Clinical trial NCT00262301: 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
Overall status: COMPLETED
Phase: PHASE3
Conditions: Hereditary Angioedema
Angioneurotic Edema
Genetic Disorders
Lead sponsor: Pharming Technologies B.V.
Sponsor class: INDUSTRY
First posted: 2005-12-06
Enrollment: 75
Sex: ALL
Minimum age (years): 16
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 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.

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

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 Deparment
city: Tirgu Mures
country: Romania
facility: Emergency County Hospital, Internal Medicin Clinica, Allergology-Immunology Department
Study officials:
affiliation: Pharming Group N.V.
name: Jan Nuijens, MD, PhD
role: STUDY_CHAIR
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: 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.
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.