Efficacy and Safety Study of DX-2930 to Prevent Acute Angioedema Attacks in Patients With Type I and Type II HAE

Shire

Status and phase: Completed; Phase 3

Conditions: Hereditary Angioedema (HAE)

Treatments: Drug: DX-2930 - 300mg/2wk; Drug: Placebo; Drug: DX-2930 - 300mg/4wk; Drug: DX-2930 - 150mg/4wk

Study type: Interventional

Funder types: Industry

Identifiers

NCT02586805

2015-003943-20 (EudraCT Number)

DX-2930-03

Details and patient eligibility

About

This is a phase 3, multicenter, randomized, double-blind, placebo-controlled trial to evaluate the efficacy and safety of DX-2930 in preventing acute angioedema attacks in patients with Type I and Type II HAE.

Enrollment: 125 patients

Sex: All

Ages: 12+ years old

Volunteers: No Healthy Volunteers

Inclusion criteria

• Males and females 12 years of age or older at time of screening
• Documented diagnosis of HAE, Type I or II
• Baseline rate of at least 1 Investigator-confirmed HAE attack per 4 weeks
• Adult subjects and caregivers of subjects under the age of 18 are willing and able to read, understand, and sign an informed consent form. Subjects age 12 to 17, whose caregiver provides informed consent, are willing and able to read, understand an dsign an assent form.
• Males and femailes who are fertile and sexually active must adhere to contraception requirements.

Exclusion criteria

• Concomitant diagnosis of another form of chronic, recurrent angioedema, such as acquired angioedema, idiopathic angioedema, or recurrent angioedema associated with urticaria.
• Participation in a prior DX-2930 study
• Treatment with any other investigational drug or exposure to an investigational device within 4 weeks prior screening
• Exposure to angiotensin-converting enzyme (ACE) inhibitors or any estrogen-containing medications within 4 weeks prior to screening.
• Exposure to androgens within 2 weeks prior to entering the run-in period.
• Use of long-term prophylactic therapy for HAE within 2 weeks prior to entering the run-in period.
• Use of short-term prophylaxis for HAE within 7 days prior to entering the run-in period.
• Any of the following liver function test abnormalities: alanine aminotransferase (ALT) > 3x upper limit of normal, or aspartate aminotransferase (AST) > 3x upper limit of normal, or total bilirubin > 2x upper limit of normal (unless the bilirubin elevation is a result of Gilbert's syndrome).
• Pregnancy or breastfeeding.

Trial design

Primary purpose: Prevention

Allocation: Randomized

Interventional model: Parallel Assignment

Masking: Quadruple Blind

---

125 participants in 4 patient groups, including a placebo group

DX-2930 300 mg every 2 weeks

Experimental group

Description:

300 mg DX-2930 administered every 2 weeks by subcutaneous injection.

Treatment:

Drug: DX-2930 - 300mg/2wk

DX-2930 300 mg every 4 weeks

Experimental group

Description:

300 mg DX-2930 administered every 4 weeks by subcutaneous injection

Treatment:

Drug: DX-2930 - 300mg/4wk

DX-2930 150 mg every 4 weeks

Experimental group

Description:

150 mg DX-2930 administered every 4 weeks by subcutaneous injection

Treatment:

Drug: DX-2930 - 150mg/4wk

Placebo

Placebo Comparator group

Description:

Placebo administered every 2 weeks by subcutaneous injection.

Treatment:

Drug: Placebo