Advanced T-cell Leukaemia (either precursor T-Lymphoblastic Leukaemia/Lymphoma or T-cell Prolymphocytic Leukaemia [T-PLL]). MedDRA version: 7.0 Level: hlgt Classification code 10024324
Conditions
Interventions
Product Name: Forodesine Hydrochloride
Product Code: BCX-1777
Pharmaceutical Form: Intravenous infusion
INN or Proposed INN: Forodesine Hydrochloride
Sponsors
BioCryst Pharmaceuticals Inc.
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: •Documented T-cell leukemia (percursor T-lymphoblastic leukaemia/lymphoma or T-cell prolymphocytic leukaemia [T-PLL]). •Failure to have responded to one or more standard regimens for their disease. •Performance status of 3 times upper limits of normal) •Adequate kidney function (calculated creatinine clearance >50 mL/min) •Negative urine pregnancy test within 2 to 7 days prior to the start of study treatment in females of childbearing potential •Females of childbearing potential and males must be willing and able to use an adequate method of contraception to avoid pregnancy for the duration of the study in such a manner that the risk of pregnancy is minimized •Signed informed consent form prior to start of any study specific procedures Are the trial subjects under 18? yes Number of subjects for this age range: F.1.2 Adults (18-64 years) yes F.1.2.1 Number of subjects for this age range F.1.3 Elderly (>=65 years) yes F.1.3.1 Number of subjects for this age range
Exclusion criteria
Exclusion criteria: •Patients with known human immunodeficiency virus (HIV) infection or human T-cell leukemia virus type 1 (HTLV-1) •Patients with known Hepatitis B and/or Hepatitis C active infection •Patients with active CMV infection •Tumor-related central nervous system (CNS) leukemia requiring active treatment •Active serious infection not controlled by oral or IV antibiotics •Treatment with any investigational antileukemic agent or chemotherapy agent within 7 days prior to study entry, unless full recovery from side effects has occurred •Rapidly progressive disease with compromised organ function judged to be life threatening by the Investigator •Concurrent treatment with other anticancer agents (corticosteroid use will not be excluded, but patient must remain on a stable dose) •Pregnant and/or lactating female •Cutaneous T-cell lymphoma (CTCL) diagnosis (including Sezary Syndrome)
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: To determine the sustained effectiveness (for a minimum of 28 days) of intravenous (IV) forodesine hydrochloride infusions in patients with advanced T-cell leukemia (precursor T-lymphoblastic leukemia/lymphoma or T-cell prolymphocytic leukemia [T-PLL]). ; Secondary Objective: •To assess the safety and tolerability of multiple infusion cycles of forodesine hydrochloride in this patient population •To evaluate the effects of forodesine hydrochloride on plasma levels of 2-deoxyguanosine (dGuo) and red blood cell (RBC) purine nucleoside phosphorylase (PNP) activity and to correlate levels with efficacy parameters •To describe the steady-state pharmacokinetics (PK) and pharmacodynamics (PD) of forodesine hydrochloride following repeat administration •To evaluate the maintenance of response and safety of forodesine hydrochloride (BCX-1777) in long-term treatment ; Primary end point(s): The proportion of patients with a sustained response will constitute the primary outcome measure for the trial. A responding patient is one with a partial or complete response. A patient with a sustained response is one responding to treatment for at least 28 days, beginning with the first laboratory measurement that shows a clinically significant response and concludes with the Cycle-6 (or Cycle-8) assessments. For precursor T-lymphoblastic leukaemia/lymphoma or T-PLL patients with circulating tumor cells, a sustained response will be based on the peripheral blood assessment; for patients who have no disease in the peripheral blood , a sustained response will be based on the bone marrow assessment. For patients with extramedullary disease, a sustained response will also be based on assessments made by additional | — |
Countries
Germany, Italy, United Kingdom
Outcome results
None listed