Adults 18 to 75, male only, with Hemophilia A. Patients with the condition only — healthy volunteers not accepted.
Results — posted to ClinicalTrials.gov
Per-arm endpoint measurements with 95% confidence intervals where reported. Source: trial results section.
Number of Participants With BAX 888-Related Adverse Events (AEs)Primary· From first dose up to end of the study (approximately 6 years)
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) is 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, physi
Group
Value
95% CI
Cohort 1: BAX 888 2.0*10^12 cp/kg
2
Cohort 2: BAX 888 6.0*10^12 cp/kg
2
Change From Baseline in Circulating Plasma FVIII Activity LevelSecondary· Baseline, up to Month 60
Change from baseline in circulating plasma FVIII activity level, based on one-stage clotting assay was assessed.
Group
Value
95% CI
Cohort 1: BAX 888 2.0*10^12 cp/kg
11.40
± 1.131
Cohort 2: BAX 888 6.0*10^12 cp/kg
248.30
± NA
Number of Participants With Clinically Significant Change From Baseline in Circulating Plasma FVIII Antigen LevelSecondary· Baseline, up to Month 60
Change from baseline in circulating plasma FVIII antigen (protein) levels were to be assessed and number of participants with clinically significant change as determined by the principal investigator (PI) were reported.
Group
Value
95% CI
Cohort 1: BAX 888 2.0*10^12 cp/kg
2
Cohort 2: BAX 888 6.0*10^12 cp/kg
0
Annualized Bleed Rate (ABR)Secondary· Up to approximately 6 years 4 months
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. ABR was calculated as (number of bleeding episodes/observed treatment period in days)\*365.25.
Group
Value
95% CI
Cohort 1: BAX 888 2.0*10^12 cp/kg
1.0
± 1.41
Cohort 2: BAX 888 6.0*10^12 cp/kg
0.5
± 0.71
Percentage of Participants With a Reduction in Consumption of Exogenous FVIIISecondary· Up to approximately 6 years 4 months
The reduction in consumption of exogenous FVIII was assessed by comparing the amount of exogenous FVIII taken at earliest time point available (prior to BAX 888 infusion) with the amount taken at the last post-infusion timepoint available, during the study. Percentage of participants with reduction in consumption of exogenous FVIII are reported.
Group
Value
95% CI
Cohort 1: BAX 888 2.0*10^12 cp/kg
0.0
Cohort 2: BAX 888 6.0*10^12 cp/kg
0.0
Number of Participants Who Developed Inhibitory Antibodies to FVIIISecondary· Up to approximately 6 years 4 months
Participants were assessed to check if they developed inhibitory antibodies to FVIII.
Group
Value
95% CI
Cohort 1: BAX 888 2.0*10^12 cp/kg
0
Cohort 2: BAX 888 6.0*10^12 cp/kg
0
Number of Participants Who Developed Total Binding Antibodies to FVIIISecondary· Up to approximately 6 years 4 months
Participants were assessed to check if they developed total binding antibodies to FVIII (Immunoglobulin G \[IgG\], Immunoglobulin M \[IgM\]).
Group
Value
95% CI
Cohort 1: BAX 888 2.0*10^12 cp/kg
0
Cohort 2: BAX 888 6.0*10^12 cp/kg
0
Number of Participants With Humoral and Cell-Mediated Immune Response to AAV8 and FVIII ProteinsSecondary· Up to approximately 6 years 4 months
The humoral (antibody-mediated) and cell-mediated immune response to adeno-associated virus (AAV8) (the vector) and FVIII proteins, was assessed. Humoral Immune Response: It is indicated by presence of specific antibodies. The anti-AAV8 binding antibodies, IgG or IgM were measured by the enzyme-linked immunosorbent assay (ELISA) method. Neutralizing antibodies were measured by a cell-based luminescent assay. Cell-mediated Immune response: The AAV8 and FVIII specific cell mediated immunity was assessed using validated interferon-γ (IFN-γ) enzyme-linked immunosorbent spot (ELISpot) assays. This
Humoral: Binding Ab to AAV8 IgG Titer
Group
Value
95% CI
Cohort 1: BAX 888 2.0*10^12 cp/kg
2
Cohort 2: BAX 888 6.0*10^12 cp/kg
1
Humoral: Neutralising Ab to AAV8 Titer
Group
Value
95% CI
Cohort 1: BAX 888 2.0*10^12 cp/kg
2
Cohort 2: BAX 888 6.0*10^12 cp/kg
1
Cell-Mediated: AAV8 Peptide Pool 1 Mean
Group
Value
95% CI
Cohort 1: BAX 888 2.0*10^12 cp/kg
0
Cohort 2: BAX 888 6.0*10^12 cp/kg
1
Cell-Mediated: AAV8 Peptide Pool 2 Mean
Group
Value
95% CI
Cohort 1: BAX 888 2.0*10^12 cp/kg
1
Cohort 2: BAX 888 6.0*10^12 cp/kg
1
Cell-Mediated: AAV8 Peptide Pool 3 Mean
Group
Value
95% CI
Cohort 1: BAX 888 2.0*10^12 cp/kg
1
Cohort 2: BAX 888 6.0*10^12 cp/kg
1
Cell-Mediated: FVIII Peptide Pool 1 Mean
Group
Value
95% CI
Cohort 1: BAX 888 2.0*10^12 cp/kg
0
Cohort 2: BAX 888 6.0*10^12 cp/kg
0
Cell-Mediated: FVIII Peptide Pool 2 Mean
Group
Value
95% CI
Cohort 1: BAX 888 2.0*10^12 cp/kg
1
Cohort 2: BAX 888 6.0*10^12 cp/kg
1
Surveillance of AAV8 Genome SheddingSecondary· Blood: Day 1, weekly at Clinic Visits between Weeks 1-15, and at Months 4 and 5; Saliva, Semen, and Stool: Day 1 and Week 1; Urine: Day 1 and Weeks 1,2,3
Surveillance of AAV8 genome shedding in blood, saliva, semen, stool and urine until two consecutive negative results were assessed.
Blood: Day 1
Group
Value
95% CI
Cohort 2: BAX 888 6.0*10^12 cp/kg
3684434.0
± NA
Blood: Week 1
Group
Value
95% CI
Cohort 1: BAX 888 2.0*10^12 cp/kg
3634.0
± NA
Cohort 2: BAX 888 6.0*10^12 cp/kg
18608.0
± 664.68
Blood: Week 2
Group
Value
95% CI
Cohort 1: BAX 888 2.0*10^12 cp/kg
2076.0
± 295.57
Cohort 2: BAX 888 6.0*10^12 cp/kg
10670.0
± 3900.40
Blood: Week 3
Group
Value
95% CI
Cohort 1: BAX 888 2.0*10^12 cp/kg
2069.0
± 741.05
Cohort 2: BAX 888 6.0*10^12 cp/kg
9845.0
± 6488.41
Blood: Week 4
Group
Value
95% CI
Cohort 1: BAX 888 2.0*10^12 cp/kg
1098.0
± NA
Cohort 2: BAX 888 6.0*10^12 cp/kg
9033.5
± 4978.74
Blood: Week 5
Group
Value
95% CI
Cohort 1: BAX 888 2.0*10^12 cp/kg
1483.0
± 1097.43
Cohort 2: BAX 888 6.0*10^12 cp/kg
7884.0
± 288.50
Blood: Week 6
Group
Value
95% CI
Cohort 1: BAX 888 2.0*10^12 cp/kg
904.0
± 335.17
Cohort 2: BAX 888 6.0*10^12 cp/kg
5372.5
± 441.94
Blood: Week 7
Group
Value
95% CI
Cohort 1: BAX 888 2.0*10^12 cp/kg
1004.0
± 656.20
Cohort 2: BAX 888 6.0*10^12 cp/kg
6808.0
± 1121.47
Adverse events — posted to ClinicalTrials.gov
Time frame: All-cause mortality: Up to approximately 6 years 4 months; SAEs and Other (Non-Serious) AEs: From first dose up to end of the study (approximately 6 years).
Reporting threshold: 5%.
Adverse-event reports describe events observed during the trial — not all are caused by the drug.
The main aim of this study is to check if there are side effects from BAX 888 and to determine the dose of BAX 888 for treating severe hemophilia A in male adults.
Participants will receive one infusion with BAX 888 at the hemophilia treatment center. During the study, participants will visit their study clinic multiple times.
Publications & conference data
8 peer-reviewed publications reference this trial (live from Europe PMC):
NCT07416526 — A Clinical Study to Evaluate the Effects of NXT007 Compared to Factor VIII Prophylaxis in Participants With Hemophilia A
· Phase 3
· recruiting
NCT07416604 — A Clinical Study to Evaluate the Effects of NXT007 Compared to Emicizumab Prophylaxis in People With Hemophilia A
· Phase 3
· recruiting
NCT07523399 — Joint Health, Balance and Quality of Life in Adults With Hemophilia A
· recruiting
NCT06833983 — To Evaluate the Clinical Study of GS1191-0445 Injection in the Treatment of Hemophilia A
· Phase 3
· recruiting
NCT06579144 — Pharmacokinetic Comparison of Efanesoctocog Alfa vs Other EHL-rFVIII Products in Participants With Severe Haemophilia A
· Phase 1
· recruiting
Other Baxalta now part of Shire trials
Trials by the same sponsor.
NCT04985682 — A Study of ADVATE in People With Hemophilia A in India
· Phase 4
· completed
NCT04578535 — A Study to Assess the Tolerability, Safety, and Pharmacokinetics of Subcutaneous Immune Globulin Infusion 10% (Human) Wi
· Phase 1
· completed
NCT04346108 — A Study of Immune Globulin Subcutaneous (Human), 20% Solution (IGSC, 20%) in Japanese Participants With Primary Immunode
· Phase 3
· completed
NCT04158934 — A Long-term Study of ADYNOVI/ADYNOVATE in Participants With Haemophilia A
· active not recruiting
NCT04394286 — A Phase 1/2 Study of SHP648, an Adeno-Associated Viral Vector for Gene Transfer in Hemophilia B Subjects
· Phase 1, PHASE2
· terminated
Publications: Europe PMC API search by NCT ID, retrieved 10 June 2026
Drug + disease cross-links: matched in real time against Drug Landscape's normalised drug + company + condition tables
Sponsor: as reported to ClinicalTrials.gov by Baxalta now part of Shire
Last refreshed: 11 September 2025
Drug Landscape aggregates and links these public records for informational use only. Always verify against the primary source before clinical or regulatory decisions. Canonical URL: https://druglandscape.com/trial/NCT03370172.