Clinical trial NCT06467084: Open-Label Safety, PK, and Efficacy Trial of Sebetralstat (KVD900) in Pediatric Patients (Ages 2-11) With HAE Type I or II
Official title: Open-Label Safety, Pharmacokinetic, and Efficacy Trial of Sebetralstat (KVD900) in Pediatric Patients (Ages 2-11) With Hereditary Angioedema Type I or II
Study type: INTERVENTIONAL
Overall status: RECRUITING
Phase: PHASE3
Conditions: Hereditary Angioedema
Keywords: KONFIDENT-KID
Lead sponsor: KalVista Pharmaceuticals, Ltd.
Sponsor class: INDUSTRY
First posted: 2024-06-20
Enrollment: 36
Sex: ALL
Minimum age (years): 2
Maximum age (years): 11
Standard ages: CHILD
Accepts healthy volunteers: no
Allocation: NON_RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

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


Eligibility criteria: Inclusion Criteria:

1. Male or female patients 2 to 11 years of age.
2. Confirmed diagnosis of HAE Type I or II.
3. For patients ≥20 kg at screening, 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 Legally Authorized Representative (LAR) provides signed informed consent and patient provides assent (when applicable).

Exclusion 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. Known hypersensitivity to sebetralstat or to any of the excipients.
9. Participation in any interventional investigational clinical trial within 4 weeks of the last dosing of investigational drug prior to the Screening Visit.

Primary outcomes:
measure: The proportion of pediatric patients with HAE Types I or II who take any sebetralstat dose, who experience any AE(s) (including fatal AEs) during the study, irrespective of uses of other medications and sebetralstat discontinuations for any reason.
timeFrame: Throughout the duration of the trial, up to 1 year.
Secondary outcomes:
measure: Caregiver Global Impression of Change (CaGI-C): Time to beginning of symptom relief defined as at least "a little better" (2 time points in a row)
timeFrame: Within 12 hours of the first IMP administration.
measure: Caregiver Global Impression of Severity (CaGI-S): Time to first incidence of decrease from baseline (2 time points in a row)
timeFrame: Within 12 hours of the first IMP administration.
measure: CaGI-S: Time to HAE attack resolution defined as "none"
timeFrame: Within 24 hours of the first IMP administration
Other outcomes:
Locations:
city: Birmingham
country: United States
facility: KalVista Investigative Site
state: Alabama
city: Scottsdale
country: United States
facility: KalVista Investigative Site
state: Arizona
city: San Diego
country: United States
facility: KalVista Investigative Site
state: California
city: Santa Monica
country: United States
facility: KalVista Investigative Site
state: California
city: Evansville
country: United States
facility: KalVista Investigative Site
state: Indiana
city: Wheaton
country: United States
facility: KalVista Investigative Site
state: Maryland
city: Saint Louis
country: United States
facility: KalVista Investigative Site
state: Missouri
city: Toledo
country: United States
facility: KalVista Investigative Site
state: Ohio
city: Hershey
country: United States
facility: KalVista Investigative Site
state: Pennsylvania
city: Dallas
country: United States
facility: KalVista Investigative Site
state: Texas
city: Edmonton
country: Canada
facility: KalVista Investigative Site
state: Alberta
city: Lille
country: France
facility: KalVista Investigative Site
city: Marseille
country: France
facility: KalVista Investigative Site
city: Paris
country: France
facility: KalVista Investigative Site
city: Frankfurt am main
country: Germany
facility: KalVista Investigative Site
city: Frankfurt am main
country: Germany
facility: KalVista Investigative Site
city: Haifa
country: Israel
facility: KalVista Investigative Site
city: Petah tikva
country: Israel
facility: KalVista Investigative Site
city: Tel Aviv
country: Israel
facility: KalVista Investigative Site
city: Milan
country: Italy
facility: KalVista Investigative Site
city: Padova
country: Italy
facility: KalVista Investigative Site
city: Rome
country: Italy
facility: KalVista Investigative Site
city: Kawagoe
country: Japan
facility: KalVista Investigative Site
city: Tokyo
country: Japan
facility: KalVista Investigative Site
Study officials:
affiliation: KalVista Pharmaceuticals, Ltd.
name: Study Director
role: STUDY_DIRECTOR
References: