Efficacy, Safety and Immunogenicity Study of Recombinant Human C1 Inhibitor for the Treatment of Acute HAE Attacks
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
1 other identifier
interventional
75
11 countries
27
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.
Trial Health
Trial Health Score
Automated assessment based on enrollment pace, timeline, and geographic reach
participants targeted
Target at below P25 for phase_3
Started Jan 2011
27 active sites
Health score is calculated from publicly available data and should be used for screening purposes only.
Trial Relationships
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Study Timeline
Key milestones and dates
First Submitted
Initial submission to the registry
August 24, 2010
CompletedFirst Posted
Study publicly available on registry
August 25, 2010
CompletedStudy Start
First participant enrolled
January 1, 2011
CompletedPrimary Completion
Last participant's last visit for primary outcome
December 1, 2012
CompletedStudy Completion
Last participant's last visit for all outcomes
March 1, 2013
CompletedResults Posted
Study results publicly available
August 7, 2015
CompletedAugust 7, 2015
July 1, 2015
1.9 years
August 24, 2010
June 10, 2015
August 3, 2015
Conditions
Keywords
Outcome Measures
Primary Outcomes (1)
Time to Beginning of Relief of Symptoms
Time to beginning of relief is the time lapsed from the beginning of the infusion of study medication to the beginning of a beneficial effect based on patient's responses to the Treatmetn Effect Questionnaire (TEQ) for the primary attack location. The beginning of relief is defined as the first timepoint at which * The patient reports any of the following answers for TEQ question 1: "A little better", "Better" or "Much better"; and; * The patient reports the following answer for TEQ question 2: "Yes"; and, * There is persistence in improvement at the next assessment time, i.e.either the same or a better response to Question 1 and "Yes" to Question 2.
Patients observed for 24 hours
Secondary Outcomes (1)
Time to Minimal Symptoms
24 hours
Study Arms (2)
rhC1INH
EXPERIMENTALPlacebo (Saline)
PLACEBO COMPARATORInterventions
One i.v. injection of rhC1INH at the dose of 50 U/kg, for patients up to 84 kg; one i.v. injection of rhC1INH at the dose of 4200U (2 vials) for patients of 84 kg body weight or greater.
One i.v. injection of saline (NaCl 0.9% w/v), equivalent in volume to the active treatment
Eligibility Criteria
You may qualify if:
- 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
You may not qualify if:
- 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
Contact the study team to confirm eligibility.
Sponsors & Collaborators
Study Sites (27)
Allergy, Asthma & Immunology, Assoc, Ltd.
Scottsdale, Arizona, 85251, United States
Allergy and Asthma Institute of the Valley
Granada Hills, California, 91344, United States
UCLA Department of Medicine Division of Clinical Immunology, David Geffen School of Medicine
Los Angeles, California, 90095, United States
USF Asthma, Allergy and Immunology Clinical Research Unit
Tampa, Florida, 33613, United States
Family Allergy and Asthma Center
Atlanta, Georgia, 30342, United States
Institute for Asthma and Allergy, P.C.
Chevy Chase, Maryland, 20815, United States
Asthma & Allergy Center - Washington University School of Medicine
St Louis, Missouri, 63141, United States
University of Cincinnati Physicians, Inc.
Cincinnati, Ohio, 45267, United States
Optimed Research, LTD
Columbus, Ohio, 43235, United States
Baker Allergy, Asthma and Dermatology Research Center, LLC
Lake Oswego, Oregon, 97035, United States
Pennsylvania State- Milton S. Hershey Medical Center
Hershey, Pennsylvania, 17033, United States
AARA Research Center
Dallas, Texas, 75231, United States
University of Texas - Medical Branch
Galveston, Texas, 77555, United States
Allergy, Asthma & Immunology Clinic, P.A.
Irving, Texas, 75063, United States
Marycliff Allergy Specialists
Spokane, Washington, 99204, United States
UMHAT "Tsaritsa Yoanna - ISUL"; Clinic of Ear-Nose-Throat Diseases
Sofia, 1527, Bulgaria
Ottawa Allergy Research Corp.
Ottawa, Ontario, KIY 4G2, Canada
Semmelweis University Faculty of Medicine, III Department of Internal Medicine
Budapest, H-1125, Hungary
Allergy, Immunology & Angioedema Center,
Tel Litwinsky, Ramat Gan, 52621, Israel
Bnei-Zion Medical Centre, Clinical Immunology and Allergy Division
Haifa, 31048, Israel
Ospedale Luigi Sacco, Azienda Ospedaliera - Polo Universitario II Divisione di Medicina Interna
Milan, 20157, Italy
P.H.U. Clinic for Dermatology, Medical University Skopje, Unit of Allergology and Clinical Immunology
Skopje, 1000, North Macedonia
Szpital Uniwersytecki w Krakowie, Oddział Kliniczny Klinik Chorób Wewnętrznych, Poradnia Alergologiczna
Krakow, 31-531, Poland
Spitalul Clinic Judeţean Mureş Secţia Clinică Medicină Internă, Compartimentul de Alergologie şi Imunologie
Târgu Mureş, 540103, Romania
Clinic for Immunology and Allergology
Belgrade, 11000, Serbia
Allergy Diagnostic & Clinical Research Unit University of Cape Town Lung Institute
Mowbray, 7700, South Africa
Wits Health Consortium (Pty) Ltd - Wits Donald Gordon Medical Centre
Parktown, 2193, South Africa
MeSH Terms
Conditions
Interventions
Condition Hierarchy (Ancestors)
Intervention Hierarchy (Ancestors)
Results Point of Contact
- Title
- Anurag Relan, Vice President Clinical Research and Medical Affairs
- Organization
- Pharming Technologies
Publication Agreements
- PI is Sponsor Employee
- No
- Restriction Type
- OTHER
- Restrictive Agreement
- Yes
Study Design
- Study Type
- interventional
- Phase
- phase 3
- Allocation
- RANDOMIZED
- Masking
- DOUBLE
- Who Masked
- PARTICIPANT, INVESTIGATOR
- Purpose
- TREATMENT
- Intervention Model
- PARALLEL
- Sponsor Type
- INDUSTRY
- Responsible Party
- SPONSOR
Study Record Dates
First Submitted
August 24, 2010
First Posted
August 25, 2010
Study Start
January 1, 2011
Primary Completion
December 1, 2012
Study Completion
March 1, 2013
Last Updated
August 7, 2015
Results First Posted
August 7, 2015
Record last verified: 2015-07