HAE Chapter-1
Research type
Research Study
Full title
A Phase II, Double-blind, Placebo-controlled, Randomized, Dose-ranging, Parallel Group Study to Evaluate the Safety and Efficacy of PHA-022121 Administered Orally for Prophylaxis Against Angioedema Attacks in Patients with Hereditary Angioedema due to C1-Inhibitor Deficiency (Type I or Type II)
IRAS ID
302606
Contact name
Sorena Kiani-Alikhan
Contact email
Sponsor organisation
Pharvaris Netherlands BV
Eudract number
2021-000227-13
Clinicaltrials.gov Identifier
Clinicaltrials.gov Identifier
153097, IND
Duration of Study in the UK
0 years, 5 months, 25 days
Research summary
This study is being carried out on patients with hereditary angioedema (HAE). 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 that produces swelling in various parts of the body, including hands, feet, intestinal wall, genitalia, face, tongue, and airway (throat).
This study will look at whether the investigational drug PHA-022121 can prevent HAE swellings (acute attacks) and will also evaluate the safety of the study drug, what the body does to the study drug, and the quality of life of the participants.
This is a Phase 2 study, the study drug has already been tested in other people, but it has not been tested in people with HAE. The study drug will be compared to a placebo (no active ingredients). Eligible patients will be randomised in a 1:1:1 ratio to receive placebo or one of two dose regimens of PHA-022121. The study is “double-blind”, so neither the patient or the study doctor will know which “study medication” (study drug or placebo) the patient receives.
Approximately, 30 adult patients with HAE type I/II will take part globally, for up to 22 weeks. The study has a screening period (up to 8 weeks), treatment period (12 weeks) and follow up period (2weeks). There will be a number of assessments (Medical history, physical exams, vital signs, ECGs, blood/urine samples) and questionnaires to be completed by patients.
Patients who completed the study may have the option of participating in an open-label extension (OLE) study (PHA022121-C302), once the study is initiated.REC name
South Central - Hampshire A Research Ethics Committee
REC reference
21/SC/0365
Date of REC Opinion
7 Jan 2022
REC opinion
Further Information Favourable Opinion