Clinical trial NCT00225147: Recombinant Human C1 Inhibitor for the Treatment of Acute Attacks in Patients With Hereditary Angioedema
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 type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE2
PHASE3
Conditions: Hereditary Angioedema
Angioneurotic Edema
Lead sponsor: Pharming Technologies B.V.
Sponsor class: INDUSTRY
First posted: 2005-09-23
Enrollment: 77
Sex: ALL
Minimum age (years): 12
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

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 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

Detailed description: A prospectively planned interim analysis will be performed on the double-blind data.

Eligibility criteria: Main Inclusion Criteria:

* Clear clinical and laboratory diagnosis of HAE
* Plasma level of functional C1INH of less than 50% of normal
* Acute abdominal, urogenital, peripheral, and/or oro-facial/pharyngeal/laryngeal HAE attack

Main Exclusion Criteria:

* Acquired angioedema
* Pregnancy or breastfeeding
* Treatment with any investigational drug within prior 30 days
* Body weight \>120 kg

Primary outcomes:
measure: Time to Beginning of Relief of Symptoms
timeFrame: up to 48 hours after study drug administration
Secondary outcomes:
measure: Time to Minimal Symptoms
timeFrame: up to 48 hours after study drug administration
Other outcomes:
Locations:
city: Leiden
country: Netherlands
facility: For information on sites please contact Pharming Medical Affairs Department
Study officials:
affiliation: Pharming Group N.V.
name: Anurag Relan, MD
role: STUDY_DIRECTOR
References:
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.
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.
citation: Riedl MA, Levy RJ, Suez D, Lockey RF, Baker JW, Relan A, Zuraw BL. Efficacy and safety of recombinant C1 inhibitor for the treatment of hereditary angioedema attacks: a North American open-label study. Ann Allergy Asthma Immunol. 2013 Apr;110(4):295-9. doi: 10.1016/j.anai.2013.02.007. Epub 2013 Mar 6.