Hereditary Angioedema
Conditions
Keywords
Angioedema, Hereditary Angioedema, HAE, Deucrictibant, Deucrictibant immediate-release, Bradykinin, Bradykinin B2 receptor, Hereditary angioedema with C1 inhibitor deficiency/dysfunction, HAE-C1INH, Hereditary angioedema with normal C1 inhibitor, HAE-nC1INH
Brief summary
This is an expanded access program (EAP) designed to allow access to deucrictibant immediate-release (IR) capsule for eligible participants with hereditary angioedema (HAE) in the United States (US) and US territories who lack satisfactory alternative treatment options for on-demand treatment of HAE attacks, as determined by their treating physician.
Detailed description
The EAP is intended to address unmet medical needs not adequately addressed by currently available therapies and is not designed to replace clinical trials conducted to support regulatory assessment and approval of the investigational product. The program will continue beyond the commercial availability of deucrictibant IR, upon regulatory approval, for up to approximately 6 months to ensure continuity of treatment for participants for whom ongoing therapy is considered appropriate. Participants will self-administer 1 capsule orally at the onset of an HAE attack. If response is inadequate or symptoms worsen or recur, a second capsule may be taken at least 4 hours after the initial dose. Capsule(s) may be taken with or without food.
Interventions
DRUGDeucrictibant IR
Deucrictibant IR capsule administered orally.
Sponsors
Pharvaris Netherlands B.V.
Eligibility
Sex/Gender
ALL
Age
12 Years to 75 Years
Inclusion criteria
* Provision of informed consent: Participants must provide written informed consent. Adolescents (≥12 to \<18 years old, or as per local law) require consent of parent or legally designated representative/guardian and must assent. If an adolescent reaches adulthood during the EAP, they must sign the adult informed consent form (ICF) to remain in the EAP. * Male or female, aged ≥12 to ≤75 years at the time of providing written informed consent/assent. * Confirmed diagnosis of HAE. 1. for participants with hereditary angioedema with normal C1 inhibitor (HAE-nC1INH): documented genetic mutation associated with HAE-nC1INH OR 2. if no documented mutation: * clinical diagnosis with family history of HAE-nC1INH, * attacks not responding to treatment with high-dose antihistamine (cetirizine 40 milligrams \[mg\]/day or equivalent high-dose second-generation antihistamine medication) and no clinical attack symptom relief if treated with corticosteroid, montelukast, and/or omalizumab. * documented effective attack symptom relief with on-demand icatibant treatment * No satisfactory treatment options are available among currently approved HAE therapies for treatment of acute attacks ((i.e. Berinert (C1 esterase inhibitor \[human\]), Ekterly (sebetralstat), Firazyr (icatibant), Kalbitor (ecallantide), Ruconest (C1 esterase inhibitor \[recombinant\])) based on inadequate response, contraindication, safety/tolerability concerns, and/or other reasons, as documented by the treating physician in the participant's medical records and maintained as part of the EAP enrollment documentation. * Not eligible for an ongoing clinical study or for whom participation in a clinical study is not possible or feasible for other reasons, including but not limited to clinical study eligibility criteria, geographic accessibility, study availability, timing considerations, participant decision to participate, or other participant-specific factors, as documented by the treating physician in the participant's medical records and maintained as part of the EAP enrollment documentation. * Residence in the US or US territories.
Exclusion criteria
* Pregnancy or nursing: any female participant who is pregnant, planning to become pregnant during the EAP, or currently breastfeeding. * Any diagnosis of angioedema other than HAE. * Significant comorbidity: any clinically significant comorbidity or systemic dysfunction (e.g., cardiovascular, gastrointestinal, renal, neurologic, respiratory) that, in the opinion of the treating physician, would interfere with the participant's safety or ability to participate in this EAP. * Severe hepatic impairment (Child-Pugh Class C). * Substance abuse: history of alcohol or drug abuse within the past year, or current evidence of substance dependence or abuse. * Prior treatment with deucrictibant IR resulting in discontinuation due to lack of efficacy, safety concerns, or tolerability issues. * Participation in another investigational drug study, or treatment with any investigational drug within 30 days or 5 half-lives (whichever is longer) prior to enrolment. * Prior gene therapy use for any indication at any time. * Current use of medications with systemic absorption that are strong CYP3A4 inhibitors (e.g., clarithromycin, itraconazole, ketoconazole, ritonavir) or strong CYP3A4 inducers (e.g., carbamazepine, phenytoin) within 30 days (or 5 half-lives, whichever longer) prior to enrolment.
Contacts
CONTACTPharvaris Medical Affairs
Outcome results
None listed