A Phase 3 interventional study of Berotralstat in Hereditary Angioedema and Pediatric, sponsored by BioCryst Pharmaceuticals. Active, not recruiting at 15 sites in 10 countries. Open to participants aged 2 Years to 11 Years. Per ClinicalTrials.gov, last updated 2026-02-25.
Sponsored by BioCryst Pharmaceuticals · Phase 3, Interventional, and Prevention
Among 11 studies testing berotralstat in Angioedemas, Hereditary — 1 open to participants now, 2 under way, 2 of them phase 3. berotralstat · Angioedemas, Hereditary.
Among the 11 studies testing berotralstat in Angioedemas, Hereditary, this one is the 8th largest by enrollment, is the only one that started in 2022, is one of 4 in phase 3, and was the 11th registered.
171 studies on the registry are indexed under Angioedemas, Hereditary; 24 are open to participants now.
Browse Angioedemas, Hereditary studies →1 of the 11 studies testing berotralstat in Angioedemas, Hereditary is open now, in 11 countries. Recruiting first, the newest first:
BioCryst Pharmaceuticals · Sites in Canada, Czechia, France and 8 more countries
By country, each count listing its studies:
berotralstat: first FDA approval 2020 (ORLADEYO), first EMA approval 2021. What it is approved for, and how studies give it, is on its page →
Of the 13 Angioedemas, Hereditary studies recruiting now, 9 (69%) set no upper age limit and 4 (31%) stop at 75 or younger; 7 take children under 18; 1 take healthy volunteers.
This study: 2 Years to 11 Years · all sexes · no healthy volunteers.
The full table on the hub →This tab as a report to print or save as PDF → With an account.
11 studies; the count open now beside each. This study's row is marked.
A study in two phases is counted once above. Listed by phase, each with its combined phases: Phase 1 4 · Phase 2 3 · Phase 3 5
First in 2015; this study’s year is marked. 1 started in 2022.
This study's enrollment of 29 is below the median of 48 across 116 interventional studies indexed under Angioedemas, Hereditary.
Median enrollment 58 across the 11 interventional studies. Of the 9 that completed or were terminated, 5 (56%) have results posted.
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11 of 11 have an industry lead sponsor. The 1 most frequent:
BioCryst Pharmaceuticals is the lead sponsor of 54 studies on the registry; 1 is open to participants now.
Of its 10 completed or terminated interventional studies of FDA-regulated products, 8 (80%) have results posted.
17 studies in Angioedemas, Hereditary by BioCryst Pharmaceuticals: 1 open now, 2 under way, 2 of them phase 3. Of 14 finished, 6 (43%) have results posted.
Among the 17 studies in Angioedemas, Hereditary by BioCryst Pharmaceuticals, this one is the 9th largest by enrollment of the 16 interventional ones that give a count, is the only one that started in 2022, is one of 5 in phase 3, and was the 17th registered.
BioCryst Pharmaceuticals’s Angioedemas, Hereditary studies by the year they started, since 2013.
None of the studies under way reaches primary completion within a year.
Among the 11 studies testing berotralstat in Angioedemas, Hereditary, from their registry updates:
Nothing recorded among them since 2026-09-25.
Among BioCryst Pharmaceuticals’s 17 in Angioedemas, Hereditary:
Nothing recorded among them since 2026-09-25.
This tab as a report to print or save as PDF → With an account.
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).
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.
Go through the criteria one by one — yes, no or not sure — and take the list to your doctor or the study team. Kept in this browser only.
Exclusion Criteria:
Underlined terms in the criteria open these too. Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
How quickly the kidneys clear creatinine, a waste product, from the blood. A study may estimate it from a blood test with age, weight and sex (often by the Cockcroft–Gault formula), or measure it using blood and urine collected over a set time. Given in mL/min; higher is better.
Two enzymes measured in a routine blood test of the liver. Higher levels can mean the liver is under strain or damaged. SGPT and SGOT are older names for ALT and AST.
Criteria usually give a limit as a multiple of the upper limit of normal (ULN), often higher when cancer has spread to the liver.
Judged by the study doctor to matter for your health or for the study. It is a doctor’s decision, so ask the study team if you are unsure whether something counts.
A recording of the heart’s electrical activity, taken with stickers on the chest, arms and legs. It takes a few minutes and does not hurt.
On an ECG, the QT interval is the time the heart’s lower chambers take to activate electrically and recover in each beat. QTc is that time adjusted for heart rate (QTcF by Fridericia’s formula, QTcB by Bazett’s). Some medicines lengthen it, and a long QTc can raise the risk of a dangerous heart rhythm.
The study may name a correction formula and a limit in milliseconds, often around 450–480.
A drug or device being studied in a clinical trial.
It may be new, or already approved and being studied for another use, dose or form. Criteria often leave out people who took part in another study of one within a recent period, commonly 30 days or five half-lives.
Creatinine clearance (CrCl)
How quickly the kidneys clear creatinine, a waste product, from the blood. A study may estimate it from a blood test with age, weight and sex (often by the Cockcroft–Gault formula), or measure it using blood and urine collected over a set time. Given in mL/min; higher is better.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
ALT and AST (liver enzymes)
Two enzymes measured in a routine blood test of the liver. Higher levels can mean the liver is under strain or damaged. SGPT and SGOT are older names for ALT and AST.
Criteria usually give a limit as a multiple of the upper limit of normal (ULN), often higher when cancer has spread to the liver.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Clinically significant
Judged by the study doctor to matter for your health or for the study. It is a doctor’s decision, so ask the study team if you are unsure whether something counts.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
ECG (electrocardiogram)
A recording of the heart’s electrical activity, taken with stickers on the chest, arms and legs. It takes a few minutes and does not hurt.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
QT and QTc
On an ECG, the QT interval is the time the heart’s lower chambers take to activate electrically and recover in each beat. QTc is that time adjusted for heart rate (QTcF by Fridericia’s formula, QTcB by Bazett’s). Some medicines lengthen it, and a long QTc can raise the risk of a dangerous heart rhythm.
The study may name a correction formula and a limit in milliseconds, often around 450–480.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Investigational drug or device
A drug or device being studied in a clinical trial.
It may be new, or already approved and being studied for another use, dose or form. Criteria often leave out people who took part in another study of one within a recent period, commonly 30 days or five half-lives.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
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.
Drug: Berotralstat
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.
Drug: Berotralstat
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.
Drug: Berotralstat
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.
Drug: Berotralstat
Administered orally once daily
Also known as: BCX7353, Orladeyo®
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.
Time frame: 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.
Time frame: 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.
Time frame: Predose at Week 2
Maximum Observed Plasma Concentration (Cmax) of Berotralstat
Cmax is the maximum observed plasma concentration of berotralstat.
Time frame: 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.
Time frame: 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.
Time frame: Predose and up to 6 hours post dose at Week 2
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.
Time frame: 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.
Time frame: 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.
Time frame: 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).
Time frame: 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.
Time frame: 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.
Time frame: 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.
Time frame: 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.
Time frame: 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.
Time frame: 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.
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
The total number of participants who discontinued the treatment due to perceived lack of efficacy of berotralstat were reported.
Time frame: 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.
Time frame: Week 1 through Week 12 and Week 1 through Week 48
A total of 29 pediatric participants were enrolled in the study at a total of 11 study sites in Austria, Canada, France, Germany, Italy, Israel, Poland, Spain and the United Kingdom (UK).
| Milestone | Pre-treatment SOC Period (12 Weeks) | 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) |
|---|---|---|---|---|---|
| Started | 29 | 0 | 0 | 0 | 0 |
| Completed | 29 | 0 | 0 | 0 | 0 |
| Not completed | 0 | 0 | 0 | 0 | 0 |
| Milestone | Pre-treatment SOC Period (12 Weeks) | 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) |
|---|---|---|---|---|---|
| Started | 0 | 7 | 9 | 9 | 4 |
| Completed | 0 | 7 | 9 | 9 | 4 |
| Not completed | 0 | 0 | 0 | 0 | 0 |
| Milestone | Pre-treatment SOC Period (12 Weeks) | 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) |
|---|---|---|---|---|---|
| Started | 0 | 7 | 9 | 9 | 4 |
| Completed | 0 | 7 | 6 | 4 | 0 |
| Not completed | 0 | 0 | 3 | 5 | 4 |
| Withdrew: Withdrawal by subject | 0 | 0 | 1 | 0 | 0 |
| Withdrew: Perceived lack of efficacy | 0 | 0 | 1 | 0 | 0 |
| Withdrew: Ongoing | 0 | 0 | 1 | 5 | 4 |
| Milestone | Pre-treatment SOC Period (12 Weeks) | 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) |
|---|---|---|---|---|---|
| Started | 0 | 7 | 6 | 4 | 0 |
| Completed | 0 | 0 | 0 | 0 | 0 |
| Not completed | 0 | 7 | 6 | 4 | 0 |
| Withdrew: Perceived lack of efficacy | 0 | 1 | 1 | 0 | 0 |
| Withdrew: Ongoing | 0 | 6 | 5 | 4 | 0 |
AUC0-last is the area under the plasma concentration-time curve from time 0 to the time of the last measurable concentration.
| nanogram* hour per milliliter (ng*h/mL) | 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) |
|---|---|---|---|---|
| Area Under the Plasma Concentration-Time Curve From Time 0 to the Last Measurable Concentration (AUC0-last) of Berotralstat | 1182 ± 48.0 | 774 ± 34.9 | 869 ± 37.1 | — |
AUC0-6 is the area under the plasma concentration-time curve from time 0 to 6 hours.
| ng*h/mL | 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) |
|---|---|---|---|---|
| Area Under the Plasma Concentration-Time Curve From Time 0 to 6 Hours Post-dose (AUC0-6) of Berotralstat | 1605 ± 19.3 | 874 ± 44.9 | 911 ± 42.1 | — |
Ctrough is the concentration at the end of a dosing interval of berotralstat.
| Nanograms per milliliter (ng/mL) | 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) |
|---|---|---|---|---|
| Concentration at the End of the Dosing Interval (Ctrough) of Berotralstat | 133 ± 45.5 | 92.9 ± 25.8 | 86.8 ± 27.4 | 106 ± 38.1 |
Cmax is the maximum observed plasma concentration of berotralstat.
| ng/mL | 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) |
|---|---|---|---|---|
| Maximum Observed Plasma Concentration (Cmax) of Berotralstat | 271 ± 40.8 | 181 ± 28.1 | 180 ± 37.7 | — |
Tlast is the time of the last measurable concentration (Clast) of berotralstat collected over the sampling interval.
| hours | 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) |
|---|---|---|---|---|
| Time of Last Measurable Plasma Concentration (Tlast) of Berotralstat | 6.00 (5.08 to 6.13) | 5.09 (4.05 to 6.00) | 6.00 (5.00 to 6.08) | — |
Tmax is the time taken to reach the maximum observed plasma concentration of berotralstat.
| hours | 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) |
|---|---|---|---|---|
| Time to Maximum Plasma Concentration (Tmax) of Berotralstat | 4.05 (3.67 to 6.00) | 2.09 (1.00 to 5.95) | 2.07 (0.92 to 4.00) | — |
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.
| Participants | 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) |
|---|---|---|---|---|
| Number of Participants With Treatment Emergent Adverse Events (TEAEs) | 6 | 7 | 9 | 3 |
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.
Results for this outcome have not been posted.
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.
Results for this outcome have not been posted.
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).
Results for this outcome have not been posted.
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.
Results for this outcome have not been posted.
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.
Results for this outcome have not been posted.
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.
Results for this outcome have not been posted.
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.
Results for this outcome have not been posted.
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.
Results for this outcome have not been posted.
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.
Results for this outcome have not been posted.
The total number of participants who discontinued the treatment due to perceived lack of efficacy of berotralstat were reported.
Results for this outcome have not been posted.
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.
Results for this outcome have not been posted.
Collected over For SOC period: 12 weeks prior to first dose For AEs: From first dose of study treatment up to approximately 73 weeks. Non-serious events are listed at a 0% frequency threshold.
| Group | Deaths | Serious | Other |
|---|---|---|---|
| Pre-treatment SOC Period | 0/29 (0%) | 0/29 (0%) | 18/29 (62.1%) |
| Cohort 1: ≥ 40 kg Body Weight (Berotralstat 150 mg) | 0/7 (0%) | 1/7 (14.3%) | 6/7 (85.7%) |
| Cohort 2: 32 to < 40 kg Body Weight (Berotralstat 108 mg) | 0/9 (0%) | 0/9 (0%) | 7/9 (77.8%) |
| Cohort 3: 24 to < 32 kg Body Weight (Berotralstat 96 mg) | 0/9 (0%) | 0/9 (0%) | 9/9 (100%) |
| Cohort 4: 12 to <24 kg Body Weight (Berotralstat 78 mg) | 0/4 (0%) | 0/4 (0%) | 3/4 (75%) |
| Event | Pre-treatment SOC Period | 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) |
|---|---|---|---|---|---|
| AccidentInjury, poisoning and procedural complications | 0/29 | 1/7 | 0/9 | 0/9 | 0/4 |
| Upper limb fractureInjury, poisoning and procedural complications | 0/29 | 1/7 | 0/9 | 0/9 | 0/4 |
| Event | Pre-treatment SOC Period | 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) |
|---|---|---|---|---|---|
| NasopharyngitisInfections and infestations | 3/29 | 1/7 | 3/9 | 4/9 | 0/4 |
| CoughRespiratory, thoracic and mediastinal disorders | 1/29 | 0/7 | 0/9 | 3/9 | 0/4 |
| Upper respiratory tract infectionInfections and infestations | 2/29 | 2/7 | 2/9 | 2/9 | 1/4 |
| GastroenteritisInfections and infestations | 0/29 | 2/7 | 0/9 | 1/9 | 0/4 |
| Viral upper respiratory tract infectionInfections and infestations | 2/29 | 2/7 | 1/9 | 0/9 | 0/4 |
| Ear infectionInfections and infestations | 2/29 | 1/7 | 0/9 | 0/9 | 1/4 |
| Viral rhinitisInfections and infestations | 0/29 | 0/7 | 0/9 | 0/9 | 1/4 |
| Oropharyngeal painRespiratory, thoracic and mediastinal disorders | 1/29 | 0/7 | 0/9 | 1/9 | 1/4 |
| Ear painEar and labyrinth disorders | 0/29 | 1/7 | 0/9 | 0/9 | 1/4 |
| ContusionInjury, poisoning and procedural complications | 1/29 | 0/7 | 0/9 | 0/9 | 1/4 |
Safety population included all participants who received at least 1 dose of berotralstat.
| Age, Continuous(years) | 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) | Total |
|---|---|---|---|---|---|
| Mean | 9.9 ± 1.35 | 8.9 ± 1.45 | 8.2 ± 1.39 | 4.5 ± 1.29 | 8.3 ± 2.12 |
| Sex: Female, Male(Participants) | 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) | Total |
|---|---|---|---|---|---|
| Female | 3 | 6 | 5 | 0 | 14 |
| Male | 4 | 3 | 4 | 4 | 15 |
| Ethnicity (NIH/OMB)(Participants) | 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) | Total |
|---|---|---|---|---|---|
| Hispanic or Latino | 0 | 0 | 0 | 0 | 0 |
| Not Hispanic or Latino | 6 | 8 | 7 | 4 | 25 |
| Unknown or Not Reported | 1 | 1 | 2 | 0 | 4 |
| Race (NIH/OMB)(Participants) | 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) | Total |
|---|---|---|---|---|---|
| American Indian or Alaska Native | 0 | 0 | 0 | 0 | 0 |
| Asian | 0 | 0 | 0 | 0 | 0 |
| Native Hawaiian or Other Pacific Islander | 0 | 0 | 0 | 0 | 0 |
| Black or African American | 0 | 0 | 0 | 0 | 0 |
| White | 6 | 8 | 6 | 2 | 22 |
| More than one race | 0 | 0 | 0 | 0 | 0 |
| Unknown or Not Reported | 1 | 1 | 3 | 2 | 7 |
PDFs hosted by the registry, ClinicalTrials.gov.
Plan to share: No
Ran from Oct 2022 to primary completion in Sep 2024, 22 months.
Results were first posted in Feb 2026, 1 year 5 months after primary completion. A results delay was requested in Sep 2025.
Of 46 industry-sponsored phase 3 interventional studies under Angioedemas, Hereditary, half reached primary completion within 22 months of starting (the median), counting those still running; a quarter within 16 and three quarters within 33. This study's 22 months is close to that.
Counting every sponsor, half of 46 studies reached it within 22 months; a quarter within 16 and three quarters within 33.
How these figures are worked out, and which studies are left out of them.
This study is active, not recruiting, as verified in Feb 2026. You cannot join it, but the record below documents what was studied.
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Studies of the drug tested here: Berotralstat