Clinical Trial Results:
CLINICAL STUDY TO EVALUATE THE EFFICACY, PHARMACOKINETICS AND SAFETY OF IMMUNOGLOBULIN INTRAVENOUS (HUMAN) 10% (NEWGAM) IN PATIENTS WITH PRIMARY IMMUNODEFICIENCY DISEASES
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Summary
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EudraCT number |
2009-011434-10 |
Trial protocol |
DE Outside EU/EEA |
Global end of trial date |
07 Jun 2012
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Results information
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Results version number |
v1(current) |
This version publication date |
30 Nov 2016
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First version publication date |
30 Nov 2016
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Other versions |
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Trial Information
Subject Disposition
Baseline Characteristics
End Points
Adverse Events
More Information
Subject Disposition
Baseline Characteristics
End Points
Adverse Events
More Information
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Trial identification
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Sponsor protocol code |
NGAM-01
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Additional study identifiers
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ISRCTN number |
ISRCTN05425999 | ||
US NCT number |
NCT01012323 | ||
WHO universal trial number (UTN) |
- | ||
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Sponsors
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Sponsor organisation name |
Octapharma AG
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Sponsor organisation address |
Seidenstraße 2, Lachen, Switzerland, CH-8853
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Public contact |
Clinical Research and Development, Octapharma Pharmazeutika Produktionsgesellschaft mbH, 43 1610320,
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Scientific contact |
Clinical Research and Development, Octapharma Pharmazeutika Produktionsgesellschaft mbH, 43 1610320,
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Paediatric regulatory details
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Is trial part of an agreed paediatric investigation plan (PIP) |
Yes
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EMA paediatric investigation plan number(s) |
EMEA-001110-PIP01-10 | ||
Does article 45 of REGULATION (EC) No 1901/2006 apply to this trial? |
No
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Does article 46 of REGULATION (EC) No 1901/2006 apply to this trial? |
No
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Results analysis stage
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Analysis stage |
Final
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Date of interim/final analysis |
31 Jul 2013
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Is this the analysis of the primary completion data? |
No
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Global end of trial reached? |
Yes
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Global end of trial date |
07 Jun 2012
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Was the trial ended prematurely? |
No
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General information about the trial
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Main objective of the trial |
The primary objective of the study is to assess the efficacy of NewGam in preventing serious bacterial infections compared to historical control data.
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Protection of trial subjects |
This trial was conducted in accordance to the principles of GCP, ensuring that the rights, safety and well-being of patients are protected and in consistency with the Declaration of Helsinki.
Inclusion and exclusion criteria were carefully defined in order to protect subjects from contraindications, interactions with other medication and safety factors associated with the investigational medicinal product. Throughout the study safety was assessed, such as occurrence of AEs, lab values, vital signs and physical examinations.
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Background therapy |
- | ||
Evidence for comparator |
- | ||
Actual start date of recruitment |
15 Jan 2010
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Long term follow-up planned |
No
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Independent data monitoring committee (IDMC) involvement? |
Yes
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Population of trial subjects
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Number of subjects enrolled per country |
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Country: Number of subjects enrolled |
Poland: 11
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Country: Number of subjects enrolled |
Germany: 2
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Country: Number of subjects enrolled |
United States: 38
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Worldwide total number of subjects |
51
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EEA total number of subjects |
13
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Number of subjects enrolled per age group |
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In utero |
0
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Preterm newborn - gestational age < 37 wk |
0
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Newborns (0-27 days) |
0
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Infants and toddlers (28 days-23 months) |
0
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Children (2-11 years) |
13
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Adolescents (12-17 years) |
13
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Adults (18-64 years) |
24
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From 65 to 84 years |
1
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85 years and over |
0
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Recruitment
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Recruitment details |
- | ||||||||||
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Pre-assignment
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Screening details |
The patients were to be recruited into three age strata (at least 2 years and less than 12 years, at least 12 years and less than 16 years of age, and at least 16 years and no greater than 75 years) and- depending on the patient’s pre-study infusion interval- on two treatment schedules (3-week or 4 week IVIG schedule ) | ||||||||||
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Period 1
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Period 1 title |
overall trial (overall period)
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Is this the baseline period? |
Yes | ||||||||||
Allocation method |
Not applicable
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Blinding used |
Not blinded | ||||||||||
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Arms
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Arm title
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NewGam | ||||||||||
Arm description |
Participants received NewGam 200-800 mg/kg body weight intravenously every 3 weeks (17 infusions) or 4 weeks (13 infusions) for 1 year. | ||||||||||
Arm type |
Experimental | ||||||||||
Investigational medicinal product name |
NewGam,human normal immunoglobulin 10%, solvent/detergent treated solution for intravenous infusion
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Investigational medicinal product code |
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Other name |
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Pharmaceutical forms |
Solution for infusion
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Routes of administration |
Intravenous use
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Dosage and administration details |
200 to 800 mg/kg body weight every 21 (+/-3) days or 28 (+/-3) days, with individual doses and intervals being dependent on the patient’s previous IVIG dose and interval before entry into the study
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Baseline characteristics reporting groups
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Reporting group title |
overall trial
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Reporting group description |
All patients exposed to treatment | ||||||||||||||||||||||||||||||||||||||||||
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End points reporting groups
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Reporting group title |
NewGam
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Reporting group description |
Participants received NewGam 200-800 mg/kg body weight intravenously every 3 weeks (17 infusions) or 4 weeks (13 infusions) for 1 year. | ||
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End point title |
rate of serious bacterial infections per person-year [1] | ||||||||
End point description |
The number of serious bacterial infections per person-year of treatment was calculated by the following formula: Total number of serious bacterial infections / patient-years on NewGam treatment. Serious bacterial infections were defined as bacteraemia/sepsis, bacterial meningitis, osteomyelitis/septic arthritis, bacterial pneumonia, and visceral abscess.
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End point type |
Primary
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End point timeframe |
Baseline to end of the study (up to 12 months)
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| Notes [1] - No statistical analyses have been specified for this primary end point. It is expected there is at least one statistical analysis for each primary end point. Justification: The primary endpoint (rate of serious bacterial infections per person-year) is presented as point estimates of the rate along with a 99% confidence interval (CI). The rate of serious bacterial infections per year was calculated by the following formula: r = Total number of serious bacterial infections/Patient years on NewGam treatment |
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| No statistical analyses for this end point | |||||||||
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Adverse events information
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Timeframe for reporting adverse events |
The condition of the patient was monitored throughout the whole study (baseline up to completion/termination of the study)
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Assessment type |
Systematic | ||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
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Dictionary used for adverse event reporting
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Dictionary name |
MedDRA | ||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
Dictionary version |
12.0
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Reporting groups
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Reporting group title |
Safety Set
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Reporting group description |
all patients who received at least part of one treatment with NewGam. | ||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
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| Frequency threshold for reporting non-serious adverse events: 5% | |||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
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Substantial protocol amendments (globally) |
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| Were there any global substantial amendments to the protocol? Yes | |||
Date |
Amendment |
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19 Aug 2010 |
Amendment 4:
• The recruitment period was prolonged and the number of sites was increased.
• Patients less than 16 years of age with a history of diabetes mellitus type II were allowed to enter the study.
• Flu vaccination including H1N1 strain during the study was allowed.
• Measles was to be reported as an SAE.
• Only AEs classified as at least possibly related to the study drug (ADRs) to be assessed as to their expectedness by the sponsor in accordance with current Octapharma drug safety procedures
• Following the adoption of a new CHMP guideline on the clinical investigation of IVIG (EMA/CHMP/BPWP/94033/2007 rev 2) , interim analysis after 6 months of treatment in 15 patients was deleted
without any consequence for the study. |
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16 Feb 2012 |
Amendment 5:
• The planned clinical end was delayed by one quarter.
• A clarification was added that patients were to be evaluated in the age group assigned at the time when they had signed the informed consent
• Secondary endpoints and safety evaluation were updated upon request of the Paediatric Committee (PDCO).
• A clarification was added that discrepancies between diary entries and eCRF entries were to be explained by investigator in source records.
• Information was added that therapeutic efficacy parameters were to be evaluated per person-year on treatment.
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Interruptions (globally) |
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| Were there any global interruptions to the trial? No | |||
Limitations and caveats |
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| Limitations of the trial such as small numbers of subjects analysed or technical problems leading to unreliable data. | |||
| None reported | |||
