Clinical Trials in Poland / NCT05453968
Active, not recruiting
Phase 3
Berotralstat Treatment in Children With Hereditary Angioedema
NCT05453968 · tracked via the Priya Life Science Poland tracker
Condition(s) studied
Hereditary AngioedemaPediatric
Investigational drug(s) / intervention(s)
Berotralstat: Administered orally once daily
Study 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).
Eligibility
Inclusion Criteria:
* 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.
Exclusion Criteria:
* 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
Primary outcome measure(s)
- Area Under the Plasma Concentration-Time Curve From Time 0 to the Last Measurable Concentration (AUC0-last) of Berotralstat — 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 — 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 — Predose at Week 2
Ctrough is the concentration at the end of a dosing interval of berotralstat. - Maximum Observed Plasma Concentration (Cmax) of Berotralstat — 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 — 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 — 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.
Trial sites (15)
| Facility | City | Region | Status |
|---|---|---|---|
| Investigative Site #1 | Vienna | Ontario | |
| Investigative Site #1 | Ottawa | Ontario | |
| Investigative Site #3 | Grenoble | France | |
| Investigative Site #2 | Marseille | France | |
| Investigative Site #1 | Paris | France | |
| Investigative Site #1 | Berlin | Germany | |
| Investigative Site #2 | Frankfurt | Germany | |
| Investigative Site #2 | Haifa | Israel | |
| Investigative Site #1 | Tel Aviv | Israel | |
| Investigative Site #1 | Padova | Italy | |
| Investigative Site #1 | Krakow | Poland | |
| Investigative Site #1 | Sângeorgiu de Mureş | Romania | |
| Investigative Site #1 | Madrid | Spain | |
| Investigative Site #2 | Málaga | Spain | |
| Investigative Site #1 | Bristol | United Kingdom |
More BioCryst Pharmaceuticals trials in Poland
Open-label Berotralstat Access to HAE Patients Previously Enrolled in Berotralstat Studies
Enrolling by invitation
Other trials for the same condition
Open-label Berotralstat Access to HAE Patients Previously Enrolled in Berotralstat Studies
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Extension Study of Oral PHA-022121 for Acute Treatment of Angioedema Attacks in Patients With Hereditary Angioedema
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STOP-HAE: A Phase 3 Study of ADX-324 in HAE
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A Study to Assess the Long-Term Safety and Efficacy of Donidalorsen in the Prophylactic Treatment of Hereditary Angioedema (HAE)
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A Long-term Study of STAR-0215 in Participants With Hereditary Angioedema
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Official registry record
This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.
View NCT05453968 on ClinicalTrials.gov ↗ ← All trials in Poland