Clinical Trial of BP1001 (L-Grb-2 Antisense Oligonucleotide) in CML, AML, ALL & MDS
Condition: Recurrent Adult Acute Myeloid Leukemia; Acute Lymphoblastic Leukemia; Myelodysplastic Syndrome; Ph1 Positive CML
MeSH terms: Leukemia; Leukemia, Myeloid; Leukemia, Myeloid, Acute; Preleukemia; Precursor Cell Lymphoblastic Leukemia-Lymphoma; Leukemia, Lymphoid; Myelodysplastic Syndromes
Study type: Interventional
Phase: Phase 1

Summary: 
      The first goal of this clinical research study is to find the highest safe dose of BP1001, a
      liposomal Growth Factor Receptor Bound Protein-2 antisense oligodeoxynucleotide (L-Grb2 AS),
      for patients with Philadelphia Chromosome positive CML, AML, ALL and MDS. The response of the
      leukemia to this treatment will also be studied. The second goal of this clinical research
      study is to evaluate the safety and toxicity of the combination of BP1001 and concurrent
      low-dose ara-C (LDAC) in patients with AML.
    

Detailed description: 
      The Philadelphia Chromosome is an unusual genetic trait found in 90-95% of patients with CML
      and approximately 20-25% of patients with ALL. 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)" plays an important role in the rapid growth of leukemic cells. The
      study drug (BP1001) may be able to inhibit the cells from making Grb-2. Researchers hope that
      without this protein, the leukemia cells will die.

      Up to 60 patients are expected to be enrolled on this study.

      Part A: Dose escalation: Each cohort will receive BP1001 at a dose higher than the previous
      group.

      Part B: Dose-expansion Cohorts: Subjects with relapsed or refractory AML will receive
      escalating doses of BP1001 concurrently with fixed low-dose ara-C (LDAC)

      The study drug is an antisense molecule complementary to the messenger RNA (mRNA) 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 process
      and is done before the study drug is administered. The liposomes (which carry the study drug)
      will be administered intravenously twice a week for 28 days. Subjects enrolled in Part B of
      the study will receive study drug twice a week for 28 days concurrently with low dose ara-C,
      self administered twice daily for 10 consecutive days.
    

Intervention name(s): BP1001; BP1001 in combination with LDAC
Intervention type(s): Drug; Drug
Intervention description(s): Study drug (BP1001) is constituted in normal saline, administered by IV on twice weekly for 28 days.; Study drug (BP1001) is constituted in normal saline, administered by IV twice weekly for 28 days. Low dose ara-C (LDAC) is self administered twice daily for 10 consecutive days during the 28 day cycle.
Other intervention name(s): Liposomal Grb-2; L-Grb-2; BP-100-1.01; Liposomal Grb-2; L-Grb-2; BP-100-1.01; low dose ara-C
Allocation: Non-Randomized
Intervention model: Single Group Assignment
Masking: None (Open Label)
Primary purpose: Treatment
Keywords: Liposomal Grb-2 treatment of CML, AML, CLL, MDS; Liposomal Grb-2 with LDAC for AML