A Phase 2 HAE Prophylaxis Study With Recombinant Human C1 Inhibitor

Study Overview
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Brief Summary
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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
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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.

Official Title
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A Phase 2 Multicenter, Randomized, Double-Blind, Placebo-Controlled, 3-Period Crossover Study to Evaluate the Efficacy and Safety of Recombinant Human C1 Inhibitor in the Prophylaxis of Angioedema Attacks in Patients With Hereditary Angioedema (HAE)

Conditions
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Hereditary Angioedema

Intervention / Treatment
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* Biological: Recombinant human C1 inhibitor
* Other: Placebo


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: 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 Exclusion Criteria: 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

Ages Eligible for Study
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Minimum Age: 13 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Prevention
Allocation: Randomized
Intervention Model: Crossover Assignment
Masking: Quadruple


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: rhC1INH twice weekly<br>rhC1INH administered twice weekly | Biological: Recombinant human C1 inhibitor<br> <br> * Other names: Conestat alfa;|
| Experimental: rhC1INH once weekly<br>rhC1INH administered once weekly | Biological: Recombinant human C1 inhibitor<br> <br> * Other names: Conestat alfa;|
| Placebo Comparator: Placebo (Saline) twice weekly<br>Placebo (Saline) administered twice weekly | Other: Placebo<br> <br> * Other names: Saline;|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Number of HAE Attacks | Average number of HAE attacks normalized to a 28 day period | 28 days | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Number of Participants With Adverse Events | Number of participants that experienced Treatment Emergent Adverse Events observed in safety population | 20 weeks | 
| Percentage of Participants Achieving at Least 50% Reduction in Number of Attacks | 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 | 28 days | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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HAE, Hereditary Angioedema, Prophylaxis