A randomized, placebo-controlled, double-blind Phase III study of the efficacy and safety of recombinant human C1 inhibitor for the treatment of acute attacks in patients with hereditary angioedema. - Phase III study of recombinant human C1 inhibitor
- Conditions
- Patients with acute attacks of hereditary angioedema.MedDRA version: 9.1 Level: LLT Classification code 10019860 Term: Hereditary angioedema
- Registration Number
- EUCTR2005-000206-31-GB
- Lead Sponsor
- Pharming Technologies B.V.
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- ot Recruiting
- Sex
- Not specified
- Target Recruitment
- 32
Screening
•Aged at least 16 years
•Signed written informed consent
•Clear clinical and laboratory diagnosis of HAE with baseline plasma level of functional C1INH of less than 50 % of normal.
Open-label treatment
•Above criteria
•Evidence for exacerbation or development of an abdominal attack and/or of facial-oro-pharyngeal angioedema and/or laryngeal angioedema and/or of genito-urinary angioedema and/or peripheral angioedema. Patients must notify and discuss symptoms with the investigator prior to travelling to the study centre.
•Onset of eligible symptoms not longer than 5 h ago.
•Patient’s VAS score of overall severity of angioedema symptoms at least at one location at the time of evaluation ( t = - 1 h) of at least 50 mm, where 0 mm means ‘no symptoms at all’ and 100 mm means ‘extremely disabling’. This inclusion criterion is set to allow suffcient room for improvement in response to study medication.
•No clear regression of angioedema signs by VAS at t = 0 h; just prior to the administration of study medication. Thus, no beginning of relief by VAS at t = 0 h (reduction of 20 mm or more) compared with VAS score at determination of eligibility, t = - 1 h.
•Above screening and treatment criteria continue to be met for subsequent eligible
attack
• Day 22 post-randomized treatment follow-up visit has occurred.
•Minimum interval of 22 Days between subsequent open-label treatments
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
F.1.3 Elderly (>=65 years) yes
F.1.3.1 Number of subjects for this age range
Screening
•A history of administration of pharmaceuticals derived from rabbits (e g. antisera, rhC1INH) in conjunction with any evidence of a relation with allergic reactions.
•A history of anaphylaxis, or severe allergies (i.e. requiring medication) to food, proteins and/or drugs.
•A history of allergic reactions to C1INH concentrates or rabbit protein.
•A diagnosis of acquired C1INH deficiency (exclude patients with AAE).
•Pregnancy, breast-feeding or current intention to become pregnant.
•Known or suspected addiction to narcotics.
•Blood donation in the past 3 months.
•Participation in another clinical study in the past 3 months or participation in two or more clinical studies prior to this study in the last 12 months.
•Any clinically significant abnormality in the routine hematology, biochemistry and urinalysis.
•Any condition or treatment that in the opinion of the investigator might interfere with the evaluation of the study objectives.
Open-label treatment
•Above criteria; no (expected) clinically significant abnormalities in routine laboratory analyses.
•Patients presenting or developing a life-threatening attack (an attack requiring immediate emergency procedures to prevent death, hypoxemia related injuries or other unfavorable outcomes).
• Any reported SAE related to (randomized or open-label) study drug administration.
• Any changes since screening and/or the moment of diagnosis of the present eligible
attack that would exclude subject based on above exclusion criteria.
• Patients presenting or developing a life-threatening attack (an attack requiring
immediate emergency procedures to prevent death, hypoxemia related injuries or
other unfavorable outcomes).
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