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Plasma Kallikrein Inhibitor

Berotralstat for Pediatric Hereditary Angioedema (APeX-P Trial)

Phase 3
Waitlist Available
Led By Matthew Buckland, MBBS, PhD, FRCP
Research Sponsored by BioCryst Pharmaceuticals
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial
Must have
Male and non-pregnant, non-lactating females 2 to < 12 years of age
Body weight ≥ 12 kg
Must not have
Concurrent diagnosis of any other type of recurrent angioedema
Known family history of sudden cardiac death
Timeline
Screening 3 weeks
Treatment Varies
Follow Up predose and multiple timepoints up to 24 hours postdose
Awards & highlights
All Individual Drugs Already Approved
No Placebo-Only Group
Pivotal Trial

Summary

This trial is testing a new drug to see if it's safe and effective in treating hereditary angioedema in children.

Who is the study for?
This trial is for children aged 2 to less than 12 with hereditary angioedema (HAE), weighing at least 12 kg, who could benefit from daily oral medication to prevent HAE attacks. It's not for kids with other types of angioedema, a family history of sudden heart death, certain liver issues, abnormal ECGs, or poor kidney function.
What is being tested?
The study tests berotralstat in young patients to find the right dose based on their weight and assess its safety. The goal is to see if this drug can help prevent swelling attacks caused by HAE in these children.
What are the potential side effects?
While specific side effects for pediatric use are being studied, berotralstat may cause stomach pain, digestive issues like diarrhea or nausea, dizziness and rash in adults. Children might experience similar side effects.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below
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I am a boy or a girl not pregnant or breastfeeding, aged 2 to under 12.
Select...
My body weight is at least 12 kg.
Select...
I have been diagnosed with Hereditary Angioedema.

Exclusion Criteria

You may be eligible for the trial if you check “No” for criteria below:
Select...
I have been diagnosed with another type of recurring swelling attacks.
Select...
A family member has died suddenly from heart issues.
Select...
My heart's electrical activity is normal, with no significant irregularities.

Timeline

Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~predose and multiple timepoints up to 24 hours postdose
This trial's timeline: 3 weeks for screening, Varies for treatment, and predose and multiple timepoints up to 24 hours postdose for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.
Primary study objectives
Pharmacokinetics: AUC0-tau
Pharmacokinetics: CL/F
Pharmacokinetics: Cmax
Other study objectives
Acceptability/palatability of berotralstat oral granules using a self-reported taste rating scale designed with images centered on taste

Side effects data

From 2022 Phase 2 & 3 trial • 387 Patients • NCT03472040
33%
Nasopharyngitis
23%
Headache
14%
Diarrhoea
14%
Abdominal pain
14%
Upper respiratory tract infection
11%
Hereditary angioedema
10%
Gastroenteritis
10%
Oropharyngeal pain
10%
Abdominal pain upper
10%
COVID-19
10%
Sinusitis
9%
Nausea
8%
Gastrooesophageal reflux disease
8%
Influenza
7%
Urinary tract infection
6%
Abdominal discomfort
5%
Dyspepsia
3%
Vomiting
2%
Abortion spontaneous
2%
Medical observation
2%
Pneumonia
2%
Enteritis
1%
Radius fracture
1%
Myocardial infarction
1%
Lower limb fracture
1%
Hepatic enzyme increased
1%
Asthma
1%
Facial paralysis
1%
Anal abscess
1%
Gastroenteritis viral
1%
Gingivitis
100%
80%
60%
40%
20%
0%
Study treatment Arm
110 mg Followed by 150 mg Berotralstat
150 mg Berotralstat

Awards & Highlights

All Individual Drugs Already Approved
Therapies where all constituent drugs have already been approved are likely to have better-understood side effect profiles.
No Placebo-Only Group
All patients enrolled in this study will receive some form of active treatment.
Pivotal Trial
The final step before approval, pivotal trials feature drugs that have already shown basic safety & efficacy.

Trial Design

1Treatment groups
Experimental Treatment
Group I: BerotralstatExperimental Treatment1 Intervention
Berotralstat administered once daily in 4 dose cohorts determined by participant weight. Cohorts 1 and 2 will enroll in parallel. After 4 participants from Cohort 1 and 2, with ≥ 2 subjects from Cohort 2, have reached Week 2, Cohort 3 will open for enrollment. Cohort 4 will open for enrollment, after ≥ 4 subjects in Cohort 3 have reached Week 2. Prior to dosing Cohort 3 and 4, available PK and safety data will be reviewed to confirm it is safe to proceed and the appropriate weight bands for each. BioCryst will notify sites when Cohorts 3 and 4 are open for enrollment.

Find a Location

Who is running the clinical trial?

BioCryst PharmaceuticalsLead Sponsor
54 Previous Clinical Trials
4,342 Total Patients Enrolled
Matthew Buckland, MBBS, PhD, FRCPPrincipal InvestigatorGreat Ormond St Hospital for Children NHS Foundation Trust
Jolanta Bernatoniene, MDPrincipal InvestigatorBristol Royal Hospital for Children

Media Library

Berotralstat (Plasma Kallikrein Inhibitor) Clinical Trial Eligibility Overview. Trial Name: NCT05453968 — Phase 3
Hereditary Angioedema Research Study Groups: Berotralstat
Hereditary Angioedema Clinical Trial 2023: Berotralstat Highlights & Side Effects. Trial Name: NCT05453968 — Phase 3
Berotralstat (Plasma Kallikrein Inhibitor) 2023 Treatment Timeline for Medical Study. Trial Name: NCT05453968 — Phase 3
~9 spots leftby Dec 2025
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