MDACC Study Summary 2003-0578

Study Summary
No. 2003-0578:.......Leukemia......Jorge Cortes......Leukemia

Study Summary Title



Study Summary Number:	2003-0578

Study Title:	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

Physician

New Patient Referral



Name:	Jorge Cortes	Patients Call:	800-392-1611 (in U.S.A.) 713-792-6161 (outside U.S.A.)

Dept:	Leukemia	Referring MD Call:	800-392-1611 (in U.S.A.) 713-792-6161 (outside U.S.A.)

Phone:	713-794-5783 Contact us about clinical trials

General Information



Disease Group:	Leukemia	Supported By:	N/A

Phase of Study:	Phase I	Return Visit:	Patients must return to clinic twice weekly.

Treatment Agents:	L-Grb-2 Antisense Oligonucleotide	Home Care:	All treatment will be administered at M.D. Anderson Cancer Center in the Clinical and Translational Research Center.

Treatment Loc:	Only at MDACC

Estimated Length of Stay in Houston:	No hospitalizations are expected.

Description/
Intervention:

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 and other disease conditions described earlier. 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.

Study Objectives / Outcomes

To determine the toxicity and tolerance of escalating doses of BP-100-1.01.

To determine the maximum tolerated dose (MTD) of BP-100-1.01.

To determine the optimal biologically active dose (OBAD), defined as a 50% reduction in Grb-2 expression in circulating leukemia cells (flow cytometry).

To determine the in vivo pharmacokinetics of BP-100-1.01.

To evaluate tumor response.

To assess the pharmacokinetic data to correlate to the historical experience in order to demonstrate the liposomal delivery vehicle performs as expected.

Study Status Information



Study Activation / Registration Date:	06/21/2010

IRB Review and Approval Date:	06/21/2010

Study Type:

Recruitment Status:	Open

Projected Accrual:	N/A

Enrollment Eligibility

If you do not meet the enrollment eligibility, there may be other treatment options for you. Please Contact the Referral Office for more information.

Inclusion Criteria:

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

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: 1) blasts in peripheral blood or bone marrow >/=15%; 2) Promyelocytes and blasts in peripheral blood or bone marrow >/=30%; 3) PB or BM basophils >/=20%; 4) Thrombocytopenia < 100x10(3)/ml, not resulting from therapy.

3) **continued from above: Blast phase is defined as >/=30% blasts in peripheral blood or bone marrow, or presence of extramedullary disease, except for liver or spleen.

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

5) Patients must have clinically adequate hepatic and renal functions as defined by: ALT<2x ULN; Serum creatinine concentration <2x ULN; Serum bilirubin <2x ULN.

6) Patients must sign an informed consent.

7) Women of child bearing age must have a negative serum or urine pregnancy test prior to the initiation of study drug.

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

9) 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 (2 weeks).

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.

Links

Registration Number: NCT01159028
Study Information on Clinical Trials Registry (clinicaltrials.gov)