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

Hereditary Angioedema Clinical Trial

Official title:

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

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT03697187
• Other study ID #: C1 1414
• Status: Completed
• Start date: June 30, 2018
• Est. completion date: July 31, 2021

Study information

• Verified date: March 2020
• Source: Pharming Technologies B.V.
• Contact: n/a
• Is FDA regulated: No
• Study type: Observational [Patient Registry]

Clinical Trial Summary

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

Description:

See below.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 152
• Est. completion date: July 31, 2021
• Est. primary completion date: June 30, 2021
• Accepts healthy volunteers: No
• Gender: All
• Age group: 13 Years and older

Eligibility

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.

Related Conditions & MeSH terms

• Angioedema
• Hereditary Angioedema

Intervention

Drug:
• rhC1INH
Recombinant human C1 inhibitor

Locations

Country	Name	City	State
United States	The US Hereditary Angioedema Association	Fairfax	Virginia

Sponsors (2)

Lead Sponsor	Collaborator
Pharming Technologies B.V.	US Hereditary Angioedema Association

Country where clinical trial is conducted

United States, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	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	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