Open-label, Long-term Safety Study of Avoralstat in Subje... | NCT02670720

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

NCT02670720

Sponsor

BioCryst Pharmaceuticals

Status

Terminated

Last Update Posted

Feb 17, 2016Estimated

Enrollment

6Actual

Phase

Phase 3

Conditions

Hereditary Angioedema
HAE

Interventions

avoralstat

Countries

Belgium
France

Protocol Section

Identification Module Status Module Sponsor/Collaborators Module Oversight Module Description Module Conditions Module Design Module Arms/Interventions Module Outcomes Module Eligibility Module Contacts/Locations Module References Module IPD Sharing Statement Module

Results Section

Participant Flow Module Baseline Characteristics Module Outcome Measures Module Adverse Events Module More Info Module

Annotation Section

Annotation Module

Document Section

Large Document Module

Derived Section

Miscellaneous Info Module Condition Browse Module Intervention Browse Module

Protocol Section

Identification Module

NCT ID

Results Section

No data available

No data is available for this block.

Annotation Section

No data available

No data is available for this block.

Document Section

No data available

No data is available for this block.

Derived Section

Miscellaneous Info Module

Version Holder

Status Module

Record Verification Date

Feb 2016

Overall Recruitment Status or Expanded Access Status

Terminated

Last Known Status

Not provided

Delayed Posting

Not provided

Why Stopped

preceding efficacy study of avoralstat in its current formulation was not significant and does not support continued development

Expanded Access Info

Start Date

Dec 2015

Primary Completion Date

Feb 2016Actual

Completion Date

Feb 2016Actual

First Submitted Date

Jan 20, 2016

First Submission Date that Met QC Criteria

Jan 28, 2016

First Posted Date

Feb 2, 2016Estimated

Results Waived

Not provided

Results First Submitted Date

Not provided

Results First Submitted that Met QC Criteria

Not provided

Results First Posted Date

Not provided

Certification/Extension (aka Delayed Results) First Submitted Date

Not provided

Certification/Extension First Submitted that Passed QC Review

Not provided

Certification/Extension First Posted Date

Not provided

Last Update Submitted Date

Feb 15, 2016

Last Update Posted Date

Feb 17, 2016Estimated

Oversight Module

Has Data Monitoring Committee (DMC)

Yes

Is FDA Regulated Drug

Not provided

Is FDA Regulated Device

Not provided

Is Unapproved Device

Not provided

Pediatric Postmarket Surveillance of a Device Product

Not provided

Product Exported from US

Not provided

FDAAA801 Violation

Not provided

Description Module

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.

Detailed Description

Not provided

Design Module

Study Type

Interventional

Number of References to an Expanded Access Study

Not provided

Expanded Access Types

Not provided

Patient Registry

Not provided

Target Follow-Up Duration

Not provided

Phases

Phase 3

Interventional Study Design

Allocation

Biospecimen

No data available

No data is available for this block.

Enrollment

6Actual

Arms/Interventions Module

Arm Groups

Label	Type	Description	Intervention Names
avoralstat	Experimental	Five avoralstat capsules (100 mg) to be taken three times daily by mouth	Drug: avoralstat

Interventions

Name	Type	Description	Arm Group Labels	Other Names
avoralstat	Drug		avoralstat	BCX4161

Outcomes Module

Primary Outcomes

Measure	Description	Time Frame
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

Measure	Description	Time Frame
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

Other Outcomes

Not provided

Eligibility Module

Eligibility 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.

Accepts Healthy Volunteers

Sex

All

Sex/Gender Based

Not provided

Sex/Gender Description

Not provided

Minimum Age

18 Years

Maximum Age

Not provided

Standard Ages

AdultOlder Adult

Study Population

Not provided

Sampling Method

Not provided

Contacts/Locations Module

Central Contacts

Not provided

Overall Officials

Name	Affiliation	Role
Henriette Farkas, MD, PhD, DSc	Semmelweis University	Principal Investigator

Locations

Facility	Status	City	State	ZIP	Country	Contacts
		Leuven			Belgium

Condition Browse Module

MeSH Terms

ID	Term
D054179	Angioedemas, Hereditary

Ancestor Terms

ID	Term
D000799	Angioedema
D014652	Vascular Diseases
D002318	Cardiovascular Diseases
D000081208	Hereditary Complement Deficiency Diseases

Browse Leaves

Not provided

Browse Branches

Not provided

Intervention Browse Module

MeSH Terms

ID	Term
C000722673	avoralstat

Ancestor Terms

Not provided

Browse Leaves

Not provided

Browse Branches

Not provided