Study to evaluate the response and efficiency of the study medication Cinryze for treatment of acute angiodema attacks (Quincke-oedema) in children less than 12 years of age with hereditary angiooedema.
- Conditions
- Acute angioedema attacks in children less than 12 years of age with hereditary angioedema.MedDRA version: 16.0Level: LLTClassification code 10037735Term: Quincke's oedemaSystem Organ Class: 100000004858Therapeutic area: Diseases [C] - Immune System Diseases [C20]
- Registration Number
- EUCTR2011-000369-11-HU
- Lead Sponsor
- ViroPharma Incorporated
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- ot Recruiting
- Sex
- All
- Target Recruitment
- 12
1. Be children (male or female), =2 and <12 years of age.
2. Be at least 10 kg of body weight.
3. Have a confirmed diagnosis of HAE with at least one of the following:
- C1 INH antigen level below normal
- Functional C1 INH level below normal
4. Have an acute HAE attack and be able to initiate study drug treatment within 8 hours after onset of symptoms.
5. Have a parent/legal guardian who is willing and able to provide written informed consent for the child to participate in the study (with assent from the child when appropriate).
Are the trial subjects under 18? yes
Number of subjects for this age range: 12
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
1. Have any active infectious illness (e.g., flu, upper respiratory tract infection, etc.).
2. Have had a prior HAE attack and/or received any C1 INH product within 7 days prior to dosing with study drug.
3. Have received therapy with antifibrinolytics (e.g., tranexamic acid), androgens (e.g., danazol, oxandrolone, stanozolol, or testosterone), ecallantide (Kalbitor®), or icatibant (Firazyr®) within 7 days prior to dosing with study drug.
4. Have a history of allergic reaction to C1 INH products, including Cinryze (or any of the components of Cinryze), or other blood products.
5. Have participated in any other investigational drug evaluation within 30 days prior to dosing with study drug, or have previously received treatment with Cinryze in this study at any time.
6. Have, as determined by the investigator and/or the sponsor’s medical monitor, any surgical or medical condition that could interfere with the administration of study drug, interpretation of study results, or compromise the safety or well-being of the subject.
7. If female, be pregnant or breastfeeding.
8. Be suspected of having an alternate explanation for their symptoms other than acute HAE attack.
9. Have a history of narcotic-seeking behavior and/or drug/alcohol abuse.
Study & Design
- Study Type
- Interventional clinical trial of medicinal product
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method Main Objective: The objectives of the study are to evaluate (1) the dose response and (2) the PK/PD of IV administration of Cinryze for the treatment of acute angioedema attacks in children above and below 25 kg and less than 12 years of age with HAE; and (3) to determine the safety and tolerability following IV administration of Cinryze in this study population.;Secondary Objective: Not applicable;Primary end point(s): The primary efficacy endpoint is the presence of unequivocal beginning of relief of the defining symptom within 4 hours following initial treatment with Cinryze. This endpoint will be determined for all subjects in the efficacy analyses, and will be presented separately for subjects in the different weight/dose categories.;Timepoint(s) of evaluation of this end point: Within 4 hours following initial treatment.
- Secondary Outcome Measures
Name Time Method Secondary end point(s): Secondary efficacy endpoints will include:<br>• Time to unequivocal beginning of relief of the defining symptom<br>• Time to complete resolution of the attack<br>These endpoints will be determined for all subjects in the efficacy analyses, and will be presented separately for subjects in the different weight/dose categories.;Timepoint(s) of evaluation of this end point: Time to complete resolution of the attack.