A Phase 2 in Adult Subjects With Hereditary Angioedema

Phase 2

Not yet recruiting

• Conditions: Hereditary Angioedema (HAE)
• Interventions: Drug: BW-20805

• Registration Number: NCT06846398
• Lead Sponsor: Shanghai Argo Biopharmaceutical Co., Ltd.

Brief Summary

A Phase 2 in Adult Subjects with Hereditary Angioedema

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 Timeline

• Status Verified: 2025-02
• Study First Submitted: 2025-02-13
• Study First Submitted QC: 2025-02-25
• Last Update Submitted: 2025-02-25
• Study First Posted: 2025-02-26
• Last Update Posted: 2025-02-26
• Study Start: 2025-02-28
• Primary Completion: 2026-06-30
• Study Completion: 2028-03-30

Recruitment & Eligibility

• Status: NOT_YET_RECRUITING
• Sex: All
• Target Recruitment: 24

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 & Design

• Study Type: INTERVENTIONAL
• Study Design: PARALLEL

Arm && Interventions

Group	Intervention	Description
Cohort 1	BW-20805	BW-20805 600 mg Q24W\*2
Cohort 2	BW-20805	BW-20805 300 mg Q24W\*2
Cohort 3	BW-20805	BW-20805 300 mg Q12W\*2

Primary Outcome Measures

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

Secondary Outcome Measures

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

Trial Locations

Locations (3)

Penn State Milton S. Hershey MC - Penn State; 🇺🇸 Hershey, Pennsylvania, United States

Peking Union Medical College Hospital; 🇨🇳 Beijing, Beijing, China

Charité - Universitätsmedizin Berlin; 🇩🇪 Berlin, Germany