Oral Forodesine Hydrochloride (BCX-1777) in Patients With Recurrent or Refractory T/NK-cell Malignancies

Study Overview
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Brief Summary
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Primary objectives are to evaluate the safety profile and tolerability of oral BCX1777 in each cohort of patients with recurrent or refractory T/NK-cell malignancies and to evaluate pharmacokinetics (PK) of oral BCX1777.

Official Title
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A Phase I, Open Label, Dose Ascending Study of BCX1777 (Oral) in Patients With Recurrent or Refractory T/NK-cell Malignancies

Conditions
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Recurrent or Refractory T/NK-cell Malignancies

Intervention / Treatment
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* Drug: forodesine hydrochloride
* Drug: forodesine hydrochloride
* Drug: forodesine hydrochloride
* Drug: forodesine hydrochloride


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: Histologically documented with histopathological diagnosis from biopsy, T/NK-cell malignancies (WHO classification). Precursor T-cell lymphoblastic leukemia/lymphoma (T-ALL/T-LBL) should be excluded in this study A patient with recurrent, relapse or refractory T/NK-cell malignancies who has received at lease one chemotherapeutic regimen Age 20 or greater Eastern Cooperative Oncology Group(ECOG) performance status:0,1. Able to be hospitalized at least for 15 days from the first dose In the case of subject with tumor cell rate of ≤25% in bone marrow, a patient who satisfies both of the following criteria. Neutrophil count: ≥1,200/mm3 Platelet count: ≥75,000/mm3 In the case of CTCL, the tumor cell rate is handled as ≤25%. Adequate Liver function: AST, ALT ≤ 5.0 x upper limit of normal (ULN) Adequate Renal function: Creatinine Clearance (using cockcroft-Gault formula) ≥ 50mL/min Life expectancy of at least 3 months A patient who has given a written informed consent prior to the start of procedures proper to this study. Exclusion Criteria: -

Ages Eligible for Study
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Minimum Age: 20 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
Intervention Model: Single Group Assignment
Masking: None (Open Label)


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: BCX1777<br> | Drug: forodesine hydrochloride<br>* Cohort 1: 100mg / body (1 x 100mg tablet once daily)<br>Drug: forodesine hydrochloride<br>* Cohort 2: 200mg / body (2 x 100mg tablets once daily)<br>Drug: forodesine hydrochloride<br>* Cohort 3: 300mg / body (3 x 100mg tablets once daily)<br>Drug: forodesine hydrochloride<br>* Cohort 4: 400mg / body (4 x 100mg tablets once daily)<br>|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| To evaluate the safety profile and tolerability of oral BCX1777 in patients with recurrent or refractory T/ NK-cell malignancies. |  | March 2010 | 
| To evaluate pharmacokinetics (PK) of oral BCX1777 |  | March 2010 | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| To evaluate pharmacodynamics (PD) of oral BCX1777 |  | March 2010 | 
| To correlate the plasma level of BCX1777 with plasma 2'-deoxyguanosine (dGuo) |  | March 2010 | 
| To evaluate the efficacy of oral BCX1777 |  | March 2010 | 
| To analyze cell surface marker in peripheral blood mononuclear cell |  | March 2010 |