A Phase 1/2 interventional study of Deucrictibant XR tablet and Placebo comparator to XR tablet in Hereditary Angioedema (HAE), Angioedema and Bradykinin-mediated Angioedema, sponsored by Institute for Asthma and Allergy. Recruiting at 1 site in United States. Open to participants aged 18 Years to 75 Years. Per ClinicalTrials.gov, last updated 2025-07-02.
Sponsored by Institute for Asthma and Allergy · Phase 1/2, Interventional, and Prevention
To assess the efficacy of prophylactic treatment with deucrictibant extended release (XR) tablet versus placebo in preventing angioedema attacks, and to also assess the efficacy of deucrictibant soft capsules as on-demand treatment versus placebo in achieving angioedema symptom relief during acute attacks.
Study Design:
The study has 2-parts, studying the efficacy of deucrictibant versus placebo for prophylactic and on-demand treatment of angioedema attacks will be assessed in adult participants with BK-AE-nC1INH. Each part of the study is designed a randomized, double-blind, placebo-controlled cross-over trial. Up to 10 patients to be enrolled.
Deucrictibant has been adopted as the international nonproprietary name (INN) for the active ingredient, previously referred to as PHA121 or PHA-022121. Deucrictibant is under development as an orally administered bradykinin B2 receptor antagonist with potential for both acute therapeutic and prophylactic use in patients with different types of BK-mediated angioedema, including HAE-1/2, HAE with normal C1INH, acquired angioedema due to C1INH deficiency, and non-hereditary BK-mediated angioedema with normal C1INH.
Throughout the study (Parts 1 and 2), a diary will be used to record all symptoms and details of angioedema attacks, including on-demand treatments, as well as Patient Reported Outcomes (PROs). Participants need to have access to FDA approved on-demand angioedema medication and be able to treat any angioedema attack that may occur using standard of care on-demand therapies. In addition to the collected data on angioedema attacks, the diary will be used as a dosing diary for the daily intake of study drug treatment in Part 1.
Part 1 will evaluate the efficacy and safety of deucrictibant administered orally for prophylaxis against angioedema attacks in patients with BK-AE-nC1INH. After providing written informed consent, participants will be screened for eligibility. Eligible participants will be enrolled in the study. Participants receiving angioedema medications for long-term prophylaxis (LTP) will discontinue and washout these medications prior to Screening.
Dosing schedule: QD administration of study drug or placebo (double blind) throughout the Treatment Period consisting of deucrictibant (40 mg strength) XR tablet and matching placebo for oral use.
The duration of an individual's participation in Part 1 is up to 24 weeks (12 weeks on each deucrictibant and placebo, in random order).
Participants will be randomized in a 1:1 ratio to receive deucrictibant 40 mg XR tablet or matching placebo once daily (QD) each for 12 weeks and then participants will be crossed over, for a total of 24 weeks of treatment.
Part 2: After completion of Part 1, a study visit will be conducted at Week 24, at which the enrolled participants will be re-randomized in Part 2 on a 1:1 ratio to a treatment sequence to receive deucrictibant 20 mg soft capsule following by matching placebo or vice versa, with one capsule given for each of 2 attacks. Participants will self-administer blinded study drug in the assigned treatment sequence at home to treat 2 non-laryngeal qualifying angioedema attacks (at least one attack symptom [skin pain, skin swelling, or abdominal pain] has reached the individual item score of ≥20 in VAS-3). The time of administration of study drug as well as rescue medication must be recorded in the patient diary.The expected duration of Part 2 is up to 16 weeks.
End-of-Treatment and End-of-Study visits will occur within 7±2 days and 28 days (-14 days), respectively, after treatment of the second attack in Part 2.
Dosing schedule: Administration of study drug for acute angioedema attacks, dosing consists of deucrictibant (20 mg strength) soft capsule and matching placebo for oral use.
The duration of treatment in Part 2 is approximately 16 weeks.
Efficacy will be assessed based upon the occurrences of angioedema attacks, which will be confirmed by Investigators. During Screening, site personnel will discuss with participants the requirements for reporting angioedema attacks, including the information that is to be captured for each angioedema attack.
A Statistical Analysis Plan (SAP) will be finalized prior to database lock and will include a more technical and detailed description of all planned statistical analyses of all study endpoints.
The analysis of safety and tolerability will be performed on the Safety Analysis Set (ie, all randomized participants who have received at least 1 dose of study drug). Treatment-emergent AEs (TEAEs) are defined as AEs that start or pre-existing AEs that have worsened after the initiation of study drug.
The number and percentage of participants with TEAEs will be summarized by treatment group by body system organ class (SOC) and preferred term using the Medical Dictionary for Regulatory Activities (MedDRA®). Clinical laboratory data, vital signs, physical examination, and ECG data will be presented in summary tables and individual listings by treatment group.
171 studies on the registry are indexed under Angioedemas, Hereditary; 24 are open to participants now.
This study's planned enrollment of 10 is below the median of 48 across 116 interventional studies indexed under Angioedemas, Hereditary.
Browse Angioedemas, Hereditary studies →This is the only study on the registry with Institute for Asthma and Allergy as lead sponsor.
Counted across the registry records on this site, refreshed daily.
Go through the criteria one by one — yes, no or not sure — and take the list to your doctor or the study team. Kept in this browser only.
Diagnosis of bradykinin-mediated angioedema based upon all of the following:
Total blood BK peptide levels following 3 days cold activation is above the diagnostic value in non-attack and/or attack period*.
*The "attack period" is defined as within 24 hours after an attack.
Exclusion Criteria:
Receiving prophylactic treatment for BK-AE-nC1INH. Participants who have previously received prophylactic therapy but have stopped can participate in this study provided a sufficiently long (≥5 half-life) is observed before the participant is screened. Exclusion includes use of:
Underlined terms in the criteria open these too. Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
Anti-inflammatory medicines such as prednisone, prednisolone and dexamethasone — not the anabolic steroids used for muscle.
Studies differ in what they allow: check which steroid medicines, routes (tablets, injections, creams, inhalers, eye drops) and doses this study accepts.
A drug or device being studied in a clinical trial.
It may be new, or already approved and being studied for another use, dose or form. Criteria often leave out people who took part in another study of one within a recent period, commonly 30 days or five half-lives.
Ways of preventing pregnancy. “Highly effective” methods fail less than 1% of the time a year when used correctly — for example an IUD or an implant.
Criteria usually say which methods count and how long to keep using them after the last dose, sometimes for male participants too. Some studies accept a partner’s vasectomy if it was confirmed successful and that person is the participant’s only sexual partner.
A planned gap after a treatment, so that its effects can lessen before the study treatment or a study assessment. Ask the study team and the doctor who prescribed it before changing any medicine.
Two enzymes measured in a routine blood test of the liver. Higher levels can mean the liver is under strain or damaged. SGPT and SGOT are older names for ALT and AST.
Criteria usually give a limit as a multiple of the upper limit of normal (ULN), often higher when cancer has spread to the liver.
The top of the normal range for a blood test, as set by the laboratory that runs it. Each lab prints its own range next to your result.
“≤ 2.5 × ULN” means the result can be up to two and a half times that top value.
A yellow substance made when red blood cells are broken down, which the liver clears. A high level can mean a problem with the liver or the bile ducts.
Criteria often allow more for people with Gilbert’s syndrome, a harmless inherited condition that raises bilirubin.
GFR (glomerular filtration rate) is how much blood the kidneys filter each minute. It is usually estimated from a blood test — eGFR, by formulas such as CKD-EPI or MDRD — and some studies ask for it to be measured directly instead. Usually given in mL/min/1.73 m².
Check the method, the units and the minimum the study states. A value of 90 or above is in the normal-or-high range, but kidney damage can show in other tests too.
Judged by the study doctor to matter for your health or for the study. It is a doctor’s decision, so ask the study team if you are unsure whether something counts.
CYP3A is a group of enzymes, mainly CYP3A4, in the liver and gut that break down many medicines. Some medicines, supplements and foods (grapefruit, St John’s wort) slow them down or speed them up, which changes how much of a study drug stays in the body.
Studies list them so they can be avoided or switched; bring a list of everything you take, including supplements.
An allergy, or another reaction of the immune system, to a substance. “Hypersensitivity to the study drug” means a known allergic reaction to it — not every side effect a medicine can have.
The other ingredients in a medicine besides the active drug, such as fillers, colours or preservatives.
Informed consent
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Corticosteroids (steroids)
Anti-inflammatory medicines such as prednisone, prednisolone and dexamethasone — not the anabolic steroids used for muscle.
Studies differ in what they allow: check which steroid medicines, routes (tablets, injections, creams, inhalers, eye drops) and doses this study accepts.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Investigational drug or device
A drug or device being studied in a clinical trial.
It may be new, or already approved and being studied for another use, dose or form. Criteria often leave out people who took part in another study of one within a recent period, commonly 30 days or five half-lives.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Contraception
Ways of preventing pregnancy. “Highly effective” methods fail less than 1% of the time a year when used correctly — for example an IUD or an implant.
Criteria usually say which methods count and how long to keep using them after the last dose, sometimes for male participants too. Some studies accept a partner’s vasectomy if it was confirmed successful and that person is the participant’s only sexual partner.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Washout period
A planned gap after a treatment, so that its effects can lessen before the study treatment or a study assessment. Ask the study team and the doctor who prescribed it before changing any medicine.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
ALT and AST (liver enzymes)
Two enzymes measured in a routine blood test of the liver. Higher levels can mean the liver is under strain or damaged. SGPT and SGOT are older names for ALT and AST.
Criteria usually give a limit as a multiple of the upper limit of normal (ULN), often higher when cancer has spread to the liver.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
ULN (upper limit of normal)
The top of the normal range for a blood test, as set by the laboratory that runs it. Each lab prints its own range next to your result.
“≤ 2.5 × ULN” means the result can be up to two and a half times that top value.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Bilirubin
A yellow substance made when red blood cells are broken down, which the liver clears. A high level can mean a problem with the liver or the bile ducts.
Criteria often allow more for people with Gilbert’s syndrome, a harmless inherited condition that raises bilirubin.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
GFR and eGFR (kidney filtering)
GFR (glomerular filtration rate) is how much blood the kidneys filter each minute. It is usually estimated from a blood test — eGFR, by formulas such as CKD-EPI or MDRD — and some studies ask for it to be measured directly instead. Usually given in mL/min/1.73 m².
Check the method, the units and the minimum the study states. A value of 90 or above is in the normal-or-high range, but kidney damage can show in other tests too.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Clinically significant
Judged by the study doctor to matter for your health or for the study. It is a doctor’s decision, so ask the study team if you are unsure whether something counts.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
CYP3A (CYP3A4) inhibitors and inducers
CYP3A is a group of enzymes, mainly CYP3A4, in the liver and gut that break down many medicines. Some medicines, supplements and foods (grapefruit, St John’s wort) slow them down or speed them up, which changes how much of a study drug stays in the body.
Studies list them so they can be avoided or switched; bring a list of everything you take, including supplements.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Hypersensitivity
An allergy, or another reaction of the immune system, to a substance. “Hypersensitivity to the study drug” means a known allergic reaction to it — not every side effect a medicine can have.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Excipients
The other ingredients in a medicine besides the active drug, such as fillers, colours or preservatives.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Deucrictibant XR tablet, 40 mg, QD, oral use for 12 weeks
Drug: Deucrictibant XR tablet
Placebo tablets, 40 mg equivalent, QD, oral use for 12 weeks
Drug: Placebo comparator to XR tablet
Single 20 capsule orally for treatment of on-demand angioedema attack, for up to 16 weeks
Drug: Deucrictibant 20 mg capsule
Placebo capsules for treatment of on-demand angioedema attacks, for up to 16 weeks
Drug: Placebo comparator to 20 mg capsule
Deucrictibant 40 mg XR tablet for prophylaxis
Also known as: Deucrictibant XR
Placebo comparator to deucrictibant 40 mg XR tablet, prophylaxis
Also known as: Placebo
Deucrictibant active drug for on-demand treatment of angioedma attacks
Also known as: Deucrictibant
Placebo comparator to Deucrictibant 20 mg capsule for on-demand treatment of angioedema attacks
Also known as: Placebo
Part-1: Incidence and quality of HAE attacks comparing active versus placebo arms.
Part-1: Time-normalized number of Investigator-confirmed moderate or severe angioedema attacks during the Treatment Period. Time-normalized number of Investigator-confirmed angioedema attacks treated with on-demand medication during the Treatment Period.
Time frame: 24 weeks
Part-2: Efficacy of active on-demand treatment versus placebo in achieving angioedema symptom relief during acute attacks
Part-2: Time to onset of symptom relief of active drug versus placebo, defined as Patient Global Impression of Change (PGI-C) rating of at least "a little better" for 2 consecutive timepoints within 12 hours post-treatment. Time to complete symptom resolution, defined as achieving PGI-S rating of "none" within 48 hours post-treatment.
Time frame: 16 weeks
Safety assessment of oral deucrictibant extended release (XR) tablet (Part 1), and deucrictibant oral on-demand tablets (Part 2).
Adverse event severity, expectedness, and relatedness of the active drug, classified based on their severity, whether they were expected, and their potential relationship to the study intervention (Parts 1 and 2).
Time frame: 40 weeks
Part-1: Change in Health-Related Quality of Life (HRQoL) Scores Between Active Drug and Placebo Periods
Assessment of quality of life will be conducted using a validated disease-specific instrument, such as the Hereditary Angioedema Quality of Life Questionnaire (HAE-QoL), and supplemented by a daily patient diary Daily diary entries will record the presence or absence of attacks, their severity, duration, body location, treatment used, time to resolution, and the impact on daily living (missed work/school, physical limitations, mood, and social activities).documenting physical, emotional, and functional impacts of each attack.
Time frame: 16 weeks
Plan to share: Yes — Access to trial Individual Participant Data (IPD) can be requested by qualified researchers engaging in independent scientific research, and will be provided following review and approval of a research proposal and Statistical Analysis Plan (SAP) and execution of a Data Sharing Agreement (DSA) at least 9 months after the publication date.
Supporting information: Study protocol, Statistical analysis plan, Informed consent form, Clinical study report, Analytic code
Time frame: After 9 months post publication date of the clinical trial data.
Access criteria: Any qualified researchers engaging in independent scientific research.
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Studies of the drug tested here: Deucrictibant