RET Inhibitor for Neoadjuvant Therapy in Locally Advanced RET-altered Thyroid Cancer

The Efficacy and Safety of RET Inhibitor for Neoadjuvant Therapy in Locally Advanced RET-altered Thyroid Cancer: a Real-world Study

This study is a multicenter observational study. Patients receive neoadjuvant therapy with RET inhibitors in the real world, and those who can undergo surgery after neoadjuvant therapy receive surgical treatment. The aim of the study is to determine the efficacy and safety of RET inhibitors for neoadjuvant therapy in locally advanced RET-altered thyroid cancer.

Study Overview

• Status: Recruiting
• Conditions: Thyroid Cancer
• Intervention / Treatment: Drug: RET Inhibitor; Procedure: Surgery

• Study Type: Observational
• Enrollment (Estimated): 48

Contacts and Locations

Study Locations

• China
  • Shanghai, China
    • Recruiting
    • Fudan Univeristy Shanghai Cancer Center
    • Contact: Yu Wang, M.D.; Phone Number: 65805 86-021-64175590; Email: neck130@hotmail.com

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Child; Adult; Older Adult
• Accepts Healthy Volunteers: N/A

• Sampling Method: Non-Probability Sample

Study Population

Patients with locally advanced thyroid cancer and RET alterations, who require RET inhibitor treatment and surgery after neoadjuvant therapy.

Description

Inclusion Criteria:

• Patients voluntarily join this study and sign an informed consent form;
• Age: ≥ 14 years old, male or female not limited;
• Locally advanced thyroid cancer diagnosed by histopathology, including papillary thyroid carcinoma, medullary thyroid carcinoma, follicular thyroid carcinoma, poorly differentiated/poorly differentiated thyroid carcinoma, etc;
• RET alterations, including fusion and mutations;

• The definition of locally advanced thyroid cancer meets at least one of the following criteria:

  1. Local advanced thyroid cancer with estimated surgical difficulty and inability to R0/1 resection;
  2. T4 thyroid cancer defined by AJCC: any size of tumor primary lesion or regional lymph node infiltration beyond the thyroid capsule to subcutaneous soft tissue, larynx, trachea, esophagus, or recurrent laryngeal nerve, tumor invasion of pre vertebral fascia or wrapping around carotid or mediastinal blood vessels;
  3. According to the imaging score, the resectable probability is less than 80% based on CT.
• At least one measurable lesion;
• For patients with distant metastasis, researchers need to determine whether patients would benefit from surgery;
• Patients voluntarily undergo tumor tissue biopsy/surgery during enrollment and withdrawal;
• Normal function of major organs.

Exclusion Criteria:

• Previously used RET inhibitors;
• There are multiple factors that affect the absorption of oral medication, such as inability to swallow, nausea and vomiting, chronic diarrhea, and intestinal obstruction;
• The patient refuses to undergo tumor tissue biopsy or surgery;
• Patients who are unsuitable for RET inhibitors or surgery.

Study Plan

How is the study designed?

Number of groups / cohorts

2

Cohorts and Interventions

Group / Cohort	Intervention / Treatment
None MTC; DTC, PDTC and ATC patients with RET fusions.	Drug: RET Inhibitor; RET inhibitors for neoadjuvant treatment; Procedure: Surgery; Operable patients receive surgery after neoadjuvant therapy
MTC; MTC patients with RET mutations	Drug: RET Inhibitor; RET inhibitors for neoadjuvant treatment; Procedure: Surgery; Operable patients receive surgery after neoadjuvant therapy

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Time Frame
R0/1 resection rate	1 year

Secondary Outcome Measures

Outcome Measure	Time Frame
Objective Response Rate, ORR	1 year
Disease Control Rate，DCR	1 year
Time to objective response，TTR	1 year
Progression Free Survival，PFS	5 years
Overall Survival，OS	5 years
Adverse Events	During RET inhibitor treatment, up to 5 years

Collaborators and Investigators

• Sponsor: Fudan University

Study record dates

Study Major Dates

• Study Start (Actual): July 18, 2024
• Primary Completion (Estimated): December 31, 2026
• Study Completion (Estimated): December 31, 2029

Study Registration Dates

• First Submitted: July 27, 2024
• First Submitted That Met QC Criteria: July 27, 2024
• First Posted (Actual): July 31, 2024

Study Record Updates

• Last Update Posted (Actual): August 2, 2024
• Last Update Submitted That Met QC Criteria: July 31, 2024
• Last Verified: July 1, 2024

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Neoplasms; Neoplasms by Site; Endocrine System Diseases; Endocrine Gland Neoplasms; Head and Neck Neoplasms; Thyroid Diseases; Thyroid Neoplasms
• Other Study ID Numbers: PATHYOID

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: UNDECIDED

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: Yes
• Studies a U.S. FDA-regulated device product: No