Clinical trial • Phase III • Immunology|Rare Disease
NAVENIBART for Hereditary angioedema|Hereditary angioedema type I|Hereditary angioedema type II
Phase III trial of NAVENIBART for Hereditary angioedema|Hereditary angioedema type I|Hereditary angioedema type II.
Overview
- Trial Therapeutic Area
- Immunology|Rare Disease
- Trial Disease
- Hereditary angioedema|Hereditary angioedema type I|Hereditary angioedema type II
- Trial Stage
- Phase III
- Drug Modality
- Monoclonal antibody
- Paediatric Trial
- Yes
- Orphan Drug
- Yes
Key dates
- Initial CTIS Submission Date
- 23-04-2025
- First CTIS Authorization Date
- 18-08-2025
Trial design
Randomised, navenibart - matching placebo (matching placebo arm). dose and schedule not specified in the ctis metadata.-controlled Phase III trial in Germany, Portugal, Austria and others.
- Randomised
- Yes
- Comparator
- Navenibart - matching placebo (matching placebo arm). Dose and schedule not specified in the CTIS metadata.
- Target Sample Size
- 93
Eligibility
Recruits 93 paediatric patients.
- Vulnerable Population
- Vulnerable population selected: adolescents are included. Assent and parent/guardian consent materials are provided (Assent forms, Parent ICF, Adolescent ICF, Young Adult ICF present in the application documents).
Inclusion criteria
- {"criterion_text":"- Documented diagnosis of HAE (Type 1 or 2). The following must be met: a.\tDocumented clinical history consistent with HAE b.\tLab findings consistent with HAE Type 1 or 2"}
- {"criterion_text":"- Experienced at least 2 HAE attacks during the Run-In period, as confirmed by an investigator based on meeting the protocol-specified definition of an HAE attack."}
Exclusion criteria
- {"criterion_text":"- Any concomitant diagnosis of another form of chronic angioedema, such as acquired C1 inhibitor deficiency, HAE with normal C1-INH (also known as HAE type 3), idiopathic angioedema, or angioedema associated with urticaria"}
- {"criterion_text":"- Use of therapies prescribed for the prevention of HAE attacks may not be used during the trial or within the below time frames prior to the Run-In Period (adult participants may be on these medications at the time of the Screening Visit, but will need to washout prior to entering the Run-In Period): a.\tLanadelumab within 70 days prior to Run-In b.\tBerotralstat within 21 days prior to Run-In c.\tGaradacimab within 90 days prior to Run-In d. Plasma-derived C1INH for LTP within 14 days prior to Run-In e.\tTranexamic acid, oral danazol, oral stanazolol, and oral oxandrolone within 3 days prior to Run-In f.\tAll other prophylactic therapies, including investigational drugs, require consultation with the Medical Monitor"}
- {"criterion_text":"- Any exposure to angiotensin-converting enzyme inhibitors or any estrogen containing medications with systemic absorption (such as hormonal contraceptives or hormone replacement therapy) within 30 days before Screening."}
Endpoints
Primary endpoints
- {"endpoint_text":"- Number of time-normalized investigator-confirmed HAE attacks during the 6-month Treatment Period.","definition_or_measurement_approach":"Count of time-normalized investigator-confirmed HAE attacks occurring during the 6-month Treatment Period (investigator confirmation as per protocol definition)."}
Secondary endpoints
- {"endpoint_text":"- Number of moderate or severe investigator-confirmed HAE attacks during the 6-month Treatment Period.","definition_or_measurement_approach":"Count of investigator-confirmed HAE attacks classified as moderate or severe during the 6-month Treatment Period."}
- {"endpoint_text":"- Number of investigator-confirmed HAE attacks that require on-demand treatment during the 6-month Treatment Period.","definition_or_measurement_approach":"Count of investigator-confirmed HAE attacks that required on-demand (acute) treatment during the 6-month Treatment Period."}
- {"endpoint_text":"- Percent reduction in monthly investigator-confirmed HAE attacks in the 6-month Treatment Period versus the Run-In Period.","definition_or_measurement_approach":"Percent change in monthly investigator-confirmed HAE attack rate comparing the 6-month Treatment Period to the Run-In Period."}
- {"endpoint_text":"- Time to first investigator-confirmed HAE attack after first and second dose","definition_or_measurement_approach":"Time-to-event (time from dosing) until first investigator-confirmed HAE attack after first and after second dose."}
- {"endpoint_text":"- The number of participants responding to treatment, defined as a ≥ 50%, ≥ 70%, or ≥ 90% reduction from the Run-In Period in investigator‑confirmed HAE attack rate (for adult participants: compared to placebo during the 6-month Treatment Period).","definition_or_measurement_approach":"Responder analysis defined by ≥50%, ≥70%, or ≥90% reduction in investigator-confirmed HAE attack rate from Run-In Period; for adults compared to placebo during 6-month Treatment Period."}
- {"endpoint_text":"- Number of participants with no investigator-confirmed HAE attacks during the 6-month Treatment Period","definition_or_measurement_approach":"Count of participants with zero investigator-confirmed HAE attacks during the 6-month Treatment Period."}
- {"endpoint_text":"- Change from baseline (Day 1) in the Angioedema Quality of Life questionnaire total score.","definition_or_measurement_approach":"Change from baseline (Day 1) in total score of the Angioedema Quality of Life questionnaire."}
- {"endpoint_text":"- Other Pre-specified Outcome Measures: Incidence of treatment‑emergent adverse events.","definition_or_measurement_approach":"Incidence (count and proportion) of treatment-emergent adverse events as pre-specified safety outcome measures."}
Recruitment
- Digital Remote Recruitment
- Yes
- Planned Sample Size
- 93
- Recruitment Window Months
- 19
- Consent Approach
- Informed consent obtained from adult participants. For minors/adolescents, parent/guardian (parent) consent and subject assent forms are provided (Assent, Parent ICF, Adolescent ICF, Young Adult ICF present). Multiple language versions and country-specific ICFs/synopses are included for participating Member States (documents present for DE, FR, IT, NL, PT, PL, HU, BG, CZ, ES and English versions).
Methods
- Patient recruitment website (Patient Recruitment Website) – online website to provide study information to potential participants.
- Display Ads (Display Ads / Display Ads Cover Sheet) – paid display advertising identified in recruitment materials.
- Paid Social Ads (Paid Social Ads; Paid Social_Cover Sheet) – social media paid advertising (e.g., Facebook/Instagram) used for recruitment.
- EU Search Ads and Keywords / Paid Search Ads – search engine marketing (search ads) targeted at potential participants.
- Navigating HAE FB and IG Pinned Posts – use of Facebook/Instagram pinned posts to inform/engage the patient community.
- Doctor-to-patient letters (Doctor to patient letter_adolescents / _adults) – letters from clinicians to patients (target: adolescents and adults).
- Brochures (Brochure adolescents / Brochure adults / Navenibart brochure) – printed or downloadable informational brochures tailored for adolescents and adults.
- Consent Navigator and Consent Flyer – materials to help potential participants navigate consent information and provide an overview.
- Country-specific ad campaigns and materials (documents labelled DE, FR, IT, HU, CZ, BG, PT, NL, ES, etc.) – recruitment content adapted/language-specific for Member States.
Geography
- Total Number Of Sites
- 33
- Total Number Of Participants
- 53
Germany
- Earliest CTIS Part Ii Submission Date
- 07-08-2025
- Latest Decision Or Authorization Date
- 23-01-2026
- Processing Time Days
- 169
- Number Of Sites
- 3
- Number Of Participants
- 7
Sites
- Site Name
- Universitaetsklinikum Frankfurt AöR
- Department Name
- Klinik für Kinder und Jugendmedizin, Angioödem-Ambulanz und interdisziplinäres HAE-Kompetenzzentrum
- 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
- Site Name
- Charite Universitaetsmedizin Berlin KöR
- Department Name
- Institut für Allergieforschung
- Principal Investigator Name
- Markus Magerl
- Principal Investigator Email
- marcus.magerl@charite.de
- Contact Person Name
- Markus Magerl
- Contact Person Email
- marcus.magerl@charite.de
- Site Name
- Medizinische Hochschule Hannover
- Department Name
- Dept. of Dermatology and Allergology
- Principal Investigator Name
- Bettina Wedi
- Principal Investigator Email
- wedi.bettina@mh-hannover.de
- Contact Person Name
- Bettina Wedi
- Contact Person Email
- wedi.bettina@mh-hannover.de
Portugal
- Earliest CTIS Part Ii Submission Date
- 18-06-2025
- Latest Decision Or Authorization Date
- 20-01-2026
- Processing Time Days
- 216
- Number Of Sites
- 2
- Number Of Participants
- 5
Sites
- Site Name
- Unidade Local De Saude De Coimbra E.P.E.
- Department Name
- Imunoalergologia
- Principal Investigator Name
- Frederico Regateiro
- Principal Investigator Email
- 10432@ulscoimbra.min-saude.pt
- Contact Person Name
- Frederico Regateiro
- Contact Person Email
- 10432@ulscoimbra.min-saude.pt
- Site Name
- Unidade Local De Saude De Santa Maria E.P.E.
- Department Name
- Imunoalergologia
- Principal Investigator Name
- Manuel Ferreira
- Principal Investigator Email
- mbrancoferreira@gmail.com
- Contact Person Name
- Manuel Ferreira
- Contact Person Email
- mbrancoferreira@gmail.com
Austria
- Earliest CTIS Part Ii Submission Date
- 08-08-2025
- Latest Decision Or Authorization Date
- 21-01-2026
- Processing Time Days
- 166
- Number Of Sites
- 2
- Number Of Participants
- 2
Sites
- Site Name
- Medical University Of Graz
- Department Name
- Dermatologie und Venerologie
- Principal Investigator Name
- Birger Kränke
- Principal Investigator Email
- birger.kraenke@meduniqraz.at
- Contact Person Name
- Birger Kränke
- Contact Person Email
- birger.kraenke@meduniqraz.at
- Site Name
- Medical University Of Vienna
- Department Name
- Deparment of Dermatology
- Principal Investigator Name
- Tamar Kinaciyan
- Principal Investigator Email
- tamar.kinaciyan@meduniwien.ac.at
- Contact Person Name
- Tamar Kinaciyan
- Contact Person Email
- tamar.kinaciyan@meduniwien.ac.at
Poland
- Earliest CTIS Part Ii Submission Date
- 14-07-2025
- Latest Decision Or Authorization Date
- 26-01-2026
- Processing Time Days
- 196
- Number Of Sites
- 5
- Number Of Participants
- 7
Sites
- Site Name
- Wojskowy Instytut Medyczny Panstwowy Instytut Badawczy
- Department Name
- Klinika Chorob Wewnetrznych, Pneumonologii, Alergologii, Immunologii Klinicznej i Chorob Rzadkich
- Principal Investigator Name
- Aleksandra Kucharczyk
- Principal Investigator Email
- akucharczyk@wim.mil.pl
- Contact Person Name
- Aleksandra Kucharczyk
- Contact Person Email
- akucharczyk@wim.mil.pl
- Site Name
- Samodzielny Publiczny Zaklad Opieki Zdrowotnej Szpital Uniwersytecki W Krakowie
- Department Name
- Imunoalergologia
- 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
- Site Name
- Szpital Uniwersytecki Nr 2 Im Dr Jana Biziela W Bydgoszczy
- Department Name
- Klinika Alergologii, Immunologii Klinicznej i Chorob Wewnetrznych
- Principal Investigator Name
- Zbigniew Bartuzi
- Principal Investigator Email
- kikalerg@cm.umk.pl
- Contact Person Name
- Zbigniew Bartuzi
- Contact Person Email
- kikalerg@cm.umk.pl
- Site Name
- Umed Clinical Trials Sp. z o.o.
- Principal Investigator Name
- Marcin Kurowski
- Principal Investigator Email
- marcin.kurowski@umed.lodz.pl
- Contact Person Name
- Marcin Kurowski
- Contact Person Email
- marcin.kurowski@umed.lodz.pl
- Site Name
- Pawlukiewicz Małgorzata Prywatny Gabinet Lekarski
- Principal Investigator Name
- Malgorzata Pawlukiewicz
- Principal Investigator Email
- mpawlu@onet.eu
- Contact Person Name
- Malgorzata Pawlukiewicz
- Contact Person Email
- mpawlu@onet.eu
Bulgaria
- Earliest CTIS Part Ii Submission Date
- 06-08-2025
- Latest Decision Or Authorization Date
- 26-01-2026
- Processing Time Days
- 173
- Number Of Sites
- 1
- Number Of Participants
- 3
Sites
- Site Name
- Diagnostics And Consultation Center Convex Ltd.
- Principal Investigator Name
- Anna Valerieva Dimitrova
- Principal Investigator Email
- anna.valerieva@gmail.com
- Contact Person Name
- Anna Valerieva Dimitrova
- Contact Person Email
- anna.valerieva@gmail.com
Netherlands
- Earliest CTIS Part Ii Submission Date
- 17-07-2025
- Latest Decision Or Authorization Date
- 21-01-2026
- Processing Time Days
- 188
- Number Of Sites
- 1
- Number Of Participants
- 2
Sites
- Site Name
- Amsterdam UMC Stichting
- Department Name
- Vascular Medicine
- Principal Investigator Name
- Danny Cohn
- Principal Investigator Email
- d.m.cohn@amsterdamumc.nl
- Contact Person Name
- Danny Cohn
- Contact Person Email
- d.m.cohn@amsterdamumc.nl
Italy
- Earliest CTIS Part Ii Submission Date
- 20-05-2025
- Latest Decision Or Authorization Date
- 19-01-2026
- Processing Time Days
- 244
- Number Of Sites
- 6
- Number Of Participants
- 8
Sites
- Site Name
- ASST Fatebenefratelli Sacco
- Department Name
- S.C. di Medicina Interna
- Principal Investigator Name
- Antonio Gidaro
- Principal Investigator Email
- gidaro.antonio@asst-fbf-sacco.it
- Contact Person Name
- Antonio Gidaro
- Contact Person Email
- gidaro.antonio@asst-fbf-sacco.it
- Site Name
- Istituti Clinici Scientifici Maugeri S.p.A. Sb
- Department Name
- Ambulatorio di orticaria angioedema
- Principal Investigator Name
- Francesca Perego
- Principal Investigator Email
- francesca.perego@icsmaugeri.it
- Contact Person Name
- Francesca Perego
- Contact Person Email
- francesca.perego@icsmaugeri.it
- Site Name
- Azienda Ospedaliera Ordine Mauriziano Di Torino
- Department Name
- S.C.D.U. Immunologia e Allergologia
- Principal Investigator Name
- Luisa Brussino
- Principal Investigator Email
- luisa.brussino@unito.it
- Contact Person Name
- Luisa Brussino
- Contact Person Email
- luisa.brussino@unito.it
- Site Name
- Policlinico San Donato S.p.A.
- Department Name
- Centro Angioedema
- Principal Investigator Name
- Andrea Zanichelli
- Principal Investigator Email
- andrea.zanichelli@unimi.it
- Contact Person Name
- Andrea Zanichelli
- Contact Person Email
- andrea.zanichelli@unimi.it
- Site Name
- Azienda Ospedaliero-Universitaria Di Cagliari
- Department Name
- SC Medicina Interna - Departimento Scienze Mediche e Sanità Pubblica
- Principal Investigator Name
- Davide Firinu
- Principal Investigator Email
- davide.firinu@unica.it
- Contact Person Name
- Davide Firinu
- Contact Person Email
- davide.firinu@unica.it
- Site Name
- Azienda Ospedaliera di Padova
- Department Name
- UOSD di 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
France
- Earliest CTIS Part Ii Submission Date
- 25-07-2025
- Latest Decision Or Authorization Date
- 23-01-2026
- Processing Time Days
- 182
- Number Of Sites
- 5
- Number Of Participants
- 7
Sites
- Site Name
- Centre Hospitalier Regional De Marseille
- Department Name
- Internal medicine
- Principal Investigator Name
- Stéphane Gayet
- Principal Investigator Email
- stephane.gayet@ap-hm.fr
- Contact Person Name
- Stéphane Gayet
- Contact Person Email
- stephane.gayet@ap-hm.fr
- Site Name
- Centre Hospitalier Universitaire Grenoble Alpes
- Department Name
- internal medicine department and clinical immunology outpatient clinic
- Principal Investigator Name
- Isabelle Boccon-Gibod
- Principal Investigator Email
- lboccon-gibod@chu-grenoble.fr
- Contact Person Name
- Isabelle Boccon-Gibod
- Contact Person Email
- lboccon-gibod@chu-grenoble.fr
- Site Name
- Assistance Publique Hopitaux De Paris
- Department Name
- Internal medicine
- Principal Investigator Name
- Olivier Fain
- Principal Investigator Email
- olivier.fain@aphp.fr
- Contact Person Name
- Olivier Fain
- Contact Person Email
- olivier.fain@aphp.fr
- Site Name
- Centre Hospitalier Universitaire De Nice
- Department Name
- Internal medicine
- Principal Investigator Name
- Pierre-Yves Jeandel
- Principal Investigator Email
- jeandel.py@chu-nice.fr
- Contact Person Name
- Pierre-Yves Jeandel
- Contact Person Email
- jeandel.py@chu-nice.fr
- Site Name
- Centre Hospitalier Universitaire De Lille
- Department Name
- Internal medicine
- Principal Investigator Name
- David Launay
- Principal Investigator Email
- david.launay@univ-lille.fr
- Contact Person Name
- David Launay
- Contact Person Email
- david.launay@univ-lille.fr
Hungary
- Earliest CTIS Part Ii Submission Date
- 03-07-2025
- Latest Decision Or Authorization Date
- 19-01-2026
- Processing Time Days
- 200
- Number Of Sites
- 1
- Number Of Participants
- 2
Sites
- Site Name
- Semmelweis University
- Department Name
- Internal medicine and Haematology
- Principal Investigator Name
- Henriette Farkas
- Principal Investigator Email
- farkas.henriette@semmelweis.hu
- Contact Person Name
- Henriette Farkas
- Contact Person Email
- farkas.henriette@semmelweis.hu
Spain
- Earliest CTIS Part Ii Submission Date
- 18-07-2025
- Latest Decision Or Authorization Date
- 23-01-2026
- Processing Time Days
- 189
- Number Of Sites
- 5
- Number Of Participants
- 7
Sites
- Site Name
- University Hospital Virgen Del Rocio S.L.
- Department Name
- Allergyc
- Principal Investigator Name
- Stefan Henning Cimbollek
- Principal Investigator Email
- stefan.henning.sspa@juntadeandalucia.es
- Contact Person Name
- Stefan Henning Cimbollek
- Contact Person Email
- stefan.henning.sspa@juntadeandalucia.es
- Site Name
- Bellvitge University Hospital
- Department Name
- Allergyc
- Principal Investigator Name
- Blanca Andres Lopez
- Principal Investigator Email
- b.andres@bellvitgehospital.cat
- Contact Person Name
- Blanca Andres Lopez
- Contact Person Email
- b.andres@bellvitgehospital.cat
- Site Name
- Hospital Universitario Nuestra Senora De Candelaria
- Department Name
- Allergyc
- Principal Investigator Name
- Ariel Callero
- Principal Investigator Email
- arielcallero@gmail.com
- Contact Person Name
- Ariel Callero
- Contact Person Email
- arielcallero@gmail.com
- Site Name
- Hospital General Universitario Gregorio Maranon
- Department Name
- Allergyc
- Principal Investigator Name
- Alicia Prieto Garcia
- Principal Investigator Email
- alicia.prieto@salud.madrid.org
- Contact Person Name
- Alicia Prieto Garcia
- Contact Person Email
- alicia.prieto@salud.madrid.org
- Site Name
- Hospital Universitario Y Politecnico La Fe
- Department Name
- Allergyc
- Principal Investigator Name
- Leah Landaveri Sánchez
- Principal Investigator Email
- landaveri_lea@gva.es
- Contact Person Name
- Leah Landaveri Sánchez
- Contact Person Email
- landaveri_lea@gva.es
Czechia
- Earliest CTIS Part Ii Submission Date
- 09-07-2025
- Latest Decision Or Authorization Date
- 01-02-2026
- Processing Time Days
- 207
- Number Of Sites
- 2
- Number Of Participants
- 3
Sites
- Site Name
- Fakultni Nemocnice V Motole
- Department Name
- Ústav imunologie 2. LF UK a FN Motol
- Principal Investigator Name
- Marta Sobotková
- Principal Investigator Email
- maria.sobotkova@fnmotol.cz
- Contact Person Name
- Marta Sobotková
- Contact Person Email
- maria.sobotkova@fnmotol.cz
- Site Name
- Fakultni Nemocnice Hradec Kralove
- Department Name
- Ustav klinické imunologie a alergologie
- Principal Investigator Name
- Irena Krčmová
- Principal Investigator Email
- irena.krcmova@fnhk.cz
- Contact Person Name
- Irena Krčmová
- Contact Person Email
- irena.krcmova@fnhk.cz
Sponsor
Primary sponsor
- Full Name
- Astria Therapeutics Inc.
- Organisation Type
- Pharmaceutical company
- Country Of Registered Address
- United States
Contract research organisations
- Name
- Medidata Solutions Inc.
- Responsibilities
- RTSM (Randomization and Trial Supply Management); additional sponsor duties codes present
- Name
- Fortrea Inc.
- Responsibilities
- Multiple operational roles including ancillary supplies management
- Name
- Labcorp Central Laboratory Services SARL / LP
- Responsibilities
- Central laboratory services (contact: GenevaCLS@labcorp.com / ctasubmissions@labcorp.com)
- Name
- Certara USA Inc.
Third parties
- {"country":"Switzerland","full_name":"Labcorp Central Laboratory Services SARL","duties_or_roles":"","organisation_type":"Pharmaceutical company"}
- {"country":"United States","full_name":"Certara USA Inc.","duties_or_roles":"","organisation_type":"Pharmaceutical company"}
- {"country":"United States","full_name":"Medidata Solutions Inc.","duties_or_roles":"RTSM (Randomization and Trial Supply Management)","organisation_type":"Non-Pharmaceutical company"}
- {"country":"United States","full_name":"Labcorp Central Laboratory Services LP","duties_or_roles":"","organisation_type":"Pharmaceutical company"}
- {"country":"United States","full_name":"Fortrea Inc.","duties_or_roles":"Ancillary supplies management","organisation_type":"Pharmaceutical company"}
Investigational products
- Investigational Product Name
- Navenibart
- Active Substance
- NAVENIBART
- Modality
- Monoclonal antibody
- Routes Of Administration
- Subcutaneous
- Route
- Subcutaneous
- Authorisation Status
- Authorised (prodAuthStatus=1)
- Orphan Designation
- Yes
- Maximum Dose
- Max daily dose 600 mg; max total amount 1200 mg (as listed in product metadata)
- Investigational Product Name
- Navenibart - matching placebo
- Modality
- Other
- Authorisation Status
- Not applicable
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