Sebetralstat for Hereditary Angioedema

(KONFIDENT-KID Trial)

Recruiting in Palo Alto (17 mi)

+20 other locations

Age: < 18

Sex: Any

Travel: May Be Covered

Time Reimbursement: Varies

Trial Phase: Phase 3

Recruiting

Sponsor: KalVista Pharmaceuticals, Ltd.

Must not be taking: ACE inhibitors, Estrogens, CYP3A4 modulators

Disqualifiers: Chronic angioedema, Poor drug response, others

No Placebo Group

Pivotal Trial (Near Approval)

Prior Safety Data

Approved in 6 Jurisdictions

Trial Summary

What is the purpose of this trial?

KVD900-303 is an open-label, multicenter clinical trial in patients aged 2 to 11 years old with HAE Type I or II.

Will I have to stop taking my current medications?

The trial requires stopping certain medications, like angiotensin-converting enzyme inhibitors and estrogen-containing medications, before the screening visit. If you're taking strong or moderate CYP3A4 inhibitors or inducers, you may also need to stop those. It's best to discuss your current medications with the trial team.

What data supports the effectiveness of the drug Sebetralstat for hereditary angioedema?

Sebetralstat, an oral plasma kallikrein inhibitor, showed promising results in a phase 2 trial for treating hereditary angioedema attacks, indicating it may effectively reduce swelling episodes. Similar drugs like berotralstat, also a kallikrein inhibitor, have been effective in preventing hereditary angioedema attacks, suggesting a potential benefit of Sebetralstat.

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Eligibility Criteria

This trial is for children aged 2-11 with Hereditary Angioedema (HAE) Types I or II who've had at least one HAE attack in the past year. A parent or guardian must consent, and caregivers need to be able to manage treatment and follow the study's diary requirements.

Inclusion Criteria

I am between 2 and 11 years old.

I have been diagnosed with HAE Type I or II.

My caregiver can store, give me my medication, and fill out the treatment diary.

Exclusion Criteria

I have been diagnosed with another type of chronic swelling.

I haven't been in a drug trial for the last 4 weeks.

I have HAE and treatments like bradykinin blockers didn't work well for me.

Trial Timeline

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive a single dose of KVD900 at 75 mg, 150 mg, or 300 mg

1 day

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

Participant Groups

The trial tests three doses of Sebetralstat (KVD900): 75 mg, 150 mg, and 300 mg. It's an open-label study where everyone knows what treatment they're getting, aiming to assess safety, how the body processes the drug, and its effectiveness against HAE.

Group I: 75 mg Dose Group Experimental Treatment 1 Intervention

Patients will take a single 75 mg dose of KVD900.

Group II: 300 mg Dose Group Experimental Treatment 1 Intervention

Patients will take a single 300 mg dose of KVD900.

Group III: 150 mg Dose Group Experimental Treatment 1 Intervention

Patients will take a single 150 mg dose of KVD900 dose.

Sebetralstat (KVD900) is already approved in European Union, United States, Switzerland, Australia, United Kingdom for the following indications:

🇪🇺 Approved in European Union as Sebetralstat for:

• Hereditary angioedema (HAE) Type I or II

🇺🇸 Approved in United States as Sebetralstat for:

• Hereditary angioedema (HAE) Type I or II

🇨🇭 Approved in Switzerland as Sebetralstat for:

• Hereditary angioedema (HAE) Type I or II

🇦🇺 Approved in Australia as Sebetralstat for:

• Hereditary angioedema (HAE) Type I or II

🇬🇧 Approved in United Kingdom as Sebetralstat for:

• Hereditary angioedema (HAE) Type I or II

Find a Clinic Near You

Research Locations Nearby Select from list below to view details:

KalVista Investigative Site Birmingham, AL

KalVista Investigative Site Scottsdale, AZ

KalVista Investigative Site San Diego, CA

KalVista Investigative Site Santa Monica, CA

More Trial Locations

Who Is Running the Clinical Trial?

KalVista Pharmaceuticals, Ltd. Lead Sponsor

References

1. England pubmed.ncbi.nlm.nih.gov

An investigational oral plasma kallikrein inhibitor for on-demand treatment of hereditary angioedema: a two-part, randomised, double-blind, placebo-controlled, crossover phase 2 trial. [2023] Guidelines recommend effective on-demand therapy for all individuals with hereditary angioedema. We aimed to assess the novel oral plasma kallikrein inhibitor, sebetralstat, which is in development, for on-demand treatment of hereditary angioedema attacks.

2. England pubmed.ncbi.nlm.nih.gov

Evaluation of patient-reported outcome measures for on-demand treatment of hereditary angioedema attacks and design of KONFIDENT, a phase 3 trial of sebetralstat. [2023] Hereditary angioedema (HAE) with C1-inhibitor deficiency (HAE-C1-INH) is characterized by recurrent, debilitating episodes of swelling. Sebetralstat, an investigational oral plasma kallikrein inhibitor, demonstrated promising efficacy for on-demand treatment of HAE-C1-INH in a phase 2 trial. We describe the multipronged approach informing the design of KONFIDENT, a phase 3 randomized, placebo-controlled, three-way crossover trial evaluating the efficacy and safety of sebetralstat in patients aged ≥12 years with HAE-C1-INH.

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

Hereditary angioedema outcomes in US patients switched from injectable long-term prophylactic medication to oral berotralstat. [2023] Berotralstat, a first-line, once-daily oral plasma kallikrein inhibitor for long-term prophylaxis of hereditary angioedema (HAE), is an effective and well-tolerated treatment option.

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] Berotralstat (BCX7353) is an oral, once-daily inhibitor of plasma kallikrein in development for the prophylaxis of hereditary angioedema (HAE) attacks.

5. 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] Berotralstat (BCX7353) is a recently approved, oral, once-daily kallikrein inhibitor for hereditary angioedema (HAE) prophylaxis. In the APeX-2 trial, berotralstat reduced HAE attack rates over 24 weeks, with a favorable safety and tolerability profile.