Study of (Telintra®) in Non-Del(5q) Myelodysplastic Syndrome

Sponsor

Telik (Industry)

Overall Status

Terminated

CT.gov ID

NCT01459159

Collaborator

(none)

162

Enrollment

Locations

16.1

Duration (Months)

23.1

Patients Per Site

1.4

Patients Per Site Per Month

Study Details

Study Description

Brief Summary

This is a multicenter, single arm open label Phase 2b Study of oral ezatiostat (Telintra®) in Patients who are RBC tranfusion dependent, Low to INT-1 IPSS risk, non-del (5q) Myelodysplastic Syndrome (MDS).

Condition or Disease	Intervention/Treatment	Phase
Myelodysplastic Syndrome (MDS)	Drug: ezatiostat hydrochloride (Telintra®)	Phase 2

Study Design

Study Type:

Interventional

Anticipated Enrollment :

162 participants

Allocation:

Non-Randomized

Intervention Model:

Single Group Assignment

Masking:

None (Open Label)

Primary Purpose:

Treatment

Official Title:

Phase 2b Study of Oral Ezatiostat Hydrochloride (Telintra®) in Patients With Low to Intermediate-1 Risk, Non-Deletion 5q Myelodysplastic Syndrome

Study Start Date :

Oct 1, 2011

Actual Primary Completion Date :

Feb 1, 2013

Actual Study Completion Date :

Feb 1, 2013

Outcome Measures

Primary Outcome Measures

Hematologic Improvement-Erythroid (HI-E) rate [At 8 weeks of treatment]
Hematologic Improvement response will be assessed per the IWG MDS response criteria (2006)
Hematologic Improvement-Erythroid (HI-E) rate [At 16 weeks of treatment]
Hematologic Improvement response will be assessed per the IWG MDS response criteria (2006)
Hematologic Improvement-Erythroid (HI-E) rate [At 24 weeks of treatment]
Hematologic Improvement response will be assessed per the IWG MDS response criteria (2006)
Hematologic Improvement-Erythroid (HI-E) rate [At 32 weeks of treatment]
Hematologic Improvement response will be assessed per the IWG MDS response criteria (2006)

Secondary Outcome Measures

RBC Transfusion independence (TI) rate [At 4, 8, 12, 16, 20, 24, 28 & 32 weeks of treatment]
Hematologic Improvement-Neutrophil (HI-N) rate [At 8, 16, 24, & 32 weeks of treatment]
Hematologic Improvement response will be assessed per the IWG MDS response criteria (2006)
Hematologic Improvement-Platelet (HI-P) rate [At 8, 16, 24, & 32 weeks of treatment]
Hematologic Improvement response will be assessed per the IWG MDS response criteria (2006)
Unilineage, bilineage, trilineage, and overall HI response rate [2 years]
Cytogenetic response rate [16 weeks, 48 weeks and at the time of first HI response]
Duration of response [2 years]
Safety of ezatiostat in this MDS population [At 4, 8, 12, 16, 20, 24, 28 & 32 weeks of treatment]
Recording and grading of AEs using NCI-CTCAE v4.03
Evaluation of the relationship between HI-E response, gene expression profiling and response-related variables [2 years]

Eligibility Criteria

Criteria

Ages Eligible for Study:

18 Years and Older

Sexes Eligible for Study:

All

Accepts Healthy Volunteers:

Inclusion Criteria:

Primary or de Novo MDS
Low to Intermediate-1 IPSS risk of MDS
ECOG performance score of 0 or 1
Documentation of significant anemia with or without additional cytopenia
Adequate kidney and liver function
Patients must have discontinued hematopoietic growth factors at least 3 weeks prior to study entry

Exclusion Criteria:

Deletion of the 5q chromosome [del(5q) MDS]
Prior allogenic bone marrow transplant for MDS
Known sensitivity to ezatiostat (injection or oral tablets)
Prior treatment with hypomethylating agent (HMA) (e.g., azacitadine, decitabine)
History of MDS IPSS risk score of greater than 1.0
Pregnant or lactating women
Any severe concurrent disease, infection or comorbidity that, in the judgement of the investigator, would make the patient inappropriate for study entry
Oral steroids greater than 10 mg per day. Exceptions: those prescribed for other conditions (such as new adrenal failure, asthma, arthritis) or brief sterioid use (such as tapered dosing for an acute non-MDS condition)
History of hepatitis B or C, or HIV

Contacts and Locations

Locations

	Site	City	State	Country	Postal Code
1	Bay Area Cancer Research Group	Concord	California	United States	94520
2	University of Colorado	Aurora	Colorado	United States	80045
3	SIU School of Medicine, Simmons Cancer Institute	Springfield	Illinois	United States	62794
4	Center for Cancer and Blood Disorders	Bethesda	Maryland	United States	20817
5	Columbia University	New York	New York	United States	10032
6	The West Clinic	Memphis	Tennessee	United States	38120
7	Vanderbilt University	Nashville	Tennessee	United States	37232