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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. |
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Register:
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EUCTR |
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Last refreshed on:
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7 February 2022 |
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Main ID: |
EUCTR2010-021781-29-IT |
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Date of registration:
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21/02/2011 |
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Prospective Registration:
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No |
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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 - ND
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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 - ND |
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Date of first enrolment:
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25/01/2011 |
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Target sample size:
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60 |
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Recruitment status: |
Not Recruiting |
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URL:
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https://www.clinicaltrialsregister.eu/ctr-search/search?query=eudract_number:2010-021781-29 |
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Study type:
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Interventional clinical trial of medicinal product |
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Study design:
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Controlled: no
Randomised: no
Open: no
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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Austria
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Bulgaria
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Denmark
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Hungary
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Ireland
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Italy
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Latvia
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Lithuania
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Norway
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Poland
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Spain
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Sweden
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United Kingdom
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Contacts
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Affiliation:
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Key inclusion & exclusion criteria
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Inclusion criteria:
Part A 1. Male, aged = 12 years 2. Severe hemophilia A defined as < 1% factor VIII concentration (FVIII:C) based on medical records and/or screening laboratory 3. > 50 ED with any high purity FVIII; 4. No current evidence of inhibitor antibody measured using the Nijmegen modified Bethesda assay [<0.6 Bethesda units (BU)/mL] within 2-3 week after last administration. Subjects may not receive FVIII within 48 h prior to the collection of samples for inhibitor testing. 5. No history of FVIII inhibitor formation. Documentation of negative result in medical records required, except for PUPs. [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 study electronic patient diary (EPD) and to document injection information during the study. 7. Written informed consent by parent/legal representative. Assent should be sought from subjects if appropriate Part B (PUPs) 8. Inclusion of PUPs may start after 10 children have accumulated 50 ED 9. Male, aged = 6 years 10. Severe hemophilia A defined as < 1% FVIII:C based on medical records and/or screening laboratory 11. no previous exposure to any FVIII 12. PUPs may be included if they will receive their first FVIII dose as regular prophylaxis or on-demand treatment for bleedings, and are willing to continue with prophylaxis. 13. Written informed consent by parent/legal representative
Are the trial subjects under 18? yes
Number of subjects for this age range:
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:
Part A and B 1. Any individuals with another bleeding disease that is different from Hemophilia A (eg, von Willebrand disease, Hemophilia B) 2. Any individual with thrombocytopenia (platelet count < 100 000/mm3) based on medical records or screening laboratory 3. Any individual with abnormal renal function (serum creatinine > 2.0 mg/dL) based on medical records or screening laboratory 4. Any individual without any documented negative inhibitor test (except for PUPs) 5. Any individual who is receiving or has received other experimental drugs within 3 months prior to study entry or has participated in a FVIII study within the last month 6. Any individual who requires any pre-medication to tolerate FVIII injections (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 8. Known hypersensitivity to active substance, mouse or hamster protein. 9. Previous participation in this study Part B only 10. For PUPs: first treatment with BAY 81-8973 for surgery
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: 9.1
Level: LLT
Classification code 10060612
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Intervention(s)
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Product Code: BAY 81-8973
Pharmaceutical Form: Powder and solvent for solution for injection
INN or Proposed INN: Coagulation factor VIII
Concentration unit: IU international unit(s)
Concentration type: equal
Concentration number: 250-
Product Code: BAY 81-8973
Pharmaceutical Form: Powder and solvent for solution for infusion
INN or Proposed INN: Coagulation factor VIII
Concentration unit: IU international unit(s)
Concentration type: equal
Concentration number: 500-
Product Code: BAY 81-8973
Pharmaceutical Form: Powder and solvent for solution for injection
INN or Proposed INN: 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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Secondary Objective: The secondary objectives are •To assess the tolerability profile of BAY 81-8973 during prophylaxis and treatment of breakthrough bleeds and safety and efficacy during surgeries, •To assess incremental recovery •To assess pharmacokinetics in a subset of 6 to 13 children in the age group of 6-12 years (participation for pharmacokinetic [PK] sampling is optional, if parents consent to it).
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Primary end point(s): The primary variable is the annualized number of all bleeds within 48 hours after a prophylaxis injection
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Main Objective: The primary objective is to evaluate the safety and efficacy of the treatment with BAY 81-8973 for prophylaxis and treatment of breakthrough bleeds in children with haemophilia A
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Secondary ID(s)
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2010-021781-29-HU
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BAY 81-8973/13400
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Source(s) of Monetary Support
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Ethics review
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Status: Approved
Approval date: 25/01/2011
Contact:
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