Clinical Trial Page

Summary
EudraCT Number:2008-008435-29
Sponsor's Protocol Code Number:3082B2-4433-WW
National Competent Authority:Greece - EOF
Clinical Trial Type:EEA CTA
Trial Status:Completed
Date on which this record was first entered in the EudraCT database:2009-10-06
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 ConcernedGreece - EOF
A.2EudraCT number2008-008435-29
A.3Full title of the trial
A Non-Randomized, Open-Label Study to Evaluate the Pharmacokinetics, Safety, and Efficacy of ReFacto AF in Previously Treated Pediatric Subjects Less Than Twelve Years of Age With Severe Hemophilia A (FVIII:C <1%)
A.4.1Sponsor's protocol code number3082B2-4433-WW
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 SponsorWyeth Research Division of Wyeth Pharmaceuticals Inc. Clinical Research and Development
B.1.3.4CountryUnited States
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 ReFacto AF
D.2.1.1.2Name of the Marketing Authorisation holderWyeth Europa Ltd
D.2.1.2Country which granted the Marketing AuthorisationEuropean Union
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.3.4Pharmaceutical 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 INNMOROCTOCOG-ALFA
D.3.9.1CAS number 284036-24-4
D.3.10 Strength
D.3.10.1Concentration unit IU international unit(s)
D.3.10.2Concentration typeequal
D.3.10.3Concentration number250
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 Yes
D.3.11.13.1Other medicinal product typeRecombinant coagulation factor VIII
D.IMP: 2
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 ReFacto AF
D.2.1.1.2Name of the Marketing Authorisation holderWyeth Europa Ltd
D.2.1.2Country which granted the Marketing AuthorisationEuropean Union
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.3.4Pharmaceutical 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 INNMOROCTOCOG-ALFA
D.3.9.1CAS number 284036-24-4
D.3.10 Strength
D.3.10.1Concentration unit IU international unit(s)
D.3.10.2Concentration typeequal
D.3.10.3Concentration number500
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 Yes
D.3.11.13.1Other medicinal product typeRecombinant coagulation factor VIII
D.IMP: 3
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 ReFacto AF
D.2.1.1.2Name of the Marketing Authorisation holderWyeth Europa Ltd
D.2.1.2Country which granted the Marketing AuthorisationEuropean Union
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.3.4Pharmaceutical 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 INNMOROCTOCOG-ALFA
D.3.9.1CAS number 284036-24-4
D.3.10 Strength
D.3.10.1Concentration unit IU international unit(s)
D.3.10.2Concentration typeequal
D.3.10.3Concentration number1000
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 Yes
D.3.11.13.1Other medicinal product typeRecombinant coagulation factor VIII
D.IMP: 4
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 ReFacto AF
D.2.1.1.2Name of the Marketing Authorisation holderWyeth Europa Ltd
D.2.1.2Country which granted the Marketing AuthorisationEuropean Union
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.3.4Pharmaceutical 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 INNMOROCTOCOG-ALFA
D.3.9.1CAS number 284036-24-4
D.3.10 Strength
D.3.10.1Concentration unit IU international unit(s)
D.3.10.2Concentration typeequal
D.3.10.3Concentration number2000
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 Yes
D.3.11.13.1Other medicinal product typeRecombinant coagulation factor VIII
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
Severe hemophilia A (FVIII:C <1%) in male subjects <12 years of age.
MedDRA Classification
E.1.2 Medical condition or disease under investigation
E.1.2Version 9.1
E.1.2Level LLT
E.1.2Classification code 10018937
E.1.2Term Haemophilia A
E.1.3Condition being studied is a rare disease Yes
E.2 Objective of the trial
E.2.1Main objective of the trial
To evaluate the pharmacokinetics and incremental recovery of ReFacto AF in pediatic subjects less than 12 years of age after a single exposure to ReFacto AF.
E.2.2Secondary objectives of the trial
To evaluate the efficacy and safety of ReFacto AF in pediatric subjects less than 12 years of age, including the frequency of less than expected therapeutic effect.
E.2.3Trial contains a sub-study No
E.3Principal inclusion criteria
1) Male subjects <12 years of age with a documented history of severe hemophilia A (FVIII:C <1%).
a) Subjects who are <6 years of age must have had at least 1 exposure day to prior FVIII products (including blood products).
b) Subjects who are ≥6 years of age must have had greater than 150 exposure days to prior FVIII products (including blood products)
E.4Principal exclusion criteria
1) For laboratory assessments, any measured Bethesda inhibitor titer ≥0.6 BU, regardless of the laboratory normal range, or any Bethesda inhibitor titer > ULN for the testing laboratory at the time of screening.
2) Any other bleeding disorder in addition to hemophilia A.
3) Treatment with any investigational drug or device within 28 days before the time of signing the parental informed consent/assent form.
4) Major surgery planned to occur during the course of the study.
5) Regular (e.g., daily; every other day) use of agents or medications known to influence platelet function such as aspirin or certain nonsteroidal anti-inflammatory drugs (NSAIDS).
6) Regular, concomitant therapy with immunomodulating drugs (e.g., intravenous immunoglobulin [IVIG], routine systemic corticosteroids), or currently receiving immune tolerance induction (ITI) for inhibitor treatment.
7) The subject is receiving treatment for HIV or hepatitis infection (exceptions may be made for subjects on a stable antiviral regimen [i.e., consistent treatment regimen for at least 3 months before signing the parental informed consent/assent form]).
8) Platelet count <100,000/µL.
9) Prothrombin time (PT) ≥1.25 x ULN, or international normalized ratio (INR) ≥1.5.
10) Known hypersensitivity to hamster protein.
11) Any condition(s) that compromises the subject’s ability to comply with and/or perform study-related activities or that poses a clinical contraindication to study participation (these conditions include, but are not limited to, inadequate medical history available to assure study eligibility; inability to properly store study medication; expectation of poor compliance in study related documentation; poor venous access), in the opinion of the investigator.
12) Unwilling or unable to follow the terms of the protocol.
E.5 End points
E.5.1Primary end point(s)
The primary safety outcome is the development of clinically significant FVIII inhibitors. Clinically significant inhibitors are defined as a central laboratory confirmed positive inhibitor (≥0.6 BU using the Nijmegen modification of the Bethesda assay present at 2 consecutive blood draws within a 6 week interval) and the occurrence of one or more of the following clinical observations:
decreased FVIII recovery, need for alternative hemostatic products, increase in number of breakthrough bleeds while on preventative or prophylactic treatment, or more than one report of Less Than Expected Therapeutic Effect (LETE) in the absence of confounding factors.
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 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) No
E.7.4Therapeutic use (Phase IV) Yes
E.8 Design of the trial
E.8.1Controlled No
E.8.1.1Randomised Information not present in EudraCT
E.8.1.2Open Information not present in EudraCT
E.8.1.3Single blind Information not present in EudraCT
E.8.1.4Double blind Information not present in EudraCT
E.8.1.5Parallel group Information not present in EudraCT
E.8.1.6Cross over Information not present in EudraCT
E.8.1.7Other Information not present in EudraCT
E.8.2 Comparator of controlled trial
E.8.2.1Other medicinal product(s) Information not present in EudraCT
E.8.2.2Placebo Information not present in EudraCT
E.8.2.3Other Information not present in EudraCT
E.8.3 The trial involves single site in the Member State concerned Yes
E.8.4 The trial involves multiple sites in the Member State concerned No
E.8.5The trial involves multiple Member States Yes
E.8.5.1Number of sites anticipated in the EEA50
E.8.6 Trial involving sites outside the EEA
E.8.6.1Trial being conducted both within and outside the EEA Yes
E.8.6.2Trial being conducted completely outside of the EEA Information not present in EudraCT
E.8.6.3If E.8.6.1 or E.8.6.2 are Yes, specify the regions in which trial sites are planned
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
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 years4
E.8.9.1In the Member State concerned months1
E.8.9.1In the Member State concerned days
E.8.9.2In all countries concerned by the trial years4
E.8.9.2In all countries concerned by the trial months1
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) Yes
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 No
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
Paediatric patients
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 45
F.4.2.2In the whole clinical trial 50
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)
N/A - Study is in usual care setting.
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 Decision2009-11-11
N.Ethics Committee Opinion of the trial applicationFavourable
N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
N.Date of Ethics Committee Opinion2010-03-22
P. End of Trial
P.End of Trial StatusCompleted
P.Date of the global end of the trial2016-04-04

Sponsors and Collaborators

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