Hereditary AngioedemaPhase 3OPuS-4NCT02670720
Open-label, Long-term Safety Study of Avoralstat in Subjects With Hereditary Angioedema
A Phase 3 study testing avoralstat in people with Hereditary Angioedema and HAE, run by BioCryst Pharmaceuticals at 3 sites in 2 countries.
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. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Hereditary Angioedema, HAE
- What is being tested?
- avoralstat
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 6 participants
- Where
- 3 sitesin 2 countries
- Study dates
- Dec 2015to Feb 2016
- Phase
- Phase 3
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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.
Who cannot join
- 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.
Trial locations and contacts
3 sites in 2 countries.
3 sites in 2 countries
About this study
Official title: OPuS-4: An Open-label Study to Evaluate the Long-term Safety of Avoralstat in Subjects With Hereditary Angioedema
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
- Being tested
- avoralstat
- Keywords
- BCX4161, avoralstat, HAE, hereditary angioedema, prophylaxis
- Medical subject headings
- Angioedemas, Hereditary
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
avoralstatDrug
Also called BCX4161
| Study group | Role | What they receive |
|---|---|---|
| avoralstat | Experimental | Five avoralstat capsules (100 mg) to be taken three times daily by mouth Receives: Drug: avoralstat |
What is being measured
The primary outcome is the main question the study is designed to answer.
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
Measured: Up to 72 weeks
Secondary outcomes (5)
Angioedema attack rate (subject-reported HAE attacks normalized for time on study)
Measured: Up to 72 weeks
Durability in response (assessed as subject-reported HAE attacks)
Measured: change over time through 72 weeks
Proportion of subjects who discontinue avoralstat with a reason of lack of efficacy
Measured: Up to 72 weeks
Quality of life as determined by the EQ-5D-5L
Measured: Up to 72 weeks
Quality of life as determined by the Angioedema Quality of Life Questionnaire
Measured: Up to 72 weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted2 Feb 2016 (estimated)
- Study startDec 2015
- Primary completionFeb 2016
- Study completionFeb 2016
Why it stopped: preceding efficacy study of avoralstat in its current formulation was not significant and does not support continued development
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Hereditary AngioedemaStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 3
- Primary purpose
- Prevention
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 6 participants (actual)
Sponsor and investigators
- Lead sponsor
- BioCryst Pharmaceuticals
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Henriette Farkas, MD, PhD, DSc, Principal investigator, Semmelweis University
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02670720
- Sponsor's protocol ID
- BCX4161-303
- Acronym
- OPuS-4
- Status last verified
- Feb 2016
- First submitted
- 20 Jan 2016
- Last updated
- 17 Feb 2016
- Results posted
- No
- Data monitoring committee
- Yes
Study at a glance
From the registry record, last updated by the sponsor on 17 Feb 2016. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- OPuS-4: An Open-label Study to Evaluate the Long-term Safety of Avoralstat in Subjects With Hereditary Angioedema
- ClinicalTrials.gov ID
- NCT02670720
- Acronym / sponsor ID
- OPuS-4 / BCX4161-303
- Conditions
- Hereditary Angioedema, HAE
- Keywords
- BCX4161, avoralstat, HAE, hereditary angioedema, prophylaxis
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- TerminatedReason stopped: preceding efficacy study of avoralstat in its current formulation was not significant and does not support continued development
- Enrolment
- 6 participants (actual)
- Lead sponsor
- BioCryst PharmaceuticalsIndustry (pharma, biotech, devices)
- Study start
- Dec 2015
- Primary completion
- Feb 2016 (actual)
- Study completion
- Feb 2016 (actual)
- First posted
- 2 Feb 2016 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Prevention
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| avoralstat | Experimental | Drug: avoralstatFive avoralstat capsules (100 mg) to be taken three times daily by mouth |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (5 listed)
- 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.
Key exclusion criteria (6 listed)
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| avoralstatAlso: BCX4161 | Drug | avoralstat |
Primary outcome
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
5 secondary outcomes
Angioedema attack rate (subject-reported HAE attacks normalized for time on study)
Time frame: Up to 72 weeks
Durability in response (assessed as subject-reported HAE attacks)
Time frame: change over time through 72 weeks
Proportion of subjects who discontinue avoralstat with a reason of lack of efficacy
Time frame: Up to 72 weeks
Quality of life as determined by the EQ-5D-5L
Time frame: Up to 72 weeks
Quality of life as determined by the Angioedema Quality of Life Questionnaire
Time frame: Up to 72 weeks
Results and safety
The study was stopped early (reason given: preceding efficacy study of avoralstat in its current formulation was not significant and does not support continued development). Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 17 Feb 2016.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Henriette Farkas, MD, PhD, DSc principal investigator
Semmelweis University
3 sites in 2 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing avoralstat in people with Hereditary Angioedema and HAE, run by BioCryst Pharmaceuticals at 3 sites in 2 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 5 inclusion and 6 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: stopped early, as of the sponsor's last update on 17 Feb 2016. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 3 sites in France, Belgium. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study was stopped early (reason given: preceding efficacy study of avoralstat in its current formulation was not significant and does not support continued development). Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
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). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 17 Feb 2016; AzTrials last refreshed its copy on 7 Nov 2026.