The medical condition to be investigated is idiopathic non-histaminergic angioedema (InH-AAE). This disease is characterized by recurrent episodes with swelling of the subcutis and mucous membranes. Patient with InH-AAE have by defenition no wheals and are nonresponsive to anti-histamine therapy.
Conditions
Interventions
Trade Name: Ruconest 2100 U powder for solution for injection
Product Name: Ruconest
Pharmaceutical Form: Powder for solution for injection
Sponsors
University Medical Center Utrecht
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: Age = 18 years. Diagnosis idiopathic non-histaminergic acquired angioedema (InH-AAE) were “non-histaminergic” is defined as following: insufficient effect of treatment with antihistamines up to 4 times the standard dose (step 2 in CSU-treatment regimen), defined as having breakthrough attacks. Minimal mean attack rate of 2 per month during the past six months despite treatment; with at least one attack in the month prior to inclusion. Normal C4 levels. Written informed consent. Are the trial subjects under 18? no 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 10 F.1.3 Elderly (>=65 years) no F.1.3.1 Number of subjects for this age range 0
Exclusion criteria
Exclusion criteria: Presence of recurrent wheals/ urticaria accompanying angioedema. Diagnosis other than InH-AAE is deemed more likely. ? ACE-inhibitor use in the past 6 months. History suggesting allergy for rabbits or rabbit derived products (such as conestat alfa). Currently trying to conceive, pregnancy and women giving breastfeeding. Inability to comply with study and follow-up procedures. Presence of clinically significant conditions that could interfere with the interpretation of the study results and or compromise the safety of the patients. Participation in an investigational drug or device trial within the last 30 days prior to screening.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: The primary objective of this exploratory study is to test if prophylactic treatment with conestat alfa decreases the frequency of angioedema attacks in patients with idiopathic non-histaminergic acquired angioedema. ;Secondary Objective: To test if prophylactic use of conestat alfa decreases disease activity in patients with InH-AAE. To test if prophylactic use of conestat alfa improves the quality of life in patients with InH-AAE. To establish the safety of prophylactic use of Conestat alfa in patients with InH-AAE. To examine potential biomarkers for disease activity, response to treatment and predictive value for response to treatment. ;Primary end point(s): The primary endpoint of this study is the mean monthly attack frequency during the 8-week treatment period as reported by the patient on the AAS form. This primary parameter will be compared with the patient reported mean monthly attack frequency over the past 6 months (primary outcome) and the mean monthly attack frequency during the 8-week observational period. ;Timepoint(s) of evaluation of this end point: Daily by AAS & at initiation visit. | — |
Secondary
| Measure | Time frame |
|---|---|
| Secondary end point(s): The mean monthly disease severity as measured by AAS28 or AAS7. The quality of life measured by AE-QoL. Changes in the UCT score. Number of adverse and serious adverse events. Levels of biomarkers.;Timepoint(s) of evaluation of this end point: AAS: daily AE-QoL&UCT: montly Adverse events: Montly during obervation and twice weekly during the 8-weeks of therapy. Biomarkers: At the first visit (V1), last visit (V18) and during the treatment period at V2, V4, V9 and V17. | — |
Countries
Netherlands
Contacts
Public ContactZonne Hofman
University Medical Center Utrecht
Outcome results
None listed