BW-20805 for Angioedema

(HAE Trial)

Recruiting at 2 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

Trial Summary

What is the purpose of this trial?

A Phase 2 in Adult Subjects with Hereditary Angioedema

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 BW-20805 (berotralstat) safe for humans?

Berotralstat, also known as BW-20805, has been shown to be generally safe in humans, with mild to moderate stomach-related side effects being the most common. Serious side effects are rare, and it is well tolerated as a once-daily oral treatment for hereditary angioedema. ^{1 2 3 4 5}

How does the drug BW-20805 differ from other treatments for angioedema?

BW-20805 is unique because it is an oral, once-daily drug that inhibits plasma kallikrein, which helps prevent angioedema attacks by blocking the production of bradykinin, a substance that can cause swelling. This oral administration is more convenient compared to traditional injectable treatments. ^{1 3 4 6 7}

Research Team

Markus Magerl, Doctor

Principal Investigator

Charite-Universitaetsmedizin Berlin - Klinik fuer Dermatologie Venerologie und Allergologie

Eligibility Criteria

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

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

Treatment Details

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.

Participant Groups

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+

Findings from Research

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]

References

1. United States 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). [2021]

2. Spain pubmed.ncbi.nlm.nih.gov

A review of oral kallikrein inhibitor berotralstat for hereditary angioedema. [2022]

3. England pubmed.ncbi.nlm.nih.gov

Long-term safety and effectiveness of berotralstat for hereditary angioedema: The open-label APeX-S study. [2021]

4. United States 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. [2021]

5. Denmark pubmed.ncbi.nlm.nih.gov

Oral berotralstat for the prophylaxis of hereditary angioedema attacks in patients in Japan: A phase 3 randomized trial. [2021]

6. Denmark pubmed.ncbi.nlm.nih.gov

Nonallergic angioedema: role of bradykinin. [2013]

7. United States pubmed.ncbi.nlm.nih.gov

Berotralstat (BCX7353): Structure-Guided Design of a Potent, Selective, and Oral Plasma Kallikrein Inhibitor to Prevent Attacks of Hereditary Angioedema (HAE). [2021]