Extension Study of Oral PHA-022121 for Acute Treatment of Angioedema Attacks in Patients With Hereditary Angioedema (RAPIDe-2)

Pharvaris

Status and phase: Enrolling; Phase 3; Phase 2

Conditions: Hereditary Angioedema - Type 2; Hereditary Angioedema Types I and II; Hereditary Angioedema With C1 Esterase Inhibitor Deficiency; C1 Esterase Inhibitor, Deficiency of; C1 Esterase Inhibitor Deficiency; C1 Esterase Inhibitor [C1-INH] Deficiency; Hereditary Angioedema Type I; C1 Inhibitor Deficiency; Hereditary Angioedema Type II; Hereditary Angioedema - Type 1; Hereditary Angioedema Attack; Hereditary Angioedema

Treatments: Drug: deucrictibant low dose; Drug: deucrictibant selected dose; Drug: deucrictibant high dose; Drug: deucrictibant medium dose

Study type: Interventional

Funder types: Industry

Identifiers

NCT05396105

PHA022121-C303

Details and patient eligibility

About

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 patients with HAE due to C1-esterase inhibitor (C1-INH) deficiency (type I/II). The study will enroll patients from Study PHA022121-C201 (NCT04618211) who elect to participate in this extension study and meet the eligibility requirements.

Full description

In Part A of the study, the double-blind treatment assignment from Study PHA022121-C201 will be maintained. The treatment in Part A will consist of 3 soft capsules per administered dose as in Study PHA022121-C201. In Part B of the study, the selected dose and formulation of deucrictibant will be administered.

The to-be-marketed deucrictibant formulation will be one single soft capsule at the strength proposed for marketing, based on the unblinding and evaluation of clinical data from Study PHA022121-C201. The duration of the treatment period (Part A plus Part B) is dependent upon the time of patient enrollment. The study is planned to continue until the availability of commercial supply, or another means of continued treatment can be provided.

Enrollment: 72 estimated patients

Sex: All

Ages: 18+ years old

Volunteers: No Healthy Volunteers

Inclusion and exclusion criteria

Key Inclusion Criteria:

1. Signed and dated informed consent form
2. Diagnosis of HAE type I or II
3. must have received at least 1 dose of study drug (including the non-attack visit) in Study PHA022121-C201.

Key Exclusion Criteria:

1. Pregnancy or breast-feeding
2. Clinically significant abnormal electrocardiogram
3. Any other systemic disease or significant disease or disorder that would interfere with the patient's safety or ability to participate in the study
4. Use of C1-esterase inhibitor, oral kallikrein inhibitors, attenuated androgens, anti-fibrinolytics, or monoclonal HAE therapy within a defined period prior to enrolment
5. History of alcohol or drug abuse within defined period, or current evidence of substance dependence or abuse
6. Discontinued from Study PHA022121-C201 after enrollment for any study drug-related safety reason.
7. 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).
8. Participation in any other investigational drug study within defined period

Trial design

Primary purpose: Treatment

Allocation: Non-Randomized

Interventional model: Sequential Assignment

Masking: Quadruple Blind

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72 participants in 4 patient groups

Part A: Low dose

Experimental group

Description:

Single low dose of deucrictibant

Treatment:

Drug: deucrictibant low dose

Part A: Medium dose

Experimental group

Description:

Single medium dose of deucrictibant

Treatment:

Drug: deucrictibant medium dose

Part A: High dose

Experimental group

Description:

Single high dose of deucrictibant

Treatment:

Drug: deucrictibant high dose

Part B: Selected dose

Experimental group

Description:

Single dose of deucrictibant

Treatment:

Drug: deucrictibant selected dose