A Phase I Clinical Trial to Study the Safety, Pharmacokinetics, and Efficacy of BP-100.1.01 (L-Grb-2 Antisense Oligonucleotide) in Patients With Refractory or Relapsed Acute Myeloid Leukemia, Philadelphia Chromosome Positive Chronic Myelogenous Leukemia, or Acute Lymphoblastic Leukemia, and Myelodysplastic Syndrome
The Philadelphia Chromosome is an unusual genetic trait found in 90-95% of patients with
CML. The protein created by this unusual trait causes normal cells within the body to become
cancer cells, and then causes these cells to grow and divide at a rapid rate. Researchers
think that the protein Growth Factor Receptor Bound Protein-2 (Grb-2) is necessary to the
growth of these cancerous cells. The drug under study may be able to prevent the cells from
making this protein. Researchers hope that without this protein, the leukemia cells will
die.
Between 18 and 30 patients are expected to be enrolled on this study. Between 3 and 6
patients will be treated at each dose level. Each new group will be treated at a dose higher
than the previous group. If the highest safe dose of the study drug is not found after the
completion of this study, additional doses or another study may be considered.
The study drug is an antisense molecule complementary to the mRNA (messenger RNA) code for
the cell's expression of the protein Grb-2. The study drug is incorporated into lipid (fat)
particles known as liposomes. This incorporation process is part of the manufacturing
procedure and is done before the drug is administered. The liposomes (which carry the study
drug) will then be injected into a vein twice a week for at least 28 days.
Interventional
Endpoint Classification: Safety Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment
Safety
Evaluate the patient tolerance to this cancer drug
30 days
Yes
Jorge Cortes, MD
Study Chair
M.D. Anderson Cancer Center
United States: Food and Drug Administration
2003-0578 (v) 08-4
NCT01159028
June 2010
Name | Location |
---|---|
M. D. Anderson Cancer Center | Houston, Texas 77030 |