A Phase IIIb randomized, double-blind, placebo-controlled study with an open-label extension evaluating the efficacy, safety and immunogenicity of recombinant human C1 inhibitor for the treatment of acute attacks of angioedema in patients with HAE - Phase III study of recombinant human C1 inhibitor
- Conditions
- Treatment of acute angioedema attacks in patients with hereditary angioedema (HAE).MedDRA version: 12.1Level: LLTClassification code 10019860Term: Hereditary angioedema
- Registration Number
- EUCTR2011-000049-19-HU
- Lead Sponsor
- Pharming Technologies B.V.
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- ot Recruiting
- Sex
- All
- Target Recruitment
- 50
Inclusion Criteria at Screening:
1. Aged at least 18 years
2. Signed written informed consent
3. Clear clinical and laboratory diagnosis of HAE with baseline plasma level of functional C1INH of less than 50% of normal
4. Willingness and ability to comply with all protocol procedures
Inclusion Criteria for randomized treatment:
1. Above screening criteria continue to be met
2. Clinical symptoms of HAE attack
3. Onset of symptoms less than 5 hours before the time at which medical evaluation
determined eligibility for inclusion
4. ‘Overall severity VAS’ score for at least one anatomical location at the time of initial
evaluation and Time 0 of at least 50 mm
5. No evidence of regression of angioedema symptoms scored with VAS at t = 0 h
compared with VAS score at the time of initial evaluation, i.e. no decrease in symptoms of angioedema scored with overall VAS at t = 0 h compared with score at the time of initial evaluation by 20 mm or more at any eligible site.
Inclusion Criteria for Open Label Treatment:
1. Above screening and randomization criteria continue to be met for subsequent eligible attack.
2. 24 hours have passed since the patient’s randomized or last open-label treatment.
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
F.1.3 Elderly (>=65 years) yes
F.1.3.1 Number of subjects for this age range
Exclusion Criteria at Screening:
Medical history of allergy to rabbits or rabbit-derived products (including rhC1INH), or positive anti-rabbit dander IgE test (cut off >0.35 kU/L; ImmunoCap® assay; Phadia or equivalent).
1. A diagnosis of acquired C1INH deficiency (AAE)
2. Pregnancy, or breastfeeding, or current intention to become pregnant
3. Treatment with any investigational drug in the past 30 days
4. Any clinically significant abnormality in the routine hematology and biochemistry that, in the opinion of the investigator, might interfere with the evaluation of study objectives
5. Any condition or treatment that, in the opinion of the investigator, might interfere with the evaluation of study objectives
Exclusion Criteria for randomized treatment:
1. Any changes since screening that would exclude patient based on above exclusion
criteria
2. Positive pregnancy test (urine or serum).
Exclusion Criteria for Open Label Treatment:
1. Any changes since screening that would exclude subject based on above exclusion
criteria.
2. Positive pregnancy test (urine or serum).
Study & Design
- Study Type
- Interventional clinical trial of medicinal product
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method
- Secondary Outcome Measures
Name Time Method