A Phase 2 HAE Prophylaxis Study With Recombinant Human C1 Inhibitor

Phase 2

Completed

• Conditions: Hereditary Angioedema
• Interventions: Biological: Recombinant human C1 inhibitor; Other: Placebo

• 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.

Study Timeline

• Study First Submitted: 2014-09-16
• Study First Submitted QC: 2014-09-19
• Study First Posted: 2014-09-25
• Study Start: 2014-12
• Primary Completion: 2016-05
• Study Completion: 2016-09
• Results First Submitted: 2017-07-26
• Status Verified: 2017-11
• Results First Submitted QC: 2017-11-07
• Last Update Submitted: 2017-11-07
• Results First Posted: 2017-12-08
• Last Update Posted: 2017-12-08

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 32

Inclusion Criteria

Patients fulfilling the following criteria at Screening are eligible for participation in the study:

1. Age 13 years or older
2. Laboratory confirmed diagnosis of HAE
3. A history of frequent HAE attacks (at least 4 attacks per month across a minimum of 3 consecutive months).
4. Female patients of childbearing potential who are sexually active must be willing to use an acceptable form of contraception.
5. Provided written informed consent (and written assent for minors)
6. Willingness and ability to comply with all protocol procedures

Exclusion Criteria

Patients who meet any of the following criteria at Screening are to be excluded from study participation:

1. Patients with medical history of allergy to rabbits or rabbit-derived products (including rhC1INH)
2. Diagnosis of acquired angioedema (AAE)
3. Patients who are pregnant, or breastfeeding, or are currently intending to become pregnant
4. Treatment with any investigational drug in the past 30 days
5. Patients with any condition or treatment that, in the opinion of the Investigator, might interfere with the evaluation of study objectives
6. Patients currently treated with angiotensin-converting enzyme (ACE) inhibitors

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: CROSSOVER

Arm && Interventions

Group	Intervention	Description
rhC1INH twice weekly	Recombinant human C1 inhibitor	rhC1INH administered twice weekly
rhC1INH once weekly	Recombinant human C1 inhibitor	rhC1INH administered once weekly
Placebo (Saline) twice weekly	Placebo	Placebo (Saline) administered twice weekly

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)

Washington University Division of Allergy and Immunology; 🇺🇸 Saint Louis, Missouri, United States

Faculty Hospital by St. Anna Brno, Department of clinical Immunology and Allergology; 🇨🇿 Brno, Czechia

PHI University Clinic of Dermatology; 🇲🇰 Skopje, Macedonia, The Former Yugoslav Republic of

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

Azienda Ospedaliera Universitaria Luigi Sacco Di Milano; 🇮🇹 Milan, Italy

Baker Allergy, Asthma and Dermatology Research Center; 🇺🇸 Lake Oswego, Oregon, United States

Ottawa Allergy Research Corp; 🇨🇦 Ottawa, Ontario, Canada

SC Centrul Clinic Mediquest SRL; 🇷🇴 Sângeorgiu de Mureş, Mures, Romania

Clinical Center Serbia; 🇷🇸 Belgrade, Serbia