Open-label study to evaluate the safety, and efficacy of recombinant human C1 inhibitor for the treatment of acute attacks in pediatric patients with hereditary angioedema, from 2 up to and including 13 years of age

Phase 1

• Conditions: Hereditary angioedema (HAE). Hereditary angioedema is characterized by angioedema localized and recurrent caused by by uncontrolled activation of the Complement systems due to congenital deficiency of Functional C1 inhibitor (C1INH). Reports in Medical the literature suggests that C1INH replacement therapy with human plasma-derived represents a safe and effective acute attacks of HA; MedDRA version: 14.1Level: PTClassification code 10019860Term: Hereditary angioedemaSystem Organ Class: 10010331 - Congenital, familial and genetic disorders; Therapeutic area: Diseases [C] - Cardiovascular Diseases [C14]

• Registration Number: EUCTR2011-000987-92-IT
• Lead Sponsor: PHARMING TECHNOLOGIES B.V.

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Study Start: 2011-12-27
• Last Update Posted: 19 February 2018

Recruitment & Eligibility

• Status: ot Recruiting
• Sex: All
• Target Recruitment: 20

Inclusion Criteria

Screening • From 2 up to and including 13 years of age • Clinical and laboratory confirmed diagnosis of HAE (baseline C1INH activity <50% of normal) • Signed written informed consent (parental permission) signed by the legal guardian(s) Treatment • Clinical symptoms of an acute HAE attack • Onset of eligible symptoms within 5 hours from the moment at which medical evaluation to determine eligibility has occurred • IS score for at least one anatomical location at the time of initial evaluation of at least 3 (moderate severity or greater) without signs of spontaneous regression • 24h or more have passed since the patient’s last study treatment
Are the trial subjects under 18? yes
Number of subjects for this age range: 5
F.1.2 Adults (18-64 years) no
F.1.2.1 Number of subjects for this age range
F.1.3 Elderly (>=65 years) no
F.1.3.1 Number of subjects for this age range

Exclusion Criteria

Screening
? A diagnosis of acquired C1INH deficiency (AAE)
? A medical history of allergy to rabbits or rabbit-derived products (including rhC1INH, antisera),
or positive anti-rabbit epithelium (dander) IgE test (cut off>0.35 kU/L in ImmunoCap assay
(Phadia, Sweden) or equivalent)
? Treatment with investigational drug in another clinical study in the last 30 days
? Any clinical significant abnormality in the physical examination and/or the routine laboratory
assessments, that in the opinion of the Investigator makes the patient unsuitable for
participation in the study
? Patient or legal guardian whose decision to participate might be unduly influenced by
perceived expectation of gain or harm by participation, such as patient or legal guardian in
detention due to official or legal order
? Any condition or treatment that in the opinion of the investigator might interfere with the
evaluation of the study objectives
Treatment
? Any changes since screening that would exclude patient based on above exclusion criteria.
? 10 HAE attacks were previously treated with study medication.
? Suspicion for an alternate explanation of the symptoms other than an acute HAE attack.
? Use of any disallowed concomitant medication since onset of acute HAE attack (see Section
8.2.1).
? Positive pregnancy test (urine or serum)

Study & Design

• Study Type: Interventional clinical trial of medicinal product
• Study Design: Not specified