Safety of Ruconest in children with HAE

• Register: EUCTR
• Last refreshed on: 4 January 2022
• Main ID: EUCTR2011-000987-92-CZ
• Date of registration: 17/04/2014
• Prospective Registration: Yes
• Primary sponsor: Pharming Technologies B.V.
• Public title: Safety of Ruconest in children with HAE
• Scientific title: Open-label, phase II, single arm study to evaluate the safety, immunogenicity, pharmacokinetics 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 - Safety of Ruconest in 2-13 year old HAE patients
• Date of first enrolment: 02/10/2014
• Target sample size: 40
• Recruitment status: Not Recruiting
• URL: https://www.clinicaltrialsregister.eu/ctr-search/search?query=eudract_number:2011-000987-92
• Study type: Interventional clinical trial of medicinal product
• Study design: Controlled: no; Randomised: no; Open: yes; Single blind: no; Double blind: no; Parallel group: no; Cross over: no; Other: no; If controlled, specify comparator, Other Medicinial Product: no; Placebo: no; Other: no
• Phase: Human pharmacology (Phase I): no Therapeutic exploratory (Phase II): yes Therapeutic confirmatory - (Phase III): no Therapeutic use (Phase IV): no

Countries of recruitment

Czech Republic, Germany, Hungary, Israel, Italy, Slovakia

Contacts

• Name: Tessa Heimap - van Rossenberg
• Affiliation: Pharming Technologies B.V.

• Name: Tessa Heimap - van Rossenberg
• Affiliation: Pharming Technologies B.V.

Key inclusion & exclusion criteria

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: 20
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
• 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


Age minimum:
Age maximum:
Gender:
Female: yes
Male: yes

Health Condition(s) or Problem(s) studied

Attacks associated with hereditary angioedema (HAE) in pediatric population 2-13 years of age.
MedDRA version: 18.1 Level: PT Classification code 10019860 Term: Hereditary angioedema System Organ Class: 10010331 - Congenital, familial and genetic disorders

Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]

Intervention(s)

Trade Name: Ruconest
Product Name: Ruconest
Pharmaceutical Form: Powder for solution for injection
INN or Proposed INN: CONESTAT ALFA
CAS Number: 80295-38-1
Concentration unit: U unit(s)
Concentration type: range
Concentration number: 50 U/kg BW-4200 U-

Primary Outcome(s)

Main Objective: To assess the clinical safety, immunogenicity and tolerability of Ruconest in the treatment of acute angioedema attacks in 2-13 year old HAE patients.

Timepoint(s) of evaluation of this end point: This will be evaluated by continuous recording adverse events.

Primary end point(s): Assessment of safety and tolerability.

Secondary Objective: To assess the pharmacokinetics (PK) and pharmacodynamics (PD) of Ruconest in the treatment of acute angioedema attacks in 2-13 year old HAE patients.

To assess the efficacy of Ruconest in the treatment of acute angioedema attacks in 2-13 year old HAE patients.

Secondary Outcome(s)

Timepoint(s) of evaluation of this end point: The clinical endpoints will be evaluated following the treatment of an attack. Blood samples for PK and PD will be taken at 5 minutes and between 2-4 hours after the Ruconest injection at the first attack.

Secondary end point(s): Time to beginning of relief, time to minimal symptoms, time to complete resolution.
Pharmacokinetic and pharmacodynamic parameters (C1INH activity and C4 in plasma) during treatment for the first attack.

Secondary ID(s)

C11209

2011-000987-92-DE

Source(s) of Monetary Support

Pharming Technologies B.V.

Ethics review

Status: Approved
Approval date: 02/10/2014
Contact:

Results

• Results available: Yes
• Date Posted: 08/03/2018
• Date Completed: 17/07/2017
• URL: https://www.clinicaltrialsregister.eu/ctr-search/trial/2011-000987-92/results