Clinical Trial Page

Summary
EudraCT Number:2008-008435-29
Sponsor's Protocol Code Number:B1831005(3082B2-4433-WW)
National Competent Authority:Bulgarian Drug Agency
Clinical Trial Type:EEA CTA
Trial Status:Completed
Date on which this record was first entered in the EudraCT database:2014-06-24
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 ConcernedBulgarian Drug Agency
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.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
The main purpose of this study is to gather information regarding the safety of ReFacto AF, and how well it works (effectiveness) in children with severe Hemophilia A under the age of 12. Approximately 50 subjects, will take part in this study. 25 subjects will be 0-6 years of age and 25 subjects will be 6 to 12 years of age.
A.4.1Sponsor's protocol code numberB1831005(3082B2-4433-WW)
A.7Trial is part of a Paediatric Investigation Plan No
A.8EMA Decision number of Paediatric Investigation Plan
B. Sponsor Information
B.Sponsor: 1
B.1.1Name of SponsorWyeth Pharmaceuticals Inc. Acting through its division Wyeth Research, a Pfizer Company
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 supportWyeth Pharmaceuticals Inc. Acting through its division Wyeth Research, a Pfizer Company
B.4.2CountryUnited States
B.5 Contact point designated by the sponsor for further information on the trial
B.5.1Name of organisationPfizer Inc.
B.5.2Functional name of contact pointClinical Trials.gov Call Center
B.5.3 Address:
B.5.3.1Street Address235 E 42nd Street
B.5.3.2Town/ cityNew York
B.5.3.3Post codeNY 10017
B.5.3.4CountryUnited States
B.5.4Telephone number0018007181021
B.5.5Fax number0013037391119
B.5.6E-mailClinicalTrials.govCallCenter@Pfizer.com
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 No
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.3.9.2Current sponsor codeNot Applicable
D.3.9.3Other descriptive nameNot Applicable
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) No
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 No
D.3.11.3.4Combination ATIMP (i.e. one involving a medical device) No
D.3.11.3.5Committee on Advanced therapies (CAT) has issued a classification for this product No
D.3.11.4Combination product that includes a device, but does not involve an Advanced Therapy No
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 Yes
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.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 No
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.3.9.2Current sponsor codeNot Applicable
D.3.9.3Other descriptive nameNot Applicable
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) No
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 No
D.3.11.3.4Combination ATIMP (i.e. one involving a medical device) No
D.3.11.3.5Committee on Advanced therapies (CAT) has issued a classification for this product No
D.3.11.4Combination product that includes a device, but does not involve an Advanced Therapy No
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 Yes
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.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 No
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.3.9.2Current sponsor codeNot Applicable
D.3.9.3Other descriptive nameNot Applicable
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) No
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 No
D.3.11.3.4Combination ATIMP (i.e. one involving a medical device) No
D.3.11.3.5Committee on Advanced therapies (CAT) has issued a classification for this product No
D.3.11.4Combination product that includes a device, but does not involve an Advanced Therapy No
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 Yes
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
Severe hemophilia A (FVIII:C <1%) in male subjects <12 years of age.
E.1.1.1Medical condition in easily understood language
Hemophilia A is a condition where blood clotting is severely impaired and which also results in internal bleeding into the muscles and joints. It is a hereditary condition only found in the male.
E.1.1.2Therapeutic area Diseases [C] - Blood and lymphatic diseases [C15]
MedDRA Classification
E.1.2 Medical condition or disease under investigation
E.1.2Version 17.0
E.1.2Level LLT
E.1.2Classification code 10018937
E.1.2Term Haemophilia A
E.1.2System Organ Class 100000004850
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 pediatric 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 50 exposure days 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 30 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.5.1.1Timepoint(s) of evaluation of this end point
The endpoint will be evaluated at the following visits: ED1, ED10-15, ED 50, ED 100 and each 6 Month visits.
E.5.2Secondary end point(s)
Evaluation of safety and efficacy in subjects less than 12 years of age
E.5.2.1Timepoint(s) of evaluation of this end point
The endpoint will be evaluated at the following visits: ED1, ED10-15, ED 50, ED 100 and each 6 Month visits.
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 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 concerned1
E.8.5The trial involves multiple Member States Yes
E.8.5.1Number of sites anticipated in the EEA45
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 No
E.8.6.3If E.8.6.1 or E.8.6.2 are Yes, specify the regions in which trial sites are planned
Bulgaria
Denmark
Finland
France
Greece
Italy
Netherlands
Poland
Romania
Serbia
Spain
Sweden
Turkey
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 years6
E.8.9.1In the Member State concerned months4
E.8.9.1In the Member State concerned days0
E.8.9.2In all countries concerned by the trial years6
E.8.9.2In all countries concerned by the trial months4
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.1.1Number of subjects for this age range: 50
F.1.1.1In Utero No
F.1.1.1.1Number of subjects for this age range: 0
F.1.1.2Preterm newborn infants (up to gestational age < 37 weeks) No
F.1.1.2.1Number of subjects for this age range: 0
F.1.1.3Newborns (0-27 days) No
F.1.1.3.1Number of subjects for this age range: 0
F.1.1.4Infants and toddlers (28 days-23 months) No
F.1.1.4.1Number of subjects for this age range: 0
F.1.1.5Children (2-11years) Yes
F.1.1.5.1Number of subjects for this age range: 50
F.1.1.6Adolescents (12-17 years) No
F.1.1.6.1Number of subjects for this age range: 0
F.1.2Adults (18-64 years) No
F.1.2.1Number of subjects for this age range: 0
F.1.3Elderly (>=65 years) No
F.1.3.1Number of subjects for this age range: 0
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 No
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 state4
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)
After the subject's Final Visit (study completion/early withdrawal) ReFacto AF will not be supplied by the sponsor.
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 Decision2014-07-04
N.Ethics Committee Opinion of the trial applicationFavourable
N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
N.Date of Ethics Committee Opinion2014-10-01
P. End of Trial
P.End of Trial StatusCompleted
P.Date of the global end of the trial2016-04-04

Sponsors and Collaborators

Medical Conditions

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