Skip to main content
OpenTrials
Completed

NCT Number: NCT00438815

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

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

Completed

Looking for future studies?

Notify Me

Key information

Conditions

Hereditary Angioedema Angioedema Angioedemas, Hereditary Cardiovascular Diseases Congenital, Hereditary, and Neonatal Diseases and Abnormalities Genetic Diseases, Inborn Hereditary Complement Deficiency Diseases Hypersensitivity Hypersensitivity, Immediate Immune System Diseases Immunologic Deficiency Syndromes Primary Immunodeficiency Diseases Skin Diseases Skin Diseases, Vascular Skin and Connective Tissue Diseases Urticaria Vascular Diseases

Age range

1 year and older

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 3

Primary location

Allergy and Immunology Associates, Scottsdale, Arizona, United States

Loading trial locations.

About this study

A total of 113 subjects were enrolled in the study. One-hundred-one (101) subjects received C1INH-nf for the treatment of 1 or more HAE attacks and were analyzed for efficacy. The study design also allowed for short-term prophylaxis with C1INH-nf prior to emergency or non-cosmetic surgical or dental procedures, and an additional 12 subjects received C1INH-nf only for this purpose. All 113 subjects were exposed to C1INH-nf and analyzed for safety.

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

Inclusion criteria

This study was open to all subjects who:

  • Completed participation in LEVP2005-1/A (NCT00289211) and were not participating in LEVP2005-1/B (NCT01005888), any time after the 3-day telephone follow-up
  • Completed participation in LEVP2005-1/B any time after the final prophylactic therapy in Part B
  • Were enrolled but not randomized in LEVP2005-1/A after Part A was closed
  • Were excluded from LEVP2005-1 for any of the following reasons:
  • Pregnancy or lactation
  • Age less than 6 years
  • Narcotic addiction
  • Presence of anti-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
  • Received blood or a blood product in the past 60 days other than C1INH-nf

Treatment and study plan

C1 esterase inhibitor [human] (C1INH-nf)

Biological

Primary outcomes

  1. Number of Hereditary Angioedema (HAE) Attacks Treated With C1INH-nf

    Time frame: Duration of the study (2.5 years)

  2. Percent of HAE Attacks With Substantial Relief of the Defining Symptom

    Time frame: Within 4 hours after initial treatment

    Subjects were to assess their symptoms every 15 minutes up to 4 hours after the initial dose or until substantial relief of the defining symptom was achieved. The conservative analysis defined substantial relief as 3 consecutive assessments of improvement of the defining symptom; any attack that did not have 3 consecutive documented reports of improvement was considered a treatment failure. In the less conservative analysis, attacks also were considered to have responded if clinical improvement of the defining symptom occurred but data were incomplete due to cessation of symptom assessments.

Secondary outcomes

  1. Time to Beginning of Substantial Relief of the Defining Symptom

    Time frame: Within 4 hours after initial treatment

    Subjects were to assess their symptoms every 15 minutes up to 4 hours after the initial dose or until substantial relief of the defining symptom was achieved. Substantial relief was defined as 3 consecutive assessments of improvement of the defining symptom. Beginning of substantial relief was considered the first of the 3 consecutive assessments.

  2. Time to Beginning of Substantial Relief of the Defining Symptom for Subjects Who Received Multiple Treatments

    Time frame: Within 4 hours after initial treatment

    For attack number 1, the number of censored observations precluded estimation of the 95% confidence interval (CI) upper bound for median time to event (subjects who did not experience beginning of substantial relief of the defining symptom within 4 hours after initial treatment were included in the analysis as censored observations). Entry of 4.0 hours indicates that data were not estimable (NE); as non-numeric data are not supported by the 95% CI field, entry of the actual result (ie, NE or >4.0) was not possible.

  3. Antigenic C1 Inhibitor (C1INH) Serum Levels

    Time frame: Pre-infusion to 1 hour post-infusion

    Change in antigenic C1INH serum levels from pre-infusion to 1 hour after the initial dose of study drug.

  4. Functional C1INH Serum Levels

    Time frame: Pre-infusion to 1 hour post-infusion

    Percent change in functional C1INH serum levels from pre-infusion to 1 hour after the initial dose of study drug. Functional C1INH serum levels are expressed as a percent of total detectable C1INH (ie, functional C1INH/total detectable C1INH).

  5. Complement C4 Serum Levels

    Time frame: Pre-infusion to 1 hour post-infusion

    Change in complement C4 serum levels from pre-infusion to 1 hour after the initial dose of study drug.

Sponsors and collaborators

Lead sponsor

Shire

Industry

Registry information

Official study title

LEVP2006-1 CHANGE 2 Trial (C1-Inhibitor in Hereditary Angioedema Nanofiltration Generation Evaluating Efficacy): Open-Label Safety/Efficacy Repeat Exposure Study of C1INH-nf (Human) in the Treatment of Acute HAE Attacks

Acronym: CHANGE 2

Important dates

Study start
2006
Primary completion
2009
Study completion
2009
First posted
Feb 22, 2007
Registry last updated
Jun 8, 2021

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

Related clinical trials

Published trials that share one or more normalized conditions with this study.