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OpenTrials
Completed

NCT Number: NCT03697187

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

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

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Key information

Conditions

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

Age range

13 year and older

Sex eligibility

All sexes

Study type

Observational

Primary location

The US Hereditary Angioedema Association

Fairfax, Virginia, 22030, United States

About this study

See below.

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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

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

Treatment and study plan

rhC1INH

Drug

Recombinant human C1 inhibitor

Other names: Ruconest

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.

    Time frame: 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 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.

    Time frame: 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.

Sponsors and collaborators

Lead sponsor

Pharming Technologies B.V.

Industry

Collaborators

  • US Hereditary Angioedema Association

Registry information

Official study title

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

Important dates

Study start
2018
Primary completion
2021
Study completion
2021
First posted
Oct 5, 2018
Registry last updated
Apr 5, 2024

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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