Study of Regorafenib in Patients With Advanced Myeloid Malignancies
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|ClinicalTrials.gov Identifier: NCT03042689|
Recruitment Status : Active, not recruiting
First Posted : February 3, 2017
Last Update Posted : September 9, 2020
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:
|Condition or disease||Intervention/treatment||Phase|
|Acute Myeloid Leukemia||Drug: Regorafenib||Phase 1|
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 Type :||Interventional (Clinical Trial)|
|Actual Enrollment :||16 participants|
|Intervention Model:||Single Group Assignment|
|Masking:||None (Open Label)|
|Official Title:||Phase I Study of Regorafenib in Patients With Advanced Myeloid Malignancies|
|Actual Study Start Date :||April 17, 2017|
|Estimated Primary Completion Date :||September 2021|
|Estimated Study Completion Date :||September 2023|
Regorafenib will be administered on a 28 day cycle Treatment will be administered on outpatient basis at a pre-determine dosage.
Other Name: Stivarga
- 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.
- Pharmacodynamic effects of regorafenib on cell signaling pathways [ Time Frame: 2 years ]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.
- 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 ]Measure changes in allele burden with regorafenib treatment using next generation sequencing.
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03042689
|United States, Massachusetts|
|Massachusetts general Hospital|
|Boston, Massachusetts, United States, 02114|
|Principal Investigator:||Gabriela Hobbs, MD||Massachusetts General Hospital|