B-cell acute lymphoblastic leukemia (B-ALL), which under WHO Guidelines is now referred to as precursor B-lymphoblastic leukemia/lymphoma MedDRA version: 8.1 Level: hlgt Classification code 10036523
Conditions
Interventions
Product Name: Forodesine Hydrochloride
Product Code: BCX-1777
Pharmaceutical Form: Intravenous infusion
Other descriptive name: Fodosine
Concentration unit: mg/ml milligram(s)/millilitre
Concentration
Sponsors
BioCryst Pharmaceuticals Inc.
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: • Documented B-cell acute lymphoblastic leukemia (B-ALL) • Must have failed at least 1 treatment regimen for B-ALL • Performance status of =2 by Eastern Cooperative Oncology Group (ECOG) criteria • Age 18 years or older • Life expectancy of at least 3 months • Adequate liver function (aspartate transaminase [AST] and/or alanine transaminase [ALT] not >3 times upper limits of normal [ULN]) • Adequate kidney function (calculated creatinine clearance >40 mL/min) • Negative serum or 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 (ICF) prior to start of any study-specific procedures Are the trial subjects under 18? no 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: • Active serious infection not controlled by oral or intravenous antibiotics • Treatment with any investigational antileukemic agent or chemotherapy agent in the last 7 days prior to study entry and lack of full recovery from side effects due to prior therapy independent of when that therapy was given • Rapidly progressive disease with compromised organ function judged to be life threatening by the Investigator • Patients with clinical evidence of active central nervous system (CNS) disease • Concurrent treatment with other anticancer agents • Pregnant and/or lactating female • Patients with known human immunodeficiency virus (HIV) infection • Patients with known active hepatitis B and/or hepatitis C infection • Hypersensitive or intolerant to any component of the study drug formulation
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: To determine the safety of repeat doses of intravenous (IV) infusion of forodesine in patients with B-ALL, which under WHO Guidelines is now referred to as precursor B-lymphoblastic leukemia/lymphoma.;Secondary Objective: • To describe the steady-state pharmacokinetics (PK) of forodesine following repeat administration • To evaluate the pharmacodynamic (PD) effects of forodesine 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 determine the efficacy of repeat doses of IV forodesine as evidenced by peripheral blood evaluations and bone-marrow evaluation • To evaluate the maintenance of response and safety of forodesine with extended treatment ;Primary end point(s): Safety: Reported adverse events presented by system organ class, preferred term, relationship to study medication and severity. Efficacy: Proportion of patients responding at Day 28. A responder is defined as a patient with either complete response, partial response or complete response in the absence of total platelet recovery. Responses to be confirmed by bone marrow evaluations. | — |
Countries
Germany
Outcome results
None listed