A Phase I/II Study of MGCD0103 (MG-0103) in Combination With Azacitidine in Patients With High-Risk Myelodysplastic Syndrome or Acute Myelogenous Leukemia
Phase I: The purpose of the first part (Phase 1) of this study is to find out what side
effects the experimental drug MG-0103 in combination with azacitidine has on your body. The
first part, or Phase, of the study will find out how much MG-0103 can be given safely along
with azacitidine to people with cancer without causing side effects that are too severe.
Patients are given MG-0103 and azacitidine and are watched closely to see what side effects
may develop and to make sure that if side effects are seen, they can be taken care of
rapidly. If the side effects are not severe, then more patients are asked to join the study
and are given the same or a slightly higher dose of MG-0103. If there are no severe side
effects, patients joining the study later on will get higher doses of MG-0103 than patients
who join earlier. All patients will get the same dose of azacitidine. This will continue
until a dose of MG-0103 is found that causes severe side effects in a high enough portion of
patients. This will be the maximum dose of MG-0103 that can be given to patients in this
study. Doses higher than that will not be given. Additional patients may be asked to join
the study and receive MG-0103 and possibly azacitidine at lower doses that did not cause
severe side effects.
Phase II: MG-0103 in combination with azacitidine may also have some effect on your disease.
The purpose of the second part (Phase 2) of this study is to find out what, if any, effect
there is. This Phase of the study will also find out more information about side effects of
this combination of drugs. In this Phase, patients will receive a slightly lower dose than
the maximum dose found in the first part of the study (which causes tolerable side effects).
If a certain effect on the disease of patients is seen, then more patients are asked to
join the study. Additional patients may also be asked to join the study and receive a lower
dose if information collected during the study suggests that this should be done.
Allocation: Non-Randomized, Endpoint Classification: Safety/Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment
Maximum tolerated dose in combination with azacitidine
1 year (anticipated)
Gregory Reid, MSc, MBA
United States: Food and Drug Administration
|Memorial Sloan-Kettering Cancer Center||New York, New York 10021|
|University of Pennsylvania||Philadelphia, Pennsylvania 19104|
|Thomas Jefferson University||Philadelphia, Pennsylvania 19107-6541|
|The Western Pennsylvania Hospital||Pittsburgh, Pennsylvania 15224|
|Thomas Jefferson University Hospital||Philadelphia, Pennsylvania 19131|
|University of Texas, MD Anderson Cancer Center||Houston, Texas 77030|
|University of Southern California||Los Angeles, California 90033|
|St. Francis Hospital & Health Center||Beech Grove, Indiana 46107|