Study to Evaluate the Clinical Efficacy and Safety of Subcutaneously Administered C1 Esterase Inhibitor for the Prevention of Angioedema Attacks in Adolescents and Adults With Hereditary Angioedema

Shire

Status and phase: Completed; Phase 3

Conditions: Hereditary Angioedema (HAE)

Treatments: Drug: C1 esterase inhibitor [human] liquid; Drug: Placebo

Study type: Interventional

Funder types: Industry

Identifiers

NCT02584959

SHP616-300

Details and patient eligibility

About

The purpose of this study is to assess the efficacy and safety of subcutaneous administration of a liquid formulation of C1 esterase inhibitor for the prevention of angioedema attacks in adolescent and adult subjects with hereditary angioedema.

Enrollment: 75 patients

Sex: All

Ages: 12+ years old

Volunteers: No Healthy Volunteers

Inclusion and exclusion criteria

The maximum duration of participation is approximately 9 months. Patients will complete a screening period of up to 21 days. Following screening, eligible patients will be randomly assigned to 1 of 3 treatment sequences. Each patient will undergo 2 14-week treatment periods for a total of 28 weeks (Treatment Period 1 and Treatment Period 2). After completing the 2 treatment periods, patients will enter a 1-month follow-up period.

Trial design

Primary purpose: Prevention

Allocation: Randomized

Interventional model: Crossover Assignment

Masking: Double Blind

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75 participants in 3 patient groups

Experimental/Placebo

Experimental group

Description:

Subjects will be randomized to receive C1 Esterase Inhibitor in the 1st Treatment period and then switch to Placebo in the 2nd treatment period.

Treatment:

Drug: Placebo

Drug: C1 esterase inhibitor [human] liquid

Placebo/Experimental

Experimental group

Description:

Subjects will be randomized to receive a placebo treatment in the 1st Treatment period and then switch to receive C1 Esterase Inhibitor in the 2nd treatment period.

Treatment:

Drug: Placebo

Drug: C1 esterase inhibitor [human] liquid

Experimental/ Experimental

Experimental group

Description:

Subjects will be randomized and receive C1 Esterase Inhibitor in both 1st as well as the 2nd treatment period

Treatment:

Drug: C1 esterase inhibitor [human] liquid