A Study to Evaluate the Clinical Efficacy and Safety of Subcutaneously Administered C1-esterase Inhibitor in the Prevention of Hereditary Angioedema



Status:Completed
Conditions:Skin and Soft Tissue Infections
Therapuetic Areas:Dermatology / Plastic Surgery
Healthy:No
Age Range:12 - Any
Updated:7/29/2017
Start Date:January 2014
End Date:October 2015

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A Double-blind, Randomized, Placebo-controlled, Cross-over Study to Evaluate the Clinical Efficacy and Safety of Subcutaneous Administration of Human Plasma-derived C1-esterase Inhibitor in the Prophylactic Treatment of Hereditary Angioedema

The aim of this study is to assess the efficacy of C1-esterase inhibitor in preventing
hereditary angioedema attacks when it is administered under the skin of subjects with
hereditary angioedema. The safety of C1-esterase inhibitor will also be assessed. Each
subject will enter a run-in period of up to 8-weeks. Subjects who complete the run-in period
and who are eligible will then enter the treatment phase which comprises two sequential
treatment periods. In the treatment phase, subjects will be randomized to one of four arms
consisting of treatment with low- or higher-volume C1-esterase inhibitor in one treatment
period and treatment with low- or higher-volume placebo in the other treatment period. The
study will measure the number of hereditary angioedema attacks that subjects experience while
receiving each treatment.


Inclusion Criteria:

Run-In Period Inclusion Criteria:

- Males or females aged 12 years or older.

- A clinical diagnosis of hereditary angioedema type I or II.

- Hereditary angioedema 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 Screening: use of a stable regimen within 3 months of Screening, with no
plans to change.

Eligibility Criteria for Entering Treatment Period 1:

- Laboratory confirmation of type I or type II hereditary angioedema, including
C1-esterase inhibitor functional activity less than 50% AND C4 antigen level below the
laboratory reference range.

- No clinically significant abnormalities as assessed using laboratory parameters.

- During participation in the run-in period, subjects must have experienced hereditary
angioedema attacks that required acute treatment, required medical attention, or
caused significant functional impairment.

Exclusion Criteria:

Run-In Period Exclusion Criteria:

- History of clinical significant arterial or venous thrombosis, or current history of a
clinically significant prothrombotic risk.

- Incurable malignancies at screening.

- Any clinical condition that will interfere with the evaluation of C1-esterase
inhibitor therapy.

- Clinically significant history of poor response to C1-esterase therapy for the
management of hereditary angioedema.

- Receiving therapy prohibited by the protocol, including medications for hereditary
angioedema prophylaxis.

- Female subjects who started taking or changed dose of any hormonal contraceptive
regimen or hormone replacement therapy (i.e., estrogen/progesterone-containing
products) within 3 months prior to the screening visit.
We found this trial at
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Campbelltown, New South Wales 2560
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Colorado Springs, Colorado 80910
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