A Study to Test the Effect of the Drug Larotrectinib in Adults and Children With NTRK-fusion Positive Solid Tumors

NCT ID: NCT02576431

Last Updated: 2025-11-12

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

COMPLETED

Clinical Phase

PHASE2

Total Enrollment

215 participants

Study Classification

INTERVENTIONAL

Study Start Date

2015-09-30

Study Completion Date

2025-09-29

Brief Summary

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This research study is done to test how well different types of cancer respond to the drug called larotrectinib. The cancer must have a change in a particular gene (NTRK1, NTRK2 or NTRK3). Larotrectinib is a drug that blocks the actions of these NTRK genes in cancer cells and can therefore be used to treat cancer.

Detailed Description

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The primary objective of this study is to investigate the efficacy of larotrectinib for the treatment of advanced solid tumors harboring a fusion of neurotrophic tyrosine receptor kinase (NTRK) of types 1-3 in children and adults.

Secondary objectives comprise the efficacy and safety of larotrectinib in different NTRK-tumor types.

Conditions

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Solid Tumors Harboring NTRK Fusion

Keywords

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Non-small cell lung cancer Thyroid cancer Sarcoma Colorectal cancer Salivary gland cancer Biliary cancer Central nervous system (CNS) Tumor Breast cancer Melanoma Neurotrophic tyrosine receptor kinase (NTRK) NTRK1 NTRK2 NTRK3 Fusion Positive TRK fusion TRKA TRKB TRKC ETV6

Study Design

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Allocation Method

NON_RANDOMIZED

Intervention Model

PARALLEL

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

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Arm 1_NSCLC

Patients with solid non-small cell lung cancer (NSCLC) harboring NTRK fusions (arm closed)

Group Type EXPERIMENTAL

BAY2757556 (Larotrectinib, Vitrakvi)

Intervention Type DRUG

Larotrectinib will be administered orally as capsule or liquid solution at a dose of 100 mg twice daily in continuing 28-days cycles.

Arm 2_Thyroid

Patients with solid thyroid tumors harboring NTRK fusions (arm closed)

Group Type EXPERIMENTAL

BAY2757556 (Larotrectinib, Vitrakvi)

Intervention Type DRUG

Larotrectinib will be administered orally as capsule or liquid solution at a dose of 100 mg twice daily in continuing 28-days cycles.

Arm 3_Sarcoma

Patients with soft-tissue sarcoma harboring NTRK fusions (arm closed)

Group Type EXPERIMENTAL

BAY2757556 (Larotrectinib, Vitrakvi)

Intervention Type DRUG

Larotrectinib will be administered orally as capsule or liquid solution at a dose of 100 mg twice daily in continuing 28-days cycles.

Arm 4_Colorectal

Patients with solid colorectal tumors harboring NTRK fusions (arm closed)

Group Type EXPERIMENTAL

BAY2757556 (Larotrectinib, Vitrakvi)

Intervention Type DRUG

Larotrectinib will be administered orally as capsule or liquid solution at a dose of 100 mg twice daily in continuing 28-days cycles.

Arm 5_Salivary

Patients with solid salivary tumors harboring NTRK fusions (arm closed)

Group Type EXPERIMENTAL

BAY2757556 (Larotrectinib, Vitrakvi)

Intervention Type DRUG

Larotrectinib will be administered orally as capsule or liquid solution at a dose of 100 mg twice daily in continuing 28-days cycles.

Arm 6_Biliary

Patients with solid biliary tumors harboring NTRK fusions (arm closed)

Group Type EXPERIMENTAL

BAY2757556 (Larotrectinib, Vitrakvi)

Intervention Type DRUG

Larotrectinib will be administered orally as capsule or liquid solution at a dose of 100 mg twice daily in continuing 28-days cycles.

Arm 7_Primary CNS

Patients with solid tumors in the primary central nervous system (CNS) harboring NTRK fusions (arm closed)

Group Type EXPERIMENTAL

BAY2757556 (Larotrectinib, Vitrakvi)

Intervention Type DRUG

Larotrectinib will be administered orally as capsule or liquid solution at a dose of 100 mg twice daily in continuing 28-days cycles.

Arm 8_Other tumors

Patients with e.g. kidney cancer, squamous cell cancer of head or neck or ovarian solid tumors harboring NTRK fusions (arm closed)

Group Type EXPERIMENTAL

BAY2757556 (Larotrectinib, Vitrakvi)

Intervention Type DRUG

Larotrectinib will be administered orally as capsule or liquid solution at a dose of 100 mg twice daily in continuing 28-days cycles.

Arm 9_Solid tumors without confirmed NTRK fusion

Patients eligible for arms 1 to 8, but with documented NTRK fusion from a laboratory where CLIA or equivalent certification cannot be confirmed by the sponsor at the time of consent (arm closed)

Group Type EXPERIMENTAL

BAY2757556 (Larotrectinib, Vitrakvi)

Intervention Type DRUG

Larotrectinib will be administered orally as capsule or liquid solution at a dose of 100 mg twice daily in continuing 28-days cycles.

Arm 10_Prospective cohort

Patients with melanoma, non secretory breast and colorectal cancer or other tumor types harboring NTRK fusions, except soft tissue sarcoma, salivary gland and thyroid cancer (arm closed)

Group Type EXPERIMENTAL

BAY2757556 (Larotrectinib, Vitrakvi)

Intervention Type DRUG

Larotrectinib will be administered orally as capsule or liquid solution at a dose of 100 mg twice daily in continuing 28-days cycles.

Arm 11_Bone health cohort

Patients with all tumor types harboring NTRK fusions, not eligible for the main prospective cohort, including patients with non-measurable disease

Group Type EXPERIMENTAL

BAY2757556 (Larotrectinib, Vitrakvi)

Intervention Type DRUG

Larotrectinib will be administered orally as capsule or liquid solution at a dose of 100 mg twice daily in continuing 28-days cycles.

Interventions

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BAY2757556 (Larotrectinib, Vitrakvi)

Larotrectinib will be administered orally as capsule or liquid solution at a dose of 100 mg twice daily in continuing 28-days cycles.

Intervention Type DRUG

Other Intervention Names

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LOXO-101

Eligibility Criteria

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Inclusion Criteria

* Locally-advanced or metastatic malignancy with an NTRK1, NTRK2, or NTRK3 gene fusion, identified through molecular assays as routinely performed at CLIA or other similarly-certified laboratories. Subjects who have an NTRK gene fusion identified in a lab where CLIA or equivalent certification cannot be confirmed by the Sponsor at the time of consent may have been enrolled in Cohort 9 as per protocol versions 1.0 - 8.0. From protocol version 9.0: CLIA or similar certification of the lab performing the fusion assay is required. However, patients may be included after discussion with the sponsor if the lab performing the fusion assay is not CLIA or similar certified.
* Subjects who have received prior standard therapy appropriate for their tumor type and stage of disease, or who have no satisfactory alternative treatments and in the opinion of the Investigator, would be unlikely to tolerate or derive clinically meaningful benefit from appropriate standard of care therapy.
* Subjects must have at least one measurable lesion as defined by RECIST v1.1 (Eisenhauer et al. 2009). Subjects with solid tumors without RECIST v1.1 measurable disease (e.g., evaluable disease only) had been eligible for enrollment to Cohort 8 as per protocol versions 1.0 - 8.0, regardless of tumor type. Subjects with primary CNS tumors should meet the following criteria:

1. Have received prior treatment including radiation and/or chemotherapy, with radiation completed \> 12 weeks prior to C1D1 of therapy, as recommended or appropriate for that CNS tumor type.
2. Have ≥ 1 site of bi-dimensionally measurable disease (confirmed by magnetic resonance imaging \[MRI\] and evaluable by RANO criteria), with the size of at least one of the measurable lesions ≥ 1 cm in each dimension and noted on more than one imaging slice.
3. Imaging study performed within 28 days before enrollment. If on steroid therapy, the dose must be stable for at least 7 days immediately before and during the imaging study.
4. Must be neurologically stable based on stable neurologic exam for 7 days prior to enrollment.

For subjects eligible for enrollment to bone health cohort, inclusion criterion 3 is modified as the following:
5. Subjects must have at least one lesion at baseline (measurable or non-measurable as defined by RECIST v1.1 or RANO criteria, as appropriate to tumor type).
6. Subjects with primary CNS tumors must be neurologically stable based on stable neurologic exam for 7 days prior to enrollment.
* At least 18 years of age
* Performance Status: Eastern Cooperative Oncology Group (ECOG) score ≤ 3. If enrolled with primary CNS tumor to be assessed by RANO, Karnofsky Performance Score (KPS) ≥ 50%.
* Tumor tissue before treatment (mandatory). If neither fresh tissue can be obtained nor archival tissue is available patients might be enrolled after consultation with the sponsor.
* Adequate organ function as defined by the following criteria:

1. Serum AST and serum ALT \< 2.5 x upper limit of normal (ULN), or AST and ALT \< 5 x ULN if liver function abnormalities are due to underlying malignancy
2. Total bilirubin \< 2.5 x ULN, except in the setting of biliary obstruction. Subjects with a known history of Gilberts Disease and an isolated elevation of indirect bilirubin are eligible
3. Serum creatinine \< 2.0 x ULN OR an estimated glomerular filtration rate ≥ 30 mL/minute using the Cockcroft-Gault formula: (140- age) x body weight (kg) x 0.85 (if female)/serum creatinine (mg/dL) x 72 with either result acceptable for enrollment.
* Ability to comply (or for guardian to ensure compliance) with outpatient treatment, laboratory monitoring, and required clinic visits for the duration of study participation.
* Willingness of men and women of reproductive potential to use double effective birth control methods, defined as one used by the subject and another by his/her partner, for the duration of treatment and for 1 month following study completion.
* For subjects eligible for enrollment to bone health cohort only: life expectancy of at least 6 months, based on investigator assessment.

Exclusion Criteria

* Investigational agent or anticancer therapy within 2 weeks prior to the planned start of larotrectinib or 5 half-lives, whichever is shorter, and without recovery of acute and/or clinically significant toxicities from that therapy.
* Prior progression while receiving approved or investigational tyrosine kinase inhibitors targeting TRK. Subjects who received less than 28 days of treatment and discontinued because of intolerance or toxicity are eligible.
* Symptomatic or unstable brain metastases. (Note: Subjects with asymptomatic brain metastases are eligible to participate in the study.) Subjects with primary CNS tumors are eligible.
* Uncontrolled concurrent malignancy that would limit assessment of efficacy of larotrectinib. Allowed conditions may include, but are not limited to in situ cancers of cervix, breast, or skin, superficial bladder cancer, limited-stage prostate cancer, and basal or squamous cancers of the skin.
* Active uncontrolled systemic bacterial, viral, or fungal infection CTCAE grade ≥ 2; unstable cardiovascular disease, or other systemic disease that would limit compliance with study procedures. Unstable cardiovascular disease is defined as:

1. In adults, persistently uncontrolled hypertension defined as systolic blood pressure (BP) \> 150 mmHg and/or diastolic BP \> 100 mmHg despite antihypertensive therapy.
2. Myocardial infarction within 3 months of screening.
3. Stroke within 3 months of screening.
* Inability to discontinue treatment with a strong CYP3A4 inhibitor or inducer
* Currently recovering from AEs/ ADRs due to previous treatments (excluding alopecia). Inclusion is only advised once the AE/ADR resolves or recovers to baseline or at least to CTCAE grade 1.
* Known or suspected hypersensitivity against the active substance or any of the ingredients of the IMP.
* Known history of HIV infection. All patients must be screened for HIV up to 28 days prior to study drug start using a blood test for HIV according to local regulations.
* HBV or HCV infection. All patients must be screened for HBV and HCV up to 28 days prior to study drug start using the routine hepatitis virus laboratorial panel. Patients positive for HBsAg or HBcAb will be eligible if they are negative for HBVDNA. Patients positive for anti-HCV antibody will be eligible if they are negative for HCV-RNA.
Minimum Eligible Age

18 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Bayer

INDUSTRY

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Locations

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Stanford Cancer Center Palo Alto

Palo Alto, California, United States

Site Status

UCLA Health Santa Monica Cancer Care

Santa Monica, California, United States

Site Status

Memorial Cancer Institute at West

Pembroke Pines, Florida, United States

Site Status

The University of Chicago Medical Center - Hyde Park - Hematology & Oncology

Chicago, Illinois, United States

Site Status

Mass General Cancer Center

Boston, Massachusetts, United States

Site Status

Dana-Farber Cancer Institute - Oncology Department

Boston, Massachusetts, United States

Site Status

Memorial Sloan Kettering Cancer Center New York - Main Campus

New York, New York, United States

Site Status

UNC Hospitals - UNC Lineberger Comprehensive Cancer Center

Chapel Hill, North Carolina, United States

Site Status

Wake Forest Baptist Health

Winston-Salem, North Carolina, United States

Site Status

Cleveland Clinic - Neurology

Cleveland, Ohio, United States

Site Status

Sidney Kimmel Cancer Center - Jefferson Health

Philadelphia, Pennsylvania, United States

Site Status

Fox Chase Cancer Center

Philadelphia, Pennsylvania, United States

Site Status

Avera Cancer Institute - Sioux Falls

Sioux Falls, South Dakota, United States

Site Status

Vanderbilt University Medical Center

Nashville, Tennessee, United States

Site Status

The University of Texas MD Anderson Cancer Center - Texas Medical Center

Houston, Texas, United States

Site Status

Inova Schar Cancer Institute

Fairfax, Virginia, United States

Site Status

Fred Hutch Cancer Center - Sloan Clinic 1

Seattle, Washington, United States

Site Status

West Virginia University

Morgantown, West Virginia, United States

Site Status

Hospital Alemán

Buenos Aires, Ciudad Auton. de Buenos Aires, Argentina

Site Status

Centro Estudios Médicos e Invest. Clínicas "Dr. N. Quirno"

Buenos Aires, Ciudad Auton. de Buenos Aires, Argentina

Site Status

Fundación Cenit para la Investigación en Neurociencias

CABA, Ciudad Auton. de Buenos Aires, Argentina

Site Status

Centro Medico Austral

TBC, Ciudad Auton. de Buenos Aires, Argentina

Site Status

Centro Médico San Roque

San Miguel de Tucumán, Tucumán Province, Argentina

Site Status

Macquarie University Hospital

Sydney, New South Wales, Australia

Site Status

Royal Darwin Hospital

Tiwi, Northern Territory, Australia

Site Status

St John of God Healthcare

Subiaco, Western Australia, Australia

Site Status

Institut Jules Bordet/Jules Bordet Instituut

Brussels, , Belgium

Site Status

Hosp. Araujo Jorge da Associação de Combate ao Câncer

Goiânia, Goiás, Brazil

Site Status

Cenantron Centro Avançado de Tratamento Oncológico, Ltda.

Belo Horizonte, Minas Gerais, Brazil

Site Status

Fundacao Pio XII - Hospital de Cancer de Barretos

Barretos/SP, São Paulo, Brazil

Site Status

Instituto do Cancer do Estado de Sao Paulo

São Paulo, São Paulo, Brazil

Site Status

Real e Benemérita Associação Portuguesa de Beneficência

São Paulo, São Paulo, Brazil

Site Status

IBCC - Instituto Brasileiro de Controle do Cancer

São Paulo, São Paulo, Brazil

Site Status

Instituto Nacional do Câncer - INCA - HC II

Rio de Janeiro, , Brazil

Site Status

INCA - Hospital do Cancer III

Rio de Janeiro, , Brazil

Site Status

Oncoclínicas Rio de Janeiro S.A

Rio de Janeiro, , Brazil

Site Status

Hospital Sirio Libanes

São Paulo, , Brazil

Site Status

Tom Baker Cancer Centre

Calgary, Alberta, Canada

Site Status

Beijing Cancer Hospital - Oncology Department

Beijing, Beijing Municipality, China

Site Status

Sun Yat-sen University Cancer Center

Guangzhou, Guangdong, China

Site Status

Sichuan University West China Hospital

Chengdu, Sichuan, China

Site Status

Zhongshan Hospital, Fudan University - Oncology Department

Shanghai, , China

Site Status

Instituto Nacional de Cancerología INC Colombia

Bogota, Cundinamarca, Colombia

Site Status

Oncomédica S.A.

Montería, Departamento de Córdoba, Colombia

Site Status

Fundación Oftalmológica de Santander Carlos Ardila Lule

Floridablanca, Santander Department, Colombia

Site Status

Fakultní Nemocnice Olomouc

Olomouc, , Czechia

Site Status

Rigshospitalet - Kræftbehandling

Copenhagen OE, , Denmark

Site Status

Hopital Jean Minjoz

Besançon, , France

Site Status

Institut Bergonie - Unicancer Nouvelle Aquitaine - Service Oncologie medicale

Bordeaux, , France

Site Status

Centre Antoine Lacassagne - Departement Oncologie

Nice, , France

Site Status

Hopital Saint Antoine APHP - Departement Oncologie

Paris, , France

Site Status

APHP-Hopital la Pitie Salpetriere-Departement oncologie

Paris, , France

Site Status

Hôpital de la Milétrie

Poitiers, , France

Site Status

Institut de Cancerologie Ouest - Saint-Herblain

Saint-Herblain, , France

Site Status

ICANS - Institut de Cancerologie de Strasbourg Europe - service oncologie medicale

Strasbourg, , France

Site Status

Charité Comprehensive Cancer Center (CCCC)

Berlin, , Germany

Site Status

All India Institute of Medical Sciences

Bhubaneswar, Odisha, India

Site Status

Jawaharlal Institute Of Postgraduate Medical Education and R

Gorimedu, Puducherry, India

Site Status

St Vincents University Hospital

Dublin, , Ireland

Site Status

A.O.R.N. San Giuseppe Moscati

Avellino, Campania, Italy

Site Status

A.O.U. di Bologna Policlinico S.Orsola Malpighi

Bologna, Emilia-Romagna, Italy

Site Status

A.S.U. Friuli Centrale - A. Regionale Coordinamento Salute

Udine, Friuli Venezia Giulia, Italy

Site Status

IRCCS Istituti Fisioterapici Ospitalieri - IFO

Rome, Lazio, Italy

Site Status

Istituto Europeo di Oncologia s.r.l

Milan, Lombardy, Italy

Site Status

Istituto Oncologico Veneto

Padua, Veneto, Italy

Site Status

Nagoya University Hospital

Nagoya, Aichi-ken, Japan

Site Status

National Cancer Center Hospital East

Kashiwa, Chiba, Japan

Site Status

Hokkaido University Hospital

Sapporo, Hokkaido, Japan

Site Status

The Cancer Institute Hospital of JFCR

Koto-ku, Tokyo, Japan

Site Status

IPO Porto

Porto, , Portugal

Site Status

Arkhangelsk Clinical Oncology Dispensary

Arkhangelsk, , Russia

Site Status

Republican Clinical Oncology Dispensary Kazan

Kazan', , Russia

Site Status

Russian Oncological Scientific Center n.a. N.N. Blokhin RAMS

Moscow, , Russia

Site Status

1st Moscow State Medical University n.a. I.M.Sechenov

Moscow, , Russia

Site Status

Clinical Diagnostical Center

Nizhny Novgorod, , Russia

Site Status

St. Petersburg Clinical Onc. Cent. of Spec. Types of Care

Saint Petersburg, , Russia

Site Status

National Cancer Center Singapore - Oncology Department

Singapore, , Singapore

Site Status

Onkologicky Ustav Svatej Alzbety, s.r.o.

Bratislava, , Slovakia

Site Status

Narodny onkologicky ustav

Bratislava, , Slovakia

Site Status

Severance Hospital, Yonsei University Health System

Seoul, Seoul Teugbyeolsi, South Korea

Site Status

Asan Medical Center-Ophthalmology

Seoul, Seoul Teugbyeolsi, South Korea

Site Status

Seoul National University Hospital

Seoul, Seoul Teugbyeolsi, South Korea

Site Status

Samsung Medical Center - Oncology Department

Seoul, , South Korea

Site Status

Institut Catala D'oncologia - Oncologia

Barcelona, L Hospitalet de Llobregat, Spain

Site Status

Hospital del Mar

Barcelona, , Spain

Site Status

Ciutat Sanitaria i Universitaria de la Vall d'Hebron

Barcelona, , Spain

Site Status

Hospital General Universitario Gregorio Maranon | Oncologia

Madrid, , Spain

Site Status

Hospital Universitario Fundacion Jimenez Diaz

Madrid, , Spain

Site Status

Centro Integral Oncológico Clara Campal

Madrid, , Spain

Site Status

Karolinska Universitetssjukhuset Solna - Tema Cancer

Stockholm, , Sweden

Site Status

Tri-Service General Hospital

Taipei, , Taiwan

Site Status

Health Ministry Of Türkiye Republic Ankara Bilkent City Hospital

Ankara, , Turkey (Türkiye)

Site Status

Trakya Univ. Tip Fak.

Edirne, , Turkey (Türkiye)

Site Status

Istanbul Universitesi Istanbul Tip Fakultesi

Istanbul, , Turkey (Türkiye)

Site Status

Istanbul Universitesi Cerrahpasa-Cerrahpasa Tip Fakultesi

Istanbul, , Turkey (Türkiye)

Site Status

TC Saglik Bakanligi Goztepe ProfDr Suleyman Yalcin Sehir Has

Istanbul, , Turkey (Türkiye)

Site Status

Izmir Katip Celebi Universitesi Ataturk Egitim ve Arastirma

Izmir, , Turkey (Türkiye)

Site Status

Erciyes Universitesi Tip Fakultesi

Kayseri, , Turkey (Türkiye)

Site Status

Countries

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Greece Hungary New Zealand Norway Poland Ukraine United Kingdom United States Argentina Australia Belgium Brazil Canada China Colombia Czechia Denmark France Germany India Ireland Italy Japan Portugal Russia Singapore Slovakia South Korea Spain Sweden Taiwan Turkey (Türkiye)

References

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Other Identifiers

Review additional registry numbers or institutional identifiers associated with this trial.

LOXO-TRK-15002

Identifier Type: OTHER

Identifier Source: secondary_id

2022-502667-38-00

Identifier Type: OTHER

Identifier Source: secondary_id

2015-003582-28

Identifier Type: EUDRACT_NUMBER

Identifier Source: secondary_id

20289

Identifier Type: -

Identifier Source: org_study_id