MDACC Study Summary 2004-0702

Study Summary
No. 2004-0702:.......Leukemia......Jorge Cortes......Leukemia

Study Summary Title



Study Summary Number:	2004-0702

Study Title:	Phase I study of BAY 43-9006 (NSC 724772) in patients with acute leukemias, myelodysplastic syndromes and chronic myeloid leukemia in blast phase

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:	weekly

Treatment Agents:	BAY 43-9006	Home Care:	none

Treatment Loc:	Only at MDACC

Estimated Length of Stay in Houston:	n/a


Description/ Intervention:	The goal of this clinical research study is to find the highest tolerated dose of BAY 43-9006 that can be given to treat hematologic (blood-related) diseases. Researchers will also compare 2 different dose schedules to find out which one may be better tolerated. The effectiveness of BAY 43-9006 on leukemia cells will also be studied.

Study Objectives / Outcomes

Primary:

To define the maximum tolerated dose and dose limiting toxicity of two different schedules of BAY 43-9006 in patients with refractory or relapsed acute leukemias.

Secondary:

To investigate the clinical activity with two different schedules of administration of BAY 43-9006 in patients with refractory or relapsed acute leukemias or myelodysplastic syndromes.

To determine the biologic effect of BAY 43-9006 on leukemia cells of patients with refractory or relapsed acute leukemias and myelodysplastic syndromes.

Study Status Information



Study Activation / Registration Date:	10/03/2005

IRB Review and Approval Date:	05/04/2005

Study Type:	Therapeutic

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) Patients with any of the following disease categories will be eligible: acute myeloid leukemia (except APL), acute lymphoblastic leukemia, myelodysplastic syndrome, chronic myeloid leukemia in blast phase.

2) Patients must have failed prior therapy with at least one cytotoxic or biologic/targeted agent ( e.g., hypomethylating agents, farnesyl transferase inhibitors, tyrosine kinase inhibitors, thalidomide, etc) for leukemias or MDS, but there is no limit as to the maximum number of prior regimens as long as other inclusion criteria are met. Prior therapies may include bone marrow transplantation. In addition, patients with CML blast phase must have received and failed or be intolerant to imatinib.

3) Age >/= 18 years.

4) ECOG performance status 0-2.

5) Patients must have normal organ function as defined below: total bilirubin </= 1.5 mg/dl; ALT (SGPT) </= 2.5 x institutional upper limit of normal; Creatinine </= 2.0 mg/dl, or creatinine clearance >/= 60 mL/min/1.73m(2) for patients with creatinine levels above 2.0 mg/dl; Cytopenias secondary to multilineage bone marrow failure is acceptable.

6) The effects of BAY 43-9006 on the developing human fetus at the recommended therapeutic dose are unknown. For this reason and because kinase inhibitors are known to be teratogenic, women of child-bearing potential and men must agree to use adequate contraception (hormonal or barrier method of birth control; abstinence) prior to study entry and for the duration of study participation. Should a woman become pregnant or suspect she is pregnant while participating in this study, she should inform her treating physician immediately.

7) Ability to understand and willingness to sign a written informed consent document.

8) In the absence of rapidly progressing disease, the interval from prior treatment to time of study drug administration should be at least 2 weeks for cytotoxic agents or at least 5 half-lives for non-cytotoxic agents. If the patient is on hydrea to control peripheral blood leukemia cell counts, the patient must be off hydrea for at least 24 hours before initiation of treatment. Persistent chronic clinically significant toxicities from prior chemotherapy must not be > grade 1. The use of hydroxyurea is allowed up to 72 hours after the start of therapy with BAY43-9006.

Exclusion Criteria:

1) Patients with APL (unless they have failed therapy with both ATRA and arsenic alone or in combination).

2) Patients with absolute blast count > 20 x 10(9)/L (unless patient has documented FLT3 ITD; patients with FLT3 ITD can be included regardless of blast count).

3) Patients may not be receiving any other investigational agents.

4) History of allergic reaction attributed to compounds of similar chemical or biologic composition to BAY 43-9006.

5) Uncontrolled intercurrent illness including, but not limited to, active uncontrolled infection, symptomatic congestive heart failure (i.e., NY Heart Association class 3 or 4), uncontrolled hypertension (i.e., sustained systolic blood pressure >/= 150 or diastolic >/=90), unstable angina pectoris, symptomatic cardiac arrhythmia requiring and not responding to medical intervention, or psychiatric illness/social situations that would limit compliance with study requirements.

6) Pregnant women are excluded from this study because BAY 43-9006 is a kinase inhibitor agent with the potential for teratogenic or abortifacient effects. Because there is an unknown but potential risk for adverse events in nursing infants secondary to treatment of the mother with BAY 43-9006, breastfeeding should be discontinued if the mother is treated with BAY 43-9006.

7) Patients with immune deficiency are at increased risk of lethal infections when treated with marrow-suppressive therapy. Therefore, HIV-positive patients receiving combination anti-retroviral therapy are excluded from the study because of possible pharmacokinetic interactions with BAY 43-9006. Appropriate studies will be undertaken in patients receiving combination anti-retroviral therapy when indicated.

8) Patients who have a donor, are eligible, and have agreed to transplant.

9) Prior therapy with BAY 43-9006.

10) Therapeutic anticoagulation. Prophylactic anticoagulation, (i.e., low-dose warfarin, catheter flushing with heparin) of venous or arterial access devices is allowed.

11) Swallowing dysfunction that impedes oral ingestion of tablets.

12) Evidence of bleeding diathesis (except that explained by low platelets associated with the primary disease).

13) Patients must not be taking the cytochrome P450 enzyme-inducing antiepileptic drugs (phenytoin, carbamazepine, or Phenobarbital), rifampin, or St. Johns Wort.

Links

Registration Number: NCT00217646
Study Information on Clinical Trials Registry (clinicaltrials.gov)