Open-Label C1 Esterase Inhibitor (C1INH-nf) for the Prevention of Acute Hereditary Angioedema (HAE) Attacks (CHANGE 3)

This study has been completed.
Sponsor:
Information provided by:
Shire
ClinicalTrials.gov Identifier:
NCT00462709
First received: April 17, 2007
Last updated: March 19, 2014
Last verified: March 2014
  Purpose

The study objective was to evaluate the safety and efficacy of prophylactic use of C1INH-nf for the prevention of acute HAE attacks.


Condition Intervention Phase
Hereditary Angioedema
Biological: C1 esterase inhibitor [human] (C1INH-nf)
Phase 3

Study Type: Interventional
Study Design: Endpoint Classification: Safety/Efficacy Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Prevention
Official Title: LEVP2006-4 CHANGE 3 Trial (C1-Inhibitor in Hereditary Angioedema Nanofiltration Generation Evaluating Efficacy): Open-Label Use of C1INH-nf (Human) for the Prophylactic Treatment to Prevent HAE Attacks and as Treatment in Acute HAE Attacks

Resource links provided by NLM:


Further study details as provided by Shire:

Primary Outcome Measures:
  • Frequency of All HAE Attacks [ Time Frame: Duration of the study ] [ Designated as safety issue: No ]
    A hereditary angioedema (HAE) attack was defined as a discrete episode during which the subject progressed from no angioedema to symptoms of angioedema.


Other Outcome Measures:
  • Antigenic C1 Inhibitor (C1INH) Serum Levels [ Time Frame: Pre-infusion to 1 hour post-infusion ] [ Designated as safety issue: No ]
    Change from pre-infusion to 1 hour post-infusion in antigenic C1INH serum levels.

  • Functional C1INH Serum Levels [ Time Frame: Pre-infusion to 1 hour post-infusion ] [ Designated as safety issue: No ]

    Change from pre-infusion to 1 hour post-infusion in functional C1INH serum levels.

    Functional C1INH serum levels are expressed as a percent of total detectable C1INH (i.e., functional C1INH/total detectable C1INH).


  • Complement C4 Serum Levels [ Time Frame: Pre-infusion to 1 hour post-infusion ] [ Designated as safety issue: No ]
    Change from pre-infusion to 1 hour post-infusion in complement C4 serum levels.


Enrollment: 146
Study Start Date: June 2006
Study Completion Date: March 2009
Primary Completion Date: March 2009 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: Open-label C1INH-nf
1,000 Units (U) of C1INH-nf administered intravenously (IV) every 3 to 7 days.
Biological: C1 esterase inhibitor [human] (C1INH-nf)

  Eligibility

Ages Eligible for Study:   1 Year and older
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

- History of at least 1 HAE attack per month or any history of laryngeal edema

In addition, this study was open to all subjects who:

  • Completed participation in LEVP2005-1/B (NCT01005888) any time after the final prophylactic therapy in Part B
  • Were enrolled but not randomized in LEVP2005-1/A (NCT00289211) after Part A was closed
  • Were enrolled and randomized in LEVP2005-1/A after LEVP2005-1/B was closed to enrollment, any time after the 3-day telephone follow-up
  • Were excluded from LEVP2005-1 for any of the following reasons:

    • Pregnancy or lactation
    • Age less than 6 years
    • Narcotic addiction
    • Presence of anti-C1 inhibitor (C1INH) autoantibodies
  • Were not enrolled in LEVP2005-1 after enrollment in LEVP2005-1 was closed, under the following circumstances:

    • Had a diagnosis of HAE: evidence of a low C4 level plus either a low C1INH antigenic level or a low C1INH functional level, or
    • Had a known HAE-causing C1INH mutation, or
    • Had a diagnosis of HAE based on a strong family history of HAE as determined by the principal investigator

Exclusion Criteria:

  • History of allergic reaction to C1INH or other blood products
  • Participated in any other investigational drug study within the past 30 days other than those sponsored by Lev Pharmaceuticals
  • Received blood or a blood product in the past 60 days other than C1INH-nf
  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: NCT00462709

  Show 48 Study Locations
Sponsors and Collaborators
Shire
Investigators
Principal Investigator: Bruce Zuraw, MD University of California, San Diego
  More Information

No publications provided by Shire

Additional publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):
Responsible Party: Chief Scientific Officer, ViroPharma
ClinicalTrials.gov Identifier: NCT00462709     History of Changes
Other Study ID Numbers: LEVP2006-4
Study First Received: April 17, 2007
Results First Received: March 31, 2010
Last Updated: March 19, 2014
Health Authority: United States: Food and Drug Administration

Keywords provided by Shire:
Hereditary angioedema
C1 esterase inhibitor (human)

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 Inactivator Proteins
Complement C1 Inhibitor Protein
Complement C1
Complement C1s
Complement Inactivating Agents
Immunosuppressive Agents
Immunologic Factors
Physiological Effects of Drugs
Pharmacologic Actions

ClinicalTrials.gov processed this record on July 20, 2014