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

NCT03697187 | Completed FDA Drug

• 1 other identifier: C1 1414
• Study Type: observational
• Target: 152
• Locations: 1 country
• Sites: 1

Brief Summary

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

Conditions

Hereditary Angioedema

Keywords

Hereditary Angioedema; HAE; Ruconest; rhC1INH; Registry; Pharming

Outcome Measures

Primary Outcomes (1)

• 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 Outcomes (1)

• 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

Study Arms (1)

Hereditary Angioedema

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

Drug: rhC1INH

Interventions

rhC1INH DRUG

Recombinant human C1 inhibitor

Also known as: Ruconest

Hereditary Angioedema

Eligibility Criteria

• Age: 13 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)
• Sampling Method: Probability Sample

Study Population

Eligible patients will be 13 years of age and older, have a current diagnosis of HAE for which they have been prescribed and are currently being treated with Ruconest.

You may qualify if:

• 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.

You may not qualify if:

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

Sponsors & Collaborators

• Pharming Technologies B.V.: lead
• US Hereditary Angioedema Association: collaborator

Study Sites (1)

The US Hereditary Angioedema Association

Fairfax, Virginia, 22030, United States

Location

MeSH Terms

Conditions

Angioedemas, Hereditary

Interventions

conestat alfa

Condition Hierarchy (Ancestors)

Angioedema; Vascular Diseases; Cardiovascular Diseases; Hereditary Complement Deficiency Diseases; Primary Immunodeficiency Diseases; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Urticaria; Skin Diseases, Vascular; Skin Diseases; Skin and Connective Tissue Diseases; Hypersensitivity, Immediate; Hypersensitivity; Immune System Diseases; Immunologic Deficiency Syndromes

Study Officials

• Anurag Relan, MD

  Pharming Technologies BV

  STUDY DIRECTOR

Study Design

• Study Type: observational
• Observational Model: COHORT
• Time Perspective: PROSPECTIVE
• Target Duration: 3 Years
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: August 9, 2018
• First Posted: October 5, 2018
• Study Start: June 30, 2018
• Primary Completion: June 30, 2021
• Study Completion: July 31, 2021
• Last Updated: April 5, 2024

Record last verified: 2020-03

Locations

US (1)