Study of Regorafenib in Patients With Advanced Myeloid Malignancies

NCT03042689 | Completed Phase 1 DMC FDA Drug

• 1 other identifier: 16-464
• Study Type: interventional
• Target: 16
• Locations: 1 country
• Sites: 1

Brief Summary

This research study is studying a drug as a possible treatment for advanced myeloid malignancies including AML (acute myeloid leukemia), MDS (myelodysplastic syndrome) and MPN (myeloproliferative neoplasms) The intervention involved in this study is:

• Regorafenib (Stivarga)

Conditions

Acute Myeloid Leukemia

Keywords

AML

Outcome Measures

Primary Outcomes (1)

• Maximum tolerated dose of regorafenib in patients with advanced myeloid malignancies

  To determine the maximum tolerated dose regorafenib in patients with advanced myeloid malignancies.

  2 years

Secondary Outcomes (4)

• Pharmacodynamic effects of regorafenib on cell signaling pathways

  2 years

• Progression Free Survival

  2 years

• Overall Survival

  2 years

• To Measure Changes In Specific Markers Of Allele Burden

  2 years

Study Arms (1)

Regorafenib

EXPERIMENTAL

Drug: Regorafenib

Interventions

Regorafenib DRUG

Regorafenib will be administered on a 28 day cycle Treatment will be administered on outpatient basis at a pre-determine dosage.

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:

• Diagnosis of relapsed/refractory advanced malignancies. Specifically: relapsed refractory refractory acute myeloid leukemia that have failed one line of prior therapy, myelodysplastic syndrome that have failed hypomethylating agents, myelofibrosis that have failed ruxolitinib or are ineligible for this therapy.
• years of age or greater.
• ECOG performance status of 0-1.
• Able to provide informed consent.
• Subjects (men and women) of childbearing potential must agree to use adequate contraception beginning at the signing of the ICF until at least 2 months after the last dose of study drug. The definition of adequate contraception will be based on the judgment of the principal investigator or a designated associate.
• Able to swallow and retain oral medication.
• Normal organ and marrow function defined as:
• Total bilirubin ≤ 1.5 x the upper limits of normal (ULN) except for patients with Gilbert's disease.
• Alanine aminotransferase (ALT) and aspartate aminotransferase (AST) ≤ 2.5 x ULN (≤ 5 x ULN for subjects with liver involvement of their cancer).
• Alkaline phosphatase limit ≤ 2.5 x ULN (≤ 5 x ULN for subjects with liver or bone involvement of their cancer).
• Serum creatinine ≤ 1.5 x the ULN.
• International normalized ratio (INR)/ Partial thromboplastin time (PTT) ≤ 1.5 x ULN. (Subjects who are prophylactically treated with an agent such as warfarin or heparin will be allowed to participate provided that no prior evidence of underlying abnormality in coagulation parameters exists.)

You may not qualify if:

• Previous assignment to treatment during this study. Subjects permanently withdrawn from study participation will not be allowed to re-enter the study.
• Systemic antineoplastic therapy in the past 14 days (excluding hydroxyurea).
• Uncontrolled hypertension (systolic pressure \>140 mmHg or diastolic pressure \> 90 mmHg 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.
• Eligible for, have a suitable donor and are willing to undergo Hematopoietic Stem Cell Transplantation (HSCT)
• Any hemorrhage or bleeding event ≥ NCI CTCAE Grade 3 within 4 weeks prior to start of study medication.
• Thrombotic, embolic, venous or arterial events, such as cerebrovascular accident (including transient ischemic attacks), deep vein thrombosis or pulmonary embolism within 6 months of informed consent.
• Previously untreated or concurrent cancer that is distinct in primary site or histology except cervical cancer in-situ, treated ductal carcinoma in situ of the breast, curatively treated non-melanoma skin carcinoma, noninvasive aerodigestive neoplasms 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 registration.
• Known history of human immunodeficiency virus (HIV) infection or current chronic or active hepatitis B or C infection requiring treatment with antiviral therapy.
• Presence of a non-healing wound, non-healing ulcer or bone fracture.

Sponsors & Collaborators

• Massachusetts General Hospital: lead
• Bayer: collaborator

Study Sites (1)

Massachusetts general Hospital

Boston, Massachusetts, 02114, United States

Location

MeSH Terms

Conditions

Leukemia, Myeloid, Acute

Interventions

regorafenib

Condition Hierarchy (Ancestors)

Leukemia, Myeloid; Leukemia; Neoplasms by Histologic Type; Neoplasms; Hematologic Diseases; Hemic and Lymphatic Diseases

Study Officials

• Gabriela Hobbs, MD

  Massachusetts General Hospital

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Gabriela Hobbs, MD

Study Record Dates

• First Submitted: January 24, 2017
• First Posted: February 3, 2017
• Study Start: April 17, 2017
• Primary Completion: September 1, 2021
• Study Completion: February 27, 2023
• Last Updated: January 8, 2026

Record last verified: 2026-01

Data Sharing

• IPD Sharing: Will not share

Locations

US (1)