Efficacy and safety of Ruconest in the prevention of angioedema attacks in patients with Hereditary Angioedema (HAE).

Phase 1

• Conditions: Prophilaxis of Angioedema Attacks on Patients with Hereditary Angioedema (HAE).; MedDRA version: 23.1Level: PTClassification code 10019860Term: Hereditary angioedemaSystem Organ Class: 10010331 - Congenital, familial and genetic disorders; MedDRA version: 20.0Level: HLGTClassification code 10027664Term: Congenital and hereditary disorders NECSystem Organ Class: 10010331 - Congenital, familial and genetic disorders; Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]

• Registration Number: EUCTR2014-002839-33-IT
• Lead Sponsor: PHARMING GROUP NV

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Results First Posted: 1900-01-01
• Study Completion: 2016-05-03
• Study Start: 2021-01-21
• Last Update Posted: 17 August 2021

Recruitment & Eligibility

• Status: ot Recruiting
• Sex: All
• Target Recruitment: 32

Inclusion Criteria

1. Males or non-pregnant females, 13 years of age or older
2. Laboratory confirmed diagnosis of HAE (functional C1INH level <50% of normal using a chromogenic assay)
3. A history of frequent HAE attacks (at least 4 attacks per month across a minimum of 3 consecutive months). HAE attack history
to be confirmed in the patient’s medical record or during intake of HAE attack history at Screening.
4. Female patients of childbearing potential who are sexually active must be willing to use an acceptable form of contraception.
Acceptable forms of contraception are defined as those with a failure rate < 1% when properly applied and include: a combination
oral pill, some intra-uterine devices, and a sterilized partner in a stable relationship. Female patients must not be pregnant,
planning to become pregnant, or be actively breastfeeding through the entire study period.
5. Provided written informed consent (and written assent for minors)
6. Willingness and ability to comply with all protocol procedures
Are the trial subjects under 18? yes
Number of subjects for this age range:
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 20
F.1.3 Elderly (>=65 years) yes
F.1.3.1 Number of subjects for this age range 5

Exclusion Criteria

1. Patients with medical history of allergy to rabbits or rabbit-derived products (including rhC1INH)
2. Diagnosis of acquired angioedema (AAE) including positive anti-C1INH antibodies with a low C1q level and/or clinical features
suggestive of AAE
3. Patients who are pregnant, or breastfeeding, or are currently intending to become pregnant
4. Treatment with any investigational drug in the past 30 days
5. Patients with any condition or treatment that, in the opinion of the Investigator, might interfere with the evaluation of study
objectives
6. Patients currently treated with angiotensin-converting enzyme (ACE) inhibitors
7. Patients who are using pdC1INH for prophylaxis or acute treatment of HAE attacks will need to discontinue therapy for at least 7
days prior to their first visit of Period 1. Patients may treat any HAE attacks between Screening and Randomization with open-label
rhC1INH, HAE-specific medications (icatibant, ecallantide), or other symptomatic medications (narcotics, anti-emetics) as required.

Study & Design

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

Primary Outcome Measures

Name	Time	Method
Main Objective: To evaluate the efficacy of rhC1INH in the prophylaxis of angioedema attacks in patients with HAE.;Secondary Objective: To evaluate the safety of rhC1INH in the prophylaxis of angioedema attacks in patients with HAE.;Primary end point(s): The primary endpoint is the number of HAE attacks during each 28 days treatment period.;Timepoint(s) of evaluation of this end point: All endpoints will be evaluated in each study period.

Secondary Outcome Measures

Name	Time	Method
Secondary end point(s): The secondary endpoint the proportion of<br>subjects who experience a clinical response, defined as a = 50% reduction in the number of attacks from the placebo treatment period to the active study drug treatment period.;Timepoint(s) of evaluation of this end point: All endpoints will be evaluated in each study period.