Neoplasms

All Conditions

Digestive System Diseases

Respiratory Tract (Lung and Bronchial) Diseases

Gland and Hormone Related Diseases

Nutritional and Metabolic Diseases

Rare Diseases

Carcinoma

Carcinoma, Neuroendocrine

Colorectal Neoplasms

Adenocarcinoma

Carcinoma, Non-Small-Cell Lung

Thyroid Neoplasms

Thyroid Cancer, Papillary

Thyroid Diseases

Intestinal Diseases

Head and Neck Neoplasms

Colonic Diseases

Lung Neoplasms

Colonic Neoplasms

Lung Diseases

Gastrointestinal Diseases

Neuroendocrine Tumors

Gastrointestinal Neoplasms

Intestinal Neoplasms

Neoplasms, Germ Cell and Embryonal

Endocrine Gland Neoplasms

Protein-Energy Malnutrition

Respiratory Tract Diseases

Thoracic Neoplasms

Digestive System Neoplasms

Respiratory Tract Neoplasms

Bronchial Neoplasms

Carcinoma, Bronchogenic

Neoplasms, Nerve Tissue

Endocrine System Diseases

Neoplasms, Glandular and Epithelial

Neoplasms by Histologic Type

Neuroectodermal Tumors

Neuroectodermal Tumors, Primitive

Adenocarcinoma, Papillary

Rectal Diseases

Neoplasms, Neuroepithelial

Bronchial Diseases

Papillary Thyroid Carcinoma

Neuroendocrine Tumor

Thyroid Cancer, Medullary

Neuroepithelioma

Antineoplastic Agents

All Drugs and Chemicals

Amino Acids

Pralsetinib

Tyrosine

Multiple doses of pralsetinib (BLU-667) for oral administration.

Oral dose of pralsetinib (BLU-667) as determined during Dose Escalation.

pralsetinib (BLU-667) is a potent and selective inhibitor of the RET mutations, fusions, and predicted resistant mutants

pralsetinib (BLU-667)

Clinical Trials

This is a Phase 1/2, open-label, first-in-human (FIH) study designed to evaluate the safety, tolerability, pharmacokinetics (PK), pharmacodynamics (PD), and preliminary antineoplastic activity of pralsetinib (BLU-667) administered orally in participants with medullary thyroid cancer (MTC), RET-altered NSCLC and other RET-altered solid tumors.

The study consists of 2 parts, a dose-escalation part (Phase 1) and an expansion part (Phase 2). Both parts will enroll participants with advanced non-resectable NSCLC, advanced non-resectable thyroid cancer and other advanced solid tumors that have progressed following standard systemic therapy, have not adequately responded to standard systemic therapy, or the participants must be intolerant to or the Investigator has determined that treatment with standard therapy is not appropriate, or there must be no accepted standard therapy for their disease.

Phase 1 (Complete):

* Advanced MTC, NSCLC or other solid tumor
* 30-600mg (PO QD or BID)

Phase 2 (400mg QD):

* Group 1: RET fusion NSCLC previously treated with a platinum chemotherapy
* Group 2: RET fusion NSCLC not previously treated for metastatic disease
* Group 3: MTC previously treated with cabozantinib and/or vandetanib
* Group 4: MTC not previously treated with cabozantinib or vandetanib
* Group 5: Other solid tumors with a RET fusion not eligible for any of the other groups and previously treated with SOC or have no acceptable SOC for their tumor type as determined by the investigator
* Group 6: Any solid tumor with a RET alteration (fusion or mutation) previously treated with a selective RET Inhibitor
* Group 7: Other solid tumors with a RET mutation previously treated with SOC
* Group 8: RET fusion NSCLC previously treated with a platinum chemotherapy (China only)
* Group 9: MTC not previously treated with systemic therapy for advanced or metastatic disease (China only)

Phase 1/2 Study of the Highly-selective RET Inhibitor, Pralsetinib (BLU-667), in Participants With Thyroid Cancer, Non-Small Cell Lung Cancer, and Other Advanced Solid Tumors

A Phase 1/2 Study of the Highly-selective RET Inhibitor, BLU-667, in Patients With Thyroid Cancer, Non-Small Cell Lung Cancer (NSCLC) and Other Advanced Solid Tumors

As assessed by Response Evaluation Criteria in Solid Tumors, Version 1.1 (RECIST v1.1) or Response Assessment in Neuro-Oncology (RANO), as appropriate per tumor type

As assessed by RECIST v1.1 or RANO, as appropriate per tumor type

Clinical Benefit Rate (CBR), Duration of Response (DOR), Disease Control Rate (DCR), Progression Free Survival (PFS)

RET gene status (i.e. gene fusion partner or primary mutation and, for MTC, whether hereditary or sporadic)

Will be measured from lead II and will be corrected for heart rate (QTc)n using Fridericia's correction factors

Hoffmann-La Roche

Spots Global Cancer Trial Database for adenocarcinoma, papillary