grupo COI - Clínicas Oncológicas Integradas

    Rio de Janeiro, 22775-001

  • Hospital Sírio Libanês

    São Paulo, São Paulo, 01308-060

  • Instituto D'Or de Pesquisa e Ensino (IDOR)

    São Paulo, São Paulo, 04543-000

  • Icesp - Instituto Do Câncer Do Estado de São Paulo

    São Paulo, 01246-000

  • Centro Paulista de Oncologia Clínica

    São Paulo, 01452-000

Other trials in Brazil

France12 sites

  • Centre Hospitalier Universitaire d'Angers

    Angers, Maine-et-Loire, 49933

  • Institut Bergonié - Centre Régional de Lutte Contre Le Cancer de Bordeaux et Sud Ouest

    Bordeaux, Aquitaine, 33076

  • Centre François Baclesse

    Caen, Calvados, 14076

  • Centre Jean Perrin - Centre Régional de Lutte contre le Cancer d'Auvergne

    Clermont-Ferrand, Puy-de-Dôme, 63011

  • Centre Georges François Leclerc

    Dijon, Côte-d'Or, 21079

  • Hopital Claude Huriez - CHU de Lille

    Lille, Nord, 59037

  • Centre Leon Berard

    Lyon, Auvergne-Rhône-Alpes, 69008

  • Assistance Publique Hôpitaux de Marseille - Hôpital Nord

    Marseille, Bouches-du-Rhône, 13915

  • Pitie Salpetriere University Hospital

    Paris, Orne, 75013

  • Centre Paul Strauss

    Strasbourg, Alsace, 67065

  • Institut Claudius Regaud

    Toulouse, Haute-Garonne, 31059

  • Gustave Roussy

    Villejuif, Val-de-Marne, 94800

Other trials in France

Spain12 sites

  • Hospital Universitari Vall d'Hebron

    Barcelona, Barcelona [Barcelona], 8035

  • Institut Català d'Oncologia (ICO) - Girona

    Girona, Girona [Gerona], 17007

  • Instituto Catalan de Oncologia - Hospital Duran i Reynals

    L'Hospitalet de Llobregat, Catalunya [Cataluña], 8907

  • Clinica Universidad de Navarra

    Madrid, Madrid, Comunidad de, 28027

  • Hospital Universitario Ramón y Cajal

    Madrid, Madrid, Comunidad de, 28034

  • Hospital Universitario 12 de Octubre

    Madrid, Madrid, Comunidad de, 28041

  • Hospital Universitario La Paz

    Madrid, Madrid, Comunidad de, 28046

  • Hospital General Universitario Gregorio Marañon

    Madrid, 28009

  • Hospital Clinico San Carlos

    Madrid, 28040

  • Hospital Universitario Virgen de la Victoria

    Málaga, Málaga, 29010

  • Clinica Universidad de Navarra

    Pamplona, Navarre, 31008

  • Hospital Universitario Miguel Servet

    Zaragoza, 50009

Other trials in Spain

Italy10 sites

  • Azienda Ospedaliera Garibaldi

    Catania, 95124

  • Fondazione IRCCS Istituto Nazionale dei Tumori

    Milan, Lombardy, 20133

  • Istituto Auxologico Italiano

    Milan, Milano, 20122

  • University of Naples Federico II

    Naples, Campania, 80131

  • Istituto Oncologico Veneto IRCCS

    Padova, Veneto, 35128

  • Azienda Ospedaliera Universitaria Pisana

    Pisa, Tuscany, 56124

  • Policlinico Umberto I

    Rome, Lazio, 00161

  • Istituto Nazionale Tumori Regina Elena

    Rome, Roma, 00144

  • Ospedale Le Scotte

    Siena, Tuscany, 53100

  • Azienda Ospedaliero-Universitaria Città della Salute e della Scienza di Torino

    Turin, Piedmont, 10126

Other trials in Italy

Germany8 sites

  • Charité Universitaetsmedizin Berlin - Campus Mitte

    Berlin, 10117

  • Universitaetsklinikum Essen

    Essen, North Rhine-Westphalia, 45122

  • Hämato-Onkologie Hamburg, Prof. Laack und Partner

    Hamburg, 20251

  • Medizinische Hochschule Hannover

    Hanover, Lower Saxony, 30625

  • Otto-von-Guericke-Universität Magdeburg

    Magdeburg, Saxony-Anhalt, 39120

  • Universitätsmedizin Johannes Gutenberg Universität Mainz

    Mainz, Rhineland-Palatinate, 55131

  • Klinikum der Universität München Großhadern

    München, Bavaria, 81337

  • Klinikum der Universität München Großhadern

    Würzburg, Bavaria, 97080

Other trials in Germany

Japan8 sites

  • National Hospital Organization Kyushu Medical Center

    Fukuoka, 810-8563

  • National Cancer Center Hospital East

    Kashiwa, Chiba, 277-8577

  • Kobe University Hospital

    Kobe, Hyōgo, 650-0017

  • Japanese Foundation for Cancer Research

    Koto, Tokyo, 135-8550

  • Aichi Cancer Center Hospital

    Nagoya, Aichi-ken, 464-8681

  • Hokkaido University Hospital

    Sapporo, Hokkaido, 060-8648

  • Osaka University Hospital

    Suita, Osaka, 565-0871

  • Yokohama City University Hospital

    Yokohama, Kanagawa, 236-0004

Other trials in Japan

India6 sites

  • Post Graduate Institute of Medical Education & Research (PGIMER)

    Chandigarh, 160012

  • Apollo Gleneagles Hospitals Kolkata

    Kolkata, West Bengal, 700054

  • HCG Manavata Cancer Centre

    Nashik, Maharashtra, 422001

  • Grant Medical Foundation - Ruby Hall Clinic

    Pune, Maharashtra, 411001

  • Deenanath Mangeshkar Hospital & Research Centre

    Pune, Maharashtra, 411004

  • Regional Cancer Centre - Thiruvananthapuram

    Thiruvananthapuram, Kerala, 695011

Other trials in India

Russia6 sites

  • Clinic Evimed

    Chelyabinsk, Chelyabinsk Oblast, 454048

  • Fed State Budgetary Inst "N.N. Blokhin Med Center of Oncology" MHRF

    Moscow, Moscow, 115478

  • Endocrinology Research Center of Rosmedtechnologies

    Moscow, Moscow, 117292

  • A. Tsyb Medical Radiological Research Center - branch of the National Medical Research Radiological

    Obninsk, Kalužskaja Oblast', 249036

  • Saint Petersburg State University

    Saint Petersburg, Sankt-Peterburg, 190020

  • Saint-Petersburg City Clinical Oncology Dispensary

    Saint Petersburg, Sankt-Peterburg, 198255

Other trials in Russia

South Korea6 sites

  • National Cancer Center

    Goyang-si, Kyǒnggi-do, 10408

  • Chungbuk National University Hospital

    Jungbuk, Chungcheongbuk-do [Chungbuk], 28644

  • Seoul National University Bundang Hospital

    Seongnam, Kyǒnggi-do, 13620

  • Seoul National University Hospital

    Seoul, Seoul-teukbyeolsi [Seoul], 3080

  • Severance Hospital, Yonsei University Health System

    Seoul, Seoul-teukbyeolsi [Seoul], 3722

  • Samsung Medical Center

    Seoul, Seoul-teukbyeolsi [Seoul], 6351

Other trials in South Korea

United Kingdom6 sites

  • Velindre Cancer Centre

    Cardiff, Cardiff [Caerdydd Gb-crd], CF14 2TL

  • Gartnavel General Hospital

    Glasgow, Glasgow City, g12OYN

  • Royal Marsden Hospital (Chelsea)

    London, Kensington and Chelsea, SW3 6JJ

  • University College London Hospital

    London, London, City of, NW1 2PG

  • Royal Marsden Hospital (Sutton)

    London, Sutton, SM2 5PT

  • Weston Park Hospital

    Sheffield, England, S10 2SJ

Other trials in United Kingdom

Australia4 sites

  • The Alfred Hospital

    Melbourne, Victoria, 3004

  • Peter MacCallum Cancer Centre

    Melbourne, Victoria, 3050

  • Sir Charles Gairdner Hospital

    Perth, Western Australia, 6009

  • Royal North Shore Hospital

    St Leonards, New South Wales, 2065

Other trials in Australia

Netherlands4 sites

  • Nederlands Kanker Instituut - Antoni van Leeuwenhoek (NKI-AVL)

    Amsterdam, North Holland, 1066 CX

  • University Medical Center Groningen

    Groningen, 9713 GR

  • Leids Universitair Medisch Centrum

    Leiden, South Holland, 2333 ZA

  • Maastricht UMC+

    Maastricht, Limburg, 6229 HX

Other trials in Netherlands

Czechia3 sites

  • Fakultní nemocnice Brno Bohunice

    Brno, Brno-město, 625 00

  • Fakultni nemocnice Olomouc

    Olomouc, 779 00

  • Fakultni nemocnice Motol

    Prague, Praha 5, 150 06

Other trials in Czechia

Greece3 sites

  • Alexandra Hospital

    Athens, Attikí, 115 28

  • University General Hospital of Heraklion

    Heraklion, Irakleío, 711 10

  • European Interbalkan Medical Center

    Thessaloniki, Thessaloniki, 570 01

Other trials in Greece

Israel3 sites

  • Hadassah Medical Center

    Jerusalem, Jerusalem, 9112001

  • Rabin Medical Center

    Petah Tikva, Central District, 4941492

  • Sheba Medical Center

    Ramat Gan, Central District, 5265601

Other trials in Israel

Poland2 sites

  • Narodowy Instytut Onkologii - Oddzial w Gliwicach

    Gliwice, Silesian Voivodeship, 44-101

  • Swietokrzyskie Centrum Onkologii, Samodzielny Publiczny Zaklad Opieki Zdrowotnej

    Kielce, Świętokrzyskie Voivodeship, 25-734

Other trials in Poland

Belgium1 site

  • Centre Hospitalier Universitaire de Liège - Domaine Universitaire du Sart Tilman

    Liège, 4000

Other trials in Belgium

Canada1 site

  • Princess Margaret Cancer Centre

    Toronto, Ontario, M5G 2M9

Other trials in Canada

About this study

Official title: 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.

Read the 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.

Being tested
Selpercatinib, Cabozantinib, Vandetanib
Keywords
medullary thyroid carcinoma, targeted therapy
Medical subject headings
Carcinoma, Medullary

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • SelpercatinibDrug

    Also called LY3527723, LOXO-292

    Administered orally

  • CabozantinibDrug

    Administered orally

  • VandetanibDrug

    Administered orally

Study groups in NCT04211337
Study groupRoleWhat they receive
Selpercatinib - Treatment A (TRT A)Experimental160 milligrams Selpercatinib administered orally (PO) twice daily (BID). Adolescent Dose: 92 milligrams per square meter (mg/m2) BID (not to exceed 160 mg BID).
Receives: Drug: Selpercatinib
Cabozantinib or Vandetanib - Treatment B (TRT B)Active comparator140 mg Cabozantinib administered orally daily (QD) or 300 mg Vandetanib administered orally QD per physician choice. Cabozantinib Adolescent Dose: 40 mg/m2. Vandetanib Adolescent Dose: * 0.7 - <0.9 - 100 mg every other day (QOD) * 0.9 - <1.2 - 100 mg QD * 1.2 - <1.6 - 7-day schedule 100 mg - 200 mg - 100 mg - 200 mg - 100 mg - 200 mg - 100 mg * ≥1.6 - 200 QD
Receives: Drug: Cabozantinib; Drug: Vandetanib

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. Progression Free Survival (PFS) by Blinded Independent Central Review (BICR)

    PFS is defined as the time from randomization until the occurrence of documented disease progression by the BICR, per Response Evaluation Criteria in Solid Tumors (RECIST 1.1) criteria, or death from any cause in the absence of BICR-documented progressive disease.

    Progressive Disease (PD) was at least a 20% increase in the sum of the diameters of target lesions, with reference being the smallest sum and an absolute increase of at least 5 mm, or unequivocal progression of non-target lesions, or 1 or more new lesions.

    Measured: Baseline to Progressive Disease or Death from Any Cause, Whichever Occurs First, Up to 39 Months

Secondary outcomes (7)

  1. Treatment Failure-Free Survival (TFFS) by Blinded Independent Committee Review (BICR)

    TFFS by BICR is defined as the time from randomization to the first occurrence of:

    • documented radiographic disease progression per RECIST 1.1 as assessed by BICR; or
    • unacceptable toxicity leading to treatment discontinuation as assessed by the investigator.

    Progressive Disease (PD) was at least a 20% increase in the sum of the diameters of target lesions, with reference being the smallest sum on study and an absolute increase of at least 5 mm, or unequivocal progression of non-target lesions, or 1 or more new lesions.

    To qualify as an event, the toxicity must be from an intolerable AE (defined as any study drug-related AE that meets protocol guidance for treatment discontinuation, with the exception of alopecia); or death (due to any cause).

    Measured: Baseline to Progressive Disease, Unacceptable Toxicity or Death from Any Cause Up to 39 Months

  2. Overall Response Rate (ORR): Percentage of Participants With Complete Response (CR) or Partial Response (PR) by BICR

    ORR is defined as the number of participants who achieved the best overall response (BOR) of CR or PR divided by the total number of participants randomized to each treatment arm. ORR per RECIST 1.1 as assessed by BICR.

    Measured: Baseline through Disease Progression or Death Up to 39 Months

  3. Duration of Response (DoR) by BICR

    DoR by BICR is defined as the time from the date that measurement criteria for complete response (CR) or partial response (PR) (whichever is first recorded) are first met by the BICR or investigator assessment, as applicable, until the first date that disease is recurrent or documented disease progression is observed, per RECIST 1.1 criteria, or the date of death from any cause in the absence of documented disease progression or recurrence. Progressive Disease (PD) was at least a 20% increase in the sum of the diameters of target lesions, with reference being the smallest sum and an absolute increase of at least 5 mm, or unequivocal progression of non-target lesions, or 1 or more new lesions.

    Measured: Date of CR or PR to Date of Disease Progression or Death Due to Any Cause Up to 39 Months

  4. Overall Survival (OS)

    Overall survival (OS) is defined as the time from randomization until death from any cause. If the participant is alive or lost to follow-up at the time of data analysis, OS data will be censored on the last date the participant is known to be alive.

    Measured: Baseline

  5. PFS2 by Investigator

    Progression-free survival 2 (PFS2) is defined as the time from randomization to disease progression (radiographic or symptomatic progression as determined by the investigator) on the next line of treatment or death from any cause in the absence of observed disease progression. If the participant is alive at the cutoff for analysis, and disease progression has not been observed, PFS2 data will be censored on the latest date of last progression-free assessment or start of the next line of treatment.

    Measured: Baseline

  6. Comparative Tolerability: Number of Weeks With High Side Effect Bother Based Score of 3 or 4 on the Functional Assessment of Cancer Therapy Item GP5 (FACT-GP5)

    Comparative tolerability defined as a comparison of the proportion of time on treatment with high side effect bother as assessed by the FACT-GP5. The FACT-GP5 is a single question used to assess the overall bother of the treatment side effects. It is scored using a 5-point rating scale (0 = not at all; 1 = a little bit; 2 = somewhat; 3 = quite a bit; and 4 = very much), where lower scores reflect less bother from treatment side effects.

    Time with high side effect bother (i.e.) score of 3 or 4 is reported here and was derived as follows: cumulative amount of time, in weeks, during which a participant reports high side effect bother divided by the total duration of therapy (weeks), derived as (date of last study treatment dose - date of first study treatment dose + 1) divided by 7.

    Measured: Baseline to Progressive Disease, Unacceptable Toxicity or Death from Any Cause Up to 39 Months

  7. 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)

    Measured: Baseline

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted26 Dec 2019
  2. Study start11 Feb 2020
  3. Primary completion22 May 2023
  4. Study completionNov 2027 (estimated)

Resources

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design
Study type
Interventional
Phase
Phase 3
Primary purpose
Treatment
Allocation
Randomised
Intervention model
Parallel groups
Masking
None (open label)
Enrolment
291 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT04211337
Sponsor's protocol ID
17478
Other IDs
J2G-MC-JZJB, 2019-001978-28, 2019
Acronym
LIBRETTO-531
Status last verified
Mar 2026
First submitted
24 Dec 2019
Last updated
19 Mar 2026
Data monitoring committee
Yes
FDA-regulated drug
Yes
FDA-regulated device
No
Individual data sharing
Yes: 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.
Expanded access
Available

Publications

  • Payakachat N, Gilligan AM, Altman D, Maeda P, Choi J, Bourke S, Speck RM, Spies E, Kopeckova K, Elisei R, Wadsley J, Krajewska J. Assessing side-effect bother, burden, and tolerability: A qualitative study exploring the content validity of the Functional Assessment of Cancer Therapy - Item GP5. J Geriatr Oncol. 2025 Sep;16(7):102304. doi: 10.1016/j.jgo.2025.102304. Epub 2025 Jul 4.

    PubMed 40617171

  • Regnault A, Bunod L, Loubert A, Brose MS, Hess LM, Maeda P, Lin Y, Speck RM, Gilligan AM, Payakachat N. Assessing tolerability with the Functional Assessment of Cancer Therapy item GP5: psychometric evidence from LIBRETTO-531, a phase 3 trial of selpercatinib in medullary thyroid cancer. J Patient Rep Outcomes. 2024 Dec 19;8(1):149. doi: 10.1186/s41687-024-00823-8.

    PubMed 39694978

  • Hadoux J, Elisei R, Brose MS, Hoff AO, Robinson BG, Gao M, Jarzab B, Isaev P, Kopeckova K, Wadsley J, Fuhrer D, Keam B, Bardet S, Sherman EJ, Tahara M, Hu MI, Singh R, Lin Y, Soldatenkova V, Wright J, Lin B, Maeda P, Capdevila J, Wirth LJ; LIBRETTO-531 Trial Investigators. Phase 3 Trial of Selpercatinib in Advanced RET-Mutant Medullary Thyroid Cancer. N Engl J Med. 2023 Nov 16;389(20):1851-1861. doi: 10.1056/NEJMoa2309719. Epub 2023 Oct 21.

    PubMed 37870969

  • 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.

    PubMed 35969032

  • 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.

    PubMed 34292174

Questions about this trial

What is this clinical trial studying?

A Phase 3 study testing Selpercatinib, Cabozantinib and Vandetanib in people with Medullary Thyroid Cancer, run by Loxo Oncology, Inc. at 143 sites in 20 countries.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 0 inclusion and 6 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: under way, not looking for participants, as of the sponsor's last update on 19 Mar 2026. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 143 sites in China, United States, Brazil, France, Spain and 15 more countries. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this phase mean?

Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

Yes. Results are posted in the registry (first posted 13 Jun 2024). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Progression Free Survival (PFS) by Blinded Independent Central Review (BICR) (time frame: Baseline to Progressive Disease or Death from Any Cause, Whichever Occurs First, Up to 39 Months). It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT04211337, which also lists 5 linked publications.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 19 Mar 2026.

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