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

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial aims to evaluate the effectiveness of lanadelumab for individuals with acquired angioedema, a condition that causes painful and recurring swelling attacks. Participants will receive lanadelumab, a monoclonal antibody, to determine if it can reduce the frequency and severity of their attacks. The trial is open-label, so all participants will receive the treatment. It suits individuals who experience frequent angioedema attacks (at least one per month) and have no family history of the condition. Participants should not have allergies to similar treatments or uncontrolled major health issues. As a Phase 4 trial, lanadelumab has already received FDA approval and demonstrated effectiveness, offering participants the opportunity to benefit from an established treatment while contributing to broader understanding.

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 is the safety track record for lanadelumab?

A previous study found lanadelumab to be safe for people with hereditary angioedema, a condition that causes sudden swelling. Participants experienced fewer attacks and felt better control over their symptoms. Some common side effects were mild, such as redness and pain at the injection site. In the largest trial, 56% of participants experienced injection site reactions, but these were generally manageable. Overall, current research suggests that lanadelumab is well-tolerated. ^{1 2 3 4 5}

Why are researchers enthusiastic about this study treatment?

Lanadelumab is unique because it targets a specific protein called kallikrein, which is involved in the overproduction of bradykinin, the peptide responsible for causing angioedema attacks. Unlike standard treatments like C1 esterase inhibitors or icatibant, which focus on different pathways or are used to treat symptoms as they occur, lanadelumab is designed to prevent attacks from happening in the first place. Researchers are excited about lanadelumab because it offers a proactive approach to managing angioedema, potentially providing patients with fewer attacks and a more consistent quality of life.

What is the effectiveness track record for lanadelumab in treating angioedema?

Studies have shown that lanadelumab effectively reduces the number and severity of hereditary angioedema (HAE) attacks. In a key study, patients experienced a significant drop in attack rates, with some reducing from about 5.8 attacks to just 0.4 attacks per month. Another study found that new users experienced a decrease in attacks from an average of 1.57 to fewer than one per month. This evidence suggests that lanadelumab can effectively help manage symptoms in people with angioedema. In this trial, all participants will receive open-label lanadelumab to further evaluate its effectiveness. ^{3 5 6 7 8}

Who Is on the Research Team?

Jonathan Bernstein, MD

Principal Investigator

5139314181

Are You a Good Fit for This Trial?

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 for a Trial Participant

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

What Are the Treatments Tested in This Trial?

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.

How Is the Trial Designed?

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+

Published Research Related to This Trial

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]

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 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]

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]

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]

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]

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 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, 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]

Citations

1. pubmed.ncbi.nlm.nih.gov pubmed.ncbi.nlm.nih.gov/39701274/

Real-World Effectiveness of Lanadelumab in Hereditary ... This real-world study demonstrates that lanadelumab long-term prophylaxis is effective in improving AFR in patients with type I/II HAE on every 2-week ...

2. jaci-inpractice.org jaci-inpractice.org/article/S2213-2198(24)01250-9/fulltext

Real-World Effectiveness of Lanadelumab in Hereditary ... In pivotal clinical trials, lanadelumab has been shown to significantly reduce the frequency and severity of hereditary angioedema attacks and ...

3. annallergy.org annallergy.org/article/S1081-1206(25)00393-X/fulltext

Lanadelumab's impact on hereditary angioedema control ... Mean HAE attack rates on lanadelumab treatment by subgroup decreased by 93% (from 5.8 to 0.4 attacks/mo) in the very high disease activity subgroup, by 75% ( ...

4. jacionline.org jacionline.org/article/S0091-6749(23)02268-6/fulltext

Final Data From the EMPOWER Study In new lanadelumab users, adjusted model-estimated mean (95% CI) HAE attack rate (attacks/month) decreased from 1.57 (0.88-2.81) pre-lanadelumab treatment to ...

5. jmcp.org jmcp.org/doi/10.18553/jmcp.2019.25.2.143

The Effectiveness and Value of Lanadelumab and C1 ... The primary outcome was investigator-confirmed HAE attack. Prophylactic treatment with Takhyzro significantly decreased the frequency of HAE ...

6. pubmed.ncbi.nlm.nih.gov pubmed.ncbi.nlm.nih.gov/40504359/

Sustained Effectiveness, Tolerability, and Safety of Long ... Real-world data from EMPOWER showed marked HAE attack rate reduction up to 36 months after initiating lanadelumab in patients newly treated with lanadelumab.

7. jacionline.org jacionline.org/article/S0091-6749(22)02067-X/fulltext

24-Month Data From the EMPOWER Study Real-world findings from EMPOWER show HAE attack rate reduction, improved QoL, and improved angioedema control after lanadelumab initiation in new lanadelumab ...

8. takhzyro.com takhzyro.com/hcp/safety-profile/help-study

Help Study: Safety Data for TAKHZYRO® (lanadelumab-flyo) Safety profile established in the largest pivotal trial prevention study in HAE ; Injection site reactions · Pain; Erythema; Bruising. 56%. 52%. 7%. 4%. 45%. 31%.