Hereditary AngioedemaNCT04428632
Oral Berotralstat Expanded Access Program
A study testing Berotralstat in people with Hereditary Angioedema and HAE, run by BioCryst Pharmaceuticals.
Trial summary
This expanded access program will provide access to berotralstat for eligible participants with hereditary angioedema in the U.S. Read more about this study
- Who is it for?
- Ages 12 years and older
- Conditions the study is for
- Hereditary Angioedema, HAE, Prophylaxis
- What is being tested?
- Berotralstat
- Type of treatment
- Pill or oral medicine
- How many people
- Not given
- Where
- No sites listed yet
- Study dates
- Start not givento not given
- Phase
- Expanded access
- 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 12 years and older
- All sexes
- Child, Adult, Older adult
Who may be able to join
- Patients must meet all of the following inclusion criteria to be eligible for participation in this program:
- At least 12 years of age
- Able to provide written, informed consent or assent
- Patients with a clinical diagnosis of HAE Type I or II who, in the opinion of their treating physician, are expected to benefit from an oral treatment for the prevention of angioedema attacks, and who are not eligible or able to access a berotralstat clinical trial
- Females must use acceptable effective contraception
Who cannot join
- Patients must meet none of the below exclusion criteria to be eligible for participation in this program:
- Pregnancy or breast-feeding
- Any clinically significant medical condition or medical history that, in the opinion of the treating physician, would interfere with the patient's safety
- Current infection with hepatitis B virus (HBV), hepatitis C virus (HCV), or human immunodeficiency virus (HIV)
About this study
Official title: Expanded Access Program With Oral Berotralstat for the Prevention of Attacks in Patients With Hereditary Angioedema
This expanded access program will provide access to berotralstat for eligible participants with hereditary angioedema in the U.S.
Read the detailed description
Physicians may request access to berotralstat for eligible participants with unmet medical need
Physicians can send email inquiries to access.us@inceptua.com or call 1-888-225-8677
Patients who may be interested should contact their physician about participation
Berotralstat (BCX7353) will be available through this expanded access program until it is commercially available
- Conditions
- Hereditary Angioedema, HAE, Prophylaxis
- Being tested
- Berotralstat
- Keywords
- Berotralstat, BCX7353, Hereditary Angioedema, HAE
- Medical subject headings
- Angioedemas, Hereditary
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
BerotralstatDrug
Also called BCX7353
One 150mg capsule administered orally once daily
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted11 Jun 2020
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
- Expanded access
Sponsor and investigators
- Lead sponsor
- BioCryst Pharmaceuticals
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04428632
- Sponsor's protocol ID
- BCX7353-308
- Status last verified
- Dec 2020
- First submitted
- 10 Jun 2020
- Last updated
- 11 Dec 2020
- Results posted
- No
Study at a glance
From the registry record, last updated by the sponsor on 11 Dec 2020. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Expanded Access Program With Oral Berotralstat for the Prevention of Attacks in Patients With Hereditary Angioedema
- ClinicalTrials.gov ID
- NCT04428632
- Acronym / sponsor ID
- BCX7353-308
- Conditions
- Hereditary Angioedema, HAE, Prophylaxis
- Keywords
- Berotralstat, BCX7353, Hereditary Angioedema, HAE
- Study type
- Expanded access
- Recruitment status
- Approved For Marketing
- Lead sponsor
- BioCryst PharmaceuticalsIndustry (pharma, biotech, devices)
- First posted
- 11 Jun 2020 (actual)
- 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 expanded access program will provide access to berotralstat for eligible participants with hereditary angioedema in the U.S.
Detailed description
Physicians may request access to berotralstat for eligible participants with unmet medical need
Physicians can send email inquiries to access.us@inceptua.com or call 1-888-225-8677
Patients who may be interested should contact their physician about participation
Berotralstat (BCX7353) will be available through this expanded access program until it is commercially available
Study design
Who may qualify
- Ages
- 12 years and older
- Sex
- All
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (5 listed)
- Patients must meet all of the following inclusion criteria to be eligible for participation in this program:
- At least 12 years of age
- Able to provide written, informed consent or assent
- Patients with a clinical diagnosis of HAE Type I or II who, in the opinion of their treating physician, are expected to benefit from an oral treatment for the prevention of angioedema attacks, and who are not eligible or able to access a berotralstat clinical trial
- Females must use acceptable effective contraception
Key exclusion criteria (4 listed)
- Patients must meet none of the below exclusion criteria to be eligible for participation in this program:
- Pregnancy or breast-feeding
- Any clinically significant medical condition or medical history that, in the opinion of the treating physician, would interfere with the patient's safety
- Current infection with hepatitis B virus (HBV), hepatitis C virus (HCV), or human immunodeficiency virus (HIV)
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 |
|---|---|---|
| BerotralstatAlso: BCX7353One 150mg capsule administered orally once daily | Drug | – |
Primary outcome
Not reported in the source record.
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 11 Dec 2020.
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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.
The record lists no contact. Use the official record to reach the sponsor.
Questions about this trial
What is this clinical trial studying?
A study testing Berotralstat in people with Hereditary Angioedema and HAE, run by BioCryst Pharmaceuticals.
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 4 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: status not confirmed, as of the sponsor's last update on 11 Dec 2020. Status can change between updates, so confirm with the study contact before making plans.
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.
Are results available for this study?
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.
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 11 Dec 2020.