Name (Synonyms) | Correlation |
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Name (Synonyms) | Correlation | |
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HP:0003125 | Reduced factor VIII activity HPO | 0.50 |
There is one clinical trial.
The purpose of this study is to evaluate the safety and determine the dose of BAX 888.
Description: An AE is defined as any untoward medical occurrence in a participant administered an investigational product (IP) that does not necessarily have a causal relationship with the treatment. A Serious adverse event (SAE) was an AE resulting in any of the following outcomes: death; life-threatening event; required or prolonged inpatient hospitalization; persistent or significant disability/incapacity; congenital anomaly. AEs include both serious and non-serious adverse events including development of FVIII inhibitory antibodies, clinically significant changes in standard laboratory parameters, physical exam, and vital signs.
Measure: Number of Participants With BAX 888-Related Adverse Events (AEs) Time: From study drug administration to 3 YearsDescription: Change from baseline in circulating plasma FVIII activity level, based on one-stage clotting assay will be assessed.
Measure: Change from Baseline in Circulating Plasma FVIII Activity Level Time: Baseline, up to approximately 3 years per participantDescription: Change from baseline in circulating plasma FVIII antigen (protein) levels will be assessed.
Measure: Change from Baseline in Circulating Plasma FVIII Antigen Level Time: Baseline, up to approximately 3 years per participantDescription: Annualized bleed rate (ABR) in comparison to before gene transfer will be assessed. A bleed is defined as subjective or objective evidence of bleeding which may or may not require treatment with FVIII. Annualized bleed rate (ABR) was calculated as (number of bleeding episodes/observed treatment period in days)*365.25.
Measure: Annualized bleed rate (ABR) Time: Throughout the study period of approximately 3 years per participantDescription: The percentage of participants with a reduction in exogenous FVIII consumption 12 months post-infusion and 3 years post-infusion compared to the historical consumption (consumption of exogenous FVIII during the 12 month period prior to BAX 888 infusion).
Measure: Percentage of Participants with a Redaction Consumption of Exogenous Factor VIII (FVIII) Time: Historical data from 12 months prior to study enrollment; and 12 months post-infusion and 3 years post-infusionDescription: Number of participants develop inhibitory antibodies to FVIII will be assessed.
Measure: Number of Participants Develop Inhibitory Antibodies to FVIII Time: Throughout the study period of approximately 3 years per participantDescription: Number of participants develop total binding antibodies to FVIII (Immunoglobulin G [IgG] and Immunoglobulin M [IgM]) will be assessed.
Measure: Number of Participants Develop Total Binding Antibodies to FVIII Time: Throughout the study period of approximately 3 years per participantDescription: Number of participants with humoral (antibody-mediated) and cell-mediated immune response to adeno-associated virus (AAV8) (the vector) and FVIII proteins will be assessed.
Measure: Number of Participants With Humoral and Cell-Mediated Immune Response to AAV8 and Factor VIII (FVIII) Proteins Time: Throughout the study period of approximately 3 years per participantDescription: Surveillance of adeno-associated virus (AAV8) genome shedding in blood, saliva, semen, urine and stool will be assessed.
Measure: Surveillance of AAV8 Genome Shedding Time: Throughout the study period of approximately 3 years per participant,or until 2 consecutive measurements are negative, which ever is sooner