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Plasma Kallikrein Inhibitor

Berotralstat for Pediatric Hereditary Angioedema (APeX-P Trial)

Q: Has Berotralstat undergone the necessary drug testing to be cleared for public use?

There is some evidence from previous clinical trials to support the efficacy of Berotralstat as well as data suggesting it is safe for use. Therefore, we have rated it a 3 on our Power scale.

Q: How many people are being asked to participate in this experiment?

That is correct. The clinical trial, which was posted on October 1st, 2020 and last updated on October 21st, 2020, is still recruiting patients. They are looking for a total of 20 people across 1 site.

Q: Are investigators still signing up new participants for this research project?

That is correct. The clinical trial, which is currently looking for 20 patients from 1 site, was posted on October 1st, 2020 and last updated 21 days ago on October 21st, 2020 according to the data available on clinicaltrials.gov

Q: Could I be a candidate for this research project?

Up to 20 children aged 2-11 can participate in this study. To be eligible, they must meet the following requirements: Be male or non-pregnant/non-lactating female, weigh more than 12 kg, have a diagnosis of HAE from a doctor, and be someone that would benefit from long term prophylaxis according to the investigator's opinion.

Q: Are people of all ages being considered for this clinical trial or are only young adults eligible?

This clinical trial is only for patients aged 2-11. There are a total of 33 studies for children and 19 for seniors.

Phase 3

Waitlist Available

Led By Jolanta Bernatoniene, MD

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

Timeline

Screening 3 weeks

Treatment Varies

Follow Up over 144 weeks

Awards & highlights

APeX-P 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.Check my eligibility

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.See study design

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.

APeX-P Trial Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below

Select...

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.

APeX-P Trial Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ over 144 weeks

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~over 144 weeks

This trial's timeline: 3 weeks for screening, Varies for treatment, and over 144 weeks for reporting.

Treatment Details

Study Objectives

Outcome measures can provide a clearer picture of what you can expect from a treatment.

Primary outcome measures

Pharmacokinetics: AUC0-tau

Pharmacokinetics: CL/F

Pharmacokinetics: Cmax

Secondary outcome measures

Frequency and severity of adverse events (AEs) and serious adverse events (SAEs)

Frequency and severity of hereditary angioedema (HAE) attacks

Other outcome measures

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%

Upper respiratory tract infection

14%

Abdominal pain

14%

Diarrhoea

11%

Hereditary angioedema

10%

Gastroenteritis

10%

Oropharyngeal pain

10%

Abdominal pain upper

10%

COVID-19

10%

Sinusitis

Nausea

Gastrooesophageal reflux disease

Influenza

Urinary tract infection

Abdominal discomfort

Dyspepsia

Vomiting

Abortion spontaneous

Medical observation

Enteritis

Pneumonia

Facial paralysis

Anal abscess

Myocardial infarction

Hepatic enzyme increased

Lower limb fracture

Radius fracture

Gastroenteritis viral

Gingivitis

Asthma

100%

80%

60%

40%

20%

Study treatment Arm

110 mg Followed by 150 mg Berotralstat

150 mg Berotralstat

APeX-P 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.

0 / 3Eligibility criteria met

Find a Location

Who is running the clinical trial?

BioCryst PharmaceuticalsLead Sponsor

53 Previous Clinical Trials

4,458 Total Patients Enrolled

Jolanta Bernatoniene, MDPrincipal InvestigatorBristol Royal Hospital for Children

Matthew Buckland, MBBS, PhD, FRCPPrincipal InvestigatorGreat Ormond St Hospital for Children NHS Foundation Trust

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

Frequently Asked Questions

These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.

Has Berotralstat undergone the necessary drug testing to be cleared for public use?

"There is some evidence from previous clinical trials to support the efficacy of Berotralstat as well as data suggesting it is safe for use. Therefore, we have rated it a 3 on our Power scale."

Answered by AI

How many people are being asked to participate in this experiment?

"That is correct. The clinical trial, which was posted on October 1st, 2020 and last updated on October 21st, 2020, is still recruiting patients. They are looking for a total of 20 people across 1 site."

Answered by AI

Are investigators still signing up new participants for this research project?

"That is correct. The clinical trial, which is currently looking for 20 patients from 1 site, was posted on October 1st, 2020 and last updated 21 days ago on October 21st, 2020 according to the data available on clinicaltrials.gov"

Answered by AI

Could I be a candidate for this research project?

"Up to 20 children aged 2-11 can participate in this study. To be eligible, they must meet the following requirements: Be male or non-pregnant/non-lactating female, weigh more than 12 kg, have a diagnosis of HAE from a doctor, and be someone that would benefit from long term prophylaxis according to the investigator's opinion."

Answered by AI