A study to investigate the effect and safety of BW-20805 in adult participants with hereditary angioedema

Overview

Sponsor-declared trial summary

Key facts

Sponsor

Shanghai Argo Biopharmaceutical Co. Ltd.

Participant type

Patients

Age range

18-64 years, 65+ years

Gender

Male and Female

Therapeutic area

Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]

Trial duration

15 May 2025 → ongoing

Decision date (initial)

2025-04-07

Transition trial

No

Low-intervention

No

Rare-disease indication

Yes

Vulnerable population

Yes

Trial design

CTIS Part I — objectives, methods, condition coding

Main objective

Scope: Prophylaxis, Safety, Others, Pharmacokinetic

To determine the prophylactic effect of BW20805 in HAE attack rate.

Secondary objectives 3

1. To evaluate the safety and tolerability of multiple doses of BW-20805.
2. To further evaluate the effect of BW-20805 in subjects with HAE
3. To assess the prekallikrein (PKK) concentration of multiple doses of BW 20805 in subjects with HAE

Conditions and MedDRA coding

Hereditary Angioedema

Eligibility criteria

Principal inclusion / exclusion criteria as submitted by sponsor

Inclusion criteria 6

1. Must have given written informed consent and be able to comply with all study requirements.
2. Males or females 18 to 70 years of age at the time of informed consent.
3. Documented diagnosis of HAE-1/HAE-2.
4. At least 2 HAE attacks within the 8-week run-in period, as confirmed by an investigator based on the protocol-specified definition.
5. Access to and ability to use ≥ 1 acute medication(s).
6. Female subjects of child-bearing potential (WOCBP) and male subjects with WOCBP partners must adhere to contraception requirements.

Exclusion criteria 6

1. 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.
2. History of allergic reaction to an oligonucleotide or N-acetylgalactosamine (GalNAc).
3. Prior treatment with any oligonucleotides (including siRNA) within 6 months if single dose or 12 months if multiple doses prior to screening.
4. Exposure to another investigational drug or biological agent within 30 days or within at least 5 half-lives (whichever is longer) prior to screening.
5. 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
6. 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 and secondary outcome measures (English text)

Primary endpoints 1

1. Change from baseline in time normalized monthly (per 4 weeks) HAE attack rate over Days 29-169.

Secondary endpoints 4

1. Incidence and severity of SAEs and AEs during the main study period, extension study period and whole study.
2. Change from baseline in timenormalized monthly (per 4 weeks) HAE attack rate to Day 169.
3. Change from baseline in time-normalized monthly (per 4 weeks) HAE attack rate during the main study period and extension period.
4. Change from baseline in plasma PKK concentration during the main study period and extension period.

Investigational products

Investigational medicinal products (IMPs), comparators, placebo, auxiliary

Test 1

Sponsors and contacts

Sponsor organisations, regulatory contacts, third parties

Shanghai Argo Biopharmaceutical Co. Ltd.

Sponsor organisation

Shanghai Argo Biopharmaceutical Co. Ltd.

Address

J2026 Room 1 203, No. 337 Shahe Road, Jiangqiao Town, Jiading District No. 337 Shahe Road Jiangqiao Town

City

Shanghai

Postcode

201803

Country

China

Scientific contact point

Organisation

Shanghai Argo Biopharmaceutical Co. Ltd.

Contact name

pengcheng shao

Public contact point

Organisation

Shanghai Argo Biopharmaceutical Co. Ltd.

Contact name

pengcheng shao

Third parties 7

Locations

4 EU/EEA countries · 8 investigational sites

By country

Investigational sites

Country notifications

Trial-start, recruitment-start, end and early-termination notifications submitted per Member State

Results and documents

Annex IV summary of results, Annex V layperson summary, and all documents registered in CTIS for this trial

Documents 55 files

Public protocol annexes, IB summaries, regulatory submissions and post-authorisation documents registered in CTIS.

Application history

3 submissions — initial application plus substantial / non-substantial modifications