Study of Ezatiostat (Telintra Tablets) for Treatment of Severe Chronic Neutropenia

Study Overview
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Brief Summary
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This is a multicenter Phase 2 randomized parallel-group study to determine the effect of Telintra treatment on severe chronic neutropenia. Patients will be randomized to Telintra or enter an observation period with an option to crossover to Telintra treatment in a 1:1 allocation.

Official Title
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Phase 2 Randomized Study of Ezatiostat Hydrochloride (Telintra™, TLK199 Tablets) for Treatment of Severe Chronic Neutropenia

Conditions
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Severe Chronic Neutropenia

Intervention / Treatment
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* Drug: Ezatiostat Hydrochloride


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: Confirmed Idiopathic Severe Chronic Neutropenia ECOG performance status of 0-2 Adequate liver and renal function Adequate Red Blood Cell and Platelet counts Exclusion Criteria: Prior treatment of SCN Non-Idiopathic types of SCN, ie. cyclic, congenital History of chromosomal abnormalities, myelodysplasia, hematologic malignancy, aplastic anemia, systemic lupus erythematosus, rheumatoid arthritis (Felty's syndrome), or other collagen diseases, and drug-induced neutropenia, autoimmune neutropenia Use of granulocyte colony stimulating factors (G-CSF), glucocorticoids, gamma globulin, lithium or investigational drug(s) within one month of enrollment History of bone marrow transplantation or stem cell support

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>4-Week dose equilibration period with Telintra followed by 4 month treatment period | Drug: Ezatiostat Hydrochloride<br>* Starting Dose 2000 mg orally per day in two divided doses with dose to increase or decrease to achieve target median ANC (Range 1,500-10,000 cells/uL)<br>* Other names: TLK199 Tablets;|
| No Intervention: 2<br>4 Month observation period with standard of care treatment and option to crossover to Telintra treatment for 4 week dose equilibration followed by 4 week treatment period | |

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Objective absolute neutrophil count (ANC) response rate |  | 18 Months | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Incidence of infections, oropharyngeal ulcers and antibiotic use |  | 18 Months | 
| Incidence and duration of hospitalizations |  | 18 Months | 
| FACT-N quality of life assessment |  | 18 Months | 
| Safety assessments |  | 18 Months | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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Hematology, Neutropenia, SCN, Severe Chronic Neutropenia, Idiopathic, 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