Recombinant Human C1 Inhibitor for the Treatment of Acute Attacks in Patients With Hereditary Angioedema

Pharming Technologies B.V.77 enrolled

Overview

Hereditary angioedema ("HAE") is a genetic disorder characterized by sudden recurrent attacks of local swelling (angioedema). These attacks are often painful and disabling, and, in some cases, life-threatening. "HAE" is caused by mutations in the "C1INH" gene that lead to a decrease in the blood level of functional "C1INH". This multi-center study was designed to assess the safety and tolerability, efficacy, and pharmacokinetics/pharmacodynamics of recombinant human C1 inhibitor ("rhC1INH") in the treatment of acute hereditary angioedema attacks. Funding Source - FDA OOPD

A prospectively planned interim analysis will be performed on the double-blind data.

Study Type

INTERVENTIONAL

Allocation

RANDOMIZED

Purpose

TREATMENT

Masking

QUADRUPLE

Enrollment

Conditions

Hereditary Angioedema Angioneurotic Edema

Interventions

Eligibility

Sex: ALLMin age: 12 Years

Medical Language ↔ Plain English

Main Inclusion Criteria: * Clear clinical and laboratory diagnosis of HAE * Plasma level of functional C1INH of less than 50% of normal * Acute abdominal, urogenital, peripheral, and/or oro-facial/pharyngeal/laryngeal HAE attack Main Exclusion Criteria: * Acquired angioedema * Pregnancy or breastfeeding * Treatment with any investigational drug within prior 30 days * Body weight \>120 kg

Outcomes

Primary Outcomes

Time to Beginning of Relief of Symptoms

The time to beginning of relief of symptoms at the location that showed the first visual analogue scale ("VAS") score decrease of at least 20 mm from baseline score with persistence to the next timepoint, assessment timepoints were taken on pre-scheduled time-points after study drug administration: baseline (0 minutes), 15 minutes, 30 minutes, 1 hour, 2 hours, 4 hours, 8 hours, 12 hours, 16 hours, 24 hours and 48 hours. Time to beginning of relief has been calculated as median time, by using the exact timepoints on which each assessment was performed.

Time frame: up to 48 hours after study drug administration

Secondary Outcomes

Time to Minimal Symptoms

The time to minimal symptoms was the time to minimal symptoms for an attack, assessed using the Visual Analogue Scale ("VAS") score. Symptoms were said to be minimal when the "VAS" score at all locations was below 20 mm. Assessment timepoints were: baseline, 15 minutes, 30 minutes, 1 hour, 2 hours, 4 hours, 8 hours, 12 hours, 16 hours, 24 hours and 48 hours. Time to minimal symtoms has been calculated by using the exact timepoints on which each assessment was performed.