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

April 3, 2024 updated by: Pharming Technologies B.V.

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

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

Study Overview

Status

Completed

Conditions

Intervention / Treatment

Detailed Description

See below.

Study Type

Observational

Enrollment (Actual)

152

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • United States
    • Virginia
      • Fairfax, Virginia, United States, 22030
        • The US Hereditary Angioedema Association

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

13 years and older (Child, Adult, Older Adult)

Accepts Healthy Volunteers

No

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.

Description

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 Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

Cohorts and Interventions

Group / Cohort
Intervention / Treatment
Hereditary Angioedema
Patients with Hereditary Angioedema who are receiving treatment with Ruconest (rhC1INH).
Drug: rhC1INH
Recombinant human C1 inhibitor
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.
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.
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.
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.
3 years

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Pharming Technologies B.V.

Collaborators

US Hereditary Angioedema Association

Investigators

  • Study Director: Anurag Relan, MD, Pharming Technologies BV

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

June 30, 2018

Primary Completion (Actual)

June 30, 2021

Study Completion (Actual)

July 31, 2021

Study Registration Dates

First Submitted

August 9, 2018

First Submitted That Met QC Criteria

October 3, 2018

First Posted (Actual)

October 5, 2018

Study Record Updates

Last Update Posted (Actual)

April 5, 2024

Last Update Submitted That Met QC Criteria

April 3, 2024

Last Verified

March 1, 2020

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • C1 1414

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

Yes

Studies a U.S. FDA-regulated device product

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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