MDACC Study Summary 2008-0249

Study Summary
No. 2008-0249:.......Leukemia......Hagop Kantarjian......Leukemia

Study Summary Title



Study Summary Number:	2008-0249

Study Title:	A Randomized, Double Blind, Placebo Controlled Study Evaluating the Efficacy and Safety of Romiplostim Treatment of Thrombocytopenia in Subjects with Low or Intermediate-1 Risk Myelodysplastic Syndrome (MDS)

Physician

New Patient Referral



Name:	Hagop Kantarjian	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:	713-792-7026 Contact us about clinical trials

General Information



Disease Group:	Leukemia	Supported By:	Amgen, Inc.

Phase of Study:	Phase II	Return Visit:	Weekly

Treatment Agents:	Placebo Romiplostim	Home Care:	N/A

Treatment Loc:	Both at MDACC & outside MDACC at one or more Collaborating Sites or Institutions

Estimated Length of Stay in Houston:	N/A


Description/ Intervention:	The goal of this clinical research study is to learn if romiplostim can help to control thrombocytopenia (low blood cell counts) in patients with low or intermediate-1 risk MDS. The safety and effectiveness of this drug will also be studied.

Study Objectives / Outcomes

Primary
To evaluate the efficacy of romiplostim for the treatment of thrombocytopenia in subjects with international prognostic scoring system (IPSS) low or intermediate-1 risk MDS as measured by the number of clinically significant bleeding events.

Secondary Efficacy

To evaluate the number of platelet transfusions
To evaluate the overall number of bleeding events
To evaluate the utilization of platelet transfusions
To evaluate platelet hematologic improvement (HI-P) per MDS IWG 2006 guidelines
To evaluate overall survival
To evaluate the incidence of patient-reported bleeding

Secondary Safety

To evaluate the overall safety, as evidenced by adverse events and clinically significant changes in laboratory findings, of romiplostim in thrombocytopenic subjects with IPSS low or intermediate-1 risk MDS
To evaluate disease progression to acute myelogenous leukemia (AML)
To evaluate the development of antibodies to romiplostim and eTPO.

Study Status Information



Study Activation / Registration Date:	09/29/2008

IRB Review and Approval Date:	06/24/2008

Study Type:	Phase Ii Or Phase I/Ii

Recruitment Status:	Open

Projected Accrual:	240

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 using the WHO classification

2) Per MDS IPSS, Low or intermediate-1 risk MDS

3) The mean of the two platelet counts taken within 4 weeks prior to randomization must be: </= 20 x 10^9/L, with no individual count >30 x 10^9/L with or without a history of bleeding, OR </= 50 x 10^9/L, with no individual count > 60 x 10^9/L with a history of bleeding. A standard of care platelet count taken prior to Informed Consent may be used as 1 of the 2 counts taken within 4 weeks prior to randomization.

4) Subjects must be >/=18 and </= 90 years of age at the time of informed consent. Subjects between 85 and 90 years of age must have been diagnosed with MDS </= 5 years from study start.

5) Eastern Cooperative Oncology Group (ECOG) performance status of 0-2.

6) Adequate liver function, as evidenced by ALT </= 3 times the laboratory normal range, AST </= 3 times the laboratory normal range and total bilirubin </= 2.0 times the laboratory normal range (Adequate liver function for patients with a confirmed diagnosis of Gilbert's Disease evidenced by ALT </= 3 times the laboratory normal range, and AST </= 3 times the laboratory normal range).

7) A serum creatinine concentration </= 2 mg/dl (</=176.8 mu mol/L)

8) Bone marrow biopsy and aspirate with cytogenetics within 3 months of the first dose of investigational product.

9) Before any study-specific procedure, the appropriate written informed consent from the subject or his/her legally acceptable representative must be obtained.

Exclusion Criteria:

1) Have ever received any disease-modifying treatment for MDS, e.g. azacitidine, decitabine, intensive chemotherapy, allogeneic transplant.

2) Previously diagnosed with intermediate-2 or high risk MDS with IPSS.

3) Prior history of leukemia, aplastic anemia, or other non-MDS related bone marrow stem cell disorder.

4) Prior history of hematopoietic stem cell transplantation.

5) Persistent peripheral blood monocytosis (>/= 3 months with an absolute monocyte count >1,000/mu L).

6) Prior malignancy (other than in situ cervical cancer, non-melanoma skin cancer, or in situ carcinoma) unless treated with curative intent and without evidence of disease for >/= 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 that currently requires anti-coagulation therapy.

11) Pregnant or breast feeding.

12) Received IL-11 within 4 weeks of screening.

13) Have previously received any thrombopoietic growth factor.

14) Receipt of G-CSF, peg-G-CSF, or GM-CSF within 4 weeks of the first dose of investigational product.

15) Planned receipt of peg-G-CSF or GM-CSF after the first dose of investigational product.

16) Subject of reproductive potential who is not using adequate contraceptive precautions, in the judgement of the investigator. Amgen recommends double barrier contraception is used for all applicable patients enrolled on this study. A double barrier method is defined as two methods of contraception, for example 2 actual barrier methods, or one actual barrier method and one hormonal method.

17) Subject has known sensitivity to any E coli-derived product (eg, Infergen, Neupogen, Somatropin, and Actimmune).

18) Subject previously has entered this study.

19) Inability to comply with study procedures.

20) Subject currently is enrolled in or has not yet completed 30 days since ending other investigational device or drug study.

21) Patient should not be taking any anticoagulant therapy, except aspirin.

Links

Registration Number: NCT00614523
Study Information on Clinical Trials Registry (clinicaltrials.gov)