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BAX 888Wiki

Developed by Shray Alag
Clinical Trial MeSH HPO Drug Gene SNP Protein Mutation


Correlated Drug Terms (0)


Name (Synonyms) Correlation

Correlated MeSH Terms (1)


Name (Synonyms) Correlation
D006467 Hemophilia A NIH 0.50

Correlated HPO Terms (1)


Name (Synonyms) Correlation
HP:0003125 Reduced factor VIII activity HPO 0.50

There is one clinical trial.

Clinical Trials


1 A Global, Open-Label, Multicenter, Phase 1/2 Study of the Safety and Dose Escalation of BAX 888, an Adeno-Associated Virus Serotype 8 (AAV8) Vector Expressing B-Domain Deleted Factor VIII (BDD-FVIII) in Severe Hemophilia A Subjects Administered a Single Intravenous Infusion

The purpose of this study is to evaluate the safety and determine the dose of BAX 888.

NCT03370172 Hemophilia A Drug: BAX 888
MeSH:Hemophilia A
HPO:Reduced factor VIII activity

Primary Outcomes

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 Years

Secondary Outcomes

Description: 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 participant

Description: 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 participant

Description: 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 participant

Description: 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-infusion

Description: 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 participant

Description: 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 participant

Description: 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 participant

Description: 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


Related HPO nodes (Using clinical trials)