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

This study has been completed.
Sponsor:
Information provided by (Responsible Party):
Shire
ClinicalTrials.gov Identifier:
NCT01095510
First received: March 24, 2010
Last updated: March 19, 2014
Last verified: March 2014
  Purpose

The objectives of the study are to:

  1. Evaluate the dose response and the pharmacokinetics (PK)/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).
  2. Determine the safety and tolerability following IV administration of Cinryze in this study population.

Condition Intervention Phase
Hereditary Angioedema
Biological: Cinryze
Phase 2

Study Type: Interventional
Study Design: Allocation: Non-Randomized
Endpoint Classification: Safety/Efficacy Study
Intervention Model: Parallel Assignment
Masking: Open Label
Primary Purpose: Treatment
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

Resource links provided by NLM:


Further study details as provided by Shire:

Primary Outcome Measures:
  • Presence of unequivocal beginning of relief of the defining symptom. [ Time Frame: within 4 hours following treatment ] [ Designated as safety issue: No ]

Secondary Outcome Measures:
  • Time to unequivocal beginning of relief of the defining symptom. [ Time Frame: within 4 hours following treatment ] [ Designated as safety issue: No ]
  • Time to complete resolution of the attack. [ Time Frame: within 1 week following treatment ] [ Designated as safety issue: No ]
  • C1INH Levels [ Time Frame: pre-infusion to 1 and 24 hours post-infusion ] [ Designated as safety issue: No ]

Enrollment: 9
Study Start Date: March 2010
Primary Completion Date: June 2013 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: Cohort 1 (≥ 10kg to ≤ 25kg)
500 Unit (U) IV single dose
Biological: Cinryze
IV single dose
Experimental: Cohort 2 (≥ 10kg to ≤ 25kg)
1000 U IV single dose
Biological: Cinryze
IV single dose
Experimental: Cohort 3 (>25kg)
1000 U IV single dose
Biological: Cinryze
IV single dose
Experimental: Cohort 4 (>25kg)
1500 U IV single dose
Biological: Cinryze
IV single dose

  Eligibility

Ages Eligible for Study:   2 Years to 11 Years
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
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 Inhibitor (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.
  Contacts and Locations
Please refer to this study by its ClinicalTrials.gov identifier: NCT01095510

Locations
United States, Colorado
Associate, Asthma & Allergy Associates, P.C.
Colorodo Springs, Colorado, United States, 80709
United States, Florida
USF Asthma, Allergy and Immunology Clinical Research Unit
Tampa, Florida, United States, 33612
United States, Maryland
Institute for Asthma and Allergy
Chevy Chase, Maryland, United States, 20815
United States, Oregon
Allergy and Asthma Research Group
Eugene, Oregon, United States, 97401
Allergy, Asthma and Dermatoligy Research Center, LLC
Lake Oswego, Oregon, United States, 97035
United States, Texas
AARA Research Center
Dallas, Texas, United States, 75231
Allergy and Asthma Research Center
San Antonio, Texas, United States, 78229
United States, Washington
MaryCliff Allergy Specialists
Spokane, Washington, United States, 99204
Germany
ViroPharma Investigational Site
Berlin, Germany
ViroPharma Investigational Site
Munich, Germany
Hungary
ViroPharma Investigational Site
Budapest, Hungary
Sponsors and Collaborators
Shire
Investigators
Study Director: Jennifer Schranz, MD ViroPharma Incorporated
  More Information

No publications provided

Responsible Party: Shire
ClinicalTrials.gov Identifier: NCT01095510     History of Changes
Other Study ID Numbers: 0624-203
Study First Received: March 24, 2010
Last Updated: March 19, 2014
Health Authority: United States: Food and Drug Administration
European Union: European Medicines Agency

Keywords provided by Shire:
HAE
Cinryze
C1 INH
C1 inhibitor
Pediatric
PK/PD

Additional relevant MeSH terms:
Angioedemas, Hereditary
Angioedema
Vascular Diseases
Cardiovascular Diseases
Urticaria
Skin Diseases, Vascular
Skin Diseases
Hypersensitivity, Immediate
Hypersensitivity
Immune System Diseases
Genetic Diseases, Inborn
Complement C1 Inhibitor Protein
Complement Inactivating Agents
Immunosuppressive Agents
Immunologic Factors
Physiological Effects of Drugs
Pharmacologic Actions

ClinicalTrials.gov processed this record on April 17, 2014