Main
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Note: This record shows only 22 elements of the WHO Trial Registration Data Set. To view changes that have been made to the source record, or for additional information about this trial, click on the URL below to go to the source record in the primary register. |
Register:
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EUCTR |
Last refreshed on:
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9 November 2020 |
Main ID: |
EUCTR2010-021781-29-LT |
Date of registration:
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28/12/2010 |
Prospective Registration:
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Yes |
Primary sponsor: |
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Public title:
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A multicenter Phase III uncontrolled open-label trial to evaluate safety and efficacy of BAY 81-8973 in children with severe haemophilia A under prophylaxis therapy
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Scientific title:
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A multicenter Phase III uncontrolled open-label trial to evaluate safety and efficacy of BAY 81-8973 in children with severe haemophilia A under prophylaxis therapy - Leopold Kids |
Date of first enrolment:
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09/05/2011 |
Target sample size:
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100 |
Recruitment status: |
Not Recruiting |
URL:
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https://www.clinicaltrialsregister.eu/ctr-search/search?query=eudract_number:2010-021781-29 |
Study type:
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Interventional clinical trial of medicinal product |
Study design:
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Controlled: no
Randomised: no
Open: yes
Single blind: no
Double blind: no
Parallel group: no
Cross over: no
Other: no
If controlled, specify comparator, Other Medicinial Product:
Placebo:
Other:
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Phase:
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Human pharmacology (Phase I): no
Therapeutic exploratory (Phase II): no
Therapeutic confirmatory - (Phase III): yes
Therapeutic use (Phase IV): no
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Countries of recruitment
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Argentina
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Austria
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Belgium
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Bulgaria
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Canada
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Denmark
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Greece
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Hungary
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Ireland
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Israel
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Italy
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Latvia
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Lithuania
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Mexico
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Norway
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Poland
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Romania
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Russian Federation
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Spain
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Sweden
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United Kingdom
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United States
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Contacts
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Name:
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CTP Team / Ref: "EU CTR" /
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Address:
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13342
Berlin
Germany |
Telephone:
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+4930300139003 |
Email:
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clinical-trials-contact@bayer.com |
Affiliation:
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Bayer AG |
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Name:
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CTP Team / Ref: "EU CTR" /
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Address:
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13342
Berlin
Germany |
Telephone:
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+4930300139003 |
Email:
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clinical-trials-contact@bayer.com |
Affiliation:
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Bayer AG |
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Key inclusion & exclusion criteria
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Inclusion criteria: Part A 1. Male, age = 12 years. Enrollment will begin with subjects 6-12 years before it is opened to all age groups. 2. Severe hemophilia A defined as < 1% factor VIII concentration (FVIII:C) based on documented prior testing or screening laboratory 3. >/= 50 ED with any FVIII concentrate 4. No current evidence of inhibitor antibody measured using the Nijmegen modified Bethesda assay [<0.6 Bethesda units (BU)/mL] within 2-3 weeks of last FVIII administration. 5. No history of FVIII inhibitor formation. Documentation of negative result in medical records required. [Subjects with a maximum historical titer of 1.0 BU on no more than 1 occasion with the classical Bethesda assay but at least 3 successive negative (<0.6 BU) results thereafter are eligible.] 6. Willingness and ability of subjects and/or parents to complete training in the use of the electronic patient diary (EPD) and to document infusions during the study. 7. Written informed consent by parent/legal representative. Assent should be sought from subjects if appropriate
Part B (PUPs) 1. Male, < 6 years 2. Severe hemophilia A defined as < 1% FVIII:C based on prior documented testing or confirmed on screening laboratory 3.a PUPs: no previous exposure to any FVIII product 3.b MTPs: have no more thank 3 EDs with any FVIII product and who have no current evidence of inhibitor antibody measured centrally using the Nijmegen-modified Bethesda assay [<0.2 Bethesda units (BU)/ ml at screening, with confirmation using local laboratory testing at baseline (<0.6 Bethesda units [BU]/ml] (as of amd7). MTPs may not receive FVIII within 48h prior to the collection of samples for inhibitor testing at the Screening and baseline visits. 3.c. MTPs: no history of FVIII inhibitor formation 4. PUPs may be included if they will receive their first FVIII dose with BAY 81-8973 for treatment of first bleeds and agree to start prophylaxis as part of their care. MTPs may be included if they agree to start prophylaxis as part of their care 5. Willingness and ability of parents to complete training in the use of the electronic patient diary (EPD) and to document all treatment during the study. 6. Written informed consent by parent/legal representative.
Are the trial subjects under 18? yes Number of subjects for this age range: 100 F.1.2 Adults (18-64 years) no F.1.2.1 Number of subjects for this age range F.1.3 Elderly (>=65 years) no F.1.3.1 Number of subjects for this age range
Exclusion criteria: Parts A and B 1. Any individual with another bleeding disorder that is different from Hemophilia A (eg, von Willebrand disease, Hemophilia B) 2. Any individual with thrombocytopenia (platelet count < 100,000/mm3) 3. Creatinine > 2x upper limit of normal or AST/ALT > 5x upper limit of normal 4. Any individual without a negative inhibitor at screening (except for PUPs) 5. Any individual who is receiving chemotherapy, immune modulatory drugs (IVIG, cyclosporine, chronic use of oral or i.v. corticosteroids), has received another investigational FVIII product within the last month, or received another experimental drug within the last 3 months. 6. Any individual who requires any pre-medication to tolerate FVIII treatment (eg, antihistamines). 7. Any individual who is unwilling to comply with study visits or other protocol requirements, for example (eg. prophylaxis treatment) or is not suitable for participation in this study for any reason, according to the Investigator’s judgment. 8. Known hypersensitivity to active substance, mouse or hamster protein. 9. Previous participation in this study
Part B only (PUPs): 10. First treatment with BAY 81-8973 for high risk bleeding situations (e.g. surgery, intracranial bleed), or requiring intensive or prolonged treatment. 11. Unable to tolerate volume of blood draws required for study participation.
Age minimum:
Age maximum:
Gender:
Female: no Male: yes
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Health Condition(s) or Problem(s) studied
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Severe Hemophilia-A (< 1% FVIII:C) MedDRA version: 20.0
Level: LLT
Classification code 10060612
Term: Hemophilia A
System Organ Class: 100000004850
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Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15]
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Intervention(s)
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Trade Name: Kovaltry Product Name: BAY 81-8973/ Kovaltry (250 IU/vial) Product Code: BAY 81-8973 Pharmaceutical Form: Powder and solvent for solution for injection INN or Proposed INN: octocog alfa CAS Number: - Current Sponsor code: BAY 81-8973 Other descriptive name: recombinant coagulation factor VIII Concentration unit: IU international unit(s) Concentration type: equal Concentration number: 250-
Trade Name: Kovaltry Product Name: BAY 81-8973/ Kovaltry (500 IU/vial) Product Code: BAY 81-8973 Pharmaceutical Form: Powder and solvent for solution for injection INN or Proposed INN: octocog alfa CAS Number: - Current Sponsor code: BAY 81-8973 Other descriptive name: recombinant coagulation factor VIII Concentration unit: IU international unit(s) Concentration type: equal Concentration number: 500-
Trade Name: Kovaltry Product Name: BAY 81-8973/ Kovaltry (1000 IU/vial) Product Code: BAY 81-8973 Pharmaceutical Form: Powder and solvent for solution for injection INN or Proposed INN: octocog alfa CAS Number: - Current Sponsor code: BAY 81-8973 Other descriptive name: recombinant coagulation factor VIII Concentration unit: IU international unit(s) Concentration type: equal Concentration number: 1000-
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Primary Outcome(s)
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Primary end point(s): The primary variable is the annualized number of total bleeds (sum of spontaneous bleeds and traumatic bleeds) during prophylaxis that occur within 48 h of the last prophylaxis infusion. Both joint and non-joint bleeding will be assessed.
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Main Objective: The primary objective is to demonstrate safety and efficacy of the treatment with BAY 81-8973 for prophylaxis and treatment of breakthrough bleeds in children with severe hemophilia A.
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Secondary Objective: The secondary objectives are - To assess the safety and efficacy of BAY 81-8973 during surgeries. - To assess incremental recovery of BAY 81-8973. - To characterize pharmacokinetics in a subset of children.
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Timepoint(s) of evaluation of this end point: Continuous throughout duration of study
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Secondary Outcome(s)
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Timepoint(s) of evaluation of this end point: Continuous throughout duration of study
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Secondary end point(s): • Subject/parents’ assessment of the response of treatment of bleeding events which is assessed as excellent, good, moderately well or poorly. • Annualized number of total bleeds (sum of spontaneous and trauma bleeds) during prophylaxis treatment • Assessment of adequacy of haemostasis during surgical interventions • Number of infusions for the treatment of a bleed • Consumption of FVIII
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Secondary ID(s)
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BAY81-8973/13400
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2010-021781-29-HU
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Source(s) of Monetary Support
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Bayer AG
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Ethics review
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Status: Approved
Approval date: 07/02/2011
Contact:
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