Study of Oral Deucrictibant Soft Capsule for On-Demand Treatment of Angioedema Attacks in Adolescents and Adults With Hereditary Angioedema

Study Purpose

This is a Phase 3, multicenter, randomized, double-blind, placebo-controlled, 2-period, 2-treatment cross-over study to evaluate the efficacy and safety of orally administered deucrictibant compared to placebo for the on-demand treatment of HAE attacks, including non-severe laryngeal attacks, in participants ≥12 to ≤75 years of age with HAE type 1 or type 2 (HAE-1/2), a proportion of whom are using long-term prophylactic medication for HAE.

Recruitment Criteria

Accepts Healthy Volunteers Healthy volunteers are participants who do not have a disease or condition, or related conditions or symptoms	No
Study Type An interventional clinical study is where participants are assigned to receive one or more interventions (or no intervention) so that researchers can evaluate the effects of the interventions on biomedical or health-related outcomes. An observational clinical study is where participants identified as belonging to study groups are assessed for biomedical or health outcomes. Searching Both is inclusive of interventional and observational studies.	Interventional
Eligible Ages	12 Years - 75 Years
Gender	All

More Inclusion & Exclusion Criteria

Inclusion Criteria:

1. Provision of written informed consent/assent. 2. Male or female, aged ≥12 to ≤75 years at the time of providing written informed consent/assent. 3. Diagnosis of HAE-1/2. 4. History of at least 2 HAE attacks in the last 3 months before screening. 5. Experience with using standard-of-care treatment to effectively manage on-demand treatment for HAE attacks. 6. Participants on long-term prophylactic therapy with plasma-derived C1-INH (danazol, anti-fibrinolytics, berotralstat, or lanadelumab) must be on a stable dose and regimen and intend to remain on the same dose for 6 months before screening and the duration of the study. OR, Participant has stopped using plasma-derived C1-INH (danazol, anti-fibrinolytics, berotralstat) at least 2 weeks or lanadelumab at least 10 weeks before screening. 7. Capable of recording, without assistance, electronic HAE diary and ePRO data using an electronic device. 8. For adolescent participants aged ≥12 and <18 years of age: body weight ≥40 kg. 9. Female participants of childbearing potential must agree to the protocol specified pregnancy testing and contraception methods.

Exclusion Criteria:

1. Any female who is pregnant, plans to become pregnant, or is breastfeeding. 2. Any diagnosis of angioedema other than HAE-1/2. 3. Any clinically significant comorbidity or systemic dysfunction that would interfere with the participant's safety or ability to participate in the study. 4. Use of attenuated androgens for short-term prophylaxis within 2 weeks before screening. 5. Abnormal hepatic function. 6. Abnormal renal function (eGFR <60 ml/min/1.73 m2). 7. History of alcohol or drug abuse within the previous year, or current evidence of substance dependence or abuse. 8. Has received prior on-demand HAE treatment with deucrictibant. 9. Currently participating in any other investigational drug study or receiving other investigational treatment within the last 30 days, or within 5 half-lives (whichever is longer) of the time of randomization. 10. Prior gene therapy for any indication at any time. 11. Use of concomitant medications with systemic absorption that are strong inhibitors of CYP3A4 or strong inducers of CYP3A4 within the last 30 days, or within 5 half-lives (whichever is longer) of the time of randomization. 12. Known hypersensitivity to study drug or any of the excipients of study drug.

Trial Details

Trial ID: This trial id was obtained from ClinicalTrials.gov, a service of the U.S. National Institutes of Health, providing information on publicly and privately supported clinical studies of human participants with locations in all 50 States and in 196 countries.	NCT06343779
Phase Phase 1: Studies that emphasize safety and how the drug is metabolized and excreted in humans. Phase 2: Studies that gather preliminary data on effectiveness (whether the drug works in people who have a certain disease or condition) and additional safety data. Phase 3: Studies that gather more information about safety and effectiveness by studying different populations and different dosages and by using the drug in combination with other drugs. Phase 4: Studies occurring after FDA has approved a drug for marketing, efficacy, or optimal use.	Phase 3
Lead Sponsor The sponsor is the organization or person who oversees the clinical study and is responsible for analyzing the study data.	Pharvaris Netherlands B.V.
Principal Investigator The person who is responsible for the scientific and technical direction of the entire clinical study.	Study Director, Pharvaris
Principal Investigator Affiliation	Pharvaris Netherlands B.V.
Agency Class Category of organization(s) involved as sponsor (and collaborator) supporting the trial.	Industry
Overall Status	Recruiting
Countries	Argentina, Australia, Austria, Bulgaria, Canada, Colombia, Czechia, France, Germany, Hong Kong, Hungary, Ireland, Italy, Japan, Korea, Republic of, Netherlands, North Macedonia, Poland, Puerto Rico, Romania, Saudi Arabia, Singapore, South Africa, Spain, Sweden, Turkey, United Kingdom, United States
Conditions The disease, disorder, syndrome, illness, or injury that is being studied.	Hereditary Angioedema, Hereditary Angioedema Type I, Hereditary Angioedema Type II, Hereditary Angioedema Types I and II, Hereditary Angioedema Attack, Hereditary Angioedema With C1 Esterase Inhibitor Deficiency, Hereditary Angioedema - Type 1, Hereditary Angioedema - Type 2, C1 Esterase Inhibitor [C1-INH] Deficiency, C1 Esterase Inhibitor Deficiency, C1 Esterase Inhibitor, Deficiency of, C1 Inhibitor Deficiency

Additional Details

The study consists of a Screening Phase during which eligibility is confirmed, a Treatment Phase in which participants will be randomized and receive double blinded study drug to treat 2 qualifying HAE attacks (i.e., 2 Treatment Periods within the Treatment Phase), and an End-of-Study Follow-up Phase after the second attack treated with study drug. In addition, for adolescent participants (age ≥12 to <18 years), PK samples are collected after administration of deucrictibant at Day 1 in a non-attack state.

Arms & Interventions

Arms

Experimental: Arm 1

Deucrictibant administered for first HAE attack, placebo administered for second HAE attack.

Experimental: Arm 2

Placebo administered for first HAE attack, deucrictibant administered for second HAE attack.

Interventions

Drug: - Deucrictibant, Placebo

Deucrictibant Soft Capsules for Oral Use

Contact a Trial Team

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Study Site, Birmingham, Alabama

Status

Recruiting

Address

Study Site

Birmingham, Alabama, 35209

Site Contact

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