Overview

This trial is active, not recruiting.

Condition hereditary angioedema types i and ii
Treatment c1-esterase inhibitor
Phase phase 3
Sponsor CSL Behring
Start date December 2014
End date September 2017
Trial size 110 participants
Trial identifier NCT02316353, 2014-001054-42, CSL830_3002

Summary

The aim of this study is to assess the long-term safety of C1-esterase inhibitor (C1-INH) in preventing hereditary angioedema (HAE) attacks when it is administered under the skin of subjects with HAE. The safety of participating subjects will be assessed for up to 54 weeks. The long-term efficacy of C1-INH will also be assessed. Each eligible subject will enter the treatment phase, wherein subjects will be randomized to treatment with either low- or medium-volume C1-INH. Subjects who have an insufficient treatment response during the study will be given an opportunity to undergo a dose increase. The study aims to enroll eligible subjects who completed study CSL830_3001 (NCT01912456). Subjects who did not participate in study CSL830_3001 may also participate, if eligible and if space permits. Subjects from the United States (US) who complete Treatment Period 2 will be allowed to participate in an Extension Period. During the Extension Period participating US subjects will continue to receive treatment with open-label CSL830 for up to an additional 88 weeks.

United States No locations recruiting
Other Countries No locations recruiting

Study Design

Allocation randomized
Endpoint classification safety/efficacy study
Intervention model parallel assignment
Masking open label
Primary purpose prevention
Arm
(Experimental)
A low-volume dose of C1-INH will be administered subcutaneously twice a week for up to 52 weeks (up to 146 weeks extension period).
c1-esterase inhibitor
(Experimental)
A medium-volume dose of C1-INH will be administered subcutaneously twice a week for up to 52 weeks (up to 146 weeks extension period).
c1-esterase inhibitor

Primary Outcomes

Measure
The person-time incidence rates of specified safety events
time frame: During the treatment and extension phases, up to 146 weeks.

Secondary Outcomes

Measure
Percentage of subjects with SAEs or other specified safety events.
time frame: During the treatment and extension phases, up to 146 weeks.
Percentage of C1-INH injections resulting in solicited AEs (injection site reactions).
time frame: During the treatment and extension phases, up to 146 weeks.
Percentage of subjects with at least 1 solicited AE (injection site reaction).
time frame: During the treatment and extension phases, up to 146 weeks.
Percentage of subjects who become seropositive for human immunodeficiency virus, hepatitis B virus, or hepatitis C virus.
time frame: From baseline through the treatment and extension phases, up to 146 weeks.
Percentage of subjects who experience < 1 HAE attack per 4-week period.
time frame: During the treatment and extension phases, up to 146 weeks.
Percentage of subjects with a ≥ 50% reduction in the time-normalized number of HAE attacks.
time frame: From baseline through the treatment and extension phases, up to 146 weeks.

Eligibility Criteria

Male or female participants at least 6 years old.

Inclusion Criteria: - Males or females aged 6 years or older. - A confirmed diagnosis of HAE type I or II. - HAE attacks over a consecutive 2-month period that required acute treatment, medical attention, or caused significant functional impairment. - For subjects who have used oral therapy for prophylaxis against HAE attacks within 3 months of first study visit: use of a stable regimen within 3 months of the first study visit. Exclusion Criteria: - Incurable malignancies. - Any clinical condition that will interfere with the evaluation of C1-INH therapy. - Clinically significant history of poor response to C1-esterase therapy for the management of HAE. - Suspected or confirmed diagnosis of acquired HAE or HAE with normal C1-INH. - Inability to have HAE managed pharmacologically with on-demand treatment.

Additional Information

Official title An Open-label, Randomized Study to Evaluate the Long-term Clinical Safety and Efficacy of Subcutaneous Administration of Human Plasma-derived C1-esterase Inhibitor in the Prophylactic Treatment of Hereditary Angioedema
Trial information was received from ClinicalTrials.gov and was last updated in November 2016.
Information provided to ClinicalTrials.gov by CSL Behring.