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

Completed

• Conditions: Hereditary Angioedema
• Interventions: Drug: rhC1INH

• Registration Number: NCT03697187
• Lead Sponsor: Pharming Technologies B.V.

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.

Study Timeline

• Study Start: 2018-06-30
• Study First Submitted: 2018-08-09
• Study First Submitted QC: 2018-10-03
• Study First Posted: 2018-10-05
• Status Verified: 2020-03
• Primary Completion: 2021-06-30
• Study Completion: 2021-07-31
• Last Update Submitted: 2024-04-03
• Last Update Posted: 2024-04-05

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 152

Inclusion Criteria

1. 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.
2. Patient is male or female and at least 13 years of age at the time of providing consent / assent.
3. Patient has been prescribed Ruconest for HAE.

Exclusion Criteria

1. Patient is receiving HAE therapy as part of a clinical trial.

Study & Design

• Study Type: OBSERVATIONAL
• Study Design: Not specified

Arm && Interventions

Group	Intervention	Description
Hereditary Angioedema	rhC1INH	Patients with Hereditary Angioedema who are receiving treatment with Ruconest (rhC1INH).

Primary Outcome Measures

Name	Time	Method
Safety analysis will consist of AEs reported per patient for up to 30 days after each single or repeated dose of Ruconest.	3 years	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.

Secondary Outcome Measures

Name	Time	Method
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.	3 years	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.

Trial Locations

Locations (1)

The US Hereditary Angioedema Association; 🇺🇸 Fairfax, Virginia, United States