Clinical trial NCT01459159: Study of (Telintra®) in Non-Del(5q) Myelodysplastic Syndrome
Official title: Phase 2b Study of Oral Ezatiostat Hydrochloride (Telintra®) in Patients With Low to Intermediate-1 Risk, Non-Deletion 5q Myelodysplastic Syndrome
Study type: INTERVENTIONAL
Overall status: TERMINATED
Phase: PHASE2
Conditions: Myelodysplastic Syndrome (MDS)
Keywords: Hematology
MDS
Myelodysplastic Syndrome
Low risk MDS
Int-1 risk MDS
Transfusion dependence
Telintra
ezatiostat
ezatiostat hydrochloride
TLK199
Glutathione
Glutathione analog
Glutathione Transferase
Glutathione Transferase P1-1 inhibitor
GST P1-1 inhibitor
Apoptosis
Differentiation
Enzyme inhibitor
non-del (5q)
non-deletion 5q
Lead sponsor: Telik
Sponsor class: INDUSTRY
First posted: 2011-10-25
Enrollment: 162
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NON_RANDOMIZED
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

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).


Eligibility criteria: 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

Primary outcomes:
measure: Hematologic Improvement-Erythroid (HI-E) rate
timeFrame: At 8 weeks of treatment
measure: Hematologic Improvement-Erythroid (HI-E) rate
timeFrame: At 16 weeks of treatment
measure: Hematologic Improvement-Erythroid (HI-E) rate
timeFrame: At 24 weeks of treatment
measure: Hematologic Improvement-Erythroid (HI-E) rate
timeFrame: At 32 weeks of treatment
Secondary outcomes:
measure: RBC Transfusion independence (TI) rate
timeFrame: At 4, 8, 12, 16, 20, 24, 28 & 32 weeks of treatment
measure: Hematologic Improvement-Neutrophil (HI-N) rate
timeFrame: At 8, 16, 24, & 32 weeks of treatment
measure: Hematologic Improvement-Platelet (HI-P) rate
timeFrame: At 8, 16, 24, & 32 weeks of treatment
measure: Unilineage, bilineage, trilineage, and overall HI response rate
timeFrame: 2 years
measure: Cytogenetic response rate
timeFrame: 16 weeks, 48 weeks and at the time of first HI response
measure: Duration of response
timeFrame: 2 years
measure: Safety of ezatiostat in this MDS population
timeFrame: At 4, 8, 12, 16, 20, 24, 28 & 32 weeks of treatment
measure: Evaluation of the relationship between HI-E response, gene expression profiling and response-related variables
timeFrame: 2 years
Other outcomes:
Locations:
city: Concord
country: United States
facility: Bay Area Cancer Research Group
state: California
city: Aurora
country: United States
facility: University of Colorado
state: Colorado
city: Springfield
country: United States
facility: SIU School of Medicine, Simmons Cancer Institute
state: Illinois
city: Bethesda
country: United States
facility: Center for Cancer and Blood Disorders
state: Maryland
city: New York
country: United States
facility: Columbia University
state: New York
city: Memphis
country: United States
facility: The West Clinic
state: Tennessee
city: Nashville
country: United States
facility: Vanderbilt University
state: Tennessee
Study officials:
affiliation: Telik
name: Gail L Brown, MD
role: STUDY_DIRECTOR
References: