Trial record 14 of 55 for:    "Hereditary angioedema"

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: June 26, 2014
Last verified: June 2014
  Purpose

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.


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 Attack 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 Attack 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 ]
  • Change in C1 Inhibitor (C1 INH) Antigen and Functional C1 INH Concentrations [ Time Frame: Pre-infusion to 1 hour and 24 hours post-infusion ] [ Designated as safety issue: No ]

Enrollment: 9
Study Start Date: March 2010
Study Completion Date: June 2013
Primary Completion Date: June 2013 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: 500 U CINRYZE (10-25 kg body weight)
Single IV dose of 500 U CINRYZE
Biological: CINRYZE
Other Name: C1 inhibitor [human]
Experimental: 1000 U CINRYZE (10-25 kg body weight)
Single IV dose of 1000 U CINRYZE
Biological: CINRYZE
Other Name: C1 inhibitor [human]
Experimental: 1000 U CINRYZE (>25 kg body weight)
Single IV dose of 1000 U CINRYZE
Biological: CINRYZE
Other Name: C1 inhibitor [human]
Experimental: 1500 U CINRYZE (>25 kg body weight)
Single IV dose of 1500 U CINRYZE
Biological: CINRYZE
Other Name: C1 inhibitor [human]

Detailed Description:

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

  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 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
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT01095510

Locations
United States, Colorado
Asthma & Allergy Associates, P.C.
Colorado Springs, Colorado, United States, 80907
United States, Florida
University of South Florida Asthma, Allergy and Immunology Clinical Research Unit
Tampa, Florida, United States, 33613
United States, Maryland
Institute for Asthma and Allergy, PC
Chevy Chase, Maryland, United States, 20815
United States, Oregon
Allergy & Asthma Research Group
Eugene, Oregon, United States, 97401
Baker Allergy, Asthma and Dermatology 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, P.A.
San Antonio, Texas, United States, 78229
United States, Washington
Marycliff Allergy Specialists
Spokane, Washington, United States, 99204
Germany
Charité Universitätsmedizin Berlin, Dept. of Dermatology and Allergy
Berlin, Germany
Klinikum rechts der Isar, Technical University Munich, ENT Clinic
Munich, Germany
Hungary
Semmelweis University, Allergy and Angioedema Outpatients Clinic, Kútvölgyi Clinical Center
Budapest, Hungary
Sponsors and Collaborators
Shire
Investigators
Study Director: Jennifer Schranz, MD Shire
  More Information

No publications provided

Responsible Party: Shire
ClinicalTrials.gov Identifier: NCT01095510     History of Changes
Other Study ID Numbers: 0624-203, 2011-000369-11
Study First Received: March 24, 2010
Results First Received: June 26, 2014
Last Updated: June 26, 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 August 28, 2014