SL-401 in Patients with Acute Myeloid Leukemia or Blastic Plasmacytoid Dendritic Cell Neoplasm

Protocol: 
AAAQ4760
Phase: 
I/II

SL-401 in Patients with Acute Myeloid Leukemia or Blastic Plasmacytoid Dendritic Cell Neoplasm

The goal of this research study is to find the safest highest dose of SL-401 that can be given to patients with AML or BPDCN. This study will also look at how SL-401 stops or slows leukemia or BPDCN growth and how SL-401 enters and leaves the body. This study will also look at certain proteins in the blood and bone marrow and how the amounts of them might change after receiving SL-401, or might be related to how well SL-401 does or does not function against the cancer. SL-401 is an investigational medication. This means that SL-401 is still being studied. It also means that the United States Food and Drug Administration (also called the FDA) does not allow it to be sold in the United States for the treatment of patients. The FDA allows SL-401 to be used only in research studies. The study will be done at about 25 centers in the United States, Canada and Europe. About 60 men and women who are at least 18 years of age will be in the study. It is anticipated that each patient will be in the study for about 6 ½ months, although the amount of time that each patient will participate will vary, depending on potential side effects and the growth or shrinkage of the cancer.

Are you Eligible? (Inclusion Criteria)

1. The patient has a diagnosis of AML or BPDCN according to WHO classification (AML; excluding acute promyelocytic leukemia [APL, FAB M3]) or confirmed by hematopathology (BPDCN).

2. The patient must meet one of the following (a) or (b) or (c):

(a) Has evidence of persistent or recurrent AML in the peripheral blood and/or bone marrow that is
refractory to, or has relapsed from, their most recent prior line of treatment.
• A prior line of treatment is considered an induction regimen if it involves an approved or investigational cytotoxic chemotherapy agent, biological agent, and/or hypomethylating agent administered alone or in a combination regimen, with the intent to induce robust cytoreduction (i.e., CR).
• The previous induction regimen may have been a SCT with intent to induce a CR.
• Consolidation and/or maintenance (including SCT) may have been given in CR/CRi, but are not counted as a line of treatment.
• Hydroxyurea will not be considered a prior line of treatment.

(b) Has previously untreated AML and is considered to be at high risk for disease progression and/or
is unlikely to derive more than transient benefit from standard therapy by having at least one of the
following:
• Treatment-related AML, except if it is associated with favorable cytogenetics (e.g., inversion 16, t(16;16), t(8;21), t(15;17)), and not a candidate for SCT in their current disease state.
• AML with antecedent hematological disease (e.g., myelodysplastic syndrome (MDS), myelofibrosis, polycythemia vera, etc.) and not a candidate for SCT.

(c) Has histological and/or cytological evidence of BPDCN by pathologic assessment at the investigative site according to World Health Organization classification (2008), that can be measured
for treatment response and is either:
• Previously untreated (allowed for Stage 1 only).
• Persistent or recurrent in the peripheral blood, bone marrow, spleen, lymph nodes, skin, or other sites after previous treatment with at least 1 line of systemic therapy for BPDCN, e.g., stem cell transplant or chemotherapy (Stage 1 or 2). A pathology specimen must be available for central pathology review for all BPDCN patients enrolled in Stage 2 (required for Stage 2 only).

3. The patient >= 18 years old.

Exclusion:
1. The patient has a diagnosis of acute promyelocytic leukemia

Specialty Area(s)

Trial Location

Herbert Irving Comprehensive Cancer Center
161 Fort Washington Ave.
Herbert Irving Pavilion
New York, NY 10032
United States