CovidResearchTrials by Shray Alag

CovidResearchTrials Covid 19 Research using Clinical Trials (Home Page)

PEGylated Recombinant Factor VIIIWiki

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 Phase 3, Prospective, Multi-center, Open Label Study to Investigate Safety, Immunogenicity and Hemostatic Efficacy of PEGylated Factor VIII (BAX 855) in Previously Untreated Patients (PUPs) < 6 Years With Severe Hemophilia A (FVIII < 1%)

The purpose of this study is to investigate safety, immunogenicity and hemostatic efficacy of PEGylated recombinant FVIII (BAX 855) in previously untreated patients (PUPs) < 6 years of age with severe hemophilia A (baseline FVIII level < 1%) and < 3 EDs to ADVATE, BAX 855 or plasma transfusion.

NCT02615691 Hemophilia A Biological: PEGylated Recombinant Factor VIII
MeSH:Hemophilia A
HPO:Reduced factor VIII activity

Primary Outcomes

Measure: Incidence of FVIII inhibitor development

Time: Throughout Part A of the study, approximately 5 years

Measure: Success rate of Immune tolerance induction (ITI)

Time: Up to 33 months

Secondary Outcomes

Description: Binding IgG and IgM antibodies to Factor VIII (FVIII), Factor VIII-Polyethylene glycol (PEG-FVIII) and Polyethylene glycol (PEG)

Measure: Binding Immunoglobulin G (IgG) and Immunoglobulin M (IgM) antibodies

Time: Throughout Part A of the study, approximately 5 years

Measure: Clinically significant adverse events (AEs) and serious adverse events (SAEs)

Time: Throughout Part A and Part B of the study, approximately 7 years

Measure: Clinically significant changes in vital signs

Time: Throughout Part A and Part B of the study, approximately 7 years

Measure: Clinically significant changes in clinical laboratory parameters

Time: Throughout Part A and Part B of the study, approximately 7 years

Measure: Annualized bleeding rate (ABR) for prophylactic and on-demand treatment

Time: Throughout Part A of the study, approximately 5 years

Measure: Number of BAX 855 infusions per bleeding episode

Time: Throughout Part A of the study, approximately 5 years

Measure: Overall hemostatic efficacy rating

Time: 24 h after initiation of treatment and at resolution of bleed

Measure: Weight-adjusted consumption of BAX 855 per month, per year and per event

Time: Throughout Part A of the study, approximately 5 years

Measure: Number of infusions per month and per year

Time: Throughout Part A of the study, approximately 5 years

Measure: Assessment of intra-, post- and perioperative hemostatic efficacy in case of surgery

Time: Surgery Day 0 up to postoperative Day 14 or discharge (whichever occurs first)

Measure: Intra- and postoperative blood loss in case of surgery

Time: Surgery Day 0 up to postoperative Day 14 or discharge (whichever occurs first)

Measure: Pharmacokinetics- Incremental recovery (IR) at baseline and over time

Time: Pre-infusion within 30 minutes; and post-infusion at 15-30 minutes and 24-48 hours

Measure: Pharmacokinetics- Half-life (T1/2) at baseline (optional)

Time: Post-infusion: 15-30 minutes and 24-48 hours

Measure: Immune tolerance induction (ITI) - Rate of partial success and failure of ITI

Time: Up to 33 months

Measure: Immune tolerance induction (ITI) - annualized bleeding rate (ABR) during ITI

Time: Up to 33 months

Measure: Immune tolerance induction (ITI) - Weight-adjusted consumption of BAX 855 per month and per year for each ITI regimen employed

Time: Up to 33 months

Measure: Immune tolerance induction (ITI) - Catheter-related complications

Time: Up to 33 months

Description: Binding IgG and IgM antibodies to Factor VIII (FVIII), Factor VIII-Polyethylene glycol (PEG-FVIII) and Polyethylene glycol (PEG)

Measure: Immune tolerance induction (ITI) -Binding Immunoglobulin G (IgG) and Immunoglobulin M (IgM) antibodies

Time: Up to 33 months

Related HPO nodes (Using clinical trials)

HP:0003125: Reduced factor VIII activity
Genes 10
F8 F8 VWF MCFD2 LMAN1 F8 MCFD2 F8 LMAN1 VWF
Protein Mutations 1
G20210A
SNP 0