Last updated on January 2019

Clinical Trial of BP1001 (Liposomal Grb2 Antisense Oligonucleotide) in Combination With LDAC in Patients With Previously Untreated AML

Brief description of study

The primary objective of this study is to assess whether the combination of BP1001 and LDAC provides greater efficacy (Complete Remission [CR], Complete Remission with incomplete hematologic recovery [CRi], Complete Remission with incomplete platelet recovery [CRp]) than LDAC alone (by historical comparison) in participants with AML that cannot or elect not to be treated with more intensive chemotherapy. Safety, pharmacokinetics, overall survival, time to response, and duration of response will also be studied.

Detailed Study Description

The Grb2 gene has been mapped to the human chromosome region 17q22-qter, a region that is duplicated in leukemias and solid tumors, which may result in an increased copy number of the Grb2 gene product. As Grb2 is important for the transformation of murine hematopoietic cells, and the proliferation of human leukemia cells that express high levels of oncogenic tyrosine kinases, inhibition of Grb2 may have a significant impact on the natural history of leukemias. 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.

Low-dose cytarabine (Ara-C) (LDAC) has been used in a variety of schedules in several Phase II trials in AML showing responses that include complete remission of disease. It is generally well tolerated and can be given in an outpatient or home care setting. Researchers hope that the combination of BP1001 and LDAC will result in greater response rates and duration of response in participants with AML that cannot or elect not to be treated with more intensive chemotherapy.

This is a Phase IIa, multicenter, study of BP1001 in combination with LDAC in participants with previously untreated AML who are not otherwise eligible for standard or high-intensity chemotherapy regimens or who have elected a low-intensity regimen.

This trial will utilize a single arm, open label, two-stage design to assess the safety profile, PK, PD, and efficacy of 60 mg/m2 of BP1001 in combination with LDAC compared to historical response rates documented for LDAC alone.

Approximately 54 evaluable participants will receive the combination of BP1001 and LDAC.

Clinical Study Identifier: NCT02781883

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University of Kansas Cancer Center

Fairway, KS United States
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Recruitment Status: Open

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