Eine Studie zur Beurteilung der Wirksamkeit und Sicherheit von Emicizumab bei Neugeborenen und Säuglingen bis zum Alter von 12 Monaten mit Hämophilie A ohne FVIII-Hemmkörper.

Trade Name: Hemlibra
Product Name: Hemlibra / Emicizumab
Product Code: RO5534262/ACE910
Pharmaceutical Form: Solution for injection
INN or Proposed INN: EMICIZUMAB
CAS Number: 1610943-06-0
Current Sponsor code: RO553-4262
Other descriptive name: HEMLIBRA
Concentration unit: mg/ml milligram(s)/millilitre
Concentration type: equal
Concentration number: 30-

Trade Name: Hemlibra
Product Name: Hemlibra / Emicizumab
Product Code: RO5534262/ACE910
Pharmaceutical Form: Solution for injection
INN or Proposed INN: EMICIZUMAB
CAS Number: 1610943-06-0
Current Sponsor code: RO553-4262
Other descriptive name: HEMLIBRA
Concentration unit: mg/ml milligram(s)/millilitre
Concentration type: equal
Concentration number: 150-

Gender:
Female: yes
Male: yes

Inclusion criteria:
Inclusion criteria:
?Age from birth to <= 12 months at time of informed consent
?Body weight >= 3 kg at time of informed consent
?Mandatory receipt of vitamin K prophylaxis according to local standard practice
?Diagnosis of severe congenital hemophilia A (intrinsic FVIII level < 1%)
?A negative test for FVIII inhibitor (i.e., < 0.6 Bethesda units [BU]/mL) locally assessed during the 2-week screening period for all patients
?For PUPs or MTPs, up to 5 days of exposure with hemophilia-related treatments, such as plasma-derived FVIII, recombinant FVIII, fresh frozen plasma, cryoprecipitate, or whole blood products
?Documentation of the details of the hemophilia-related treatments received since birth
?Documentation of the details of the bleeding episodes since birth
?For patients from birth to < 3 months of age at the time of study entry: no evidence of active ICH, as confirmed by a negative cranial ultrasound at screening irrespective of delivery mode
?Adequate hematologic, hepatic, and renal function

Are the trial subjects under 18? yes
Number of subjects for this age range: 50
F.1.2 Adults (18-64 years) no
F.1.2.1 Number of subjects for this age range 0
F.1.3 Elderly (>=65 years) no
F.1.3.1 Number of subjects for this age range 0

Exclusion criteria:
Exclusion criteria
?Inherited or acquired bleeding disorder other than severe hemophilia A
?Use of systemic immunomodulators (e.g., interferon) at enrollment or planned use
?during the study
?Receipt of any of the following:
? An investigational drug to treat or reduce the risk of hemophilic bleeds within 5 drug-elimination half-lives of last drug administration
? A non-hemophilia-related investigational drug within the last 30 days or 5 drug-elimination half-lives, whichever is shorter
? An investigational drug concurrently
? Prior use of emicizumab prophylaxis including investigational or commercial emicizumab
?Current active severe bleed, such as ICH
?Planned surgery during the study
?History of clinically significant hypersensitivity associated with monoclonal antibody therapies or components of the emicizumab injection
? Patients who are at high risk for TMA (e.g., have a previous medical or family history of TMA, such as thrombotic thrombocytopenic
purpura, atypical hemolytic uremic syndrome) in the investigator's judgment
?Previous or current treatment for thromboembolic disease or signs of thromboembolic disease
?Any hereditary or acquired maternal condition that may predispose the patient to thrombotic events
?Other diseases (e.g., certain autoimmune diseases) that may increase risk of bleeding or thrombosis
?Known infection with HIV, hepatitis B virus, or hepatitis C virus
?Serious infection requiring antibiotics or antiviral treatments within 14 days prior to screening
?Concurrent disease, treatment, abnormality in clinical laboratory tests, vital signs measurements, or physical examination findings that could interfere with the conduct of the study or that would, in the opinion of the investigator or Sponsor, preclude the patient?s safe participation in and completion of the study or interpretation of the study results
?Unwillingness of the parent or caregiver to allow receipt of blood or blood products, or any standard-of-care treatment for a life-threatening condition
?Any other medical, social, or other condition that may prevent adequate compliance with the study protocol in the opinion of the investigator