Clinical Trial Results:
An open-label exploratory Phase II study of the safety and prophylactic effect of a weekly 50 U/kg rC1INH treatment in asymptomatic patients with hereditary C1INH deficiency (HAE)
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Summary
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EudraCT number |
2009-010736-18 |
Trial protocol |
HU |
Global end of trial date |
19 Apr 2010
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Results information
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Results version number |
v1(current) |
This version publication date |
26 Sep 2018
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First version publication date |
26 Sep 2018
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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 |
C1 1207
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Additional study identifiers
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ISRCTN number |
- | ||
US NCT number |
NCT00851409 | ||
WHO universal trial number (UTN) |
- | ||
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Sponsors
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Sponsor organisation name |
Pharming Technologies BV
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Sponsor organisation address |
Darwinweg 24, Leiden, Netherlands, 2333CR
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Public contact |
Anurag Relan, MD, Pharming Technologies BV, +31 715247400, medical-information@pharming.com
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Scientific contact |
Anurag Relan, MD, Pharming Technologies BV, +31 715247400, medical-information@pharming.com
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Paediatric regulatory details
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Is trial part of an agreed paediatric investigation plan (PIP) |
No
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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 |
19 Apr 2010
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Is this the analysis of the primary completion data? |
Yes
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Primary completion date |
19 Apr 2010
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Global end of trial reached? |
Yes
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Global end of trial date |
19 Apr 2010
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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 |
To evaluate the occurrence of HAE attacks under prophylactic administration of rhC1INH (50 U/kg, once a week)
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Protection of trial subjects |
Breakthrough angioedema acute attacks were allowed to be treated with rhC1INH at 50 U/kg with the provision of a second 50 U/kg dose. The maximum allowed total number of rhC1INH administrations within this study was 15 per patient.
For the purpose of this study, patients were recruited in 3 Phases. The study commenced with the simultaneous recruitment of 4 patients (Phase 1). Safety Laboratory Data from the 4th treatment visit was analyzed for clinically significant abnormalities. If no safety problems were identified in the Phase 1 patients, the next 4 patients (Phase 2) started.
Safety Laboratory Data from the last treatment visit from the Phase 1 patients along with Safety Laboratory Data from the 4th treatment visit from Phase 2 patients were analyzed for clinically significant abnormalities. If no safety problems were identified in the Phase 1 and Phase 2 patients, then the last 17 patients (Phase 3) were able to start.
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Background therapy |
- | ||
Evidence for comparator |
- | ||
Actual start date of recruitment |
26 Aug 2009
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Long term follow-up planned |
No
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Independent data monitoring committee (IDMC) involvement? |
No
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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 |
Israel: 7
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Country: Number of subjects enrolled |
Romania: 10
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Country: Number of subjects enrolled |
Poland: 8
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Worldwide total number of subjects |
25
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EEA total number of subjects |
18
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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) |
0
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Adolescents (12-17 years) |
0
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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 |
Twenty-five patients from five countries (Italy, Hungary, Romania, Poland and Israel) with a confirmed HAE diagnosis, of at least 18 years old (men and women) were planned to be included in the study. | ||||||||||
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Pre-assignment
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Screening details |
Confirmed diagnosis of HAE with baseline plasma level of functional C1 INH activity of less than 50% of normal, and/or proven HAE mutation in C1INH gene and occurrence of HAE attacks at least fortnight. In total: 25 screened, 25 eligible, 25 treated and 24 completed patients, from Israel, Romania and Poland. | ||||||||||
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Period 1
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Period 1 title |
Treatment 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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Treatment period | ||||||||||
Arm description |
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Arm type |
Experimental | ||||||||||
Investigational medicinal product name |
Recombinant Human C1 Inhibitor
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Investigational medicinal product code |
rhC1INH
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Other name |
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Pharmaceutical forms |
Powder for solution for infusion
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Routes of administration |
Intravenous use
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Dosage and administration details |
rhC1INH at 50 U/kg
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Period 2
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Period 2 title |
Follow up
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Is this the baseline period? |
No | ||||||||||
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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Follow up | ||||||||||
Arm description |
After the last treatment patients were followed up for 42 days. One study visit took place 7 days after the last study treatment and one visit 42 days after last study treatment. | ||||||||||
Arm type |
No intervention | ||||||||||
Investigational medicinal product name |
No investigational medicinal product assigned in this arm
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Baseline characteristics reporting groups
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Reporting group title |
Treatment period
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Reporting group description |
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End points reporting groups
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Reporting group title |
Treatment period
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Reporting group description |
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Reporting group title |
Follow up
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Reporting group description |
After the last treatment patients were followed up for 42 days. One study visit took place 7 days after the last study treatment and one visit 42 days after last study treatment. | ||
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End point title |
Incidence of documented HAE attacks during treatment period [1] | ||||||||
End point description |
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End point type |
Primary
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End point timeframe |
8 weeks
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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: As this was an open-label single arm study, no statistical analyses could be provided. The pre defined endpoint was the number of HAE attacks during the treatment period. No comparison has been against a control or placebo group, so therefore no statistical analyses could be presented. |
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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 |
All treatment emergent adverse events have been listed, including any events that happened in the follow up period.
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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 |
13
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Reporting groups
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Reporting group title |
Treatment phase
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Reporting group description |
Administrations of rhC1INH were given once weekly over an 8-week period. | ||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
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| Frequency threshold for reporting non-serious adverse events: 4% | |||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
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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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24 Sep 2009 |
This amendment is written to make the protocol more clear and to rectify any discrepancies in the text body or between the text body and appendix. Also to reflect changes that occurred in the time between the first final protocol and this amendment 1. |
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10 Dec 2009 |
This amendment was written to clarify the methodology for immunology testing. To avoid inconsistencies, the body text needed adaptation in some points. |
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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 | |||
