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

Shire

Status and phase: Completed; Phase 3

Conditions: Hereditary Angioedema (HAE)

Treatments: Biological: CINRYZE 1000; Biological: CINRYZE 500

Study type: Interventional

Funder types: Industry

Identifiers

NCT02052141

SHP616-301 (Other Identifier)

2013-002453-29 (EudraCT Number)

0624-301

Details and patient eligibility

About

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.

Enrollment: 12 patients

Sex: All

Ages: 6 to 11 years old

Volunteers: No Healthy Volunteers

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.

Trial design

Primary purpose: Prevention

Allocation: Randomized

Interventional model: Crossover Assignment

Masking: Single Blind

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12 participants in 2 patient groups

500/1000

Experimental group

Description:

500 Units of CINRYZE administered by IV injection twice per week for 12 weeks followed by 1000 Units of CINRYZE administered by IV injection twice per week for 12 weeks

Treatment:

Biological: CINRYZE 500

Biological: CINRYZE 1000

1000/500

Experimental group

Description:

1000 Units of CINRYZE administered by IV injection twice per week for 12 weeks followed by 500 Units of CINRYZE administered by IV injection twice per week for 12 weeks

Treatment:

Biological: CINRYZE 500

Biological: CINRYZE 1000