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

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ClinicalTrials.gov Identifier: NCT01095510
Recruitment Status : Completed
First Posted : March 30, 2010
Results First Posted : July 25, 2014
Last Update Posted : August 5, 2015
Sponsor:
Information provided by (Responsible Party):
Shire

March 24, 2010
March 30, 2010
June 26, 2014
July 25, 2014
August 5, 2015
March 2010
June 2013   (Final data collection date for primary outcome measure)
Presence of Unequivocal Beginning of Relief of the Defining Attack Symptom [ Time Frame: Within 4 hours following treatment ]
Presence of unequivocal beginning of relief of the defining symptom. [ Time Frame: Within 4 hours following treatment ]
Complete list of historical versions of study NCT01095510 on ClinicalTrials.gov Archive Site
  • Time to Unequivocal Beginning of Relief of the Defining Attack Symptom [ Time Frame: Within 4 hours following treatment ]
  • Time to Complete Resolution of the Attack [ Time Frame: 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.
  • Time to unequivocal beginning of relief of the defining symptom. [ Time Frame: Within 4 hours following treatment ]
  • Time to complete resolution of the attack. [ Time Frame: Within 1 week following treatment ]
  • C1INH Levels [ Time Frame: pre-infusion to 1 and 24 hours post-infusion ]
Not Provided
Not Provided
 
CINRYZE for the Treatment of Hereditary Angioedema Attacks in Children Under the Age of 12
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.
Each subject received CINRYZE for treatment of a single acute angioedema attack.
Interventional
Phase 2
Allocation: Non-Randomized
Intervention Model: Parallel Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Hereditary Angioedema
Biological: CINRYZE
Other Name: C1 inhibitor [human]
  • Experimental: 500 U CINRYZE (10-25 kg body weight)
    Single IV dose of 500 U CINRYZE
    Intervention: Biological: CINRYZE
  • Experimental: 1000 U CINRYZE (10-25 kg body weight)
    Single IV dose of 1000 U CINRYZE
    Intervention: Biological: CINRYZE
  • Experimental: 1000 U CINRYZE (>25 kg body weight)
    Single IV dose of 1000 U CINRYZE
    Intervention: Biological: CINRYZE
  • Experimental: 1500 U CINRYZE (>25 kg body weight)
    Single IV dose of 1500 U CINRYZE
    Intervention: Biological: CINRYZE
Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Completed
9
12
June 2013
June 2013   (Final data collection date for primary outcome measure)

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.
Sexes Eligible for Study: All
2 Years to 11 Years   (Child)
No
Contact information is only displayed when the study is recruiting subjects
Germany,   Hungary,   United States
 
 
NCT01095510
0624-203
2011-000369-11 ( EudraCT Number )
No
Not Provided
Not Provided
Shire
Shire
Not Provided
Study Director: Jennifer Schranz, MD Shire
Shire
June 2014

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