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

NCT06467084 | Completed Phase 3 Results FDA Drug

• 1 other identifier: KVD900-303
• Study Type: interventional
• Target: 36
• Locations: 7 countries
• Sites: 24

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.

Conditions

Hereditary Angioedema

Keywords

KONFIDENT-KID

Outcome Measures

Primary Outcomes (1)

• Number of Participants With Treatment-emergent Adverse Events (TEAEs)

  A TEAE was defined as an adverse event (AE) that met any of the following conditions: (1) began on or after the first dose of IMP, (2) began before the first dose of IMP and increased in severity on or after first dose, (3) was completely missing a start date and the stop date, (4) was completely missing a start date and the stop date was on or after the first dose of IMP. An on-treatment TEAE was defined as any TEAE occurring within 3 days of IMP administration. A treatment-related TEAE was an AE considered related to the IMP by the investigator. A serious AE was defined as any untoward medical occurrence that at any dose resulted in death, was life-threatening, required in-patient hospitalization or prolongation, resulted in significant disability/incapacity, was a congenital anomaly/birth defect, or was an important medical event (based upon medical and scientific judgment).

  From first dose of IMP until the Final/Early Termination (ET) Visit, up to a maximum of 54 weeks.

Secondary Outcomes (1)

• Plasma Concentrations of Sebetralstat

  At 0.5 hours (±5 min), 2 hours (±15 min), and 4 hours (±15 min) post-dose. PK assessments for sebetralstat 300 mg ED were conducted at the Enrollment Visit, and for 600 mg ED at the Dose Increase Visit (up to 12.6 months after Enrollment Visit).

Study Arms (3)

150 mg Dose Group

OTHER

Patients will take a single 150 mg dose of KVD900.

Drug: KVD900 150 mg

300 mg Dose Group

OTHER

Patients will take a single 300 mg dose of KVD900.

Drug: KVD900 300 mg

600 mg Dose Group

OTHER

Patients will take a single 600 mg dose of KVD900.

Drug: KVD900 600 mg

Interventions

KVD900 150 mg DRUG

KVD900 Tablet 150 mg (2 x 75 mg)

150 mg Dose Group

KVD900 300 mg DRUG

KVD900 Tablet 300 mg (1 x 300 mg)

300 mg Dose Group

KVD900 600 mg DRUG

KVD900 Tablet 600 mg (2 x 300 mg)

600 mg Dose Group

Eligibility Criteria

• Age: 2 Years - 11 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17)

You may qualify if:

• 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).

You may not qualify if:

• 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.

Sponsors & Collaborators

• KalVista Pharmaceuticals, Ltd.: lead

Study Sites (24)

KalVista Investigative Site

Birmingham, Alabama, 35209, United States

Location

KalVista Investigative Site

Scottsdale, Arizona, 85251, United States

Location

KalVista Investigative Site

San Diego, California, 92123, United States

Location

KalVista Investigative Site

Santa Monica, California, 90404, United States

Location

KalVista Investigative Site

Evansville, Indiana, 47715, United States

Location

KalVista Investigative Site

Wheaton, Maryland, 20902, United States

Location

KalVista Investigative Site

St Louis, Missouri, 63141, United States

Location

KalVista Investigative Site

Toledo, Ohio, 43560, United States

Location

KalVista Investigative Site

Hershey, Pennsylvania, 17011, United States

Location

KalVista Investigative Site

Dallas, Texas, 75231, United States

Location

KalVista Investigative Site

Edmonton, Alberta, T6G 2B7, Canada

Location

KalVista Investigative Site

Lille, 59000, France

Location

KalVista Investigative Site

Marseille, 13005, France

Location

KalVista Investigative Site

Paris, 75012, France

Location

KalVista Investigative Site

Frankfurt am Main, 60590, Germany

Location

KalVista Investigative Site

Frankfurt am Main, 60596, Germany

Location

KalVista Investigative Site

Haifa, 31048, Israel

Location

KalVista Investigative Site

Petah Tikva, 4920235, Israel

Location

KalVista Investigative Site

Tel Aviv, 6423906, Israel

Location

KalVista Investigative Site

Milan, 20097, Italy

Location

KalVista Investigative Site

Padova, 35128, Italy

Location

KalVista Investigative Site

Rome, 00133, Italy

Location

KalVista Investigative Site

Kawagoe, 350-8550, Japan

Location

KalVista Investigative Site

Tokyo, 113-8431, Japan

Location

MeSH Terms

Conditions

Angioedemas, Hereditary

Interventions

sebetralstat

Condition Hierarchy (Ancestors)

Angioedema; Vascular Diseases; Cardiovascular Diseases; Hereditary Complement Deficiency Diseases; Primary Immunodeficiency Diseases; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Urticaria; Skin Diseases, Vascular; Skin Diseases; Skin and Connective Tissue Diseases; Hypersensitivity, Immediate; Hypersensitivity; Immune System Diseases; Immunologic Deficiency Syndromes

Results Point of Contact

• Title: Vice President Clinical
• Organization: KalVista Pharmaceuticals Ltd

clinicalstudies@kalvista.com

1 (857) 999-0075

Study Officials

• Study Director

  KalVista Pharmaceuticals, Ltd.

  STUDY DIRECTOR

Publication Agreements

• PI is Sponsor Employee: No
• Restrictive Agreement: Yes

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: NON RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: June 14, 2024
• First Posted: June 20, 2024
• Study Start: August 1, 2024
• Primary Completion: January 15, 2026
• Study Completion: January 15, 2026
• Last Updated: August 5, 2026
• Results First Posted: August 5, 2026

Record last verified: 2026-01

Data Sharing

• IPD Sharing: Will not share

Locations

US (10); JP (2); DE (2); IT (3); CA (1); FR (3); IL (3)