Open-Label C1 Esterase Inhibitor (C1INH-nf) for the Treatment of Acute Hereditary Angioedema (HAE) Attacks (CHANGE 2)

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

NCT00438815

LEVP2006-1

Details and patient eligibility

About

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

Full description

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.

Enrollment: 113 patients

Sex: All

Ages: 1+ year old

Volunteers: No Healthy Volunteers

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

Trial design

Primary purpose: Treatment

Allocation: N/A

Interventional model: Single Group Assignment

Masking: None (Open label)

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113 participants in 1 patient group

Open-label C1INH-nf

Experimental group

Description:

1,000 Units (U) of C1INH-nf administered intravenously. If there was no response to treatment 60 minutes after the first dose, a second 1,000 U dose could be administered.

Treatment:

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