A Phase 2 in Adult Subjects With Hereditary Angioedema (HAE)

A Phase 2, Open-label, Multicenter Study to Assess the Efficacy and Safety of BW-20805 in Adult Subjects With Hereditary Angioedema

A Phase 2 in Adult Subjects with Hereditary Angioedema

Study Overview

• Status: Active, not recruiting
• Conditions: Hereditary Angioedema (HAE)
• Intervention / Treatment: Drug: BW-20805; Drug: BW-20805; Drug: BW-20805

Detailed Description

A Phase 2, Open-label, Multicenter Study to Assess the Efficacy and Safety of BW-20805 in Adult Subjects with Hereditary Angioedema

• Study Type: Interventional
• Enrollment (Actual): 25
• Phase: Phase 2

Contacts and Locations

Study Locations

• China
  • Beijing Municipality
    • Beijing, Beijing Municipality, China, 100730
      • Peking Union Medical College Hospital
  • Gansu
    • Lanzhou, Gansu, China, 730000
      • The First Hospital of Lanzhou University
  • Hubei
    • Wuhan, Hubei, China, 130030
      • Tongji Hospital, Tongji Medical College of Huazhong University of Science and Technology
  • Shandong
    • Yantai, Shandong, China, 264000
      • Yantai Yuhuangding Hospital
  • Sichaun
    • Chengdu, Sichaun, China, 610041
      • West China Hospital, Sichuan University
  • Yunnan
    • Kunming, Yunnan, China, 650032
      • First Affiliated Hospital of Kunming Medical University
• Germany
  • Rhineland-Palatinate
    • Mainz, Rhineland-Palatinate, Germany, 55101
      • Universitätsmedizin der Johannes Gutenberg
  • State of Berlin
    • Berlin, State of Berlin, Germany, 12200
      • Charité - Universitätsmedizin Berlin
• Italy
  • Catania
    • Catania, Catania, Italy, 95125
      • Azienda Ospedaliero Universitaria Policlinico G. Rodolico San Marco - Ospedale San Marco
  • Florence
    • Florence, Florence, Italy, 50134
      • Azienda Ospedaliero-Universitaria Careggi
  • PD
    • Padova, PD, Italy, 35128
      • Azienda Ospedale - Universita di Padova
• Poland
  • Lesser Poland Voivodeship
    • Krakow, Lesser Poland Voivodeship, Poland, 31-066
      • SP ZOZ Szpital Uniwersytecki w Krakowie
• Spain
  • Sevilla
    • Seville, Sevilla, Spain, 41013
      • Hospital Universitario Virgen del Rocio
• United States
  • Maryland
    • Silver Spring, Maryland, United States, 20902
      • Institute for Asthma and Allergy - Wheaton
  • Missouri
    • St Louis, Missouri, United States, 63141
      • Washington University School of Medicine
  • North Carolina
    • Durham, North Carolina, United States, 27705
      • Duke University Medicine Center
  • Pennsylvania
    • Hershey, Pennsylvania, United States, 17033
      • Penn State Milton S. Hershey MC - Penn State
  • Virginia
    • Falls Church, Virginia, United States, 22031
      • Inova Clinical Trials and Research Center

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Adult; Older Adult
• Accepts Healthy Volunteers: No

Description

Inclusion Criteria:

• Must have given written informed consent and be able to comply with all study requirements.
• Males or females 18 to 70 years of age at the time of informed consent.
• Documented diagnosis of HAE-1/HAE-2.
• At least 2 HAE attacks within the 8-week run-in period, as confirmed by an investigator based on the protocol-specified definition
• Access to and ability to use ≥ 1 acute medication(s)
• Female subjects must be non-pregnant;non-lactating, and either surgically sterile
• Male subjects with WOCBP partners, dual contraception is required if no surgically sterile

Exclusion Criteria:

• Any clinically significant medical or psychiatric condition or medical history that, in the opinion of the Investigator, makes the subject unsuitable for participation in the study
• 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
• History or presence of carcinoma within 5 years prior to screening, except for basal or squamous cell carcinoma of the skin or carcinoma in situ of the cervix that has been successfully treated.
• Have undergone major surgery within 3 months prior to screening.
• History of clinically significant arterial or venous thrombosis, or current clinically significant prothrombotic or bleeding risk
• History of allergic reaction to an oligonucleotide or N-acetylgalactosamine
• Prior treatment with any oligonucleotides within 6 months if single dose or 12 months if multiple doses prior to screening.
• Exposure to another investigational drug or biological agent within 30 days or within at least 5 half-lives prior to screening.
• With ANY of the abnormalities in clinical laboratory tests at screening and run-in period
• Clinically significant findings on 12-lead electrocardiogram that would place the patient at risk or interfere with participation in the study at screening.
• Positive for Human immunodeficiency virus (HIV), HBsAg, Hepatitis C or syphilis infection
• Active infection requiring systemic antiviral or antimicrobial therapy that will not be completed prior to dosing.

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: Randomized
• Interventional Model: Parallel Assignment
• Masking: None (Open Label)

Number of Arms

3

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: Cohort 1; BW-20805 600 mg Q24W*2	Drug: BW-20805; SC administrations of BW-20805 600 mg on Day1 to Day673; Drug: BW-20805; SC administrations of BW-20805 300 mg every 24 weeks on Day1 to Day169;Day337 to Day673; Drug: BW-20805; SC administrations of BW-20805 300 mg every 12 weeks on Day 1 to Day 589
Experimental: Cohort 2; BW-20805 300 mg Q24W*2	Drug: BW-20805; SC administrations of BW-20805 600 mg on Day1 to Day673; Drug: BW-20805; SC administrations of BW-20805 300 mg every 24 weeks on Day1 to Day169;Day337 to Day673; Drug: BW-20805; SC administrations of BW-20805 300 mg every 12 weeks on Day 1 to Day 589
Experimental: Cohort 3; BW-20805 300 mg Q12W*2	Drug: BW-20805; SC administrations of BW-20805 600 mg on Day1 to Day673; Drug: BW-20805; SC administrations of BW-20805 300 mg every 24 weeks on Day1 to Day169;Day337 to Day673; Drug: BW-20805; SC administrations of BW-20805 300 mg every 12 weeks on Day 1 to Day 589

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
To determine the prophylactic effect of in HAE attack rate.	Change in time-normalized monthly (per 4 weeks) HAE attack rate from baseline to Day169	from baseline to Day169

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
To evaluate the safety and tolerability of multiple doses of BW-20805.	Incidence and severity of SAEs and AEs up to 96 weeks ( the main study period, extension study period and whole study)	up to 96 weeks

Collaborators and Investigators

• Sponsor: Shanghai Argo Biopharmaceutical Co., Ltd.
• Investigators: Principal Investigator: Markus Magerl, Doctor, Charite-Universitaetsmedizin Berlin - Klinik fuer Dermatologie Venerologie und Allergologie

Study record dates

Study Major Dates

• Study Start (Actual): February 28, 2025
• Primary Completion (Estimated): June 30, 2026
• Study Completion (Estimated): March 30, 2028

Study Registration Dates

• First Submitted: February 13, 2025
• First Submitted That Met QC Criteria: February 25, 2025
• First Posted (Actual): February 26, 2025

Study Record Updates

• Last Update Posted (Actual): April 17, 2026
• Last Update Submitted That Met QC Criteria: April 16, 2026
• Last Verified: March 1, 2026

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Hereditary Complement Deficiency Diseases; Primary Immunodeficiency Diseases; Vascular Diseases; Cardiovascular Diseases; Genetic Diseases, Inborn; Immune System Diseases; Hypersensitivity, Immediate; Hypersensitivity; Immunologic Deficiency Syndromes; Skin Diseases; Urticaria; Skin Diseases, Vascular; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Skin and Connective Tissue Diseases; Angioedema; Angioedemas, Hereditary
• Other Study ID Numbers: BW-20805-2001

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: Yes
• Studies a U.S. FDA-regulated device product: No
• product manufactured in and exported from the U.S.: No