Please see informed consent.

• Register: EUCTR
• Last refreshed on: 9 November 2020
• Main ID: EUCTR2010-021781-29-LV
• Date of registration: 25/01/2011
• Prospective Registration: Yes
• Primary sponsor: Bayer HealthCare AG
• Public title: Please see informed consent.
• Scientific title: 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
• Date of first enrolment: 08/04/2011
• Target sample size: 75
• Recruitment status: Not Recruiting
• URL: https://www.clinicaltrialsregister.eu/ctr-search/search?query=eudract_number:2010-021781-29
• Study type: Interventional clinical trial of medicinal product
• Study design: 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: Number of treatment arms in the trial: 1
• Phase: Human pharmacology (Phase I): no Therapeutic exploratory (Phase II): no Therapeutic confirmatory - (Phase III): yes Therapeutic use (Phase IV): no

Countries of recruitment

Austria, Bulgaria, Canada, Denmark, Hungary, Ireland, Israel, Italy, Latvia, Lithuania, Norway, Poland, Romania, Serbia, Spain, Sweden, United Kingdom, United States

Contacts

• Name: CTP Team / Ref: "EU CTR" / S102 -
• Affiliation: Bayer HealthCare AG

• Name: CTP Team / Ref: "EU CTR" / S102 -
• Affiliation: Bayer HealthCare AG

Key inclusion & exclusion criteria

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 and/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 and under
2. Severe hemophilia A defined as < 1% FVIII:C based on prior documented testing and confirmed on screening laboratory
3. No previous exposure to any FVIII product
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.
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: 75
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 documented negative inhibitor test based on medical records or screening laboratory test (except for PUPs)
5. Any individual who is receiving chemotherapy, immune modulatory drugs (IVIG, cyclosporine, chronic use of oral or i.v. corticosteroids), has participated in another FVIII study 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

Health Condition(s) or Problem(s) studied

Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15]

Severe Hemophilia-A (< 1% FVIII:C)
MedDRA version: 13.1 Level: LLT Classification code 10060612 Term: Hemophilia A System Organ Class: 10010331 - Congenital, familial and genetic disorders

Intervention(s)

Product Name: BAY 81-8973 (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-

Product Name: BAY 81-8973 (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-

Product Name: BAY 81-8973 (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-

Primary Outcome(s)

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 severe hemophilia A.

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.

Timepoint(s) of evaluation of this end point: Continuous throughout duration of study

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 assess pharmacokinetic parameters in a subset of children.

Secondary Outcome(s)

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

Timepoint(s) of evaluation of this end point: Continuous throughout duration of study

Secondary ID(s)

2010-021781-29-HU

BAY81-8973/13400

Source(s) of Monetary Support

Bayer HealthCare AG

Ethics review

Status: Approved
Approval date: 17/03/2011
Contact:

Results

• Results available: Yes
• Date Posted: 22/03/2020
• URL: https://www.clinicaltrialsregister.eu/ctr-search/trial/2010-021781-29/results