Recombinant Human C1 Inhibitor for the Treatment of Acute Attacks in Patients With Hereditary Angioedema
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|ClinicalTrials.gov Identifier: NCT00225147|
Recruitment Status : Completed
First Posted : September 23, 2005
Results First Posted : August 31, 2012
Last Update Posted : February 22, 2013
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 lead to a decrease in the blood level of functional "C1INH". This multi-center study was designed to assess the safety and tolerability, efficacy, and pharmacokinetics/pharmacodynamics of recombinant human C1 inhibitor ("rhC1INH") in the treatment of acute hereditary angioedema attacks.
Funding Source - FDA OOPD
|Condition or disease||Intervention/treatment||Phase|
|Hereditary Angioedema Angioneurotic Edema||Drug: Recombinant Human C1 Inhibitor Drug: placebo||Phase 2 Phase 3|
|Study Type :||Interventional (Clinical Trial)|
|Actual Enrollment :||77 participants|
|Intervention Model:||Parallel Assignment|
|Masking:||Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)|
|Official Title:||A Randomized, Placebo-controlled, Double Blind Phase II/III Study of the Safety and Efficacy of Recombinant Human C1 Inhibitor for the Treatment of Acute Attacks in Patients With Hereditary Angioedema|
|Study Start Date :||July 2005|
|Actual Primary Completion Date :||October 2009|
|Actual Study Completion Date :||January 2010|
Experimental: 100 IU/kg rhC1INH
100 IU/kg Recombinant human C1 inhibitor
Drug: Recombinant Human C1 Inhibitor
Experimental: 50 IU/kg rhC1INH
50 IU/kg Recombinant human C1 inhibitor
Drug: Recombinant Human C1 Inhibitor
|Placebo Comparator: Saline||
- Time to Beginning of Relief of Symptoms [ Time Frame: up to 48 hours after study drug administration ]The time to beginning of relief of symptoms at the location that showed the first visual analogue scale ("VAS") score decrease of at least 20 mm from baseline score with persistence to the next timepoint, assessment timepoints were taken on pre-scheduled time-points after study drug administration: baseline (0 minutes), 15 minutes, 30 minutes, 1 hour, 2 hours, 4 hours, 8 hours, 12 hours, 16 hours, 24 hours and 48 hours. Time to beginning of relief has been calculated as median time, by using the exact timepoints on which each assessment was performed.
- Time to Minimal Symptoms [ Time Frame: up to 48 hours after study drug administration ]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 timepoints were: baseline, 15 minutes, 30 minutes, 1 hour, 2 hours, 4 hours, 8 hours, 12 hours, 16 hours, 24 hours and 48 hours. Time to minimal symtoms has been calculated by using the exact timepoints on which each assessment was performed.
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT00225147
|For information on sites please contact Pharming Medical Affairs Department|
|Leiden, Netherlands, 2300 AL|
|Study Director:||Anurag Relan, MD||Pharming Group N.V.|