Back to resultsRecombinant Human C1 Inhibitor for the Treatment of Acute Attacks in Patients With Hereditary Angioedema
Primary Purpose
Hereditary Angioedema, Angioneurotic Edema, Genetic Disorders
Status
Completed
Phase
Phase 3
Locations
International
Study Type
Interventional
Intervention
recombinant human C1 inhibitor
Placebo
Sponsored by
About this trial
This is an interventional treatment trial for Hereditary Angioedema
Eligibility Criteria
Inclusion Criteria: Clear clinical and laboratory diagnosis of HAE Baseline plasma level of functional C1INH of less than 50% of normal Evidence for exacerbation or development of a severe abdominal, oro-facial/ pharyngeal/ laryngeal, genito-urinary and/or peripheral HAE attack Exclusion Criteria: Acquired angioedema Pregnancy or breastfeeding Participation in another clinical study within prior 3 months
Sites / Locations
- For information on sites, please contact Pharming Medical Affairs Deparment
- Emergency County Hospital, Internal Medicin Clinica, Allergology-Immunology Department
Arms of the Study
Arm 1
Arm 2
Arm Type
Experimental
Placebo Comparator
Arm Label
100 IU/kg "rhC1INH"
Saline
Arm Description
100 IU/kg recombinant human C1 inhibitor
Saline solution
Outcomes
Primary Outcome Measures
Time to Beginning of Relief of Symptoms
The time to beginning of relief of symptoms has been assessed by using a patient-reported visual analogue scale ("VAS") ranging from 0 mm (no symptoms at all) to 100 mm (extremely disabling). Time to beginning of relief of symptoms at the location that showed first "VAS" score decrease of at least 20 mm from baseline score (t= 0 min) to the next assessment time-point). Assessment time-points were taken on pre-scheduled time-points after drug administration: baseline (0 minutes), 15 minutes, 30 minutes, 1 hour, 2 hours, 4 hours, 8 hours, 12 hours, 16 hours, 24 hours, 48 hours. Time to beginning of relief has been calculated as median time, by using the exact time-points on which each assessment was performed.
Secondary Outcome Measures
Time to Minimal Symptoms
the time to minimal symptoms was the time to minimal symptoms for an attack, assessed using the Visual Analogue Scale ("VAS") score. Symptoms were said to be minimal when the "VAS" score at all locations was below 20 mm. Assessment time-points were: baseline (0 minutes), 15 minutes, 30 minutes, 1 hour, 2 hours, 4 hours, 8 hours, 12 hours, 16 hours, 24 hours, 48 hours. Time to minimal symptoms has been calculated by using the exact time-points on which each assessment was performed.
Full Information
NCT ID
NCT00262301
First Posted
December 1, 2005
Last Updated
September 27, 2012
Sponsor
Pharming Technologies B.V.
1. Study Identification
Unique Protocol Identification Number
NCT00262301
Brief Title
Recombinant Human C1 Inhibitor for the Treatment of Acute Attacks in Patients With Hereditary Angioedema
Official Title
A Randomized, Placebo-controlled, Double-blind Phase III Study of the Efficacy and Safety of Recombinant Human C1 Inhibitor for the Treatment of Acute Attacks in Patients With Hereditary Angioedema
Study Type
Interventional
2. Study Status
Record Verification Date
September 2012
Overall Recruitment Status
Completed
Study Start Date
June 2004 (undefined)
Primary Completion Date
July 2009 (Actual)
Study Completion Date
October 2009 (Actual)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Pharming Technologies B.V.
4. Oversight
Data Monitoring Committee
Yes
5. Study Description
Brief Summary
Hereditary angioedema ("HAE") is a genetic disorder characterized by sudden recurrent attacks of local swelling (angioedema). These attacks are often painful and disabling, and, in some cases, life-threatening. "HAE" is caused by mutations in the "C1INH" gene that leads to a decrease in the blood level of functional "C1INH". This multi-center study was designed to assess the safety and tolerability, efficacy and pharmacodynamics/ pharmacokinetics of recombinant human C1 inhibitor ("rhC1INH") in the treatment of acute hereditary angioedema attacks.
Detailed Description
A prospectively planned interim analysis will be performed on the double-blind data.
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Hereditary Angioedema, Angioneurotic Edema, Genetic Disorders
7. Study Design
Primary Purpose
Treatment
Study Phase
Phase 3
Interventional Study Model
Parallel Assignment
Masking
ParticipantCare ProviderInvestigatorOutcomes Assessor
Allocation
Randomized
Enrollment
75 (Actual)
8. Arms, Groups, and Interventions
Arm Title
100 IU/kg "rhC1INH"
Arm Type
Experimental
Arm Description
100 IU/kg recombinant human C1 inhibitor
Arm Title
Saline
Arm Type
Placebo Comparator
Arm Description
Saline solution
Intervention Type
Drug
Intervention Name(s)
recombinant human C1 inhibitor
Other Intervention Name(s)
"rhC1INH", Ruconest, conestat alfa
Intervention Description
IV
Intervention Type
Drug
Intervention Name(s)
Placebo
Other Intervention Name(s)
saline, physiological salt solution
Intervention Description
IV
Primary Outcome Measure Information:
Title
Time to Beginning of Relief of Symptoms
Description
The time to beginning of relief of symptoms has been assessed by using a patient-reported visual analogue scale ("VAS") ranging from 0 mm (no symptoms at all) to 100 mm (extremely disabling). Time to beginning of relief of symptoms at the location that showed first "VAS" score decrease of at least 20 mm from baseline score (t= 0 min) to the next assessment time-point). Assessment time-points were taken on pre-scheduled time-points after drug administration: baseline (0 minutes), 15 minutes, 30 minutes, 1 hour, 2 hours, 4 hours, 8 hours, 12 hours, 16 hours, 24 hours, 48 hours. Time to beginning of relief has been calculated as median time, by using the exact time-points on which each assessment was performed.
Time Frame
up to 48 hours after study drug administration
Secondary Outcome Measure Information:
Title
Time to Minimal Symptoms
Description
the time to minimal symptoms was the time to minimal symptoms for an attack, assessed using the Visual Analogue Scale ("VAS") score. Symptoms were said to be minimal when the "VAS" score at all locations was below 20 mm. Assessment time-points were: baseline (0 minutes), 15 minutes, 30 minutes, 1 hour, 2 hours, 4 hours, 8 hours, 12 hours, 16 hours, 24 hours, 48 hours. Time to minimal symptoms has been calculated by using the exact time-points on which each assessment was performed.
Time Frame
up to 48 hours after study drug administration
10. Eligibility
Sex
All
Minimum Age & Unit of Time
16 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria:
Clear clinical and laboratory diagnosis of HAE
Baseline plasma level of functional C1INH of less than 50% of normal
Evidence for exacerbation or development of a severe abdominal, oro-facial/ pharyngeal/ laryngeal, genito-urinary and/or peripheral HAE attack
Exclusion Criteria:
Acquired angioedema
Pregnancy or breastfeeding
Participation in another clinical study within prior 3 months
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Jan Nuijens, MD, PhD
Organizational Affiliation
Pharming Group N.V.
Official's Role
Study Chair
Facility Information:
Facility Name
For information on sites, please contact Pharming Medical Affairs Deparment
City
Leiden
ZIP/Postal Code
2300 AL
Country
Netherlands
Facility Name
Emergency County Hospital, Internal Medicin Clinica, Allergology-Immunology Department
City
Tirgu Mures
ZIP/Postal Code
541103
Country
Romania
12. IPD Sharing Statement
Citations:
PubMed Identifier
20920772
Citation
Zuraw B, Cicardi M, Levy RJ, Nuijens JH, Relan A, Visscher S, Haase G, Kaufman L, Hack CE. Recombinant human C1-inhibitor for the treatment of acute angioedema attacks in patients with hereditary angioedema. J Allergy Clin Immunol. 2010 Oct;126(4):821-827.e14. doi: 10.1016/j.jaci.2010.07.021.
Results Reference
result
PubMed Identifier
22909164
Citation
Moldovan D, Reshef A, Fabiani J, Kivity S, Toubi E, Shlesinger M, Triggiani M, Montinaro V, Cillari E, Realdi G, Cancian M, Visscher S, Zanichelli A, Relan A, Cicardi M. Efficacy and safety of recombinant human C1-inhibitor for the treatment of attacks of hereditary angioedema: European open-label extension study. Clin Exp Allergy. 2012 Jun;42(6):929-35. doi: 10.1111/j.1365-2222.2012.03984.x.
Results Reference
result
PubMed Identifier
28284978
Citation
Bernstein JA, Relan A, Harper JR, Riedl M. Sustained response of recombinant human C1 esterase inhibitor for acute treatment of hereditary angioedema attacks. Ann Allergy Asthma Immunol. 2017 Apr;118(4):452-455. doi: 10.1016/j.anai.2017.01.029. Epub 2017 Mar 9.
Results Reference
derived
Learn more about this trial
Recombinant Human C1 Inhibitor for the Treatment of Acute Attacks in Patients With Hereditary Angioedema
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