Clinical Trial Page

Summary
EudraCT Number:2005-000206-31
Sponsor's Protocol Code Number:C1 1304-01
National Competent Authority:Spain - AEMPS
Clinical Trial Type:EEA CTA
Trial Status:Completed
Date on which this record was first entered in the EudraCT database:2010-03-15
Trial results View results
Index
A. PROTOCOL INFORMATION
B. SPONSOR INFORMATION
C. APPLICANT IDENTIFICATION
D. IMP IDENTIFICATION
D.8 INFORMATION ON PLACEBO
E. GENERAL INFORMATION ON THE TRIAL
F. POPULATION OF TRIAL SUBJECTS
G. INVESTIGATOR NETWORKS TO BE INVOLVED IN THE TRIAL
N. REVIEW BY THE COMPETENT AUTHORITY OR ETHICS COMMITTEE IN THE COUNTRY CONCERNED
P. END OF TRIAL
A. Protocol Information
A.1Member State ConcernedSpain - AEMPS
A.2EudraCT number2005-000206-31
A.3Full title of the trial
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.
A.3.2Name or abbreviated title of the trial where available
Phase III study of recombinant human C1 inhibitor
A.4.1Sponsor's protocol code numberC1 1304-01
A.7Trial is part of a Paediatric Investigation Plan Information not present in EudraCT
A.8EMA Decision number of Paediatric Investigation Plan
B. Sponsor Information
B.Sponsor: 1
B.1.1Name of SponsorLaboratorios Dr. Esteve, S.A.
B.1.3.4CountrySpain
B.3.1 and B.3.2Status of the sponsorCommercial
B.4 Source(s) of Monetary or Material Support for the clinical trial:
B.4.1Name of organisation providing support
B.4.2Country
B.5 Contact point designated by the sponsor for further information on the trial
B.5.1Name of organisation
B.5.2Functional name of contact point
B.Sponsor: 2
B.1.1Name of SponsorPharming Tecnologies B.V.
B.1.3.4CountryNetherlands
B.3.1 and B.3.2Status of the sponsorNon-Commercial
B.4 Source(s) of Monetary or Material Support for the clinical trial:
B.4.1Name of organisation providing support
B.4.2Country
B.5 Contact point designated by the sponsor for further information on the trial
B.5.1Name of organisation
B.5.2Functional name of contact point
D. IMP Identification
D.IMP: 1
D.1.2 and D.1.3IMP RoleTest
D.2 Status of the IMP to be used in the clinical trial
D.2.1IMP to be used in the trial has a marketing authorisation No
D.2.5The IMP has been designated in this indication as an orphan drug in the Community Yes
D.2.5.1Orphan drug designation numberEU/3/01/036
D.3 Description of the IMP
D.3.1Product nameRecombinant human C1 inhibitor
D.3.2Product code rhC1INH
D.3.4Pharmaceutical form Powder for solution for infusion
D.3.4.1Specific paediatric formulation Information not present in EudraCT
D.3.7Routes of administration for this IMPIntravenous use
D.3.11 The IMP contains an:
D.3.11.1Active substance of chemical origin No
D.3.11.2Active substance of biological/ biotechnological origin (other than Advanced Therapy IMP (ATIMP) Yes
The IMP is a:
D.3.11.3Advanced Therapy IMP (ATIMP) Information not present in EudraCT
D.3.11.3.1Somatic cell therapy medicinal product No
D.3.11.3.2Gene therapy medical product No
D.3.11.3.3Tissue Engineered Product Information not present in EudraCT
D.3.11.3.4Combination ATIMP (i.e. one involving a medical device) Information not present in EudraCT
D.3.11.3.5Committee on Advanced therapies (CAT) has issued a classification for this product Information not present in EudraCT
D.3.11.4Combination product that includes a device, but does not involve an Advanced Therapy Information not present in EudraCT
D.3.11.5Radiopharmaceutical medicinal product No
D.3.11.6Immunological medicinal product (such as vaccine, allergen, immune serum) No
D.3.11.7Plasma derived medicinal product No
D.3.11.8Extractive medicinal product No
D.3.11.9Recombinant medicinal product Information not present in EudraCT
D.3.11.10Medicinal product containing genetically modified organisms No
D.3.11.11Herbal medicinal product No
D.3.11.12Homeopathic medicinal product No
D.3.11.13Another type of medicinal product Information not present in EudraCT
D.8 Information on Placebo
E. General Information on the Trial
E.1 Medical condition or disease under investigation
E.1.1Medical condition(s) being investigated
Patients with acute attacks of hereditary angioedema
MedDRA Classification
E.1.2 Medical condition or disease under investigation
E.1.2Version 8
E.1.2Level LLT
E.1.2Classification code 10019860
E.1.3Condition being studied is a rare disease Yes
E.2 Objective of the trial
E.2.1Main objective of the trial
To demonstrate the efficay of rhC1INH in the treatment of acute attacks in patients with HAE.
E.2.2Secondary objectives of the trial
To assess the safety and tolerability of rhC1INH in symptomatic patients with HAE.
To assess the safety, tolerability, efficacy as well as PK and PD of rhC1INH in symptomatic patients with HAE.
E.2.3Trial contains a sub-study No
E.3Principal inclusion criteria
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 sufficient 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.
•Day 22 post treatment follow-up visit has occurred.
Minimum interval of 22 days between subsequent open label treatments.
E.4Principal exclusion criteria
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).
Open Label Treatment
• Any reported SAE related to (randomized or open-label) study drug administration
• Any changes since screening and/or moment of diagnosis 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).
E.5 End points
E.5.1Primary end point(s)
Time to the beginning of relief based on patient’s VAS scores.
This variable will be based on the overall VAS for each eligible location (abdominal and/or facial-oro-pharyngeal-laryngeal and/or genito-urinary and/or peripheral). Time to the beginning of relief is defined as the first assessment time at which the overall VAS decreases by at least 20 mm with respect to baseline (t = 0 h). For single attacks involving more than one (eligible) location, time to the beginning of relief is based on a decrease of at least 20 mm for any location. For single attacks involving more than one (eligible; at least 50 mm at t = - 1 h) location, the location with the earliest response will be used for statistical analysis.
E.6 and E.7 Scope of the trial
E.6Scope of the trial
E.6.1Diagnosis No
E.6.2Prophylaxis No
E.6.3Therapy Yes
E.6.4Safety Yes
E.6.5Efficacy Yes
E.6.6Pharmacokinetic Yes
E.6.7Pharmacodynamic Yes
E.6.8Bioequivalence No
E.6.9Dose response No
E.6.10Pharmacogenetic No
E.6.11Pharmacogenomic No
E.6.12Pharmacoeconomic No
E.6.13Others No
E.7Trial type and phase
E.7.1Human pharmacology (Phase I) No
E.7.1.1First administration to humans No
E.7.1.2Bioequivalence study No
E.7.1.3Other No
E.7.1.3.1Other trial type description
E.7.2Therapeutic exploratory (Phase II) No
E.7.3Therapeutic confirmatory (Phase III) Yes
E.7.4Therapeutic use (Phase IV) No
E.8 Design of the trial
E.8.1Controlled No
E.8.1.1Randomised No
E.8.1.2Open No
E.8.1.3Single blind No
E.8.1.4Double blind No
E.8.1.5Parallel group No
E.8.1.6Cross over No
E.8.1.7Other No
E.8.2 Comparator of controlled trial
E.8.2.1Other medicinal product(s) No
E.8.2.2Placebo No
E.8.2.3Other No
E.8.3 The trial involves single site in the Member State concerned No
E.8.4 The trial involves multiple sites in the Member State concerned Yes
E.8.4.1Number of sites anticipated in Member State concerned4
E.8.5The trial involves multiple Member States Yes
E.8.5.1Number of sites anticipated in the EEA19
E.8.6 Trial involving sites outside the EEA
E.8.6.1Trial being conducted both within and outside the EEA No
E.8.6.2Trial being conducted completely outside of the EEA Information not present in EudraCT
E.8.7Trial has a data monitoring committee Yes
E.8.8 Definition of the end of the trial and justification where it is not the last visit of the last subject undergoing the trial
E.8.9 Initial estimate of the duration of the trial
E.8.9.1In the Member State concerned years1
E.8.9.1In the Member State concerned months6
E.8.9.1In the Member State concerned days
E.8.9.2In all countries concerned by the trial years2
E.8.9.2In all countries concerned by the trial months0
F. Population of Trial Subjects
F.1 Age Range
F.1.1Trial has subjects under 18 Yes
F.1.1.1In Utero No
F.1.1.2Preterm newborn infants (up to gestational age < 37 weeks) No
F.1.1.3Newborns (0-27 days) No
F.1.1.4Infants and toddlers (28 days-23 months) No
F.1.1.5Children (2-11years) No
F.1.1.6Adolescents (12-17 years) Yes
F.1.2Adults (18-64 years) Yes
F.1.3Elderly (>=65 years) Yes
F.2 Gender
F.2.1Female Yes
F.2.2Male Yes
F.3 Group of trial subjects
F.3.1Healthy volunteers No
F.3.2Patients Yes
F.3.3Specific vulnerable populations Yes
F.3.3.1Women of childbearing potential not using contraception No
F.3.3.2Women of child-bearing potential using contraception Yes
F.3.3.3Pregnant women No
F.3.3.4Nursing women No
F.3.3.5Emergency situation No
F.3.3.6Subjects incapable of giving consent personally No
F.3.3.7Others No
F.4 Planned number of subjects to be included
F.4.1In the member state5
F.4.2 For a multinational trial
F.4.2.1In the EEA 30
F.4.2.2In the whole clinical trial 30
G. Investigator Networks to be involved in the Trial
N. Review by the Competent Authority or Ethics Committee in the country concerned
N.Competent Authority Decision Authorised
N.Date of Competent Authority Decision2005-01-27
N.Ethics Committee Opinion of the trial applicationFavourable
N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
N.Date of Ethics Committee Opinion2005-01-14
P. End of Trial
P.End of Trial StatusCompleted
P.Date of the global end of the trial2007-11-13

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