EUCTR2010-020113-85-FR进行中（未招募）1 期
A Multicentre, Interventional, Non-randomized, Open-label, Single-group Phase III Study to evaluate Plasma-Derived Antihaemophilic Factor/von Willebrand Factor Concentrate (Biostate®) for Immune Tolerance Induction in Male Paediatric Subjects with Haemophilia A (=2%) who have Developed High-titre Antibodies to Factor VIII (Factor VIII Inhibitors) - SWIFT-ITI
适应症
试验速览
- 阶段
- 1 期
- 状态
- 进行中（未招募）
- 入组人数
- 1
研究概览
简要总结
暂无简介。
研究设计
- 研究类型
- Interventional clinical trial of medicinal product
入排标准
- 性别
- Male
入选标准
- •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: 20
- •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
排除标准
- •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 Product, Dose, and Mode of Administration:
- •The IMP Biostate will be intravenously (i.v.) administered at a daily dose of 200 international units (IU)/kg body weight (b.w.), preferably split into 2 doses of 100 IU/kg b.w. per day.
- •For subjects with an undetectable inhibitor titre (<0.6 BU/mL) at 2 consecutive assessments, tested at intervals of 2 weeks (±3 days), and normal recovery (=66% of predicted) for 6-8 weeks, the daily Biostate dose will be reduced by 20 IU/kg b.w. (ie, by 10% of the initial daily ITI dose), if possible, every 2-4 weeks down to a dose of 100 IU/kg b.w., provided the recovery remains normal during that time (assessed weekly).
- •After further gradual reduction of the dose and extension of the administration interval (at the discretion of the investigator), Biostate will be administered as prophylaxis at a daily dose of 50 IU/kg b.w. on 3 days per week (about every second day).
- •Any daily dose >100 IU/kg b.w. should preferably be evenly split into 2 doses per day. Daily doses of 100 IU/kg b.w. or lower will be administered once daily.
- •Bleeding complications during the tolerization period should be treated according to centre’s standard of care. Study treatment should not be interrupted for surgical procedures.
研究者
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