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

• Register: EUCTR
• Last refreshed on: 16 November 2020
• Main ID: EUCTR2010-021781-29-SE
• Date of registration: 28/12/2010
• Prospective Registration: Yes
• Primary sponsor: Bayer HealthCare AG
• Public 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
• 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 - Leopold Kids
• Date of first enrolment: 01/02/2011
• 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:
• 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" /
• Affiliation: Bayer HealthCare AG

• Name: CTP Team / Ref: "EU CTR" /
• 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 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.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 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

Health Condition(s) or Problem(s) studied

Severe Hemophilia-A (< 1% FVIII:C)
MedDRA version: 17.0 Level: LLT Classification code 10060612 Term: Hemophilia A System Organ Class: 100000004850

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

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)

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.

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

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.

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

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: 01/02/2011
Contact:

Results

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