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Active, not recruitingNCT03213704Updated Jun 17, 2026Results posted

Larotrectinib in Treating Patients With Relapsed or Refractory Advanced Solid Tumors, Non-Hodgkin Lymphoma, or Histiocytic Disorders With NTRK Fusions (A Pediatric MATCH Treatment Trial)

A Phase 2 interventional study of Biospecimen Collection and Bone Marrow Aspiration and Biopsy in Advanced Malignant Solid Neoplasm, Recurrent Ependymoma and Recurrent Ewing Sarcoma/Peripheral Primitive Neuroectodermal Tumor, sponsored by National Cancer Institute (NCI). Active, not recruiting at 124 sites in 2 countries. Open to participants aged 12 Months to 21 Years. Per ClinicalTrials.gov, last updated 2026-06-17.

Sponsored by National Cancer Institute (NCI) · Phase 2, Interventional, and Treatment

Phase
Phase 2
Study type
Interventional
Enrollment
9
Allocation
Not applicable
Ages
12 Months to 21 Years
Sex
All
01

Study summary

This phase II Pediatric MATCH trial studies how well larotrectinib works in treating patients with solid tumors, non-Hodgkin lymphoma, or histiocytic disorders with NTRK fusions that may have spread from where it first started to nearby tissue, lymph nodes, or distant parts of the body (advanced) and have come back (relapased) or does not respond to treatment (refractory). Larotrectinib may stop the growth of cancer cells by blocking some of the enzymes needed for cell growth.

Read the detailed description

PRIMARY OBJECTIVE:

I. To determine the objective response rate (ORR; complete response + partial response) in pediatric patients treated with larotrectinib sulfate (LOXO-101 [larotrectinib]) with advanced solid tumors (including central nervous system [CNS] tumors), non-Hodgkin lymphomas or histiocytic disorders harboring NTRK 1/2/3 fusions.

SECONDARY OBJECTIVES:

I. To estimate the progression free survival in pediatric patients treated with LOXO-101 (larotrectinib) with advanced solid tumors (including CNS tumors), non-Hodgkin lymphomas or histiocytic disorders with NTRK 1/2/3 fusions.

II. To obtain additional information about the tolerability of LOXO-101 (larotrectinib) in children with relapsed or refractory cancer.

III. To provide preliminary estimates of the pharmacokinetics of LOXO-101 (larotrectinib) in children with relapsed or refractory cancer.

EXPLORATORY OBJECTIVE:

I. To explore approaches to profiling changes in tumor genomics over time through evaluation of circulating tumor deoxyribonucleic acid (DNA).

OUTLINE:

Patients receive larotrectinib sulfate orally (PO) or via nasogastric (NG)- or gastric (G)-tube twice per day (BID) on days 1-28. Cycles repeat every 28 days for up to 2 years in the absence of disease progression or unacceptable toxicity. Patients undergo a computed tomography (CT) scan, magnetic resonance imaging (MRI), an x-ray, bone scan, and/or iobenguane (MIBG) scintigraphy during screening and on study. Patients also undergo bone marrow aspiration and/or biopsy during screening and may undergo blood sample collection on study.

After completion of study treatment, patients are followed up at 30 days, then periodically thereafter.

02

Conditions studied

  • Advanced Malignant Solid Neoplasm
  • Recurrent Ependymoma
  • Recurrent Ewing Sarcoma/Peripheral Primitive Neuroectodermal Tumor
  • Recurrent Glioma
  • Recurrent Hepatoblastoma
  • Recurrent Kidney Wilms Tumor
  • Recurrent Langerhans Cell Histiocytosis
  • Recurrent Malignant Germ Cell Tumor
  • Recurrent Malignant Glioma
  • Recurrent Malignant Solid Neoplasm
  • Recurrent Medulloblastoma
  • Recurrent Neuroblastoma
  • Recurrent Non-Hodgkin Lymphoma
  • Recurrent Osteosarcoma
  • Recurrent Rhabdoid Tumor
  • Recurrent Rhabdomyosarcoma
  • Recurrent Soft Tissue Sarcoma
  • Refractory Ependymoma
  • Refractory Ewing Sarcoma/Peripheral Primitive Neuroectodermal Tumor
  • Refractory Glioma
  • Refractory Hepatoblastoma
  • Refractory Langerhans Cell Histiocytosis
  • Refractory Malignant Germ Cell Tumor
  • Refractory Malignant Glioma
  • Refractory Malignant Solid Neoplasm
  • Refractory Medulloblastoma
  • Refractory Neuroblastoma
  • Refractory Non-Hodgkin Lymphoma
  • Refractory Osteosarcoma
  • Refractory Primary Central Nervous System Neoplasm
  • Refractory Rhabdoid Tumor
  • Refractory Rhabdomyosarcoma
  • Refractory Soft Tissue Sarcoma
03

In context

Ependymoma

224 studies on the registry are indexed under Ependymoma; 35 are open to participants now.

This study's enrollment of 9 is below the median of 28 across 174 interventional studies indexed under Ependymoma.

Browse Ependymoma studies →

Lead sponsor

National Cancer Institute (NCI) is the lead sponsor of 3,556 studies on the registry; 336 are open to participants now.

Of its 411 completed or terminated interventional studies of FDA-regulated products, 373 (91%) have results posted.

Browse National Cancer Institute (NCI) studies →

Counted across the registry records on this site, refreshed daily.

04

Who can participate

Ages eligible
12 Months to 21 Years
Sexes eligible
All
Accepts healthy volunteers
No

Go through the criteria one by one — yes, no or not sure — and take the list to your doctor or the study team. Kept in this browser only.

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 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
    • lesions that do not meet the measurement requirements for (RECIST) 1.1
  • >= 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 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
    • : 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 (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 (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 (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 disease will be eligible for study provided they meet the blood counts (may receive transfusions provided they are not known to be to red cell or platelet transfusions); these patients will not be evaluable for hematologic toxicity
  • or radioisotope (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
  • (sum of conjugated + unconjugated) =\< 1.5 x (ULN) for age (within 7 days prior to enrollment)
  • Serum glutamate pyruvate transaminase () (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 ( [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 ; assent, when appropriate, will be obtained according to institutional guidelines

Exclusion criteria

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 may not participate unless they have agreed to use an effective method for the duration of study treatment
  • Patients receiving 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 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 (GVHD) post bone marrow transplant are not eligible for this trial
  • Patients who are currently receiving drugs that are strong inducers or inhibitors of 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 , 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
Terms in these criteria, in plain language(22)

Underlined terms in the criteria open these too. Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.

Measurable disease

The amount of disease can be tracked with a defined measurement, so the study can see whether it shrinks or grows. It might be a tumour measured on a scan or, in some blood cancers such as myeloma, a marker measured in blood or urine.

The criteria or the study team say which measurement counts — having no tumour visible on a scan does not by itself rule you out.

Leptomeningeal disease

Cancer that has spread to the leptomeninges, the thin layers covering the brain and spinal cord, and the fluid between them. It is not the same as brain metastases.

Criteria often treat it separately, and many studies leave it out even where treated brain metastases are allowed.

RECIST

The standard rules for measuring tumours on CT or MRI scans, to tell whether a cancer is shrinking, stable or growing during treatment.

“Measurable disease per RECIST 1.1” usually means at least one tumour 10 mm or more at its longest on a CT scan, or a lymph node 15 mm or more across its short axis.

Karnofsky performance status (KPS)

A 0–100 scale of how well someone can carry out everyday activities. 100 is no complaints and no signs of illness; 70 is able to look after yourself but not to work or keep up normal activity; 50 is needing considerable help and frequent medical care.

Studies often ask for 60 or 70 and above. Your doctor gives the score.

Platelets

Blood cells that help blood clot. Too few raises the risk of bleeding and bruising. A usual adult range is about 150–400 × 10⁹/L (150,000–400,000 per µL); each lab has its own reference range.

Corticosteroids (steroids)

Anti-inflammatory medicines such as prednisone, prednisolone and dexamethasone — not the anabolic steroids used for muscle.

Studies differ in what they allow: check which steroid medicines, routes (tablets, injections, creams, inhalers, eye drops) and doses this study accepts.

GVHD (graft-versus-host disease)

After a transplant from a donor, the donor’s immune cells attack the recipient’s body, often the skin, gut or liver.

Brain metastases

Cancer deposits in the brain from a cancer that started elsewhere in the body (CNS metastases include the spinal cord).

Studies differ: some leave out any brain metastases, some accept ones treated and stable for some weeks. Check what this study allows.

Absolute neutrophil count (ANC)

The number of neutrophils, white blood cells that help fight infection, in a given amount of blood. Too few raises the risk of infection.

It is a count, not a percentage of the white cells. Limits are written in units that mean the same: ≥ 1.5 × 10⁹/L is ≥ 1,500 per mm³ (µL). Each lab has its own reference range.

Metastases

Cancer that has spread from where it began to other parts of the body. “Metastatic” cancer is cancer that has spread this way.

Relapsed and refractory

Relapsed: the illness came back after treatment had worked. Refractory: it did not respond to treatment, or stopped responding while still on it.

Creatinine clearance (CrCl)

How quickly the kidneys clear creatinine, a waste product, from the blood. A study may estimate it from a blood test with age, weight and sex (often by the Cockcroft–Gault formula), or measure it using blood and urine collected over a set time. Given in mL/min; higher is better.

GFR and eGFR (kidney filtering)

GFR (glomerular filtration rate) is how much blood the kidneys filter each minute. It is usually estimated from a blood test — eGFR, by formulas such as CKD-EPI or MDRD — and some studies ask for it to be measured directly instead. Usually given in mL/min/1.73 m².

Check the method, the units and the minimum the study states. A value of 90 or above is in the normal-or-high range, but kidney damage can show in other tests too.

Bilirubin

A yellow substance made when red blood cells are broken down, which the liver clears. A high level can mean a problem with the liver or the bile ducts.

Criteria often allow more for people with Gilbert’s syndrome, a harmless inherited condition that raises bilirubin.

ULN (upper limit of normal)

The top of the normal range for a blood test, as set by the laboratory that runs it. Each lab prints its own range next to your result.

“≤ 2.5 × ULN” means the result can be up to two and a half times that top value.

ALT and AST (liver enzymes)

Two enzymes measured in a routine blood test of the liver. Higher levels can mean the liver is under strain or damaged. SGPT and SGOT are older names for ALT and AST.

Criteria usually give a limit as a multiple of the upper limit of normal (ULN), often higher when cancer has spread to the liver.

CTCAE grade

The US National Cancer Institute’s scale of how severe an adverse event is — any unwanted medical event during a study, such as a symptom, an illness or an abnormal test result, whether or not a treatment caused it. Whether it did is judged separately.

1
Mild.
2
Moderate.
3
Severe.
4
Life-threatening.
5
Death due to the event.

“Side effects of earlier treatment resolved to grade 1 or less” means they have settled to mild or gone.

Informed consent

Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.

You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.

Reproductive potential

Able to have children. For women this usually means from the first period until menopause, unless permanently unable to through surgery; for men, unless permanently sterile. Read who the line is about — participants, women, men, or their partners.

Criteria about it usually ask for contraception during the study and for a time after it.

Contraception

Ways of preventing pregnancy. “Highly effective” methods fail less than 1% of the time a year when used correctly — for example an IUD or an implant.

Criteria usually say which methods count and how long to keep using them after the last dose, sometimes for male participants too. Some studies accept a partner’s vasectomy if it was confirmed successful and that person is the participant’s only sexual partner.

Investigational drug or device

A drug or device being studied in a clinical trial.

It may be new, or already approved and being studied for another use, dose or form. Criteria often leave out people who took part in another study of one within a recent period, commonly 30 days or five half-lives.

CYP3A (CYP3A4) inhibitors and inducers

CYP3A is a group of enzymes, mainly CYP3A4, in the liver and gut that break down many medicines. Some medicines, supplements and foods (grapefruit, St John’s wort) slow them down or speed them up, which changes how much of a study drug stays in the body.

Studies list them so they can be avoided or switched; bring a list of everything you take, including supplements.

05

Study design

Phase
Phase 2
Primary purpose
Treatment
Allocation
Not applicable
Intervention model
Single group
Masking
None (open label)
Enrollment
9 participants (actual)

Study arms

  • Experimental
    Treatment (larotrectinib sulfate)

    Patients receive larotrectinib sulfate PO or via NG- or G-tube BID on days 1-28. Cycles repeat every 28 days for up to 2 years in the absence of disease progression or unacceptable toxicity. Patients undergo a CT scan, MRI, an x-ray, bone scan, and/or MIBG scintigraphy during screening and on study. Patients also undergo bone marrow aspiration and/or biopsy during screening and may undergo blood sample collection on study.

    Procedure: Biospecimen Collection · Procedure: Bone Marrow Aspiration and Biopsy · Procedure: Bone Scan · Procedure: Computed Tomography · Drug: Larotrectinib Sulfate · Procedure: Magnetic Resonance Imaging · Procedure: Radionuclide Imaging · Procedure: X-Ray Imaging

Interventions

  • ProcedureBiospecimen Collection

    Undergo blood sample collection

    Also known as: Biological Sample Collection, Biospecimen Collected, Sample Collection, Specimen Collection

  • ProcedureBone Marrow Aspiration and Biopsy

    Undergo a bone marrow aspiration and/or biopsy

  • ProcedureBone Scan

    Undergo a bone scan

    Also known as: Bone Scintigraphy

  • ProcedureComputed Tomography

    Undergo a CT scan

    Also known as: CAT, CAT Scan, Computed Axial Tomography, Computerized Axial Tomography, Computerized axial tomography (procedure), Computerized Tomography, Computerized Tomography (CT) scan, CT, CT Scan, Diagnostic CAT Scan, Diagnostic CAT Scan Service Type, tomography

  • DrugLarotrectinib Sulfate

    Given PO or via nasogastric- or gastric-tube

    Also known as: ARRY 470 Sulfate, LOXO 101 Sulfate, LOXO-101 Sulfate, Vitrakvi

  • ProcedureMagnetic Resonance Imaging

    Undergo MRI

    Also known as: Magnetic Resonance, Magnetic Resonance Imaging (MRI), Magnetic resonance imaging (procedure), Magnetic Resonance Imaging Scan, Medical Imaging, Magnetic Resonance / Nuclear Magnetic Resonance, MR, MR Imaging, MRI, MRI Scan, MRIs, NMR Imaging, NMRI, Nuclear Magnetic Resonance Imaging, sMRI, Structural MRI

  • ProcedureRadionuclide Imaging

    Undergo a MIBG scintigraphy

    Also known as: Gamma Scan, NM, Nuclear Medicine, nuclear medicine scan, radioimaging, Radionuclide Scanning, Scan, Scintigraphy

  • ProcedureX-Ray Imaging

    Undergo an x-ray

    Also known as: Conventional X-Ray, Diagnostic Radiology, Medical Imaging, X-Ray, Plain film radiographs, Radiographic Imaging, Radiographic imaging procedure (procedure), Radiography, RG, Static X-Ray, X-Ray

06

What researchers measure

Primary outcomes

  1. Objective Response Rate

    A responder is defined as a patient who achieves a best response of partial response or complete response on the study. Response rates will be calculated as the percent of evaluable patients who are responders. The revised Response Evaluation Criteria in Solid Tumors (RECIST) guideline (version 1.1) was used to determine response and progression in this study, with specific criteria outlined for the different subtypes of tumors (e.g., 2-dimensional measurements for central nervous system (CNS) tumors).

    Time frame: Up to 2 years from study entry

Secondary outcomes

  1. Progression Free Survival (PFS)

    The Kaplan-Meier method will be used to estimate the 6 month PFS. PFS is defined as time from initiation of protocol treatment to disease progression, recurrence, death from any cause, or date of last contact.

    Time frame: Up to 6 months from study entry

  2. Percentage of Patients Experiencing Grade 3 or Higher Adverse Events

    Percentage of patients experiencing grade 3 or higher adverse events will be evaluated according to National Cancer Institute Common Terminology Criteria for Adverse Events version 5.0.

    Time frame: Up to 2 years from study entry

Other outcomes

  1. Pharmacokinetics of Larotrectinib

    A descriptive analysis of pharmacokinetic parameters of LOXO-101 (larotrectinib) will be performed to define systemic exposure, drug clearance, and other pharmacokinetic parameters. The pharmacokinetic parameters will be summarized with simple summary statistics, including means, medians, ranges, and standard deviations (if numbers and distribution permit). All these analyses will be descriptive and exploratory and hypotheses generating in nature.

    Time frame: Pre-dose, 1, 2, 4, and 6-8 hours after morning dose of day 1 cycle 1 and pre-dose of day 1 cycle 2

  2. Ability to Detect NTRK Fusions in Circulating Cell-free Tumor Deoxyribonucleic Acid in Plasma

    A descriptive analysis of the exploratory aims will be performed and will be summarized with simple summary statistics. All of these analyses will be descriptive in nature.

    Time frame: Up to 5 years

07

Results

Posted Oct 16, 2025

Participant flow

Participant flow — Overall Study
MilestoneTreatment (Larotrectinib Sulfate)
Started9
Completed1
Not completed8
Withdrew: Physician decision3
Withdrew: Protocol violation1
Withdrew: Progressive disease4

Outcome measures

PrimaryObjective Response Rate

A responder is defined as a patient who achieves a best response of partial response or complete response on the study. Response rates will be calculated as the percent of evaluable patients who are responders. The revised Response Evaluation Criteria in Solid Tumors (RECIST) guideline (version 1.1) was used to determine response and progression in this study, with specific criteria outlined for the different subtypes of tumors (e.g., 2-dimensional measurements for central nervous system (CNS) tumors).

Time frame:
Up to 2 years from study entry
Reported as:
Number · percentage of participants
Objective Response Rate
percentage of participantsTreatment (Larotrectinib Sulfate)
Objective Response Rate33 (14.2 to 60.2)
SecondaryProgression Free Survival (PFS)

The Kaplan-Meier method will be used to estimate the 6 month PFS. PFS is defined as time from initiation of protocol treatment to disease progression, recurrence, death from any cause, or date of last contact.

Time frame:
Up to 6 months from study entry
Reported as:
Number · percentage of participants
Progression Free Survival (PFS)
percentage of participantsTreatment (Larotrectinib Sulfate)
Progression Free Survival (PFS)76 (33.2 to 93.5)
SecondaryPercentage of Patients Experiencing Grade 3 or Higher Adverse Events

Percentage of patients experiencing grade 3 or higher adverse events will be evaluated according to National Cancer Institute Common Terminology Criteria for Adverse Events version 5.0.

Time frame:
Up to 2 years from study entry
Reported as:
Number · percentage of participants
Percentage of Patients Experiencing Grade 3 or Higher Adverse Events
percentage of participantsTreatment (Larotrectinib Sulfate)
Percentage of Patients Experiencing Grade 3 or Higher Adverse Events44 (13.7 to 78.8)
Other pre-specifiedPharmacokinetics of Larotrectinib

A descriptive analysis of pharmacokinetic parameters of LOXO-101 (larotrectinib) will be performed to define systemic exposure, drug clearance, and other pharmacokinetic parameters. The pharmacokinetic parameters will be summarized with simple summary statistics, including means, medians, ranges, and standard deviations (if numbers and distribution permit). All these analyses will be descriptive and exploratory and hypotheses generating in nature.

Time frame:
Pre-dose, 1, 2, 4, and 6-8 hours after morning dose of day 1 cycle 1 and pre-dose of day 1 cycle 2

Results for this outcome have not been posted.

Other pre-specifiedAbility to Detect NTRK Fusions in Circulating Cell-free Tumor Deoxyribonucleic Acid in Plasma

A descriptive analysis of the exploratory aims will be performed and will be summarized with simple summary statistics. All of these analyses will be descriptive in nature.

Time frame:
Up to 5 years

Results for this outcome have not been posted.

Adverse events

Collected over Adverse Events monitored/assessed from enrollment to 30 days after the end of treatment, up to 2 years. All-Cause Mortality monitored/assessed up to 5 years. Non-serious events are listed at a 0% frequency threshold.

Adverse event summary by group
GroupDeathsSeriousOther
Treatment (Larotrectinib Sulfate)3/9 (33.3%)2/9 (22.2%)7/9 (77.8%)
Most frequent serious events
Most frequent serious events
EventTreatment (Larotrectinib Sulfate)
DiarrheaGastrointestinal disorders1/9
NauseaGastrointestinal disorders1/9
Disease progressionGeneral disorders1/9
FeverGeneral disorders1/9
SepsisInfections and infestations1/9
Neutrophil count decreasedInvestigations1/9
Depressed level of consciousnessNervous system disorders1/9
HypotensionVascular disorders1/9
HypoglycemiaMetabolism and nutrition disorders1/9
Most frequent other events
Showing 10 of 84
Most frequent other events
EventTreatment (Larotrectinib Sulfate)
VomitingGastrointestinal disorders5/9
White blood cell decreasedInvestigations5/9
AnemiaBlood and lymphatic system disorders4/9
Lymphocyte count decreasedInvestigations4/9
HyperglycemiaMetabolism and nutrition disorders4/9
HypocalcemiaMetabolism and nutrition disorders4/9
Sinus tachycardiaCardiac disorders3/9
NauseaGastrointestinal disorders3/9
FatigueGeneral disorders3/9
FeverGeneral disorders3/9

Baseline characteristics

Age, Categorical
Age, Categorical(Participants)Treatment (Larotrectinib Sulfate)
<=18 years8
Between 18 and 65 years1
>=65 years0
Age, Continuous
Age, Continuous(years)Treatment (Larotrectinib Sulfate)
Mean9.3 ± 6.5
Sex: Female, Male
Sex: Female, Male(Participants)Treatment (Larotrectinib Sulfate)
Female3
Male6
Ethnicity (NIH/OMB)
Ethnicity (NIH/OMB)(Participants)Treatment (Larotrectinib Sulfate)
Hispanic or Latino6
Not Hispanic or Latino3
Unknown or Not Reported0
Race (NIH/OMB)
Race (NIH/OMB)(Participants)Treatment (Larotrectinib Sulfate)
American Indian or Alaska Native0
Asian0
Native Hawaiian or Other Pacific Islander0
Black or African American3
White3
More than one race0
Unknown or Not Reported3
Region of Enrollment
Region of Enrollment(participants)Treatment (Larotrectinib Sulfate)
United States9
08

Study locations

124 sites

Showing the first 100 of 124 sites across 2 countries. Choose a country and city above to see the rest.

09

References and documents

Study documents

PDFs hosted by the registry, ClinicalTrials.gov.

10

Timeline

Tracking since Sep 25, 2026
ElapsedEstimatedEstimated, not yet reachedSimilar studies: half by the dot; the band is the middle halfHalf of similar studies reached it by the dot; the band is the middle half, from a quarter to three quarters

Ran from Aug 2017 to primary completion in Sep 2024, 85 months.

Results were first posted in Oct 2025, 1 year after primary completion.

Of 34 non-industry phase 2 interventional studies under Ependymoma, half reached primary completion within 66 months of starting (the median), counting those still running; a quarter within 47 and three quarters within 108. This study's 85 months is above that.

Counting every sponsor, half of 35 studies reached it within 67 months; a quarter within 52 and three quarters within 108.

How these figures are worked out, and which studies are left out of them.

Registered
Jul 10, 2017
First posted
Jul 11, 2017
StartActual
Aug 23, 2017
Primary completionActual
Sep 30, 2024
CompletionEst.
Oct 8, 2026
Results first posted
Oct 16, 2025
Last update
Jun 17, 2026
What these dates mean
Start.
When the first participant enrolled; while estimated, when the study expects to open to recruitment.
Primary completion.
When the last participant was examined or treated to collect data for the study's main question, its primary outcome. A study that stopped early gives the date its data collection stopped.
Completion.
When the last participant's last visit took place, for every outcome, secondary ones and side effects included: the end of the study. Often later than primary completion, sometimes the same day.
Actual and Est.
Actual means the registry says the date has happened. Est. (estimated) is a date the sponsor plans or expects. It can still move, and the sponsor is expected to change it to actual once it has passed.
No changes since tracking began. The registry record was last updated on Jun 17, 2026, before this site started recording changes on Sep 25, 2026. Its history is on ClinicalTrials.gov ↗
11

Registry details

Key details

Study ID
NCT03213704
Lead sponsor
National Cancer Institute (NCI)
Responsible party
Sponsor
First posted
Jul 11, 2017
Start date
Aug 23, 2017
Primary completion
Sep 30, 2024
Completion
Oct 8, 2026 (estimated)
Results posted
Oct 16, 2025
Last update
Jun 17, 2026

Study contacts

Katherine A Janeway
principal investigator · Children's Oncology Group

Oversight

Data monitoring committee
No
FDA-regulated drug
Yes
FDA-regulated device
No
View the source record on ClinicalTrials.gov ↗

Not currently enrolling

This study is active, not recruiting, as verified in May 2026. You cannot join it, but the record below documents what was studied.

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Discussion

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