EDEMA2: Evaluation of DX-88's Effect in Mitigating Angioedema

Study Overview
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Brief Summary
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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.

Official Title
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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

Conditions
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Hereditary Angioedema (HAE)

Intervention / Treatment
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* Drug: DX-88 (ecallantide)


Participation Criteria
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Eligibility Criteria
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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

Ages Eligible for Study
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Minimum Age: 10 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Allocation: Non-Randomized
Intervention Model: Single Group Assignment
Masking: None (Open Label)


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: 5mg/m2 DX-88 IV<br>5mg/m2 DX-88 (ecallantide)administered intravenously | Drug: DX-88 (ecallantide)<br>* solution for injection 10 mg/mL<br>|
| Experimental: 10mg/m2 DX-88 IV<br>10mg/m2 DX-88(ecallantide)administered intravenously | Drug: DX-88 (ecallantide)<br>* solution for injection 10 mg/mL<br>|
| Experimental: 20mg/m2 DX-88 IV<br>20mg/m2 DX-88 (ecallantide) administered intravenously | Drug: DX-88 (ecallantide)<br>* solution for injection 10 mg/mL<br>|
| Experimental: 30 mg DX-88 SC<br>30mg DX-88(ecallantide)administered subcutaneously | Drug: DX-88 (ecallantide)<br>* solution for injection 10 mg/mL<br>|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Proportion of attacks treated with successful outcome | successful outcome is defined as attack resolution begun within 4 hrs after treatment and maintained for 24 hours | 24 hours | 
| Proportion of attacks with a partial response | partial response is defined as an initial response to dosing followed by a relapse within 4 to 24 hours | 24 hours | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| 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 |