Open-label, Long-term Safety Study of Avoralstat in Subjects With Hereditary Angioedema

Hereditary Angioedema Clinical Trial

— OPuS-4  

Official title:

OPuS-4: An Open-label Study to Evaluate the Long-term Safety of Avoralstat in Subjects With Hereditary Angioedema

Clinical Trial Details — Status: Terminated

Administrative data

• NCT number: NCT02670720
• Other study ID #: BCX4161-303
• Status: Terminated
• Phase: Phase 3
• First received: January 20, 2016
• Last updated: February 15, 2016
• Start date: December 2015
• Est. completion date: February 2016

Study information

• Verified date: February 2016
• Source: BioCryst Pharmaceuticals
• Contact: n/a
• Is FDA regulated: No
• Health authority: Germany: Federal Institute for Drugs and Medical DevicesUnited Kingdom: Medicines and Healthcare Products Regulatory AgencyFrance: Agence Nationale de Sécurité du Médicament et des produits de santéItaly: The Italian Medicines AgencyHungary: National Institute of PharmacyBelgium: Federal Agency for Medicines and Health Products, FAMHP
• Study type: Interventional

Clinical Trial Summary

This is an open-label study designed to evaluate the long-term safety of prophylactic avoralstat (500 mg three times daily) when given to approximately 150 patients with hereditary angioedema (HAE) for a duration of up to 72 weeks. The study will also evaluate the long-term efficacy and impact on quality of life of avoralstat prophylactic treatment.

Recruitment information / eligibility

• Status: Terminated
• Enrollment: 6
• Est. completion date: February 2016
• Est. primary completion date: February 2016
• Accepts healthy volunteers: No
• Gender: Both
• Age group: 18 Years and older

Eligibility

Inclusion Criteria:

• Males and non-pregnant, non-lactating females age = 18 years.

• Provide written, informed consent.

• Prior completion of an avoralstat therapeutic study OR a confirmed diagnosis of HAE Type 1 or HAE Type 2.

• Access to appropriate medication for the treatment of acute HAE attacks.

• Adequate contraception.

Exclusion Criteria:

• Females who are pregnant or breast feeding.

• Clinically significant medical condition or medical history.

• Abnormal screening ECG, laboratory or urinalysis finding that is clinically significant.

• Investigational drug exposure within 30 days (except avoralstat).

• History of or current alcohol or drug abuse.

• HIV or active HBV or HCV infection.

Study Design

Endpoint Classification: Safety Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Prevention

Related Conditions & MeSH terms

• Angioedema
• HAE
• Hereditary Angioedema

Intervention

Drug:
• avoralstat

Locations

Country	Name	City	State
n/a

Sponsors (1)

Lead Sponsor	Collaborator
BioCryst Pharmaceuticals

Countries where clinical trial is conducted

Belgium,  France, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Proportion of subjects who discontinue due to a treatment-emergent adverse event, who experience a treatment-emergent serious AE, who experience a Grade 3 or 4 AE and who experience Grade 3 or 4 laboratory abnormalities		Up to 72 weeks	No
Secondary	Angioedema attack rate (subject-reported HAE attacks normalized for time on study)		Up to 72 weeks	No
Secondary	Durability in response (assessed as subject-reported HAE attacks)		change over time through 72 weeks	No
Secondary	Proportion of subjects who discontinue avoralstat with a reason of lack of efficacy		Up to 72 weeks	No
Secondary	Quality of life as determined by the EQ-5D-5L		Up to 72 weeks	No
Secondary	Quality of life as determined by the Angioedema Quality of Life Questionnaire		Up to 72 weeks	No