Acute Myeloid LeukemiaPhase 1NCT03042689
Study of Regorafenib in Patients With Advanced Myeloid Malignancies
A Phase 1 study testing Regorafenib in people with Acute Myeloid Leukemia, run by Massachusetts General Hospital at 1 site in United States.
Trial 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) Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Acute Myeloid Leukemia
- What is being tested?
- Regorafenib
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 16 participants
- Where
- 1 siteUnited States
- Study dates
- 17 Apr 2017to 27 Feb 2023
- Phase
- Phase 1
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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.
- 18 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.
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- 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.)
Who cannot join
- 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.
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- 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.
- Major surgical procedure or significant traumatic injury within 28 days before start of study medication.
- Renal failure requiring hemo-or peritoneal dialysis.
- Seizure disorder requiring medication.
- Persistent proteinuria ≥ Grade 3 NCI-CTCAE v4.0 (> 3.5 g/24 hrs, measured by urine protein: creatinine ratio on a random urine sample).
- Interstitial lung disease with ongoing signs and symptoms at the time of informed consent.
- Pleural effusion or ascites that causes respiratory compromise (≥ NCI-CTCAE version 4.0 Grade 2 dyspnea).
- History of organ allograft (including corneal transplant). Known or suspected allergy or hypersensitivity to any of the study drugs, study drug classes, or excipients of the formulations given during the course of this trial.
- Any malabsorption condition.
- Women who are pregnant or breast-feeding, or intend to become pregnant during the study. Women of childbearing potential must have a negative serum pregnancy test performed within 7 days prior to the start of study drug. Post-menopausal women (defined as no menses for at least 1 year) and surgically sterilized women are not required to undergo a pregnancy test.
- Substance abuse, medical, psychological or social conditions that may interfere with the subject's participation in the study or evaluation of the study results.
- Presence of unresolved toxicities from prior anticancer therapy, defined as having not resolved to NCI CTCAE v4.03 grade 0 or 1 with the exception of alopecia. Subjects with irreversible toxicity that is not reasonably expected to be exacerbated by regorafenib (e.g. hearing loss, neuropathy) may be included after consultation with the principal investigator.
- Uncontrolled inter-current illness including, but not limited to, ongoing or active infection, symptomatic congestive heart failure, uncontrolled hypertension, unstable angina pectoris, uncontrolled cardiac arrhythmia, or psychiatric illness/social situations that would limit compliance with study requirements
Trial locations and contacts
1 site in 1 country.
United States1 site
Massachusetts general Hospital
Boston, Massachusetts, 02114
About this study
Official title: Phase I Study of Regorafenib in Patients With Advanced Myeloid Malignancies
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)
Read the detailed description
This research study is a Phase I clinical trial, which tests the safety of an investigational drug and also tries to define the appropriate dose of the investigational drug to use for further studies. "Investigational" means that the drug is being studied.
The FDA (the U.S. Food and Drug Administration) has not approved Regorafenib for this specific disease but it has been approved for other uses.
In this research study, the investigators are looking for the best dose of Regorafenib to treat the participant's disease with. Researchers will also look at how effective the study drug is in treating the participant's disease and if any side effects occur.
- Conditions
- Acute Myeloid Leukemia
- Being tested
- Regorafenib
- Keywords
- AML
- Medical subject headings
- Leukemia, Myeloid, Acute
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
RegorafenibDrug
Also called Stivarga
Regorafenib will be administered on a 28 day cycle Treatment will be administered on outpatient basis at a pre-determine dosage.
| Study group | Role | What they receive |
|---|---|---|
| Regorafenib | Experimental | Not described Receives: Drug: Regorafenib |
What is being measured
The primary outcome is the main question the study is designed to answer.
Maximum tolerated dose of regorafenib in patients with advanced myeloid malignancies
To determine the maximum tolerated dose regorafenib in patients with advanced myeloid malignancies.
Measured: 2 years
Secondary outcomes (4)
Pharmacodynamic effects of regorafenib on cell signaling pathways
To assess pharmacodynamic effect of regorafenib on cell signaling pathways such as FLT-3, RAS and other specific mutations found in each patient's leukemia.
Measured: 2 years
Progression Free Survival
Measured: 2 years
Overall Survival
Measured: 2 years
To Measure Changes In Specific Markers Of Allele Burden
Measure changes in allele burden with regorafenib treatment using next generation sequencing.
Measured: 2 years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted3 Feb 2017 (estimated)
- Study start17 Apr 2017
- Primary completion1 Sep 2021
- Study completion27 Feb 2023
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Acute Myeloid LeukemiaStudies of the same condition, open and under way.
- More trials run by Massachusetts General HospitalThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 16 participants (actual)
Sponsor and investigators
- Lead sponsor
- Massachusetts General Hospital
- Sponsor type
- University, hospital or other
- Collaborators
- Bayer
- Responsible party
- Principal investigator, Gabriela Hobbs, Massachusetts General Hospital
- Investigators
- Gabriela Hobbs, MD, Principal investigator, Massachusetts General Hospital
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03042689
- Sponsor's protocol ID
- 16-464
- Status last verified
- Jan 2026
- First submitted
- 24 Jan 2017
- Last updated
- 8 Jan 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 8 Jan 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Phase I Study of Regorafenib in Patients With Advanced Myeloid Malignancies
- ClinicalTrials.gov ID
- NCT03042689
- Acronym / sponsor ID
- 16-464
- Conditions
- Acute Myeloid Leukemia
- Keywords
- AML
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 16 participants (actual)
- Lead sponsor
- Massachusetts General HospitalUniversity, hospital or other
- Collaborators
- Bayer
- Study start
- 17 Apr 2017 (actual)
- Primary completion
- 1 Sep 2021 (actual)
- Study completion
- 27 Feb 2023 (actual)
- First posted
- 3 Feb 2017 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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)
Detailed description
This research study is a Phase I clinical trial, which tests the safety of an investigational drug and also tries to define the appropriate dose of the investigational drug to use for further studies. "Investigational" means that the drug is being studied.
The FDA (the U.S. Food and Drug Administration) has not approved Regorafenib for this specific disease but it has been approved for other uses.
In this research study, the investigators are looking for the best dose of Regorafenib to treat the participant's disease with. Researchers will also look at how effective the study drug is in treating the participant's disease and if any side effects occur.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Regorafenib | Experimental | Drug: Regorafenib |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (12 listed)
- 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.
- 18 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.
Key exclusion criteria (27 listed)
- 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:
- Evidence or history of bleeding diathesis or coagulopathy.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| RegorafenibAlso: StivargaRegorafenib will be administered on a 28 day cycle Treatment will be administered on outpatient basis at a pre-determine dosage. | Drug | Regorafenib |
Primary outcome
Maximum tolerated dose of regorafenib in patients with advanced myeloid malignancies
Time frame: 2 years
To determine the maximum tolerated dose regorafenib in patients with advanced myeloid malignancies.
4 secondary outcomes
Pharmacodynamic effects of regorafenib on cell signaling pathways
Time frame: 2 years
Progression Free Survival
Time frame: 2 years
Overall Survival
Time frame: 2 years
To Measure Changes In Specific Markers Of Allele Burden
Time frame: 2 years
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 8 Jan 2026.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Gabriela Hobbs, MD principal investigator
Massachusetts General Hospital
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing Regorafenib in people with Acute Myeloid Leukemia, run by Massachusetts General Hospital at 1 site in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 12 inclusion and 27 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 8 Jan 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Maximum tolerated dose of regorafenib in patients with advanced myeloid malignancies (time frame: 2 years). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 8 Jan 2026.