A Phase 2 HAE Prophylaxis Study With Recombinant Human C1 Inhibitor
- Conditions
- Hereditary Angioedema
- Registration Number
- NCT02247739
- Lead Sponsor
- Pharming Technologies B.V.
- Brief Summary
Primary Objective:
To evaluate the efficacy of recombinant human C1 inhibitor (rhC1INH) in the prophylaxis of angioedema attacks in patients with HAE
Secondary Objective:
To evaluate the safety and immunogenicity of recombinant human C1 inhibitor (rhC1INH) in the prophylaxis of angioedema attacks in patients with HAE
- Detailed Description
Study Design:
This is a multi-center, randomized, double-blind, placebo-controlled, 3-period crossover study of rhC1INH in prophylaxis of angioedema attacks in patients with HAE.
Medical screening (clinical and laboratory parameters) will be performed and patient medical history specific to HAE attacks will be collected to assess eligibility. Each patient will receive three 4 week periods of treatment twice weekly.
Recruitment & Eligibility
- Status
- COMPLETED
- Sex
- All
- Target Recruitment
- 32
Patients fulfilling the following criteria at Screening are eligible for participation in the study:
- Age 13 years or older
- Laboratory confirmed diagnosis of HAE
- A history of frequent HAE attacks (at least 4 attacks per month across a minimum of 3 consecutive months).
- Female patients of childbearing potential who are sexually active must be willing to use an acceptable form of contraception.
- Provided written informed consent (and written assent for minors)
- Willingness and ability to comply with all protocol procedures
Patients who meet any of the following criteria at Screening are to be excluded from study participation:
- Patients with medical history of allergy to rabbits or rabbit-derived products (including rhC1INH)
- Diagnosis of acquired angioedema (AAE)
- Patients who are pregnant, or breastfeeding, or are currently intending to become pregnant
- Treatment with any investigational drug in the past 30 days
- Patients with any condition or treatment that, in the opinion of the Investigator, might interfere with the evaluation of study objectives
- Patients currently treated with angiotensin-converting enzyme (ACE) inhibitors
Study & Design
- Study Type
- INTERVENTIONAL
- Study Design
- CROSSOVER
- Primary Outcome Measures
Name Time Method Number of HAE Attacks 28 days Average number of HAE attacks normalized to a 28 day period
- Secondary Outcome Measures
Name Time Method Number of Participants With Adverse Events 20 weeks Number of participants that experienced Treatment Emergent Adverse Events observed in safety population
Percentage of Participants Achieving at Least 50% Reduction in Number of Attacks 28 days Percentage of participants achieving at least 50% reduction in the number of attacks normalized to a 28-day period as compared to the placebo treatment period
Trial Locations
- Locations (10)
University of South Florida Asthma, Allergy and Immunology Clinical Research Unit
🇺🇸Tampa, Florida, United States
University of South Florida, Asthma, Allergy & Immunology Clinical Research Unit
🇺🇸Tampa, Florida, United States
Washington University Division of Allergy and Immunology
🇺🇸Saint Louis, Missouri, United States
Baker Allergy, Asthma and Dermatology Research Center
🇺🇸Lake Oswego, Oregon, United States
Ottawa Allergy Research Corp
🇨🇦Ottawa, Ontario, Canada
Faculty Hospital by St. Anna Brno, Department of clinical Immunology and Allergology
🇨🇿Brno, Czechia
Azienda Ospedaliera Universitaria Luigi Sacco Di Milano
🇮🇹Milan, Italy
PHI University Clinic of Dermatology
🇲🇰Skopje, Macedonia, The Former Yugoslav Republic of
SC Centrul Clinic Mediquest SRL
🇷🇴Sângeorgiu de Mureş, Mures, Romania
Clinical Center Serbia
🇷🇸Belgrade, Serbia
University of South Florida Asthma, Allergy and Immunology Clinical Research Unit🇺🇸Tampa, Florida, United States