This trial is active, not recruiting.

Condition acute myeloid leukemia
Sponsor Taipei Medical University WanFang Hospital
Start date April 2008
Trial size 100 participants
Trial identifier NCT01142375, Leukemia-001


Acute leukemias are commonly seen in elderly and have no effective therapy. In recent years, small molecule inhibitors have shown a tremendous promise in cancer treatment such as chronic myeloid leukemia and lung cancer. Thus, in this proposal, we intend to use our novel patented "indole" compound to conjugated with hydroxamic acid in histone deacetylases inhibitor, suberoylanilide hydroxamic acid and further modify its structure to generate novel small-molecule anticancer compounds. By screening acute leukemic cell lines, we will look for compounds that have shown potential for future drug development. In our preliminary studies, we have identified a novel compound, MPT0E001, to have marked growth inhibitory activity, induce apoptosis and downregulate c-Myc protein level. We intend to use MPT0E001 as a basis to develop novel compounds and test them in multiple leukemic cell lines and primary leukemia samples (Specific aim 1) and identify the mechanisms for downregulation of c-Myc and other pathways such as NF-kappaB and Akt (Specific aim 2). Once we have identified the lead compounds, we will use murine model to assess the acute toxicity profiles in different dose ranges and examine the effects of blood counts and vital organs. After toxicity study, we will test the lead compounds using in vivo xenogenic murine model using both cell lines and primary leukemia. Using this approach, we hope to develop novel compounds that are able to be used in future leukemic therapy.

United States No locations recruiting
Other countries No locations recruiting

Study Design

Observational model cohort
Time perspective prospective

Eligibility Criteria

Male or female participants of any age.

Inclusion Criteria: - Acute Myeloid Leukemia Exclusion Criteria: - Hematologic malignancies other than AML

Additional Information

Official title Development and Exploration of the Feasibility of Using Locally Synthesized Small Molecule Inhibitors to Treat Human Acute Leukemia Cells
Principal investigator Hsin-Gjin Eugene Liu
Trial information was received from ClinicalTrials.gov and was last updated in June 2010.
Information provided to ClinicalTrials.gov by Taipei Medical University WanFang Hospital.