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

Phase 3

Completed

Conditions: Hereditary Angioedema (HAE)

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

Registration Number: NCT02586805

Lead Sponsor: Shire

Brief Summary: 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.

Detailed Description: Not available

Recruitment & Eligibility

Status: COMPLETED

Sex: All

Target Recruitment: 125

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.

Study & Design

Study Type: INTERVENTIONAL

Study Design: PARALLEL

Arm && Interventions

Group	Intervention	Description
DX-2930 150 mg every 4 weeks	DX-2930 - 150mg/4wk	150 mg DX-2930 administered every 4 weeks by subcutaneous injection
DX-2930 300 mg every 2 weeks	DX-2930 - 300mg/2wk	300 mg DX-2930 administered every 2 weeks by subcutaneous injection.
Placebo	Placebo	Placebo administered every 2 weeks by subcutaneous injection.
DX-2930 300 mg every 4 weeks	DX-2930 - 300mg/4wk	300 mg DX-2930 administered every 4 weeks by subcutaneous injection

Primary Outcome Measures

Name	Time	Method
Rate of Investigator Confirmed Hereditary Angioedema (HAE) Attacks During Treatment Period	From Day 0 to Day 182	HAE attack was defined as a discrete episode during which the participant progressed from no angioedema to symptoms of angioedema. Rate of investigator confirmed HAE attacks was analyzed using a generalized linear model (GLM) for count data assuming a poisson distribution with a log link function and Pearson chi-square scaling of standard errors to account for potential overdispersion. The logarithm of time in days each subject was observed during the treatment period was used as an offset variable in the model.

Secondary Outcome Measures

Name	Time	Method
Rate of Investigator Confirmed Hereditary Angioedema (HAE) Attack Requiring Acute Treatment	From Day 0 to Day 182	HAE attack was defined as a discrete episode during which the participant progressed from no angioedema to symptoms of angioedema. Rate of investigator confirmed HAE attack was analyzed using the GLM for count data assuming a poisson distribution with a log link function and Pearson chi-square scaling of standard errors to account for potential overdispersion. The logarithm of time in days each subject was observed during the treatment period was used as an offset variable in the model.
Rate of Investigator Confirmed Hereditary Angioedema (HAE) Attacks During Day 14 Through Day 182	From Day 14 to Day 182	HAE attack was defined as a discrete episode during which the participant progressed from no angioedema to symptoms of angioedema. Rate of investigator confirmed HAE attacks during day 14 after study drug administration through day 182 was analyzed by the same poisson regression model as in the primary endpoint analysis.
Rate of Moderate or Severe Investigator Confirmed Hereditary Angioedema (HAE) Attacks	From Day 0 to Day 182	HAE attack was defined as a discrete episode during which the participant progressed from no angioedema to symptoms of angioedema. Moderate and severe investigator-confirmed HAE attacks were the attacks that were moderate or severe as per the HAE attack assessment and reporting procedures (HAARP) defined severity. The overall severity of attack was determined by the investigator using following definitions: mild (transient or mild discomfort), moderate (mild to moderate limitation in activity), severe (marked limitation in activity). Rate of moderate or severe investigator confirmed HAE attack was analyzed using the GLM for count data assuming a poisson distribution with a log link function and Pearson chi-square scaling of standard errors to account for potential overdispersion. The logarithm of time in days each subject was observed during the treatment period was used as an offset variable in the model.

Trial Locations

Locations (41): Clinical Research Center of Alabama, LLC
🇺🇸
Birmingham, Alabama, United States
Medical Research of Arizona
🇺🇸
Scottsdale, Arizona, United States
UC San Diego School of Medicine
🇺🇸
San Diego, California, United States
AIRE Medical of Los Angeles
🇺🇸
Santa Monica, California, United States
Allergy & Asthma Clinical Research
🇺🇸
Walnut Creek, California, United States
IMMUNOe International Health & Research Centers
🇺🇸
Centennial, Colorado, United States
Asthma and Allergy Associates, P.C.
🇺🇸
Colorado Springs, Colorado, United States
University of South Florida
🇺🇸
Tampa, Florida, United States
University of Kansas Medical Center
🇺🇸
Kansas City, Kansas, United States
Institute of Asthma & Allergy, P.C.
🇺🇸
Chevy Chase, Maryland, United States
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Clinical Research Center of Alabama, LLC
🇺🇸Birmingham, Alabama, United States