Oral PHA-022121 for Hereditary Angioedema
(HAE CHAPTER-1 Trial)
What You Need to Know Before You Apply
What is the purpose of this trial?
This trial tests a new oral medication, PHA-022121, to determine its safety and effectiveness in preventing attacks in people with hereditary angioedema (HAE), a condition that causes sudden swelling. In the first part, participants receive either a low dose, a high dose, or a placebo (a pill with no active medicine) to compare results. In the second part, all participants receive the high dose. The trial seeks individuals diagnosed with HAE type I or II who have experienced at least three swelling attacks in the last three months or two during the screening period. As a Phase 2 trial, this research focuses on measuring the treatment's effectiveness in an initial, smaller group of people.
Will I have to stop taking my current medications?
The trial requires that you stop using certain medications like C1-esterase inhibitors, oral kallikrein inhibitors, attenuated androgens, anti-fibrinolytics, or monoclonal HAE therapy before enrolling. The protocol does not specify the exact time period for stopping these medications.
Is there any evidence suggesting that this trial's treatments are likely to be safe?
Research shows that deucrictibant, the main ingredient in PHA-022121, has been tested for safety in people with hereditary angioedema (HAE). In earlier studies, patients generally tolerated deucrictibant well. Safety checks, including physical exams, blood tests, and heart monitoring, found no major safety issues. Another study confirmed its long-term safety for preventing HAE attacks.
Moreover, deucrictibant has shown good results in both preventing and treating HAE attacks as needed. This indicates that the treatment is safe for people with HAE in different situations. While side effects can occur, these studies have reported no serious side effects.12345Why are researchers excited about this trial's treatments?
Unlike current treatments for hereditary angioedema, which often involve injections or infusions, PHA-022121 offers an oral option, making it more convenient for patients. This investigational drug, deucrictibant, works by inhibiting the bradykinin B2 receptor, a novel approach compared to traditional C1 esterase inhibitors and kallikrein inhibitors. Researchers are excited about the potential for PHA-022121 to provide rapid relief of symptoms through a less invasive delivery method, potentially enhancing patient comfort and adherence.
What evidence suggests that this trial's treatments could be effective for hereditary angioedema?
Research has shown that PHA-022121, also known as deucrictibant, may help treat hereditary angioedema (HAE). In one study, 86.9% of patients experienced complete resolution of swelling attacks within 24 hours. Another study suggested the drug could benefit those with a type of swelling related to HAE. Long-term research has also indicated that deucrictibant is safe and effective for managing HAE attacks. In this trial, participants will receive either a low or high dose of deucrictibant, or a placebo, to evaluate its effectiveness in preventing attacks in people with hereditary angioedema.13678
Who Is on the Research Team?
Marc Riedl, MD
Principal Investigator
UC San Diego, La Jolla, California, United States
Emel Aygören-Pürsün, MD
Principal Investigator
University Hospital Frankfurt - Goethe University, Frankfurt, Germany
Are You a Good Fit for This Trial?
This trial is for people with Hereditary Angioedema (HAE) types I or II who've had at least 3 attacks in the last 3 months, or a minimum of 2 during screening. They must be able to use standard acute attack meds and sign consent forms. It's not for pregnant/breastfeeding individuals, those with significant health issues that could affect safety/participation, recent drug abuse history, abnormal kidney/liver function, or if they've used certain HAE treatments/drugs recently.Inclusion Criteria
Exclusion Criteria
Timeline for a Trial Participant
Screening
Participants are screened for eligibility to participate in the trial
Treatment Part 1
Participants are randomized to receive either a low dose, high dose of PHA-022121, or placebo for 12 weeks
Treatment Part 2
Participants continue in a single open-label arm receiving a high dose of PHA-022121 for 30 months
Follow-up
Participants are monitored for safety and effectiveness after treatment
What Are the Treatments Tested in This Trial?
Interventions
- PHA-022121
- Placebo
Find a Clinic Near You
Who Is Running the Clinical Trial?
Pharvaris Netherlands B.V.
Lead Sponsor