A clinical trial investigating safety and efficacy of a long-acting factor VIIIin children (age <12 years) with severe hemophila A

• Conditions: severe hemophilia A (<1% FVIII:C); MedDRA version: 18.0Level: SOCClassification code 10010331Term: Congenital, familial and genetic disordersSystem Organ Class: 10010331 - Congenital, familial and genetic disorders; MedDRA version: 18.0Level: LLTClassification code 10060612Term: Hemophilia ASystem Organ Class: 10010331 - Congenital, familial and genetic disorders; Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15]

• Registration Number: EUCTR2012-004434-42-Outside-EU/EEA
• Lead Sponsor: Bayer HealthCare AG

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Study Start: 2013-05-30
• Last Update Posted: 14 September 2015

Recruitment & Eligibility

• Status: A
• Sex: Male
• Target Recruitment: 60

Inclusion Criteria

1. Male, age <12 years to be enrolled in 2 subgroups:
- age 6 to <12 years
- age < 6 years

Subjects in the expansion group are to be <6 years of age.

2. Severe hemophilia A defined as < 1% factor VIII concentration (FVIII:C) by measurement at the time of screening or from reliable prior documentation (eg, measurement in other clinical trials, results from approved clinical laboratory, or
diagnostic genetic testing)

3. > 50 ED with any FVIII concentrate(s) (plasma derived or recombinant)

4. Willingness and ability of subjects and/or parents to complete training in the use of the
EPD and to document infusions during the study

5. Written informed consent by parent/legal representative. Assent should be sought from
subjects, if appropriate
Are the trial subjects under 18? yes
Number of subjects for this age range: 60
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

1. Current evidence of inhibitor to FVIII measured using the Nijmegen-modified Bethesda assay (>0.6 BU/mL) at the time of screening (central laboratory). Subjects should not have received FVIII within 72 h prior to the collection of screening
samples and should have had FVIII administered within the prior 2-3 weeks.

2. History or presence of Factor VIII inhibitors. Inhibitor to FVIII is defined as a titer
>0.6 BU/mL or clinical history suggestive of inhibitor requiring modification of treatment. (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 subsequent negative results [<0.6 BU] are eligible.)

3. Any other inherited or acquired bleeding disorder in addition to hemophilia A

4. Platelet count < 100,000/mm3

5. Creatinine > 2x upper limit of normal

6. Aspartate aminotransferase (AST) or Alanine aminotransferase (ALT) > 5x upper
limit of normal

7. Known hypersensitivity to the drug substance, or any of its components (eg, mouse or
hamster protein)

8. The subject is currently participating in another investigational drug study, or has participated in a clinical study involving an investigational drug within 30 days of study entry. Subjects who are currently participating in an investigational study in
which they are treated with a currently marketed FVIII concentrate are not excluded. Subjects currently treated with BAY 81-8973 may continue treatment with the product
up to the start of Visit 1.

9. Any individual who is receiving chemotherapy, immune modulatory drugs other than anti-retroviral chemotherapy, or chronic use of oral or intravenous (IV) corticosteroids (> 14 days) within the last 3 months.

10. The subject is identified by the investigator as being unable or unwilling to perform study procedures.

11. Previous assignment to treatment during this study.

Study & Design

• Study Type: Interventional clinical trial of medicinal product
• Study Design: Not specified