Clinical trial NCT03157128: A Study of Selpercatinib (LOXO-292) in Participants With Advanced Solid Tumors, RET Fusion-Positive Solid Tumors, and Medullary Thyroid Cancer (LIBRETTO-001)
Official title: A Phase 1/2 Study of Oral Selpercatinib (LOXO-292) in Patients With Advanced Solid Tumors, Including RET Fusion-Positive Solid Tumors, Medullary Thyroid Cancer, and Other Tumors With RET Activation (LIBRETTO-001)
Study type: INTERVENTIONAL
Overall status: ACTIVE_NOT_RECRUITING
Phase: PHASE1
PHASE2
Conditions: Non-Small Cell Lung Cancer
Medullary Thyroid Cancer
Colon Cancer
Any Solid Tumor
Keywords: LOXO-292
KIF5B-RET
M918T
CCDC6-RET
RET-PTC1
NCOA4-RET
RET-PTC
RET-PTC3
RET-PTC4
PRKAR1A-RET
RET-PTC2
GOLGA5-RET
RET-PTC5
ERC1-RET
KTN1-RET
RET-PTC8
HOOK3-RET
PCM1-RET
TRIM24-RET
RET-PTC6
TRIM27-RET
TRIM33-RET
RET-PTC7
AKAP13-RET
FKBP15-RET
SPECC1L-RET
TBL1XR1-RET
BCR-RET
FGRF1OP-RET
RFG8-RET
RET-PTC9
ACBD5-RET
MYH13-RET
CUX1-RET
KIAA1468-RET
FRMD4A-RET
SQSTM1-RET
AFAP1L2-RET
PPFIBP2-RET
EML4-RET
PARD3-RET
G533C
C609F
C609G
C609R
C609S
C609Y
C611F
C611G
C611S
C611Y
C611W
C618F
C618R
C618S
C620F
C620R
C620S
C630R
C630Y
D631Y
C634F
C634G
C634R
C634S
C634W
C634Y
K666E
E768D
L790F
V804L
V804M
A883F
S891A
R912P
CLIP1-RET
Y806C
RET fusion
RET alteration
RET mutation
RET rearrangement
RET translocation
Neoplasms by Site
Neoplasms
Non-Small Cell Lung Cancer
Lung Neoplasms
Carcinoma, Non-Small-Cell Lung
Cancer of Lung
Cancer of the Lung
Lung Cancer
Neoplasms, Lung
Neoplasms, Pulmonary
Pulmonary Cancer
Pulmonary Neoplasms
Respiratory Tract Neoplasms
Lung Diseases
Respiratory Tract Diseases
Carcinoma, Bronchogenic
Bronchial Neoplasms
Medullary Thyroid Cancer
Papillary Thyroid Cancer
Thyroid Diseases
Thyroid Neoplasms
Cancer of the Thyroid
Cancer of Thyroid
Neoplasms, Thyroid
Thyroid Ademona
Thyroid Cancer
Thyroid Carcinoma
Endocrine System Diseases
Endocrine Gland Neoplasms
Head and Neck Neoplasms
Thoracic Neoplasms
CNS tumor
Primary CNS tumor
Cancer of Colon
Cancer of the Colon
Colon Cancer
Colon Neoplasms
Colonic Cancer
Neoplasms, Colonic
Malignant tumor of Breast
Mammary Cancer
Mammary Carcinoma, Human
Mammary Neoplasm, Human
Neoplasms, Breast
Tumors, Breast
Human Mammary Carcinoma
Malignant Neoplasm of Breast
Breast Carcinoma
Breast Tumors
Cancer of the Breast
Breast Neoplasms
Breast Cancer
RET Inhibitor
MTC
NSCLC
selpercatinib
neo-adjuvant treatment in early stage NSCLC
Lead sponsor: Eli Lilly and Company
Sponsor class: INDUSTRY
Collaborators: name: Loxo Oncology, Inc.
First posted: 2017-05-17
Enrollment: 857
Sex: ALL
Minimum age (years): 12
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

Brief summary: This is an open-label, first-in-human study designed to evaluate the safety, tolerability, pharmacokinetics (PK) and preliminary anti-tumor activity of selpercatinib (also known as LOXO-292) administered orally to participants with advanced solid tumors, including rearranged during transfection (RET)-fusion-positive solid tumors, medullary thyroid cancer (MTC) and other tumors with RET activation.

Detailed description: This is an open-label, multi-center Phase 1/2 study in participants with advanced solid tumors, including RET fusion-positive solid tumors, MTC, and other tumors with RET activation. The trial will be conducted in 2 parts: Phase 1 (dose escalation - completed) and phase 2 (dose expansion). Participants with advanced cancer are eligible if they have progressed on or are intolerant to available standard therapies, or no standard or available curative therapy exists, or in the opinion of the Investigator, they would be unlikely to tolerate or derive significant clinical benefit from appropriate standard of care therapy, or they declined standard therapy. A dose of 160 milligrams (mg) twice a day (BID) has been selected as the recommended phase 2 dose (RP2D). Approximately 875 participants with advanced solid tumors harboring a RET gene alteration in tumor and/or blood will be enrolled to one of seven phase 2 cohorts:

* Cohort 1: Advanced RET fusion positive solid tumor other than NSCLC or thyroid cancer for participants who progressed on or intolerant to first line therapy (open)
* Cohort 2: Advanced RET fusion positive solid tumor other than NSCLC or thyroid cancer for treatment naïve participants (open)
* Cohort 3: Advanced RET-mutant MTC participants who progressed on or intolerant to first line therapy (closed)
* Cohort 4: Advanced RET-mutant MTC participants who are treatment naïve (closed)
* Cohort 5: Advanced RET-altered solid tumor for participants other than NSCLC or thyroid cancer and RET-mutant MEN2 spectrum tumors (e.g. pheochromocytoma) otherwise ineligible for cohorts 1-4. See details in inclusion/exclusion criteria (open)
* Cohort 6: Participants otherwise eligible for Cohorts 1-5 who discontinued another RET inhibitor due to intolerance may be eligible with prior Sponsor approval (closed)
* Cohort 7: RET fusion positive early-stage non-small cell lung cancer (NSCLC) participants who are candidates for definitive surgery. Participants will receive selpercatinib in a neoadjuvant and adjuvant setting. Participants will be followed for disease recurrence for up to 5 years from the date of surgery (closed)

Eligibility criteria: Key Inclusion Criteria:

For Phase 1:

* Participants with a locally advanced or metastatic solid tumor that:
* Has progressed on or is intolerant to standard therapy, or
* For which no standard therapy exists, or in the opinion of the Investigator, are not candidates for or would be unlikely to tolerate or derive significant clinical benefit from standard therapy, or
* Decline standard therapy
* Prior multikinase inhibitors (MKIs) with anti-RET activity are allowed
* A RET gene alteration is not required initially. Once adequate PK exposure is achieved, evidence of RET gene alteration in tumor and/or blood is required as identified through molecular assays, as performed for clinical evaluation
* Measurable or non-measurable disease as determined by RECIST 1.1 or RANO as appropriate to tumor type
* Eastern Cooperative Oncology Group (ECOG) score of 0, 1, or 2 or Lansky Performance Score (LPS) greater than or equal to (≥) 40 percent (%) (age less than \[\<\] 16 years) with no sudden deterioration 2 weeks prior to the first dose of study treatment
* Adequate hematologic, hepatic and renal function
* Life expectancy of at least 3 months

For Phase 2: As for phase 1 with the following modifications:

* For Cohort 1: Participants must have received prior standard therapy appropriate for their tumor type and stage of disease, or in the opinion of the Investigator, would be unlikely to tolerate or derive clinical benefit from appropriate standard of care therapy
* Cohorts 1 and 2:

  * Enrollment will be restricted to participants with evidence of a RET gene alteration in tumor
  * At least one measurable lesion as defined by RECIST 1.1 or RANO, as appropriate to tumor type and not previously irradiated
* Cohorts 3 and 4: Enrollment closed
* Cohort 5:

  * Cohorts 1-4 without measurable disease
  * MCT not meeting the requirements for Cohorts 3 or 4
  * MTC syndrome spectrum cancers (e.g., MTC, pheochromocytoma), cancers with neuroendocrine features/differentiation, or poorly differentiated thyroid cancers with other RET alteration/activation may be allowed with prior Sponsor approval
  * cfDNA positive for a RET gene alteration not known to be present in a tumor sample
* Cohort 6: Participants who otherwise are eligible for Cohorts 1, 2 or 5 who discontinued another RET inhibitor may be eligible with prior Sponsor approval
* Cohort 7: Participants with a histologically confirmed stage IB-IIIA NSCLC and a RET fusion; determined to be medically operable and tumor deemed resectable by a thoracic surgical oncologist, without prior systemic treatment for NSCLC

Key Exclusion Criteria (Phase 1 and Phase 2):

* Phase 2 Cohorts 1 and 2: an additional known oncogenic driver
* Cohorts 3 and 4: Enrollment closed
* Cohorts 1, 2 and 5: prior treatment with a selective RET inhibitor Notes: Participants otherwise eligible for Cohorts 1, 2, and 5 who discontinued another selective RET inhibitor may be eligible for Phase 2 Cohort 6 with prior Sponsor approval
* Investigational agent or anticancer therapy (including chemotherapy, biologic therapy, immunotherapy, anticancer Chinese medicine or other anticancer herbal remedy) within 5 half-lives or 2 weeks (whichever is shorter) prior to planned start of LOXO-292 (selpercatinib). In addition, no concurrent investigational anti-cancer therapy is permitted Note: Potential exception for this exclusion criterion will require a valid scientific justification and approval from the Sponsor
* Major surgery (excluding placement of vascular access) within 2 weeks prior to planned start of LOXO-292 (selpercatinib)
* Radiotherapy with a limited field of radiation for palliation within 1 week of planned start of LOXO-292 (selpercatinib), with the exception of participants receiving radiation to more than 30% of the bone marrow or with a wide field of radiation, which must be completed at least 4 weeks prior to the first dose of study treatment
* Any unresolved toxicities from prior therapy greater than Common Terminology Criteria for Adverse Events (CTCAE) Grade 1 at the time of starting study treatment with the exception of alopecia and Grade 2, prior platinum-therapy related neuropathy
* Symptomatic primary CNS tumor, metastases, leptomeningeal carcinomatosis, or untreated spinal cord compression. Participants are eligible if neurological symptoms and CNS imaging are stable and steroid dose is stable for 14 days prior to the first dose of LOXO-292 (selpercatinib) and no CNS surgery or radiation has been performed for 28 days, 14 days if stereotactic radiosurgery (SRS)
* Clinically significant active cardiovascular disease or history of myocardial infarction within 6 months prior to planned start of LOXO-292 (selpercatinib) or prolongation of the QT interval corrected (QTcF) greater than (\>) 470 milliseconds (msec)

  * Participants with implanted pacemakers may enter the study without meeting QTc criteria due to nonevaluable measurement if it is possible to monitor for QT changes.
  * Participants with bundle branch block may be considered for study entry if QTc is appropriate by a formula other than Fridericia's and if it is possible to monitor for QT changes.
* Required treatment with certain strong cytochrome P450 3A4 (CYP3A4) inhibitors or inducers and certain prohibited concomitant medications
* Phase 2 Cohort 7 (neoadjuvant treatment): Participant must not have received prior systemic therapy for NSCLC.

Primary outcomes:
measure: Phase 1: MTD
timeFrame: The first 28 days of treatment (Cycle 1)
measure: Phase 1: RP2D
timeFrame: The first 28 days of treatment (Cycle 1) and every cycle (28 days) for approximately 12 months (or earlier if the participant discontinues from the study)
measure: Phase 2: Objective Response Rate
timeFrame: Approximately every 8 weeks for one year, then every 12 weeks, and 7 days after the last dose (for up to 2 years) in participants who have not progressed.
Secondary outcomes:
measure: Phase 1: Number of Participants with a Treatment-Related Adverse Event(s) (TRAE[s])
timeFrame: From the time of informed consent, for approximately 24 months (or earlier if the participant discontinues from the study), and through Safety Follow-up (28 days after the last dose)
measure: Phase 1: Number of Participants with an Abnormal Laboratory or Physical Exam Result(s)
timeFrame: From the time of informed consent, for approximately 24 months (or earlier if the participant discontinues from the study), and through Safety Follow-up (28 days after the last dose)
measure: Phase 1: Overall Response Rate (ORR) based on RECIST 1.1 or RANO, as Appropriate to Tumor Type
timeFrame: Approximately every 8 weeks for one year, then every 12 weeks, 7 days after the last dose (for up to 2 years) in participants who have not progressed
measure: Phase 2: ORR (by Investigator)
timeFrame: Approximately every 8 weeks for one year, then every 12 weeks, 7 days after the last dose (for up to 2 years) in participants who have not progressed
measure: Phase 2: Best Change in Tumor Size from Baseline (by IRC and Investigator)
timeFrame: Approximately every 8 weeks for one year, then every 12 weeks, 7 days after the last dose (for up to 2 years) in participants who have not progressed
measure: Phase 2: Duration of Response (DOR; by IRC and Investigator)
timeFrame: Approximately every 8 weeks for one year, then every 12 weeks, 7 days after the last dose (for up to 2 years) in participants who have not progressed
measure: Phase 2: Central Nervous System (CNS) ORR (by IRC)
timeFrame: Approximately every 8 weeks for one year, then every 12 weeks, 7 days after the last dose (for up to 2 years) in participants who have not progressed
measure: Phase 2: CNS DOR (by IRC)
timeFrame: Approximately every 8 weeks for one year, then every 12 weeks, 7 days after the last dose (for up to 2 years) in participants who have not progressed
measure: Phase 2: Time to Any and Best Response (by IRC and Investigator)
timeFrame: every 8 weeks for one year, then every 12 weeks, 7 days after the last dose (for up to 2 years) in participants who have not progressed
measure: Phase 2: CBR (by IRC and Investigator)
timeFrame: Approximately every 8 weeks for one year, then every 12 weeks, 7 days after the last dose (for up to 2 years) in participants who have not progressed
measure: Phase 2: PFS (by IRC and Investigator)
timeFrame: Approximately every 8 weeks for one year, then every 12 weeks, 7 days after the last dose (for up to 2 years) in participants who have not progressed
measure: Phase 2: Overall Survival (OS)
timeFrame: Approximately every 8 weeks for one year, then every 12 weeks, 7 days after the last dose (for up to 2 years) in participants who have not progressed
measure: Phase 2: Percentage of Participants with any Serious Adverse Event (SAE[s])
timeFrame: From the time of informed consent, for approximately 24 months (or earlier if the participant discontinues from the study), and through Safety Follow-up (28 days after the last dose)
measure: Phase 1 and 2: Pharmacokinetics (PK): Area Under the Plasma Concentration-Time Curve of LOXO-292 (Selpercatinib)
timeFrame: Cycle 1 Day 1 through Cycle 5 Day 1 (Cycle = 28 days)
measure: Phase 1 and 2: PK: Maximum Concentration (Cmax) of LOXO-292 (Selpercatinib)
timeFrame: Cycle 1 Day 1 through Cycle 5 Day 1 (Cycle = 28 days)
Other outcomes:
Locations:
city: Scottsdale
country: United States
facility: Mayo Clinic of Scottsdale
state: Arizona
city: Duarte
country: United States
facility: City of Hope National Medical Center
state: California
city: Los Angeles
country: United States
facility: UCLA Medical Center
state: California
city: Newport Beach
country: United States
facility: Hoag Memorial Hospital Presbyterian
state: California
city: Oakland
country: United States
facility: Kaiser Permanente
state: California
city: Orange
country: United States
facility: Irvine Medical Center
state: California
city: San Diego
country: United States
facility: University of California - San Diego
state: California
city: San Francisco
country: United States
facility: UCSF Medical Center at Mission Bay
state: California
city: Walnut Creek
country: United States
facility: Kaiser Permanente Medical Center
state: California
city: Denver
country: United States
facility: Sarah Cannon Research Institute at HealthOne
state: Colorado
city: New Haven
country: United States
facility: Yale Cancer Center
state: Connecticut
city: Jacksonville
country: United States
facility: Mayo Clinic in Florida
state: Florida
city: Pembroke
country: United States
facility: Memorial Hospital Pembroke
state: Florida
city: Atlanta
country: United States
facility: Emory University
state: Georgia
city: Chicago
country: United States
facility: University of Chicago Medicine-Comprehensive Cancer Center
state: Illinois
city: New Orleans
country: United States
facility: Ochsner Clinic Foundation
state: Louisiana
city: Baltimore
country: United States
facility: University of Maryland Medical Center
state: Maryland
city: Baltimore
country: United States
facility: Johns Hopkins University
state: Maryland
city: Boston
country: United States
facility: Massachusetts General Hospital
state: Massachusetts
city: Boston
country: United States
facility: Dana-Farber Cancer Institute
state: Massachusetts
city: Ann Arbor
country: United States
facility: University of Michigan
state: Michigan
city: Grand Rapids
country: United States
facility: START Midwest
state: Michigan
city: Rochester
country: United States
facility: Mayo Clinic
state: Minnesota
city: Saint Louis
country: United States
facility: Washington University Medical School
state: Missouri
city: Las Vegas
country: United States
facility: Comprehensive Cancer Centers of Nevada
state: Nevada
city: Buffalo
country: United States
facility: Roswell Park Cancer Institute
state: New York
city: New York
country: United States
facility: NYU Langone
state: New York
city: New York
country: United States
facility: Memorial Sloan Kettering Cancer Center
state: New York
city: Chapel Hill
country: United States
facility: University of North Carolina
state: North Carolina
city: Cleveland
country: United States
facility: Cleveland Clinic Foundation
state: Ohio
city: Columbus
country: United States
facility: Ohio State University Hospital
state: Ohio
city: Portland
country: United States
facility: Oregon Health and Science University
state: Oregon
city: Philadelphia
country: United States
facility: University of Pennsylvania Hospital
state: Pennsylvania
city: Philadelphia
country: United States
facility: Thomas Jefferson University
state: Pennsylvania
city: Nashville
country: United States
facility: Sarah Cannon Research Institute SCRI
state: Tennessee
city: Nashville
country: United States
facility: Vanderbilt University Medical Center
state: Tennessee
city: Dallas
country: United States
facility: University of Texas Southwestern Medical Center at Dallas
state: Texas
city: Houston
country: United States
facility: University of Texas MD Anderson Cancer Center
state: Texas
city: Salt Lake City
country: United States
facility: Huntsman Cancer Institute
state: Utah
city: Fairfax
country: United States
facility: USO-Virginia Cancer Specialists, PC
state: Virginia
city: Madison
country: United States
facility: University of Wisconsin-Madison Hospital and Health Clinic
state: Wisconsin
city: St Leonards
country: Australia
facility: Royal North Shore Hospital
state: New South Wales
city: Melbourne
country: Australia
facility: Peter MacCallum Cancer Centre
state: Victoria
city: Vancouver
country: Canada
facility: BC Cancer Vancouver
state: British Columbia
city: Copenhagen
country: Denmark
facility: Rigshospitalet
city: Bordeaux
country: France
facility: Institut Bergonié - Centre Régional de Lutte Contre Le Cancer de Bordeaux et Sud Ouest
state: Aquitaine
city: Lyon
country: France
facility: Centre Leon Berard
state: Rhône-Alpes
city: Marseille
country: France
facility: APHM Hôpital de la Timone
city: Montpellier Cedex 5
country: France
facility: Institut du Cancer de Montpellier - Val d'aurelle
city: Villejuif Cedex
country: France
facility: Gustave Roussy
city: Paris
country: France
facility: Hôpital Européen Georges Pompidou
state: Île-de-France
city: Wuerzburg
country: Germany
facility: Universitätsklinikum Würzburg A. ö. R.
state: Bayern
city: Köln
country: Germany
facility: Universitätsklinikum Köln
state: Nordrhein-Westfalen
city: Hong Kong
country: Hong Kong
facility: Prince of Wales Hospital
state: Shatin, New Territories
city: Ramat Gan
country: Israel
facility: Sheba Medical Center
state: HaMerkaz
city: Jerusalem
country: Israel
facility: Shaare Zedek Medical Center
state: Yerushalayim
city: Beer-Sheva
country: Israel
facility: Soroka Medical Center - Pediatric Outpatient Clinic
city: Jerusalem
country: Israel
facility: Hadassah Medical Center
city: Milano
country: Italy
facility: Istituto Nazionale dei Tumori
state: Lombardie
city: Nagoya
country: Japan
facility: Nagoya University Hospital
state: Aichi
city: Kashiwa
country: Japan
facility: National Cancer Center Hospital East
state: Chiba
city: Sapporo
country: Japan
facility: Hokkaido University Hospital
state: Hokkaido
city: Akashi
country: Japan
facility: Hyogo Cancer Center
state: Hyogo
city: Kanazawa
country: Japan
facility: Kanazawa University Hospital
state: Ishikawa
city: Osaka Sayama-shi
country: Japan
facility: Kindai University Hospital
state: Osaka
city: Nagaizumi-cho,Sunto-gun
country: Japan
facility: Tominaga Hospital
state: Shizuoka
city: Chuo-ku
country: Japan
facility: National Cancer Center Hospital
state: Tokyo
city: Koto
country: Japan
facility: Japanese Foundation for Cancer Research
state: Tokyo
city: Yonago
country: Japan
facility: Tottori University Hospital
state: Tottori
city: Fukuoka
country: Japan
facility: National Hospital Organization Kyushu Cancer Center
city: Okayama
country: Japan
facility: Okayama University Hospital
city: Osaka
country: Japan
facility: Osaka City General Hospital
city: Goyang-si
country: Korea, Republic of
facility: National Cancer Center
state: Kyǒnggi-do
city: Seongnam
country: Korea, Republic of
facility: Seoul National University Bundang Hospital
state: Kyǒnggi-do
city: Seoul
country: Korea, Republic of
facility: Severance Hospital, Yonsei University Health System
state: Seoul-teukbyeolsi [Seoul]
city: Seoul
country: Korea, Republic of
facility: Asan Medical Center
state: Seoul-teukbyeolsi [Seoul]
city: Seoul
country: Korea, Republic of
facility: Samsung Medical Center
city: Singapore
country: Singapore
facility: National Cancer Centre Singapore
state: Central Singapore
city: Barcelona
country: Spain
facility: Hospital Universitari Vall d'Hebron
state: Barcelona [Barcelona]
city: Madrid
country: Spain
facility: Hospital Universitario Fundación Jiménez Díaz
city: Madrid
country: Spain
facility: Hospital Madrid Norte Sanchinarro
city: Luzern 16
country: Switzerland
facility: Kantonsspital Luzern
state: Luzern
city: Taichung
country: Taiwan
facility: Taichung Veterans General Hospital
city: Taipei
country: Taiwan
facility: National Taiwan University Hospital
city: London
country: United Kingdom
facility: Royal Marsden Hospital
Study officials:
affiliation: Eli Lilly and Company
name: Call 1-877-CTLILLY (1-877-285-4559) or 1-317-615-4559 Mon - Fri 9 AM - 5 PM Eastern time (UTC/GMT - 5 hours, EST)
role: STUDY_DIRECTOR
References:
citation: Subbiah V, Wolf J, Konda B, Kang H, Spira A, Weiss J, Takeda M, Ohe Y, Khan S, Ohashi K, Soldatenkova V, Szymczak S, Sullivan L, Wright J, Drilon A. Tumour-agnostic efficacy and safety of selpercatinib in patients with RET fusion-positive solid tumours other than lung or thyroid tumours (LIBRETTO-001): a phase 1/2, open-label, basket trial. Lancet Oncol. 2022 Oct;23(10):1261-1273. doi: 10.1016/S1470-2045(22)00541-1. Epub 2022 Sep 12.
citation: Rolfo C, Hess LM, Jen MH, Peterson P, Li X, Liu H, Lai Y, Sugihara T, Kiiskinen U, Vickers A, Summers Y. External control cohorts for the single-arm LIBRETTO-001 trial of selpercatinib in RET+ non-small-cell lung cancer. ESMO Open. 2022 Aug;7(4):100551. doi: 10.1016/j.esmoop.2022.100551. Epub 2022 Aug 2.
citation: Subbiah V, Gainor JF, Oxnard GR, Tan DSW, Owen DH, Cho BC, Loong HH, McCoach CE, Weiss J, Kim YJ, Bazhenova L, Park K, Daga H, Besse B, Gautschi O, Rolfo C, Zhu EY, Kherani JF, Huang X, Kang S, Drilon A. Intracranial Efficacy of Selpercatinib in RET Fusion-Positive Non-Small Cell Lung Cancers on the LIBRETTO-001 Trial. Clin Cancer Res. 2021 Aug 1;27(15):4160-4167. doi: 10.1158/1078-0432.CCR-21-0800. Epub 2021 Jun 4.
citation: Wirth LJ, Sherman E, Robinson B, Solomon B, Kang H, Lorch J, Worden F, Brose M, Patel J, Leboulleux S, Godbert Y, Barlesi F, Morris JC, Owonikoko TK, Tan DSW, Gautschi O, Weiss J, de la Fouchardiere C, Burkard ME, Laskin J, Taylor MH, Kroiss M, Medioni J, Goldman JW, Bauer TM, Levy B, Zhu VW, Lakhani N, Moreno V, Ebata K, Nguyen M, Heirich D, Zhu EY, Huang X, Yang L, Kherani J, Rothenberg SM, Drilon A, Subbiah V, Shah MH, Cabanillas ME. Efficacy of Selpercatinib in RET-Altered Thyroid Cancers. N Engl J Med. 2020 Aug 27;383(9):825-835. doi: 10.1056/NEJMoa2005651.
citation: Drilon A, Oxnard GR, Tan DSW, Loong HHF, Johnson M, Gainor J, McCoach CE, Gautschi O, Besse B, Cho BC, Peled N, Weiss J, Kim YJ, Ohe Y, Nishio M, Park K, Patel J, Seto T, Sakamoto T, Rosen E, Shah MH, Barlesi F, Cassier PA, Bazhenova L, De Braud F, Garralda E, Velcheti V, Satouchi M, Ohashi K, Pennell NA, Reckamp KL, Dy GK, Wolf J, Solomon B, Falchook G, Ebata K, Nguyen M, Nair B, Zhu EY, Yang L, Huang X, Olek E, Rothenberg SM, Goto K, Subbiah V. Efficacy of Selpercatinib in RET Fusion-Positive Non-Small-Cell Lung Cancer. N Engl J Med. 2020 Aug 27;383(9):813-824. doi: 10.1056/NEJMoa2005653.