Safety and Efficacy Study of CINRYZE for Prevention of Angioedema Attacks in Children Ages 6-11 With Hereditary Angioedema

Phase 3CompletedNCT02052141

Shire12 enrolled

Overview

Primary Objective - To assess the relative efficacy of two dose levels of CINRYZE (500 Units and 1000 Units) administered by intravenous (IV) injection every 3 or 4 days to prevent angioedema attacks in children 6 to 11 years of age with hereditary angioedema (HAE). Secondary Objectives - To assess the safety and tolerability, characterize the pharmacokinetics (PK) and pharmacodynamics (PD), and assess the immunogenicity of two dose levels of CINRYZE administered by IV injection in children 6 to 11 years of age with HAE.

Study Type

INTERVENTIONAL

Allocation

RANDOMIZED

Purpose

PREVENTION

Masking

SINGLE

Enrollment

Conditions

Hereditary Angioedema (HAE)

Interventions

CINRYZE 500BIOLOGICAL

500 Units of CINRYZE administered by IV injection

CINRYZE 1000BIOLOGICAL

1000 Units of CINRYZE administered by IV injection

Eligibility

Sex: ALLMin age: 6 YearsMax age: 11 Years

Medical Language ↔ Plain English

Inclusion Criteria: * Diagnosis of Type I or Type II HAE. * History of angioedema attacks. Exclusion Criteria: * History of bleeding or clotting abnormality. * Diagnosis of acquired angioedema or known to have C1 INH antibodies. * History of allergic reaction to C1 esterase inhibitor or other blood products. * Receipt of any experimental agents other than those required for prevention or treatment of angioedema attacks within 30 days prior to screening.

Locations (8)

Asthma and Allergy Associates, P.C

Colorado Springs, Colorado, United States

Nevada Access to Research and Education Society

Las Vegas, Nevada, United States

Oregon Allergy Associates

Eugene, Oregon, United States

Klinikum der J.W. Goethe Universitat

Frankfurt, Germany

Outcomes

Primary Outcomes

Normalized Number of Angioedema Attacks Per Month in a Treatment Period

Angioedema attack was defined as the participant-reported indication of symptoms or signs such as swelling or pain at any location following a report of no swelling or pain on the previous day. Manifestations of an attack that progress from one site to another, prior to complete resolution, was considered a single attack. Attacks that began to regress and then worsened before complete resolution was also considered one attack. Attacks that began then appeared to resolve and then reappeared without a symptom-free calendar day reported after the appearance of resolution were considered 1 attack. Any events of swelling due to trauma or symmetrical nonpainful swelling of the lower extremities were not considered an angioedema attack. The number of attacks was normalized for the number of days participants participated in a given period and expressed as the monthly frequency.

Time frame: From start of treatment up to 12 weeks during each treatment period

Secondary Outcomes

Cumulative Attack-severity Score of Angioedema Attacks Normalized Per Month in a Treatment Period

Severity of the angioedema attack sign/symptom was characterized as None: no symptom; Mild: noticeable symptom but easily tolerated by the participant and did not interfere with routine activities; Moderate: symptom interfered with the participant's ability to attend school or participate in family life and social/recreational activities; Severe: symptom significantly limited the participant's ability to attend school or participate in family life and social/recreational activities. Symptom severity score was assigned as Mild = 1, Moderate = 2 and Severe = 3. Cumulative attack severity score was the sum of the maximum symptom severity scores recorded for each angioedema attack in a treatment period. Cumulative attack-severity score normalized per month \[(raw score/number of days of participation in that treatment period)\*30.4\] was reported here. Cumulative attack-severity score normalized per month ranged from 0 to 10.4 and higher scores represent worse symptoms.

Time frame: From start of treatment up to 12 weeks during each treatment period

Cumulative Daily-severity Score of Angioedema Attacks Normalized Per Month in a Treatment Period

Data from ClinicalTrials.gov

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Normalized Number of Angioedema Attacks Per Month Requiring Acute Treatment in a Treatment Period

Angioedema attack was defined as the participant-reported indication of symptoms or signs such as swelling or pain at any location following a report of no swelling or pain on the previous day. Manifestations of an attack that progress from one site to another, prior to complete resolution, was considered a single attack. Attacks that began to regress and then worsened before complete resolution was also considered one attack. Attacks that began then appeared to resolve and then reappeared without a symptom-free calendar day reported after the appearance of resolution were considered 1 attack. Any events of swelling due to trauma or symmetrical nonpainful swelling of the lower extremities were not considered an angioedema attack. The number of attacks requiring acute treatment was normalized for the number of days participants participated in a given period and expressed as the monthly frequency.

Time frame: From start of treatment up to 12 weeks during each intervention period