KVD900 for Hereditary Angioedema
Recruiting in Palo Alto (17 mi)
+52 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: Anti-coagulants, Anti-platelets
Disqualifiers: Bleeding disorders
No Placebo Group
Pivotal Trial (Near Approval)
Prior Safety Data
Approved in 2 jurisdictions
Trial Summary
What is the purpose of this trial?This trial is testing a medication called KVD900 in teenagers with a rare condition that causes sudden swelling. The goal is to see how the medication behaves in their bodies and if it can help manage their symptoms.
Will I have to stop taking my current medications?
The trial does not specify if you need to stop taking your current medications, but you cannot participate if you are taking any anti-coagulant or anti-platelet agents.
What data supports the effectiveness of the drug KVD900 for hereditary angioedema?
Research shows that KVD900, an oral plasma kallikrein inhibitor, has promising potential as an on-demand treatment for hereditary angioedema, as it helps control the swelling and pain associated with the condition.
12345Is KVD900 (sebetralstat) safe for humans?
Sebetralstat, also known as KVD900, is being tested for safety in a phase 3 trial for hereditary angioedema, and earlier trials have shown promising safety results. While specific safety data for KVD900 is limited, similar drugs like berotralstat have shown mild to moderate side effects, mostly related to the stomach, with minimal serious issues reported.
15678Eligibility Criteria
Adolescents aged 12-17 with Hereditary Angioedema (HAE) types I or II can join this trial if they're already in the KVD900-302 study. They must be willing to provide samples, complete forms and diaries, and have consent from a legal representative.Inclusion Criteria
I am willing to provide samples, complete forms, and follow the study's requirements.
I am between 12 and 17 years old.
Patient is currently participating in KVD900-302
+1 more
Exclusion Criteria
I have a bleeding disorder or am taking blood-thinning medication.
Trial Timeline
Screening
Participants are screened for eligibility to participate in the trial
2-4 weeks
Treatment
Participants receive KVD900 to investigate its pharmacokinetic profile
6 weeks
Follow-up
Participants are monitored for safety and effectiveness after treatment
4 weeks
Participant Groups
The trial is testing how the body processes KVD900, a drug for HAE. It's specifically looking at its pharmacokinetics—how the drug is absorbed, distributed, metabolized, and excreted—in adolescents.
2Treatment groups
Experimental Treatment
Group I: KVD900 600 mgExperimental Treatment1 Intervention
Group II: Experimental: KVD900 300 mgExperimental Treatment1 Intervention
KVD900 is already approved in European Union, United States for the following indications:
🇪🇺 Approved in European Union as sebetralstat for:
- Hereditary Angioedema (HAE)
🇺🇸 Approved in United States as sebetralstat for:
- Hereditary Angioedema (HAE)
Find a Clinic Near You
Research Locations NearbySelect from list below to view details:
KalVista Investigative SiteLittle Rock, AR
KalVista Investigative SiteChevy Chase, MD
KalVista Investigative SiteScottsdale, AZ
KalVista Investigative SiteOverland Park, KS
More Trial Locations
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Who Is Running the Clinical Trial?
KalVista Pharmaceuticals, Ltd.Lead Sponsor
References
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.
Hereditary angioedema with normal C1 inhibitor in a French cohort: Clinical characteristics and response to treatment with icatibant. [2018]The clinical characteristics and icatibant-treatment outcomes of patients with hereditary angioedema with normal C1 inhibitor (HAE-nC1 INH) are limited.
Social costs of icatibant self-administration vs. health professional-administration in the treatment of hereditary angioedema in Spain. [2021]Icatibant is the only subcutaneous treatment for acute Type I and Type II hereditary angioedema with C1-esterase inhibitor deficiency (HAE-C1-INH) licensed for self-administration in Europe.
Pharmacological suppression of the kallikrein kinin system with KVD900: An orally available plasma kallikrein inhibitor for the on-demand treatment of hereditary angioedema. [2022]Hereditary angioedema (HAE) is a rare genetic disease that leads to recurrent episodes of swelling and pain caused by uncontrolled plasma kallikrein (PKa) activity. Current guidelines recommend ready availability of on-demand HAE treatments that can be administered early upon attack onset. This report describes the pharmacological and pharmacodynamic properties of the novel oral small-molecule PKa inhibitor KVD900 as a potential on-demand treatment for HAE.
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.
A review of oral kallikrein inhibitor berotralstat for hereditary angioedema. [2022]Orladeyo, a once-daily oral formulation of berotralstat (formerly BCX-7353), is a novel oral small-molecule drug developed by BioCryst Pharmaceuticals for the prevention of hereditary angioedema (HAE) attacks. It was first approved by the U.S. Food and Drug Administration (FDA) in 2020, and in 2021 also gained approval for marketing in Japan and the European Union. Preclinical and phase I studies showed promising efficacy and safety, and several multicenter international Angioedema Prophylaxis (APeX) phase II and III trials have since been initiated to further evaluate berotralstat. The ongoing phase III APeX-2 trial showed a 67% reduction in HAE attacks at the standard 150-mg dosing. Mild to moderate gastrointestinal side effects are most commonly seen and minimal serious adverse effects have been reported. Other first-line therapies for HAE prophylaxis rely on burdensome subcutaneous or intravenous routes. Thus far berotralstat has shown to be effective and well tolerated for HAE prophylaxis with the convenience of once-daily oral dosing.
An evidence-based review of the potential role of icatibant in the treatment of acute attacks in hereditary angioedema type I and II. [2021]Icatibant, a first-in-class B2 bradykinin receptor antagonist, appears to have a favorable efficacy and safety profile for the treatment of acute attacks of hereditary angioedema in adults.
A Retrospective Analysis of Long-Term Prophylaxis with Berotralstat in Patients with Hereditary Angioedema and Acquired C1-Inhibitor Deficiency-Real-World Data. [2023]Hereditary angioedema (HAE) and acquired C1-inhibitor deficiency (AAE-C1-INH) are orphan diseases. Berotralstat is a recently licensed long-term prophylaxis (LTP) and the first oral therapy for HAE patients. No approved therapies exist for AAE-C1-INH patients. This study is the first to report real-world clinical data of patients with AAE-C1-INH and HAE who received Berotralstat. All patients treated with Berotralstat were included in this retrospective, bi-centric study. Data was collected from patients' attack calendars and the angioedema quality of life (AE-QoL) and angioedema control test (AECT) questionnaires before treatment, and at 3, 6, and 12 months after treatment and was then analyzed. Twelve patients were included, 3 patients with AAE-C1-INH, 7 patients with HAE type I, and 2 patients with HAE-nC1-INH. One patient (HAE I) quit treatment. Berotralstat was associated with fewer attacks in all groups. After 6 months of treatment, a median decrease of attacks per month was noted for HAE type I patients (3.3 to 1.5) and AAE-C1-INH patients (2.3 to 1.0). No aerodigestive attacks were noted for AAE-C1-INH patients. For HAE-nC1-INH patients, a mean decrease from 3.8 to 1.0 was noted (3 months). For HAE I patients, the total AE-QoL lowered a mean of 24.1 points after 6 months, for HAE-nC1-HAE patients 8.0 points, and for AAE-C1-INH patients 13.7 points. AECT scores increased for HAE I patients (mean: 7.1), HAE-nC1-INH patients (9.0), and AAE-C1-INH patients (4.2) after 6 months. Patients with HAE, HAE-nC1-INH, and AAE-C1-INH treated with Berotralstat showed reduced angioedema attacks and improved AE-QoL and AECT scores.