A Prospective Cohort Study of Pralsetinib or Anlotinib in the Treatment of Locally Advanced and/or Metastatic Medullary Thyroid Carcinoma With RET Gene Mutations

NCT07048964 | Not Yet Recruiting

• 1 other identifier: 22/284-3486
• Study Type: observational
• Target: 40
• Locations: 0 countries
• Sites: N/A

Brief Summary

This trial is a prospective, observational Phase II clinical study. For patients with locally advanced and/or metastatic medullary thyroid carcinoma with RET gene mutations who require systemic treatment, they are randomly assigned to either the Pralsetinib or Anlotinib observation cohort based on their clinical treatment choices. The treatment continues until disease progression or the occurrence of intolerable adverse reactions. At the same time, the correlation between the efficacy and safety of the drugs and the RET gene mutation subtypes is analyzed, and the resistance mechanisms of Anlotinib and Pralsetinib are preliminarily explored to provide more evidence for the clinical treatment of patients with locally advanced or metastatic MTC in China.

Conditions

Medullary Thyroid Cancer (MTC)

Outcome Measures

Primary Outcomes (1)

• To evaluate the objective response rate (ORR) of Pralsetinib or Anlotinib in patients with RET gene mutation-related locally advanced or metastatic medullary thyroid carcinoma (MTC) who require systemic treatment

  Defined as complete response (CR) or partial response (PR) according to RECIST v1.1

  24 months

Secondary Outcomes (6)

• Progression-free survival (PFS) as assessed by Investigator

  24 months

• Duration of Response (DOR)

  24 months

• Disease control rate (DCR)

  24 months

• Changes in blood calcitonin (CT) and carcinoembryonic antigen (CEA)

  24 months

• Safety: Type incidence and severity (as graded by NCI CTCAE v 4.0) of Pralsetinib or Anlotinib

  24 months

Other Outcomes (2)

• To explore the correlation of different RET gene mutation subtypes and other tumor tissue biomarkers with the efficacy and safety of drug treatment

  24 months

• Preliminary exploration of resistance mechanisms in Chinese patients with locally advanced or metastatic RET-mutant medullary thyroid carcinoma (MTC) requiring systemic therapy after treatment with pralsetinib.

  24 months

Study Arms (2)

Drug: Pralsetinib

Drug: Anlotinib

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)
• Sampling Method: Non-Probability Sample

Study Population

Patients with RET gene mutation-related locally advanced or metastatic medullary thyroid carcinoma (MTC) who require systemic treatment.

You may qualify if:

• Age ≥18 years.
• Pathologically confirmed medullary thyroid carcinoma (MTC).
• Patients with locally advanced or metastatic MTC who require systemic treatment.
• Disease progression within 14 months before the screening visit.
• Presence of RET gene mutations based on tumor tissue and/or blood evaluations conducted by the study center.
• No prior treatment with pralsetinib and/or anlotinib and/or cabozantinib and/or vandetanib.
• Eastern Cooperative Oncology Group (ECOG) performance status (PS) score of 0-1.
• Expected survival time ≥ 3 months
• Signed informed consent to participate in this study.

You may not qualify if:

• The patient meets any of the following criteria within 14 days before the first administration of the study drug:
• Platelet count \< 75 × 10\^9/L.
• Absolute neutrophil count (ANC) \< 1.0 × 10\^9/L.
• Hemoglobin \< 9.0 g/dL, with the possibility of elevating hemoglobin to 9.0 g/dL or higher through red blood cell transfusion and erythropoietin use, provided such treatment is completed at least 2 weeks before the first administration of the study drug.
• Aspartate aminotransferase (AST) or alanine aminotransferase (ALT) \> 3 × upper limit of normal (ULN) without liver metastases; \> 5 × ULN with liver metastases.
• Total bilirubin \> 1.5 × ULN, \> 3 × ULN with liver metastases; in the case of Gilbert's syndrome, total bilirubin \> 3 × ULN and direct bilirubin \> 1.5 × ULN.
• Estimated (Cockcroft-Gault formula) or measured creatinine clearance \< 60 mL/min.
• The patient's QTcF \> 470 msec. The patient has a history of long QT syndrome or torsades de pointes (TdP). The patient has a family history of long QT syndrome.
• The patient has clinically significant, uncontrolled cardiovascular diseases, including congestive heart failure classified as New York Heart Association (NYHA) Class III or IV; myocardial infarction or unstable angina within 6 months; uncontrolled hypertension; or clinically significant uncontrolled arrhythmias, including bradyarrhythmias that may lead to QT prolongation (e.g., second-degree atrioventricular block type II or third-degree atrioventricular block).
• The patient has metastatic central nervous system (CNS) tumors or primary CNS tumors with progressive neurological symptoms or requiring an increased dose of corticosteroids to control CNS disease. If corticosteroid treatment is needed for CNS disease, the dosing must be stable within the two weeks prior to C1D1.
• The patient has symptomatic interstitial lung disease or interstitial pneumonia, including radiation pneumonitis (i.e., affecting daily activities or requiring therapeutic intervention).
• The patient has received anti-tumor therapy within 14 days or five half-lives of the study drug prior to the first administration.
• The patient has received granulocyte colony-stimulating factor (G-CSF) support therapy within 14 days prior to the first administration of the study drug.
• The patient has undergone major surgery (excluding central venous catheterization, tumor biopsy, and gastrostomy tube insertion) within 14 days before the first administration of the study drug.
• The patient has been diagnosed with or requires treatment for another primary malignancy within the past 3 years, except for completely resected basal cell and squamous cell carcinoma of the skin, localized prostate cancer after curative treatment, and any in situ carcinoma that has been fully resected.

Sponsors & Collaborators

• Cancer Institute and Hospital, Chinese Academy of Medical Sciences: lead

MeSH Terms

Conditions

Carcinoma, Medullary

Condition Hierarchy (Ancestors)

Carcinoma, Neuroendocrine; Neuroendocrine Tumors; Neuroectodermal Tumors; Neoplasms, Germ Cell and Embryonal; Neoplasms by Histologic Type; Neoplasms; Adenocarcinoma; Carcinoma; Neoplasms, Glandular and Epithelial; Neoplasms, Ductal, Lobular, and Medullary; Neoplasms, Nerve Tissue

Study Design

• Study Type: observational
• Observational Model: COHORT
• Time Perspective: PROSPECTIVE
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: May 23, 2025
• First Posted: July 3, 2025
• Study Start: August 1, 2025
• Primary Completion: February 28, 2026
• Study Completion (Estimated): February 28, 2027
• Last Updated: July 3, 2025

Record last verified: 2025-06