A Study Using Regorafenib as Second or Third Line Therapy in Metastatic Medullary Thyroid Cancer

NCT02657551 | Active Not Recruiting Phase 2 Results DMC

• 1 other identifier: 15-350
• Study Type: interventional
• Target: 8
• Locations: 1 country
• Sites: 2

Brief Summary

This research study is studying a targeted therapy as a possible treatment for thyroid cancer. A targeted therapy is a type of treatment that uses drugs or other substances to identify and attack specific types of cancer cells with less harm to normal cells. \- The name of the study intervention involved in this study is regorafenib.

Conditions

Thyroid Cancer

Keywords

Thyroid Cancer

Outcome Measures

Primary Outcomes (2)

• 10-month Progression-free Survival (PFS) Rate [MTC Cohort]

  10-month PFS Rate is the proportion of participants ramaing alive and progression free at 10 months. Progression-free survival based on the Kaplan-Meier method is defined as the duration of time from study entry to documented disease progression (PD) or death. Per RECIST 1.1 criteria: progressive disease (PD) is at least a 20% increase in the sum of longest diameter (LD) of target lesions taking as reference the smallest sum LD recorded since the treatment started or the appearance of one or more new lesions. PD for the evaluation of non-target lesions is the appearance of one or more new lesions and/or unequivocal progression of non-target lesions.

  Disease was evaluated through imaging scan on baseline, Cycle 1 Day 1, end of treatment and during follow-up. Relevant to this endpoint is 10 Months

• Response Rate [Differentiated Thyroid Cancer (DTC)] DATA NOT MATURE YET

  The response rate is the proportion of participants achieving XX based on RECIST 1.1 criteria defined per protocol section 11.1.4 for target lesions.

  2 Years

Secondary Outcomes (2)

• Grade 3-5 Treatment-related Toxicity Rate [MTC Cohort]

  AEs were evaluated on treatment cycle 1 day 1 of week 1, 2 and 3, and cycle 2 day 1 of week 1 and 3, and cycle 3 and beyond day 1 of week 1. For this study cohort, participants were evaluated for AEs for the maximum treatment duration up to 24 months.

• Quality of Life (QOL) DATA NOT MATURE YET

  Reported cycle 1 and 2 on day 1 of week 1 and 3, cycle 3 and beyond on day 1 of week 1, EOT, and follow-up.

Study Arms (1)

Regorafenib

EXPERIMENTAL

Regorafenib tablets 80mg orally, once daily at predetermined dosage for 21 days per cycle

Drug: Regorafenib

Interventions

Regorafenib DRUG

Regorafenib is a small molecule inhibitor of multiple membrane-bound and intracellular kinases involved in normal cellular functions and in pathologic processes.

Also known as: Stivarga

Regorafenib

Eligibility Criteria

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

You may qualify if:

• Subjects must be able to understand and be willing to sign the written informed consent form. A signed informed consent form must be appropriately obtained prior to the conduct of any trial-specific procedure.
• Age ≥ 18 years.
• Life expectancy of at least 12 weeks (3 months).
• Eastern Cooperative Oncology Group performance status of ≤1.
• Histologically or cytologically confirmed diagnosis of metastatic medullary thyroid cancer.
• Documented disease progression within 6 months prior to study registration, as defined by RECIST criteria.
• Must have at least 1 site of measurable disease by RECIST criteria, by version 1.1.
• Archival tissue block or unstained slides (from primary or metastatic site) must be available, otherwise fresh tissue biopsy sample will be collected.
• Any number of prior chemotherapies and targeted therapies are allowed.
• Patients must have received at least one prior line of targeted therapy.
• Adequate bone marrow, liver and renal function as assessed by the following laboratory requirements within 3 weeks prior to study registration:
• Total bilirubin ≤ 1.5 x the upper limits of normal (ULN)
• Alanine aminotransferase (ALT) and aspartate amino-transferease (AST) ≤ 2.5 x ULN (≤ 5 x ULN for subjects with liver involvement of their cancer)
• Alkaline phosphastase limit ≤ 2.5 x ULN (≤ 5 x ULN for subjects with liver involvement of their cancer)
• Serum creatinine ≤ 1.5 x the ULN

You may not qualify if:

• Prior treatment with regorafenib.
• Previous assignment to treatment during this study. Subjects permanently withdrawn from study participation will not be allowed to re-enter study.
• Uncontrolled hypertension (systolic pressure \>140 mm Hg or diastolic pressure \> 90 mm Hg \[NCI-CTCAE v4.0\] on repeated measurement) despite optimal medical management.
• Active or clinically significant cardiac disease including:
• Congestive heart failure - New York Heart Association (NYHA) \> Class II.
• Active coronary artery disease.
• Cardiac arrhythmias requiring anti-arrhythmic therapy other than beta blockers or digoxin.
• Unstable angina (anginal symptoms at rest), new-onset angina within 3 months before randomization, or myocardial infarction within 6 months before randomization.
• Evidence or history of bleeding diathesis or coagulopathy.
• Any hemorrhage or bleeding event ≥ NCI CTCAE Grade 3 within 4 weeks prior to start of study medication.
• Subjects with thrombotic, embolic, venous, or arterial events, such as cerebrovascular accident (including transient ischemic attacks) deep vein thrombosis or pulmonary embolism within 6 months of start of study treatment.
• Subjects with any previously untreated or concurrent cancer that is distinct in primary site or histology except cervical cancer in-situ, treated basal cell carcinoma, or superficial bladder tumor. Subjects surviving a cancer that was curatively treated and without evidence of disease for more than 3 years before registration are allowed. All cancer treatments must be completed at least 3 years prior to study entry (i.e., signature date of the informed consent form).
• Patients with pheochromocytoma.
• Known history of human immunodeficiency virus (HIV) infection or current chronic or active hepatitis B or C infection requiring treatment with antiviral therapy.
• Ongoing infection \> Grade 2 NCI-CTCAE v4.0.

Sponsors & Collaborators

• Dana-Farber Cancer Institute: lead
• Bayer: collaborator

Study Sites (2)

Yale Cancer Center

New Haven, Connecticut, 06520-8028, United States

Location

Dana-Farber Cancer Institute

Boston, Massachusetts, 02115, United States

Location

Related Publications (1)

• Ettrich TJ, Seufferlein T. Regorafenib. Recent Results Cancer Res. 2018;211:45-56. doi: 10.1007/978-3-319-91442-8_3.

  PMID: 30069758 DERIVED

MeSH Terms

Conditions

Thyroid Neoplasms

Interventions

regorafenib

Condition Hierarchy (Ancestors)

Endocrine Gland Neoplasms; Neoplasms by Site; Neoplasms; Head and Neck Neoplasms; Endocrine System Diseases; Thyroid Diseases

Results Point of Contact

• Title: Kartik Seghal
• Organization: Dana-Farber Cancer Institute

Kartik_Sehgal@dfci.harvard.edu

000-000-0000

Study Officials

• Kartik Seghal, MD

  Dana-Farber Cancer Institute

  PRINCIPAL INVESTIGATOR

Publication Agreements

• PI is Sponsor Employee: Yes

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Principal Investigator

Study Record Dates

• First Submitted: November 9, 2015
• First Posted: January 18, 2016
• Study Start: January 1, 2016
• Primary Completion: December 1, 2025
• Study Completion: December 1, 2025
• Last Updated: September 11, 2025
• Results First Posted: November 15, 2023

Record last verified: 2025-08

Data Sharing

• IPD Sharing: Will not share

Locations

US (2)