Phase 3 N=125 Randomized Quadruple-blind Prevention

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

Hereditary Angioedema (HAE)

Bottom Line

View on ClinicalTrials.gov: NCT02586805 ↗

Enrolled (actual)

125

Serious AEs

4.8%

Results posted

Apr 2018

Primary outcome: Primary: Rate of Investigator Confirmed Hereditary Angioedema (HAE) Attacks During Treatment Period — 1.967; 0.480; 0.526; 0.257 Attacks per 4 weeks — p=<0.001

Study Design & Population

Study type: Interventional
Phase: Phase 3
Interventions: DX-2930 - 300mg/2wk (Drug); DX-2930 - 300mg/4wk (Drug); DX-2930 - 150mg/4wk (Drug); Placebo (Drug)
Age: Pediatric, Adult, Older Adult · 12+ yrs
Sex: All
Sponsor: Shire
Primary completion: Apr 2017

Outcome Measures

Outcome	Result	p-value
PRIMARY Rate of Investigator Confirmed Hereditary Angioedema (HAE) Attacks During Treatment Period	1.967; 0.480; 0.526; 0.257	<0.001 sig
SECONDARY Rate of Investigator Confirmed Hereditary Angioedema (HAE) Attack Requiring Acute Treatment	1.637; 0.314; 0.423; 0.208	<0.001 sig
SECONDARY Rate of Moderate or Severe Investigator Confirmed Hereditary Angioedema (HAE) Attacks	1.216; 0.359; 0.325; 0.202	<0.001 sig
SECONDARY Rate of Investigator Confirmed Hereditary Angioedema (HAE) Attacks During Day 14 Through Day 182	1.988; 0.445; 0.489; 0.218	<0.001 sig

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.

Eligibility Criteria

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.

View full record on ClinicalTrials.gov →

Data sourced from ClinicalTrials.gov (NCT02586805). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.