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Safety and Efficacy Study of CINRYZE for Prevention of Angioedema Attacks in Children Ages 6-11 With Hereditary Angioedema

This study is ongoing, but not recruiting participants.
Sponsor:
Information provided by (Responsible Party):
Shire
ClinicalTrials.gov Identifier:
NCT02052141
First received: January 30, 2014
Last updated: September 19, 2016
Last verified: September 2016
  Purpose

Primary Objective - To assess the relative efficacy of two dose levels of CINRYZE (500 Units and 1000 Units) administered by intravenous (IV) injection every 3 or 4 days to prevent angioedema attacks in children 6 to 11 years of age with hereditary angioedema (HAE).

Secondary Objectives - To assess the safety and tolerability, characterize the pharmacokinetics (PK) and pharmacodynamics (PD), and assess the immunogenicity of two dose levels of CINRYZE administered by IV injection in children 6 to 11 years of age with HAE.


Condition Intervention Phase
Hereditary Angioedema (HAE)
Biological: CINRYZE 500
Biological: CINRYZE 1000
Phase 3

Study Type: Interventional
Study Design: Allocation: Randomized
Endpoint Classification: Safety/Efficacy Study
Intervention Model: Crossover Assignment
Masking: Single Blind (Subject)
Primary Purpose: Prevention
Official Title: A Phase 3, Multicenter, Randomized, Single-Blind, Dose-Ranging, Crossover Study to Evaluate the Safety and Efficacy of Intravenous Administration of CINRYZE® (C1 Esterase Inhibitor [Human]) for the Prevention of Angioedema Attacks in Children 6 to 11 Years of Age With Hereditary Angioedema

Resource links provided by NLM:


Further study details as provided by Shire:

Primary Outcome Measures:
  • Number of angioedema attacks in each treatment period [ Time Frame: 12 weeks ] [ Designated as safety issue: No ]

Secondary Outcome Measures:
  • Number of subjects with adverse events by dose group [ Time Frame: 12 weeks ] [ Designated as safety issue: Yes ]
  • Change from baseline in C1 inhibitor (C1 INH) and C4 concentrations during each treatment period [ Time Frame: Pre-dose to 1 hour post-dose at Visit 1, Week 6, and Week 12 ] [ Designated as safety issue: No ]
  • Number of subjects with C1 INH antibodies [ Time Frame: 28 weeks ] [ Designated as safety issue: Yes ]

Estimated Enrollment: 12
Study Start Date: February 2014
Estimated Study Completion Date: June 2017
Estimated Primary Completion Date: May 2017 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: 500/1000
500 Units of CINRYZE administered by IV injection twice per week for 12 weeks followed by 1000 Units of CINRYZE administered by IV injection twice per week for 12 weeks
Biological: CINRYZE 500
500 Units of CINRYZE administered by IV injection
Biological: CINRYZE 1000
1000 Units of CINRYZE administered by IV injection
Experimental: 1000/500
1000 Units of CINRYZE administered by IV injection twice per week for 12 weeks followed by 500 Units of CINRYZE administered by IV injection twice per week for 12 weeks
Biological: CINRYZE 500
500 Units of CINRYZE administered by IV injection
Biological: CINRYZE 1000
1000 Units of CINRYZE administered by IV injection

  Eligibility

Ages Eligible for Study:   6 Years to 11 Years   (Child)
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  • Diagnosis of Type I or Type II HAE.
  • History of angioedema attacks.

Exclusion Criteria:

  • History of bleeding or clotting abnormality.
  • Diagnosis of acquired angioedema or known to have C1 INH antibodies.
  • History of allergic reaction to C1 esterase inhibitor or other blood products.
  • Receipt of any experimental agents other than those required for prevention or treatment of angioedema attacks within 30 days prior to screening.
  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: NCT02052141

Locations
United States, Colorado
Asthma and Allergy Associates, P.C
Colorado Springs, Colorado, United States, 80907
United States, Nevada
Nevada Access to Research and Education Society
Las Vegas, Nevada, United States, 89106
United States, Oregon
Oregon Allergy Associates
Eugene, Oregon, United States, 97401
Germany
Klinikum der J.W. Goethe Universitat
Frankfurt, Germany, 60590
HZRM Hamophilie Zentrum Rhein Main GmbH
Mörfelden-Walldorf, Germany, 64546
Israel
Tel Aviv Sourasky Medical Center
Tel Aviv, Israel, 64239
Mexico
Instituto Nacional de Pediatria
Mexico City, Mexico, 04530
Romania
Clinical County Hospital Mures
Targu-Mures, Romania, 540072
United Kingdom
Royal Manchester Children's Hospital
Manchester, United Kingdom, M13 9WL
Sponsors and Collaborators
Shire
Investigators
Study Director: Jennifer Schranz, MD Shire
  More Information

Responsible Party: Shire
ClinicalTrials.gov Identifier: NCT02052141     History of Changes
Other Study ID Numbers: 0624-301  2013-002453-29  SHP616-301 
Study First Received: January 30, 2014
Last Updated: September 19, 2016
Health Authority: Romania: National Agency for Medicines and Medical Devices
Italy: The Italian Medicines Agency
United States: Food and Drug Administration
United Kingdom: Medicines and Healthcare Products Regulatory Agency
Germany: Paul-Ehrlich-Institut
Mexico: Federal Commission for Protection Against Health Risks

Keywords provided by Shire:
Pediatric
C1 inhibitor
HAE
Cinryze
C1 INH
Prevention

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 C1 Inactivator Proteins
Complement Inactivating Agents
Immunosuppressive Agents
Immunologic Factors
Physiological Effects of Drugs

ClinicalTrials.gov processed this record on September 23, 2016