New hope for kids with rare cancers: targeted drug larotrectinib tested in small trial
NCT ID NCT03213704
First seen Jun 27, 2026 · Last updated Jun 27, 2026
Summary
This phase 2 trial is testing the drug larotrectinib in children and young adults (ages 1 to 21) with advanced solid tumors, non-Hodgkin lymphoma, or histiocytic disorders that have a specific genetic change called an NTRK fusion. The cancers have either come back after treatment or not responded to standard therapy. The goal is to see if larotrectinib can shrink tumors or stop them from growing by blocking enzymes that cancer cells need to grow.
What this could mean
Our plain-language read of the trial. This is informational only, not medical advice or a prediction.
- Active substance
- Larotrectinib (a targeted cancer drug that blocks enzymes helping cancer cells grow)
- What this could lead to
- If it works, this could provide a new treatment option for children with certain hard-to-treat cancers that have a specific genetic change (NTRK fusion).
- What could go wrong
- This is a very small, early-phase trial (only 9 participants) and results may not apply to all patients. The drug may not shrink tumors or improve survival, and side effects are possible.
This is an AI summary of the original study and may miss details. Read our disclaimer.
Study facts
What this study's own registry entry says, in plain language.
- Phase
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Phase 2
Tests whether the treatment actually works, and watches for side effects, in a larger group.
- Participants
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9 people
The number who actually took part.
- Started
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Aug 2017
- Expected to finish
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Oct 2026
An estimate. End dates often move.
- Lead sponsor
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A government research agency
The lead sponsor is the US National Institutes of Health.
Who can take part
This study's own entry requirements. Only the study team can say for certain whether you qualify.
- Ages
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12 months to 21 years
- Sex
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Anyone
- Healthy volunteers
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Not accepted
This study is not open to healthy volunteers. The entry requirements below say who it is open to.
Show the full entry requirements Hide the full entry requirements
Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.
Inclusion Criteria: * APEC1621SC: Patient must have enrolled onto APEC1621SC and must have been given a treatment assignment to Molecular Analysis for Therapy Choice (MATCH) to APEC1621A based on the presence of an actionable mutation as defined in APEC1621SC * Patients must be \>= than 12 months and =\< 21 years of age at the time of study enrollment * Patients must have radiographically measurable disease at the time of study enrollment; patients with neuroblastoma who do not have measurable disease but have MIBG+ evaluable disease are eligible; measurable disease in patients with CNS involvement is defined as any lesion that is at minimum 10 mm in one dimension on standard MRI or CT; Note: The following do not qualify as measurable disease: * Malignant fluid collections (e.g., ascites, pleural effusions) * Bone marrow infiltration except that detected by MIBG scan for neuroblastoma * Lesions only detected by nuclear medicine studies (e.g., bone, gallium or positron emission tomography \[PET\] scans) except as noted for neuroblastoma * Elevated tumor markers in plasma or cerebrospinal fluid (CSF) * Previously radiated lesions that have not demonstrated clear progression post radiation * Leptomeningeal lesions that do not meet the measurement requirements for Response Evaluation Criteria in Solid Tumors (RECIST) 1.1 * Karnofsky \>= 50% for patients \> 16 years of age and Lansky \>= 50 for patients =\< 16 years of age; Note: neurologic deficits in patients with CNS tumors must have been relatively stable for at least 7 days prior to study enrollment; patients who are unable to walk because of paralysis, but who are up in a wheelchair, will be considered ambulatory for the purpose of assessing the performance score * Patients must have fully recovered from the acute toxic effects of all prior anti-cancer therapy and must meet the following minimum duration from prior anti-cancer directed therapy prior to enrollment; if after the required time frame, the numerical eligibility criteria are met, e.g. blood count criteria, the patient is considered to have recovered adequately * Cytotoxic chemotherapy or other anti-cancer agents known to be myelosuppressive; for agents not listed, the duration of this interval must be discussed with the study chair and the study-assigned research coordinator prior to enrollment * \>= 21 days after the last dose of cytotoxic or myelosuppressive chemotherapy (42 days if prior nitrosourea) * Anti-cancer agents not known to be myelosuppressive (e.g. not associated with reduced platelet or absolute neutrophil \[ANC\] counts): \>= 7 days after the last dose of agent; for agents not listed, the duration of this interval must be discussed with the study chair and the study-assigned research coordinator prior to enrollment * Antibodies: \>= 21 days must have elapsed from infusion of last dose of antibody, and toxicity related to prior antibody therapy must be recovered to grade =\< 1 * Corticosteroids: if used to modify immune adverse events related to prior therapy, \>= 14 days must have elapsed since last dose of corticosteroid * Hematopoietic growth factors: \>= 14 days after the last dose of a long-acting growth factor (e.g. pegfilgrastim) or 7 days for short-acting growth factor; for growth factors that have known adverse events occurring beyond 7 days after administration, this period must be extended beyond the time during which adverse events are known to occur; the duration of this interval must be discussed with the study chair and the study-assigned research coordinator * Interleukins, interferons and cytokines (other than hematopoietic growth factors): \>= 21 days after the completion of interleukins, interferon or cytokines (other than hematopoietic growth factors) * Stem cell Infusions (with or without total body irradiation \[TBI\]): * Allogeneic (non-autologous) bone marrow or stem cell transplant, or any stem cell infusion including donor leukocyte infusion (DLI) or boost infusion: \>= 84 days after infusion and no evidence of graft versus host disease (GVHD) * Autologous stem cell infusion including boost infusion: \>= 42 days * Cellular therapy: \>= 42 days after the completion of any type of cellular therapy (e.g. modified T cells, NK cells, dendritic cells, etc.) * Radiation therapy (XRT)/external beam irradiation including protons: \>= 14 days after local XRT; \>= 150 days after TBI, craniospinal XRT or if radiation to \>= 50% of the pelvis; \>= 42 days if other substantial brain metastases (BM) radiation; Note: radiation may not be delivered to "measurable disease" tumor site(s) being used to follow response to subprotocol treatment * Radiopharmaceutical therapy (e.g., radiolabeled antibody, 131I-MIBG): \>= 42 days after systemically administered radiopharmaceutical therapy * Patients must not have received prior exposure to other NTRK inhibitors including but not limited to LOXO-101 (larotrectinib), entrectinib (RXDX-101), DS6051, PLX7486 * For patients with solid tumors without known bone marrow involvement: * Peripheral absolute neutrophil count (ANC) \>= 1000/mm\^3 (within 7 days prior to enrollment) * Platelet count \>= 100,000/mm\^3 (transfusion independent, defined as not receiving platelet transfusions for at least 7 days prior to enrollment) (within 7 days prior to enrollment) * Patients with known bone marrow metastatic disease will be eligible for study provided they meet the blood counts (may receive transfusions provided they are not known to be refractory to red cell or platelet transfusions); these patients will not be evaluable for hematologic toxicity * Creatinine clearance or radioisotope glomerular filtration rate (GFR) \>= 70 ml/min/1.73 m\^2 (within 7 days prior to enrollment) or * A serum creatinine based on age/gender as follows (within 7 days prior to enrollment): * Age: 1 to \< 2 years; maximum serum creatinine (mg/dL); male: 0.6 female: 0.6 * Age: 2 to \< 6 years; maximum serum creatinine (mg/dL); male: 0.8 female: 0.8 * Age: 6 to \< 10 years; maximum serum creatinine (mg/dL); male: 1 female: 1 * Age: 10 to \< 13 years; maximum serum creatinine (mg/dL); male: 1.2 female: 1.2 * Age: 13 to \< 16 years; maximum serum creatinine (mg/dL); male: 1.5 female: 1.4 * Age: \>= 16 years; maximum serum creatinine (mg/dL); male: 1.7 female: 1.4 * Bilirubin (sum of conjugated + unconjugated) =\< 1.5 x upper limit of normal (ULN) for age (within 7 days prior to enrollment) * Serum glutamate pyruvate transaminase (SGPT) (alanine aminotransferase \[ALT\]) =\< 135 U/L (within 7 days prior to enrollment); (for the purpose of this study, the ULN for SGPT is 45 U/L) * Serum albumin \>= 2 g/dL (within 7 days prior to enrollment) * Patients with seizure disorder may be enrolled if on anti-convulsants and well controlled * Nervous system disorders (Common Terminology Criteria for Adverse Events \[CTCAE\] version \[v\] 5.0) resulting from prior therapy must be =\< grade 2, with the exception of decreased tendon reflect (DTR); any grade of DTR is eligible * All patients and/or their parents or legally authorized representatives must sign a written informed consent; assent, when appropriate, will be obtained according to institutional guidelines Exclusion Criteria: * Pregnant or breast-feeding women will not be entered on this study due to risks of fetal and teratogenic adverse events as seen in animal/human studies; pregnancy tests must be obtained in girls who are post-menarchal; males or females of reproductive potential may not participate unless they have agreed to use an effective contraceptive method for the duration of study treatment * Patients receiving corticosteroids who have not been on a stable or decreasing dose of corticosteroid for at least 7 days prior to enrollment are not eligible; if used to modify immune adverse events related to prior therapy, \>= 14 days must have elapsed since last dose of corticosteroid * Patients who are currently receiving another investigational drug are not eligible * Patients who are currently receiving other anti-cancer agents are not eligible * Patients who are receiving cyclosporine, tacrolimus or other agents to prevent graft-versus-host disease (GVHD) post bone marrow transplant are not eligible for this trial * Patients who are currently receiving drugs that are strong inducers or inhibitors of CYP3A4 are not eligible; strong inducers or inhibitors of CYP3A4 should be avoided from 14 days prior to enrollment to the end of the study; Note: CYP3A4 inducing anti-epileptic drugs and dexamethasone for CNS tumors or metastases, on a stable dose, are allowed * Patients who have received prior therapy with a specific inhibitor of TRK (including but not limited to entrectinib \[RXDX-101\], DS-6051b, and PLX7486) are not eligible * Patients who have an uncontrolled infection are not eligible * Patients who have received a prior solid organ transplantation are not eligible * Patients who in the opinion of the investigator may not be able to comply with the safety monitoring requirements of the study are not eligible
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Conditions
The condition(s) this trial relates to.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.
Contacts and locations
Locations
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AdventHealth Orlando
Orlando, Florida, 32803, United States
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Albany Medical Center
Albany, New York, 12208, United States
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Alfred I duPont Hospital for Children
Wilmington, Delaware, 19803, United States
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Alliance for Childhood Diseases/Cure 4 the Kids Foundation
Las Vegas, Nevada, 89135, United States
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Arkansas Children's Hospital
Little Rock, Arkansas, 72202-3591, United States
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Arnold Palmer Hospital for Children
Orlando, Florida, 32806, United States
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Ascension Saint Vincent Indianapolis Hospital
Indianapolis, Indiana, 46260, United States
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BI-LO Charities Children's Cancer Center
Greenville, South Carolina, 29605, United States
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Banner Children's at Desert
Mesa, Arizona, 85202, United States
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Banner University Medical Center - Tucson
Tucson, Arizona, 85719, United States
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Baylor College of Medicine/Dan L Duncan Comprehensive Cancer Center
Houston, Texas, 77030, United States
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Blank Children's Hospital
Des Moines, Iowa, 50309, United States
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Bronson Methodist Hospital
Kalamazoo, Michigan, 49007, United States
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C S Mott Children's Hospital
Ann Arbor, Michigan, 48109, United States
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Cardinal Glennon Children's Medical Center
St Louis, Missouri, 63104, United States
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Carolinas Medical Center/Levine Cancer Institute
Charlotte, North Carolina, 28203, United States
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Children's Healthcare of Atlanta - Arthur M Blank Hospital
Atlanta, Georgia, 30329, United States
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Children's Hospital Colorado
Aurora, Colorado, 80045, United States
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Children's Hospital Los Angeles
Los Angeles, California, 90027, United States
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Children's Hospital New Orleans
New Orleans, Louisiana, 70118, United States
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Children's Hospital and Medical Center of Omaha
Omaha, Nebraska, 68114, United States
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Children's Hospital of Alabama
Birmingham, Alabama, 35233, United States
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Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, 19104, United States
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Children's Hospital of Pittsburgh of UPMC
Pittsburgh, Pennsylvania, 15224, United States
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Children's Hospital of San Antonio
San Antonio, Texas, 78207, United States
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Children's Hospital of The King's Daughters
Norfolk, Virginia, 23507, United States
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Children's Hospital of Wisconsin
Milwaukee, Wisconsin, 53226, United States
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Children's Hospitals and Clinics of Minnesota - Minneapolis
Minneapolis, Minnesota, 55404, United States
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Children's Mercy Hospitals and Clinics
Kansas City, Missouri, 64108, United States
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Children's National Medical Center
Washington D.C., District of Columbia, 20010, United States
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Cincinnati Children's Hospital Medical Center
Cincinnati, Ohio, 45229, United States
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Cleveland Clinic Foundation
Cleveland, Ohio, 44195, United States
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Cook Children's Medical Center
Fort Worth, Texas, 76104, United States
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Corewell Health Grand Rapids Hospitals - Helen DeVos Children's Hospital
Grand Rapids, Michigan, 49503, United States
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Dayton Children's Hospital
Dayton, Ohio, 45404, United States
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Dell Children's Medical Center of Central Texas
Austin, Texas, 78723, United States
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Driscoll Children's Hospital
Corpus Christi, Texas, 78411, United States
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Duke University Medical Center
Durham, North Carolina, 27710, United States
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East Tennessee Childrens Hospital
Knoxville, Tennessee, 37916, United States
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Eastern Maine Medical Center
Bangor, Maine, 04401, United States
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Geisinger Medical Center
Danville, Pennsylvania, 17822, United States
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Hackensack University Medical Center
Hackensack, New Jersey, 07601, United States
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Johns Hopkins All Children's Hospital
St. Petersburg, Florida, 33701, United States
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Johns Hopkins University/Sidney Kimmel Cancer Center
Baltimore, Maryland, 21287, United States
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Kaiser Permanente Downey Medical Center
Downey, California, 90242, United States
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Kaiser Permanente-Oakland
Oakland, California, 94611, United States
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Legacy Emanuel Children's Hospital
Portland, Oregon, 97227, United States
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Loma Linda University Medical Center
Loma Linda, California, 92354, United States
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Lurie Children's Hospital-Chicago
Chicago, Illinois, 60611, United States
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Madigan Army Medical Center
Tacoma, Washington, 98431, United States
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Marshfield Medical Center-Marshfield
Marshfield, Wisconsin, 54449, United States
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Mayo Clinic in Rochester
Rochester, Minnesota, 55905, United States
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Medical City Dallas Hospital
Dallas, Texas, 75230, United States
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Memorial Sloan Kettering Cancer Center
New York, New York, 10065, United States
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Mercy Hospital Saint Louis
St Louis, Missouri, 63141, United States
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Methodist Children's Hospital of South Texas
San Antonio, Texas, 78229, United States
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Michigan State University
East Lansing, Michigan, 48823, United States
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Miller Children's and Women's Hospital Long Beach
Long Beach, California, 90806, United States
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Mission Hospital
Asheville, North Carolina, 28801, United States
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Montefiore Medical Center - Moses Campus
The Bronx, New York, 10467, United States
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Morristown Medical Center
Morristown, New Jersey, 07960, United States
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NYP/Columbia University Medical Center/Herbert Irving Comprehensive Cancer Center
New York, New York, 10032, United States
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NYP/Weill Cornell Medical Center
New York, New York, 10065, United States
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Nationwide Children's Hospital
Columbus, Ohio, 43205, United States
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Nemours Children's Clinic - Pensacola
Pensacola, Florida, 32504, United States
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Nemours Children's Clinic-Jacksonville
Jacksonville, Florida, 32207, United States
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Nemours Children's Hospital
Orlando, Florida, 32827, United States
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New York Medical College
Valhalla, New York, 10595, United States
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Nicklaus Children's Hospital
Miami, Florida, 33155, United States
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Norton Children's Hospital
Louisville, Kentucky, 40202, United States
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Novant Health Presbyterian Medical Center
Charlotte, North Carolina, 28204, United States
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OSF Children's Hospital of Illinois
Peoria, Illinois, 61637, United States
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Ochsner Medical Center Jefferson
New Orleans, Louisiana, 70121, United States
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Oregon Health and Science University
Portland, Oregon, 97239, United States
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Primary Children's Hospital
Salt Lake City, Utah, 84113, United States
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Prisma Health Richland Hospital
Columbia, South Carolina, 29203, United States
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ProMedica Toledo Hospital/Russell J Ebeid Children's Hospital
Toledo, Ohio, 43606, United States
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Providence Alaska Medical Center
Anchorage, Alaska, 99508, United States
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Providence Sacred Heart Medical Center and Children's Hospital
Spokane, Washington, 99204, United States
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Rainbow Babies and Childrens Hospital
Cleveland, Ohio, 44106, United States
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Riley Hospital for Children
Indianapolis, Indiana, 46202, United States
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Rocky Mountain Hospital for Children-Presbyterian Saint Luke's Medical Center
Denver, Colorado, 80218, United States
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Roswell Park Cancer Institute
Buffalo, New York, 14263, United States
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Saint Joseph's Hospital/Children's Hospital-Tampa
Tampa, Florida, 33607, United States
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Saint Jude Children's Research Hospital
Memphis, Tennessee, 38105, United States
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Saint Luke's Cancer Institute - Boise
Boise, Idaho, 83712, United States
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Saint Mary's Medical Center
West Palm Beach, Florida, 33407, United States
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Saint Peter's University Hospital
New Brunswick, New Jersey, 08901, United States
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San Jorge Children's Hospital
San Juan, 00912, Puerto Rico
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Sanford Broadway Medical Center
Fargo, North Dakota, 58122, United States
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Sanford USD Medical Center - Sioux Falls
Sioux Falls, South Dakota, 57117-5134, United States
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Scott and White Memorial Hospital
Temple, Texas, 76508, United States
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Seattle Children's Hospital
Seattle, Washington, 98105, United States
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Sinai Hospital of Baltimore
Baltimore, Maryland, 21215, United States
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Southern Illinois University School of Medicine
Springfield, Illinois, 62702, United States
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State University of New York Upstate Medical University
Syracuse, New York, 13210, United States
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Stony Brook University Medical Center
Stony Brook, New York, 11794, United States
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Summerlin Hospital Medical Center
Las Vegas, Nevada, 89144, United States
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Sunrise Hospital and Medical Center
Las Vegas, Nevada, 89109, United States
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The Children's Hospital at TriStar Centennial
Nashville, Tennessee, 37203, United States
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The Steven and Alexandra Cohen Children's Medical Center of New York
New Hyde Park, New York, 11040, United States
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UCSF Benioff Children's Hospital Oakland
Oakland, California, 94609, United States
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UCSF Medical Center-Mission Bay
San Francisco, California, 94158, United States
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UF Health Cancer Institute - Gainesville
Gainesville, Florida, 32610, United States
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UT MD Anderson Cancer Center
Houston, Texas, 77030, United States
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UT Southwestern/Simmons Cancer Center-Dallas
Dallas, Texas, 75390, United States
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University Medical Center of Southern Nevada
Las Vegas, Nevada, 89102, United States
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University Pediatric Hospital
San Juan, 00926, Puerto Rico
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University of California Davis Comprehensive Cancer Center
Sacramento, California, 95817, United States
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University of Chicago Comprehensive Cancer Center
Chicago, Illinois, 60637, United States
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University of Iowa/Holden Comprehensive Cancer Center
Iowa City, Iowa, 52242, United States
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University of Miami Miller School of Medicine-Sylvester Cancer Center
Miami, Florida, 33136, United States
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University of Minnesota/Masonic Cancer Center
Minneapolis, Minnesota, 55455, United States
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University of Mississippi Medical Center
Jackson, Mississippi, 39216, United States
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University of Nebraska Medical Center
Omaha, Nebraska, 68198, United States
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University of Oklahoma Health Sciences Center
Oklahoma City, Oklahoma, 73104, United States
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University of Rochester
Rochester, New York, 14642, United States
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University of Wisconsin Carbone Cancer Center - University Hospital
Madison, Wisconsin, 53792, United States
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VCU Massey Comprehensive Cancer Center
Richmond, Virginia, 23298, United States
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Valley Children's Hospital
Madera, California, 93636, United States
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Vanderbilt University/Ingram Cancer Center
Nashville, Tennessee, 37232, United States
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Washington University School of Medicine
St Louis, Missouri, 63110, United States
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West Virginia University Healthcare
Morgantown, West Virginia, 26506, United States
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Yale University
New Haven, Connecticut, 06520, United States
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