Clinical Trial of L-Grb-2 Antisense Oligonucleotide in CML, AML, ALL & MDS
Recruitment status was Recruiting
|First Received Date ICMJE||July 7, 2010|
|Last Updated Date||July 8, 2010|
|Start Date ICMJE||June 2010|
|Primary Completion Date||Not Provided|
|Current Primary Outcome Measures ICMJE
||Safety [ Time Frame: 30 days ] [ Designated as safety issue: Yes ]
Evaluate the patient tolerance to this cancer drug
|Original Primary Outcome Measures ICMJE||Same as current|
|Change History||Complete list of historical versions of study NCT01159028 on ClinicalTrials.gov Archive Site|
|Current Secondary Outcome Measures ICMJE
||Efficacy [ Time Frame: 30 days ] [ Designated as safety issue: No ]
Measure the reduction in leukemic blast cells
|Original Secondary Outcome Measures ICMJE||Same as current|
|Current Other Outcome Measures ICMJE||Not Provided|
|Original Other Outcome Measures ICMJE||Not Provided|
|Brief Title ICMJE||Clinical Trial of L-Grb-2 Antisense Oligonucleotide in CML, AML, ALL & MDS|
|Official Title ICMJE||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 goal of this clinical research study is to find the highest safe dose of a liposomal Growth Factor Receptor Bound Protein-2 antisense oligodeoxynucleotide (L-Grb2 AS) that can be given as treatment for patients with Philadelphia Chromosome positive CML, AML, CLL and MDS. The trade name of this drug is BP-100-1.01. The response of the leukemia to this treatment will also be studied. In addition, the time needed for the body to process this drug will be evaluated.
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.
|Study Type ICMJE||Interventional|
|Study Phase||Phase 1|
|Study Design ICMJE||Endpoint Classification: Safety Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
|Condition ICMJE||Safety and Pharmacokinetics of L-Grb-2 in Treating CML, AML, CLL and MDS|
|Intervention ICMJE||Drug: BP-100-1.01 (Liposomal Grb-2)
5 mg/meter squared, dose is constituted in normal saline, administered by IV. Treatment given twice a week for four weeks.
|Study Arm (s)||Not Provided|
|Publications *||Not Provided|
* Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
|Recruitment Status ICMJE||Recruiting|
|Estimated Enrollment ICMJE||18|
|Completion Date||Not Provided|
|Primary Completion Date||Not Provided|
|Eligibility Criteria ICMJE||
One of the following parameters is required to meet criteria for accelerated phase CML:
Blast phase is defined as ≥30% blasts in peripheral blood or bone marrow, or presence of extramedullary disease, except for liver or spleen.
188.8.131.52 Patients with CML must have demonstrated resistance and/or intolerance to therapy with at least 2 tyrosine kinase inhibitors (TKI) 184.108.40.206 AML and Ph+ ALL should have received at least one prior treatment regimen and either failed to achieve response or relapsed on treatment.
220.127.116.11 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
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.
|Ages||18 Years to 70 Years|
|Accepts Healthy Volunteers||No|
|Location Countries ICMJE||United States|
|NCT Number ICMJE||NCT01159028|
|Other Study ID Numbers ICMJE||2003-0578 (v) 08-4|
|Has Data Monitoring Committee||Yes|
|Responsible Party||Peter Nielsen, President & CEO, Bio-Path Holdings, Inc.|
|Study Sponsor ICMJE||Bio-Path Holdings, Inc.|
|Collaborators ICMJE||Not Provided|
|Information Provided By||Bio-Path Holdings, Inc.|
|Verification Date||April 2010|
ICMJE Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP