Phase 2 Study Comparing Two Dose Schedules of Telintra™ in Myelodysplastic Syndrome (MDS)

Study Overview
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Brief Summary
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This is a randomized, open label, multicenter, Phase 2 study comparing two dose schedules of Telintra in patients with Low or Intermediate-1 risk MDS. Patients at least 18 years of age presenting with histologically confirmed Low to Intermediate-1 risk MDS with documented significant cytopenia for at least two months by the IWG criteria are eligible.

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

Conditions
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Myelodysplastic Syndrome (MDS)

Intervention / Treatment
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* Drug: Ezatiostat Hydrochloride


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: Primary or de novo MDS Low or intermediate-1 MDS ECOG performance status 0 or 1 Documented significant anemia with or without neutropenia and/or thrombocytopenia Adequate kidney and liver function Patients must have discontinued hematopoietic growth factors at least 3 weeks prior to study entry Exclusion Criteria: Prior allogenic bone marrow transplant for MDS History of MDS IPSS score 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 e.g. prednisone >10 mg per day History of active hepatitis B or C Known history of HIV

Ages Eligible for Study
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Minimum Age: 18 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: None (Open Label)


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: 1<br>Dose Schedule 1: Two weeks treatment with Telintra 3000 mg per day in two divided doses followed by one week with no treatment per three week cycle. | Drug: Ezatiostat Hydrochloride<br>* Dose Schedule 1-3000 mg orally per day in two divided doses for two weeks followed by 1 week with no treatment per three week cycle. Dose Schedule 2-2000 mg orally per day in two divided doses for three weeks followed by 1 week with no treatment per four week cycle.<br>* Other names: ezatiostat tablets;|
| Experimental: 2<br>Dose Schedule 2: Three weeks treatment with Telintra 2000 mg per day in two divided doses followed by one week with no treatment per four week cycle. | Drug: Ezatiostat Hydrochloride<br>* Dose Schedule 1-3000 mg orally per day in two divided doses for two weeks followed by 1 week with no treatment per three week cycle. Dose Schedule 2-2000 mg orally per day in two divided doses for three weeks followed by 1 week with no treatment per four week cycle.<br>* Other names: ezatiostat tablets;|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Hematologic Improvement-Erythroid (HI-E) rate |  | 24 Weeks | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Hematologic Improvement-Neutrophil (HI-N) rate, the Hematologic Improvement-Platelet (HI-P) rate, overall hematologic response rate, safety profile and Quality of Life assessments. |  | 24 Weeks | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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Hematology, MDS, Myelodysplastic Syndrome, Low Risk MDS, Intermediate-1 risk MDS, Int-1 risk MDS, Telintra, ezatiostat hydrochloride, ezatiostat, TLK199, Glutathione, Glutathione analog, Glutathione Transferase, Glutathione Transferase inhibitor, Glutathione Transferase P1-1 inhibitor, GSTp1-1 inhibitor, Apoptosis, Differentiation, Enzyme inhibitor