RAPIDe-1
Research type
Research Study
Full title
A Phase II, double-blind, placebo-controlled, Randomized, cross-over, dose-ranging study of oral PHA-022121 for Acute treatment of angioedema attacks in Patients with hereditary angioedema due to C1-Inhibitor Deficiency type I and II (RAPIDe-1)
IRAS ID
291420
Contact name
Sorena Kiani-Alikhan
Contact email
Sponsor organisation
Pharvaris Netherlands BV
Eudract number
2020-003445-11
Clinicaltrials.gov Identifier
Duration of Study in the UK
1 years, 2 months, 0 days
Research summary
Hereditary Angioedema is estimated to affect between 1 in 10,000 and 1 in 150,000 individuals. The disease manifests as oedema attacks most commonly in the limbs, face, intestinal tract, urogenital region, and airways. Acute episodes of HAE often occur without warning and may be precipitated by triggers such as minor trauma, oestrogens, dental procedures, pregnancy and stress. Attacks often lead to discomfort, pain, nausea, in cases of intestinal involvement, and limitations in activities of life. Attacks can become life-threatening in case of airway obstruction and asphyxiation if the attack remains untreated.
There are currently two treatment approaches to the management of HAE: acute (on-demand) treatment of attacks and prevention of attacks with short or long-term prophylactic therapy. Acute attacks can still occur on some patients on prophylactic treatment and in patients who are not on prophylaxis as their attacks are not sufficiently frequent to indicate prophylaxis. Subcutaneous icatibant is the only available B2 receptor antagonist indicated for acute attack treatment of HAE type I/II, and is currently marketed and recommended as a first-line treatment option. However, this medication requires subcutaneous injections, and is associated with pain at the injection site and local side effects. The other treatment option for acute attacks are C1-Inhibitor replacement therapy that requires obtaining intravenous access and in most cases emergency department attendance. Therefore, there is a strong unmet medical need for an efficacious orally bioavailable drug that could expand patient access to on demand.
This study aims to evaluate the efficacy of three oral doses (low, medium, and high) of PHA-022121 versus placebo to achieve angioedema symptom relief in adult patients with HAE type I/II. Approximately 54 participants with HAE will take part at 30 study centres globally. Participation will last approximately 30 weeks and with around 6 visits to the study site.
REC name
East of England - Cambridge South Research Ethics Committee
REC reference
21/EE/0025
Date of REC Opinion
30 Mar 2021
REC opinion
Further Information Favourable Opinion