Pharmacokinetic, Efficacy, and Safety Study of Recombinant Factor VIII Single Chain (rVIII-SingleChain) in Children With Severe Hemophilia A
Condition: Congenital Hemophilia A
MeSH terms: Hemophilia A
Intervention: rVIII-SingleChain
Intervention MeSH terms: Factor VIII
Phase: Phase 3
Study type: Interventional
Primary purpose: Treatment
Intervention model: Single Group Assignment
Masking: None (Open Label)
Enrollment: 84.0
Sex: Male
Maximum age: 11 Years
Healthy volunteers: No

Summary:

      This is an international, multicenter, open-label study to assess the efficacy, safety, and
      pharmacokinetic (PK) profile of rVIII-SingleChain in pediatric patients with severe
      hemophilia A. A minimum of 25 previously treated subjects ≥ 6 to < 12 years of age and at
      least 25 subjects < 6 years of age who have undergone > 50 exposure days (EDs) with a
      previous Factor VIII (FVIII) product are planned to be enrolled. Subjects will be assigned to
      either an on-demand or prophylaxis treatment regimen and will receive rVIII-SingleChain at a
      dose to be determined by the investigator. Hemostatic efficacy will be assessed by the
      subject/caregiver and the investigator who will assess overall efficacy by a 4-point scale.
    

Eligibility criteria:

        Inclusion Criteria:

          -  Diagnosis of severe hemophilia A defined as < 1% Factor VIII (FVIII) concentration
             (FVIII:C) documented in medical records,

          -  Males < 12 years of age,

          -  Subjects who have received > 50 EDs with a FVIII product,

          -  Prior PK data (at least incremental recovery and half-life) from previous FVIII
             exposure for subjects participating in the PK part

          -  Investigator believes that the subject is willing and able to adhere to all protocol
             requirements. Investigator believes that the subject's parent(s) or legally acceptable
             representative(s) is / are willing and able to adhere to all protocol requirements.

        Exclusion Criteria:

          -  Any history of or current FVIII inhibitors

          -  Use of an Investigational Medical Product (IMP) within 30 days prior to the first
             rVIII-SingleChain administration,

          -  Administration of any cryoprecipitate, whole blood or plasma within 30 days prior to
             administration of rVIII-SingleChain,

          -  Known hypersensitivity (allergic reaction or anaphylaxis) to any FVIII product or
             hamster protein,

          -  Subject currently receiving IV immunomodulating agents such as immunoglobulin or
             chronic systemic corticosteroid treatment,

          -  Subject with serum aspartate aminotransferase (AST) or serum alanine aminotransferase
             (ALT) values >5 times (x) the upper limit of normal (ULN) at Screening,

          -  Subjects with serum creatinine values >2 x ULN at Screening,

          -  Evidence of thrombosis, including deep vein thrombosis, stroke, pulmonary embolism,
             myocardial infarction and arterial embolus within 3 months before Day 1,

          -  Experienced life-threatening bleeding episode or had major surgery or an orthopedic
             surgical procedure during the 3 months before rVIII-SingleChain administration.