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Open-label, Long-term Safety Study of Avoralstat in Subjects With Hereditary Angioedema (OPuS-4)

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ClinicalTrials.gov Identifier: NCT02670720
Recruitment Status : Terminated (preceding efficacy study of avoralstat in its current formulation was not significant and does not support continued development)
First Posted : February 2, 2016
Last Update Posted : February 17, 2016
Sponsor:
Information provided by (Responsible Party):
BioCryst Pharmaceuticals

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.

Condition or disease Intervention/treatment Phase
Hereditary Angioedema HAE Drug: avoralstat Phase 3

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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 6 participants
Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Prevention
Official Title: OPuS-4: An Open-label Study to Evaluate the Long-term Safety of Avoralstat in Subjects With Hereditary Angioedema
Study Start Date : December 2015
Actual Primary Completion Date : February 2016
Actual Study Completion Date : February 2016

Resource links provided by the National Library of Medicine


Arm Intervention/treatment
Experimental: avoralstat
Five avoralstat capsules (100 mg) to be taken three times daily by mouth
Drug: avoralstat
Other Name: BCX4161




Primary Outcome Measures :
  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 [ Time Frame: Up to 72 weeks ]

Secondary Outcome Measures :
  1. Angioedema attack rate (subject-reported HAE attacks normalized for time on study) [ Time Frame: Up to 72 weeks ]
  2. Durability in response (assessed as subject-reported HAE attacks) [ Time Frame: change over time through 72 weeks ]
  3. Proportion of subjects who discontinue avoralstat with a reason of lack of efficacy [ Time Frame: Up to 72 weeks ]
  4. Quality of life as determined by the EQ-5D-5L [ Time Frame: Up to 72 weeks ]
  5. Quality of life as determined by the Angioedema Quality of Life Questionnaire [ Time Frame: Up to 72 weeks ]


Information from the National Library of Medicine

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Ages Eligible for Study:   18 Years and older   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

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.

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02670720


Locations
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Belgium
Leuven, Belgium
France
Grenoble, France
Lille, France
Sponsors and Collaborators
BioCryst Pharmaceuticals
Investigators
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Principal Investigator: Henriette Farkas, MD, PhD, DSc Semmelweis University
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Responsible Party: BioCryst Pharmaceuticals
ClinicalTrials.gov Identifier: NCT02670720    
Other Study ID Numbers: BCX4161-303
First Posted: February 2, 2016    Key Record Dates
Last Update Posted: February 17, 2016
Last Verified: February 2016
Keywords provided by BioCryst Pharmaceuticals:
BCX4161
avoralstat
HAE
hereditary angioedema
prophylaxis
Additional relevant MeSH terms:
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Angioedema
Angioedemas, Hereditary
Vascular Diseases
Cardiovascular Diseases
Urticaria
Skin Diseases, Vascular
Skin Diseases
Hypersensitivity, Immediate
Hypersensitivity
Immune System Diseases
Genetic Diseases, Inborn