Clinical trial • Phase II • Rare Disease
BW-20805 for Hereditary angioedema | HAE type I/II
Phase II trial of BW-20805 for Hereditary angioedema | HAE type I/II. open-label. 14 participants.
Overview
- Trial Therapeutic Area
- Rare Disease
- Trial Disease
- Hereditary angioedema | HAE type I/II
- Trial Stage
- Phase II
- Drug Modality
- Oligonucleotide
Key dates
- Initial CTIS Submission Date
- 20-12-2024
- First CTIS Authorization Date
- 07-04-2025
Trial design
open-label Phase II trial in Spain, Italy, Germany and others.
- Open Label
- Yes
- Target Sample Size
- 14
- Trial Duration For Participant
- 225
Eligibility
Recruits 14 Vulnerable population flag selected. Participants must provide written informed consent. Dedicated informed consent forms for pregnant participants and pregnant partners are present in the submitted documents (e.g. 'Pregnant Partner ICF', 'Pregnant Participant ICF'). No assent for minors is specified (study enrolment restricted to ages 18-70)..
- Pregnancy Exclusion
- Female subjects of child-bearing potential (WOCBP) and male subjects with WOCBP partners must adhere to contraception requirements.
- Vulnerable Population
- Vulnerable population flag selected. Participants must provide written informed consent. Dedicated informed consent forms for pregnant participants and pregnant partners are present in the submitted documents (e.g. 'Pregnant Partner ICF', 'Pregnant Participant ICF'). No assent for minors is specified (study enrolment restricted to ages 18-70).
Inclusion criteria
- {"criterion_text":"- Must have given written informed consent and be able to comply with all study requirements."}
- {"criterion_text":"- Males or females 18 to 70 years of age at the time of informed consent."}
- {"criterion_text":"- Documented diagnosis of HAE-1/HAE-2."}
- {"criterion_text":"- At least 2 HAE attacks within the 8-week run-in period, as confirmed by an investigator based on the protocol-specified definition."}
- {"criterion_text":"- Access to and ability to use ≥ 1 acute medication(s)."}
- {"criterion_text":"- Female subjects of child-bearing potential (WOCBP) and male subjects with WOCBP partners must adhere to contraception requirements."}
Exclusion criteria
- {"criterion_text":"- Any concomitant diagnosis of another form of chronic angioedema, such as acquired angioedema, HAE with normal C1-INH, idiopathic angioedema, or recurrent angioedema associated with urticaria."}
- {"criterion_text":"- History of allergic reaction to an oligonucleotide or N-acetylgalactosamine (GalNAc)."}
- {"criterion_text":"- Prior treatment with any oligonucleotides (including siRNA) within 6 months if single dose or 12 months if multiple doses prior to screening."}
- {"criterion_text":"- Exposure to another investigational drug or biological agent within 30 days or within at least 5 half-lives (whichever is longer) prior to screening."}
- {"criterion_text":"- ANY of the following abnormalities in clinical laboratory tests at screening and run-in period (within 7 days prior to dosing), a. Platelet count < LLN b. Alanine aminotransferase (ALT) or aspartate aminotransferase (AST)﹥3 × upper limit of normal (ULN) c. Total bilirubin﹥1.5 × ULN unless due to Gilbert’s syndrome d. Calculated creatinine clearance ≤ 60 mL/min (Cockcroft-Gault equation). e. APTT﹥1.5 × ULN"}
- {"criterion_text":"- Any of the following medications prior to the run-in period: a. Angiotensin-converting enzyme inhibitors (ACEIs) or any estrogen-containing medications with systemic absorption within 4 weeks. b. Any exposure to androgens within 1 week. c. Prophylactic treatment of HAE attacks: - lanadelumab within 10 weeks. - berotralstat within 3 weeks. - all other prophylactic therapies, within 1 week."}
Endpoints
Primary endpoints
- {"endpoint_text":"- Change from baseline in time normalized monthly (per 4 weeks) HAE attack rate over Days 29-169.","definition_or_measurement_approach":"Change from baseline in time-normalized monthly (per 4 weeks) HAE attack rate measured over Days 29-169."}
Secondary endpoints
- {"endpoint_text":"- Incidence and severity of SAEs and AEs during the main study period, extension study period and whole study.","definition_or_measurement_approach":"Reporting and categorisation of serious adverse events (SAEs) and adverse events (AEs) during main study, extension period and across the whole study."}
- {"endpoint_text":"- Change from baseline in timenormalized monthly (per 4 weeks) HAE attack rate to Day 169.","definition_or_measurement_approach":"Change from baseline in time-normalized monthly (per 4 weeks) HAE attack rate measured to Day 169."}
- {"endpoint_text":"- Change from baseline in time-normalized monthly (per 4 weeks) HAE attack rate during the main study period and extension period.","definition_or_measurement_approach":"Change from baseline in time-normalized monthly (per 4 weeks) HAE attack rate measured during the main study period and extension period."}
- {"endpoint_text":"- Change from baseline in plasma PKK concentration during the main study period and extension period.","definition_or_measurement_approach":"Change from baseline in plasma prekallikrein (PKK) concentration measured during the main study period and extension period."}
Recruitment
- Planned Sample Size
- 14
- Recruitment Window Months
- 35
- Consent Approach
- Written informed consent is required from each participant. Main informed consent forms (ICFs) and patient information sheets are provided in multiple languages (English patient-facing documents plus country-specific ICFs and SIS in Spanish, Italian, German, Polish). There are dedicated ICFs for pregnant participants and pregnant partners. No mention of parental consent or assent (study enrols adults 18-70).
Methods
- Spain: Recruitment arrangements and outreach materials provided (documents: K1_BW-20805-2001_ES_Recruitment arrangements; K2 outreach text, patient study guide, study flyer) — channels include outreach text and study flyers targeted at potential participants with HAE in Spain.
- Italy: Recruitment arrangements, outreach text, patient study guide and study flyer present (documents: K_BW-20805-2001_IT_Recruitment arrangements; K_BW-20805-2001_IT_Outreach Text; K_BW-20805-2001_IT_Patient Study Guide; K_BW-20805-2001_IT_StudyFlyer) — channels include outreach text, patient study guide and flyers targeted at Italian participants.
- Germany: Recruitment and informed consent procedure and outreach materials present (documents: K_BW-20805-2001_DE_Recruitment and Informed consent procedure; K_BW-20805-2001_DE_Outreach Text_German; Patient Study Guide; Study Flyer) — channels include outreach text, study flyers and center-driven recruitment materials targeted at German participants.
- Poland: Recruitment arrangements, outreach text, patient study guide and flyer present (documents: K1_BW-20805-2001_PL_Recruitment arrangements; K2_BW-20805-2001_PL_Recruitment material_Flyer; K2 outreach text; Patient Study Guide) — channels include outreach text, patient study guide and flyers targeted at Polish participants.
Geography
- Total Number Of Sites
- 8
- Total Number Of Participants
- 10
Spain
- Earliest CTIS Part Ii Submission Date
- 04-04-2025
- Latest Decision Or Authorization Date
- 24-10-2025
- Processing Time Days
- 203
- Number Of Sites
- 1
- Number Of Participants
- 2
Sites
- Site Name
- University Hospital Virgen Del Rocio S.L.
- Department Name
- Allergy Department
- Principal Investigator Name
- Stefan Cimbollek
- Principal Investigator Email
- stefan.henning.sspa@juntadeandalucia.es
- Contact Person Name
- Stefan Cimbollek
- Contact Person Email
- stefan.henning.sspa@juntadeandalucia.es
Italy
- Earliest CTIS Part Ii Submission Date
- 17-01-2025
- Latest Decision Or Authorization Date
- 23-10-2025
- Processing Time Days
- 279
- Number Of Sites
- 3
- Number Of Participants
- 3
Sites
- Site Name
- Azienda Ospedaliero Universitaria Careggi
- Department Name
- Department of Experimental and Clinical Medicine
- Principal Investigator Name
- Alessandra Vultaggio
- Principal Investigator Email
- alessandra.vultaggio@unifi.it
- Contact Person Name
- Alessandra Vultaggio
- Contact Person Email
- alessandra.vultaggio@unifi.it
- Site Name
- Azienda Ospedaliero-Universitaria Policlinico G. Rodolico-San Marco Di Catania
- Department Name
- Medicina Interna
- Principal Investigator Name
- Francesco Giardino
- Principal Investigator Email
- f.giardino@policlinico.unict.it
- Contact Person Name
- Francesco Giardino
- Contact Person Email
- f.giardino@policlinico.unict.it
- 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
- 31-03-2025
- Latest Decision Or Authorization Date
- 22-10-2025
- Processing Time Days
- 205
- Number Of Sites
- 3
- Number Of Participants
- 4
Sites
- Site Name
- Universitaetsmedizin der Johannes Gutenberg-Universitaet Mainz KöR
- Department Name
- Dermatologie
- 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
- Medizinische Hochschule Hannover
- Department Name
- Klinik für Dermatologie, Allergologie und Venerologie
- 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
- Site Name
- Charite Universitaetsmedizin Berlin KöR
- Department Name
- Institut für Allergieforschung
- Principal Investigator Name
- Markus Magerl
- Principal Investigator Email
- markus.magerl@charite.de
- Contact Person Name
- Markus Magerl
- Contact Person Email
- markus.magerl@charite.de
Poland
- Earliest CTIS Part Ii Submission Date
- 08-04-2025
- Latest Decision Or Authorization Date
- 29-10-2025
- Processing Time Days
- 204
- Number Of Sites
- 1
- Number Of Participants
- 1
Sites
- Site Name
- Samodzielny Publiczny Zaklad Opieki Zdrowotnej Szpital Uniwersytecki W Krakowie
- Department Name
- Centrum Alergologii – Poradnia Alergologiczna
- 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
Sponsor
Primary sponsor
- Full Name
- Shanghai Argo Biopharmaceutical Co. Ltd.
- Organisation Type
- Pharmaceutical company
- Country Of Registered Address
- China
Contract research organisations
- Name
- Fortrea Inc.
- Responsibilities
- [{"code":1},{"code":11},{"code":12},{"code":2},{"code":5},{"code":6},{"code":8}]
- Name
- Labcorp Pharmaceutical Research And Development (Shanghai) Co. Ltd.
- Responsibilities
- [{"code":15,"value":"bio samples storage"},{"code":4}]
Third parties
- {"country":"United States","full_name":"Labcorp Central Laboratory Services LP","duties_or_roles":"[{\"code\":15,\"value\":\"bio samples storage\"}]","organisation_type":"Pharmaceutical company"}
- {"country":"Sweden","full_name":"Viedoc Technologies AB","duties_or_roles":"[{\"code\":3},{\"code\":7}]","organisation_type":"Non-Pharmaceutical company"}
- {"country":"China","full_name":"Wuxi Apptec (Shanghai) Co. Ltd.","duties_or_roles":"[{\"code\":4}]","organisation_type":"Pharmaceutical company"}
- {"country":"Switzerland","full_name":"Labcorp Central Laboratory Services SARL","duties_or_roles":"[{\"code\":15,\"value\":\"bio sample storage\"}]","organisation_type":"Pharmaceutical company"}
- {"country":"United States","full_name":"Fortrea Inc.","duties_or_roles":"[{\"code\":1},{\"code\":11},{\"code\":12},{\"code\":2},{\"code\":5},{\"code\":6},{\"code\":8}]","organisation_type":"Pharmaceutical company"}
- {"country":"China","full_name":"Labcorp Pharmaceutical Research And Development (Shanghai) Co. Ltd.","duties_or_roles":"[{\"code\":15,\"value\":\"bio samples storage\"},{\"code\":4}]","organisation_type":"Pharmaceutical company"}
- {"country":"Belgium","full_name":"Taikun Pharmaceutical Technology","duties_or_roles":"[{\"code\":14}]","organisation_type":"Pharmaceutical company"}
Investigational products
- Investigational Product Name
- BW-20805 Injection
- Active Substance
- BW-20805
- Modality
- Oligonucleotide
- Routes Of Administration
- Subcutaneous
- Route
- Subcutaneous
- Maximum Dose
- 600 mg
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