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

Primary Purpose

Hereditary Angioedema (HAE)

Status

Completed

Phase

Phase 2

Locations

International

Study Type

Interventional

Intervention

CINRYZE

About this trial

This is an interventional treatment trial for Hereditary Angioedema (HAE) focused on measuring CINRYZE, C1 INH, HAE, C1 inhibitor, Pediatric, PK/PD

Eligibility Criteria

2 Years - 11 Years (Child)All SexesDoes not accept healthy volunteers

Inclusion Criteria:

To be eligible for this protocol, subjects must:

Be at least 10 kg of body weight.
Have a confirmed diagnosis of HAE.
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:

Have any active infectious illness.
Have had a prior HAE attack and/or received any C1 INH product within 7 days prior to dosing with study drug.
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.
Have a history of allergic reaction to C1 INH products, including CINRYZE (or any of the components of CINRYZE), or other blood products.
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.

Sites / Locations

Asthma & Allergy Associates, P.C.
University of South Florida Asthma, Allergy and Immunology Clinical Research Unit
Institute for Asthma and Allergy, PC
Allergy & Asthma Research Group
Baker Allergy, Asthma and Dermatology Research Center, LLC
AARA Research Center
Allergy and Asthma Research Center, P.A.
Marycliff Allergy Specialists
Charité Universitätsmedizin Berlin, Dept. of Dermatology and Allergy
Klinikum rechts der Isar, Technical University Munich, ENT Clinic
Semmelweis University, Allergy and Angioedema Outpatients Clinic, Kútvölgyi Clinical Center

Arms of the Study

Arm 1

Arm 2

Arm 3

Arm 4

Arm Type

Experimental

Arm Label

500 U CINRYZE (10-25 kg body weight)

1000 U CINRYZE (10-25 kg body weight)

1000 U CINRYZE (>25 kg body weight)

1500 U CINRYZE (>25 kg body weight)

Arm Description

Single IV dose of 500 U CINRYZE

Single IV dose of 1000 U CINRYZE

Single IV dose of 1500 U CINRYZE

Outcomes

Primary Outcome Measures

Presence of Unequivocal Beginning of Relief of the Defining Attack Symptom

Secondary Outcome Measures

Time to Unequivocal Beginning of Relief of the Defining Attack Symptom

Time to Complete Resolution of the Attack

Change in C1 Inhibitor (C1 INH) Antigen and Functional C1 INH Concentrations

Data was not reported due to change in planned analysis.

Full Information

NCT ID

NCT01095510

First Posted

March 24, 2010

Last Updated

May 11, 2021

Sponsor

Shire

1. Study Identification

Unique Protocol Identification Number

NCT01095510

Brief Title

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

Official Title

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

Study Type

Interventional

2. Study Status

Record Verification Date

May 2021

Overall Recruitment Status

Completed

Study Start Date

June 2, 2010 (Actual)

Primary Completion Date

April 17, 2012 (Actual)

Study Completion Date

April 17, 2012 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title

Sponsor

Name of the Sponsor

Shire

4. Oversight

Data Monitoring Committee

5. Study Description

Brief Summary

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.

Detailed Description

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

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study

Hereditary Angioedema (HAE)

Keywords

CINRYZE, C1 INH, HAE, C1 inhibitor, Pediatric, PK/PD

7. Study Design

Primary Purpose

Treatment

Study Phase

Phase 2

Interventional Study Model

Parallel Assignment

Masking

None (Open Label)

Allocation

Non-Randomized

Enrollment

9 (Actual)

8. Arms, Groups, and Interventions

Arm Title

500 U CINRYZE (10-25 kg body weight)

Arm Type

Experimental

Arm Description

Single IV dose of 500 U CINRYZE

Arm Title

1000 U CINRYZE (10-25 kg body weight)

Arm Type

Experimental

Arm Description

Single IV dose of 1000 U CINRYZE

Arm Title

1000 U CINRYZE (>25 kg body weight)

Arm Type

Experimental

Arm Description

Single IV dose of 1000 U CINRYZE

Arm Title

1500 U CINRYZE (>25 kg body weight)

Arm Type

Experimental

Arm Description

Single IV dose of 1500 U CINRYZE

Intervention Type

Biological

Intervention Name(s)

CINRYZE

Other Intervention Name(s)

C1 inhibitor [human]

Primary Outcome Measure Information:

Title

Presence of Unequivocal Beginning of Relief of the Defining Attack Symptom

Time Frame

Within 4 hours following treatment

Secondary Outcome Measure Information:

Title

Time to Unequivocal Beginning of Relief of the Defining Attack Symptom

Time Frame

Within 4 hours following treatment

Title

Time to Complete Resolution of the Attack

Time Frame

Within 1 week following treatment

Title

Change in C1 Inhibitor (C1 INH) Antigen and Functional C1 INH Concentrations

Description

Data was not reported due to change in planned analysis.

Time Frame

Pre-dose, 2, 4, 8 hours post dose on Day 1; Day 2, 3, 5, 8

10. Eligibility

Sex

All

Minimum Age & Unit of Time

2 Years

Maximum Age & Unit of Time

11 Years

Accepts Healthy Volunteers

Eligibility Criteria

Inclusion Criteria: To be eligible for this protocol, subjects must: Be at least 10 kg of body weight. Have a confirmed diagnosis of HAE. 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: Have any active infectious illness. Have had a prior HAE attack and/or received any C1 INH product within 7 days prior to dosing with study drug. 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. Have a history of allergic reaction to C1 INH products, including CINRYZE (or any of the components of CINRYZE), or other blood products. 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.

Overall Study Officials:

First Name & Middle Initial & Last Name & Degree

Study Director

Organizational Affiliation

Takeda

Official's Role

Study Director

Facility Information:

Facility Name

Asthma & Allergy Associates, P.C.

City

Colorado Springs

State/Province

Colorado

ZIP/Postal Code

80907

Country

United States

Facility Name

University of South Florida Asthma, Allergy and Immunology Clinical Research Unit

City

Tampa

State/Province

Florida

ZIP/Postal Code

33613

Country

United States

Facility Name

Institute for Asthma and Allergy, PC

City

Chevy Chase

State/Province

Maryland

ZIP/Postal Code

20815

Country

United States

Facility Name

Allergy & Asthma Research Group

City

Eugene

State/Province

Oregon

ZIP/Postal Code

97401

Country

United States

Facility Name

Baker Allergy, Asthma and Dermatology Research Center, LLC

City

Lake Oswego

State/Province

Oregon

ZIP/Postal Code

97035

Country

United States

Facility Name

AARA Research Center

City

Dallas

State/Province

Texas

ZIP/Postal Code

75231

Country

United States

Facility Name

Allergy and Asthma Research Center, P.A.

City

San Antonio

State/Province

Texas

ZIP/Postal Code

78229

Country

United States

Facility Name

Marycliff Allergy Specialists

City

Spokane

State/Province

Washington

ZIP/Postal Code

99204

Country

United States

Facility Name

Charité Universitätsmedizin Berlin, Dept. of Dermatology and Allergy

City

Berlin

Country

Germany

Facility Name

Klinikum rechts der Isar, Technical University Munich, ENT Clinic

City

Munich

Country

Germany

Facility Name

Semmelweis University, Allergy and Angioedema Outpatients Clinic, Kútvölgyi Clinical Center

City

Budapest

Country

Hungary

12. IPD Sharing Statement

Citations:

PubMed Identifier

26171584

Citation

Lumry W, Soteres D, Gower R, Jacobson KW, Li HH, Chen H, Schranz J. Safety and efficacy of C1 esterase inhibitor for acute attacks in children with hereditary angioedema. Pediatr Allergy Immunol. 2015 Nov;26(7):674-80. doi: 10.1111/pai.12444. Epub 2015 Aug 11.

Results Reference

result

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

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