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Hereditary AngioedemaPhase 2/3APeX-SNCT03472040

A Long Term Safety Study of BCX7353 in Hereditary Angioedema

A Phase 2/3 study testing BCX7353 in people with Hereditary Angioedema and HAE, run by BioCryst Pharmaceuticals at 87 sites in 20 countries.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Official record
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Trial summary

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). 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?
BCX7353
Type of treatment
Pill or oral medicine
Could I receive a placebo?
No, everyone receives an active treatment
How many people
387 participants
Where
87 sitesin 20 countries
Study dates
16 Feb 2018to 27 Apr 2022
Phase
Phase 2/3
Are there results?
Yes, posted in the registrySummary in the doctors' view

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
  • No healthy volunteers
  • Child, Adult, Older adult

Who may be able to join

  • 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

Who cannot join

  • 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)

Trial locations and contacts

87 sites in 20 countries.

87 sites in 20 countries

United States42 sites

  • Study Center

    Ann Arbor, Michigan, 48106

  • Study Center

    Austin, Texas, 78731

  • Study Center

    Bentonville, Arkansas, 72712

  • Study Center

    Birmingham, Alabama, 35209

  • Study Center

    Boston, Massachusetts, 02114

  • Study Center

    Centennial, Colorado, 80112

  • Study Center

    Charlotte, North Carolina, 28277

  • Study Center

    Chevy Chase, Maryland, 20815

  • Study Center

    Cincinnati, Ohio, 45231

  • Study Center

    Clackamas, Oregon, 97015

  • Study Center

    Colorado Springs, Colorado, 80907

  • Study Center

    Columbus, Ohio, 43235

  • Study Center

    Dallas, Texas, 75231

  • Study Center

    Durham, North Carolina, 27705

  • Study Center

    East Providence, Rhode Island, 02914

  • Study Center

    Evansville, Indiana, 47713

  • Study Center

    Grand Rapids, Michigan, 49506

  • Study Center

    Greer, South Carolina, 29651

  • Study Center

    Happy Valley, Oregon, 97086

  • Study Center

    Hershey, Pennsylvania, 17033

  • Study Center

    Indianapolis, Indiana, 46202

  • Study Center

    Irving, Texas, 75063

  • Study Center

    Lincoln, Nebraska, 68505

  • Study Center

    Little Rock, Arkansas, 72205

  • Study Center

    Louisville, Kentucky, 40215

  • Study Center

    Madison, Mississippi, 39110

  • Study Center

    Marietta, Georgia, 30060

  • Study Center

    Milwaukee, Wisconsin, 53226

  • Study Center

    Murray, Utah, 84107

  • Study Center

    Normal, Illinois, 61761

  • Study Center

    Overland Park, Kansas, 66210

  • Study Center

    Rochester, Minnesota, 55905

  • Study Center

    San Antonio, Texas, 78229

  • Study Center

    San Diego, California, 92123

  • Study Center

    Scottsdale, Arizona, 85251

  • Study Center

    Seattle, Washington, 98115

  • Study Center

    Spokane, Washington, 99202

  • Study Center

    St Louis, Missouri, 63141

  • Study Center

    Tampa, Florida, 33613

  • Study Center

    Walnut Creek, California, 94598

  • Study Center

    Waterbury, Connecticut, 06708

  • Study Center

    Wheat Ridge, Colorado, 80033

Other trials in United States

Australia6 sites

  • Study Center

    Adelaide

  • Study Center

    Campbelltown

  • Study Center

    Camperdown

  • Study Center

    Melbourne

  • Study Center

    Murdoch

  • Study Center

    Nedlands

Other trials in Australia

United Kingdom6 sites

  • Study center

    Birmingham

  • Study Center

    Bristol

  • Study Center

    Cambridge

  • Study Center

    London

  • Study Center

    Plymouth

  • Study Center

    Southampton

Other trials in United Kingdom

Israel4 sites

  • Study Center

    Ashkelon

  • Study Center

    Haifa

  • Study Center

    Tel Aviv

  • Study Center

    Tel HaShomer

Other trials in Israel

South Korea4 sites

  • Study Center

    Daegu

  • Study Center

    Donggu

  • Study Center

    Gyeonggi-do

  • Study Center

    Seoul

Other trials in South Korea

France3 sites

  • Study Center

    Grenoble

  • Study Center

    Lille

  • Study Center

    Paris

Other trials in France

Germany3 sites

  • Study Center

    Berlin

  • Study Center

    Frankfurt

  • Study Center

    Ulm

Other trials in Germany

Italy3 sites

  • Study Center

    Milan

  • Study Center

    Padova

  • Study Center

    Salerno

Other trials in Italy

Austria2 sites

  • Study Center

    Graz

  • Study Center

    Vienna

Other trials in Austria

New Zealand2 sites

  • Study Center

    Auckland

  • Study Center

    Wellington

Other trials in New Zealand

Serbia2 sites

  • Study Center

    Belgrade

  • Study Center

    Niš

Other trials in Serbia

Spain2 sites

  • Study Center

    Barcelona

  • Study Center

    Madrid

Other trials in Spain

Denmark1 site

  • Study Center

    Odense

Other trials in Denmark

Hong Kong1 site

  • Study Center

    Central

Other trials in Hong Kong

Hungary1 site

  • Study Center

    Budapest

Other trials in Hungary

North Macedonia1 site

  • Study Center

    Skopje

Other trials in North Macedonia

Poland1 site

  • Study Center

    Krakow

Other trials in Poland

Slovakia1 site

  • Study Center

    Martin

Other trials in Slovakia

South Africa1 site

  • Study Center

    Cape Town

Other trials in South Africa

Switzerland1 site

  • Study Center

    Zurich

Other trials in Switzerland

About this study

Official title: 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).

Being tested
BCX7353
Keywords
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.

  • BCX7353Drug

    BCX7353 mg oral capsules administered once daily

Study groups in NCT03472040
Study groupRoleWhat they receive
BCX7353 150 mg once dailyExperimentalNot described
Receives: Drug: BCX7353

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. Safety & Tolerability

    The number and percentage of subjects with treatment-emergent adverse events.

    Measured: Up to 96 weeks (US) / 216 weeks (Rest of World (ROW)).

Secondary outcomes (4)

  1. Incidence of Acute Attacks of Angioedema in Subjects During Treatment

    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.

    Measured: Up to 96 weeks (US) / 216 weeks (ROW)

  2. The Durability of Response to Treatment

    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.

    Measured: Up to 96 weeks (US) / 216 weeks (ROW)

  3. Patient Reported Quality of Life (QoL) During Treatment

    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.

    Measured: Up to 96 weeks (US) / 216 weeks (ROW)

  4. Patient's Satisfaction With Medication During Long Term Administration of Berotralstat

    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.

    Measured: Up to 96 weeks (US) / 216 weeks (ROW)

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted21 Mar 2018
  2. Study start16 Feb 2018
  3. Primary completion27 Apr 2022
  4. Study completion27 Apr 2022

Resources

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 2/3
Primary purpose
Prevention
Allocation
Not applicable
Intervention model
Single group
Masking
None (open label)
Enrolment
387 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT03472040
Sponsor's protocol ID
BCX7353-204
Acronym
APeX-S
Status last verified
May 2023
First submitted
27 Feb 2018
Last updated
18 Jun 2023
Data monitoring committee
Yes
FDA-regulated drug
Yes
FDA-regulated device
No
Individual data sharing
No

Publications

  • Farkas H, Peter JG, Stobiecki M, Anderson J, Aygoren-Pursun E, Hagin D, Jesenak M, Kessel A, Kiani-Alikhan S, Kinaciyan T, Manning M, Reshef A, Wu A, Iocca HA, Johnston DT, Noble L, Tomita D, Banerji A. Long-term safety and efficacy of once-daily berotralstat in patients with hereditary angioedema: APeX-S final results. Ann Allergy Asthma Immunol. 2025 Sep;135(3):311-319.e6. doi: 10.1016/j.anai.2025.06.004. Epub 2025 Jun 7.

    PubMed 40490218

  • Peter JG, Desai B, Tomita D, Collis P, Stobiecki M. Assessment of HAE prophylaxis transition from androgen therapy to berotralstat: A subset analysis of the APeX-S trial. World Allergy Organ J. 2023 Nov 6;16(11):100841. doi: 10.1016/j.waojou.2023.100841. eCollection 2023 Nov.

    PubMed 38020288

  • Riedl MA, Soteres D, Sublett JW, Desai B, Tomita D, Collis P, Bernstein JA; APeX-S Study Investigators. Hereditary angioedema outcomes in US patients switched from injectable long-term prophylactic medication to oral berotralstat. Ann Allergy Asthma Immunol. 2024 Apr;132(4):505-511.e1. doi: 10.1016/j.anai.2023.11.016. Epub 2023 Nov 24.

    PubMed 38006972

  • Farkas H, Balla Z. A review of berotralstat for the treatment of hereditary angioedema. Expert Rev Clin Immunol. 2023 Feb;19(2):145-153. doi: 10.1080/1744666X.2023.2150611. Epub 2022 Nov 29.

    PubMed 36408587

  • Riedl MA, Neville D, Cloud B, Desai B, Bernstein JA. Shared decision-making in the management of hereditary angioedema: An analysis of patient and physician perspectives. Allergy Asthma Proc. 2022 Sep 1;43(5):397-405. doi: 10.2500/aap.2022.43.220050. Epub 2022 Jul 12.

    PubMed 35820771

  • Farkas H, Stobiecki M, Peter J, Kinaciyan T, Maurer M, Aygoren-Pursun E, Kiani-Alikhan S, Wu A, Reshef A, Bygum A, Fain O, Hagin D, Huissoon A, Jesenak M, Lindsay K, Panovska VG, Steiner UC, Zubrinich C, Best JM, Cornpropst M, Dix D, Dobo SM, Iocca HA, Desai B, Murray SC, Nagy E, Sheridan WP. Long-term safety and effectiveness of berotralstat for hereditary angioedema: The open-label APeX-S study. Clin Transl Allergy. 2021 Jun;11(4):e12035. doi: 10.1002/clt2.12035.

    PubMed 34161665

Questions about this trial

What is this clinical trial studying?

A Phase 2/3 study testing BCX7353 in people with Hereditary Angioedema and HAE, run by BioCryst Pharmaceuticals at 87 sites in 20 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 7 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 Jun 2023. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 87 sites in United States, Australia, United Kingdom, Israel, South Korea and 15 more countries. 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 combined 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.

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?

Yes. Results are posted in the registry (first posted 18 Jun 2023). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.

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?

Safety & Tolerability (time frame: Up to 96 weeks (US) / 216 weeks (Rest of World (ROW)).). 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 NCT03472040, which also lists 6 linked publications.

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 Jun 2023.

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