Larotrectinib (LOXO-101) Clinical Trials

Size	Price	Qty
5mg	USD 147
25mg	USD 297

Clinical Trials

Numerous clinical trials spanning Early Phase 1 through Phase 4 evaluate larotrectinib across oncology indications, primarily focusing on NTRK fusion-positive adult and pediatric solid tumors, high-grade gliomas, differentiated thyroid carcinomas, and refractory acute leukemias. Sponsored by pharmaceutical entities as well as academic and cooperative networks, these studies encompass pediatric Phase 1/2 trials and adult Phase 2 basket studies. Portfolio recruitment statuses currently range from actively recruiting and active, not recruiting to post-marketing approval evaluations.

NCT Number	Recruitment	Conditions	Sponsor/Collaborators	Start Date	Phases
NCT05725200	RECRUITING	Metastatic Colorectal Cancer	Oslo University Hospital	2022-09-27	PHASE2
NCT07010393	NOT_YET_RECRUITING	Thyroid Neoplasms	Fujian Medical University	2025-07-01	PHASE4
NCT04879121	RECRUITING	Locally Advanced Malignant Solid Neoplasm; Metastatic Malignant Solid Neoplasm	M.D. Anderson Cancer Center	2021-04-30	PHASE2
NCT06563999	RECRUITING	Lung Cancer Stage III; Mutation	Sun Yat-sen University	2024-11-01	PHASE2
NCT02465060	ACTIVE_NOT_RECRUITING	Advanced Lymphoma; Advanced Malignant Solid Neoplasm; Bladder Carcinoma; Breast Carcinoma; Cervical Carcinoma; Colon Carcinoma; Colorectal Carcinoma; Endometrial Carcinoma; Esophageal Carcinoma; Exocrine Pancreas Carcinoma; Gastric Carcinoma; Glioma; Head and Neck Carcinoma; Hematopoietic and Lymphoid Cell Neoplasm; Kidney Carcinoma; Liver Carcinoma; Lung Carcinoma; Lymphoma; Malignant Uterine Corpus Neoplasm; Malignant Uterine Neoplasm; Melanoma; Multiple Myeloma; Ovarian Carcinoma; Prostate Carcinoma; Rectal Carcinoma; Recurrent Bladder Carcinoma; Recurrent Breast Carcinoma; Recurrent Cervical Carcinoma; Recurrent Colon Carcinoma; Recurrent Colorectal Carcinoma; Recurrent Esophageal Carcinoma; Recurrent Gastric Carcinoma; Recurrent Glioma; Recurrent Head and Neck Carcinoma; Recurrent Liver Carcinoma; Recurrent Lung Carcinoma; Recurrent Lymphoma; Recurrent Malignant Solid Neoplasm; Recurrent Malignant Uterine Corpus Neoplasm; Recurrent Melanoma; Recurrent Multiple Myeloma; Recurrent Ovarian Carcinoma; Recurrent Pancreatic Carcinoma; Recurrent Prostate Carcinoma; Recurrent Rectal Carcinoma; Recurrent Skin Carcinoma; Recurrent Thyroid Gland Carcinoma; Refractory Lymphoma; Refractory Malignant Solid Neoplasm; Refractory Multiple Myeloma; Skin Carcinoma; Thyroid Gland Carcinoma	National Cancer Institute (NCI)	2015-08-17	PHASE2
NCT06195228	RECRUITING	Advanced Thyroid Cancer Patients Who Received Target Therapy	Fudan University	2020-01-01	PHASE4
NCT04655404	RECRUITING	High Grade Glioma; Diffuse Intrinsic Pontine Glioma	Nationwide Children's Hospital	2021-04-08	EARLY_PHASE1
NCT05783323	RECRUITING	Differentiated Thyroid Cancer; Pediatric Cancer; Cancer; Cancer, Thyroid	Children's Hospital of Philadelphia	2024-02-14	PHASE2
NCT02576431	COMPLETED	Solid Tumors Harboring NTRK Fusion	Bayer	2015-09-30	PHASE2
NCT03155620	ACTIVE_NOT_RECRUITING	Advanced Malignant Solid Neoplasm; Ann Arbor Stage III Non-Hodgkin Lymphoma; Ann Arbor Stage IV Non-Hodgkin Lymphoma; Histiocytic Sarcoma; Juvenile Xanthogranuloma; Langerhans Cell Histiocytosis; Malignant Glioma; Recurrent Childhood Rhabdomyosarcoma; Recurrent Ependymoma; Recurrent Ewing Sarcoma; Recurrent Glioma; Recurrent Hepatoblastoma; Recurrent Langerhans Cell Histiocytosis; Recurrent Malignant Germ Cell Tumor; Recurrent Malignant Solid Neoplasm; Recurrent Medulloblastoma; Recurrent Neuroblastoma; Recurrent Non-Hodgkin Lymphoma; Recurrent Osteosarcoma; Recurrent Peripheral Primitive Neuroectodermal Tumor; Recurrent Primary Central Nervous System Neoplasm; Recurrent Rhabdoid Tumor; Recurrent Soft Tissue Sarcoma; Refractory Ewing Sarcoma; Refractory Glioma; Refractory Hepatoblastoma; Refractory Langerhans Cell Histiocytosis; Refractory Malignant Germ Cell Tumor; Refractory Malignant Solid Neoplasm; Refractory Medulloblastoma; Refractory Neuroblastoma; Refractory Non-Hodgkin Lymphoma; Refractory Osteosarcoma; Refractory Peripheral Primitive Neuroectodermal Tumor; Refractory Primary Central Nervous System Neoplasm; Refractory Rhabdoid Tumor; Refractory Rhabdomyosarcoma; Rhabdoid Tumor; Stage III Osteosarcoma AJCC v7; Stage III Soft Tissue Sarcoma AJCC v7; Stage IV Osteosarcoma AJCC v7; Stage IV Soft Tissue Sarcoma AJCC v7; Stage IVA Osteosarcoma AJCC v7; Stage IVB Osteosarcoma AJCC v7; Wilms Tumor	National Cancer Institute (NCI)	2017-07-31	PHASE2
NCT06482086	RECRUITING	Locally Advanced Thyroid Gland Carcinoma	West China Hospital	2021-06-01	PHASE2
NCT03213704	ACTIVE_NOT_RECRUITING	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	National Cancer Institute (NCI)	2017-08-23	PHASE2
NCT03834961	ACTIVE_NOT_RECRUITING	Central Nervous System Neoplasm; Infantile Fibrosarcoma; Recurrent Acute Leukemia; Refractory Acute Leukemia; Solid Neoplasm	Children's Oncology Group	2019-10-25	PHASE2
NCT02637687	ACTIVE_NOT_RECRUITING	Solid Tumors Harboring NTRK Fusion	Bayer	2015-12-16	PHASE1; PHASE2
NCT06390852	ACTIVE_NOT_RECRUITING	Advanced Lymphoma; Advanced Malignant Solid Neoplasm; Refractory Lymphoma; Refractory Malignant Solid Neoplasm; Refractory Multiple Myeloma	National Cancer Institute (NCI)	2017-08-21	PHASE2
NCT04655404	Recruiting	High Grade Glioma\|Diffuse Intrinsic Pontine Glioma	Nationwide Children''s Hospital	2021-04-08	Early Phase 1
NCT04814667	Active not recruiting	Solid Tumor Adult\|Locally Advanced Solid Tumor\|Metastatic Cancer	Centre Leon Berard\|Bayer	2021-02-23	--
NCT04142437	Recruiting	Locally Advanced or Metastatic Solid Tumor Harboring an NTRK Gene Fusion	Bayer	2020-04-03	--
NCT03834961	Active not recruiting	Central Nervous System Neoplasm\|Infantile Fibrosarcoma\|Recurrent Acute Leukemia\|Refractory Acute Leukemia\|Solid Neoplasm	Children''s Oncology Group\|National Cancer Institute (NCI)	2019-10-25	Phase 2
NCT02122913	COMPLETED	Solid Tumors Harboring NTRK Fusion	Bayer	2014-05-04	PHASE1
NCT02637687	Active not recruiting	Solid Tumors Harboring NTRK Fusion	Bayer	2015-12-16	Phase 1\|Phase 2
NCT02576431	Active not recruiting	Solid Tumors Harboring NTRK Fusion	Bayer	2015-09-30	Phase 2

(data from https://clinicaltrials.gov, updated on 2023-02-21)

Check the Larotrectinib (LOXO-101) product page for in-depth specifications, including solubility, stock solutions, MOA, and working concentrations.

Compliance for Clinical Use

Mechanism and Biochemical Profile

Larotrectinib is an ATP-competitive, highly selective inhibitor of tropomyosin-related kinase family receptors that blocks receptor autophosphorylation and downstream oncogenic signal transduction pathways. By suppressing cellular proliferation and inducing apoptosis in TRK-dependent malignant cells, this targeted inhibition halts tumor progression in adult and pediatric patients with solid tumors and central nervous system neoplasms harboring NTRK gene fusions.

Appendix RUO and cGMP Quality Standards

Quality Dimension	RUO (Research Use Only)	cGMP (Current Good Manufacturing Practice)
Clinical Applicability	Prohibited in human clinical trials or medical diagnostics.	Mandatory for human clinical trials (Phase I–III) and therapies.
Regulatory Status	Non-regulated grade; exempt from drug manufacturing laws.	Legally enforced by health authorities (e.g., FDA, EMA, NMPA).
Facility Environment	Unclassified analytical or research laboratories.	Validated Cleanrooms (ISO Class 5–8) with continuous monitoring.
Quality Control	Basic purity and activity testing.	Rigorous safety release testing (Sterility, Endotoxin, Mycoplasma).
Process Validation	Basic equipment calibration; no process validation required.	Full qualification (IQ/OQ/PQ) and complete batch records.
Quality Assurance	Vendor self-declared without required formal QMS.	Mandatory QA/QC unit, Change Control, CAPA, and vendor audits.
Regulatory Impact	High risk of IND rejection if used as a critical raw material.	Required for IND/NDA filings, supported by Drug Master Files (DMF).

Footnotes

Regulatory Note: Governed by FDA (21 CFR Parts 210/211/312), EMA (EudraLex Vol 4), ICH Guidelines (E6/Q7/Q9/Q10), and compendial standards (USP <71>/<85>/<1043>).