KVD824 for Long-Term Prophylactic Treatment of HAE Types I/II Attacks
Research type
Research Study
Full title
A Randomized, Double-Blind, Placebo-Controlled, Phase 2 Trial to Evaluate the Efficacy and Safety of Three Dose Levels of KVD824, an Oral Plasma Kallikrein Inhibitor, for Long-Term Prophylactic Treatment of Hereditary Angioedema Type I or II
IRAS ID
299456
Contact name
Sorena Kiani-Alikhan
Contact email
Sponsor organisation
Kalvista Pharmaceuticals Ltd
Eudract number
2021-000136-59
Duration of Study in the UK
1 years, 3 months, 1 days
Research summary
Hereditary angioedema (HAE) is a rare and potentially life-threatening genetic disease affecting 1 to 2 people per 100,000 people worldwide. It is caused by a deficiency in the C1-esterase inhibitor protein and is characterised by unpredictable tissue swelling due to an increase in vascular permeability (the capacity of a blood vessel wall to allow small molecules to pass through).
There can be triggers for HAE attacks; however, most HAE attacks are unpredictable and occur without an obvious trigger.
Current treatment of HAE involves the inhibition of plasma kallikrein activation, which is a major cause of HAE attacks. The study drug, KVD824, is a potent and selective small-molecule inhibitor of plasma kallikrein, and when administered orally has achieved sustained dose-dependent blockade of plasma kallikrein activity.
KVD824 was evaluated in two Phase 1 studies. KVD824 achieved adequate plasma concentrations with sustained plasma kallikrein suppression over 12 to 14 hour intervals.
Sponsor is KalVista Pharmaceuticals.
This study is to evaluate the efficacy and safety of KVD824 for long-term prophylactic treatment of HAE (type I or II). Approximately 48 patients will be enrolled into the study globally. Participants will be randomly assigned (like flipping a coin) to KVD824 (300, 600 or 900 mg) modified release tablet or placebo (an inactive substance which resembles the study drug but have no medical value) taken twice daily. Patients and investigators will not know what dose patients have been assigned to but will know the number of tablets taken daily. Treatment will last up to 12 weeks.
Participants will be asked to attend approximately 5 visits over a total duration of up to 30 weeks. Study procedures will include physical examinations, vital signs, Electrocardiogram, blood samples, and the completion of an eDiary for dosing, attacks, and quality of life questionnaires.REC name
London - Chelsea Research Ethics Committee
REC reference
21/LO/0517
Date of REC Opinion
21 Sep 2021
REC opinion
Further Information Favourable Opinion