Hereditary Angioedema, HAEPhase 3APeX-JNCT03873116

Study to Evaluate the Efficacy and Safety of BCX7353 as an Oral Treatment for the Prevention of HAE Attacks in Japan

A Phase 3 study testing BCX7353 capsules in people with Hereditary Angioedema, HAE, run by BioCryst Pharmaceuticals at 10 sites in Japan.

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 a phase 3, multi-center, randomized, double-blind, placebo-controlled trial to evaluate the efficacy and safety of oral BCX7353 in preventing acute angioedema attacks in patients with Type I and Type II HAE who live in Japan. Read more about this study

Who is it for?: Ages 12 years and older
Conditions the study is for: Hereditary Angioedema, HAE
What is being tested?: BCX7353 capsules
Type of treatment: Pill or oral medicine
Could I receive a placebo?: Yes, some participants may receive a placebo
How many people: 19 participants
Where: 10 sitesJapan
Study dates: 27 Dec 2018to 8 Jul 2021
Phase: Phase 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

A clinical diagnosis of hereditary angioedema (HAE) Type 1 or Type 2, defined as having a C1-INH functional level and a C4 level below the lower limit of the normal (LLN) reference range, as assessed during the Screening period.
Access to and ability to use one or more acute medications approved by the relevant competent authority for the treatment of acute attacks of HAE
Subjects must be medically appropriate for on-demand treatment as the sole medicinal management for their HAE during the study.
Subjects must have a specified number of expert-confirmed attacks during the run-in period of 56 days from the Screening visit.
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
Severe hypersensitivity to multiple medicinal products or severe hypersensitivity/ anaphylaxis with unclear etiology
Use of C1-INH within 14 days or use of androgens or tranexamic acid within 28 days prior to the Screening visit for prophylaxis of HAE attacks, or initiation of these drugs during the study
Current participation in any other investigational drug study or received another investigational drug within 30 days of the Screening visit
Prior enrollment in a BCX7353 study

Trial locations and contacts

10 sites in 1 country.

City, postcode or hospital

10 sites in 1 country

Japan10 sites

Study Site
Chiba
Study Site
Gunma
Study Site
Hokkaido
Study Site
Nagoya
Study Site
Osaka
Study Center
Saga
Study Site
Saitama
Study Site
Shimane
Study Site
Shizuoka
Study Site
Tokyo

Other trials in Japan

About this study

Official title: A Phase 3, Randomized, Double-blind, Placebo-controlled, Parallel-group Study to Evaluate the Efficacy and Safety of Two Dose Levels of BCX7353 as an Oral Treatment for the Prevention of Attacks in Subjects With Hereditary Angioedema

Conditions: Hereditary Angioedema, HAE
Being tested: BCX7353 capsules, Placebo oral capsule
Keywords: BCX7353
Medical subject headings: Angioedemas, Hereditary

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

BCX7353 capsulesDrug
BCX7353 capsules administered orally once daily
BCX7353 capsulesDrug
BCX7353 capsules administered orally once daily
Placebo oral capsuleDrug
Matching placebo capsules administered orally once daily

Study groups in NCT03873116
Study group	Role	What they receive
BCX7353 110mg once daily	Experimental	BCX7353 capsules administered orally once daily Receives: Drug: BCX7353 capsules
BCX7353 150mg once daily	Experimental	BCX7353 capsules administered orally once daily Receives: Drug: BCX7353 capsules
Placebo	Placebo comparator	Matching placebo oral capsules administered orally once daily Receives: Drug: Placebo oral capsule

What is being measured

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

Part 1: The Rate of Expert-confirmed HAE Attacks During Dosing in the Entire 24-week Treatment Period (Day 1 to Day 168)
The angioedema event rate and the treatment comparisons between each berotralstat dose and placebo in the rate of expert-confirmed angioedema events during the entire dosing period was analyzed using a negative binomial regression model. The number of expert-confirmed angioedema events was included as the dependent variable, the treatment was included as a fixed effect, the stratification variable (baseline monthly angioedema event rate) and study (for the combined study analysis) were included as covariates, and the logarithm of duration on treatment was included as an offset variable. The estimated rate of angioedema events for each treatment group, the treatment differences expressed as the angioedema event rate ratio (berotralstat) over placebo rate ratio), and their associated 95% confidence intervals (CIs) were provided from the negative binomial regression model.
Measured: 24 weeks
Part 2: To Evaluate the Long-term Safety and Tolerability of Berotralstat 110 and 150 mg in Subjects With HAE
The safety data was assessed for the safety population for subjects who entered Part 2, and includes TEAEs that occurred in Part 1 and Part 2 for these subjects with a data cut-off date of 10-April-2020. TEAEs are defined as AEs that occurred on or after first dose of study treatment, whether in Part 1 or 2, and were assigned to the relevant treatment depending on when the TEAE began (Part 1 or Part 2 treatment). TEAEs were assessed for severity (graded) using the Division of Microbiology and Infectious Disease (DMID) criteria for grading AEs. TEAEs not covered by the DMID criteria were assessed as Mild (Grade 1), Moderate (Grade 2), Severe (Grade 3) or Life-threatening (Grade 4).
Measured: Part 1: 24 weeks (Week 0 to 24 to 52), Part 2: 28 weeks (Week 24 to 52)
Part 3: To Evaluate the Long-term Safety and Tolerability of Berotralstat Administered QD Over a 52- to up to 104-week Administration Period in Subjects With HAE.
The safety data was assessed for the safety population for subjects who entered Part 3, and includes TEAEs that occurred in Part 3 for these subjects.
Measured: Part 3: Week 52 to up to Week 104.

Secondary outcomes (8)

Part 1: Proportion of Days With Angioedema Symptoms Through 24 Weeks.
Assessment of number and proportion of days subjects had angioedema symptoms from expert-confirmed angioedema events during Part 1.
Measured: 24 weeks
Part 1: Rate of Expert-confirmed Angioedema Events During Dosing in the Effective Treatment Period
The rate of expert-confirmed angioedema events for the effective treatment period gives an analysis of the efficacy of active treatment after berotralstat had reached steady-state concentrations, given the effective half-life of 150 mg berotralstat in Study BCX7353-106 (Study 106) of 89 hours.
Measured: Day 8 through to 24 weeks
Part 1: Change From Baseline in Angioedema Quality of Life (AE-QoL) Questionnaire at Week 24 (Total Score)
Change in Quality of Life, on a 1-100 scale, where higher scores indicate more impairment and a decrease (change with a negative value) in AE-QoL questionnaire scores indicates an improvement in the subject's QoL. The minimum clinically important difference (MCID) for the AE-QoL questionnaire is -6 (total score).
Measured: Baseline and 24 weeks
Part 2: To Assess the Effectiveness of Berotralstat Over a 24- to 52-week Period
Monthly Attack Rate was defined as the total number of investigator-confirmed 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. The end of Month 6 was defined as the start of Part 2 treatment. For crossover subjects receiving active treatment following placebo, months were adjusted according to the date of the first dose of active treatment.
Baseline investigator-confirmed attack rate was defined as the total number of investigator-confirmed HAE attacks experienced in the period between screening and first dose of study drug adjusted for the length of a month (defined as 28 days) and the number of days during that period.
Measured: 28 weeks (Week 24 to 52)
Part 2: To Evaluate QoL Following Berotralstat Administration Over a 24- to 52-week Period.
Change in Quality of Life, on a 1-100 scale, where higher scores indicate more impairment and a decrease (change with a negative value) in AE-QoL questionnaire scores indicates an improvement in the subject's QoL. The minimum clinically important difference (MCID) for the AE-QoL questionnaire is -6 (total score). The AE-QoL is only validated for adults; however, data were collected on all adult and adolescent study subjects.
Measured: 28 Weeks (Week 24 to 52)
Part 3: To Assess the Effectiveness of Berotralstat Over a 52- to up to 104-week Administration Period
Adjusted subject-reported event rate was defined as (total number of adjusted subject-reported HAE events experienced in the period between the Week 52 visit and end of study [or the last dose date/time in Part 3 + 24 hours for subjects who discontinued drug in Part 3]) * 28/(date of last dose in Part 3 - date of Week 52 visit + 1).
Measured: 52 weeks (Week 52 to 104)
Part 3: To Evaluate QoL Following Berotralstat Administration Over a 52- to up to 104-week Period
Change in Quality of Life, on a 1-100 scale, where higher scores indicate more impairment and a decrease (change with a negative value) in AE-QoL questionnaire scores indicates an improvement in the subject's QoL. The minimum clinically important difference (MCID) for the AE-QoL questionnaire is -6 (total score). For subjects who received active treatment following placebo, visits were adjusted according to the date of the first dose of active treatment.
Measured: 52 Weeks (Week 52 to 104)
Part 3: To Evaluate Subject's Satisfaction With Berotralstat During 52- to 104-week Administration Period
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. Scales 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. For subjects who received active treatment following placebo, visits were adjusted according to the date of the first dose of active treatment.
Measured: 52 Weeks (Week 52 to 104)

Timeline

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

27 Dec 20188 Jul 2021

First posted13 Mar 2019
Study start27 Dec 2018
Primary completion8 Jul 2021
Study completion8 Jul 2021

Resources

Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
Publications (4)Papers the sponsor has linked to this study.
Other trials for Hereditary Angioedema, HAEStudies 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: Randomised
Intervention model: Parallel groups
Masking: Triple blind
Who is masked: Participant, Care provider, Investigator
Enrolment: 19 participants (actual)

IDs, dates and publications

ClinicalTrials.gov ID: NCT03873116
Sponsor's protocol ID: BCX7353-301
Acronym: APeX-J
Status last verified: Feb 2024
First submitted: 11 Mar 2019
Last updated: 19 Jul 2024
Results posted: Yes, on ClinicalTrials.gov
Data monitoring committee: Yes
FDA-regulated drug: No
FDA-regulated device: No
Individual data sharing: No

Publications

Honda D, Hide M, Fukuda T, Koga K, Morita E, Moriwaki S, Sasaki Y, Suzuki Y, Collis P, Johnston DT, Tomita D, Desai B, Ohsawa I. Berotralstat for long-term prophylaxis of hereditary angioedema in Japan: Parts 2 and 3 of the randomized APeX-J Phase III trial. World Allergy Organ J. 2024 Mar 2;17(3):100882. doi: 10.1016/j.waojou.2024.100882. eCollection 2024 Mar.
PubMed 38445295
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
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
Ohsawa I, Honda D, Suzuki Y, Fukuda T, Kohga K, Morita E, Moriwaki S, Ishikawa O, Sasaki Y, Tago M, Chittick G, Cornpropst M, Murray SC, Dobo SM, Nagy E, Van Dyke S, Reese L, Best JM, Iocca H, Collis P, Sheridan WP, Hide M. Oral berotralstat for the prophylaxis of hereditary angioedema attacks in patients in Japan: A phase 3 randomized trial. Allergy. 2021 Jun;76(6):1789-1799. doi: 10.1111/all.14670. Epub 2020 Dec 23.
PubMed 33247955

Want to know more?

The sponsor has not listed a contact. The official record may have more details.

Talk to your doctor first and take this page with you. Only the study team can confirm whether you can join.

Questions to ask the study team

What is the study trying to find out?
What would I need to do, and how often would I visit?
Could I receive a placebo instead of the treatment?
What are the possible risks and benefits for me?
Will it cost me anything? Are travel or time paid for?
Can I keep seeing my own doctor during the study?
What happens when the study ends?

See locations

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All Hereditary Angioedema, HAE trials

Study at a glance

From the registry record, last updated by the sponsor on 19 Jul 2024. Recruitment status is as the sponsor reported it: confirm with the study team.

Official title: A Phase 3, Randomized, Double-blind, Placebo-controlled, Parallel-group Study to Evaluate the Efficacy and Safety of Two Dose Levels of BCX7353 as an Oral Treatment for the Prevention of Attacks in Subjects With Hereditary Angioedema
ClinicalTrials.gov ID: NCT03873116
Acronym / sponsor ID: APeX-J / BCX7353-301
Conditions: Hereditary Angioedema, HAE
Keywords: BCX7353
Phase: Phase 3
Study type: Interventional
Recruitment status: Completed
Enrolment: 19 participants (actual)
Lead sponsor: BioCryst PharmaceuticalsIndustry (pharma, biotech, devices)
Study start: 27 Dec 2018 (actual)
Primary completion: 8 Jul 2021 (actual)
Study completion: 8 Jul 2021 (actual)
First posted: 13 Mar 2019 (actual)
Results: Posted 4 Mar 2021

Clinical summary

The research question as the sponsor registered it. This describes what the study plans to test, not what it found.

Study design

Primary purpose: Prevention
Allocation: Randomised
Intervention model: Parallel groups
Masking: Triple blind (participant, care provider, investigator)
Number of arms: 3

Arm	Type	Receives
BCX7353 110mg once daily	Experimental	Drug: BCX7353 capsulesBCX7353 capsules administered orally once daily
BCX7353 150mg once daily	Experimental	Drug: BCX7353 capsulesBCX7353 capsules administered orally once daily
Placebo	Placebo comparator	Drug: Placebo oral capsuleMatching placebo oral capsules administered orally once daily

Who may qualify

Ages: 12 years and older
Sex: All
Healthy volunteers: Not accepted
Age groups: Child, Adult, Older adult

Key inclusion criteria (6 listed)

A clinical diagnosis of hereditary angioedema (HAE) Type 1 or Type 2, defined as having a C1-INH functional level and a C4 level below the lower limit of the normal (LLN) reference range, as assessed during the Screening period.
Access to and ability to use one or more acute medications approved by the relevant competent authority for the treatment of acute attacks of HAE
Subjects must be medically appropriate for on-demand treatment as the sole medicinal management for their HAE during the study.
Subjects must have a specified number of expert-confirmed attacks during the run-in period of 56 days from the Screening visit.
Acceptable effective contraception

Key exclusion criteria (7 listed)

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
Severe hypersensitivity to multiple medicinal products or severe hypersensitivity/ anaphylaxis with unclear etiology
Use of C1-INH within 14 days or use of androgens or tranexamic acid within 28 days prior to the Screening visit for prophylaxis of HAE attacks, or initiation of these drugs during the study

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
BCX7353 capsulesBCX7353 capsules administered orally once daily	Drug	BCX7353 110mg once daily
BCX7353 capsulesBCX7353 capsules administered orally once daily	Drug	BCX7353 150mg once daily
Placebo oral capsuleMatching placebo capsules administered orally once daily	Drug	Placebo

Primary outcome

Part 1: The Rate of Expert-confirmed HAE Attacks During Dosing in the Entire 24-week Treatment Period (Day 1 to Day 168)
Time frame: 24 weeks
The angioedema event rate and the treatment comparisons between each berotralstat dose and placebo in the rate of expert-confirmed angioedema events during the entire dosing period was analyzed using a negative binomial regression model. The number of expert-confirmed angioedema events was included as the dependent variable, the treatment was included as a fixed effect, the stratification variable (baseline monthly angioedema event rate) and study (for the combined study analysis) were included as covariates, and the logarithm of duration on treatment was included as an offset variable. The estimated rate of angioedema events for each treatment group, the treatment differences expressed as the angioedema event rate ratio (berotralstat) over placebo rate ratio), and their associated 95% confidence intervals (CIs) were provided from the negative binomial regression model.
Part 2: To Evaluate the Long-term Safety and Tolerability of Berotralstat 110 and 150 mg in Subjects With HAE
Time frame: Part 1: 24 weeks (Week 0 to 24 to 52), Part 2: 28 weeks (Week 24 to 52)
The safety data was assessed for the safety population for subjects who entered Part 2, and includes TEAEs that occurred in Part 1 and Part 2 for these subjects with a data cut-off date of 10-April-2020. TEAEs are defined as AEs that occurred on or after first dose of study treatment, whether in Part 1 or 2, and were assigned to the relevant treatment depending on when the TEAE began (Part 1 or Part 2 treatment). TEAEs were assessed for severity (graded) using the Division of Microbiology and Infectious Disease (DMID) criteria for grading AEs. TEAEs not covered by the DMID criteria were assessed as Mild (Grade 1), Moderate (Grade 2), Severe (Grade 3) or Life-threatening (Grade 4).
Part 3: To Evaluate the Long-term Safety and Tolerability of Berotralstat Administered QD Over a 52- to up to 104-week Administration Period in Subjects With HAE.
Time frame: Part 3: Week 52 to up to Week 104.
The safety data was assessed for the safety population for subjects who entered Part 3, and includes TEAEs that occurred in Part 3 for these subjects.

8 secondary outcomes

Part 1: Proportion of Days With Angioedema Symptoms Through 24 Weeks.
Time frame: 24 weeks
Part 1: Rate of Expert-confirmed Angioedema Events During Dosing in the Effective Treatment Period
Time frame: Day 8 through to 24 weeks
Part 1: Change From Baseline in Angioedema Quality of Life (AE-QoL) Questionnaire at Week 24 (Total Score)
Time frame: Baseline and 24 weeks
Part 2: To Assess the Effectiveness of Berotralstat Over a 24- to 52-week Period
Time frame: 28 weeks (Week 24 to 52)
Part 2: To Evaluate QoL Following Berotralstat Administration Over a 24- to 52-week Period.
Time frame: 28 Weeks (Week 24 to 52)
Part 3: To Assess the Effectiveness of Berotralstat Over a 52- to up to 104-week Administration Period
Time frame: 52 weeks (Week 52 to 104)
Part 3: To Evaluate QoL Following Berotralstat Administration Over a 52- to up to 104-week Period
Time frame: 52 Weeks (Week 52 to 104)
Part 3: To Evaluate Subject's Satisfaction With Berotralstat During 52- to 104-week Administration Period
Time frame: 52 Weeks (Week 52 to 104)

Results and safety

Results posted to the registry on 4 Mar 2021, as reported by the sponsor. Figures are copied from the record, not re-analysed. Full results on ClinicalTrials.gov.

Participant flow

Group	Started	Completed
Berotralstat 110mg Once Daily	3	3
Berotralstat 110mg QD, Followed by Berotralstat 150 mg QD	3	3
Berotralstat 110mg QD After Placebo	2	2
Berotralstat 110mg QD, Followed by Berotralstat 150 mg QD, After Placebo	1	1
Berotralstat 150mg Once Daily	7	7
Berotralstat 150mg QD After Placebo	3	2

Primary outcome: Part 1: The Rate of Expert-confirmed HAE Attacks During Dosing in the Entire 24-week Treatment Period (Day 1 to Day 168)

Time frame: 24 weeks. Mean (Standard Deviation). Unit: Angioedema event rate per 28 days

Measure	Berotralstat 110mg Once Daily n = 6	Berotralstat 150mg Once Daily n = 7	Placebo n = 6
Value	1.961 (1.3021)	1.089 (0.9168)	2.734 (1.6359)

Berotralstat 110mg Once Daily vs Placebo: negative binomial regression model: -24.6 (95% CI -50.1 to 14.0), p = 0.181, negative binomial regression model
Berotralstat 150mg Once Daily vs Placebo: negative binomial regression model: -49.1 (95% CI -67.5 to -20.4), p = 0.003, negative binomial regression model

Population analysed: The intent to treat (ITT) population included all randomized subjects, regardless of whether study treatment was administered. This population was the primary population for the analysis of the efficacy and health outcomes data.

Primary outcome: Part 2: To Evaluate the Long-term Safety and Tolerability of Berotralstat 110 and 150 mg in Subjects With HAE

Time frame: Part 1: 24 weeks (Week 0 to 24 to 52), Part 2: 28 weeks (Week 24 to 52). Count of participants. Unit: Participants

Measure	Berotralstat 110mg Once Daily n = 6	Berotralstat 110mg QD After Placebo n = 2	Berotralstat 150mg Once Daily n = 7	Berotralstat 150mg QD After Placebo n = 2	Placebo n = 6
TEAE	6	1	7	2	6
Drug-related TEAE	2	0	3	0	2
SAE	2	0	0	0	0
Drug-related SAE	0	0	0	0	0
Grade 3 or 4 TEAE	0	0	1	0	1
Drug-related grade 3 or 4 TEAE	0	0	1	0	0
TEAE leading to interruption of study drug	1	0	2	0	0
TEAE leading to discontinuation of study drug	0	0	1	0	1

Population analysed: The safety population included all subjects who received at least 1 capsule of study treatment.

Primary outcome: Part 3: To Evaluate the Long-term Safety and Tolerability of Berotralstat Administered QD Over a 52- to up to 104-week Administration Period in Subjects With HAE.

Time frame: Part 3: Week 52 to up to Week 104.. Count of participants. Unit: Participants

Measure	Berotralstat 150mg Once Daily n = 11
TEAE	6
Drug-related TEAE	0
SAE	1
Drug-related SAE	0
Grade 3 or 4 TEAE	1
Drug-related grade 3 or 4 TEAE	0
TEAE leading to interruption of study drug	0
TEAE leading to discontinuation of study drug	0

8 secondary outcomes are reported in the full results.

Adverse events

Time frame: Adverse Events (AEs) were assessed and recorded from the time that the Informed consent form was signed until the last follow-up visit, approximately 3 weeks following the last dose of study drug, or until the AE was resolved or the subject was in a clinically stable condition with regard to the AE. AEs were assigned to the relevant treatment depending on when the AE began; i.e., Part 1 or Part 2 and 3 treatment.. Other adverse events reported at 5% frequency or more

Group	Serious adverse events	Other adverse events	Deaths
Berotralstat 110mg Once Daily	2 of 8 (25%)	8 of 8 (100%)	0 of 8
Berotralstat 150mg Once Daily	1 of 9 (11.1%)	9 of 9 (100%)	0 of 9
Placebo	0 of 6 (0%)	6 of 6 (100%)	0 of 6

Loading the posted results from ClinicalTrials.gov…

Research links

Official record on ClinicalTrials.govLast updated by the sponsor 19 Jul 2024.
Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
Derived from this study: PubMed 38445295Honda D, Hide M, Fukuda T, Koga K, Morita E, Moriwaki S, Sasaki Y, Suzuki Y, Collis P, Johnston DT, Tomita D, Desai B, Ohsawa I. Berotralstat for long-term prophylaxis of hereditary angioedema in Japan: Parts 2 and 3 of the randomized APeX-J Phase III trial. World Allergy Organ J. 2024 Mar 2;17(3):100882. doi: 10.1016/j.waojou.2024.100882. eCollection 2024 Mar.
Derived from this study: PubMed 36408587Farkas 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.
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.
Derived from this study: PubMed 33247955Ohsawa I, Honda D, Suzuki Y, Fukuda T, Kohga K, Morita E, Moriwaki S, Ishikawa O, Sasaki Y, Tago M, Chittick G, Cornpropst M, Murray SC, Dobo SM, Nagy E, Van Dyke S, Reese L, Best JM, Iocca H, Collis P, Sheridan WP, Hide M. Oral berotralstat for the prophylaxis of hereditary angioedema attacks in patients in Japan: A phase 3 randomized trial. Allergy. 2021 Jun;76(6):1789-1799. doi: 10.1111/all.14670. Epub 2020 Dec 23.
Individual participant dataSharing planned: No

Related trials for Hereditary Angioedema, HAE

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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.

Isao Ohsawa principal investigator

Saiyu Soka Hospital

10 sites in 1 country: see every site and its status.

Questions about this trial

What is this clinical trial studying?

A Phase 3 study testing BCX7353 capsules in people with Hereditary Angioedema, HAE, run by BioCryst Pharmaceuticals at 10 sites in Japan.

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 6 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 19 Jul 2024. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 10 sites in Japan. 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 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 4 Mar 2021). 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?

Part 1: The Rate of Expert-confirmed HAE Attacks During Dosing in the Entire 24-week Treatment Period (Day 1 to Day 168) (time frame: 24 weeks). The study lists 3 primary outcomes in all. 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 NCT03873116, which also lists 4 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 19 Jul 2024.