A long-term study to evaluate if KVD900 is safe and effective intreating attacks in patients with Hereditary Angioedema.

Phase 1

• Conditions: Hereditary Angioedema Type I or II; MedDRA version: 21.0Level: LLTClassification code 10080956Term: Hereditary angioedema type ISystem Organ Class: 10010331 - Congenital, familial and genetic disorders; MedDRA version: 26.0Level: LLTClassification code 10080960Term: Hereditary angioedema type IISystem Organ Class: 10010331 - Congenital, familial and genetic disorders; Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15]

• Registration Number: EUCTR2021-001176-42-AT
• Lead Sponsor: KalVista Pharmaceuticals Ltd

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Study Start: 2022-07-20
• Last Update Posted: 15 April 2024

Recruitment & Eligibility

• Status: ot Recruiting
• Sex: All
• Target Recruitment: 150

Inclusion Criteria

Patients may roll over from KVD900-301.

1) Confirmed diagnosis of HAE type I or II at any time in the medical history
2) Patient has had at least 2 documented HAE attacks within 3 months prior to the Enrollment Visit.
3) If a patient is receiving long-term prophylactic treatment with one of the protocol-allowed therapies, they must have been on a stable dose and regimen for at least 3 months prior to the Enrollment Visit.
4) Male or female patients 12 years of age and older.
5) Patients must meet the contraception requirements.
6) Patients must be able to swallow trial tablets whole.
7) Patients, as assessed by the Investigator, must be able to appropriately receive and store IMP, and be able to read, understand, and complete the eDiary.
8) Investigator believes that the patient is willing and able to adhere to all protocol requirements.
9) Patient provides signed informed consent or assent (when applicable). A parent or LAR must also provide signed informed consent when required.
Are the trial subjects under 18? yes
Number of subjects for this age range: 12
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 134
F.1.3 Elderly (>=65 years) yes
F.1.3.1 Number of subjects for this age range 4

Exclusion Criteria

1) Discontinued from the KVD900-301 trial for reasons of non-compliance, withdrawal of consent, or safety.
2) Presence of any safety concerns that would preclude participation in the open-label trial as determined by the investigator.
3) Any concomitant diagnosis of another form of chronic angioedema, such as acquired C1 inhibitor deficiency, HAE with normal C1-INH (previously known as HAE type III), idiopathic angioedema, or angioedema associated with urticaria.
4) A clinically significant history of poor response to bradykinin receptor 2 (BR2) blocker, C1-INH therapy, or plasma kallikrein inhibitor therapy for the management of HAE, in the opinion of the Investigator.
5) Use of attenuated androgens (e.g., stanozolol, danazol, oxandrolone, methyltestosterone, testosterone), or anti-fibrinolytics (e.g., tranexamic acid) within 28 days prior to the Enrollment Visit.
6) Use of ACE inhibitors within 7 days prior to the Enrollment Visit.
7) Any estrogen-containing medications with systemic absorption (such as oral contraceptives including ethinylestradiol or hormonal replacement therapy) within 7 days prior to the Enrollment Visit.
8) Inadequate organ function, including but not limited to:
a) Alanine aminotransferase (ALT) >2x ULN
b) Aspartate aminotransferase (AST) >2x ULN
c) Bilirubin direct >1.25x ULN
d) INR >1.2
e) Clinically significant hepatic impairment defined as a Child-Pugh B or C
9) 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.
10) History of substance abuse or dependence that would interfere with the completion of the trial, as determined by the Investigator.
11) Known hypersensitivity to KVD900 or to any of the excipients.
12) Participation in any gene therapy treatment or trial for HAE.
13) Participation in any interventional investigational clinical trial, including an investigational COVID-19 vaccine trial, within 4 weeks of the last dosing of investigational drug prior to the Enrollment Visit.
14) Any pregnant or breastfeeding patient.

Study & Design

• Study Type: Interventional clinical trial of medicinal product
• Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
Main Objective: To assess the safety of long-term administration of KVD900 in adolescent and adult patients with HAE type I or II. ;Secondary Objective: - To assess the long-term efficacy of KVD900 in the treatment of attacks in adolescent and adult patients with HAE type I or II.<br><br>- To assess the safety and efficacy of KVD900 when used as short-term prophylaxis in adolescent and adult patients with HAE types I or II. ;Primary end point(s): The proportion of patients with at least one AE in adolescent and adult patients with HAE type I or II who have taken at least one dose of IMP. ;Timepoint(s) of evaluation of this end point: AE's are assessed throughout the trial.

Secondary Outcome Measures

Name	Time	Method
Secondary end point(s): PGI-C: time to beginning of symptom relief defined as at least '' a little better'' (2 time points in a row) within 12 hours of initial dose of IMP administration. <br><br>PGI-S: time to first incidence of 2 time points in a row decrease from baseline within 12 hours of initial dose of IMP administration. <br><br>PGI-S: time to HAE attack resolution, defined as ''none'' within 24 hours of initial dose of IMP administration. ;Timepoint(s) of evaluation of this end point: - Within 12 hours of initial dose of IMP administration. <br><br>- Within 12 hours of initial dose of IMP administration. <br><br>- Within 24 hours of initial dose of IMP administration.