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

Shire

Status and phase: Completed; Phase 3

Conditions: Hereditary Angioedema

Treatments: Biological: C1 esterase inhibitor [human] (C1INH-nf)

Study type: Interventional

Funder types: Industry

Identifiers

NCT00462709

LEVP2006-4

Details and patient eligibility

About

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

Enrollment: 146 patients

Sex: All

Ages: 1+ year old

Volunteers: No Healthy Volunteers

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

Trial design

Primary purpose: Prevention

Allocation: N/A

Interventional model: Single Group Assignment

Masking: None (Open label)

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146 participants in 1 patient group

Open-label C1INH-nf

Experimental group

Description:

1,000 Units (U) of C1INH-nf administered intravenously (IV) every 3 to 7 days.

Treatment:

Biological: C1 esterase inhibitor [human] (C1INH-nf)