Extension Study of Oral PHA-022121 for Acute Treatment of Angioedema Attacks in Patients With Her… (NCT05396105) | Clinical Trial Compass
By InvitationPhase 2/3
Extension Study of Oral PHA-022121 for Acute Treatment of Angioedema Attacks in Patients With Hereditary Angioedema
United States, Argentina, Australia150 participantsStarted 2022-12-28
Plain-language summary
This study evaluates the safety and efficacy of long-term on-demand treatment with orally administered deucrictibant for acute hereditary angioedema (HAE) attacks, including laryngeal attacks. The study will enroll participants from Study PHA022121-C201 (NCT04618211), Study PHA022121-C306 (NCT06343779) and deucrictibant treatment naïve HAE-nC1INH adult participants who elect to participate in this extension study and meet the eligibility requirements.
Who can participate
Age range
12 Years
Sex
ALL
See this in plain English?
AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.
Inclusion criteria
. Provision of the signed informed consent form by the participant and/ or legally designated representative. If the participant is a minor (i.e., \<18 years of age or as determined by local law), consent will be obtained from the participant's parent/legally designated representative/guardian and signed assent will be obtained from the participant, per country regulations.
. For participants from Study C201, received at least one dose of study drug (including the non-attack visit) in Study C201. For participants from Study C306, participant was randomized (and for adolescent participants ≥12 to \<18 years received a dose of study drug in a non-attack state at Visit 1) and completed Study C306, with 2 attacks treated, or after closure of that study by the Sponsor. Enrollment of adolescents (≥12 to \<18 years or age of adulthood as defined locally) from these studies is with consideration of local age requirements.
. Female participants of childbearing potential (or who become of childbearing potential during the study) must agree to the protocol-specified pregnancy testing and to be abstinent from heterosexual intercourse or to use an acceptable contraception method as defined in the protocol and as available locally from enrollment until 30 days after the last study drug administration.
Questions worth asking your doctor
Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.
1Based on my diagnosis and history, is this trial worth exploring for me — or is there a standard treatment we should try first?
2What does this trial's phase tell us about how much is already known about its safety and benefit?
3What would taking part actually involve for me — visits, tests, time, and travel?
4What are the known and possible risks or side effects I should weigh, and how would they be monitored?
5If this trial isn't the right fit, what other options or trials would you suggest I look into?
Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.
Questions for the trial coordinator
The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.
1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?
A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.
What they're measuring
1
Treatment-emergent Adverse Events (TEAEs), treatment-related TEAEs, treatment-emergent serious adverse events (TESAEs), treatment-related TESAEs, and TEAEs leading to deucrictibant discontinuation
Timeframe: From enrollment through study completion, up to 54 months (dependent on time of enrollment).
2
Heart Rate
Timeframe: From enrollment through study completion, up to 54 months (dependent on time of enrollment).
3
Blood pressure
Timeframe: From enrollment through study completion, up to 54 months (dependent on time of enrollment).
4
Body temperature
Timeframe: From enrollment through study completion, up to 54 months (dependent on time of enrollment).
5
Clinical laboratory tests
Timeframe: From enrollment through study completion, up to 54 months (dependent on time of enrollment).
6
Electrocardiograms
Timeframe: From enrollment through study completion, up to 54 months (dependent on time of enrollment).
. In the opinion of the Investigator, the participant (and parent/caregiver for adolescent participants) is willing and able to comply with the protocol.
. Adult participants with HAE type III (HAE-nC1INH) who are deucrictibant-treatment naïve, must meet all of the following:
. Documented genetic mutation associated with HAE-nC1INH as listed in the Hereditary Angioedema Association (HAEA) and World Allergy Organization (WAO)/European Academy of Allergy and Clinical Immunology (EAACI) Guidelines.
. If no documented mutation: clinical diagnosis with family history of HAE-nC1INH and documented elevations of bradykinin levels in blood. (US, UK and Canada only).
Exclusion criteria
. Any female who is pregnant, plans to become pregnant, or is breast-feeding.
. Any other systemic disease (e.g., cardiovascular, gastrointestinal, renal, respiratory, neurological) or significant disease or disorder that, in the opinion of the Investigator, would interfere with the participant's safety or ability to participate in the study.
. Use of lanadelumab for long-term HAE prophylactic therapy within 12 weeks prior to enrollment in Part A.
. Participants who have recently used short or long-term HAE prophylaxis or on-demand HAE treatment will not be excluded from the study provided the following washout period is observed (i.e., study screening or enrollment/rollover should be delayed allowing for washout):
. 2-week washout period before enrollment should be respected for participants who have used any C1-INH product, oral kallikrein inhibitors, attenuated androgens, or anti-fibrinolytics for long-term prophylactic HAE therapy.
. 1-week washout period before enrollment should be respected for participants who have used plasma derived C1-INH concentrates (Berinert, Cinryze, Haegarda) for on-demand treatment or short-term prophylaxis.
. 24-hour washout period before enrollment should be respected for participants who have used recombinant C1-INH (Ruconest) for on-demand treatment or short-term prophylaxis.
. History of alcohol or drug abuse within the previous year, or current evidence of substance dependence or abuse
Physical Examination
Timeframe: From enrollment through study completion, up to 54 months (dependent on time of enrollment).