a phase iii, crossover trial evaluating the efficacy and safety of kvd900 (sebetralstat) for on-demand treatment of angioedema attacks in adolescent and adult patients with hereditary angioedema (hae)

Conditions: Hereditary Angioedema

Summary: this study is a randomized, double-blind, placebo-controlled, phase iii, three-way crossover clinical trial evaluating the efficacy and safety of kvd900, in the treatment of hereditary angioedema attacks in adolescent and adult patients


Eligibility criteria:
inclusion criteria: * male or female patients 12 years of age and older. * confirmed diagnosis of hae type i or ii at any time in the medical history. * patient has access to and ability to use conventional on-demand treatment for hae attacks. * if a patient is receiving long-term prophylactic treatment with one of the protocol-allowed therapies, they must be on a stable dose and regimen for at least 3 months prior to the screening visit (except for danazol, which requires a stable dose and regimen for 6 months prior to the screening visit). patient must be willing to remain on a stable dose and regimen for the duration of the trial. * patient's last dose of attenuated androgens other than danazol was at least 28 days prior to randomization. * patient: 1. has had at least 2 documented hae attacks within 3 months prior to screening or randomization; or 2. is a completer of the kvd824-201 trial within 3 months prior to randomization and meets all other entry criteria to enroll in kvd900-301 * patients must meet the contraception requirements. * patients must be able to swallow trial tablets whole. * patients, as assessed by the investigator, must be able to appropriately receive and store imp, and be able to read, understand, and complete the electronic diary (ediary). * investigator believes that the patient is willing and able to adhere to all protocol requirements. * patient provides signed informed consent or assent (when applicable). a parent or legally authorized representative (lar) must also provide signed informed consent when required. exclusion criteria: * any concomitant diagnosis of another form of chronic angioedema, such as acquired c1-inhibitor deficiency, hae with normal c1-inh (previously known as hae type iii), idiopathic angioedema, or angioedema associated with urticaria. * a clinically significant history of poor response to bradykinin receptor 2 (br2) blocker, c1-inh therapy or plasma kallikrein inhibitor therapy for the management of hae, in the opinion of the investigator. * use of angiotensin-converting enzyme (ace) inhibitors after the screening visit or within 7 days prior to randomization. * any estrogen containing medications with systemic absorption (such as oral contraceptives including ethinylestradiol or hormonal replacement therapy) within 7 days prior to the screening visit. * patients who require sustained use of strong cytochrome p450 3a4 (cyp3a4) inhibitors or inducers. * inadequate organ function, including but not limited to: 1. alanine aminotransferase (alt) \>2x upper limit of normal (uln) 2. aspartate aminotransferase (ast) \>2x uln 3. bilirubin direct \>1.25x uln 4. international normalized ratio (inr) \>1.2 5. clinically significant hepatic impairment defined as a child-pugh b or c * any clinically significant comorbidity or systemic dysfunction, which in the opinion of the investigator, would jeopardize the safety of the patient by participating in the trial. * history of substance abuse or dependence that would interfere with the completion of the trial, as determined by the investigator. * known hypersensitivity to kvd900 or placebo or to any of the excipients. * prior participation in trial kvd900-201. * participation in any gene therapy treatment or trial for hae. * participation in any interventional investigational clinical trial (with the exception of kvd824-201), including an investigational covid-19 vaccine trial, within 4 weeks of the last dosing of investigational drug prior to screening. * any pregnant or breastfeeding patient.

Keywords: konfident sebetralstat