Overview

This trial is active, not recruiting.

Condition hereditary angioedema (hae)
Treatments cinryze 500, cinryze 1000
Phase phase 3
Sponsor Shire
Start date February 2014
End date May 2017
Trial size 12 participants
Trial identifier NCT02052141, 0624-301, 2013-002453-29, SHP616-301

Summary

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.

United States No locations recruiting
Other Countries No locations recruiting

Study Design

Allocation randomized
Endpoint classification safety/efficacy study
Intervention model crossover assignment
Masking single blind (subject)
Primary purpose prevention
Arm
(Experimental)
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
cinryze 500
500 Units of CINRYZE administered by IV injection
cinryze 1000
1000 Units of CINRYZE administered by IV injection
(Experimental)
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
cinryze 500
500 Units of CINRYZE administered by IV injection
cinryze 1000
1000 Units of CINRYZE administered by IV injection

Primary Outcomes

Measure
Number of angioedema attacks in each treatment period
time frame: 12 weeks

Secondary Outcomes

Measure
Number of subjects with adverse events by dose group
time frame: 12 weeks
Change from baseline in C1 inhibitor (C1 INH) and C4 concentrations during each treatment period
time frame: Pre-dose to 1 hour post-dose at Visit 1, Week 6, and Week 12
Number of subjects with C1 INH antibodies
time frame: 28 weeks

Eligibility Criteria

Male or female participants from 6 years up to 11 years old.

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.

Additional Information

Official title A Phase 3, Multicenter, Randomized, Single-Blind, Dose-Ranging, Crossover Study to Evaluate the Safety and Efficacy of Intravenous Administration of CINRYZE® (C1 Esterase Inhibitor [Human]) for the Prevention of Angioedema Attacks in Children 6 to 11 Years of Age With Hereditary Angioedema
Trial information was received from ClinicalTrials.gov and was last updated in October 2016.
Information provided to ClinicalTrials.gov by Shire.