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

Phase 2

Terminated

• Conditions: Myelodysplastic Syndrome (MDS)

• Registration Number: NCT01459159
• Lead Sponsor: Telik

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).

Detailed Description

Not available

Study Timeline

• Study Start: 2011-10
• Study First Submitted: 2011-10-11
• Study First Submitted QC: 2011-10-21
• Study First Posted: 2011-10-25
• Primary Completion: 2013-02
• Study Completion: 2013-02
• Status Verified: 2013-11
• Last Update Submitted: 2013-11-20
• Last Update Posted: 2013-11-25

Recruitment & Eligibility

• Status: TERMINATED
• Sex: All
• Target Recruitment: 162

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

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Primary Outcome Measures

Name	Time	Method
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

Name	Time	Method
Duration of response	2 years
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)
Evaluation of the relationship between HI-E response, gene expression profiling and response-related variables	2 years
Unilineage, bilineage, trilineage, and overall HI response rate	2 years
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)
RBC Transfusion independence (TI) rate	At 4, 8, 12, 16, 20, 24, 28 & 32 weeks of treatment
Cytogenetic response rate	16 weeks, 48 weeks and at the time of first HI response
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

Trial Locations

Locations (7)

Bay Area Cancer Research Group; 🇺🇸 Concord, California, United States

Center for Cancer and Blood Disorders; 🇺🇸 Bethesda, Maryland, United States

University of Colorado; 🇺🇸 Aurora, Colorado, United States

The West Clinic; 🇺🇸 Memphis, Tennessee, United States

Columbia University; 🇺🇸 New York, New York, United States

Vanderbilt University; 🇺🇸 Nashville, Tennessee, United States

SIU School of Medicine, Simmons Cancer Institute; 🇺🇸 Springfield, Illinois, United States