RAPIDe-2
Research type
Research Study
Full title
A Phase II/III, Extension Study of Orally Administered PHA-022121 for Acute Treatment of Angioedema Attacks in Patients with Hereditary Angioedema due to C1-Inhibitor Deficiency (Type I or Type II)
IRAS ID
1005340
Contact name
Eivind Omli
Contact email
Sponsor organisation
Pharvaris Netherlands BV
Eudract number
2021-006906-58
Research summary
PHVS416 (PHA-022121 soft capsules) is being developed for the treatment of hereditary angioedema (HAE). The main purpose of this research study is to test the long-term safety of 3 different doses of PHVS416 in treating HAE attacks. HAE is a very rare genetic disease. In most cases, HAE patients have a defect in the gene that controls a blood protein called “C1 Inhibitor". Lack or low level of C1 inhibitor in the blood causes a biochemical imbalance characterized by an increased formation of the chemical bradykinin. Bradykinin produces the symptoms of HAE, such as swelling and pain in various parts of the body. The study drug is designed to block the effects of too much bradykinin to avoid progression of attacks and lead to the resolution of attacks. There are already medicines available to treat the manifestations of HAE attacks, that are injected either into a vein or under the skin. Up to 72 subjects with HAE who previously participated in the RAPIDe-1 study will take part in this research study at approximately 30 study sites globally. The study will be conducted in two parts. In Part A of the study, patients will continue to receive the same dose of study drug as the dose you were receiving in the RAPIDe-1 study: 10 mg, 20 mg, or 30 mg. Once all the data from the RAPIDe-1 study have been collected and analysed, the sponsor will decide which of the 3 doses used in RAPIDe-1 will be used for Part B of the study. Once this selected dose has been approved by the MHRA and Central Ethics Committee patients will move to Part B of the study where all participants in the study will receive that dose of study drug. This study is planned to continue until the study drug (PHVS416) is available for commercial supply or another means of continued treatment can be provided.
REC name
London - Riverside Research Ethics Committee
REC reference
22/LO/0409
Date of REC Opinion
20 Jul 2022
REC opinion
Further Information Unfavourable Opinion