Clinical trial • Phase III • Haematology | Rare Disease
BEROTRALSTAT for Hereditary angioedema
Phase III trial of BEROTRALSTAT for Hereditary angioedema. open-label. 79 participants.
Overview
- Trial Therapeutic Area
- Haematology | Rare Disease
- Trial Disease
- Hereditary angioedema
- Trial Stage
- Phase III
- Drug Modality
- Small molecule
- Paediatric Trial
- Yes
Key dates
- Initial CTIS Submission Date
- 16-05-2024
- First CTIS Authorization Date
- 17-07-2024
Trial design
open-label Phase III trial in Czechia, Poland, Slovakia and others.
- Open Label
- Yes
- Target Sample Size
- 79
Eligibility
Recruits 79 paediatric patients.
- Pregnancy Exclusion
- Pregnancy or breastfeeding
- Vulnerable Population
- Participant or parent/ legally designated representative (for participants < 18 years of age) able to provide written informed consent. For participants < 18 years of age, based on the child’s age and local regulatory requirements, participant assent will be collected as appropriate.
Inclusion criteria
- {"criterion_text":"- Males and non-pregnant, non-lactating females who are currently enrolled as a subject or were previously enrolled and did not discontinue due to an adverse event or due to noncompliance in BioCryst-sponsored Study 302, 204, or 304."}
- {"criterion_text":"- Participant or parent/ legally designated representative (for participants < 18 years of age) able to provide written informed consent. For participants < 18 years of age, based on the child’s age and local regulatory requirements, participant assent will be collected as appropriate."}
- {"criterion_text":"- Would benefit from continued berotralstat treatment in the opinion of the investigator"}
- {"criterion_text":"- Female subjects must meet at least 1 of the following requirements: a. Be a WOCBP (defined as a female following menarche and prior to becoming post-menopausal who has not had a hysterectomy or bilateral salpingectomy and bilateral oophorectomy) who agrees to use at least an acceptable effective contraceptive method during the study. b. Be a woman of nonchildbearing potential (defined as postmenopausal [no menses for at least 12 months without an alternative medical cause] or has had a hysterectomy or a bilateral salpingectomy and bilateral oophorectomy)."}
- {"criterion_text":"- In the opinion of the investigator, the subject is expected to adequately comply with all required study procedures"}
Exclusion criteria
- {"criterion_text":"- Any condition or situation, including medical history or changes in medical history, which, in the opinion of the investigator or sponsor, would interfere with the subject's safety or ability to participate in the study"}
- {"criterion_text":"- Dementia, altered mental status, or any psychiatric condition that would prohibit the understanding or rendering of informed consent."}
- {"criterion_text":"- Hypersensitivity to the active substance or to any of the excipients in the IMP"}
- {"criterion_text":"- Use of other medications for long-term prophylaxis of HAE attacks at the Baseline visit or any time during the study. These include: C1-INH, tranexamic acid, androgens, or lanadelumab"}
- {"criterion_text":"- Use of any other investigational medicinal product at the Baseline visit or any time during the study."}
- {"criterion_text":"- Pregnancy or breastfeeding"}
- {"criterion_text":"- Subjects with an immediate family relationship to either sponsor employees, the investigator, or employees of the study site who are named on the delegation log."}
- {"criterion_text":"- Subjects who are held in an institution by a governmental or judicial order."}
Endpoints
Primary endpoints
- {"endpoint_text":"- Number and proportion of subjects with a treatment-related TEAE\n- Number and proportion of subjects who experience a serious adverse event (SAE)\n- Number and proportion of subjects who experience a treatment-related Grade 3 or 4 TEAE\n- Number and proportion of subjects who experience a treatment-related Grade 3 or 4 treatment-emergent chemistry abnormality\n- Number and proportion of subjects who discontinue due to a TEAE","definition_or_measurement_approach":"Primary endpoints are safety outcome counts and proportions (number and proportion of subjects meeting each listed safety outcome as recorded during study treatment/safety reporting)."}
Secondary endpoints
- {"endpoint_text":"- Another mechanism is available to provide drug to the subject(eg, market access), or until the sponsor discontinues all global commercialization and development of berotralstat for the prevention of angioedema attacks, whichever comes first","definition_or_measurement_approach":"Time/condition-based endpoint: study treatment continued until an alternative access mechanism is available or until sponsor discontinues global development/commercialization for HAE prevention, whichever occurs first."}
Recruitment
- Planned Sample Size
- 79
- Recruitment Window Months
- 124
- Consent Approach
- Participant or parent/ legally designated representative (for participants < 18 years of age) able to provide written informed consent. For participants < 18 years of age, based on the child’s age and local regulatory requirements, participant assent will be collected as appropriate. Country- and age-specific subject information and informed consent/assent documents are provided (examples in the submitted documents include Czech, Slovak, Polish, Spanish, French, Italian and English versions and specific adolescent/assent ICFs).
Geography
- Total Number Of Sites
- 9
- Total Number Of Participants
- 45
Czechia
- Earliest CTIS Part Ii Submission Date
- 17-07-2024
- Latest Decision Or Authorization Date
- 05-06-2025
- Processing Time Days
- 323
- Number Of Sites
- 2
- Number Of Participants
- 6
Sites
- Site Name
- Fakultni Nemocnice U Sv Anny V Brne
- Department Name
- Ustav klinicke imunologie a alergologie
- Principal Investigator Name
- Roman Hakl
- Principal Investigator Email
- roman.hakl@fnusa.cz
- Contact Person Name
- Roman Hakl
- Contact Person Email
- roman.hakl@fnusa.cz
- Site Name
- Fakultni Nemocnice Plzen
- Department Name
- Oddělení klinické Alergologie a Imunologie
- Principal Investigator Name
- Martina Vachová
- Principal Investigator Email
- vachovam@fnplzen.cz
- Contact Person Name
- Martina Vachová
- Contact Person Email
- vachovam@fnplzen.cz
Poland
- Earliest CTIS Part Ii Submission Date
- 17-07-2024
- Latest Decision Or Authorization Date
- 18-03-2026
- Processing Time Days
- 609
- Number Of Sites
- 1
- Number Of Participants
- 13
Sites
- Site Name
- Samodzielny Publiczny Zaklad Opieki Zdrowotnej Szpital Uniwersytecki W Krakowie
- Department Name
- Centrum alergologii
- Principal Investigator Name
- Marcin Stobiecki
- Principal Investigator Email
- marcin.stobiecki@uj.edu.pl
- Contact Person Name
- Marcin Stobiecki
- Contact Person Email
- marcin.stobiecki@uj.edu.pl
Slovakia
- Earliest CTIS Part Ii Submission Date
- 19-07-2024
- Latest Decision Or Authorization Date
- 10-06-2025
- Processing Time Days
- 326
- Number Of Sites
- 1
- Number Of Participants
- 12
Sites
- Site Name
- Univerzitna Nemocnica Martin
- Department Name
- Centrum pre hereditarny angioedem
- Principal Investigator Name
- Milos Jesenak
- Principal Investigator Email
- sekrlps@unm.sk
- Contact Person Name
- Milos Jesenak
- Contact Person Email
- sekrlps@unm.sk
Spain
- Earliest CTIS Part Ii Submission Date
- 16-09-2025
- Latest Decision Or Authorization Date
- 16-09-2025
- Number Of Sites
- 1
- Number Of Participants
- 1
Sites
- Site Name
- Hospital Universitario La Paz
- Department Name
- Allergy (Paediatric)
- Principal Investigator Name
- Elsa Phillips
- Principal Investigator Email
- elsa.phillips@salud.madrid.org
- Contact Person Name
- Elsa Phillips
- Contact Person Email
- elsa.phillips@salud.madrid.org
Italy
- Earliest CTIS Part Ii Submission Date
- 22-10-2025
- Latest Decision Or Authorization Date
- 18-03-2026
- Processing Time Days
- 147
- Number Of Sites
- 1
- Number Of Participants
- 3
Sites
- Site Name
- Azienda Ospedaliera di Padova
- Department Name
- UOSD Allergologia
- Principal Investigator Name
- Riccardo Senter
- Principal Investigator Email
- riccardo.senter@aopd.veneto.it
- Contact Person Name
- Riccardo Senter
- Contact Person Email
- riccardo.senter@aopd.veneto.it
Germany
- Earliest CTIS Part Ii Submission Date
- 01-09-2025
- Latest Decision Or Authorization Date
- 18-03-2026
- Processing Time Days
- 198
- Number Of Sites
- 1
- Number Of Participants
- 3
Sites
- Site Name
- Goethe University Frankfurt
- Department Name
- Interdisciplinary Comprehensive Care Centre for Hereditary Angioedema
- Principal Investigator Name
- Emel Aygören-Pürsün
- Principal Investigator Email
- aygoeren@em.uni-frankfurt.de
- Contact Person Name
- Emel Aygören-Pürsün
- Contact Person Email
- aygoeren@em.uni-frankfurt.de
France
- Earliest CTIS Part Ii Submission Date
- 04-09-2025
- Latest Decision Or Authorization Date
- 18-03-2026
- Processing Time Days
- 195
- Number Of Sites
- 2
- Number Of Participants
- 7
Sites
- Site Name
- Trousseau Hospital
- Department Name
- Allergology
- Principal Investigator Name
- Melisande Bourgoin-heck
- Principal Investigator Email
- elisande.bourgoing-heck@aphp.fr
- Contact Person Name
- Melisande Bourgoin-heck
- Contact Person Email
- elisande.bourgoing-heck@aphp.fr
- Site Name
- Centre Hospitalier Universitaire Grenoble Alpes
- Department Name
- Paediatric immuno-haemato-oncology
- Principal Investigator Name
- Anne Pagnier
- Principal Investigator Email
- APagnier@chu-grenoble.fr
- Contact Person Name
- Anne Pagnier
- Contact Person Email
- APagnier@chu-grenoble.fr
Sponsor
Primary sponsor
- Full Name
- Biocryst Pharmaceuticals Inc.
- Organisation Type
- Pharmaceutical company
- Country Of Registered Address
- United States
Contract research organisations
- Name
- Pharpoint Research Inc.
- Responsibilities
- Sponsor duties codes: [10, 6, 7]
- Name
- AMS Advanced Medical Services Limited
- Responsibilities
- Sponsor duties codes: [1, 12]
Third parties
- {"country":"United States","full_name":"Pharpoint Research Inc.","duties_or_roles":"sponsorDuties codes: [10, 6, 7] (as recorded in CTIS third party entry)","organisation_type":"Pharmaceutical company"}
- {"country":"United Kingdom","full_name":"AMS Advanced Medical Services Limited","duties_or_roles":"sponsorDuties codes: [1, 12] (as recorded in CTIS third party entry)","organisation_type":"Pharmaceutical company"}
Investigational products
- Investigational Product Name
- Berotralstat / BEROTRALSTAT
- Active Substance
- BEROTRALSTAT
- Modality
- Small molecule
- Routes Of Administration
- Oral
- Route
- Oral
- Authorisation Status
- Investigational medicinal product (IMP); MIA number reported for IMP11566/00001 (as recorded)
- Dose Levels
- Reported max daily dose amounts in submission: 150 mg; 108 mg; 96 mg; 78 mg; 66 mg
- Maximum Dose
- 150 mg
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