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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


Phase 1
18 Years
70 Years
Open (Enrolling)
Both
Safety and Pharmacokinetics of L-Grb-2 in Treating CML, AML, CLL and MDS

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Trial Information

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.


Inclusion Criteria:



- 4.1.1 Male or female patients 18 years of age or older 4.1.2 A diagnosis of
refractory or relapsed acute myeloid leukemia, or Ph+ CML, in chronic, accelerated or
blast phase, or acute lymphoblastic leukemia, or myelodysplastic syndrome.

One of the following parameters is required to meet criteria for accelerated phase CML:

- Blasts in Peripheral Blood or Bone Marrow ≥15%

- Promyelocytes and Blasts in Peripheral Blood or Bone Marrow ≥30%

- PB or BM basophils ≥20%

- Thrombocytopenia <100 x 103/ml, not resulting from therapy

Blast phase is defined as ≥30% blasts in peripheral blood or bone marrow, or presence of
extramedullary disease, except for liver or spleen.

4.1.2.1 Patients with CML must have demonstrated resistance and/or intolerance to therapy
with at least 2 tyrosine kinase inhibitors (TKI) 4.1.2.2 AML and Ph+ ALL should have
received at least one prior treatment regimen and either failed to achieve response or
relapsed on treatment.

4.1.2.3 MDS patients should have failed prior therapy with a hypomethylating agent or, if
associated with a 5q- chromosomal abnormality, lenalidomide. Patients with 5q- unable to
receive or intolerant to lenalidomide are also eligible.

4.1.3 Patients must have clinically adequate hepatic and renal functions as defined by:

ALT<2x ULN Serum creatinine concentration <2x ULN Serum bilirubin <2x ULN 4.1.4 Patients
must sign an informed consent. 4.1.5 Women of childbearing age must have a negative serum
or urine pregnancy test prior to the initiation of study drug.

4.1.6 Barrier contraceptive precautions are to be used throughout the trial by all study
participants of child bearing potential.

4.1.7 Patients must be off anti-cancer therapy for at least two weeks prior to study
entry, with the exception of hydroxyurea or anagrelide (24 hours), TKI (5 days), and
interferon (two weeks).

4.1.8 Patients with ECOG Performance of 0-2

Exclusion Criteria:

4.2.1 Serious intercurrent medical illnesses, which would interfere with the ability of
the patient to carry out the treatment program.

4.2.2 Pregnant or breastfeeding women 4.2.3 Patients who have uncontrolled active
infection 4.2.4 Patients who have received another Investigational product within the
longer of 14 days or 5 half-lives of the previous product.

-

Type of Study:

Interventional

Study Design:

Endpoint Classification: Safety Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment

Outcome Measure:

Safety

Outcome Description:

Evaluate the patient tolerance to this cancer drug

Outcome Time Frame:

30 days

Safety Issue:

Yes

Principal Investigator

Jorge Cortes, MD

Investigator Role:

Study Chair

Investigator Affiliation:

M.D. Anderson Cancer Center

Authority:

United States: Food and Drug Administration

Study ID:

2003-0578 (v) 08-4

NCT ID:

NCT01159028

Start Date:

June 2010

Completion Date:

Related Keywords:

  • Safety and Pharmacokinetics of L-Grb-2 in Treating CML, AML, CLL and MDS
  • Liposomal Grb-2 treatment of CML
  • Precursor Cell Lymphoblastic Leukemia-Lymphoma
  • Leukemia, Myelogenous, Chronic, BCR-ABL Positive
  • Myelodysplastic Syndromes
  • Philadelphia Chromosome

Name

Location

M. D. Anderson Cancer CenterHouston, Texas  77030