Berotralstat for Pediatrics

Phase-Based Progress Estimates
2
Effectiveness
3
Safety
Investigative Site #1, Tel Aviv, Israel
Pediatrics+3 More
Berotralstat - Drug
Eligibility
< 18
All Sexes
What conditions do you have?
Select

Study Summary

The purpose of this study is to evaluate the pharmacokinetics (PK) and safety of berotralstat to determine the appropriate weight-based dose for pediatric participants 2 to < 12 years old for prophylactic treatment to prevent attacks of hereditary angioedema (HAE).

Eligible Conditions

  • Pediatrics
  • Hereditary Angioedema (HAE)

Treatment Effectiveness

Effectiveness Progress

2 of 3
This is further along than 85% of similar trials

Other trials for Pediatrics

Study Objectives

3 Primary · 2 Secondary · Reporting Duration: Over 144 weeks

Over 144 weeks
Frequency and severity of adverse events (AEs) and serious adverse events (SAEs)
Over 48 weeks
Frequency and severity of hereditary angioedema attacks (HAE) attacks
Hour 24
Pharmacokinetics: AUC0-tau
Pharmacokinetics: CL/F
Pharmacokinetics: Cmax
Day 1
Acceptability/palatability of berotralstat oral granules using a self-reported taste rating scale designed with images centered on taste

Trial Safety

Safety Progress

3 of 3
This is further along than 85% of similar trials

Other trials for Pediatrics

Trial Design

1 Treatment Group

Berotralstat
1 of 1
Experimental Treatment

20 Total Participants · 1 Treatment Group

Primary Treatment: Berotralstat · No Placebo Group · Phase 3

Berotralstat
Drug
Experimental Group · 1 Intervention: Berotralstat · Intervention Types: Drug

Trial Logistics

Trial Timeline

Approximate Timeline
Screening: ~3 weeks
Treatment: Varies
Reporting: over 144 weeks
Closest Location: Investigative Site #1 · Ottawa, Canada
Photo of Ottawa  1Photo of Ottawa  2Photo of Ottawa  3
2022First Recorded Clinical Trial
1 TrialsResearching Pediatrics
1 CompletedClinical Trials

Eligibility Criteria

Age < 18 · All Participants · 4 Total Inclusion Criteria

Mark “yes” if the following statements are true for you:
You are male and non-pregnant, non-lactating females aged 2 to < 12 years.
You have a clinical diagnosis of HAE.
The participant would benefit from long term oral HAE prophylaxis.

About The Reviewer

Michael Gill preview

Michael Gill - B. Sc.

First Published: October 9th, 2021

Last Reviewed: August 12th, 2022

Michael Gill holds a Bachelors of Science in Integrated Science and Mathematics from McMaster University. During his degree he devoted considerable time modeling the pharmacodynamics of promising drug candidates. Since then, he has leveraged this knowledge of the investigational new drug ecosystem to help his father navigate clinical trials for multiple myeloma, an experience which prompted him to co-found Power Life Sciences: a company that helps patients access randomized controlled trials.