MDACC Study Summary 2008-0081

Study Summary
No. 2008-0081:.......Leukemia......Stefan Faderl......Leukemia

Study Summary Title



Study Summary Number:	2008-0081

Study Title:	Phase 2 Randomized Study Comparing Two Dose Schedules of Ezatiostat Hydrochloride (Telintra™, TLK199 Tablets) in Low to Intermediate-1 Risk Myelodysplastic Syndrome (MDS)

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:	Telik, Inc.

Phase of Study:	Phase II	Return Visit:	Outpatient treatment visits are scheduled on days 1 and 22 (29) followed by every 21 (28) days +/- 3 days thereafter for patients receiving Telintra on dose schedule 1 (dose schedule 2).

Treatment Agents:	TLK199	Home Care:	Follow up labs can be done at home. Patients will keep a diary while on therapy.

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

Estimated Length of Stay in Houston:	No hospitalizations should be required as this in an oral treatment.


Description/ Intervention:	The goal of this clinical research is to compare 2 different dose schedules of Telintra (ezatiostat hydrochloride) to learn if the study drug can help to control MDS. The safety of this drug will also be studied.

Study Objectives / Outcomes

Primary:

To evaluate and compare the Hematologic Improvement-Erythroid (HI-E) rate.

Secondary:

To evaluate and compare the Hematologic Improvement-Neutrophil (HI-N) rate.
To evaluate and compare the Hematologic Improvement-Platelet (HI-P) rate.
To evaluate and compare the unilineage, bilineage, trilineage, and overall hematologic response rate.
To evaluate the safety profile of each treatment arm.
To evaluate and compare the Functional Assessment of Cancer Therapy-Anemia (FACT-An).

Study Status Information



Study Activation / Registration Date:	04/01/2009

IRB Review and Approval Date:	05/29/2008

Study Type:	Phase Ii Or Phase I/Ii

Recruitment Status:	Closed

Projected Accrual:	86

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) Histologically confirmed diagnosis of primary or de novo MDS using the World Health Organization (WHO) classification.

2) Low or intermediate-1 risk MDS using the International Prognostic Scoring System (IPSS).

3) World Health Organization (WHO) classification types of: refractory anemia (RA), refractory anemia with excess of blasts (RAEB-1), refractory anemia with ringed sideroblasts (RARS), refractory cytopenia with multilineage dysplasia (RCMD), refractory cytopenia with multilineage dysplasia with ringed sideroblasts (RCMD-RS), MDS-Unclassified (MDS-U), MDS/Myeloproliferative disorder-unclassified (MDS/MPD-U) without leukocytosis, and MDS with deletion of chromosome 5q (MDS-del5q).

4) >/= 18 years of age.

5) Eastern Cooperative Oncology Group (ECOG) performance status of 0 or 1.

6) Prior treatment with experimental or conventional treatment other than blood products (i.e., chemotherapeutic, immunosuppressive, cytoprotective or demethylating agents), at least a four-week (28 days) washout period is required.

7) Documented significant cytopenia for at least two months, defined by a) or b) and in addition, may have c) and/or d): a) Red blood cell (RBC) transfusion dependence is defined as requiring four or more units within an eight-week period for symptomatic anemia with a hemoglobin value of </= 9 g/dL, or b) baseline hemoglobin < 10 g/dL (defined as an average of at least two baseline values not influenced by RBC transfusions for at least seven days prior to study entry) and in addition may have,

8) (cont'd from 7) c) Thrombocytopenia with an average of two or more platelet counts </= 50,000/mcl (not influenced by platelet transfusions for at least 3 days) or a clinically significant hemorrhage requiring platelet transfusions, and/or d) absolute neutrophil count (ANC) </= 1,000/mcl, or a prior infection requiring intravenous (IV) antibiotics.

9) Adequate organ function as indicated by the following parameters: a) serum bilirubin </= 2.5 mg/dL (except when Gilbert's syndrome or MDS-related hemolysis is clearly documented and other liver function tests are normal); b) AST and ALT </= 3 times the upper limit of normal (ULN); c) serum creatinine </= 2 mg/dL or a calculated creatinine clearance of >/= 60 mL/min; blood urea nitrogen (BUN) </= 25 mg/100 mL.

10) Patients must have discontinued hematopoietic growth factors: erythropoietin, granulocyte colony stimulating factors (G-CSF), or granulocyte macrophage colony stimulating factors (GM-CSF) at least three weeks (21 days) prior to study entry.

11) Ineligible or unwilling to undergo stem cell bone marrow transplantation.

12) Women of childbearing potential and men capable of reproduction willing to practice effective method of birth control.

13) Written informed consent.

14) Willingness to comply with study procedures and follow-up.

Exclusion Criteria:

1) Prior allogeneic bone marrow transplant for MDS.

2) Known hypersensitivity to ezatiostat hydrochloride (Telintra, TLK199 Product) intravenous (IV) or ezatiostat hydrochloride (Telintra, TLK199 Tablets) oral (PO).

3) A history of prior malignancy, except for adequately treated carcinoma in situ of uterine cervix, basal cell or squamous cell skin cancer, or other cancers (e.g., breast, prostate) for which the patient has been disease-free for at least three years.

4) Myelodysplastic syndrome evolving from a pre-existing myeloproliferative disorder or autoimmune disease, or secondary to prior treatment with radiation or chemotherapy.

5) History of MDS IPSS score > 1.

6) Pregnant or lactating women.

7) Leptomeningeal or leukemic meningitis.

8) Any severe concurrent disease, infection, or comorbidity that, in the judgment of the Investigator, would make the patient inappropriate for study entry.

9) Psychiatric disorders that would preclude obtaining informed consent or the ability to participate in an ongoing research study.

10) Oral steroids (prednisone or equivalent) > 10 mg daily (concomitant steroids or hormones as related treatment for MDS or neoplasms are not allowed). Exceptions: Steroids or hormones at </= 10 mg daily for other conditions (e.g., new adrenal failure, asthma or arthritis) are allowed.

11) Active infection requiring therapy with IV antibiotics or antifungal therapy. Prior or concurrent history of one or more opportunistic infections (e.g., cytomegalovirus, pneumocystis carinii, aspergillosis, histoplasmosis, or mycobacteria other than TB) within the past six months.

12) History of documented active Hepatitis C (HCV).

13) History of documented active Hepatitis B (HBV).

14) Known history of HIV positivity (regardless of immune status).

15) Clinically significant bleeding within two weeks prior to study entry (e.g., GI bleeding, intracranial hemorrhage).

Links

Registration Number: NCT00700206
Study Information on Clinical Trials Registry (clinicaltrials.gov)