BW-20805 for Angioedema

(HAE Trial)

Recruiting at 25 trial locations

Overseen By Ying Zhang, Master

Age: 18+

Sex: Any

Trial Phase: Phase 2

Sponsor: Shanghai Argo Biopharmaceutical Co., Ltd.

Must not be taking: Oligonucleotides, Investigational drugs

Disqualifiers: Chronic angioedema, Carcinoma, Thrombosis, others

No Placebo Group All trial participants will receive the active study treatment (no placebo)

Prior Safety Data This treatment has passed at least one previous human trial

Compensation: Varies

Number of Visits: 12

Duration: 24 weeks

24 Participants Needed

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial explores a new treatment called BW-20805 (experimental treatment) for individuals with hereditary angioedema, a condition causing sudden swelling in various body parts. The trial investigates different dosages and schedules of the medication to determine the most effective option. It involves three groups, each receiving a different amount or timing of the medication. Suitable participants have experienced at least two angioedema attacks in the past two months and have a confirmed diagnosis of hereditary angioedema. As a Phase 2 trial, this research focuses on measuring the treatment's effectiveness in an initial, smaller group, offering participants a chance to contribute to significant advancements in treatment.

Will I have to stop taking my current medications?

The trial information does not specify if you need to stop taking your current medications. However, it mentions that you need access to at least one acute medication, so you might be able to continue some treatments. It's best to discuss your specific medications with the trial team.

Is there any evidence suggesting that BW-20805 is likely to be safe for humans?

Research has shown that BW-20805 is safe for people. Studies found that this treatment was well-tolerated, with no serious side effects, no dropouts due to side effects, and no reported deaths. Previous patients experienced no major issues that would raise concerns about the safety of BW-20805. ^{1 2 3 4 5}

Why do researchers think this study treatment might be promising for angioedema?

BW-20805 is unique because it offers a new approach to treating angioedema, which is commonly managed with treatments like C1 inhibitors, bradykinin receptor antagonists, and antihistamines. Unlike these standard options, BW-20805 is being administered in varying doses and intervals, potentially offering a more tailored approach to managing the condition. Researchers are particularly excited about its novel dosing schedule—600 mg every 24 weeks or 300 mg every 12 or 24 weeks—allowing for potentially longer-lasting effects and fewer administrations. This could mean a significant improvement in convenience and quality of life for patients dealing with angioedema.

What evidence suggests that BW-20805 might be an effective treatment for angioedema?

Research shows that BW-20805, a new treatment being tested in this trial, is designed to block a protein called prekallikrein (PKK), which plays a major role in hereditary angioedema (HAE). In earlier studies, BW-20805 lowered PKK levels for up to 24 weeks. Lowering PKK is important because high levels are linked to HAE symptoms. Early results suggest this treatment could help prevent swelling attacks in HAE patients by addressing the root cause. While more information is needed, these findings offer promising evidence for its potential to manage hereditary angioedema. ^{1 3 6 7 8}

Who Is on the Research Team?

Markus Magerl, Doctor

Principal Investigator

Charite-Universitaetsmedizin Berlin - Klinik fuer Dermatologie Venerologie und Allergologie

Are You a Good Fit for This Trial?

This trial is for adults with Hereditary Angioedema, a condition causing sudden swelling in various parts of the body. Specific eligibility criteria are not provided, but typically participants must meet certain health standards and may be required to have a confirmed diagnosis of the condition.

Inclusion Criteria

I am a man and will use birth control with my partner who can have children.

Must have given written informed consent and be able to comply with all study requirements

At least 2 HAE attacks within the 8-week run-in period, as confirmed by an investigator based on the protocol-specified definition

Exclusion Criteria

I haven't taken any oligonucleotide drugs in the last 6 to 12 months.

With ANY of the abnormalities in clinical laboratory tests at screening and run-in period

I haven't taken any experimental drugs within the last 30 days.

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive BW-20805 at varying doses depending on cohort assignment

24 weeks

Visits every 12 or 24 weeks depending on cohort

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

Extension

Participants may continue to receive BW-20805 to evaluate long-term safety and tolerability

up to 96 weeks

What Are the Treatments Tested in This Trial?

Interventions

• BW-20805

Trial Overview The trial is testing BW-20805, which is likely a new treatment or medication for Hereditary Angioedema. As it's a Phase 2 trial, it focuses on evaluating the effectiveness and safety of this intervention in people who have the condition.

How Is the Trial Designed?

Group I: Cohort 3 Experimental Treatment 1 Intervention

BW-20805 300 mg Q12W\*2

Group II: Cohort 2 Experimental Treatment 1 Intervention

BW-20805 300 mg Q24W\*2

Group III: Cohort 1 Experimental Treatment 1 Intervention

BW-20805 600 mg Q24W\*2

Who Is Running the Clinical Trial?

Shanghai Argo Biopharmaceutical Co., Ltd.

Lead Sponsor

Trials

2

Recruited

160+

Published Research Related to This Trial

In a 48-week phase 3 trial involving 108 patients with hereditary angioedema, berotralstat significantly reduced the monthly attack rates from an average of 3.06 to 1.06 for the 150 mg dose, demonstrating its effectiveness in HAE prophylaxis.

The treatment was well-tolerated, with most adverse events being mild or moderate, and no serious drug-related side effects reported, indicating a favorable safety profile for long-term use.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Randomized Trial of the Efficacy and Safety of Berotralstat (BCX7353) as an Oral Prophylactic Therapy for Hereditary Angioedema: Results of APeX-2 Through 48 Weeks (Part 2). Wedner, HJ., Aygören-Pürsün, E., Bernstein, J., et al.[2021]

Orladeyo (berotralstat) is an effective once-daily oral medication for preventing hereditary angioedema (HAE) attacks, showing a 67% reduction in attacks in the ongoing phase III APeX-2 trial.

The drug has demonstrated a favorable safety profile with mostly mild to moderate gastrointestinal side effects and minimal serious adverse effects, making it a convenient alternative to traditional subcutaneous or intravenous therapies.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

A review of oral kallikrein inhibitor berotralstat for hereditary angioedema. Gao, Y., Hwang, J., Hwang, G., et al.[2022]

In a 24-week phase 3 trial involving 121 patients with hereditary angioedema (HAE), berotralstat significantly reduced the frequency of HAE attacks, with a reduction to 1.31 attacks per month at the 150 mg dose compared to 2.35 attacks per month in the placebo group.

Berotralstat was found to be safe and well tolerated, with no serious drug-related adverse events reported, although some patients experienced mild side effects like abdominal pain and diarrhea.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Oral once-daily berotralstat for the prevention of hereditary angioedema attacks: A randomized, double-blind, placebo-controlled phase 3 trial. Zuraw, B., Lumry, WR., Johnston, DT., et al.[2021]

Citations

1. argobiopharma.com argobiopharma.com/news/156.html

Argo Biopharma to Present Positive Phase I Results of ... BW-20805 is an investigational siRNA therapy that targets and inhibits prekallikrein (PKK), a well-validated target for hereditary angioedema ( ...

2. clinicaltrials.gov clinicaltrials.gov/study/NCT06846398

Study Details | NCT06846398 | A Phase 2 in Adult Subjects ... ... Efficacy and Safety of BW-20805 in Adult Subjects With Hereditary Angioedema. Conditions. Hereditary Angioedema (HAE). Hereditary Angioedema (HAE). Hereditary ...

3. sciencedirect.com sciencedirect.com/science/article/pii/S108112062500537X

r094 hereditary angioedema prophylaxis with prekallikrein ... The Phase 1 study results provide the evidence that BW-20805 is safe and significantly reduces PKK levels in a dose-dependent manner for 24 weeks. This ...

4. clinicaltrials.eu clinicaltrials.eu/inn/bw-20805/

BW-20805: A New Treatment for Hereditary Angioedema ... This Phase 2, open-label, multicenter clinical trial aims to evaluate the efficacy and safety of BW-20805 in adult patients with HAE. The study explores ...

5. synapse.patsnap.com synapse.patsnap.com/drug/394cb5cb4eef49c5b89bea62933bd99e

BW-20805 - Drug Targets, Indications, Patents A randomized, double-blind, placebo-controlled phase I safety, tolerability, pharmacokinetics, and pharmacodynamics study of BW-20805 as a single dose in ...

6. annallergy.org annallergy.org/article/S1081-1206(25)00537-X/fulltext

HEREDITARY ANGIOEDEMA PROPHYLAXIS WITH ... BW-20805 was safe and well tolerated, with no serious adverse events (SAEs) related to study drug, no AE leading to discontinuation, no deaths were reported.

7. rarediseaseadvisor.com rarediseaseadvisor.com/reports/bw20805-sustains-prekallikrein-suppression-phase-1-hae-trial/

BW-20805 Sustains Prekallikrein Suppression in Phase 1 ... BW-20805 produced a dose-dependent reduction in plasma prekallikrein levels sustained for 24 weeks, supporting potential HAE prevention.

8. anzctr.org.au anzctr.org.au/Trial/Registration/TrialReview.aspx?ACTRN=12623000689673

actrn12623000689673 The primary outcomes are to evaluate the safety and tolerability of a single dose of BW-20805 in healthy subject. ... The safety laboratory data will be ...