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

Phase 3

Recruiting

• Conditions: Hereditary Angioedema
• Interventions: Drug: KVD900 75 mg; Drug: KVD900 150 mg; Drug: KVD900 300 mg

• Registration Number: NCT06467084
• Lead Sponsor: KalVista Pharmaceuticals, Ltd.

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.

Detailed Description

Not available

Study Timeline

• Study First Submitted: 2024-06-14
• Study First Submitted QC: 2024-06-19
• Study First Posted: 2024-06-20
• Study Start: 2024-06-24
• Status Verified: 2024-12
• Last Update Submitted: 2024-12-20
• Last Update Posted: 2024-12-27
• Primary Completion: 2027-08-31
• Study Completion: 2027-08-31

Recruitment & Eligibility

• Status: RECRUITING
• Sex: All
• Target Recruitment: 24

Inclusion 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 Legally Authorized Representative (LAR) provides signed informed consent and patient provides assent (when applicable).

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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.

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Study & Design

• Study Type: INTERVENTIONAL
• Study Design: PARALLEL

Arm && Interventions

Group	Intervention	Description
75 mg Dose Group	KVD900 75 mg	Patients will take a single 75 mg dose of KVD900.
150 mg Dose Group	KVD900 150 mg	Patients will take a single 150 mg dose of KVD900 dose.
300 mg Dose Group	KVD900 300 mg	Patients will take a single 300 mg dose of KVD900.

Primary Outcome Measures

Name	Time	Method
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.	Throughout the duration of the trial, up to 1 year.

Secondary Outcome Measures

Name	Time	Method
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)	Within 12 hours of the first IMP administration.
Caregiver Global Impression of Severity (CaGI-S): Time to first incidence of decrease from baseline (2 time points in a row)	Within 12 hours of the first IMP administration.
CaGI-S: Time to HAE attack resolution defined as "none"	Within 24 hours of the first IMP administration

Trial Locations

Locations (1)

KalVista Investigative Site; 🇮🇹 Rome, Italy