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

Shire26 enrolled

Overview

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

Subjects were given diary cards and instructed to document all HAE attacks on a daily basis. Subjects evaluated their symptoms over the previous 24 hours, noting the severity and duration of swelling at each of 5 locations (abdominal, genitourinary, facial, respiratory \[including laryngeal\], and/or extremity). The study design also allowed for administration of open-label C1INH-nf (1,000 U of C1INH-nf administered IV \[repeated after 60 minutes, if necessary\] for treatment of laryngeal angioedema or if deemed necessary by the investigator; 1,000 U of C1INH-nf administered IV \[single dose\] prior to emergency surgical procedures). A total of 26 subjects were enrolled in the study. One subject received open-label C1INH-nf but withdrew prior to randomization. Another subject was randomized but withdrew prior to receiving study drug. Twenty-four (24) subjects were randomized and treated with blinded study drug. In total, 25 subjects received at least 1 dose of study drug and were analyzed for safety; all 25 subjects were exposed to C1INH-nf and 23 subjects were exposed to placebo.

Study Type

INTERVENTIONAL

Allocation

RANDOMIZED

Purpose

PREVENTION

Masking

DOUBLE

Enrollment

Conditions

Hereditary Angioedema

Interventions

C1 esterase inhibitor [human] (C1INH-nf)BIOLOGICAL

Placebo (saline)DRUG

Eligibility

Sex: ALLMin age: 6 Years

Medical Language ↔ Plain English

Inclusion Criteria: * Documented HAE * Normal C1q level * Relatively frequent angioedema attacks (at least 2 per month on average) Exclusion Criteria: * Low C1q level * B-cell malignancy * Presence of anti-C1INH autoantibody * History of allergic reaction to C1INH or other blood products * Narcotic addiction * Current participation in any other investigational drug study or within the past 30 days * Participation in a C1 esterase inhibitor trial, or received blood or a blood product in the past 90 days * Pregnancy or lactation * Any clinically significant medical condition, such as renal failure, that in the opinion of the investigator would interfere with the subject's ability to participate in the study

Locations (16)

Allergy and Immunology Associates

Scottsdale, Arizona, United States

University of California, San Diego

San Diego, California, United States

Outcomes

Primary Outcomes

Number of Hereditary Angioedema (HAE) Attacks During Each Prophylactic Therapy Period

An HAE attack was defined as the subject-reported indication of swelling at any location following a report of no swelling on the previous day. Analyses include observed attack counts and normalized attack counts (i.e., the number of attacks observed during each therapy period, normalized for the number of days the subject participated in that period).

Time frame: 12 weeks

Secondary Outcomes

Number of Subject Withdrawals During Each Prophylactic Therapy Period

At the end of each therapy period, each subject was assigned a yes/no drop-out status. A drop-out was defined as a subject who did not have a Week 12 visit record.

Time frame: 12 weeks

Average Severity of HAE Attacks During Each Prophylactic Therapy Period

All attacks in each therapy period were assigned a value of 1 (mild), 2 (moderate), or 3 (severe). Attack severity was considered the highest value assigned by the subject to any swelling location during the attack. Average severity was set to 0 if there was no attack in a period.

Time frame: 12 weeks

Average Duration of HAE Attacks During Each Prophylactic Therapy Period

The duration of an attack was measured from the first report of swelling at any one of the five locations (abdominal, genitourinary, facial, respiratory \[including laryngeal\], or extremity) until the first subsequent report of "no swelling" at all five locations.

Time frame: 12 weeks

Number of Open-label C1INH-nf Infusions Required During Each Prophylactic Therapy Period

The study design allowed for subjects to be treated with open-label C1INH-nf for laryngeal angioedema, if deemed necessary by the investigator, or prior to emergency surgical procedures.

Data from ClinicalTrials.gov

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