MDACC Study Summary 2011-0253

Study Summary
No. 2011-0253:.......Leukemia......Stefan Faderl......Leukemia

Study Summary Title



Study Summary Number:	2011-0253

Study Title:	A Phase I Clinical Study of CWP232291 in Patients with Relapsed or Refractory Acute Myeloid Leukemia (AML) or Chronic Myelomonocytic Leukemia-2 (CMML-2), Myelodysplastic Syndrome Having Failed Hypomethylating Treatment (MDS), and High-Risk Myelofibrosis (MF)

Physician

New Patient Referral



Name:	Stefan Faderl	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:	713-745-4613 Contact us about clinical trials

General Information



Disease Group:	Leukemia	Supported By:	JW Pharmaceutical Corporation

Phase of Study:	Phase I	Return Visit:

Treatment Agents:	CWP232291	Home Care:

Treatment Loc:	Both at MDACC & and Other Sites

Estimated Length of Stay in Houston:

Description/
Intervention:

The goal of this clinical research study is to learn the highest tolerable dose
of the drug CWP232291 that can be given to patients with relapsed or refractory
AML CMML-2, MDS, or MF disease. The safety of CWP232291 will also be studied to
understand the best way to give the drug and also see what the best dose will
be for patients with hematologic disease.

CWP232291 is designed to target and block the function of messenger molecules
in cancer cells. This may slow the growth of tumors or kill the cancer cells.
This is the first study using CWP232291 in humans.

Study Objectives / Outcomes

Primary Objective:
The primary objective of this study is to determine the MTD of CWP232291 in patients with advanced myeloid neoplasms, when administered by intravenous (IV) bolus injection daily for 7 days, followed by 2 weeks without study drug.

Secondary Objectives:
· To characterize the safety profile of CWP232291
· To determine the plasma pharmacokinetic (PK) profile of CWP232291 and CWP232204
· To assess the preliminary antileukemic activity (efficacy) of CWP232291 in patients with AML, CMML-2, MDS and MF.
To explore biomarkers of CWP232291, including survivin and beta-catenin

Study Status Information



Study Activation / Registration Date:

IRB Review and Approval Date:	10/18/2011

Study Type:	Phase I

Recruitment Status:	Open

Projected Accrual:	36 - 48

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) Able to understand and willing to sign an informed consent form (ICF) prior to initiation of any study-specific procedure and treatment

2) Must be 18 years of age or older.

3) A pathologically confirmed diagnosis of AML or CMML-2 by World Health Organization (WHO) classification that is relapsed or refractory or for which no current therapies are anticipated to result in a durable remission, or MDS by WHO classification that are RAEB-1 or RAEB-2 and that have failed at least three cycles of hypomethylating therapy, or primary (PMF), post-polycythemia vera (PPMF) or post-essential thrombocythemia (PTMF) MF by WHO classification are high-risk category by the Dynamic International Prognostic Scoring System (DIPSS Plus), have >/=1% circulating blasts, and have failed treatment with ruxolitinib.

4) Eastern Cooperative Oncology Group (ECOG) performance score 0-2.

5) 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 noncytotoxic agents.If a patient is on hydroxyurea to control peripheral blood leukemic cell counts, the patients must have discontinued hydroxyurea for at least 24 hours before initiation of treatment with study drug. Persistent clinically significant toxicities from prior chemotherapy must not be greater than grade 1.

6) Adequate renal function: Serum creatinine </= 2.0 mg/dL

7) Adequate hepatic function: Total bilirubin <1.5 x upper limit of normal (ULN), unless considered due to Gilbert's syndrome. Alkaline phosphatase (AP) </=2.5 x ULN, unless considered due to organ involvement of target disease. Aspartate transaminase (AST) or alanine transaminase (ALT) </=3 x ULN, unless considered due to organ involvement of target disease.

8) Women of child-bearing potential (i.e., women who are pre-menopausal or not surgically sterile) must use acceptable contraceptive methods (abstinence, intrauterine device [IUD], oral contraceptive, or double barrier device), and must have a negative serum or urine pregnancy test within 2 weeks prior to beginning treatment on this trial. Sexually active men must also use acceptable contraceptive methods for the duration of time on study.

9) Able to adhere to the study visit schedule and other protocol requirements.

Exclusion Criteria:

1) Uncontrolled intercurrent illness including, but not limited to, uncontrolled infection, symptomatic congestive heart failure (CHF), cardiac arrhythmia, or psychiatric illness/social situation that would limit compliance with study requirements.

2) Active heart disease including myocardial infarction (MI) within previous 3 months, symptomatic coronary artery disease (CAD), arrhythmias not controlled by medication, or uncontrolled CHF.

3) Active central nervous system (CNS) disease.

4) Therapy with any other standard or investigational treatment for hematologic malignancy (except hydroxyurea, as mentioned in the inclusion criteria).

5) Therapy with anticoagulant or antithrombotic agents (including aspirin) within 7 days prior to study drug administration.

6) History of gastrointestinal (GI) hemorrhage.

7) Known positive status for human immunodeficiency virus (HIV) and/or active hepatitis B or C.

8) Pregnant or nursing women. Pregnant and nursing patients are excluded because the effects of CWP232991 on a fetus or nursing child are unknown.

9) Patients eligible for bone marrow transplant, regardless of age.

10) Patients with FLT3 ITD positive AML or AML patients with other cytogenetic abnormalities who are eligible for trials of other targeted investigational agents from which the investigator feels there is greater benefit.

Links

Registration Number: NCT01398462
Study Information on Clinical Trials Registry (clinicaltrials.gov)