Chronic neutropenia MedDRA version: 20.0 Level: LLT Classification code 10066702 Term: Chronic neutropenia System Organ Class: 100000013471
Conditions
Interventions
Product Name: ANF RhoTM (pegfilgrastim ANF)
Product Code: ANF RhoTM
Pharmaceutical Form: Solution for injection
INN or Proposed INN: PEGFILGRASTIM
CAS Number: 208265-92-3
Current Sponsor code: ANF-RHO
Sponsors
Prolong Pharmaceuticals, LLC
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: 1) Patients 12 years of age or older 2) Patients with established Chronic, cyclic or idiopathic severe neutropenia defined as Median ANC =65 years) yes F.1.3.1 Number of subjects for this age range 1
Exclusion criteria
Exclusion criteria: A subject will not be enrolled if they meet any of the following criteria: 1) Evidence of chromosomal abnormalities, myelodysplasia, hematologic malignancy, aplastic anemia, systemic lupus erythematosus, or rheumatoid arthritis (Felty’s syndrome) or if the neutropenia was drug-induced 2) Progressive malignant disease or malignancy history 3) Presence of macrophage activation syndrome before the diagnosis of neutropenia 4) Presence of a permanent proteinuria before the diagnosis of neutropenia 5) Clinical Significant Abnormal Renal, Cardiac, Hepatic Function or Blood Coagulation 6) Patient has a chronic infection such as hepatitis B virus(HBV), hepatitis C Virus (HCV) or Human immunodeficiency virus( HIV) or history of tuberculosis 7) Association with anemia <8 g/dL or thrombocytopenia (except iron deficiency anemia inflammatory except Shwachman syndrome) before the diagnosis of neutropenia 8) Drug abuse, substance abuse, or alcohol abuse 9) Use of any other investigational drug at the time of enrollment, or within 5 half-lives prior to enrollment, whichever is longer 10) Patients unwilling and/or who are not capable of ensuring compliance with the provisions of the study protocol 11) Pregnant or breastfeeding women where pregnancy is defined as the state of a female after conception and until the termination of gestation, confirmed by a positive serum hCG laboratory test 12) Women of childbearing potential, defined as all women physiologically capable of becoming pregnant, UNLESS they are using a highly effective method of birth control (i.e., one that results in a less than 1% per year failure rate when used consistently and correctly, such as implants, injectables, combined oral contraceptives and intrauterine devices (IUDs)). Periodic abstinence (e.g., calendar, ovulation, symptothermal, post ovulation methods) is not acceptable 13) For patients with glycogen storage disease and G6PC3 neutropenia, Pulmonary hypertension should be determined by ECHO and if positive patient is not eligible for the study
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: Primary: • To assess the response rate to ANF-Rho following a 6-month treatment period (induction and maintenance periods) in patients with Chronic Neutropenia o Complete response is defined as the achievement of Median ANC of =1.0 x 109/L over 6 months total treatment period o Partial response is defined as at least 100% increase in ANC relative to baseline and achievement of Median ANC = 0.5 x 109/L and ANC < 1 x 109/L over 6 months total treatment period ;Secondary Objective: Secondary: • To determine the pharmacokinetic profile of ANF-Rho in patients with Chronic Neutropenia (CN) • To determine the rate of subject reported infection-related morbidities (infections, hospitalizations and antibiotic use) in CN patients treated with ANF-Rho • To evaluate the effect of ANF-Rho on the Quality of Life and Bone Pain in CN patients Safety • To evaluate safety and tolerability of subcutaneous injections of ANF-Rho in CN patients;Primary end point(s): Primary Endpoint • Response rate to ANF-Rho following a 6-month treatment period in patients with Chronic Neutropenia o Complete response: Achievement of Median ANC of =1.0 x 109/L over 6-month total treatment period o Partial response: At least 100% increase in Median ANC relative to baseline ANC value and achievement of Median ANC =0.5 x 109/L and < 1 x 109/L over 6 months total treatment period;Timepoint(s) of evaluation of this end point: 6 months | — |
Secondary
| Measure | Time frame |
|---|---|
| Secondary end point(s): Secondary Endpoints • Pharmacokinetic profile as determined from blood plasma concentrations over time • Type, Incidence and duration of infection-related events, including hospitalization and use of antibiotic medications • Quality of life and bone pain measured by SF-36 and bone pain questionnaire Safety • Changes in vital signs, electrocardiographic assessments, clinical signs, and bio-analytical measures (e.g., blood chemistry, hematology), and reported adverse events;Timepoint(s) of evaluation of this end point: 6 months | — |
Countries
France
Contacts
Public ContactHEIDET-PIANELLI Véronique
CLINACT
Outcome results
None listed