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CHAPTER-4 – A long term trial of deucrictibant treatment in prophylaxis against HAE attacks in adults and adolescents
EU CTIS ID: 2024-516248-24-00
What this study is testing
To evaluate the safety and tolerability of deucrictibant 40 mg extended-release (XR) tablet in long-term prophylactic treatment of hereditary angioedema (HAE)
- Therapeutic confirmatory (Phase III)
A plain-language read of the study's public EU CTIS listing. The study team confirms the details.
Who can take part
You may be able to join if
- Provision of written informed consent. At the time of signing informed consent, male and female participants must be aged ≥12 years. If the participant is an adolescent (ie, aged ≥12 to <18 years or as determined by local law, ≥40 kg), written assent will be obtained from the participant and consent will be obtained from the participant’s parent/legal guardian. A participant who is an adolescent will sign the informed consent form if they reach the age of 18 or as determined by local law during their participation in the study
- Participants who previously participated in HAE studies with deucrictibant on-demand or prophylactic treatment may be eligible to be screened for this study. Participants in Study PHA022121-305 must have completed all study drug treatment and assessments in the double-blind Treatment Period of the previous study
- Participants who have not previously had their HAE diagnosis confirmed by a central laboratory in a Pharvaris-sponsored study with deucrictibant must meet the following requirements: a. Diagnosis of HAE based upon all of the following: • Documented clinical history consistent with HAE (cutaneous or submucosal, nonpruritic swelling without accompanying urticaria) • At least one of the following: 1. Age at reported onset of first angioedema symptoms ≤30 years 2. Family history consistent with HAE 3. C1q within normal range b. Diagnostic testing results to confirm HAE: • C1 esterase inhibitor (C1INH) functional level <50% of the normal level must be shown by chromogenic assay performed by the central laboratory as part of the Screening procedures.
- For Non rollover participants: History of at least 1 attack in the last 3 consecutive months prior to Screening
- Participant is assessed by the Investigator to have reliable access and ability to use standard of care on-demand treatments to effectively manage acute HAE attacks
- Investigator considers that the participant (and parent/caregiver for adolescent participants) is willing and able to adhere to all protocol requirements, including the participant being capable of and compliant with data recording into an eDiary
You likely can't join if
- Any diagnosis of angioedema other than HAE
- History of epilepsy and/or other significant neurological diseases
- Any clinically significant and uncontrolled gastrointestinal dysfunction (eg, chronic diarrhea, inflammatory bowel disease) which impacts study drug absorption
- History of alcohol or drug abuse within the previous year, or current evidence of substance dependence or abuse
- Use of concomitant medications with systemic absorption and foods that are moderate and strong inhibitors of cytochrome P450 (CYP)3A4 such as clarithromycin, erythromycin, diltiazem, itraconazole, ketoconazole, ritonavir, verapamil, and grapefruit juice or strong inducers of CYP3A4 such as carbamazepine, phenytoin, and rifampin within the last 30 days or within 5 half-lives (whichever is longer) of the time of enrollment
- Known hypersensitivity to deucrictibant or any of the excipients of study drug
See the full eligibility criteria
Who can join
- Provision of written informed consent. At the time of signing informed consent, male and female participants must be aged ≥12 years. If the participant is an adolescent (ie, aged ≥12 to <18 years or as determined by local law, ≥40 kg), written assent will be obtained from the participant and consent will be obtained from the participant’s parent/legal guardian. A participant who is an adolescent will sign the informed consent form if they reach the age of 18 or as determined by local law during their participation in the study
- Participants who previously participated in HAE studies with deucrictibant on-demand or prophylactic treatment may be eligible to be screened for this study. Participants in Study PHA022121-305 must have completed all study drug treatment and assessments in the double-blind Treatment Period of the previous study
- Participants who have not previously had their HAE diagnosis confirmed by a central laboratory in a Pharvaris-sponsored study with deucrictibant must meet the following requirements: a. Diagnosis of HAE based upon all of the following: • Documented clinical history consistent with HAE (cutaneous or submucosal, nonpruritic swelling without accompanying urticaria) • At least one of the following: 1. Age at reported onset of first angioedema symptoms ≤30 years 2. Family history consistent with HAE 3. C1q within normal range b. Diagnostic testing results to confirm HAE: • C1 esterase inhibitor (C1INH) functional level <50% of the normal level must be shown by chromogenic assay performed by the central laboratory as part of the Screening procedures.
- For Non rollover participants: History of at least 1 attack in the last 3 consecutive months prior to Screening
- Participant is assessed by the Investigator to have reliable access and ability to use standard of care on-demand treatments to effectively manage acute HAE attacks
- Investigator considers that the participant (and parent/caregiver for adolescent participants) is willing and able to adhere to all protocol requirements, including the participant being capable of and compliant with data recording into an eDiary
- Female participants of childbearing potential (or who become of childbearing potential during the study) must agree to the protocol specified pregnancy testing and use an acceptable contraception method defined in the protocol from enrollment until 30 days after the last study drug administration
What rules you out
- Any diagnosis of angioedema other than HAE
- History of epilepsy and/or other significant neurological diseases
- Any clinically significant and uncontrolled gastrointestinal dysfunction (eg, chronic diarrhea, inflammatory bowel disease) which impacts study drug absorption
- History of alcohol or drug abuse within the previous year, or current evidence of substance dependence or abuse
- Use of concomitant medications with systemic absorption and foods that are moderate and strong inhibitors of cytochrome P450 (CYP)3A4 such as clarithromycin, erythromycin, diltiazem, itraconazole, ketoconazole, ritonavir, verapamil, and grapefruit juice or strong inducers of CYP3A4 such as carbamazepine, phenytoin, and rifampin within the last 30 days or within 5 half-lives (whichever is longer) of the time of enrollment
- Known hypersensitivity to deucrictibant or any of the excipients of study drug
- Participation in a clinical study with any other investigational drug within the last 30 days or within 5 half-lives of the investigational drug at ICF signature (whichever was longer) or prior gene therapy for any indication at any time
- Participants who discontinued from previous studies with deucrictibant prophylactic and/or on demand treatment due to safety reasons or compliance issues that, in the opinion of the Investigator, would interfere with the participant’s safety or compliance to participate in the study. Participants who previously received deucrictibant prophylactic and/or on-demand treatment and recently completed a pregnancy may be eligible to participate in this study. Participants may be eligible for this study if the previous study with deucrictibant prophylactic and/or on-demand treatment has been terminated by the Sponsor prior to their completion of the study
- Exposure to angiotensin-converting enzyme (ACE) inhibitors or any estrogen-containing medications with systemic absorption (such as oral contraceptives or hormonal replacement therapy) within 4 weeks of Screening
- For Rollover participants who have completed participation in studies with deucrictibant prophylactic treatment prior to Screening and Non rollover participants: Receiving prophylactic treatment for HAE within the time periods before Screening as indicated below: a. Long-term prophylactic therapy for HAE (C1INH, oral kallikrein inhibitors, or anti-fibrinolytics) within 2 weeks prior to Screening b. Long-term prophylactic therapy for HAE with attenuated androgens within 4 weeks prior to Screening c. Long-term prophylactic monoclonal antibody therapy for HAE (ie, lanadelumab) within 5 half-lives prior to Screening d. Short-term prophylaxis for HAE within 7 days prior to Screening
- Any females who are pregnant, plan to become pregnant, or are currently breast-feeding
- Abnormal hepatic function (aspartate aminotransferase [AST] >2× upper limit of normal [ULN], alanine aminotransferase [ALT] >2× ULN, or total bilirubin >1.5× ULN or any hepatic impairment via Child-Pugh Scoring System. Participants with Gilbert’s syndrome, defined as isolated increase of total bilirubin ≤3× ULN and AST and ALT within the normal range, are not excluded
- Moderate to severe renal impairment (estimated glomerular filtration rate [eGFR] <60 mL/min/1.73 m2)
- Any clinically significant history of angina, myocardial infarction, syncope, stroke, left ventricular hypertrophy or cardiomyopathy, uncontrolled hypertension, bradycardia, or any other clinically significant cardiovascular abnormality within the previous year that, in the opinion of the Investigator, would interfere with the participant's safety or ability to participate in the study
The study team makes the final eligibility decision.
Where it's taking place
- China
- United States
- New Zealand
- Brazil
- Puerto Rico
- Switzerland
- Qatar
- South Africa
- Canada
- Singapore
- North Macedonia
- Hong Kong
- Saudi Arabia
- Japan
- Turkey
- Korea, Republic of
- United Kingdom
- Colombia
- Argentina
Questions & answers
Do participants get paid in this trial?
This listing doesn't specify compensation. Many trials still reimburse travel or offer a stipend, so it's worth asking the study team when you connect.
Is it free to join, and do I need insurance?
Searching and applying through BridgeMD is free. In clinical trials the study-related treatment and visits are generally provided at no cost to you. You usually don't need insurance to take part - confirm specifics with the study team.
How long does this study last?
The listing doesn't state an exact length. The study team walks you through the schedule and number of visits before you decide to enroll.
Who can join this trial?
This study is enrolling all sexes, 18-64 years, 65+ years, 0-17 years. The study team makes the final eligibility decision.
Where is this trial taking place?
Study sites include China; United States; New Zealand; Brazil; Puerto Rico; Switzerland and 13 more location(s). Enter your location above to see the nearest site and check your eligibility.
Explore other conditions
BridgeMD is an information and trial-matching tool - not medical advice, and not the study sponsor. Details come from EU CTIS; the study team decides eligibility.