Clinical trial NCT01095510: 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
Overall status: COMPLETED
Phase: PHASE2
Conditions: Hereditary Angioedema (HAE)
Keywords: CINRYZE
C1 INH
HAE
C1 inhibitor
Pediatric
PK/PD
Lead sponsor: Takeda
Sponsor class: INDUSTRY
First posted: 2010-03-30
Enrollment: 9
Sex: ALL
Minimum age (years): 2
Maximum age (years): 11
Standard ages: CHILD
Accepts healthy volunteers: no
Allocation: NON_RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

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.

Eligibility criteria: 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.

Primary outcomes:
measure: Presence of Unequivocal Beginning of Relief of the Defining Attack Symptom
timeFrame: Within 4 hours following treatment
Secondary outcomes:
measure: Time to Unequivocal Beginning of Relief of the Defining Attack Symptom
timeFrame: Within 4 hours following treatment
measure: Time to Complete Resolution of the Attack
timeFrame: Within 1 week following treatment
measure: Change in C1 Inhibitor (C1 INH) Antigen and Functional C1 INH Concentrations
timeFrame: Pre-dose, 2, 4, 8 hours post dose on Day 1; Day 2, 3, 5, 8
Other outcomes:
Locations:
city: Colorado Springs
country: United States
facility: Asthma & Allergy Associates, P.C.
state: Colorado
city: Tampa
country: United States
facility: University of South Florida Asthma, Allergy and Immunology Clinical Research Unit
state: Florida
city: Chevy Chase
country: United States
facility: Institute for Asthma and Allergy, PC
state: Maryland
city: Eugene
country: United States
facility: Allergy & Asthma Research Group
state: Oregon
city: Lake Oswego
country: United States
facility: Baker Allergy, Asthma and Dermatology Research Center, LLC
state: Oregon
city: Dallas
country: United States
facility: AARA Research Center
state: Texas
city: San Antonio
country: United States
facility: Allergy and Asthma Research Center, P.A.
state: Texas
city: Spokane
country: United States
facility: Marycliff Allergy Specialists
state: Washington
city: Berlin
country: Germany
facility: Charité Universitätsmedizin Berlin, Dept. of Dermatology and Allergy
city: Munich
country: Germany
facility: Klinikum rechts der Isar, Technical University Munich, ENT Clinic
city: Budapest
country: Hungary
facility: Semmelweis University, Allergy and Angioedema Outpatients Clinic, Kútvölgyi Clinical Center
Study officials:
affiliation: Takeda
name: Study Director
role: STUDY_DIRECTOR
References:
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.