A Long Term Safety Study of BCX7353 in Hereditary Angioedema (APeX-S)

May 23, 2023 updated by: BioCryst Pharmaceuticals

An Open-label Study to Evaluate the Long-term Safety of Daily Oral BCX7353 in Subjects With Type I and II Hereditary Angioedema

This is an open-label study to evaluate the long term safety and effectiveness of oral treatment with BCX7353 in preventing acute angioedema attacks in patients with Type I and Type II Hereditary Angioedema (HAE).

Study Overview

Status

Status

Indicates the current recruitment status or the expanded access status.
Completed

Conditions

Conditions

The disease, disorder, syndrome, illness, or injury that is being studied. On ClinicalTrials.gov, conditions may also include other health-related issues, such as lifespan, quality of life, and health risks.

Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.

Study Type

Study Type

Describes the nature of a clinical study. Study types include interventional studies (also called clinical trials), observational studies (including patient registries), and expanded access.
Interventional

Enrollment (Actual)

Enrollment

The number of participants in a clinical study. The "anticipated" enrollment is the target number of participants that the researchers need for the study.
387

Phase

Phase

The stage of a clinical trial studying a drug or biological product, based on definitions developed by the U.S. Food and Drug Administration (FDA). The phase is based on the study's objective, the number of participants, and other characteristics. There are five phases: Early Phase 1 (formerly listed as Phase 0), Phase 1, Phase 2, Phase 3, and Phase 4. Not Applicable is used to describe trials without FDA-defined phases, including trials of devices or behavioral interventions.
  • Phase 2
  • Phase 3

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • Australia
      • Adelaide, Australia
        • Study Center
      • Campbelltown, Australia
        • Study Center
      • Camperdown, Australia
        • Study Center
      • Melbourne, Australia
        • Study Center
      • Murdoch, Australia
        • Study Center
      • Nedlands, Australia
        • Study Center
  • Austria
      • Graz, Austria
        • Study Center
      • Vienna, Austria
        • Study Center
  • Denmark
      • Odense, Denmark
        • Study Center
  • France
      • Grenoble, France
        • Study Center
      • Lille, France
        • Study Center
      • Paris, France
        • Study Center
  • Germany
      • Berlin, Germany
        • Study Center
      • Frankfurt, Germany
        • Study Center
      • Ulm, Germany
        • Study Center
  • Hong Kong
      • Central, Hong Kong
        • Study Center
  • Hungary
      • Budapest, Hungary
        • Study Center
  • Israel
      • Ashkelon, Israel
        • Study Center
      • Haifa, Israel
        • Study Center
      • Tel Aviv, Israel
        • Study Center
      • Tel HaShomer, Israel
        • Study Center
  • Italy
      • Milan, Italy
        • Study Center
      • Padova, Italy
        • Study Center
      • Salerno, Italy
        • Study Center
  • Korea, Republic of
      • Daegu, Korea, Republic of
        • Study Center
      • Donggu, Korea, Republic of
        • Study Center
      • Gyeonggi-do, Korea, Republic of
        • Study Center
      • Seoul, Korea, Republic of
        • Study Center
  • New Zealand
      • Auckland, New Zealand
        • Study Center
      • Wellington, New Zealand
        • Study Center
  • North Macedonia
      • Skopje, North Macedonia
        • Study Center
  • Poland
      • Kraków, Poland
        • Study Center
  • Serbia
      • Belgrade, Serbia
        • Study Center
      • Niš, Serbia
        • Study Center
  • Slovakia
      • Martin, Slovakia
        • Study Center
  • South Africa
      • Cape Town, South Africa
        • Study Center
  • Spain
      • Barcelona, Spain
        • Study Center
      • Madrid, Spain
        • Study Center
  • Switzerland
      • Zürich, Switzerland
        • Study Center
  • United Kingdom
      • Birmingham, United Kingdom
        • Study Center
      • Bristol, United Kingdom
        • Study Center
      • Cambridge, United Kingdom
        • Study Center
      • London, United Kingdom
        • Study Center
      • Plymouth, United Kingdom
        • Study Center
      • Southampton, United Kingdom
        • Study Center
  • United States
    • Alabama
      • Birmingham, Alabama, United States, 35209
        • Study Center
    • Arizona
      • Scottsdale, Arizona, United States, 85251
        • Study Center
    • Arkansas
      • Bentonville, Arkansas, United States, 72712
        • Study Center
      • Little Rock, Arkansas, United States, 72205
        • Study Center
    • California
      • San Diego, California, United States, 92123
        • Study Center
      • Walnut Creek, California, United States, 94598
        • Study Center
    • Colorado
      • Centennial, Colorado, United States, 80112
        • Study Center
      • Colorado Springs, Colorado, United States, 80907
        • Study Center
      • Wheat Ridge, Colorado, United States, 80033
        • Study Center
    • Connecticut
      • Waterbury, Connecticut, United States, 06708
        • Study Center
    • Florida
      • Tampa, Florida, United States, 33613
        • Study Center
    • Georgia
      • Marietta, Georgia, United States, 30060
        • Study Center
    • Illinois
      • Normal, Illinois, United States, 61761
        • Study Center
    • Indiana
      • Evansville, Indiana, United States, 47713
        • Study Center
      • Indianapolis, Indiana, United States, 46202
        • Study Center
    • Kansas
      • Overland Park, Kansas, United States, 66210
        • Study Center
    • Kentucky
      • Louisville, Kentucky, United States, 40215
        • Study Center
    • Maryland
      • Chevy Chase, Maryland, United States, 20815
        • Study Center
    • Massachusetts
      • Boston, Massachusetts, United States, 02114
        • Study Center
    • Michigan
      • Ann Arbor, Michigan, United States, 48106
        • Study Center
      • Grand Rapids, Michigan, United States, 49506
        • Study Center
    • Minnesota
      • Rochester, Minnesota, United States, 55905
        • Study Center
    • Mississippi
      • Madison, Mississippi, United States, 39110
        • Study Center
    • Missouri
      • Saint Louis, Missouri, United States, 63141
        • Study Center
    • Nebraska
      • Lincoln, Nebraska, United States, 68505
        • Study Center
    • North Carolina
      • Charlotte, North Carolina, United States, 28277
        • Study Center
      • Durham, North Carolina, United States, 27705
        • Study Center
    • Ohio
      • Cincinnati, Ohio, United States, 45231
        • Study Center
      • Columbus, Ohio, United States, 43235
        • Study Center
    • Oregon
      • Clackamas, Oregon, United States, 97015
        • Study Center
      • Happy Valley, Oregon, United States, 97086
        • Study Center
    • Pennsylvania
      • Hershey, Pennsylvania, United States, 17033
        • Study Center
    • Rhode Island
      • East Providence, Rhode Island, United States, 02914
        • Study Center
    • South Carolina
      • Greer, South Carolina, United States, 29651
        • Study Center
    • Texas
      • Austin, Texas, United States, 78731
        • Study Center
      • Dallas, Texas, United States, 75231
        • Study Center
      • Irving, Texas, United States, 75063
        • Study Center
      • San Antonio, Texas, United States, 78229
        • Study Center
    • Utah
      • Murray, Utah, United States, 84107
        • Study Center
    • Washington
      • Seattle, Washington, United States, 98115
        • Study Center
      • Spokane, Washington, United States, 99202
        • Study Center
    • Wisconsin
      • Milwaukee, Wisconsin, United States, 53226
        • Study Center

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Eligibility Criteria

The key requirements that people who want to participate in a clinical study must meet or the characteristics they must have. Eligibility criteria consist of both inclusion criteria (which are required for a person to participate in the study) and exclusion criteria (which prevent a person from participating). Types of eligibility criteria include whether a study accepts healthy volunteers, has age or age group requirements, or is limited by sex.

Ages Eligible for Study

12 years and older (Child, Adult, Older Adult)

Accepts Healthy Volunteers

No

Description

Key Inclusion Criteria:

  • Subjects with HAE Type I or II who either have participated in a previous BCX7353 study or, in selected countries, in the opinion of the Investigator are expected to derive benefit from an oral treatment for the prevention of angioedema attacks.
  • Access to appropriate medication for treatment of acute attacks
  • Acceptable effective contraception
  • Written informed consent

Key Exclusion Criteria:

  • Pregnancy or breast-feeding
  • Any clinically significant medical condition or medical history that, in the opinion of the Investigator or Sponsor, would interfere with the subject's safety or ability to participate in the study
  • Any laboratory parameter abnormality that, in the opinion of the Investigator, is clinically significant and relevant for this study
  • Discontinuation of study drug due to a hypersensitivity reaction BCX7353 in a prior study
  • Severe hypersensitivity to multiple medicinal products or severe hypersensitivity/ anaphylaxis with unclear etiology
  • Unacceptable noncompliance in a previous BCX7353 study (if applicable) as assessed by the Sponsor or Investigator
  • Investigational drug exposure, other than BCX7353, within 30 days prior to the screening visit (or baseline if no screening visit)

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Prevention
  • Allocation: N/A
  • Interventional Model: Single Group Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / Arm

Participant Group / Arm

A group or subgroup of participants in a clinical trial that receives a specific intervention/treatment, or no intervention, according to the trial's protocol.
Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.
Experimental: BCX7353 150 mg once daily
Drug: BCX7353
BCX7353 mg oral capsules administered once daily

What is the study measuring?

Primary Outcome Measures

Primary Outcome Measures

In a clinical study's protocol, the planned outcome measure that is the most important for evaluating the effect of an intervention/treatment. Most clinical studies have one primary outcome measure, but some have more than one.
Outcome Measure
Measure Description
Time Frame
Safety & Tolerability
Time Frame: Up to 96 weeks (US) / 216 weeks (Rest of World (ROW)).
The number and percentage of subjects with treatment-emergent adverse events.
Up to 96 weeks (US) / 216 weeks (Rest of World (ROW)).

Secondary Outcome Measures

Secondary Outcome Measures

In a clinical study's protocol, a planned outcome measure that is not as important as the primary outcome measure for evaluating the effect of an intervention but is still of interest. Most clinical studies have more than one secondary outcome measure.
Outcome Measure
Measure Description
Time Frame
Incidence of Acute Attacks of Angioedema in Subjects During Treatment
Time Frame: Up to 96 weeks (US) / 216 weeks (ROW)
Number of 'adjusted' attacks were assessed. Adjusted attacks included at least 1 symptom of swelling, had a response of 'no' to the diary question, 'In retrospect, could there be an alternative explanation for your symptoms other than an HAE attack (i.e., allergic reaction, viral cold etc.)?', and were considered unique (attack began > 24 hours from the end of the prior attack). Any attack that began within 24 hours from the end of a prior attack was combined with the prior attack.
Up to 96 weeks (US) / 216 weeks (ROW)
The Durability of Response to Treatment
Time Frame: Up to 96 weeks (US) / 216 weeks (ROW)
To evaluate if the rate of attacks remains consistent (durable) over time, the monthly attack rate was assessed at 0 to 24 weeks, 24 to 48 weeks, 48 to 96 weeks and 96 weeks until the end of the study. Monthly attack rate was defined as the total number of adjusted HAE attacks experienced during the treatment period adjusted for the length of a month (defined as 28 days) and the number of days the subject was on treatment during that month.
Up to 96 weeks (US) / 216 weeks (ROW)
Patient Reported Quality of Life (QoL) During Treatment
Time Frame: Up to 96 weeks (US) / 216 weeks (ROW)
Quality of Life (QoL) specific to hereditary angioedema (HAE) was assessed at baseline and at each study visit until the end of the study. The questionnaire (i.e. AE-QoL) consisted of 17 questions spanning 4 domains (functioning, fatigue/mood, fear/shame, and nutrition). Each AE-QoL question had 5 answer options (scored 1-5), with lower and higher scores indicting less and more adverse impact, respectively. Per-subject scores for each domain were computed using the appropriate scoring algorithm applied to the question response scores for each domain. Per-subject total scores (including all 4 domains) were similarly computed using the question response scores for all 17 questions. The outputs from the scoring algorithm were normalized on a scale ranging from 0 (less adverse impact) to 100 (most adverse impact). The Mean change from baseline (CFB) in AE-QoL total score over time is presented below.
Up to 96 weeks (US) / 216 weeks (ROW)
Patient's Satisfaction With Medication During Long Term Administration of Berotralstat
Time Frame: Up to 96 weeks (US) / 216 weeks (ROW)
The Treatment Satisfaction Questionnaire for Medication (TSQM) was completed by subjects at baseline and at each study visit until the end of the study. TSQM scores consisted of 14 items of which 13 items were made up of 3 specific scales (Effectiveness, Side Effects, and Convenience) and 1 global satisfaction scale (Global Satisfaction). At baseline, TSQM questionnaires were completed based on subject's satisfaction with usual medications. At all other time points for collection of TSQM, subjects were asked about their level of satisfaction or dissatisfaction with the study drug. Scale scores were calculated for each scale and were transformed into scores ranging from 0 to 100, with higher scores indicating higher satisfaction. TSQM score and corresponding change from baseline values were calculated at each visit. Note: Subjects in Hong Kong did not complete the TSQM.
Up to 96 weeks (US) / 216 weeks (ROW)

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Sponsor

The organization or person who initiates the study and who has authority and control over the study.
BioCryst Pharmaceuticals

Investigators

Investigators

A researcher involved in a clinical study. Related terms include site principal investigator, site sub-investigator, study chair, study director, and study principal investigator.
  • Principal Investigator: Henriette Farkas, MD, Semmelweis University, Budapest, Hungary

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

General Publications

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

Study Start

The actual date on which the first participant was enrolled in a clinical study. The "anticipated" study start date is the date that the researchers think will be the study start date.
February 16, 2018

Primary Completion (Actual)

Primary Completion

The date on which the last participant in a clinical study was examined or received an intervention to collect final data for the primary outcome measure. Whether the clinical study ended according to the protocol or was terminated does not affect this date. For clinical studies with more than one primary outcome measure with different completion dates, this term refers to the date on which data collection is completed for all the primary outcome measures. The "anticipated" primary completion date is the date that the researchers think will be the primary completion date for the study.
April 27, 2022

Study Completion (Actual)

Study Completion

The date on which the last participant in a clinical study was examined or received an intervention/treatment to collect final data for the primary outcome measures, secondary outcome measures, and adverse events (that is, the last participant's last visit). The "anticipated" study completion date is the date that the researchers think will be the study completion date.
April 27, 2022

Study Registration Dates

First Submitted

First Submitted

The date on which the study sponsor or investigator first submitted a study record to ClinicalTrials.gov. There is typically a delay of a few days between the first submitted date and the record's availability on ClinicalTrials.gov (the first posted date).
February 27, 2018

First Submitted That Met QC Criteria

First Submitted That Met QC Criteria

The date on which the study sponsor or investigator first submits a study record that is consistent with National Library of Medicine (NLM) quality control (QC) review criteria. The sponsor or investigator may need to revise and submit a study record one or more times before NLM's QC review criteria are met. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
March 14, 2018

First Posted (Actual)

First Posted

The date on which the study record was first available on ClinicalTrials.gov after National Library of Medicine (NLM) quality control (QC) review has concluded. There is typically a delay of a few days between the date the study sponsor or investigator submitted the study record and the first posted date.
March 21, 2018

Study Record Updates

Last Update Posted (Estimated)

Last Update Posted

The most recent date on which changes to a study record were made available on ClinicalTrials.gov. There may be a delay between when the changes were submitted to ClinicalTrials.gov by the study's sponsor or investigator (the last update submitted date) and the last update posted date.
June 19, 2023

Last Update Submitted That Met QC Criteria

Last Update Submitted That Met QC Criteria

The most recent date on which the study sponsor or investigator submitted changes to a study record that are consistent with National Library of Medicine (NLM) quality control (QC) review criteria. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
May 23, 2023

Last Verified

Last Verified

The most recent date on which the study sponsor or investigator confirmed the information about a clinical study on ClinicalTrials.gov as accurate and current. If a study with a recruitment status of recruiting; not yet recruiting; or active, not recruiting has not been confirmed within the past 2 years, the study's recruitment status is shown as unknown.
May 1, 2023

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

Other Study ID Numbers

Identifiers or ID numbers other than the NCT number that are assigned to a clinical study by the study's sponsor, funders, or others. These numbers may include unique identifiers from other trial registries and National Institutes of Health grant numbers.
  • BCX7353-204

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

NO

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

Yes

Studies a U.S. FDA-regulated device product

No

product manufactured in and exported from the U.S.

Yes

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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Conditions

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