EDEMA2: An Open-Label Study to Assess the Efficacy and Tolerability of Repeated Doses of DX-88 (Recombinant Plasma Kallikrein Inhibitor) in Patients With Hereditary Angioedema
Trial Snapshot
- Phase
- Phase 2
- Status
- Completed
- Sponsor
- Shire
- Enrollment
- 77
- Primary Endpoint
- Proportion of attacks treated with successful outcome
Study Overview
Brief Summary
EDEMA2 is an open-label, Phase 2 dose-ranging study designed to assess the safety and efficacy of repeated dosing of DX-88 (recombinant plasma kallikrein inhibitor) in Patients with Hereditary Angioedema.
Study Design
- Study Type
- Interventional
- Allocation
- Non Randomized
- Intervention Model
- Single Group
- Primary Purpose
- Treatment
- Masking
- None
Eligibility Criteria
- Ages
- 10 Years to — (Child, Adult, Older Adult)
- Sex
- All
- Accepts Healthy Volunteers
- No
Inclusion Criteria
- •10 years of age or older
- •Documented diagnosis of HAE (Type I or II)
- •Patient reported to a study site no later than 4 hours following patient recognition of the onset of the attack
- •Willing and able to give informed consent
Exclusion Criteria
- •Patients with a serious intercurrent illness or serious active infection
- •Patient with serum creatinine greater than 110% the upper limit of normal or liver transaminases 2 times the upper limit of normal
- •Receipt of an investigational drug or device, within 30 days prior to study treatment
- •Pregnancy or breastfeeding
- •Diagnosis of acquired angioedema (AAE)
- •Patients who had not completed their Day-7 follow-up procedures for a previously treated attack
Arms & Interventions
30 mg DX-88 SC
30mg DX-88(ecallantide)administered subcutaneously
Intervention: DX-88 (ecallantide) (Drug)
5mg/m2 DX-88 IV
5mg/m2 DX-88 (ecallantide)administered intravenously
Intervention: DX-88 (ecallantide) (Drug)
10mg/m2 DX-88 IV
10mg/m2 DX-88(ecallantide)administered intravenously
Intervention: DX-88 (ecallantide) (Drug)
20mg/m2 DX-88 IV
20mg/m2 DX-88 (ecallantide) administered intravenously
Intervention: DX-88 (ecallantide) (Drug)
Outcomes
Primary Outcomes
Proportion of attacks treated with successful outcome
Time Frame: 24 hours
successful outcome is defined as attack resolution begun within 4 hrs after treatment and maintained for 24 hours
Proportion of attacks with a partial response
Time Frame: 24 hours
partial response is defined as an initial response to dosing followed by a relapse within 4 to 24 hours
Secondary Outcomes
- Proportion of patients who respond to a 2nd dose of ecallantide after an initial, partial response(24 hours)
- Time to resolution onset of each acute attack, as determined by patient report(24 hours)