Twelve-month study on the immunogenicity (i.e. the ability of the study medication to provoke an immune response), safety and efficacy of the study medication Zarzio®/Filgrastim HEXAL® in patients with severe chronic neutropenia

Phase 1

• Conditions: Severe chronic neutropenia (i.e. congenital neutropenia, cyclic neutropenia and chronic idiopathic neutropenia); MedDRA version: 18.0Level: PTClassification code 10051645Term: Idiopathic neutropeniaSystem Organ Class: 10005329 - Blood and lymphatic system disorders; MedDRA version: 18.0Level: PTClassification code 10053176Term: Cyclic neutropeniaSystem Organ Class: 10005329 - Blood and lymphatic system disorders; MedDRA version: 18.0Level: LLTClassification code 10069819Term: Congenital neutropeniaSystem Organ Class: 100000004850; Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15]

• Registration Number: EUCTR2011-001118-32-SE
• Lead Sponsor: Sandoz GmbH

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Study Start: 2011-12-06
• Last Update Posted: 7 November 2016

Recruitment & Eligibility

• Status: ot Recruiting
• Sex: All
• Target Recruitment: 40

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 = 18 years of age at the day of inclusion
3. Written informed consent of patient before any assessment is performed

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 40
F.1.3 Elderly (>=65 years) no
F.1.3.1 Number of subjects for this age range 0

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 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, postovulation methods) is not acceptable.

Study & Design

• Study Type: Interventional clinical trial of medicinal product
• Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
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<br>• To evaluate the efficacy of Sandoz’ filgrastim in patients with severe chronic neutropenia in terms of changes in the absolute neutrophil count<br>;Primary end point(s): n/a, only descriptive statistics;Timepoint(s) of evaluation of this end point: n/a

Secondary Outcome Measures

Name	Time	Method
Secondary end point(s): n/a, only descriptive statistics;Timepoint(s) of evaluation of this end point: n/a