Trial Outcomes & Findings for Immunogenicity, Safety, and Efficacy of Zarzio®/Filgrastim HEXAL® in Patients With Severe Chronic Neutropenia (NCT NCT01859637)
NCT ID: NCT01859637
Last Updated: 2016-03-28
Results Overview
Incidence of anti-rhG-CSF antibodies was monitored. Patients were screened for anti-rhG-CSF antibodies at screening and at each study except visit 02 (start of treatment = baseline). Evaluation of immune response to rhG-CSF administration was made by a three-step procedure comprising a validated binding antibody screening and confirmatory radioimmunoprecipitation assay (RIP) and a validated cell-based neutralization antibody assay (NAB).
TERMINATED
PHASE4
6 participants
screening, 3, 6, 9 and 12 months
2016-03-28
Participant Flow
First patient in: 05-Jul-2011, last patient in: 12-Feb-2013. 2 sites (medical school and university hospital)
Participant milestones
| Measure |
Zarzio®/Filgrastim HEXAL® (EP2006)
Open label single arm. All patients received Zarzio® subcutaneously dosed as per recommendations in Summary of Product Characteristics (SmPC).
|
|---|---|
|
Overall Study
STARTED
|
6
|
|
Overall Study
COMPLETED
|
6
|
|
Overall Study
NOT COMPLETED
|
0
|
Reasons for withdrawal
Withdrawal data not reported
Baseline Characteristics
Immunogenicity, Safety, and Efficacy of Zarzio®/Filgrastim HEXAL® in Patients With Severe Chronic Neutropenia
Baseline characteristics by cohort
| Measure |
Zarzio®/Filgrastim HEXAL®
n=6 Participants
Open label single arm. All patients received Zarzio®/Filgrastim HEXAL® subcutaneously dosed as per recommendations in SmPC.
|
|---|---|
|
Age, Continuous
|
45.0 years
n=5 Participants
|
|
Sex: Female, Male
Female
|
5 Participants
n=5 Participants
|
|
Sex: Female, Male
Male
|
1 Participants
n=5 Participants
|
|
Race/Ethnicity, Customized
Caucasian
|
5 participants
n=5 Participants
|
|
Race/Ethnicity, Customized
Latin American
|
1 participants
n=5 Participants
|
|
Region of Enrollment
Sweden
|
4 participants
n=5 Participants
|
|
Region of Enrollment
Germany
|
2 participants
n=5 Participants
|
|
Severe Chronic Neutropenia (SCN) diagnosis
Congenital neutropenia
|
1 participants
n=5 Participants
|
|
Severe Chronic Neutropenia (SCN) diagnosis
Cyclic neutropenia
|
1 participants
n=5 Participants
|
|
Severe Chronic Neutropenia (SCN) diagnosis
Idiopathic neutropenia
|
4 participants
n=5 Participants
|
|
Granulocyte Colony Stimulating Factor (G-CSF) pretreatment
Yes
|
6 participants
n=5 Participants
|
|
Granulocyte Colony Stimulating Factor (G-CSF) pretreatment
No
|
0 participants
n=5 Participants
|
|
Height
|
165.0 cm
n=5 Participants
|
|
Weight
|
67.0 kg
n=5 Participants
|
PRIMARY outcome
Timeframe: screening, 3, 6, 9 and 12 monthsPopulation: Safety population (SAF): All patients with at least one dose Zarzio®/Filgrastim HEXAL® and at least one post-baseline safety assessment. All six patients were screened for anti-rhG-CSF antibodies at all six study visits, except for one missing assessment (no sample was taken for patient 0204 at Visit 03, which was an optional visit).
Incidence of anti-rhG-CSF antibodies was monitored. Patients were screened for anti-rhG-CSF antibodies at screening and at each study except visit 02 (start of treatment = baseline). Evaluation of immune response to rhG-CSF administration was made by a three-step procedure comprising a validated binding antibody screening and confirmatory radioimmunoprecipitation assay (RIP) and a validated cell-based neutralization antibody assay (NAB).
Outcome measures
| Measure |
Zarzio®/Filgrastim HEXAL®
n=6 Participants
All patients received open-label Zarzio®/Filgrastim HEXAL®
|
|---|---|
|
Incidence of Anti- Recombinant Human Granulocyte Colony Stimulating Factor (rhG-CSF) Antibodies
Visit 1 - negative
|
6 participants
|
|
Incidence of Anti- Recombinant Human Granulocyte Colony Stimulating Factor (rhG-CSF) Antibodies
Visit 3 - negative
|
5 participants
|
|
Incidence of Anti- Recombinant Human Granulocyte Colony Stimulating Factor (rhG-CSF) Antibodies
Visit 4 - negative
|
6 participants
|
|
Incidence of Anti- Recombinant Human Granulocyte Colony Stimulating Factor (rhG-CSF) Antibodies
Visit 5 - negative
|
6 participants
|
|
Incidence of Anti- Recombinant Human Granulocyte Colony Stimulating Factor (rhG-CSF) Antibodies
Visit 6 - negative
|
6 participants
|
|
Incidence of Anti- Recombinant Human Granulocyte Colony Stimulating Factor (rhG-CSF) Antibodies
Visit 7 - negative
|
6 participants
|
SECONDARY outcome
Timeframe: 12 monthsPopulation: Safety population (SAF): all patients with at least one dose Zarzio®/Filgrastim HEXAL® and at least one post-baseline safety assessment
Patients experiencing AEs by system organ class and preferred term (PT) and number of events. Patients with more than one AE coded to the same PT were counted once per PT
Outcome measures
| Measure |
Zarzio®/Filgrastim HEXAL®
n=6 Participants
All patients received open-label Zarzio®/Filgrastim HEXAL®
|
|---|---|
|
Number of Participants With Adverse Events (AEs)
|
6 participants
|
SECONDARY outcome
Timeframe: Participants were followed for a duration of 12 months and ANC was assessed at baseline, week 6, Month 3, Month 6, Month 9 and Month 12.Population: Safety population (SAF): all patients with at least one dose Zarzio®/Filgrastim HEXAL® and at least one post-baseline safety assessment
To evaluate the efficacy of Zarzio®/Filgrastim HEXAL® in patients with SCN in terms of changes in absolute neutrophile count (ANC). Change from each visit to baseline in ANC for all patients is calculated.
Outcome measures
| Measure |
Zarzio®/Filgrastim HEXAL®
n=6 Participants
All patients received open-label Zarzio®/Filgrastim HEXAL®
|
|---|---|
|
Change in Absolute Neutrophile Count (ANC)
ANC Visit 6 Month 9 to Baseline (Visit 2)
|
1.506 10^9 cells/L
Interval -9.6 to 14.7
|
|
Change in Absolute Neutrophile Count (ANC)
ANC Visit 1 Screening to Baseline (Visit 2)
|
-1.252 10^9 cells/L
Interval -10.9 to 2.84
|
|
Change in Absolute Neutrophile Count (ANC)
ANC Visit 3 Week 6 to Baseline (Visit 2)
|
6.873 10^9 cells/L
Interval -11.2 to 16.4
|
|
Change in Absolute Neutrophile Count (ANC)
ANC Visit 4 Month 3 to Baseline (Visit 2)
|
-0.198 10^9 cells/L
Interval -11.0 to 25.5
|
|
Change in Absolute Neutrophile Count (ANC)
ANC Visit 5 Month 6 to Baseline (Visit 2)
|
-0.711 10^9 cells/L
Interval -10.8 to 12.38
|
|
Change in Absolute Neutrophile Count (ANC)
ANC Visit 7 Month 12 to Baseline (Visit 2)
|
1.579 10^9 cells/L
Interval -10.8 to 18.87
|
Adverse Events
Open-label Zarzio®/Filgrastim HEXAL®
Serious adverse events
| Measure |
Open-label Zarzio®/Filgrastim HEXAL®
n=6 participants at risk
All patients received open-label Zarzio®/Filgrastim HEXAL®
|
|---|---|
|
Renal and urinary disorders
Nephrolithiasis
|
16.7%
1/6 • Number of events 1 • 12 months
|
Other adverse events
| Measure |
Open-label Zarzio®/Filgrastim HEXAL®
n=6 participants at risk
All patients received open-label Zarzio®/Filgrastim HEXAL®
|
|---|---|
|
Infections and infestations
Nasopharyngitis
|
50.0%
3/6 • Number of events 5 • 12 months
|
|
Infections and infestations
Genital infection fugal
|
16.7%
1/6 • Number of events 1 • 12 months
|
|
Infections and infestations
Gingivitis
|
16.7%
1/6 • Number of events 1 • 12 months
|
|
Infections and infestations
Groin abscess
|
16.7%
1/6 • Number of events 1 • 12 months
|
|
Infections and infestations
Herpes virus infection
|
16.7%
1/6 • Number of events 1 • 12 months
|
|
Infections and infestations
Mucosal infection
|
16.7%
1/6 • Number of events 2 • 12 months
|
|
Infections and infestations
Oral herpes
|
16.7%
1/6 • Number of events 1 • 12 months
|
|
Infections and infestations
Pneumonia
|
16.7%
1/6 • Number of events 1 • 12 months
|
|
Infections and infestations
Rash pustular
|
16.7%
1/6 • Number of events 4 • 12 months
|
|
Infections and infestations
Sinusitis
|
16.7%
1/6 • Number of events 1 • 12 months
|
|
Infections and infestations
Subcutaneous abscess
|
16.7%
1/6 • Number of events 1 • 12 months
|
|
Infections and infestations
Tonsilitis
|
16.7%
1/6 • Number of events 1 • 12 months
|
|
Infections and infestations
Upper respiratory tract infection
|
16.7%
1/6 • Number of events 1 • 12 months
|
|
General disorders
Fatigue
|
33.3%
2/6 • Number of events 2 • 12 months
|
|
General disorders
Local swelling
|
16.7%
1/6 • Number of events 1 • 12 months
|
|
General disorders
Malaise
|
16.7%
1/6 • Number of events 1 • 12 months
|
|
Nervous system disorders
Headache
|
50.0%
3/6 • Number of events 3 • 12 months
|
|
Skin and subcutaneous tissue disorders
Alopecia
|
16.7%
1/6 • Number of events 1 • 12 months
|
|
Skin and subcutaneous tissue disorders
Pruritus
|
16.7%
1/6 • Number of events 1 • 12 months
|
|
Skin and subcutaneous tissue disorders
Skin induration
|
16.7%
1/6 • Number of events 1 • 12 months
|
|
Gastrointestinal disorders
Abdominal pain
|
16.7%
1/6 • Number of events 1 • 12 months
|
|
Gastrointestinal disorders
Diarrhoea
|
16.7%
1/6 • Number of events 1 • 12 months
|
|
Gastrointestinal disorders
Mouth ulceration
|
16.7%
1/6 • Number of events 2 • 12 months
|
|
Gastrointestinal disorders
Vomiting
|
16.7%
1/6 • Number of events 1 • 12 months
|
|
Investigations
Neutrophil count decreased
|
16.7%
1/6 • Number of events 1 • 12 months
|
|
Musculoskeletal and connective tissue disorders
Arthralgia
|
16.7%
1/6 • Number of events 1 • 12 months
|
|
Surgical and medical procedures
Tooth extraction
|
16.7%
1/6 • Number of events 1 • 12 months
|
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place
Restriction type: GT60