Clinical trial • Phase III • Haematology
SEBETRALSTAT for Hereditary angioedema (Type I or II)
Phase III trial of SEBETRALSTAT for Hereditary angioedema (Type I or II). open-label. 75 participants.
Overview
- Trial Therapeutic Area
- Haematology
- Trial Disease
- Hereditary angioedema (Type I or II)
- Trial Stage
- Phase III
- Drug Modality
- Small molecule
- Paediatric Trial
- Yes
- Orphan Drug
- Yes
Key dates
- Initial CTIS Submission Date
- 23-11-2023
- First CTIS Authorization Date
- 26-02-2024
Trial design
open-label Phase III trial in Austria, Italy, Portugal and others.
- Open Label
- Yes
- Target Sample Size
- 75
Eligibility
Recruits 75 paediatric patients.
- Pregnancy Exclusion
- Any pregnant or breastfeeding patient.
- Vulnerable Population
- Includes adolescents aged 12–17 (vulnerable population). The patient must provide signed informed consent or assent when applicable; a parent or legally authorised representative (LAR) must also provide signed informed consent when required. Country-specific assent and parental ICF documents are provided (separate adolescent assent forms and parental ICFs listed in the documentation). The Investigator assesses the patient's ability to consent/assent and to complete study procedures (e.g., eDiary).
Inclusion criteria
- {"criterion_text":"- Patients may roll over from KVD900-301.\n- Patient provides signed informed consent or assent (when applicable). A parent or LAR must also provide signed informed consent when required.\n- Confirmed diagnosis of HAE type I or II at any time in the medical history\n- Patient has had at least 2 documented HAE attacks within 3 months prior to the Enrollment Visit\n- If a patient is receiving long-term prophylactic treatment with one of the protocol-allowed therapies, they must have been on a stable dose and regimen for at least 3 months prior to the Enrollment Visit\n- Male or female patients 12 years of age and older.\n- Patients must meet the contraception requirements.\n- Patients must be able to swallow trial tablets whole.\n- Patients, as assessed by the Investigator, must be able to appropriately receive and store IMP, and be able to read, understand, and complete the eDiary.\n- Investigator believes that the patient is willing and able to adhere to all protocol requirements."}
Exclusion criteria
- {"criterion_text":"- Discontinued from the KVD900-301 trial for reasons of non-compliance, withdrawal of consent, or safety.\n- History of substance abuse or dependence that would interfere with the completion of the trial, as determined by the Investigator.\n- Known hypersensitivity to KVD900 or to any of the excipients.\n- Participation in any gene therapy treatment or trial for HAE.\n- Participation in any interventional investigational clinical trial, including an investigational COVID-19 vaccine trial, within 4 weeks of the last dosing of investigational drug prior to the Enrollment Visit.\n- Any pregnant or breastfeeding patient.\n- Presence of any safety concerns that would preclude participation in the open-label trial as determined by the investigator.\n- Any concomitant diagnosis of another form of chronic angioedema, such as acquired C1 inhibitor deficiency, HAE with normal C1-INH (previously known as HAE type III), idiopathic angioedema, or angioedema associated with urticaria.\n- A clinically significant history of poor response to bradykinin receptor 2 (BR2) blocker, C1-INH therapy, or plasma kallikrein inhibitor therapy for the management of HAE, in the opinion of the Investigator.\n- Use of attenuated androgens (e.g., stanozolol, danazol, oxandrolone, methyltestosterone, testosterone), or anti-fibrinolytics (e.g., tranexamic acid) within 28 days prior to the Enrollment Visit.\n- Use of ACE inhibitors within 7 days prior to the Enrollment Visit.\n- Any estrogen-containing medications with systemic absorption (such as oral contraceptives including ethinylestradiol or hormonal replacement therapy) within 7 days prior to the Enrollment Visit.\n- Inadequate organ function, including but not limited to: a) Alanine aminotransferase (ALT) >2x ULN b) Aspartate aminotransferase (AST) >2x ULN c) Bilirubin direct >1.25x ULN d) INR >1.2 e) Clinically significant hepatic impairment defined as a Child-Pugh B or C\n- Any clinically significant comorbidity or systemic dysfunction, which in the opinion of the Investigator, would jeopardize the safety of the patient by participating in the trial."}
Endpoints
Primary endpoints
- {"endpoint_text":"- Frequencies and percentages of patients with AEs, AEs within 2 days of IMP administration, serious AEs, and AEs causing premature discontinuation","definition_or_measurement_approach":"Measured as frequencies and percentages of patients with adverse events (including AEs within 2 days of IMP administration), serious adverse events, and AEs causing premature discontinuation (per scheduled assessments and safety reporting)."}
- {"endpoint_text":"- Number and percentage of patients with normal or abnormal laboratory results at each scheduled visit","definition_or_measurement_approach":"Measured as counts and percentages of patients with normal versus abnormal laboratory results at each scheduled visit."}
- {"endpoint_text":"- Number and percentage of patients with normal or abnormal vital sign results at each scheduled visit","definition_or_measurement_approach":"Measured as counts and percentages of patients with normal versus abnormal vital sign measurements at each scheduled visit."}
Secondary endpoints
- {"endpoint_text":"- PGI-C: time to beginning of symptom relief defined as at least '' a little better'' (2 time points in a row) within 12 hours of initial dose of IMP administration.","definition_or_measurement_approach":"Patient Global Impression of Change (PGI-C): time from initial IMP dose to first occurrence of at least 'a little better' on two consecutive time points within 12 hours."}
- {"endpoint_text":"- PGI-S: time to first incidence of 2 time points in a row decrease from baseline within 12 hours of initial dose of IMP administration.","definition_or_measurement_approach":"Patient Global Impression of Severity (PGI-S): time from initial IMP dose to first occurrence of a decrease from baseline on two consecutive time points within 12 hours."}
- {"endpoint_text":"- PGI-S: time to HAE attack resolution defined as ''none'' within 24 hours of initial dose of IMP administration.","definition_or_measurement_approach":"PGI-S: time from initial IMP dose to resolution of HAE attack (PGI-S = 'none') within 24 hours."}
Recruitment
- Registry Or Advocacy Recruitment
- True: site and patient advocacy contact list referenced (no specific organisations named in the public documents)
- Digital Remote Recruitment
- True: use of email communications (patient concierge emails, Scout email communications), digital eDiary for patients, and electronic communications/materials (flip-charts, appointment reminder emails) referenced in country documents
- Planned Sample Size
- 75
- Recruitment Window Months
- 44
- Consent Approach
- Patients provide signed informed consent; adolescents (12–17 years) provide assent where applicable and a parent or legally authorised representative (LAR) must also sign parental ICFs when required. Country- and age-specific assent and parental ICF documents are provided (examples in the dossier: assent forms and parental ICFs for 12–13 and 14–17 years; main ICFs and parental ICFs available in multiple country languages).
Methods
- Site and patient advocacy contact lists (document: Site_and_Patient_Advocacy_Contact_List_for_ICF_Public)
- Country-specific recruitment arrangements documents (K1/Recruiment-Arrangements PDFs per country)
- GP letter (e.g. K2_KVD900-302_GP_Letter_HU for Hungary)
- Patient Concierge communications including welcome and unable-to-reach emails (Hungary documents)
- Appointment reminder cards and patient cards (Hungary documents)
- Scout email communications and ScoutPass reloadable voucher materials (Hungary documents)
- IA Flip-Chart recruitment materials (Spain and PK-substudy flip-charts listed)
- Digital communications via email and site contact (many country-specific documents reference email contact details)
Geography
- Total Number Of Sites
- 26
- Total Number Of Participants
- 75
Austria
- Earliest CTIS Part Ii Submission Date
- 13-12-2023
- Latest Decision Or Authorization Date
- 03-03-2025
- Processing Time Days
- 446
- Number Of Sites
- 1
- Number Of Participants
- 3
Sites
- Site Name
- Medical University Of Vienna
- Department Name
- Department 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
Italy
- Earliest CTIS Part Ii Submission Date
- 13-12-2023
- Latest Decision Or Authorization Date
- 28-02-2025
- Processing Time Days
- 443
- Number Of Sites
- 4
- Number Of Participants
- 8
Sites
- Site Name
- Azienda Ospedaliera Policlinico Universitario Tor Vergata
- Department Name
- Dipartimento di Scienze Mediche, UOC Reumatologia
- Principal Investigator Name
- Paola Triggianese
- Principal Investigator Email
- paola.triggianese@ptvonline.it
- Contact Person Name
- Paola Triggianese
- Contact Person Email
- paola.triggianese@ptvonline.it
- Site Name
- Azienda Ospedaliera Ospedali Riuniti Villa Sofia Cervello
- Department Name
- UOC di Patologia Clinica – Centro Angioedema
- Principal Investigator Name
- Francesco Arcoleo
- Principal Investigator Email
- farcoleo@villasofia.it
- Contact Person Name
- Francesco Arcoleo
- Contact Person Email
- farcoleo@villasofia.it
- Site Name
- Azienda Ospedale-Universita Padova
- Department Name
- Clinica Medica 1 Dipartimento di Medicina dei Sistemi UOSD Allergologia
- Principal Investigator Name
- Mauro Cancian
- Principal Investigator Email
- mcancian@unipd.it
- Contact Person Name
- Mauro Cancian
- Contact Person Email
- mcancian@unipd.it
- Site Name
- IRCCS Policlinico San Donato
- Department Name
- U.O. di Medicina Interna
- Principal Investigator Name
- Andrea Zanichelli
- Principal Investigator Email
- andrea.zanichelli@unimi.it
- Contact Person Name
- Andrea Zanichelli
- Contact Person Email
- andrea.zanichelli@unimi.it
Portugal
- Earliest CTIS Part Ii Submission Date
- 13-12-2023
- Latest Decision Or Authorization Date
- 26-02-2025
- Processing Time Days
- 442
- Number Of Sites
- 1
- Number Of Participants
- 4
Sites
- Site Name
- Unidade Local de Saude de Sao Joao E.P.E.
- Department Name
- Immunoallergology Service
- Principal Investigator Name
- Eunice Dias de Castro
- Principal Investigator Email
- eunicediascastro@gmail.com
- Contact Person Name
- Eunice Dias de Castro
- Contact Person Email
- eunicediascastro@gmail.com
Spain
- Earliest CTIS Part Ii Submission Date
- 13-12-2023
- Latest Decision Or Authorization Date
- 27-02-2025
- Processing Time Days
- 442
- Number Of Sites
- 3
- Number Of Participants
- 9
Sites
- Site Name
- Hospital Universitari Vall D Hebron
- Department Name
- Servicio de Alergia
- Principal Investigator Name
- Mar Guilarte Clavero
- Principal Investigator Email
- mguilarte@vhebron.net
- Contact Person Name
- Mar Guilarte Clavero
- Contact Person Email
- mguilarte@vhebron.net
- Site Name
- Bellvitge University Hospital
- Department Name
- Servicio de Alergia
- Principal Investigator Name
- Ramón Lleonart Bellfill
- Principal Investigator Email
- rlleonart@gmail.com
- Contact Person Name
- Ramón Lleonart Bellfill
- Contact Person Email
- rlleonart@gmail.com
- Site Name
- Hospital Universitario La Paz
- Department Name
- Servicio de Alergia
- Principal Investigator Name
- Teresa Caballero Molina
- Principal Investigator Email
- tercaballero@gmail.com
- Contact Person Name
- Teresa Caballero Molina
- Contact Person Email
- tercaballero@gmail.com
Romania
- Earliest CTIS Part Ii Submission Date
- 13-12-2023
- Latest Decision Or Authorization Date
- 03-03-2025
- Processing Time Days
- 446
- Number Of Sites
- 1
- Number Of Participants
- 1
Sites
- Site Name
- Centrul Clinic Mediquest S.R.L.
- Department Name
- Allergology and clinical immunology
- Principal Investigator Name
- Noemi Bara
- Principal Investigator Email
- noemi.bara@yahoo.com
- Contact Person Name
- Noemi Bara
- Contact Person Email
- noemi.bara@yahoo.com
Hungary
- Earliest CTIS Part Ii Submission Date
- 13-12-2023
- Latest Decision Or Authorization Date
- 27-02-2025
- Processing Time Days
- 442
- Number Of Sites
- 1
- Number Of Participants
- 2
Sites
- Site Name
- Semmelweis University
- Department Name
- Belgyogyaszati es Hematologiai Klinika
- Principal Investigator Name
- Henriette FARKAS
- Principal Investigator Email
- farkas.henriette@med.semmelweis-univ.hu
- Contact Person Name
- Henriette FARKAS
- Contact Person Email
- farkas.henriette@med.semmelweis-univ.hu
Bulgaria
- Earliest CTIS Part Ii Submission Date
- 13-12-2023
- Latest Decision Or Authorization Date
- 04-03-2025
- Processing Time Days
- 447
- Number Of Sites
- 1
- Number Of Participants
- 3
Sites
- Site Name
- Alexandrovska University Hospital
- Department Name
- Allergology clinic
- Principal Investigator Name
- Maria Staevska
- Principal Investigator Email
- mari66sta@gmail.com
- Contact Person Name
- Maria Staevska
- Contact Person Email
- mari66sta@gmail.com
Netherlands
- Earliest CTIS Part Ii Submission Date
- 13-12-2023
- Latest Decision Or Authorization Date
- 05-03-2025
- Processing Time Days
- 448
- Number Of Sites
- 1
- Number Of Participants
- 4
Sites
- Site Name
- Academisch Medisch Centrum
- Principal Investigator Name
- Danny Michael Cohn
- Principal Investigator Email
- HAE@amsterdamumc.nl
- Contact Person Name
- Danny Michael Cohn
- Contact Person Email
- HAE@amsterdamumc.nl
Greece
- Earliest CTIS Part Ii Submission Date
- 13-12-2023
- Latest Decision Or Authorization Date
- 04-03-2025
- Processing Time Days
- 447
- Number Of Sites
- 2
- Number Of Participants
- 8
Sites
- Site Name
- Athens Naval Hospital
- Department Name
- Allergiology Department
- Principal Investigator Name
- Fotios Psarros
- Principal Investigator Email
- psarros@allergy.gr
- Contact Person Name
- Fotios Psarros
- Contact Person Email
- psarros@allergy.gr
- Site Name
- Laiko General Hospital Of Athens
- Department Name
- Allergiology Department
- Principal Investigator Name
- Nikolaos Mikos
- Principal Investigator Email
- mikosnikos@gmail.com
- Contact Person Name
- Nikolaos Mikos
- Contact Person Email
- mikosnikos@gmail.com
Poland
- Earliest CTIS Part Ii Submission Date
- 13-12-2023
- Latest Decision Or Authorization Date
- 02-03-2025
- Processing Time Days
- 445
- Number Of Sites
- 2
- Number Of Participants
- 8
Sites
- Site Name
- Samodzielny Publiczny Zaklad Opieki Zdrowotnej Szpital Uniwersytecki W Krakowie
- Department Name
- Ambulatoria Uniwersyteckie Zespół Poradni Specjalistycznych SU Centrum Alergologii
- Principal Investigator Name
- Marcin Stobiecki
- Principal Investigator Email
- centrumalergologii@su.krakow.pl
- Contact Person Name
- Marcin Stobiecki
- Contact Person Email
- centrumalergologii@su.krakow.pl
- Site Name
- Samodzielny Publiczny Zaklad Opieki Zdrowotnej Centralny Szpital Kliniczny Uniwersytetu Medycznego W Lodzi
- Department Name
- Klinika Immunologii i Alergii
- Principal Investigator Name
- Marcin Maciej Kurowski
- Principal Investigator Email
- marcin.kurowski@umed.lodz.pl
- Contact Person Name
- Marcin Maciej Kurowski
- Contact Person Email
- marcin.kurowski@umed.lodz.pl
Slovakia
- Earliest CTIS Part Ii Submission Date
- 13-12-2023
- Latest Decision Or Authorization Date
- 27-02-2025
- Processing Time Days
- 441
- Number Of Sites
- 1
- Number Of Participants
- 3
Sites
- Site Name
- Univerzitna Nemocnica Martin
- Department Name
- Klinika detí a dorastu
- Principal Investigator Name
- Miloš Jeseňák
- Principal Investigator Email
- jesenak@gmail.com
- Contact Person Name
- Miloš Jeseňák
- Contact Person Email
- jesenak@gmail.com
France
- Earliest CTIS Part Ii Submission Date
- 13-12-2023
- Latest Decision Or Authorization Date
- 08-08-2025
- Processing Time Days
- 604
- Number Of Sites
- 4
- Number Of Participants
- 10
Sites
- Site Name
- Centre Hospitalier Universitaire Grenoble Alpes
- Department Name
- Service de Médecine Interne
- Principal Investigator Name
- Laurence BOUILLET
- Principal Investigator Email
- lbouillet@chu-grenoble.fr
- Contact Person Name
- Laurence BOUILLET
- Contact Person Email
- lbouillet@chu-grenoble.fr
- Site Name
- Centre Hospitalier Universitaire De Lille
- Department Name
- Unité Pédiatrique de Rhumatologie
- Principal Investigator Name
- Héloïse REUMAUX
- Principal Investigator Email
- heloise.reumaux@chu-lille.fr
- Contact Person Name
- Héloïse REUMAUX
- Contact Person Email
- heloise.reumaux@chu-lille.fr
- Site Name
- Assistance Publique Hopitaux De Paris
- Department Name
- Service de Médecine Interne Bâtiment de l'Horloge
- 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 Lille (second site)
- Department Name
- Service De Médecine Interne
- 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
Germany
- Earliest CTIS Part Ii Submission Date
- 13-12-2023
- Latest Decision Or Authorization Date
- 06-02-2026
- Processing Time Days
- 786
- Number Of Sites
- 4
- Number Of Participants
- 12
Sites
- Site Name
- Universitaetsmedizin Der Johannes Gutenberg-Universitaet Mainz
- Department Name
- Haut-und Poliklinik Abteilung für Dermatologie und Allergologie
- Principal Investigator Name
- Petra Staubach-Renz
- Principal Investigator Email
- petra.staubach@unimedizin-mainz.de
- Contact Person Name
- Petra Staubach-Renz
- Contact Person Email
- petra.staubach@unimedizin-mainz.de
- Site Name
- HZRM Haemophilie-Zentrum Rhein Main GmbH
- Department Name
- Diagnostik, Therapie und Erforschung von Gerinnungsstörungen, Immundefekten und HAE
- Principal Investigator Name
- Carmen Escuriola Ettingshausen
- Principal Investigator Email
- inmaculada.martinez@hzrm.de
- Contact Person Name
- Carmen Escuriola Ettingshausen
- Contact Person Email
- inmaculada.martinez@hzrm.de
- Site Name
- Charite Universitaetsmedizin Berlin KöR
- Department Name
- Institute of Allergology (IFA)
- Principal Investigator Name
- Markus Magerl
- Principal Investigator Email
- markus.magerl@charite.de
- Contact Person Name
- Markus Magerl
- Contact Person Email
- markus.magerl@charite.de
- Site Name
- Universitaetsklinikum Frankfurt AöR
- Department Name
- Klinik für Kinder- und Jugendmeizin
- Principal Investigator Name
- Emel Aygören-Pürsün
- Principal Investigator Email
- emel.aygoeren@kgu.de
- Contact Person Name
- Emel Aygören-Pürsün
- Contact Person Email
- emel.aygoeren@kgu.de
Sponsor
Primary sponsor
- Full Name
- Kalvista Pharmaceuticals Limited
- Organisation Type
- Pharmaceutical company
- Country Of Registered Address
- United Kingdom
Contract research organisations
- Name
- Ppd Inc.
- Responsibilities
- Clinical operations, monitoring, data management and other operational sponsor duties; CSR Writing and multiple listed responsibilities
- Name
- Medidata Solutions International Limited
- Responsibilities
- IVRS / randomisation (IVRS30 – treatment randomisation)
- Name
- PPD Global Central Labs / PPD Global Clinical Labs
- Responsibilities
- Laboratory services (central lab activities)
- Name
- Arriello s.r.o.
- Responsibilities
- Aggregate report and line listing submission activities; safety reporting contact (email kalvista.safety@arriello.com)
- Name
- Mayo Collaborative Services LLC
- Responsibilities
- C1-esterase inhibitor activity and antigen testing
- Name
- York Bioanalytical Solutions Limited
- Responsibilities
- Bioanalytical testing services
Third parties
- {"country":"Czechia","full_name":"Arriello s.r.o.","duties_or_roles":"Aggregate report and line listing submission activities; (additional sponsor duties codes present)","organisation_type":"Pharmaceutical company"}
- {"country":"United States","full_name":"Mayo Collaborative Services LLC","duties_or_roles":"C1-esterase inhibitor activity (functional level) and C1-esterase inhibitor protein (antigen)","organisation_type":"Laboratory/Research/Testing facility"}
- {"country":"United Kingdom","full_name":"Medidata Solutions International Limited","duties_or_roles":"IVRS30 – treatment randomisation; (additional sponsor duty code present)","organisation_type":"Pharmaceutical company"}
- {"country":"United States","full_name":"Ppd Inc.","duties_or_roles":"Multiple sponsor duties including CSR Writing and other study operational responsibilities (codes listed in dossier)","organisation_type":"Pharmaceutical company"}
- {"country":"Belgium","full_name":"PPD Global Central Labs","duties_or_roles":"Central laboratory services (sponsor duty code 4)","organisation_type":"Pharmaceutical company"}
- {"country":"United States","full_name":"PPD Global Clinical Labs","duties_or_roles":"Central laboratory services (sponsor duty code 4)","organisation_type":"Industry (lab)"}
- {"country":"United Kingdom","full_name":"York Bioanalytical Solutions Limited","duties_or_roles":"Bioanalytical laboratory services (sponsor duty code 4)","organisation_type":"Laboratory/Research/Testing facility"}
Investigational products
- Investigational Product Name
- KVD900 (ORODISPERSIBLE TABLET)
- Active Substance
- SEBETRALSTAT
- Modality
- Small molecule
- Routes Of Administration
- ORAL
- Route
- ORAL
- Authorisation Status
- Authorised
- Orphan Designation
- Yes
- Maximum Dose
- 900 mg
- Investigational Product Name
- KVD900 (FILM-COATED TABLET)
- Active Substance
- SEBETRALSTAT
- Modality
- Small molecule
- Routes Of Administration
- ORAL
- Route
- ORAL
- Authorisation Status
- Authorised
- Orphan Designation
- Yes
- Maximum Dose
- 1800 mg
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