A clinical trial to study the safety and efficacy of PEGylated Factor VIII (BAX 855) in previously untreated patients (PUPs) 6 years with severe hemophilia A (FVIII 1%)
- Conditions
- Health Condition 1: D688- Other specified coagulation defects
- Registration Number
- CTRI/2021/04/032837
- Lead Sponsor
- Baxalta Innovations GmbH
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- Closed to Recruitment of Participants
- Sex
- Not specified
- Target Recruitment
- 0
1.Subject is < 6 years old at the time of screening 2. Subject is previously untreated with < 3 EDs to ADVATE, BAX 855 or Fresh Frozen Plasma
(FFP) at any time prior to screening 3.
Subject has severe hemophilia A (FVIII < 1%) as determined by the central laboratory, or a historical FVIII level < 1% as determined at any local laboratory, optionally supported by an additional FVIII gene mutation consistent with severe hemophilia A 4.
Subject is immune competent with a CD4+ count > 200 cells/mm3, as confirmed by central laboratory at screening 5. Parent or legally authorized representative is willing and able to comply with the requirements of the protocol
Additional inclusion criteria for Part B (ITI): 1 Parent or legal representative has/have voluntarily provided signed informed consent for ITI portion 2 Subject has a confirmed positive high titer inhibitor ( > 5.00 BU) or has a positive confirmed low titer inhibitor (� 0.6 BU) as determined by the central laboratory based on a second repeat blood sample with a) poorly controlled bleeding despite increased BAX 855 doses, or b) requires bypassing agents to treat bleeding episodes
1 Subject has detectable FVIII inhibitory antibodies (�0.6 BU using the Nijmegen modification of the Bethesda assay) as confirmed by central laboratory at screening
2 S2.Subject has a history of FVIII inhibitory antibodies (� 0.6 BU using the Nijmegen modification of the Bethesda assay or the Bethesda assay) at any time prior to screening
3 Subject has been diagnosed with an inherited or acquired hemostatic defect other than
hemophilia A (eg, qualitative platelet defect or von Willebrandââ?¬•s disease)
4 Subject has been previously treated with cryoprecipitate or any type of FVIII concentrate other
than ADVATE, BAX 855v or FFP, or was administered ADVATE,BAX 855 or FFP for � 3 Eds at any time prior to screening.
5 Subject has received any kind of blood-transfusion such as PRBC, platelets or plasma prior to
screening at any time prior to screening
6 The subjectââ?¬•s weight is < 5 kg
7 Subjectââ?¬•s platelet count is < 100,000/mL
8 Subject has known hypersensitivity towards mouse or hamster proteins, PEG or Tween 80
9 Subject has severe chronic hepatic dysfunction [eg, > 5 times upper limit of normal alanine
aminotransferase (ALT), aspartate aminotransferase (AST), or a documented INR > 1.5] in his
medical history or at the time of screening
10 Subject has severe renal impairment (serum creatinine > 1.5 times the upper limit of normal)
11 Subject has current or recent ( < 30 days) use of other PEGylated drugs prior to study participation or is scheduled to use such drugs during study participation
1212.Subject is scheduled to receive during the course of the study a systemic immunomodulating
drug (e.g. corticosteroid agents at a dose equivalent to hydrocortisone greater than 10 mg/day, or
�±-interferon) other than anti-retroviral chemotherapy
13 Subject has participated in another clinical study involving an IP or investigational device within 30 days prior to enrollment or is scheduled to participate in another clinical study involving an IP or investigational device during the course of this study
14 14.Parent or legally authorized representative has a medical, psychiatric, or cognitive illness or recreational drug/alcohol use that, in the opinion of the investigator, would affect subject safety or compliance
15 Parent, legally authorized representative or subject are a member of the team conducting this
study or is in a dependent relationship with one of the study team members. Dependent
relationships include close relatives (ie, children, partner/spouse, siblings, parents) as well as
employees of the investigator or site personnel conducting the study.
Additional exclusion criteria for Part B (ITI)
1 Spontaneous disappearance of the inhibitor prior to ITI
2 FVIII inhibitor titer � 0.6 BU is not confirmed by a second new blood sample drawn within
2 weeks of study site notification of inhibitor and determined at the central laboratory
3 Inability or unwillingness to comply with the protocol
Study & Design
- Study Type
- Interventional
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method Incidence of FVIII inhibitor development Success rate of Immune tolerance induction (ITI)Timepoint: Throughout Part A of the study, approximately 5 years <br/ ><br> Up to 33 months <br/ ><br>
- Secondary Outcome Measures
Name Time Method