12-Week Safety and Efficacy Study of BCX4161 as an Oral Prophylaxis Against HAE Attacks

Hereditary Angioedema Clinical Trial

— OPuS-2  

Official title:

OPuS-2: A Multicenter, Randomized, Double-blind, Placebo-controlled, Parallel Group Study to Evaluate the Efficacy and Safety of Two Dose Levels of BCX4161 for 12 Weeks as an Oral Prophylaxis Treatment for Attacks of Hereditary Angioedema

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT02303626
• Other study ID #: BCX4161-301
• Status: Completed
• Phase: Phase 2/Phase 3
• First received: November 24, 2014
• Last updated: February 15, 2016
• Start date: November 2014
• Est. completion date: January 2016

Study information

• Verified date: February 2016
• Source: BioCryst Pharmaceuticals
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial Summary

This study will evaluate the safety and efficacy of an oral treatment, BCX4161, in preventing acute attacks in subjects with hereditary angioedema (HAE). Eligible subjects will be randomized to receive one of two doses of BCX4161 or placebo for 12 weeks. The study will compare the number of acute attacks in each treatment group, as well as a number of other clinical outcomes, and the safety and tolerability of each dose of BCX4161 compared to placebo.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 110
• Est. completion date: January 2016
• Est. primary completion date: January 2016
• Accepts healthy volunteers: No
• Gender: All
• Age group: 18 Years and older

Eligibility

Key Inclusion Criteria:

1. A clinical diagnosis of HAE type I or II

2. Documented HAE attacks within a defined calendar period; in the absence of documented HAE attacks, subjects will be required to enter a run-in period to document attacks

3. Access to acute attack medications

4. Sexually active women of child-bearing potential and sexually active men must utilize highly effective contraception

Key Exclusion Criteria:

1. Women who are pregnant or breast-feeding

2. Any clinical condition or medical history that would interfere with the subject's safety or ability to participate in the study

3. Use of C1INH or tranexamic acid for prophylaxis of HAE attacks

4. Current participation in any other investigational drug study or within the last 30 days

5. History of or current alcohol or drug abuse

6. Infection with hepatitis B, hepatitis C or HIV

Related Conditions & MeSH terms

• Angioedema
• HAE
• Hereditary Angioedema

Intervention

Drug:
• BCX4161

• Placebo

Locations

Country	Name	City	State
n/a

Sponsors (1)

Lead Sponsor	Collaborator
BioCryst Pharmaceuticals

Countries where clinical trial is conducted

United States,  Belgium,  Canada,  France,  Germany,  Hungary,  Italy,  United Kingdom, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	The mean acute angioedema attack rate		12 weeks
Secondary	Number of attack-free days		12 weeks
Secondary	Number of subjects who are attack-free		12 weeks
Secondary	Disease activity, as measured by the 84-day Angioedema Activity Score		12 weeks
Secondary	Quality of Life, as measured by the Angioedema Quality of Life Questionnaire		12 weeks
Secondary	Incidence and severity of adverse events and laboratory abnormalities		12 weeks
Secondary	Quality of Life, as measured by the EuroQoL five-dimensional, 5-level Questionnaire		12 weeks