Pediatric Clinical Trial
— APeX-POfficial title:
A Phase 3 Study to Evaluate the Safety and Pharmacokinetics of Berotralstat Prophylaxis in Children With Hereditary Angioedema Who Are 2 to < 12 Years of Age
Verified date | March 2024 |
Source | BioCryst Pharmaceuticals |
Contact | n/a |
Is FDA regulated | No |
Health authority | |
Study type | Interventional |
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).
Status | Active, not recruiting |
Enrollment | 29 |
Est. completion date | July 2027 |
Est. primary completion date | July 2025 |
Accepts healthy volunteers | No |
Gender | All |
Age group | 2 Years to 11 Years |
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 Exclusion Criteria: - Concurrent diagnosis of any other type of recurrent angioedema - Known family history of sudden cardiac death - Creatinine clearance using the modified Schwartz formula of = 30 mL/min/1.73 m2 - Aspartate aminotransferase or alanine aminotransferase value = 3 × the upper limit of the age-appropriate normal reference range value - Clinically significant abnormal ECG including but not limited to, a corrected QT interval calculated 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 |
Country | Name | City | State |
---|---|---|---|
Austria | Investigative Site #1 | Vienna | |
Canada | Investigative Site #1 | Ottawa | Ontario |
France | Investigative Site #3 | Grenoble | |
France | Investigative Site #2 | Marseille | |
France | Investigative Site #1 | Paris | |
Germany | Investigative Site #1 | Berlin | |
Germany | Investigative Site #2 | Frankfurt | |
Israel | Investigative Site #2 | Haifa | |
Israel | Investigative Site #1 | Tel Aviv | |
Italy | Investigative Site #1 | Padova | |
Poland | Investigative Site #1 | Kraków | |
Romania | Investigative Site #1 | Sângeorgiu De Mures | |
Spain | Investigative Site #1 | Madrid | |
Spain | Investigative Site #2 | Málaga | |
United Kingdom | Investigative Site #1 | Bristol |
Lead Sponsor | Collaborator |
---|---|
BioCryst Pharmaceuticals |
Austria, Canada, France, Germany, Israel, Italy, Poland, Romania, Spain, United Kingdom,
Type | Measure | Description | Time frame | Safety issue |
---|---|---|---|---|
Other | Acceptability/palatability of berotralstat oral granules using a self-reported taste rating scale designed with images centered on taste | TASTY; 7-point scale [0 "worst" to 6 "best"] | Time of first dose (Day 1) | |
Primary | Pharmacokinetics: Cmax | Maximum plasma concentration of berotralstat | Predose and multiple timepoints up to 24 hours postdose | |
Primary | Pharmacokinetics: AUC0-tau | Area under the plasma concentration berotralstat time curve from time zero to the end of dosing (tau) | Predose and multiple timepoints up to 24 hours postdose | |
Primary | Pharmacokinetics: CL/F | Apparent oral clearance of berotralstat | Predose and multiple timepoints up to 24 hours postdose | |
Secondary | Frequency and severity of adverse events (AEs) and serious adverse events (SAEs) | Over 144 weeks | ||
Secondary | Frequency and severity of hereditary angioedema (HAE) attacks | Over 48 weeks |
Status | Clinical Trial | Phase | |
---|---|---|---|
Completed |
NCT04176822 -
Designing Animated Movie for Preoperative Period
|
N/A | |
Completed |
NCT01981954 -
A Clinical Study to Investigate How Solifenacin Fluid is Taken up, How Long it Stays in the Body and How Effective and Safe it is in Treating Children Aged From 6 Months to Less Than 5 Years With Symptoms of Neurogenic Detrusor Overactivity (NDO)
|
Phase 3 | |
Completed |
NCT02199626 -
SECOND GENERATION COLON CAPSULE ENDOSCOPY (CCE-2) IN PEDIATRIC CROHN'S DISEASE (CD).
|
Phase 4 | |
Recruiting |
NCT04679792 -
Postual Control in Pediatric Chiari I Malformation
|
||
Recruiting |
NCT06061159 -
The ED95 of Single Intravenous Bolus Remimazolam Besylate in Pediatric Patients During General Anesthesia Induction
|
Phase 4 | |
Not yet recruiting |
NCT02182648 -
Bispectral Index Guiding Etomidate Used in Children Aged 3 to 10 for Clinical Anesthesia Induction
|
Phase 4 | |
Active, not recruiting |
NCT04176640 -
Quantifying Brain Injury on Computed Tomography in Hospitalized Children
|
||
Active, not recruiting |
NCT06146452 -
Micronutrient Status in Pediatric Palliative Care
|
||
Completed |
NCT04041180 -
Impact on the Length of Stay in Incentive Spirometry and Pain in the Decompensation of Sickle Cell Disease: .
|
||
Not yet recruiting |
NCT06165003 -
Examination of Nurses' Use of Broselow Tape in Non-resuscitation Pediatric Emergency Department Practices
|
N/A | |
Recruiting |
NCT02286297 -
Pediatric Endotracheal Intubation During Resuscitation
|
N/A | |
Unknown status |
NCT00613184 -
Comparison of Nylon Flocked Swabs and Saline Aspirates for Detection Respiratory Viruses
|
N/A | |
Recruiting |
NCT00265772 -
Comparison of a Nutritional Anti-Inflammatory Treatment to Steroids for Pediatric Crohn's Disease - the Molecular Basis
|
Phase 4 | |
Completed |
NCT04106063 -
A New Speech, Spatial, and Qualities of Hearing Scale Short-Form for Deaf Children
|
||
Not yet recruiting |
NCT06376643 -
Augmented Reality to Support Cardiopulmonary Resuscitation
|
N/A | |
Not yet recruiting |
NCT05511233 -
ERAS Protocol in Newborns: CARES Study
|
N/A | |
Not yet recruiting |
NCT04127968 -
Efficacy and Safety of Vitamin A Treatment for Children With Sepsis
|
N/A | |
Active, not recruiting |
NCT04472286 -
Healthy Bones, Healthy Life: Habitual Physical Activity on Bone & Metabolic Health in Pediatric Cancer Survivors
|
||
Recruiting |
NCT04988516 -
Virtual Reality Distraction for Anxiety (VR-IMAGINE)
|
N/A | |
Recruiting |
NCT05909878 -
Virtual Reality Distraction for Anxiety (VR-IMAGINE at HSJ)
|
N/A |