CINRYZE for the Treatment of Hereditary Angioedema Attacks in Children Under the Age of 12

May 11, 2021 updated by: Shire

Open-Label, Single-Dose Study to Evaluate the Response and Pharmacokinetics/Pharmacodynamics of Different Doses of CINRYZE® [C1 Inhibitor (Human)] For Treatment of Acute Angioedema Attacks in Children Less Than 12 Years of Age With Hereditary Angioedema

The objectives of this study were to evaluate: (1) the dose response and (2) the pharmacokinetics (PK) and pharmacodynamics (PD) of intravenous (IV) administration of CINRYZE for the treatment of acute angioedema attacks in children above and below 25 kg and less than 12 years of age with hereditary angioedema (HAE); and (3) to determine the safety and tolerability following IV administration of CINRYZE in this study population.

Study Overview

Status

Completed

Conditions

Intervention / Treatment

Detailed Description

Each subject received CINRYZE for treatment of a single acute angioedema attack.

Study Type

Interventional

Enrollment (Actual)

9

Phase

  • Phase 2

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

  • Germany
      • Berlin, Germany
        • Charité Universitätsmedizin Berlin, Dept. of Dermatology and Allergy
      • Munich, Germany
        • Klinikum rechts der Isar, Technical University Munich, ENT Clinic
  • Hungary
      • Budapest, Hungary
        • Semmelweis University, Allergy and Angioedema Outpatients Clinic, Kútvölgyi Clinical Center
  • United States
    • Colorado
      • Colorado Springs, Colorado, United States, 80907
        • Asthma & Allergy Associates, P.C.
    • Florida
      • Tampa, Florida, United States, 33613
        • University of South Florida Asthma, Allergy and Immunology Clinical Research Unit
    • Maryland
      • Chevy Chase, Maryland, United States, 20815
        • Institute for Asthma and Allergy, PC
    • Oregon
      • Eugene, Oregon, United States, 97401
        • Allergy & Asthma Research Group
      • Lake Oswego, Oregon, United States, 97035
        • Baker Allergy, Asthma and Dermatology Research Center, LLC
    • Texas
      • Dallas, Texas, United States, 75231
        • AARA Research Center
      • San Antonio, Texas, United States, 78229
        • Allergy and Asthma Research Center, P.A.
    • Washington
      • Spokane, Washington, United States, 99204
        • Marycliff Allergy Specialists

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

2 years to 11 years (CHILD)

Accepts Healthy Volunteers

No

Genders Eligible for Study

All

Description

Inclusion Criteria:

To be eligible for this protocol, subjects must:

  1. Be at least 10 kg of body weight.
  2. Have a confirmed diagnosis of HAE.
  3. Have an acute HAE attack and be able to initiate treatment within 8 hours after onset of symptoms.

Exclusion Criteria:

To be eligible for this protocol, subjects must not:

  1. Have any active infectious illness.
  2. Have had a prior HAE attack and/or received any C1 INH product within 7 days prior to dosing with study drug.
  3. Have received therapy with antifibrinolytics (e.g., tranexamic acid), androgens (e.g., danazol, oxandrolone, stanozolol, or testosterone), ecallantide (Kalbitor®), or icatibant (Firazyr®) within 7 days prior to dosing with study drug.
  4. Have a history of allergic reaction to C1 INH products, including CINRYZE (or any of the components of CINRYZE), or other blood products.
  5. Have participated in any other investigational drug evaluation within 30 days prior to dosing with study drug, or have previously received treatment with CINRYZE in this study at any time.

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

  • Primary Purpose: TREATMENT
  • Allocation: NON_RANDOMIZED
  • Interventional Model: PARALLEL
  • Masking: NONE

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
EXPERIMENTAL: 500 U CINRYZE (10-25 kg body weight)
Single IV dose of 500 U CINRYZE
Biological: CINRYZE
Other Names:
  • C1 inhibitor [human]
EXPERIMENTAL: 1000 U CINRYZE (10-25 kg body weight)
Single IV dose of 1000 U CINRYZE
Biological: CINRYZE
Other Names:
  • C1 inhibitor [human]
EXPERIMENTAL: 1000 U CINRYZE (>25 kg body weight)
Single IV dose of 1000 U CINRYZE
Biological: CINRYZE
Other Names:
  • C1 inhibitor [human]
EXPERIMENTAL: 1500 U CINRYZE (>25 kg body weight)
Single IV dose of 1500 U CINRYZE
Biological: CINRYZE
Other Names:
  • C1 inhibitor [human]

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Time Frame
Presence of Unequivocal Beginning of Relief of the Defining Attack Symptom
Time Frame: Within 4 hours following treatment
Within 4 hours following treatment

Secondary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Time to Unequivocal Beginning of Relief of the Defining Attack Symptom
Time Frame: Within 4 hours following treatment
Within 4 hours following treatment
Time to Complete Resolution of the Attack
Time Frame: Within 1 week following treatment
Within 1 week following treatment
Change in C1 Inhibitor (C1 INH) Antigen and Functional C1 INH Concentrations
Time Frame: Pre-dose, 2, 4, 8 hours post dose on Day 1; Day 2, 3, 5, 8
Data was not reported due to change in planned analysis.
Pre-dose, 2, 4, 8 hours post dose on Day 1; Day 2, 3, 5, 8

Collaborators and Investigators

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

Sponsor

Shire

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

General Publications

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 2, 2010

Primary Completion (ACTUAL)

April 17, 2012

Study Completion (ACTUAL)

April 17, 2012

Study Registration Dates

First Submitted

March 24, 2010

First Submitted That Met QC Criteria

March 26, 2010

First Posted (ESTIMATE)

March 30, 2010

Study Record Updates

Last Update Posted (ACTUAL)

June 3, 2021

Last Update Submitted That Met QC Criteria

May 11, 2021

Last Verified

May 1, 2021

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • 0624-203
  • 2011-000369-11 (EUDRACT_NUMBER)

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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