MDACC Study Summary 2010-0041

Study Summary
No. 2010-0041:.......Leukemia......Guillermo Garcia-Manero......Leukemia

Study Summary Title



Study Summary Number:	2010-0041

Study Title:	A multi-center, randomized, double-blind, placebo-controlled clinical trial of deferasirox in patients with myelodysplastic syndromes (low/int-1risk) and transfusional iron overload (TELESTO)

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:	Novartis Pharmaceuticals

Phase of Study:	Phase III	Return Visit:

Treatment Agents:	Deferasirox	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 study the effectiveness of the drug Exjade® (deferasirox) in controlling MDS. Researchers will also study the effect of the study drug/placebo on the heart, kidneys, and liver.

Study Objectives / Outcomes

The purpose of this study is to demonstrate in low/int-1 risk MDS patients, treated as per standard practice, the clinical superiority of deferasirox to placebo, while rigorously monitoring relevant clinical parameters (cardiac and liver function and transformation to AML) potentially affected by iron overload complications. The primary objective is to compare deferasirox to placebo with regard to event-free survival (a composite primary endpoint including death and non-fatal events related to cardiac and liver function) in low and int-1 risk MDS patients with transfusional iron overload.

The key secondary objective is to compare overall survival.

The other secondary objectives are to evaluate:

Change in endocrine function (thyroid and glycemic control)
Change in hematological function (MDS progression, progression to AML, and transfusion requirements)
Change in serum ferritin level
Change in cardiac function
Safety, in particular to assess the levels of increased risk for pre-specified adverse events (renal dysfunction, neutropenia, thrombocytopenia, gastrointestinal bleeding, and laboratory abnormalities) that would be clinically unacceptable in the context of the level of benefit that is likely to be provided by iron chelation using deferasirox in MDS patients with iron overload

Study Status Information



Study Activation / Registration Date:	09/02/2010

IRB Review and Approval Date:	09/02/2010

Study Type:	Phase Iii

Recruitment Status:	Open

Projected Accrual:	630

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) Male or female patients, greater than or equal to 18 years of age.

2) Patient must weigh between 35-135 kg

3) Patients with low or intermediate (int-1) risk MDS, as determined by IPSS score. This must be confirmed by a bone marrow examination within 6 months prior to study entry and must be hematologically stable.

4) Ferritin > 1000 mcg/L and at screening. Serum ferritin will be measured at Screening Visit 1 and Screening Visit 2 (at least 14 days apart) and the mean value will be used for eligibility criteria.

5) History of transfusion of 15 to 75 PRBC units.

6) Anticipated to be transfused with at least 8 units of PRBC annually during the study.

7) Sexually active pre-menopausal female patients must use double-barrier contraception, oral contraceptive plus barrier contraceptive, or must have undergone clinically documented total hysterectomy and/or oophorectomy, tubal ligation.

Exclusion Criteria:

1) More than 3 years since patient began receiving regular transfusions (2 units per 8 weeks or 4 units received in a 3 month period).

2) Creatinine Clearance <40 ml/min

3) Serum creatinine > 1.5 x ULN at screening

4) Significant proteinuria as indicated by a urinary protein/creatinine ratio > 0.5 mg/mg in a non-first void urine sample at Visit 1 or Visit 2 (or alternatively in two of three samples obtained for screening).

5) ECOG performance status > 2.

6) Left ventricular ejection fraction < 50% by echocardiography as per the central reading assessment.

7) A history of hospitalization for congestive heart failure.

8) Systemic diseases which would prevent study treatment (e.g.uncontrolled hypertension, cardiovascular, renal, hepatic, metabolic, etc.).

9) Clinical or laboratory evidence of active Hepatitis B or Hepatitis C (HBsAg in the absence of HBsAb OR HCV Ab positive with HCV RNA positive).

10) History of HIV positive test result (ELISA or Western blot).

11) Treatment with systemic investigational drug within 4 weeks or topical investigational drug within 7 days of study start.

12) ALT or AST > 3.5 × ULN at screening.

13) Total bilirubin > 1.5 x ULN at screening.

14) Diagnosis of liver cirrhosis (either established diagnosis or diagnosis by liver biopsy or central ultrasound reading).

15) Patients participating in another clinical trial other than an observational registry study.

16) Patients with a history of another malignancy within the past five years, with the exception of basal skin carcinoma or cervical carcinoma in situ or completely resected colonic polyps carcinoma in situ.

17) History of non-compliance to medical regimens, or patients who are considered potentially unreliable and/or not cooperative.

18) Presence of a surgical or medical condition which might significantly alter the absorption, distribution, metabolism or excretion of study drug.

19) Pregnant, intending-to-become pregnant, or breast-feeding patients.

20) History of drug or alcohol abuse within the 12 months prior to enrollment.

21) More than 6 months of cumulative iron chelation therapy (such as daily deferasirox (Exjade) or deferiprone or 5x/week deferoxamine

22) Intermittent deferoxamine doses in association with blood transfusions are not exclusionary regardless of duration of such treatment.

Links

Registration Number: NCT00940602
Study Information on Clinical Trials Registry (clinicaltrials.gov)