Lanadelumab for Angioedema

Overseen By Jonathan Bernstein, MD

Age: 18+

Sex: Any

Trial Phase: Phase 4

Sponsor: Bernstein Clinical Research Center

Disqualifiers: Anaphylaxis, Uncontrolled systemic disease, Pregnancy, 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: Unknown

Duration: 12 months

5 Participants Needed

Trial Summary

What is the purpose of this trial?

use of lanadelumab in patients with acquired 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. It's best to discuss this with the trial coordinators or your doctor.

What data supports the effectiveness of the drug Lanadelumab for treating angioedema?

Research shows that Lanadelumab is effective in preventing attacks of hereditary angioedema (HAE), with 77% of patients being attack-free in a study compared to 3% with a placebo. It also improved the quality of life for patients over a long-term period. ^{1 2 3 4 5}

Is lanadelumab safe for humans?

Lanadelumab is generally well tolerated in humans, with the most common side effects being mild and temporary reactions at the injection site. It has a low potential for causing immune reactions and is considered a safe option for preventing hereditary angioedema attacks in patients aged 12 and older. ^{1 3 6 7 8}

How is the drug Lanadelumab different from other treatments for angioedema?

Lanadelumab is unique because it is a fully human monoclonal antibody that targets and inhibits plasma kallikrein, which helps prevent hereditary angioedema attacks. Unlike some other treatments, it is administered subcutaneously (under the skin) and is designed for long-term prevention rather than on-demand treatment. ^{1 3 5 8 9}

Research Team

Jonathan Bernstein, MD

Principal Investigator

5139314181

Eligibility Criteria

This trial is for patients with acquired angioedema who experience at least one attack per month and have specific lab findings like decreased C1INH, C4, and C1q levels. It's not open to pregnant or breastfeeding women, those with hereditary angioedema types 1 or 2, people allergic to biologics, or anyone with uncontrolled systemic diseases.

Inclusion Criteria

I had at least one attack per month before starting TAA.

I have AAE with recurrent attacks, no skin hives, and specific blood test findings.

Exclusion Criteria

I am not pregnant or breastfeeding.

I have Hereditary Angioedema Type 1 or 2.

History of anaphylaxis or hypersensitivity to biologics

Timeline

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive open-label lanadelumab for prophylaxis of acquired angioedema

12 months

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

Treatment Details

Interventions

• Lanadelumab

Trial Overview The study tests Lanadelumab (300 mg), a medication intended for long-term prevention of swelling attacks in acquired angioedema. Participants will receive this treatment to see how effective it is in reducing the frequency of their symptoms.

Participant Groups

Group I: open-label administration Experimental Treatment 1 Intervention

all subjects receive open-label lanadelumab

Who Is Running the Clinical Trial?

Bernstein Clinical Research Center

Lead Sponsor

Trials

4

Recruited

90+

Findings from Research

Lanadelumab significantly reduced the monthly attack rate of hereditary angioedema (HAE) from the first dose, with rates of 0.41-0.76 compared to 2.04 for placebo, indicating its strong efficacy in preventing attacks.

The treatment was well-tolerated, with similar rates of adverse events during the initial and later phases of the study, suggesting that lanadelumab is a safe long-term option for HAE management.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Lanadelumab demonstrates rapid and sustained prevention of hereditary angioedema attacks. Riedl, MA., Maurer, M., Bernstein, JA., et al.[2021]

Long-term treatment with lanadelumab significantly improved health-related quality of life (HRQoL) in patients with hereditary angioedema (HAE), with nearly half of rollover patients achieving a clinically meaningful improvement in their quality of life scores.

A high percentage of patients reported controlled disease and excellent treatment response, indicating that lanadelumab not only reduces attack rates but also enhances overall patient satisfaction and productivity.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Long-term lanadelumab treatment improves health-related quality of life in patients with hereditary angioedema. Lumry, WR., Maurer, M., Weller, K., et al.[2023]

In a study involving 212 patients with hereditary angioedema, lanadelumab significantly reduced the mean attack rate by 87.4%, with patients being attack-free for an average of 97.7% of the treatment days.

The treatment was well-tolerated, with most adverse events being mild and resolving quickly; no serious treatment-related adverse events or deaths were reported, indicating a favorable safety profile for long-term use.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Long-term prevention of hereditary angioedema attacks with lanadelumab: The HELP OLE Study. Banerji, A., Bernstein, JA., Johnston, DT., et al.[2022]

Lanadelumab is an effective and safe treatment for long-term prophylaxis of hereditary angioedema (HAE), with a significant reduction in the number of attacks observed in all 9 patients studied.

Both standard (300 mg every 2 weeks) and extended (300 mg every 4 weeks) dosing regimens were equally effective, with 56% of patients achieving complete remission from attacks, indicating flexibility in treatment options.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Using an extended treatment regimen of lanadelumab in the prophylaxis of hereditary angioedema: a single-centre experience. Abuzakouk, M., Ghorab, O., Al-Hameli, H., et al.[2022]

In a controlled study, 77% of patients receiving 300 mg of lanadelumab every 2 weeks were attack-free for the last 16 weeks, compared to only 3% in the placebo group, demonstrating its efficacy in preventing hereditary angioedema (HAE) attacks.

The most common side effects were mild injection-site reactions, indicating that lanadelumab is generally safe and could shift treatment from on-demand to a preventive approach for HAE.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Lanadelumab to treat hereditary angioedema. Wedi, B.[2019]

Lanadelumab (Takhzyro™) is an effective monoclonal antibody that significantly reduces the frequency of hereditary angioedema (HAE) attacks in patients aged 12 and older, as demonstrated in the pivotal HELP trial.

The treatment is generally well tolerated, with mild and transient injection-site reactions being the most common side effects, and it offers the convenience of self-administration every two weeks.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Lanadelumab: A Review in Hereditary Angioedema. Syed, YY.[2020]

Lanadelumab, a monoclonal antibody, effectively prevents attacks of hereditary angioedema (HAE) by inhibiting the formation of bradykinin, with a significant reduction in attack rates observed in a study of 257 subjects, including 233 patients with HAE.

The pharmacokinetics of lanadelumab show a long half-life of 14.8 days and a dosing regimen of 300 mg every two weeks leads to effective suppression of cHMWK formation, demonstrating its efficacy across different body weight groups.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Pharmacokinetics, Pharmacodynamics, and Exposure-Response of Lanadelumab for Hereditary Angioedema. Wang, Y., Marier, JF., Kassir, N., et al.[2021]

In a study of 34 patients with hereditary angioedema, switching to lanadelumab resulted in significant improvement in disease control, with angioedema control test scores increasing from 7.5 to 14.9, indicating a shift from poorly controlled to well-controlled disease.

The study demonstrated that patients could gradually extend their injection intervals from 30 to 32.5 days without losing efficacy, suggesting that lanadelumab can effectively minimize treatment burden while maintaining control of symptoms.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Lanadelumab Efficacy, Safety, and Injection Interval Extension in HAE: A Real-Life Study. Buttgereit, T., Vera, C., Weller, K., et al.[2022]

Lanadelumab (Takhzyro™) is a fully human monoclonal antibody that effectively inhibits plasma kallikrein, which is responsible for causing hereditary angioedema (HAE) attacks due to excessive bradykinin production.

In a phase III trial involving patients aged 12 years and older with type I or II HAE, lanadelumab significantly reduced the frequency of HAE attacks compared to placebo, leading to its recent approval in the USA for HAE prevention.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Lanadelumab: First Global Approval. Syed, YY.[2021]

References

1. Denmark pubmed.ncbi.nlm.nih.gov

Lanadelumab demonstrates rapid and sustained prevention of hereditary angioedema attacks. [2021]

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

Long-term lanadelumab treatment improves health-related quality of life in patients with hereditary angioedema. [2023]

3. Denmark pubmed.ncbi.nlm.nih.gov

Long-term prevention of hereditary angioedema attacks with lanadelumab: The HELP OLE Study. [2022]

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

Using an extended treatment regimen of lanadelumab in the prophylaxis of hereditary angioedema: a single-centre experience. [2022]

5. Spain pubmed.ncbi.nlm.nih.gov

Lanadelumab to treat hereditary angioedema. [2019]

6. New Zealand pubmed.ncbi.nlm.nih.gov

Lanadelumab: A Review in Hereditary Angioedema. [2020]

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

Pharmacokinetics, Pharmacodynamics, and Exposure-Response of Lanadelumab for Hereditary Angioedema. [2021]

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

Lanadelumab Efficacy, Safety, and Injection Interval Extension in HAE: A Real-Life Study. [2022]

9. New Zealand pubmed.ncbi.nlm.nih.gov

Lanadelumab: First Global Approval. [2021]