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

NCT02670720 | Terminated Phase 3 DMC

• 1 other identifier: BCX4161-303
• Study Type: interventional
• Target: 6
• Locations: 2 countries
• Sites: 3

Brief 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.

Conditions

Hereditary Angioedema; HAE

Keywords

BCX4161; avoralstat; HAE; hereditary angioedema; prophylaxis

Outcome Measures

Primary Outcomes (1)

• 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

Secondary Outcomes (5)

• Angioedema attack rate (subject-reported HAE attacks normalized for time on study)

  Up to 72 weeks

• Durability in response (assessed as subject-reported HAE attacks)

  change over time through 72 weeks

• Proportion of subjects who discontinue avoralstat with a reason of lack of efficacy

  Up to 72 weeks

• Quality of life as determined by the EQ-5D-5L

  Up to 72 weeks

• Quality of life as determined by the Angioedema Quality of Life Questionnaire

  Up to 72 weeks

Study Arms (1)

avoralstat

EXPERIMENTAL

Five avoralstat capsules (100 mg) to be taken three times daily by mouth

Drug: avoralstat

Interventions

avoralstat DRUG

Also known as: BCX4161

avoralstat

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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.

You may not qualify if:

• 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.

Sponsors & Collaborators

• BioCryst Pharmaceuticals: lead

Study Sites (3)

Unknown Facility

Leuven, Belgium

Location

Unknown Facility

Grenoble, France

Location

Unknown Facility

Lille, France

Location

MeSH Terms

Conditions

Angioedemas, Hereditary

Interventions

avoralstat

Condition Hierarchy (Ancestors)

Angioedema; Vascular Diseases; Cardiovascular Diseases; Hereditary Complement Deficiency Diseases; Primary Immunodeficiency Diseases; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Urticaria; Skin Diseases, Vascular; Skin Diseases; Skin and Connective Tissue Diseases; Hypersensitivity, Immediate; Hypersensitivity; Immune System Diseases; Immunologic Deficiency Syndromes

Study Officials

• Henriette Farkas, MD, PhD, DSc

  Semmelweis University

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: NA
• Masking: NONE
• Purpose: PREVENTION
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: January 20, 2016
• First Posted: February 2, 2016
• Study Start: December 1, 2015
• Primary Completion: February 1, 2016
• Study Completion: February 1, 2016
• Last Updated: February 17, 2016

Record last verified: 2016-02

Locations

BE (1); FR (2)