Clinical trial NCT01188564: Efficacy, Safety and Immunogenicity Study of Recombinant Human C1 Inhibitor for the Treatment of Acute HAE Attacks
Official title: A Phase III Randomized, Double-blind, Placebo-controlled Study With an Open-label Extension Evaluating the Efficacy, Safety and Immunogenicity of Recombinant Human C1 Inhibitor for the Treatment of Acute Attacks of Angioedema in Patients With HAE
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE3
Conditions: Hereditary Angioedema
Keywords: Hereditary Angioedema
HAE
Angioedema
Recombinant C1 Inhibitor
rhC1INH
Lead sponsor: Pharming Technologies B.V.
Sponsor class: INDUSTRY
First posted: 2010-08-25
Enrollment: 75
Sex: ALL
Minimum age (years): 13
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

Brief summary: This study is being conducted to confirm the efficacy, safety, and immunogenicity of recombinant human C1 inhibitor (rhC1INH) at a dose of 50 U/kg when used for the treatment of acute angioedema attacks in Hereditary Angioedema (HAE) patients.

Detailed description: HAE is characterized by recurrent localized angioedema caused by uncontrolled activation of the complement and contact systems due to a congenital deficiency of functional C1 inhibitor.

rhC1INH has been developed to offer a more widely available therapeutic alternative to the existing plasma-derived C1INH (pdC1INH) products that have been used in the treatment of acute angioedema attacks patients with HAE.

Patients who have qualified for enrollment in advance and who present to a study center within 5 hours of onset of an attack will be evaluated for eligibility. 75 eligible patients will be randomized (3:2) to receive an intravenous infusion of rhC1INH or saline in a double-blind fashion. Open-label rhC1INH may be provided as rescue medication to patients who do not experience the beginning of relief within 4 hours or who experience life-threatening oropharyngeal-laryngeal angioedema symptoms.

Any patient having received a randomized treatment will be allowed to receive treatment with rhC1INH in an open-label fashion for subsequent eligible attacks.

Eligibility criteria: Inclusion Criteria:

* Aged at least 13 years
* Signed written informed consent
* Clear clinical and laboratory diagnosis of HAE with baseline plasma level of functional C1INH of less than 50% of normal
* Willingness and ability to comply with all protocol procedures
* Clinical symptoms of an eligible HAE attack with onset less than 5 hours before the time of initial evaluation

Exclusion Criteria:

* Medical history of allergy to rabbits or rabbit-derived products (including rhC1INH), or positive anti-rabbit dander IgE test (cut off \>0.35 kU/L; ImmunoCap® assay; Phadia or equivalent).
* A diagnosis of acquired C1INH deficiency (AAE)
* Pregnancy, or breastfeeding, or current intention to become pregnant
* Treatment with any investigational drug in the past 30 days
* Known or suspected addiction to drug and/or alcohol abuse
* Suspicion for an alternate explanation of the symptoms other than acute HAE attack

Primary outcomes:
measure: Time to Beginning of Relief of Symptoms
timeFrame: Patients observed for 24 hours
Secondary outcomes:
measure: Time to Minimal Symptoms
timeFrame: 24 hours
Other outcomes:
Locations:
city: Scottsdale
country: United States
facility: Allergy, Asthma & Immunology, Assoc, Ltd.
state: Arizona
city: Granada Hills
country: United States
facility: Allergy and Asthma Institute of the Valley
state: California
city: Los Angeles
country: United States
facility: UCLA Department of Medicine Division of Clinical Immunology, David Geffen School of Medicine
state: California
city: Tampa
country: United States
facility: USF Asthma, Allergy and Immunology Clinical Research Unit
state: Florida
city: Atlanta
country: United States
facility: Family Allergy and Asthma Center
state: Georgia
city: Chevy Chase
country: United States
facility: Institute for Asthma and Allergy, P.C.
state: Maryland
city: St. Louis
country: United States
facility: Asthma & Allergy Center - Washington University School of Medicine
state: Missouri
city: Cincinnati
country: United States
facility: University of Cincinnati Physicians, Inc.
state: Ohio
city: Columbus
country: United States
facility: Optimed Research, LTD
state: Ohio
city: Lake Oswego
country: United States
facility: Baker Allergy, Asthma and Dermatology Research Center, LLC
state: Oregon
city: Hershey
country: United States
facility: Pennsylvania State- Milton S. Hershey Medical Center
state: Pennsylvania
city: Dallas
country: United States
facility: AARA Research Center
state: Texas
city: Galveston
country: United States
facility: University of Texas - Medical Branch
state: Texas
city: Irving
country: United States
facility: Allergy, Asthma & Immunology Clinic, P.A.
state: Texas
city: Spokane
country: United States
facility: Marycliff Allergy Specialists
state: Washington
city: Sofia
country: Bulgaria
facility: UMHAT "Tsaritsa Yoanna - ISUL"; Clinic of Ear-Nose-Throat Diseases
city: Ottawa
country: Canada
facility: Ottawa Allergy Research Corp.
state: Ontario
city: Budapest
country: Hungary
facility: Semmelweis University Faculty of Medicine, III Department of Internal Medicine
city: Tel Hashomer
country: Israel
facility: Allergy, Immunology & Angioedema Center,
state: Ramat Gan
city: Haifa
country: Israel
facility: Bnei-Zion Medical Centre, Clinical Immunology and Allergy Division
city: Milan
country: Italy
facility: Ospedale Luigi Sacco, Azienda Ospedaliera - Polo Universitario II Divisione di Medicina Interna
city: Skopje
country: Macedonia, The Former Yugoslav Republic of
facility: P.H.U. Clinic for Dermatology, Medical University Skopje, Unit of Allergology and Clinical Immunology
city: Krakow
country: Poland
facility: Szpital Uniwersytecki w Krakowie, Oddział Kliniczny Klinik Chorób Wewnętrznych, Poradnia Alergologiczna
city: Târgu-Mureş
country: Romania
facility: Spitalul Clinic Judeţean Mureş Secţia Clinică Medicină Internă, Compartimentul de Alergologie şi Imunologie
city: Belgrade
country: Serbia
facility: Clinic for Immunology and Allergology
city: Mowbray
country: South Africa
facility: Allergy Diagnostic & Clinical Research Unit University of Cape Town Lung Institute
city: Parktown
country: South Africa
facility: Wits Health Consortium (Pty) Ltd - Wits Donald Gordon Medical Centre
Study officials:
References: