Hereditary AngioedemaPhase 2OPuS-1NCT01984788
Safety and Efficacy of Oral BCX4161 as a Prophylactic Treatment for HAE
A Phase 2 study testing BCX4161 in people with Hereditary Angioedema, run by BioCryst Pharmaceuticals at 6 sites in 2 countries.
Trial summary
The purpose of this study is to determine whether BCX4161 given as a daily oral prophylactic treatment is safe and effective in reducing the number of acute attacks in patients with hereditary angioedema. Read more about this study
- Who is it for?
- Ages 18 to 65 years
- Conditions the study is for
- Hereditary Angioedema
- What is being tested?
- BCX4161
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 24 participants
- Where
- 6 sitesin 2 countries
- Study dates
- Nov 2013to May 2014
- Phase
- Phase 2
- 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 to 65 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Written informed consent
- Clinical and laboratory diagnosis of Hereditary Angioedema Type I or II
- An average angioedema attack frequency of 1/week
- Acceptable birth control measures
Who cannot join
- Concurrent use of defined treatments for prophylaxis
- Pregnancy or breast-feeding
- Clinically significant medical condition, laboratory abnormality or medical history
Trial locations and contacts
6 sites in 2 countries.
6 sites in 2 countries
Germany5 sites
Dr Marcus Maurer
Berlin
Dr Emel Aygoren-Pursun
Frankfurt
Dr Petra Staubach
Mainz
Dr Inmaculada Martinez-Saguer
Mörfelden-Walldorf
Dr Murat Bas
München
About this study
Official title: A Phase 2a Double-Blind Placebo-Controlled 2-Period Crossover Study to Evaluate the Safety and Efficacy of BCX4161 as a Prophylactic Treatment to Reduce the Frequency of Attacks in Subjects With Hereditary Angioedema
The purpose of this study is to determine whether BCX4161 given as a daily oral prophylactic treatment is safe and effective in reducing the number of acute attacks in patients with hereditary angioedema.
- Conditions
- Hereditary Angioedema
- Being tested
- BCX4161, Placebo
- Keywords
- Oral 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.
BCX4161Drug
PlaceboDrug
| Study group | Role | What they receive |
|---|---|---|
| BCX4161 | Active comparator | 400 mg TID for 28 days Receives: Drug: BCX4161 |
| Placebo | Placebo comparator | TID for 28 days Receives: Drug: Placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
Number of acute angioedema attacks
Measured: Over 28 days of treatment
Secondary outcomes (4)
Incidence and severity of adverse events and laboratory abnormalities
Measured: Over 28 days of treatment
Number of attack free days
Measured: Over 28 days of treatment
Angioedema Quality of Life scores
Measured: Over 28 days of treatment
Angioedema activity scores
Measured: Over 28 days of treatment
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted15 Nov 2013 (estimated)
- Study startNov 2013
- Primary completionMay 2014
- Study completionMay 2014
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)Papers the sponsor has linked to this study.
- 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 2
- Primary purpose
- Prevention
- Allocation
- Randomised
- Intervention model
- Crossover
- Masking
- Double blind
- Who is masked
- Participant, Investigator
- Enrolment
- 24 participants (actual)
Sponsor and investigators
- Lead sponsor
- BioCryst Pharmaceuticals
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Marcus Maurer, MD, Principal investigator, Charite University, Berlin, Germany
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01984788
- Sponsor's protocol ID
- BCX4161-203
- Acronym
- OPuS-1
- Status last verified
- Aug 2014
- First submitted
- 5 Nov 2013
- Last updated
- 18 Aug 2014
- Results posted
- No
- Data monitoring committee
- Yes
Publications
Beard N, Frese M, Smertina E, Mere P, Katelaris C, Mills K. Interventions for the long-term prevention of hereditary angioedema attacks. Cochrane Database Syst Rev. 2022 Nov 3;11(11):CD013403. doi: 10.1002/14651858.CD013403.pub2.
PubMed 36326435
Study at a glance
From the registry record, last updated by the sponsor on 18 Aug 2014. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase 2a Double-Blind Placebo-Controlled 2-Period Crossover Study to Evaluate the Safety and Efficacy of BCX4161 as a Prophylactic Treatment to Reduce the Frequency of Attacks in Subjects With Hereditary Angioedema
- ClinicalTrials.gov ID
- NCT01984788
- Acronym / sponsor ID
- OPuS-1 / BCX4161-203
- Conditions
- Hereditary Angioedema
- Keywords
- Oral Prophylaxis
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 24 participants (actual)
- Lead sponsor
- BioCryst PharmaceuticalsIndustry (pharma, biotech, devices)
- Study start
- Nov 2013
- Primary completion
- May 2014 (actual)
- Study completion
- May 2014 (actual)
- First posted
- 15 Nov 2013 (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.
The purpose of this study is to determine whether BCX4161 given as a daily oral prophylactic treatment is safe and effective in reducing the number of acute attacks in patients with hereditary angioedema.
Study design
- Primary purpose
- Prevention
- Allocation
- Randomised
- Intervention model
- Crossover
- Masking
- Double blind (participant, investigator)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| BCX4161 | Active comparator | Drug: BCX4161400 mg TID for 28 days |
| Placebo | Placebo comparator | Drug: PlaceboTID for 28 days |
Who may qualify
- Ages
- 18 to 65 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (4 listed)
- Written informed consent
- Clinical and laboratory diagnosis of Hereditary Angioedema Type I or II
- An average angioedema attack frequency of 1/week
- Acceptable birth control measures
Key exclusion criteria (3 listed)
- Concurrent use of defined treatments for prophylaxis
- Pregnancy or breast-feeding
- Clinically significant medical condition, laboratory abnormality or medical history
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 |
|---|---|---|
| BCX4161 | Drug | BCX4161 |
| Placebo | Drug | Placebo |
Primary outcome
Number of acute angioedema attacks
Time frame: Over 28 days of treatment
4 secondary outcomes
Incidence and severity of adverse events and laboratory abnormalities
Time frame: Over 28 days of treatment
Number of attack free days
Time frame: Over 28 days of treatment
Angioedema Quality of Life scores
Time frame: Over 28 days of treatment
Angioedema activity scores
Time frame: Over 28 days of treatment
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 18 Aug 2014.
- Derived from this study: PubMed 36326435Beard N, Frese M, Smertina E, Mere P, Katelaris C, Mills K. Interventions for the long-term prevention of hereditary angioedema attacks. Cochrane Database Syst Rev. 2022 Nov 3;11(11):CD013403. doi: 10.1002/14651858.CD013403.pub2.
Related trials for Hereditary Angioedema
RecruitingPhase 3
Donidalorsen Treatment in Children With Hereditary Angioedema
Hereditary Angioedema (HAE)
RecruitingObservational study
A Study of Takhzyro in Teenagers and Adults With Hereditary Angioedema (HAE) in South Korea
Hereditary Angioedema (HAE)
Active, not recruitingPhase 3
HAELO: A Phase 3 Study to Evaluate NTLA-2002 in Participants With Hereditary Angioedema (HAE)
Hereditary Angioedema
RecruitingPhase 3
Study of IV Human Plasma-derived C1 Esterase Inhibitor Concentrate in Patients With Congenital C1-INH Deficiency for Treatment and Pre-procedure Preventing of Acute Hereditary Angioedema Attacks
Acute Hereditary Angio Edema
Active, not recruitingPhase 1/2
NTLA-2002 in Adults With Hereditary Angioedema (HAE)
Hereditary Angioedema
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.
Marcus Maurer, MD principal investigator
Charite University, Berlin, Germany
6 sites in 2 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing BCX4161 in people with Hereditary Angioedema, run by BioCryst Pharmaceuticals at 6 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 4 inclusion and 3 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: finished, as of the sponsor's last update on 18 Aug 2014. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 6 sites in Germany, United Kingdom. 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. Some participants in this study may receive a placebo. 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 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
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 is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
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?
Number of acute angioedema attacks (time frame: Over 28 days of treatment). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT01984788, which also lists 1 linked publication.
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 18 Aug 2014.