Patient Registry to Evaluate the Real-world Safety of Ruconest®

Study Overview
=================
Brief Summary
-----------------
This is a prospective, real-world, observational patient registry for patients with HAE who are receiving treatment with Ruconest for HAE.

Detailed Description
-----------------
See below.

Official Title
-----------------
An Observational Patient Registry to Evaluate the Real-world Safety of Ruconest® (C1 Esterase Inhibitor [Recombinant]) for the Treatment of Hereditary Angioedema

Conditions
-----------------
Hereditary Angioedema

Intervention / Treatment
-----------------
* Drug: rhC1INH


Participation Criteria
=================
Eligibility Criteria
-----------------
Inclusion Criteria: Patient provides informed consent as documented on the Institutional Review Board (IRB) approved informed consent document (ICF). For patients aged between 13 and 17 years old, the method of consent with or without assent will be determined by the IRB. Patient is male or female and at least 13 years of age at the time of providing consent / assent. Patient has been prescribed Ruconest for HAE. Exclusion Criteria: 1. Patient is receiving HAE therapy as part of a clinical trial.

Ages Eligible for Study
-----------------
Minimum Age: 13 Years

Sexes Eligible for Study
-----------------
All

Accepts Healthy Volunteers
-----------------
No

Study Plan
=================
How is the study designed?
-----------------

Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Hereditary Angioedema<br>Patients with Hereditary Angioedema who are receiving treatment with Ruconest (rhC1INH). | Drug: rhC1INH<br>* Recombinant human C1 inhibitor<br>* Other names: Ruconest;|

What is the study measuring?
-----------------
Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Safety analysis will consist of AEs reported per patient for up to 30 days after each single or repeated dose of Ruconest. | In addition, extent of exposure to Ruconest and concomitant medications taken for HAE will be summarized. Concomitant medications will be limited to those specifically indicated for treatment AEs or treatment of symptoms of HAE, for example C1-inhibitors, epinephrine, IV fluids, etc. AEs will be summarized as incidence per 10,000 person-days where each patient counts only once for multiple events of the same System Organ Class and preferred terms within the 30-day evaluation period. The person-time duration for each AE for each patient is the time period (in days) between the dose of Ruconest and AE onset. | 3 years | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| AEs occurring during pregnancy or lactation and for breastfed infants will be summarized separately using the same methods as described in the primary outcome measure. | Any pregnant woman who treats with Ruconest while on the Registry will be followed up to twelve weeks post-delivery or termination. Those treated with Ruconest while actively nursing will be followed until end of lactation. | 3 years | 

 Terms related to the study
=================
Keywords Provided by Centre Hospitalier Valida
-----------------
HAE, Hereditary Angioedema, Ruconest, rhC1INH, Registry, Pharming