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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 : December 12, 2018
Sponsor:
Information provided by (Responsible Party):
Shire

Tracking Information
First Submitted Date  ICMJE March 24, 2010
First Posted Date  ICMJE March 30, 2010
Results First Submitted Date  ICMJE June 26, 2014
Results First Posted Date  ICMJE July 25, 2014
Last Update Posted Date December 12, 2018
Actual Study Start Date  ICMJE June 2, 2010
Actual Primary Completion Date April 17, 2012   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: June 26, 2014)
Presence of Unequivocal Beginning of Relief of the Defining Attack Symptom [ Time Frame: Within 4 hours following treatment ]
Original Primary Outcome Measures  ICMJE
 (submitted: March 26, 2010)
Presence of unequivocal beginning of relief of the defining symptom. [ Time Frame: Within 4 hours following treatment ]
Change History Complete list of historical versions of study NCT01095510 on ClinicalTrials.gov Archive Site
Current Secondary Outcome Measures  ICMJE
 (submitted: July 14, 2015)
  • 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.
Original Secondary Outcome Measures  ICMJE
 (submitted: March 26, 2010)
  • 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 ]
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title  ICMJE CINRYZE for the Treatment of Hereditary Angioedema Attacks in Children Under the Age of 12
Official Title  ICMJE 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
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.
Study Type  ICMJE Interventional
Study Phase  ICMJE Phase 2
Study Design  ICMJE Allocation: Non-Randomized
Intervention Model: Parallel Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Condition  ICMJE Hereditary Angioedema (HAE)
Intervention  ICMJE Biological: CINRYZE
Other Name: C1 inhibitor [human]
Study Arms  ICMJE
  • 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
Publications * 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.

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Completed
Actual Enrollment  ICMJE
 (submitted: November 15, 2013)
9
Original Estimated Enrollment  ICMJE
 (submitted: March 26, 2010)
12
Actual Study Completion Date  ICMJE April 17, 2012
Actual Primary Completion Date April 17, 2012   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

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.
Sex/Gender  ICMJE
Sexes Eligible for Study: All
Ages  ICMJE 2 Years to 11 Years   (Child)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE Contact information is only displayed when the study is recruiting subjects
Listed Location Countries  ICMJE Germany,   Hungary,   United States
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT01095510
Other Study ID Numbers  ICMJE 0624-203
2011-000369-11 ( EudraCT Number )
Has Data Monitoring Committee No
U.S. FDA-regulated Product Not Provided
IPD Sharing Statement  ICMJE Not Provided
Responsible Party Shire
Study Sponsor  ICMJE Shire
Collaborators  ICMJE Not Provided
Investigators  ICMJE
Study Director: Study Director Shire
PRS Account Shire
Verification Date November 2018

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