Hereditary AngioedemaPhase 3APeX-PNCT05453968
Berotralstat Treatment in Children With Hereditary Angioedema
A Phase 3 study testing Berotralstat in people with Hereditary Angioedema and Pediatric, run by BioCryst Pharmaceuticals at 15 sites in 10 countries.
Trial summary
The purpose of this study is to evaluate the pharmacokinetics (PK), safety and effectiveness of berotralstat to determine the appropriate weight-based dose for pediatric participants 2 to < 12 years of age for prophylactic treatment to prevent attacks of hereditary angioedema (HAE). Read more about this study
- Who is it for?
- Ages 2 to 11 years
- Conditions the study is for
- Hereditary Angioedema, Pediatric
- What is being tested?
- Berotralstat
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 29 participants
- Where
- 15 sitesin 10 countries
- Study dates
- 25 Oct 2022to Feb 2027
- 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 2 to 11 years
- All sexes
- No healthy volunteers
- Child
Who may be able to join
- Male and non-pregnant, non-lactating females 2 to < 12 years of age
- Body weight ≥ 12 kg
- Clinical diagnosis of HAE
- In the opinion of the investigator, the participant would benefit from long term oral HAE prophylaxis
- For subjects who are not currently receiving prophylaxis for HAE, documented history of >= 2 HAE attacks in the 6 months prior to the enrollment visit.
Who cannot join
- Concurrent diagnosis of any other type of recurrent angioedema
- Known family history of sudden cardiac death at a young age (< 40 years of age)
- Creatinine clearance using the modified Schwartz formula of ≤ 30 mL/min/1.73 m^2
- Aspartate aminotransferase or alanine aminotransferase value ≥ 3 × the upper limit of the age-appropriate normal reference range value
- Clinically significant abnormal electrocardiogram (ECG) including but not limited to, a corrected QT interval using Fridericia's correction > 450 msec, or ventricular and/or atrial premature contractions that are more frequent than occasional, and/or as couplets or higher in grouping
- Current participation in any other investigational drug study or received another investigational drug within 30 days of enrollment
Trial locations and contacts
15 sites in 10 countries.
15 sites in 10 countries
France3 sites
Investigative Site #3
Grenoble
Investigative Site #2
Marseille
Investigative Site #1
Paris
About this study
Official title: A Phase 3 Study to Evaluate the Safety and Pharmacokinetics of Berotralstat Prophylaxis in Children With Hereditary Angioedema Who Are 2 to < 12 Years of Age
The purpose of this study is to evaluate the pharmacokinetics (PK), safety and effectiveness of berotralstat to determine the appropriate weight-based dose for pediatric participants 2 to < 12 years of age for prophylactic treatment to prevent attacks of hereditary angioedema (HAE).
Read the detailed description
This is a single-arm, 3-part, open-label study designed to evaluate the PK, safety, and effectiveness of berotralstat weight-based treatment for the prevention of HAE attacks in pediatric participants 2 to < 12 years of age. Participation in this study is expected to be a minimum of 12 weeks in the standard of care (SOC) period, and in the berotralstat period, it is expected to be 12 weeks in Part 1, 36 weeks in Part 2, and 96 weeks in Part 3 of the study.
Participants were enrolled into 4 cohorts; participant weight at baseline was used to determine assignment to each cohort with the higher weight cohorts (Cohorts 1 and 2) enrolling first and in parallel. Safety assessments and PK modelling from all available PK data were then used to confirm the dose and weight bands for sequentially enrolling Cohorts 3 and 4. The safety and effectiveness of berotralstat in this population was summarized using descriptive statistical methods.
- Conditions
- Hereditary Angioedema, Pediatric
- Being tested
- Berotralstat
- Keywords
- Berotralstat, BCX7353, Orladeyo, once a day, Hereditary angioedema (HAE), Pediatric, kallikrein inhibitor, Oral
- 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, Orladeyo®
Administered orally once daily
| Study group | Role | What they receive |
|---|---|---|
| Cohort 1: ≥ 40 kg body weight (Berotralstat 150 mg) | Experimental | Participants received 150 milligram (mg) berotralstat capsule orally once daily for up to 144 weeks. Dose modifications were permitted due to weight changes, PK results, or safety. Receives: Drug: Berotralstat |
| Cohort 2: 32 to < 40 kg body weight (Berotralstat 108 mg) | Experimental | Participants received 108 mg berotralstat granules orally once daily for up to 144 weeks. Dose modifications were permitted due to weight changes, PK results, or safety. Receives: Drug: Berotralstat |
| Cohort 3: 24 to < 32 kg body weight (Berotralstat 96 mg) | Experimental | Participants received 96 mg berotralstat granules orally once daily for up to 144 weeks. Dose modifications were permitted due to weight changes, PK results, or safety. Receives: Drug: Berotralstat |
| Cohort 4: 12 to <24 kg body weight (Berotralstat 78 mg) | Experimental | Participants received 78 mg berotralstat granules orally once daily for up to 144 weeks. Dose modifications were permitted due to weight changes, PK results, or safety. Receives: Drug: Berotralstat |
What is being measured
The primary outcome is the main question the study is designed to answer.
Area Under the Plasma Concentration-Time Curve From Time 0 to the Last Measurable Concentration (AUC0-last) of Berotralstat
AUC0-last is the area under the plasma concentration-time curve from time 0 to the time of the last measurable concentration.
Measured: Week 2
Area Under the Plasma Concentration-Time Curve From Time 0 to 6 Hours Post-dose (AUC0-6) of Berotralstat
AUC0-6 is the area under the plasma concentration-time curve from time 0 to 6 hours.
Measured: Predose and up to 6 hours post dose at Week 2
Concentration at the End of the Dosing Interval (Ctrough) of Berotralstat
Ctrough is the concentration at the end of a dosing interval of berotralstat.
Measured: Predose at Week 2
Maximum Observed Plasma Concentration (Cmax) of Berotralstat
Cmax is the maximum observed plasma concentration of berotralstat.
Measured: Predose and up to 6 hours post dose at Week 2
Time of Last Measurable Plasma Concentration (Tlast) of Berotralstat
Tlast is the time of the last measurable concentration (Clast) of berotralstat collected over the sampling interval.
Measured: Predose and up to 6 hours post dose at Week 2
Time to Maximum Plasma Concentration (Tmax) of Berotralstat
Tmax is the time taken to reach the maximum observed plasma concentration of berotralstat.
Measured: Predose and up to 6 hours post dose at Week 2
Secondary outcomes (12)
Number of Participants With Treatment Emergent Adverse Events (TEAEs)
An adverse event (AE) is any untoward medical occurrence in a clinical study participant. No causal relationship with study drug or with the clinical study itself is implied. An AE could be an unfavorable and unintended sign, symptom (including an abnormal laboratory finding), syndrome, or illness that developed or worsened during the clinical study. A serious adverse event (SAE) is any untoward medical occurrence resulting in death, is life-threatening, requires inpatient hospitalization or prolongation of existing hospitalization, results in persistent or significant disability/incapacity, results in a congenital anomaly/birth defect, or other medically important event. TEAEs are AEs that occurred on/after first dose of berotralstat through 30 days post discontinuation of study treatment.
Measured: From first dose of study treatment up to approximately 73 weeks
Number of Adjusted Hereditary Angioedema (HAE) Attacks
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 (that is, allergic reaction, viral cold etc.)?",were considered unique (attack began > 24 hours from the end of the prior attack), and if the entire adjusted attack was untreated, it had a duration of > 24 hours. Any attack that began within 24 hours from the end of a prior attack was combined with the prior attack.
Measured: Week 1 through Week 12 and Week 1 through Week 48
Rate of Adjusted HAE Attacks
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 (that is, allergic reaction, viral cold etc.)?",were considered unique (attack began > 24 hours from the end of the prior attack), and if the entire adjusted attack was untreated, it had a duration of > 24 hours. Any attack that began within 24 hours from the end of a prior attack was combined with the prior attack.
The adjusted attack rate was calculated as the number of adjusted attacks observed during a given period and standardized to number of attacks per month, where 1 month is defined as a 28-day (4 week) period.
Measured: Week 1 through Week 12 and Week 1 through Week 48
Duration of Adjusted HAE Attack Symptoms
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 (that is, allergic reaction, viral cold etc.)?",were considered unique (attack began > 24 hours from the end of the prior attack), and if the entire adjusted attack was untreated, it had a duration of > 24 hours. Any attack that began within 24 hours from the end of a prior attack was combined with the prior attack. The duration of each participant-reported attack was calculated in hours, based on the start, and stop date and time of the adjusted attack (time the attack finished).
Measured: Week 1 through Week 12 and Week 1 through Week 48
Incidence of Adjusted HAE Attack Based on Anatomical Location
Adjusted attacks were assessed based on anatomical location. 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 (that is, allergic reaction, viral cold etc.)?", were considered unique (attack began > 24 hours from the end of the prior attack), and if the entire adjusted attack was untreated, it had a duration of > 24 hours. Any attack that began within 24 hours from the end of a prior attack was combined with the prior attack.
Anatomical locations were categorized as abdominal-only, non-abdominal peripheral, mixed, and laryngeal attacks.
Measured: Week 1 through Week 12 and Week 1 through Week 48
Number of Adjusted Attacks Requiring On-Demand Treatment
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 (that is, allergic reaction, viral cold etc.)?", were considered unique (attack began > 24 hours from the end of the prior attack), and if the entire adjusted attack was untreated, it had a duration of > 24 hours. Any attack that began within 24 hours from the end of a prior attack was combined with the prior attack.
Number of adjusted attacks treated with targeted HAE medications was assessed.
Measured: Week 1 through Week 12 and Week 1 through Week 48
Proportion of Adjusted Attacks Requiring On-Demand Treatment
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 (that is, allergic reaction, viral cold etc.)?", were considered unique (attack began > 24 hours from the end of the prior attack), and if the entire adjusted attack was untreated, it had a duration of > 24 hours. Any attack that began within 24 hours from the end of a prior attack was combined with the prior attack.
Proportion of adjusted attacks treated with targeted HAE medications was assessed.
Measured: Week 1 through Week 12 and Week 1 through Week 48
Number of Days With Angioedema Symptoms
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 (that is, allergic reaction, viral cold etc)?",were considered unique (attack began > 24 hours from the end of the prior attack),and if the entire adjusted attack was untreated, it had a duration of > 24 hours. Any attack that began within 24 hours from the end of a prior attack was combined with the prior attack.
The number of days with angioedema symptoms is the number of the days during the reporting period for which at least 1 symptom is reported during an adjusted HAE attack based on the start date and resolution date of an attack.
Measured: Week 1 through Week 12 and Week 1 through Week 48
Proportion of Days With Angioedema Symptoms
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 (that is, allergic reaction, viral cold etc)?",were considered unique (attack began > 24 hours from the end of the prior attack),and if the entire adjusted attack was untreated, it had a duration of > 24 hours. Any attack that began within 24 hours from the end of a prior attack was combined with the prior attack.
The proportion of days with angioedema symptoms was based on the number of days with reported symptoms and the number of days the participant was on treatment, where the number of days with angioedema symptoms is the number of the days during the reporting period for which at least 1 symptom is reported during an adjusted HAE attack based on the start date and resolution date of an attack.
Measured: Week 1 through Week 12 and Week 1 through Week 48
Assessment of Adjusted HAE Attack Severity
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 (that is, allergic reaction, viral cold etc.)?", were considered unique (attack began > 24 hours from the end of the prior attack), and if the entire adjusted attack was untreated, it had a duration of > 24 hours. Any attack that began within 24 hours from the end of a prior attack was combined with the prior attack. The severity was assessed by the parent/caregiver as negligible, mild, moderate or severe.
Measured: Week 1 through Week 12 and Week 1 through Week 48
Number of Participants Who Discontinued Treatment Due to Perceived Lack of Efficacy
The total number of participants who discontinued the treatment due to perceived lack of efficacy of berotralstat were reported.
Measured: Week 1 through Week 12 and Week 1 through Week 48
Number of Hospitalizations and Clinic Visits Due to HAE
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 (that is, allergic reaction, viral cold etc.)?", were considered unique (attack began > 24 hours from the end of the prior attack), and if the entire adjusted attack was untreated, it had a duration of > 24 hours. Any attack that began within 24 hours from the end of a prior attack was combined with the prior attack.
Number of adjusted attacks that required hospitalization or clinic visits are reported.
Measured: Week 1 through Week 12 and Week 1 through Week 48
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted12 Jul 2022
- Study start25 Oct 2022
- Primary completion11 Sep 2024
- Study completionFeb 2027 (estimated)
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 (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 3
- Primary purpose
- Prevention
- Allocation
- Non-randomised
- Intervention model
- Single group
- Model description
- This is a sequential, 3-part, open-label study. Participants were assigned a cohort based on weight at baseline.
- Masking
- None (open label)
- Enrolment
- 29 participants (actual)
Sponsor and investigators
- Lead sponsor
- BioCryst Pharmaceuticals
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Jolanta Bernatoniene, MD, Principal investigator, Bristol Royal Hospital for Children
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05453968
- Sponsor's protocol ID
- BCX7353-304
- Other IDs
- 2021-005932-50
- Acronym
- APeX-P
- Status last verified
- Feb 2026
- First submitted
- 30 Jun 2022
- Last updated
- 25 Feb 2026
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Publications
Bernatoniene J, Bourgoin-Heck M, Cancian M, Yang W, Hagin D, Pagnier A, Stobiecki M, Kinaciyan T, Phillips-Angles E, Gayet S, Bara NA, Hunter J, Mateescu E, DeSpirito M, Johnston D, Long D, Iocca H, Petroni D, Aygoren-Pursun E. Oral berotralstat for hereditary angioedema prophylaxis in patients aged 2 to <12 years: APeX-P interim results. Ann Allergy Asthma Immunol. 2025 Dec;135(6):681-688.e3. doi: 10.1016/j.anai.2025.07.012. Epub 2025 Jul 25.
PubMed 40716738
Study at a glance
From the registry record, last updated by the sponsor on 25 Feb 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase 3 Study to Evaluate the Safety and Pharmacokinetics of Berotralstat Prophylaxis in Children With Hereditary Angioedema Who Are 2 to < 12 Years of Age
- ClinicalTrials.gov ID
- NCT05453968
- Acronym / sponsor ID
- APeX-P / BCX7353-304
- Conditions
- Hereditary Angioedema, Pediatric
- Keywords
- Berotralstat, BCX7353, Orladeyo, once a day, Hereditary angioedema (HAE), Pediatric, kallikrein inhibitor, Oral
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- Active, not recruiting
- Enrolment
- 29 participants (actual)
- Lead sponsor
- BioCryst PharmaceuticalsIndustry (pharma, biotech, devices)
- Study start
- 25 Oct 2022 (actual)
- Primary completion
- 11 Sep 2024 (actual)
- Study completion
- Feb 2027 (estimated)
- First posted
- 12 Jul 2022 (actual)
- Results
- Posted 25 Feb 2026
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 evaluate the pharmacokinetics (PK), safety and effectiveness of berotralstat to determine the appropriate weight-based dose for pediatric participants 2 to < 12 years of age for prophylactic treatment to prevent attacks of hereditary angioedema (HAE).
Detailed description
This is a single-arm, 3-part, open-label study designed to evaluate the PK, safety, and effectiveness of berotralstat weight-based treatment for the prevention of HAE attacks in pediatric participants 2 to < 12 years of age. Participation in this study is expected to be a minimum of 12 weeks in the standard of care (SOC) period, and in the berotralstat period, it is expected to be 12 weeks in Part 1, 36 weeks in Part 2, and 96 weeks in Part 3 of the study.
Participants were enrolled into 4 cohorts; participant weight at baseline was used to determine assignment to each cohort with the higher weight cohorts (Cohorts 1 and 2) enrolling first and in parallel. Safety assessments and PK modelling from all available PK data were then used to confirm the dose and weight bands for sequentially enrolling Cohorts 3 and 4. The safety and effectiveness of berotralstat in this population was summarized using descriptive statistical methods.
Study design
- Primary purpose
- Prevention
- Allocation
- Non-randomised
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 4
- Model description
- This is a sequential, 3-part, open-label study. Participants were assigned a cohort based on weight at baseline.
| Arm | Type | Receives |
|---|---|---|
| Cohort 1: ≥ 40 kg body weight (Berotralstat 150 mg) | Experimental | Drug: BerotralstatParticipants received 150 milligram (mg) berotralstat capsule orally once daily for up to 144 weeks. Dose modifications were permitted due to weight changes, PK results, or safety. |
| Cohort 2: 32 to < 40 kg body weight (Berotralstat 108 mg) | Experimental | Drug: BerotralstatParticipants received 108 mg berotralstat granules orally once daily for up to 144 weeks. Dose modifications were permitted due to weight changes, PK results, or safety. |
| Cohort 3: 24 to < 32 kg body weight (Berotralstat 96 mg) | Experimental | Drug: BerotralstatParticipants received 96 mg berotralstat granules orally once daily for up to 144 weeks. Dose modifications were permitted due to weight changes, PK results, or safety. |
| Cohort 4: 12 to <24 kg body weight (Berotralstat 78 mg) | Experimental | Drug: BerotralstatParticipants received 78 mg berotralstat granules orally once daily for up to 144 weeks. Dose modifications were permitted due to weight changes, PK results, or safety. |
Who may qualify
- Ages
- 2 to 11 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (5 listed)
- Male and non-pregnant, non-lactating females 2 to < 12 years of age
- Body weight ≥ 12 kg
- Clinical diagnosis of HAE
- In the opinion of the investigator, the participant would benefit from long term oral HAE prophylaxis
- For subjects who are not currently receiving prophylaxis for HAE, documented history of >= 2 HAE attacks in the 6 months prior to the enrollment visit.
Key exclusion criteria (6 listed)
- Concurrent diagnosis of any other type of recurrent angioedema
- Known family history of sudden cardiac death at a young age (< 40 years of age)
- Creatinine clearance using the modified Schwartz formula of ≤ 30 mL/min/1.73 m^2
- Aspartate aminotransferase or alanine aminotransferase value ≥ 3 × the upper limit of the age-appropriate normal reference range value
- Clinically significant abnormal electrocardiogram (ECG) including but not limited to, a corrected QT interval using Fridericia's correction > 450 msec, or ventricular and/or atrial premature contractions that are more frequent than occasional, and/or as couplets or higher in grouping
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: BCX7353, Orladeyo®Administered orally once daily | Drug | Cohort 1: ≥ 40 kg body weight (Berotralstat 150 mg), Cohort 2: 32 to < 40 kg body weight (Berotralstat 108 mg), Cohort 3: 24 to < 32 kg body weight (Berotralstat 96 mg), Cohort 4: 12 to <24 kg body weight (Berotralstat 78 mg) |
Primary outcome
Area Under the Plasma Concentration-Time Curve From Time 0 to the Last Measurable Concentration (AUC0-last) of Berotralstat
Time frame: Week 2
AUC0-last is the area under the plasma concentration-time curve from time 0 to the time of the last measurable concentration.
Area Under the Plasma Concentration-Time Curve From Time 0 to 6 Hours Post-dose (AUC0-6) of Berotralstat
Time frame: Predose and up to 6 hours post dose at Week 2
AUC0-6 is the area under the plasma concentration-time curve from time 0 to 6 hours.
Concentration at the End of the Dosing Interval (Ctrough) of Berotralstat
Time frame: Predose at Week 2
Ctrough is the concentration at the end of a dosing interval of berotralstat.
Maximum Observed Plasma Concentration (Cmax) of Berotralstat
Time frame: Predose and up to 6 hours post dose at Week 2
Cmax is the maximum observed plasma concentration of berotralstat.
Time of Last Measurable Plasma Concentration (Tlast) of Berotralstat
Time frame: Predose and up to 6 hours post dose at Week 2
Tlast is the time of the last measurable concentration (Clast) of berotralstat collected over the sampling interval.
Time to Maximum Plasma Concentration (Tmax) of Berotralstat
Time frame: Predose and up to 6 hours post dose at Week 2
Tmax is the time taken to reach the maximum observed plasma concentration of berotralstat.
12 secondary outcomes
Number of Participants With Treatment Emergent Adverse Events (TEAEs)
Time frame: From first dose of study treatment up to approximately 73 weeks
Number of Adjusted Hereditary Angioedema (HAE) Attacks
Time frame: Week 1 through Week 12 and Week 1 through Week 48
Rate of Adjusted HAE Attacks
Time frame: Week 1 through Week 12 and Week 1 through Week 48
Duration of Adjusted HAE Attack Symptoms
Time frame: Week 1 through Week 12 and Week 1 through Week 48
Incidence of Adjusted HAE Attack Based on Anatomical Location
Time frame: Week 1 through Week 12 and Week 1 through Week 48
Number of Adjusted Attacks Requiring On-Demand Treatment
Time frame: Week 1 through Week 12 and Week 1 through Week 48
Proportion of Adjusted Attacks Requiring On-Demand Treatment
Time frame: Week 1 through Week 12 and Week 1 through Week 48
Number of Days With Angioedema Symptoms
Time frame: Week 1 through Week 12 and Week 1 through Week 48
Proportion of Days With Angioedema Symptoms
Time frame: Week 1 through Week 12 and Week 1 through Week 48
Assessment of Adjusted HAE Attack Severity
Time frame: Week 1 through Week 12 and Week 1 through Week 48
Number of Participants Who Discontinued Treatment Due to Perceived Lack of Efficacy
Time frame: Week 1 through Week 12 and Week 1 through Week 48
Number of Hospitalizations and Clinic Visits Due to HAE
Time frame: Week 1 through Week 12 and Week 1 through Week 48
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 25 Feb 2026.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Derived from this study: PubMed 40716738Bernatoniene J, Bourgoin-Heck M, Cancian M, Yang W, Hagin D, Pagnier A, Stobiecki M, Kinaciyan T, Phillips-Angles E, Gayet S, Bara NA, Hunter J, Mateescu E, DeSpirito M, Johnston D, Long D, Iocca H, Petroni D, Aygoren-Pursun E. Oral berotralstat for hereditary angioedema prophylaxis in patients aged 2 to <12 years: APeX-P interim results. Ann Allergy Asthma Immunol. 2025 Dec;135(6):681-688.e3. doi: 10.1016/j.anai.2025.07.012. Epub 2025 Jul 25.
- Individual participant dataSharing planned: No
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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.
Jolanta Bernatoniene, MD principal investigator
Bristol Royal Hospital for Children
15 sites in 10 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing Berotralstat in people with Hereditary Angioedema and Pediatric, run by BioCryst Pharmaceuticals at 15 sites in 10 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 5 inclusion and 6 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: under way, not looking for participants, as of the sponsor's last update on 25 Feb 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 15 sites in France, Germany, Israel, Spain, Austria and 5 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 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 25 Feb 2026). 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?
Area Under the Plasma Concentration-Time Curve From Time 0 to the Last Measurable Concentration (AUC0-last) of Berotralstat (time frame: Week 2). The study lists 6 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 NCT05453968, 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 25 Feb 2026.