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

Sponsor:

Collaborators:

Information provided by (Responsible Party):

，

Tracking Information

First Submitted Date ^ICMJE

August 9, 2018

First Posted Date ^ICMJE

October 5, 2018

Last Update Posted Date

October 5, 2018

Actual Study Start Date ^ICMJE

July 16, 2018

Estimated Primary Completion Date

July 16, 2021 (Final data collection date for primary outcome measure)

Current Primary Outcome Measures ^ICMJE

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.

Original Primary Outcome Measures ^ICMJE

Same as current

Current Secondary Outcome Measures ^ICMJE

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.

Descriptive Information

Brief Title ^ICMJE

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

Official Title ^ICMJE

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

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 Type ^ICMJE

Observational [Patient Registry]

Study Phase

Study Design ^ICMJE

Allocation:
Intervention Model:
Intervention Model Description:
Masking: Observational [Patient Registry]
Masking Description:
Primary Purpose:

Condition ^ICMJE

Intervention ^ICMJE

Drug: rhC1INH
Recombinant human C1 inhibitor

Study Arms

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

Recruitment Information

Recruitment Status ^ICMJE

Recruiting

Estimated Enrollment ^ICMJE

303

Original Estimated Enrollment ^ICMJE

Same as current

Estimated Study Completion Date

August 16, 2021

Estimated Primary Completion Date

July 16, 2021 (Final data collection date for primary outcome measure)

Eligibility Criteria ^ICMJE

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.

Sex/Gender

Sexes Eligible for Study:

All

Ages

13 Years and older (Adult, Older Adult)

Accepts Healthy Volunteers

Listed Location Countries ^ICMJE

United States

Removed Location Countries

Administrative Information

Has Data Monitoring Committee

U.S. FDA-regulated Product

Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No

IPD Sharing Statement

Plan to Share IPD:

Responsible Party

，

Study Sponsor ^ICMJE

Pharming Technologies B.V.

Collaborators ^ICMJE

Investigators ^ICMJE

Study Director: Anurag Relan, MD Pharming Technologies BV

PRS Account

Verification Date

October 2018

^ICMJE Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP

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