Dose-ranging Study of Oral PHA-022121 for Acute Treatment of Angioedema Attacks in Patients With Hereditary Angioedema (RAPIDe-1)

Pharvaris

Status and phase: Completed; Phase 2

Conditions: Hereditary Angioedema - Type 2; Hereditary Angioedema Types I and II; Hereditary Angioedema With C1 Esterase Inhibitor Deficiency; Hereditary Angioedema Type I; Hereditary Angioedema Type II; C1 Inhibitor Deficiency; C1 Esterase Inhibitor Deficiency; Hereditary Angioedema - Type 1; Hereditary Angioedema Attack; Hereditary Angioedema

Treatments: Drug: Placebo; Drug: Deucrictibant

Study type: Interventional

Funder types: Industry

Identifiers

NCT04618211

2020-003445-11 (EudraCT Number)

PHA022121-C201

Details and patient eligibility

About

This study evaluates the efficacy of orally administered deucrictibant for the acute treatment of attacks in patients with hereditary angioedema (HAE). Eligible subjects are randomized to one of three single doses of deucrictibant and placebo. The study will compare symptom relief (skin pain, skin swelling, abdominal pain) during HAE attacks and safety of each dose of deucrictibant with placebo.

Full description

In Part I of the study, patients in non-attack state receive the assigned active single dose of deucrictibant at the study center to assess pharmacokinetics (the way the body absorbs, distributes, and gets rid of the drug) and safety. In Part II of the study, patients self-administer blinded study drug at home to treat three HAE attacks with deucrictibant or placebo (cross-over).

Enrollment: 74 patients

Sex: All

Ages: 18 to 75 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. Documented history of HAE attacks: at least three in the last 4 months, or at least two in the last 2 months prior to screening
4. Reliable access and experience to use standard of care acute attack medications

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 enrollment
5. Positive serology for HIV or active infection with hepatitis B virus or hepatitis C virus
6. Abnormal hepatic function
7. Abnormal renal function
8. History of alcohol or drug abuse within defined period, or current evidence of substance dependence or abuse
9. History of documented severe hypersensitivity to any medicinal product
10. Participation in any other investigational drug study within defined period

Trial design

Primary purpose: Treatment

Allocation: Randomized

Interventional model: Crossover Assignment

Masking: Quadruple Blind

---

74 participants in 3 patient groups

Low dose/placebo

Other group

Description:

Single low dose of deucrictibant or placebo

Treatment:

Drug: Placebo

Drug: Deucrictibant

Medium dose/placebo

Other group

Description:

Single medium dose of deucrictibant or placebo

Treatment:

Drug: Placebo

Drug: Deucrictibant

High dose/placebo

Other group

Description:

Single high dose of deucrictibant or placebo

Treatment:

Drug: Placebo

Drug: Deucrictibant