Severe chronic neutropenia (i.e. congenital neutropenia, cyclic neutropenia and chronic idiopathic neutropenia) MedDRA version: 14.1 Level: PT Classification code 10051645 Term: Idiopathic neutropenia System Organ Class: 10005329 - Blood and lymphatic system disorders MedDRA version: 14.1 Level: LLT Classification code 10069819 Term: Congenital neutropenia System Organ Class: 10010331 - Congenital, familial and genetic disorders MedDRA version: 14.1 Level: PT Classification code 10053176 Term
Conditions
Interventions
Trade Name: Filgrastim Hexal
Pharmaceutical Form: Solution for injection/infusion in pre-filled syringe
INN or Proposed INN: FILGRASTIM
CAS Number: 121181-53-1
Concentration unit: µg microgram(s)
Conc
Sponsors
Sandoz GmbH
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: 1. Patients with established congenital, cyclic or idiopathic severe chronic neutropenia having an indication for treatment with Sandoz’ filgrastim according to the SmPC of the product 2. Patients of any age at the day of inclusion 3. Written informed consent of patient and/or patient's legally authorized representative before any assessment is performed, applicable Are the trial subjects under 18? yes Number of subjects for this age range: 15 F.1.2 Adults (18-64 years) yes F.1.2.1 Number of subjects for this age range 15 F.1.3 Elderly (>=65 years) yes F.1.3.1 Number of subjects for this age range 0
Exclusion criteria
Exclusion criteria: 1. Chemotherapy-induced neutropenia 2. Neutropenia in combination with confirmed diagnosis of autoimmune disease, e.g. rheumatoid arthritis, Felty’s syndrome, or systemic lupus erythematosus 3. Myelodysplastic syndrome or leukemia 4. Thrombocytopenia (platelets < 50.000/mm3) or anemia (hemoglobin < 8 g/dl) with the exception of patients with Shwachman-Diamond syndrome, glycogen storage disease 1b, or Barth's syndrome 5. Sickle cell disease 6. History of malignancy of any organ system, treated or untreated, with the exception of localized basal cell carcinoma of the skin 7. For patients with congenital severe chronic neutropenia only: Any cytogenetic aberrations in bone marrow aspirates with results not older than six months suspicious for malignant transformation 8. Known or suspected hypersensitivity to rhG-CSF products 9. Known or suspected hypersensitivity to any of the excipients of Sandoz’ filgrastim product 10. Positive result of anti-rhG-CSF antibody assessment at screening 11. Absolute and relative contraindications as specified in the SmPC of Sandoz’ filgrastim 12. Drug abuse, substance abuse, or alcohol abuse 13. Use of any other investigational drug at the time of enrollment, or within 30 days or 5 half-lives prior to enrollment, whichever is longer 14. Patients unwilling and/or who are not capable of ensuring compliance with the provisions of the study protocol 15. 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 16. Women of child-bearing potential, defined as all women physiologically capable of becoming pregnant, UNLESS they are using an effective method of birth control (i.e. hormonal implants or injectables, combined oral contraceptives and hormonal intrauterine devices). For women of child-bearing potential who will not use a hormonal method named above it is acceptable to use a double barrier method. Adequate double-barrier methods of contraception include: condom (by the partner) combined with a diaphragm with spermicidal foam/gel/etc., an intrauterine device (copper), a sponge or spermicide. Periodic abstinence (e.g. calendar, ovulation, symptothermal, postovulation methods) is not acceptable.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: The primary objective of this study is to evaluate the immunogenicity of long-term treatment of SCN patients with Sandoz’ filgrastim in terms of the incidence of anti-rhG-CSF antibodies.;Secondary Objective: • To assess the incidence and severity of adverse events • To evaluate the efficacy of Sandoz’ filgrastim in patients with severe chronic neutropenia in terms of changes in the absolute neutrophil count ;Primary end point(s): n/a, only descriptive statistics;Timepoint(s) of evaluation of this end point: n/a | — |
Secondary
| Measure | Time frame |
|---|---|
| Secondary end point(s): n/a, only descriptive statistics;Timepoint(s) of evaluation of this end point: n/a | — |
Countries
Germany, Sweden
Contacts
Public ContactClaudia Sailer
Sandoz Biopharmaceuticals Development
Outcome results
None listed