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

Hereditary Angioedema (HAE) Clinical Trial

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

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT01095510
• Other study ID #: 0624-203
• Secondary ID: 2011-000369-11
• Status: Completed
• Phase: Phase 2
• Start date: June 2, 2010
• Est. completion date: April 17, 2012

Study information

• Verified date: May 2021
• Source: Takeda
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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.

Description:

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

Recruitment information / eligibility

• Status: Completed
• Enrollment: 9
• Est. completion date: April 17, 2012
• Est. primary completion date: April 17, 2012
• Accepts healthy volunteers: No
• Gender: All
• Age group: 2 Years to 11 Years

Eligibility

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.

Related Conditions & MeSH terms

• Angioedema
• Hereditary Angioedema (HAE)

Intervention

Biological:
• CINRYZE

Locations

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

Sponsors (1)

Lead Sponsor	Collaborator
Shire

Countries where clinical trial is conducted

United States,  Germany,  Hungary, 

References & Publications (1)

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 Au — View Citation

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Presence of Unequivocal Beginning of Relief of the Defining Attack Symptom		Within 4 hours following treatment
Secondary	Time to Unequivocal Beginning of Relief of the Defining Attack Symptom		Within 4 hours following treatment
Secondary	Time to Complete Resolution of the Attack		Within 1 week following treatment
Secondary	Change in C1 Inhibitor (C1 INH) Antigen and Functional C1 INH Concentrations	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