Clinical Trial of BP1001 (L-Grb-2 Antisense Oligonucleotide) in CML, AML, ALL & MDS

• Status: Completed
• Trial end date: 2017-03-30
• Target enrollment: 0
• Participant gender: All

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.

• Phase: Phase 1
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Bio-Path Holdings, Inc.

Criteria

Inclusion Criteria

1. Male or female patients 18 years of age or older

2. A diagnosis of refractory or relapsed AML, 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.

3. Patients with CML must have demonstrated resistance and/or intolerance to therapy with at least 2 tyrosine kinase inhibitors (TKI)

4. Patients with AML and ALL should have received at least 1 prior treatment regimen and either failed to achieve response or relapsed on treatment

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

6. Have clinically adequate hepatic and renal functions as defined by:

• ALT<2x ULN

• Serum creatinine concentration <2x ULN

• Serum bilirubin <2x ULN

7. Patients must sign an informed consent

8. Women of childbearing age must have a negative serum or urine pregnancy test prior to the initiation of study drug.

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

10. Have not received anti-cancer therapy for at least 2 weeks prior to study entry, with the exception of low dose ara-C (LDAC) given as subcutaneous injections (no less than 15 days prior), hydroxyurea or anagrelide (no less than 24 hours prior), TKI (no less than 5 days prior), and interferon (no less than 2 weeks prior)

11. Have an ECOG Performance of 0-2

12. Have a life-expectancy ≥3 months

Exclusion Criteria

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

2. Pregnant or breastfeeding women

3. Patients who have uncontrolled active infection

4. Patients who have received another investigational product within the longer of 14 days or 5 half-lives of the previous product

5. Any history of adverse reaction or hypersensitivity to LDAC

Part B: BP1001 with Concurrent LDAC Dose-Expansion Cohorts

Enrollment in the dose-expansion cohorts (DEC) will be limited to only those patients with a diagnosis of refractory or relapsed AML(except acute promyelocytic leukemia) or those who are refractory to at least 1 prior therapy regimen and no more than 1 prior salvage regimen.