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Extension Study of Oral PHA-022121 for Acute Treatment of Angioedema Attacks in Patients with Hereditary Angioedema

Study Purpose

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, in participants with HAE type 1 or type 2 (HAE-1/2). The study will enroll patients from Study PHA022121-C201 (NCT04618211) and Study PHA022121-C306 (NCT06343779) who elect to participate in this extension study and meet the eligibility requirements.

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 and Over
Gender All
More Inclusion & Exclusion Criteria

Key

Inclusion Criteria:

1. Signed and dated informed consent form. 2. Diagnosis of HAE type I or
  • II. 3.
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. 4. In the opinion of the Investigator, the participant (and parent/caregiver for adolescent participants) is willing and able to comply with the protocol. Key

Exclusion Criteria:

1. Pregnancy or breast-feeding. 2. Any other systemic disease or significant disease or disorder that would interfere with the patient's safety or ability to participate in the study. 3. Use of lanadelumab for long-term HAE prophylactic therapy within 12 weeks prior to enrollment in Part A. 4. For Part A: Use of C1-esterase inhibitor, oral kallikrein inhibitors, attenuated androgens, anti-fibrinolytics, or monoclonal HAE therapy within a defined period prior to enrolment. For Part B: If a participant is receiving long-term prophylactic therapy with one of the following medications indicated for HAE: plasma-derived C1-INH, danazol at less than or equal to 200 mg/day, anti-fibrinolytics, berotralstat, or lanadelumab, they must be on a stable dose and regimen for at least 3 months before screening and intends to remain on the same dose for the duration of the study. 5. History of alcohol or drug abuse within defined period, or current evidence of substance dependence or abuse. 6. Participation in any other investigational drug study within defined period. 7. Discontinued from parent study after enrollment for any study drug-related safety reason. 8. Use of concomitant medications that are potent CYP3A4 inhibitors (e.g., clarithromycin, erythromycin, itraconazole, ketoconazole, ritonavir, grapefruit) or potent CYP3A4 inducers (e.g., phenytoin, rifampicin, St. John's Wort).

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.

NCT05396105
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 2/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
Principal Investigator Affiliation Pharvaris Netherlands B.V.
Agency Class

Category of organization(s) involved as sponsor (and collaborator) supporting the trial.

Industry
Overall Status Enrolling by invitation
Countries Bulgaria, Canada, Czechia, France, Germany, Hungary, Israel, Italy, Poland, Spain, 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

Part A of the study will enroll adult participants from Study PHA022121-C201. The double-blind treatment assignment from Study PHA022121-C201 will be maintained. Part B will include participants rolling over from Part A and additionally enroll participants from Study PHA022121-C201 who did not participate in Part A, and participants from Study PHA022121-C306 who elect to participate in this extension study and meet the eligibility requirements.

Arms & Interventions

Arms

Experimental: Part B: Selected dose

Single dose of deucrictibant

Interventions

Drug: - deucrictibant selected dose

deucrictibant soft capsule for oral use

Contact a Trial Team

If you are interested in learning more about this trial, find the trial site nearest to your location and contact the site coordinator via email or phone. We also strongly recommend that you consult with your healthcare provider about the trials that may interest you and refer to our terms of service below.

Study site, Birmingham, Alabama

Status

Address

Study site

Birmingham, Alabama, 35209

Study site, Scottsdale, Arizona

Status

Address

Study site

Scottsdale, Arizona, 85258

Study site, Santa Monica, California

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Address

Study site

Santa Monica, California, 90404

Study site, Walnut Creek, California

Status

Address

Study site

Walnut Creek, California, 94598

Study site, Colorado Springs, Colorado

Status

Address

Study site

Colorado Springs, Colorado, 80907

Study site, Chevy Chase, Maryland

Status

Address

Study site

Chevy Chase, Maryland, 20815

International Sites

Study site, Sofia, Bulgaria

Status

Address

Study site

Sofia, , 1431

Study site, Sofia, Bulgaria

Status

Address

Study site

Sofia, , 1680

Study site, Montréal, Quebec, Canada

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Address

Study site

Montréal, Quebec,

Study site, Brno, Czechia

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Address

Study site

Brno, , 65691

Study site, Grenoble, France

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Address

Study site

Grenoble, , 38043

Study site, Paris, France

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Address

Study site

Paris, , 75010

Study site, Berlin, Germany

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Address

Study site

Berlin, , 10114

Study site, Frankfurt am Main, Germany

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Address

Study site

Frankfurt am Main, , 60596

Study site, Budapest, Hungary

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Address

Study site

Budapest, , 1088

Study site, Ashkelon, Israel

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Address

Study site

Ashkelon, , 78278

Study site, Napoli, Italy

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Address

Study site

Napoli, , 80131

Study site, Kraków, Poland

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Address

Study site

Kraków, , 31-503

Study site, Barcelona, Spain

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Address

Study site

Barcelona, , 08035

Study site, Barcelona, Spain

Status

Address

Study site

Barcelona, , 08907

Study site, Madrid, Spain

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Address

Study site

Madrid, , 28007

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