임상 시험 Nct 페이지

Summary
EudraCT Number:2004-005051-34
Sponsor's Protocol Code Number:42-73-305 / WIL1-0305
National Competent Authority:Belgium - FPS Health-DGM
Clinical Trial Type:EEA CTA
Trial Status:Completed
Date on which this record was first entered in the EudraCT database:2007-05-31
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 ConcernedBelgium - FPS Health-DGM
A.2EudraCT number2004-005051-34
A.3Full title of the trial
Efficacy and safety study of vWF SD-35-DH (WILFACTIN) in children under 6 years of age
A.3.2Name or abbreviated title of the trial where available
NOT APPLICABLE
A.4.1Sponsor's protocol code number42-73-305 / WIL1-0305
A.5.1ISRCTN (International Standard Randomised Controlled Trial) NumberNOT APPLICABLE
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 SponsorLFB BIOTECHNOLOGIES
B.1.3.4CountryFrance
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
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 Yes
D.2.1.1.1Trade name WILFACTIN
D.2.1.1.2Name of the Marketing Authorisation holderLFB BIOMEDICAMENTS
D.2.1.2Country which granted the Marketing AuthorisationFrance
D.2.5The IMP has been designated in this indication as an orphan drug in the Community No
D.2.5.1Orphan drug designation number
D.3 Description of the IMP
D.3.1Product nameWILFACTIN
D.3.2Product code VWF SD-35-DH
D.3.4Pharmaceutical form Powder and solvent for solution for injection
D.3.4.1Specific paediatric formulation Information not present in EudraCT
D.3.7Routes of administration for this IMPIntravenous use
D.3.8 to D.3.10 IMP Identification Details (Active Substances)
D.3.8INN - Proposed INNhuman von Willebrand factor
D.3.10 Strength
D.3.10.1Concentration unit IU/ml international unit(s)/millilitre
D.3.10.2Concentration typeequal
D.3.10.3Concentration number1
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 Yes
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 No
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
Von Willebrand disease
MedDRA Classification
E.1.2 Medical condition or disease under investigation
E.1.2Classification code 10047715
E.1.3Condition being studied is a rare disease No
E.2 Objective of the trial
E.2.1Main objective of the trial
Main objective : to evaluate, in children presenting with inherited von Willebrand factor (VWF) deficiencies, the biological and clinical efficacy of WILFACTIN for the treatment of bleeding episodes and for the prevention of haemorrhages during surgery or invasive procedures when desmopressin is ineffective or contraindicated.
E.2.2Secondary objectives of the trial
Secondary objectives : to evaluate the safety, especially the immunological safety, of WILFACTIN in a paediatric population.
E.2.3Trial contains a sub-study No
E.3Principal inclusion criteria
- age < 6 years
- VWF:RCo < 20% (IU/dl) (except type 2N)
E.4Principal exclusion criteria
- positive VWF and/or FVIII inhibitors
- platelets < 100 x 10exp9/l (except for type 2B patients)
E.5 End points
E.5.1Primary end point(s)
- The biological efficacy of WILFACTIN will be evaluated based on VWF recovery after administration of 100 IU/kg at inclusion of the study and without bleeding ongoing,
- Efficacy of treatment for bleeding episodes will be rated at the end of the episode by the doctor, using the following scale: "excellent", "good", "moderate", "none".
- Efficacy of treatment during surgery will be rated at the end of hospitalisation by the doctor, using the same scale. Blood loss and use of transfusions will be recorded and analysed in comparison to normal subject.
- Efficacy of long-term prophylaxis treatment with vWF SD-35-DH will be judged by the absence of spontaneous bleeding episodes or the clear reduction of haemorrhages.
E.6 and E.7 Scope of the trial
E.6Scope of the trial
E.6.1Diagnosis No
E.6.2Prophylaxis Yes
E.6.3Therapy Yes
E.6.4Safety Yes
E.6.5Efficacy Yes
E.6.6Pharmacokinetic No
E.6.7Pharmacodynamic No
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 Yes
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 EEA1
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 No
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
The end of the trial is designed to be the date of the last biological result obtained at the last visit of the last subject undergoing the trial (approximately 1 month after the last visit of the last subject).
E.8.9 Initial estimate of the duration of the trial
E.8.9.1In the Member State concerned years9
E.8.9.1In the Member State concerned months6
E.8.9.1In the Member State concerned days0
E.8.9.2In all countries concerned by the trial years5
E.8.9.2In all countries concerned by the trial months6
E.8.9.2In all countries concerned by the trial days0
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) Yes
F.1.1.4Infants and toddlers (28 days-23 months) Yes
F.1.1.5Children (2-11years) Yes
F.1.1.6Adolescents (12-17 years) No
F.1.2Adults (18-64 years) No
F.1.3Elderly (>=65 years) No
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 No
F.3.3.3Pregnant women No
F.3.3.4Nursing women No
F.3.3.5Emergency situation Yes
F.3.3.6Subjects incapable of giving consent personally Yes
F.3.3.6.1Details of subjects incapable of giving consent
children < 6 years
F.3.3.7Others No
F.4 Planned number of subjects to be included
F.4.1In the member state4
F.4.2 For a multinational trial
F.4.2.1In the EEA 10
F.4.2.2In the whole clinical trial 15
F.5 Plans for treatment or care after the subject has ended the participation in the trial (if it is different from the expected normal treatment of that condition)
At the end of the study, the patients will have the choice to continue the treatment with the marketed product or to use another treatment available in their country
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 Decision2005-04-18
N.Ethics Committee Opinion of the trial applicationFavourable
N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
N.Date of Ethics Committee Opinion2005-01-20
P. End of Trial
P.End of Trial StatusCompleted

스폰서 및 공동 작업자

건강 상태

약물 개입

3
구독하다