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
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.Summary of results:This summary describes the results of a clinical study that tested an investigational medicine called deucrictibant in adults with hereditary angioedema (HAE).
This summary is written for people without a medical background.
The results are for information only and should not replace advice from your doctor.
Key Highlights
Why was this Study done?
Hereditary angioedema (HAE) is a rare condition that causes repeated swelling attacks in different parts of the body. These attacks can be painful and sometimes life‑threatening.
Many current treatments require injections. There is a need for easy‑to‑use oral (tablet) treatments.
This study was done to find out:
• if deucrictibant is safe and well tolerated
• if it can reduce the number of swelling attacks
What conditions did the people in study have?
Male and female participants, aged ≥18 and ≤75 years at enrollment, with a diagnosis of HAE type 1or type 2 and documented history of at least 3 HAE attacks in the 3 consecutive months or prior to screening.
The screening period was up to 8 weeks.
Who took part in the study?
• 34 adults took part in the main study
• Ages ranged from 18 to 75 years
• All participants had Type 1 or Type 2 HAE
• Participants had frequent attacks (at least 3 recent attacks)
When did the study take place?
Duration of treatment:
The planned duration of treatment in Part 1 was 12 weeks.
The planned duration of treatment in Part 2 was 30 months.
The study started in March 2022 and ended in June 2025.
How was the study done?
This study had two parts:
Part 1 (12 weeks)
Participants were randomly assigned to:
• deucrictibant 10 mg twice daily
• deucrictibant 20 mg twice daily
• placebo (inactive capsule)
Part 2 (long‑term)
• All participants received deucrictibant 20 mg twice daily
• Treatment continued for up to 30 months
Participants recorded symptoms daily in an electronic diary.
What were the main questions?
Researchers wanted to know:
• Does deucrictibant reduce HAE attacks?
• Is it safe for long‑term use?
What were the main results?
Did the medicine work?
Yes — deucrictibant reduced the number of HAE attacks.
Results after 12 weeks (Part 1):
• Placebo: 1.93 attacks/month
• Deucrictibant 10 mg: 0.40 attacks/month (79.24% reduction)
• Deucrictibant 20 mg: 0.30 attacks/month (84.46% reduction)
More people had fewer or no attacks:
• Many participants had large reductions in attacks
• Some participants had no attacks
• No participants on placebo were attack‑free
Long‑term results (Part 2):
• Average attacks: 0.12 per month
• About 48% of participants had no attacks
Other benefits:
• fewer severe attacks
• fewer attacks needing emergency medication
• improved quality of life
What do the safety results tell us?
The study demonstrated that deucrictibant was well tolerated and efficacious for long-term prophylactic treatment in adult patients with HAE.
Was the medicine safe?
Yes — deucrictibant was generally safe and well tolerated.
Part 1:
• Side effects were similar across all groups
• Most were mild or moderate
• No serious side effects related to the medicine
• No participant stopped treatment due to side effects
Part 2:
• No new safety concerns were identified
• Most side effects remained mild or manageable
Common side effects included:
• headache
• nausea
• dizziness
• mild laboratory changes
What do the results mean?
This study showed that deucrictibant:
• significantly reduced HAE attacks
• worked in both short‑term and long‑term use
• was safe and well tolerated
How has this study helped people?
Improvements in efficacy and quality of life that were observed in Part 1 were maintained in Part 2, and there was no change in the overall safety profile of deucrictibant over the median duration of exposure of 657 days in Part 2.
These results suggest that deucrictibant may be a convenient oral treatment option for preventing HAE attacks.
However, more studies may still be needed.
Thank you to everyone who took part
We would like to thank everyone who took part in this study for being part of the research.
Where can I find more information about this study?
You can find more detailed information on these websites:
• https://gbr01.safelinks.protection.outlook.com/?url=https%3A%2F%2Ftrack.pstmrk.it%2F3ts%2Fclinicaltrials.gov%2FNBTI%2FGsLGAQ%2FAQ%2Fee8be3fa-04cc-48f1-91c0-880ca32071aa%2F2%2FooftMQnXkf&data=05%7C02%7Champshirea.rec%40hra.nhs.uk%7Cfe921d4e51324aa4e1a408dee4052a33%7C8e1f0acad87d4f20939e36243d574267%7C0%7C0%7C639198908003985403%7CUnknown%7CTWFpbGZsb3d8eyJFbXB0eU1hcGkiOnRydWUsIlYiOiIwLjAuMDAwMCIsIlAiOiJXaW4zMiIsIkFOIjoiTWFpbCIsIldUIjoyfQ%3D%3D%7C0%7C%7C%7C&sdata=EiC6JWnAv%2BBfkyl6lfopECbPFZfBT5zZGikwvm9UwsE%3D&reserved=0
• https://gbr01.safelinks.protection.outlook.com/?url=https%3A%2F%2Ftrack.pstmrk.it%2F3ts%2Fwww.clinicaltrialsregister.eu%252Fctr-search%252Fsearch%2FNBTI%2FGsLGAQ%2FAQ%2Fee8be3fa-04cc-48f1-91c0-880ca32071aa%2F3%2FCkS88oF2Cm&data=05%7C02%7Champshirea.rec%40hra.nhs.uk%7Cfe921d4e51324aa4e1a408dee4052a33%7C8e1f0acad87d4f20939e36243d574267%7C0%7C0%7C639198908004006434%7CUnknown%7CTWFpbGZsb3d8eyJFbXB0eU1hcGkiOnRydWUsIlYiOiIwLjAuMDAwMCIsIlAiOiJXaW4zMiIsIkFOIjoiTWFpbCIsIldUIjoyfQ%3D%3D%7C0%7C%7C%7C&sdata=IPtTO2y6WMu6vbcZtP3wntd9F1W0ctw7Ux4lpFd6kQs%3D&reserved=0REC 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