Open-Label Safety, PK, and Efficacy Trial of Sebetralstat (KVD900) in Pediatric Patients (Ages 2-11) With HAE Type I or II

Inclusion criteria

• Male or female patients 2 to 11 years of age.
• Confirmed diagnosis of HAE Type I or II.
• For patients ≥20 kg at screening, patient has had at least 1 documented HAE attack in the last year prior to screening.
• Caregiver, as assessed by the Investigator, must be able to appropriately store and administer IMP and be able to read, understand, and complete the diary.
• Investigator believes that the patient and caregiver are willing and able to adhere to all protocol requirements.
• Parent or Legally Authorized Representative (LAR) provides signed informed consent and patient provides assent (when applicable).

Exclusion criteria

• Any concomitant diagnosis of another form of chronic angioedema, such as acquired C1 inhibitor deficiency, HAE with normal C1-INH, idiopathic angioedema, or angioedema associated with urticaria.
• A clinically significant history of poor response to bradykinin receptor 2 blocker, C1-INH therapy, or plasma kallikrein inhibitor therapy for the management of HAE, in the opinion of the Investigator.
• Patient weighs <9.5 kg.
• Use of angiotensin-converting enzyme inhibitors after the Screening Visit.
• 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 or moderate CYP3A4 inducers.
• 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.
• Known hypersensitivity to sebetralstat or to any of the excipients.
• Participation in any interventional investigational clinical trial within 4 weeks of the last dosing of investigational drug prior to the Screening Visit.