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

JRCT ID: jRCT2031240670

Registered date:12/02/2025

Basic Information

• Recruitment status: Recruiting
• Health condition(s) or Problem(s) studied: Hereditary Angioedema
• Date of first enrollment: 03/02/2025
• Target sample size: 24
• Countries of recruitment: United States,Japan,Canada,Japan,France,Japan,Germany,Japan,Italy,Japan,Israel,Japan
• Study type: Interventional
• Intervention(s): Each hereditary angioedema attack is treated with up to three oral doses of Sebetralstat 75 mg (75 mgx1 tablet), 150 mg (75 mgx2 tablets), or 300 mg (300 mgx1 tablet) based on the weight of the patient.

Outcome(s)

• Primary Outcome: To evaluate the safety and tolerability of sebetralstat in pediatric patients aged 2 to <12 years of age with HAE Type I or II.

Secondary Outcome

- To evaluate the pharmacokinetics (PK) of sebetralstat in pediatric patients with HAE Type I or II.
- To evaluate the clinical efficacy of sebetralstat for the on-demand treatment of HAE attacks in pediatric patients with HAE Type I or II.

Key inclusion & exclusion criteria

• Age minimum: >= 2age old
• Age maximum: < 12age old
• Gender: Both

Include criteria

1) Male or female patients 2 to 11 years of age.
2) Confirmed diagnosis of HAE Type I or II
3) Patient has had at least 1 documented HAE attack in the last year prior to screening.
4) 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.
5) Investigator believes that the patient and caregiver are willing and able to adhere to all protocol requirements.
6) Parent or LAR provides signed informed consent and patient provides assent (when applicable).

Exclude criteria

1) 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.
2) 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.
3) Patient weighs <9.5 kg.
4) Use of angiotensin-converting enzyme inhibitors after the Screening Visit.
5) 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.
6) Patients who require sustained use of strong cytochrome P450 3A4 (CYP3A4) inhibitors or inducers or moderate CYP3A4 inducers.
7) 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.
8) Pregnant or breastfeeding.
9) Known hypersensitivity to sebetralstat or to any of the excipients.
10) Participation in any interventional investigational clinical trial within 4 weeks of the last dosing of investigational drug prior to the Screening Visit.

Related Information

• Primary Sponsor: Smith Michael
• Secondary ID(s): NCT06467084