This is a clinical study to investigate how well Biostate works in treatment of male patients below the age of 12 years who have a clotting factor deficiency that is aggravated by the development of antibodies. The antibodies are directed against the clotting factor that is given for replacement therapy and usually make therapy unsuccessful. The treatment used in this study is called immune tollerance therapy.

• Conditions: Male paediatric subjects with Haemophilia A who developed high titre antibodies to human coagulation Factor VIII; MedDRA version: 14.1Level: LLTClassification code 10018941Term: Haemophilia NOSSystem Organ Class: 10010331 - Congenital, familial and genetic disorders; Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15]

• Registration Number: EUCTR2010-020113-85-IT
• Lead Sponsor: CSL BEHRING S.P.A.

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Study Start: 2012-03-07
• Last Update Posted: 14 April 2014

Recruitment & Eligibility

• Status: ot Recruiting
• Sex: Male
• Target Recruitment: 20

Inclusion Criteria

1.Male subjects diagnosed with haemophilia A (=2% FVIII level in the absence of factor replacement, according to their medical history). 2.Age 28 days to <12 years. 3.ITI treatment can be initiated if: a.An inhibitor level of >5 and <200 BU/mL was confirmed in 2 repeated tests by the central laboratory during the screening period and no waiting period is required as judged by the investigator. b.An inhibitor level of >5 and <200 BU/mL was confirmed in 2 repeated tests by the central laboratory during the screening period and the inhibitor titre decreased from the peak titre during a waiting period (maximum 11 months) added at the discretion of the investigator. c.A subject has pre-existing inhibitors, determined no more than 11 months prior to the Screening visit, which decreased from the peak titre, and an inhibitor level of >5 and <200 BU/mL was confirmed in 2 repeated tests by the central laboratory during the screening period. A waiting period could still be added if the total duration from inhibitor diagnosis until start of ITI treatment (ie, including the screening period) does not exceed 12 months. 4.The subject and/or his legally acceptable representative understand the nature of the study and have given written informed consent to participate in the study. 5.Sufficient peripheral venous access or central venous line.
Are the trial subjects under 18? yes
Number of subjects for this age range: 15
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. The subject has received ITI previously. 2. Subjects with a historical peak inhibitor titre of =200 BU/mL. 3. Concomitant treatment with drugs with immunosuppressive side effects (eg, systemic corticosteroids), azathioprine, cyclophosphamide, high dose immunoglobulin or the use of a protein A column or plasmapheresis and interferons. 4. High risk of cardiovascular, cerebrovascular, or other thromboembolic events (excluding catheter thrombosis) as judged by the investigator. 5.Subjects who are human immunodeficiency virus (HIV)-1 or HIV-2 positive (as reported in the medical records or determined at screening). 6.The subject has evidence or a history (within the previous 12 months) of abuse of any drug substance, licit or illicit. 7.The subject has a known or suspected hypersensitivity or has previous evidence of severe side effects to von Willebrand factor (VWF)/FVIII or FVIII concentrates or human albumin. 8.The subject has participated in a clinical study or used an investigational compound (eg, a new chemical entity not approved for clinical use) in the past 3 months, unless the study was for haemophilia A and the subject developed an inhibitor (then, a wash-out period of at least 4 weeks must be applied), or is planning to enter such a study during the study period. 9.Subjects or legal guardians/representatives with suspected inability (eg, language problems) or unwillingness to comply with study procedures. 10.The subject has an acute or chronic medical condition other than haemophilia A, which may, in the opinion of the investigator, affect the conduct of the study. 11.Mental condition rendering the subject (or the subject’s legally acceptable representative) unable to understand the nature, scope and possible consequences of the study). 12.Any condition that is likely to interfere with evaluation of the investigational medicinal product (IMP) or satisfactory conduct of the study. 13.Employee at the study site, or spouse/partner or relative of the investigator or subinvestigators.

Study & Design

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