MDACC Study Summary 2006-0722

Study Summary
No. 2006-0722:.......Leukemia......Guillermo Garcia-Manero......Leukemia

Study Summary Title



Study Summary Number:	2006-0722

Study Title:	A Randomized, Double Blind, Placebo Controlled Study Evaluating the Efficacy and Safety of Romiplostim Treatment of Subjects with Low or Intermediate Risk Myelodysplastic Syndrome (MDS) Receiving Hypomethylating Agents

Physician

New Patient Referral



Name:	Guillermo Garcia-Manero	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:	713-745-3428 Contact us about clinical trials

General Information



Disease Group:	Leukemia	Supported By:	N/A

Phase of Study:	Phase II	Return Visit:	1 to 3 times weekly

Treatment Agents:	Romiplostim	Home Care:	N/A

Treatment Loc:	Independent Multicenter Arrangements

Estimated Length of Stay in Houston:	N/A


Description/ Intervention:	The goal of this clinical research study is to learn how well Romiplostim, given at different dose levels along with decitabine, can increase the number of platelets in the blood of patients with MDS.

Study Objectives / Outcomes

Primary
The primary objective of the study is to evaluate the effect of romiplostim on the
incidence of clinically-significant thrombocytopenia (grade 3 or 4 or receipt of platelet
transfusions) in subjects with low or intermediate risk MDS receiving a hypomethylating
agent.

Secondary

To evaluate the safety and tolerability of Romiplostim administration in combination with a hypomethylating agent.
To evaluate the proportion of patients receiving hypomethylating agent treatment at the prescribed dose and schedule.
To evaluate the proportion of patients achieving an overall response (CR + PR), complete (CR) or partial (PR) response at the end of the treatment period.
To evaluate the utilization rate of platelet transfusions.

Exploratory

To evaluate the effect of the combination of Romiplostim and hypomethylating agent treatment on therapeutic responses based on the modified International Working Group (IWG) criteria.
To evaluate changes in patient reported outcomes (PRO) assessments.
To evaluate the subject incidence of major or minor cytogenetic responses based on the modified IWG criteria.
To evaluate the effect of Romiplostim on bleeding events.
To evaluate long-term safety and tolerability of Romiplostim in low, intermediate-1 intermediate-2 risk MDS who have or are receiving concurrent treatment with hypomethylating agents.

Study Status Information



Study Activation / Registration Date:	01/23/2007

IRB Review and Approval Date:	09/20/2006

Study Type:	Phase Ii Or Phase I/Ii

Recruitment Status:	Closed

Projected Accrual:	60

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) Diagnosis of MDS by bone marrow biopsy based on the World Health Organization (WHO) classification

2) Low, Intermediate-1 or Intermediate-2 risk category MDS using the IPSS

3) Eastern Cooperative Oncology (ECOG) performance status of 0-2

4) Subjects must be greater than or equal to 18 years of age at the time of obtaining informed consent

5) Adequate Liver Function, as evidenced by a serum bilirubin less than or equal to 1.5 times the laboratory normal range (except for patients with a confirmed diagnosis of Gilbert's Disease) or an ALT or AST less than or equal to 3 times the laboratory normal range

6) A serum creatinine concentration less than or equal to 2 mg/dl

7) Part A only: Planned to receive azacitidine 75 mg/m^2 by subcutaneous administration each day for 7 days for at least 4 cycles from the time of enrollment

8) Part B only: Planned to receive decitabine according to the approved label or at a dose of 20 mg/m2 IV over 1 hour for 5 days every 4 weeks for at least 4 cycles from the time of enrollment

9) Before any study-specific procedure, the appropriate written informed consent must be obtained

Exclusion Criteria:

1) Part A only: Prior exposure to greater than 3 cycles of azacitidine

2) Part B only: Prior exposure to greater than 3 cycles of decitabine

3) Exposure to a hypomethylating agent within the last 30 days

4) Prior history of leukemia or aplastic anemia

5) Prior history of bone marrow transplantation

6) Prior malignancy (other than in situ cervical cancer, controlled prostate cancer, or basal cell cancer of the skin) unless treated with curative intent and without evidence of disease for greater than or equal to 3 years before randomization

7) Active or uncontrolled infections

8) Unstable angina, congestive heart failure [NYHA > class II], uncontrolled hypertension [diastolic >100 mmHg], uncontrolled cardiac arrhythmia, or recent (within 1 year) myocardial infarction

9) History of arterial thrombosis ( eg, stroke or transient ischemic attack) in the past year

10) History of venous thrombosis and currently requires anti-coagulation therapy

11) Received IL-11 within 4 weeks of screening

12) Less than 4 weeks since receipt of any therapeutic drug or device that is not FDA approved for any indication

13) Have previously received any other thrombopoietic growth factor

14) Subject is pregnant (eg, positive HCG test) or is breast-feeding.

15) Subject of reproductive potential who are not using adequate contraceptive precautions in the judgment of the investigator.

16) Subject has known hypersensitivity to any recombinant E coli-derived product (eg, Infergen�, Neupogen�, Somatropin, and Actimmune).

17) Subject previously has enrolled in this study

18) Subject will not be available for follow-up assessment.

19) Subject has any kind of disorder that compromises the ability of the subject to give written informed consent (and does not have a legally-acceptable representative) or subject is unable to comply with study procedures

Links

Registration Number: NCT00321711
Study Information on Clinical Trials Registry (clinicaltrials.gov)