Trial Outcomes & Findings for A Study to Evaluate Prospective Efficacy and Safety Data of Current FIX Prophylaxis Replacement Therapy in Adult Hemophilia B Subjects (FIX:C≤2%) or Current FVIII Prophylaxis Replacement Therapy in Adult Hemophilia A Subjects (FVIII:C≤1%) (NCT NCT03587116)

Results Overview

ABR per participant was calculated as number of bleeds over number of days from baseline visit (Day 1 of the study) to end of study multiplication(\*)365.25 days. All bleeds included treated and untreated bleeds. A treated bleed was defined as an event necessitating administration of coagulation factor within 72 hours of signs or symptoms of bleeding. An untreated bleed was defined as an event not necessitating administration of coagulation factor within 72 hours of signs or symptoms of bleeding. In this outcome measure, ABR for all and treated bleeds during prospective data collection period in hemophilia B participants per efficacy analysis set was reported. Abbreviations used: AAV6 = adeno-associated virus 6; AAV-Spark100 = Bioengineered AAV capsid, derived from a naturally occurring AAV serotype; nAb = neutralizing antibodies.

Recruitment status

COMPLETED

Study phase

PHASE3

Target enrollment

212 participants

Primary outcome timeframe

During prospective data collection period (from baseline visit of the study [Day 1] up to end of study follow-up [maximum follow-up: 1269 days])

Results posted on

2025-11-06

Participant Flow

Participant milestones

Participant milestones
Measure	Standard of Care FIX Replacement Therapy (Hemophilia B) Participants with moderately severe to severe hemophilia B who administered their own current Factor IX (FIX) replacement therapy in the usual healthcare setting were included. No investigational product was administered as a part of this study.	Standard of Care FVIII Replacement Therapy (Hemophilia A) Participants with moderately severe to severe hemophilia A who administered their own current Factor VIII (FVIII) replacement therapy in the usual healthcare setting were included. No investigational product was administered as a part of this study.
Overall Study STARTED	111	101
Overall Study Safety Analysis Set	111	101
Overall Study Efficacy Analysis Set	110	96
Overall Study Per-protocol Analysis Set	107	81
Overall Study Protocol Amendment 5 Analysis Set	39	24
Overall Study COMPLETED	107	99
Overall Study NOT COMPLETED	4	2

Reasons for withdrawal

Reasons for withdrawal
Measure	Standard of Care FIX Replacement Therapy (Hemophilia B) Participants with moderately severe to severe hemophilia B who administered their own current Factor IX (FIX) replacement therapy in the usual healthcare setting were included. No investigational product was administered as a part of this study.	Standard of Care FVIII Replacement Therapy (Hemophilia A) Participants with moderately severe to severe hemophilia A who administered their own current Factor VIII (FVIII) replacement therapy in the usual healthcare setting were included. No investigational product was administered as a part of this study.
Overall Study Lost to Follow-up	0	1
Overall Study No Longer Met Eligibility Criteria	0	1
Overall Study Withdrawal by Subject	4	0

Baseline Characteristics

A Study to Evaluate Prospective Efficacy and Safety Data of Current FIX Prophylaxis Replacement Therapy in Adult Hemophilia B Subjects (FIX:C≤2%) or Current FVIII Prophylaxis Replacement Therapy in Adult Hemophilia A Subjects (FVIII:C≤1%)

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Standard of Care FIX Replacement Therapy (Hemophilia B) n=111 Participants Participants with moderately severe to severe hemophilia B who administered their own current FIX replacement therapy in the usual healthcare setting were included. No investigational product was administered as a part of this study.	Standard of Care FVIII Replacement Therapy (Hemophilia A) n=101 Participants Participants with moderately severe to severe hemophilia A who administered their own current FVIII replacement therapy in the usual healthcare setting were included. No investigational product was administered as a part of this study.	Total n=212 Participants Total of all reporting groups
Age, Customized 18-44 Years	93 Participants n=49 Participants	84 Participants n=50 Participants	177 Participants n=50 Participants
Age, Customized 45-64 Years	18 Participants n=49 Participants	17 Participants n=50 Participants	35 Participants n=50 Participants
Sex: Female, Male Female	0 Participants n=49 Participants	0 Participants n=50 Participants	0 Participants n=50 Participants
Sex: Female, Male Male	111 Participants n=49 Participants	101 Participants n=50 Participants	212 Participants n=50 Participants
Ethnicity (NIH/OMB) Hispanic or Latino	4 Participants n=49 Participants	4 Participants n=50 Participants	8 Participants n=50 Participants
Ethnicity (NIH/OMB) Not Hispanic or Latino	91 Participants n=49 Participants	76 Participants n=50 Participants	167 Participants n=50 Participants
Ethnicity (NIH/OMB) Unknown or Not Reported	16 Participants n=49 Participants	21 Participants n=50 Participants	37 Participants n=50 Participants
Race (NIH/OMB) American Indian or Alaska Native	0 Participants n=49 Participants	0 Participants n=50 Participants	0 Participants n=50 Participants
Race (NIH/OMB) Asian	18 Participants n=49 Participants	17 Participants n=50 Participants	35 Participants n=50 Participants
Race (NIH/OMB) Native Hawaiian or Other Pacific Islander	0 Participants n=49 Participants	0 Participants n=50 Participants	0 Participants n=50 Participants
Race (NIH/OMB) Black or African American	2 Participants n=49 Participants	6 Participants n=50 Participants	8 Participants n=50 Participants
Race (NIH/OMB) White	90 Participants n=49 Participants	78 Participants n=50 Participants	168 Participants n=50 Participants
Race (NIH/OMB) More than one race	1 Participants n=49 Participants	0 Participants n=50 Participants	1 Participants n=50 Participants
Race (NIH/OMB) Unknown or Not Reported	0 Participants n=49 Participants	0 Participants n=50 Participants	0 Participants n=50 Participants

PRIMARY outcome

Timeframe: During prospective data collection period (from baseline visit of the study [Day 1] up to end of study follow-up [maximum follow-up: 1269 days])

Population: Efficacy analysis set: participants who signed an informed consent form (ICF), had their blood sample collected and assayed for AAV-Spark100 or AAV6 immunity testing, who were subsequently identified as nAb negative (negative for nAb to AAV-Spark100 for the hemophilia B cohort), met the inclusion/ exclusion criteria and who participated in the prospective data collection phase as part of their usual healthcare setting.

ABR per participant was calculated as number of bleeds over number of days from baseline visit (Day 1 of the study) to end of study multiplication(\*)365.25 days. All bleeds included treated and untreated bleeds. A treated bleed was defined as an event necessitating administration of coagulation factor within 72 hours of signs or symptoms of bleeding. An untreated bleed was defined as an event not necessitating administration of coagulation factor within 72 hours of signs or symptoms of bleeding. In this outcome measure, ABR for all and treated bleeds during prospective data collection period in hemophilia B participants per efficacy analysis set was reported. Abbreviations used: AAV6 = adeno-associated virus 6; AAV-Spark100 = Bioengineered AAV capsid, derived from a naturally occurring AAV serotype; nAb = neutralizing antibodies.

Outcome measures

Outcome measures
Measure	Standard of Care FIX Replacement Therapy (Hemophilia B) n=110 Participants Participants with moderately severe to severe hemophilia B who administered their own current FIX replacement therapy in the usual healthcare setting were included. No investigational product was administered as a part of this study.	Standard of Care FVIII Replacement Therapy (Hemophilia A) Participants with moderately severe to severe hemophilia A who administered their own current FVIII replacement therapy in the usual healthcare setting were included. No investigational product was administered as a part of this study.
Annualized Bleeding Rate (ABR) for Treated Bleeds and All Bleeds During Prospective Data Collection Period in Hemophilia B Participants: Efficacy Analysis Set Treated bleeds	3.61 Bleeds per year Standard Deviation 7.634	—
Annualized Bleeding Rate (ABR) for Treated Bleeds and All Bleeds During Prospective Data Collection Period in Hemophilia B Participants: Efficacy Analysis Set All bleeds	4.46 Bleeds per year Standard Deviation 9.446	—

PRIMARY outcome

Timeframe: During prospective data collection period (from baseline visit of the study [Day 1] up to end of study follow-up [maximum follow-up: 1269 days])

Population: Per-Protocol analysis set: all participants who signed an ICF, had their blood sample collected and assayed for AAV-Spark100 or AAV6 immunity testing, who were subsequently identified as nAb negative (negative for nAb to AAV-Spark100 for the hemophilia B cohort), met the inclusion/ exclusion criteria and who participated and completed at least 6 months of the prospective data collection phase as part of their usual healthcare setting.

ABR per participant was calculated as number of bleeds over number of days from baseline visit (Day 1 of the study) to end of study\*365.25 days. All bleeds included treated and untreated bleeds. A treated bleed was defined as an event necessitating administration of coagulation factor within 72 hours of signs or symptoms of bleeding. An untreated bleed was defined as an event not necessitating administration of coagulation factor within 72 hours of signs or symptoms of bleeding. In this outcome measure, ABR for all and treated bleeds during prospective data collection period in hemophilia B participants per protocol analysis set was reported.

Outcome measures

Outcome measures
Measure	Standard of Care FIX Replacement Therapy (Hemophilia B) n=107 Participants Participants with moderately severe to severe hemophilia B who administered their own current FIX replacement therapy in the usual healthcare setting were included. No investigational product was administered as a part of this study.	Standard of Care FVIII Replacement Therapy (Hemophilia A) Participants with moderately severe to severe hemophilia A who administered their own current FVIII replacement therapy in the usual healthcare setting were included. No investigational product was administered as a part of this study.
ABR for Treated Bleeds and All Bleeds During Prospective Data Collection Period in Hemophilia B Participants: Per-protocol Analysis Set Treated bleeds	3.71 Bleeds per year Standard Deviation 7.717	—
ABR for Treated Bleeds and All Bleeds During Prospective Data Collection Period in Hemophilia B Participants: Per-protocol Analysis Set All bleeds	4.56 Bleeds per year Standard Deviation 9.557	—

PRIMARY outcome

Timeframe: During prospective data collection period (from baseline visit of the study [Day 1] up to end of study follow-up [maximum follow-up: 1269 days])

Population: Protocol Amendment 5 analysis set: all participants enrolled under Protocol Amendment 5 and afterwards, who fulfilled the inclusion/exclusion criteria and had prospective data collected.

ABR per participant was calculated as number of bleeds over number of days from baseline visit (Day 1 of the study) to end of study\*365.25 days. All bleeds included treated and untreated bleeds. A treated bleed was defined as an event necessitating administration of coagulation factor within 72 hours of signs or symptoms of bleeding. An untreated bleed was defined as an event not necessitating administration of coagulation factor within 72 hours of signs or symptoms of bleeding. In this outcome measure, ABR for all and treated bleeds during prospective data collection period in hemophilia B participants per protocol amendment 5 analysis set was reported.

Outcome measures

Outcome measures
Measure	Standard of Care FIX Replacement Therapy (Hemophilia B) n=39 Participants Participants with moderately severe to severe hemophilia B who administered their own current FIX replacement therapy in the usual healthcare setting were included. No investigational product was administered as a part of this study.	Standard of Care FVIII Replacement Therapy (Hemophilia A) Participants with moderately severe to severe hemophilia A who administered their own current FVIII replacement therapy in the usual healthcare setting were included. No investigational product was administered as a part of this study.
ABR for Treated Bleeds and All Bleeds During Prospective Data Collection Period in Hemophilia B Participants: Protocol Amendment 5 Analysis Set Treated bleeds	4.21 Bleeds per year Standard Deviation 10.609	—
ABR for Treated Bleeds and All Bleeds During Prospective Data Collection Period in Hemophilia B Participants: Protocol Amendment 5 Analysis Set All bleeds	4.78 Bleeds per year Standard Deviation 10.887	—

PRIMARY outcome

Timeframe: During retrospective data collection period (12 months before screening collected in the hemophilia history case report form [CRF])

Population: Protocol Amendment 5 analysis set: all participants enrolled under Protocol Amendment 5 and afterwards, who fulfilled the inclusion/exclusion criteria and had retrospective data collected. Here, "Overall Number of Participants Analyzed" signifies number of participants evaluable for this outcome measure.

ABR per participant was calculated as number of bleeds over number of days from baseline visit (Day 1 of the study) to end of study\*365.25 days. All bleeds included treated and untreated bleeds. A treated bleed was defined as an event necessitating administration of coagulation factor within 72 hours of signs or symptoms of bleeding. An untreated bleed was defined as an event not necessitating administration of coagulation factor within 72 hours of signs or symptoms of bleeding. In this outcome measure, ABR for all and treated bleeds during retrospective data collection period in hemophilia B participants per protocol amendment 5 analysis set was reported.

Outcome measures

Outcome measures
Measure	Standard of Care FIX Replacement Therapy (Hemophilia B) n=38 Participants Participants with moderately severe to severe hemophilia B who administered their own current FIX replacement therapy in the usual healthcare setting were included. No investigational product was administered as a part of this study.	Standard of Care FVIII Replacement Therapy (Hemophilia A) Participants with moderately severe to severe hemophilia A who administered their own current FVIII replacement therapy in the usual healthcare setting were included. No investigational product was administered as a part of this study.
ABR for Treated Bleeds and All Bleeds During Retrospective Data Collection Period in Hemophilia B Participants: Protocol Amendment 5 Analysis Set Treated bleeds	5.42 Bleeds per year Standard Deviation 8.163	—
ABR for Treated Bleeds and All Bleeds During Retrospective Data Collection Period in Hemophilia B Participants: Protocol Amendment 5 Analysis Set All bleeds	6.79 Bleeds per year Standard Deviation 10.390	—

PRIMARY outcome

Timeframe: From start of retrospective data collection period (12 months before screening collected in hemophilia history CRF) up to end of prospective data collection follow-up of period (maximum follow-up:1269 days), for a total of approximately 4.5 years

Population: Protocol Amendment 5 analysis set: all participants enrolled under Protocol Amendment 5 and afterwards, who fulfilled the inclusion/exclusion criteria and had retrospective and prospective data collected. Here, "Overall Number of Participants Analyzed" signifies number of participants evaluable for this outcome measure.

ABR per participant was calculated as number of bleeds over number of days from baseline visit (Day 1 of the study) to end of study\*365.25 days. All bleeds included treated and untreated bleeds. A treated bleed was defined as an event necessitating administration of coagulation factor within 72 hours of signs or symptoms of bleeding. An untreated bleed was defined as an event not necessitating administration of coagulation factor within 72 hours of signs or symptoms of bleeding. In this outcome measure, ABR for all and treated bleeds during retrospective and prospective data collection period in hemophilia B participants per protocol amendment 5 analysis set was reported.

Outcome measures

Outcome measures
Measure	Standard of Care FIX Replacement Therapy (Hemophilia B) n=38 Participants Participants with moderately severe to severe hemophilia B who administered their own current FIX replacement therapy in the usual healthcare setting were included. No investigational product was administered as a part of this study.	Standard of Care FVIII Replacement Therapy (Hemophilia A) Participants with moderately severe to severe hemophilia A who administered their own current FVIII replacement therapy in the usual healthcare setting were included. No investigational product was administered as a part of this study.
ABR for Treated Bleeds and All Bleeds From the Combined Retrospective and Prospective Data Collection Period in Hemophilia B Participants: Protocol Amendment 5 Analysis Set All bleeds	5.48 Bleeds per year Standard Deviation 8.284	—
ABR for Treated Bleeds and All Bleeds From the Combined Retrospective and Prospective Data Collection Period in Hemophilia B Participants: Protocol Amendment 5 Analysis Set Treated bleeds	4.65 Bleeds per year Standard Deviation 7.589	—

PRIMARY outcome

Timeframe: During prospective data collection period (from baseline visit of the study [Day 1] up to end of study follow-up [maximum follow-up: 948 days])

Population: Efficacy analysis set: participants who signed an ICF, had their blood sample collected and assayed for AAV-Spark100 or AAV6 immunity testing, who were subsequently identified as nAb negative (negative for nAb to AAV6 for the hemophilia A cohort), met the inclusion/ exclusion criteria and who participated in the prospective data collection phase as part of their usual healthcare setting.

ABR per participant was calculated as number of bleeds over number of days from baseline visit (Day 1 of the study) to end of study\*365.25 days. All bleeds included treated and untreated bleeds. A treated bleed was defined as an event necessitating administration of coagulation factor within 72 hours of signs or symptoms of bleeding. An untreated bleed was defined as an event not necessitating administration of coagulation factor within 72 hours of signs or symptoms of bleeding. In this outcome measure, ABR for all and treated bleeds during prospective data collection period in hemophilia A participants per efficacy analysis set was reported.

Outcome measures

Outcome measures
Measure	Standard of Care FIX Replacement Therapy (Hemophilia B) n=96 Participants Participants with moderately severe to severe hemophilia B who administered their own current FIX replacement therapy in the usual healthcare setting were included. No investigational product was administered as a part of this study.	Standard of Care FVIII Replacement Therapy (Hemophilia A) Participants with moderately severe to severe hemophilia A who administered their own current FVIII replacement therapy in the usual healthcare setting were included. No investigational product was administered as a part of this study.
ABR for Treated Bleeds and All Bleeds During Prospective Data Collection Period in Hemophilia A Participants: Efficacy Analysis Set Treated bleeds	4.87 Bleeds per year Standard Deviation 7.246	—
ABR for Treated Bleeds and All Bleeds During Prospective Data Collection Period in Hemophilia A Participants: Efficacy Analysis Set All bleeds	6.10 Bleeds per year Standard Deviation 10.578	—

PRIMARY outcome

Timeframe: During prospective data collection period (from baseline visit of the study [Day 1] up to end of study follow-up [maximum follow-up: 948 days])

Population: Per-Protocol analysis set: all participants who signed an ICF, had their blood sample collected and assayed for AAV-Spark100 or AAV6 immunity testing, who were subsequently identified as nAb negative (negative for nAb to AAV6 for the hemophilia A cohort), met the inclusion/ exclusion criteria and who participated and completed at least 6 months of the prospective data collection phase as part of their usual healthcare setting.

ABR per participant was calculated as number of bleeds over number of days from baseline visit (Day 1 of the study) to end of study\*365.25 days. All bleeds included treated and untreated bleeds. A treated bleed was defined as an event necessitating administration of coagulation factor within 72 hours of signs or symptoms of bleeding. An untreated bleed was defined as an event not necessitating administration of coagulation factor within 72 hours of signs or symptoms of bleeding. In this outcome measure, ABR for all and treated bleeds during prospective data collection period in hemophilia A participants per protocol analysis set was reported.

Outcome measures

Outcome measures
Measure	Standard of Care FIX Replacement Therapy (Hemophilia B) n=81 Participants Participants with moderately severe to severe hemophilia B who administered their own current FIX replacement therapy in the usual healthcare setting were included. No investigational product was administered as a part of this study.	Standard of Care FVIII Replacement Therapy (Hemophilia A) Participants with moderately severe to severe hemophilia A who administered their own current FVIII replacement therapy in the usual healthcare setting were included. No investigational product was administered as a part of this study.
ABR for Treated Bleeds and All Bleeds During Prospective Data Collection Period in Hemophilia A Participants: Per-protocol Analysis Set Treated bleeds	3.82 Bleeds per year Standard Deviation 5.665	—
ABR for Treated Bleeds and All Bleeds During Prospective Data Collection Period in Hemophilia A Participants: Per-protocol Analysis Set All bleeds	4.33 Bleeds per year Standard Deviation 6.700	—

PRIMARY outcome

Timeframe: During prospective data collection period (from baseline visit of the study [Day 1] up to end of study follow-up [maximum follow-up: 948 days])

Population: Protocol Amendment 5 analysis set: all participants enrolled under Protocol Amendment 5 and afterwards, who fulfilled the inclusion/exclusion criteria and had prospective data collected.

ABR per participant was calculated as number of bleeds over number of days from baseline visit (Day 1 of the study) to end of study\*365.25 days. All bleeds included treated and untreated bleeds. A treated bleed was defined as an event necessitating administration of coagulation factor within 72 hours of signs or symptoms of bleeding. An untreated bleed was defined as an event not necessitating administration of coagulation factor within 72 hours of signs or symptoms of bleeding. In this outcome measure, ABR for all and treated bleeds during prospective data collection period in hemophilia A participants per protocol amendment 5 analysis set was reported.

Outcome measures

Outcome measures
Measure	Standard of Care FIX Replacement Therapy (Hemophilia B) n=24 Participants Participants with moderately severe to severe hemophilia B who administered their own current FIX replacement therapy in the usual healthcare setting were included. No investigational product was administered as a part of this study.	Standard of Care FVIII Replacement Therapy (Hemophilia A) Participants with moderately severe to severe hemophilia A who administered their own current FVIII replacement therapy in the usual healthcare setting were included. No investigational product was administered as a part of this study.
ABR for Treated Bleeds and All Bleeds During Prospective Data Collection Period in Hemophilia A Participants: Protocol Amendment 5 Analysis Set Treated bleeds	7.62 Bleeds per year Standard Deviation 9.907	—
ABR for Treated Bleeds and All Bleeds During Prospective Data Collection Period in Hemophilia A Participants: Protocol Amendment 5 Analysis Set All bleeds	10.86 Bleeds per year Standard Deviation 16.692	—

PRIMARY outcome

Timeframe: During retrospective data collection period (12 months before screening collected in the hemophilia history CRF)

Population: Protocol Amendment 5 analysis set: all participants enrolled under Protocol Amendment 5 and afterwards, who fulfilled the inclusion/exclusion criteria and had retrospective data collected.

ABR per participant was calculated as number of bleeds over number of days from baseline visit (Day 1 of the study) to end of study\*365.25 days. All bleeds included treated and untreated bleeds. A treated bleed was defined as an event necessitating administration of coagulation factor within 72 hours of signs or symptoms of bleeding. An untreated bleed was defined as an event not necessitating administration of coagulation factor within 72 hours of signs or symptoms of bleeding. In this outcome measure, ABR for all and treated bleeds during retrospective data collection period in hemophilia A participants per protocol amendment 5 analysis set was reported.

Outcome measures

Outcome measures
Measure	Standard of Care FIX Replacement Therapy (Hemophilia B) n=24 Participants Participants with moderately severe to severe hemophilia B who administered their own current FIX replacement therapy in the usual healthcare setting were included. No investigational product was administered as a part of this study.	Standard of Care FVIII Replacement Therapy (Hemophilia A) Participants with moderately severe to severe hemophilia A who administered their own current FVIII replacement therapy in the usual healthcare setting were included. No investigational product was administered as a part of this study.
ABR for Treated Bleeds and All Bleeds During Retrospective Data Collection Period in Hemophilia A Participants: Protocol Amendment 5 Analysis Set Treated bleeds	9.83 Bleeds per year Standard Deviation 13.656	—
ABR for Treated Bleeds and All Bleeds During Retrospective Data Collection Period in Hemophilia A Participants: Protocol Amendment 5 Analysis Set All bleeds	14.29 Bleeds per year Standard Deviation 18.208	—

PRIMARY outcome

Timeframe: From start of retrospective data collection period (12 months before screening collected in hemophilia history CRF) up to end of prospective data collection follow-up of period (maximum follow-up:948 days), for a total of approximately 3.6 years

Population: Protocol Amendment 5 analysis set: all participants enrolled under Protocol Amendment 5 and afterwards, who fulfilled the inclusion/exclusion criteria and had retrospective and prospective data collected.

ABR per participant was calculated as number of bleeds over number of days from baseline visit (Day 1 of the study) to end of study\*365.25 days. All bleeds included treated and untreated bleeds. A treated bleed was defined as an event necessitating administration of coagulation factor within 72 hours of signs or symptoms of bleeding. An untreated bleed was defined as an event not necessitating administration of coagulation factor within 72 hours of signs or symptoms of bleeding. In this outcome measure, ABR for all and treated bleeds during retrospective and prospective data collection period in hemophilia A participants per protocol amendment 5 analysis set was reported.

Outcome measures

Outcome measures
Measure	Standard of Care FIX Replacement Therapy (Hemophilia B) n=24 Participants Participants with moderately severe to severe hemophilia B who administered their own current FIX replacement therapy in the usual healthcare setting were included. No investigational product was administered as a part of this study.	Standard of Care FVIII Replacement Therapy (Hemophilia A) Participants with moderately severe to severe hemophilia A who administered their own current FVIII replacement therapy in the usual healthcare setting were included. No investigational product was administered as a part of this study.
ABR for Treated Bleeds and All Bleeds From the Combined Retrospective and Prospective Data Collection Period in Hemophilia A Participants: Protocol Amendment 5 Analysis Set Treated bleeds	9.01 Bleeds per year Standard Deviation 11.908	—
ABR for Treated Bleeds and All Bleeds From the Combined Retrospective and Prospective Data Collection Period in Hemophilia A Participants: Protocol Amendment 5 Analysis Set All bleeds	12.77 Bleeds per year Standard Deviation 16.290	—

SECONDARY outcome

Timeframe: During prospective data collection period (from baseline visit of the study [Day 1] up to end of study follow-up [maximum follow-up: 1269 days])

Population: Efficacy analysis set: participants who signed an ICF, had their blood sample collected and assayed for AAV-Spark100 or AAV6 immunity testing, who were subsequently identified as nAb negative (negative for nAb to AAV-Spark100 for the hemophilia B cohort), met the inclusion/ exclusion criteria and who participated in the prospective data collection phase as part of their usual healthcare setting.

AIR per participant was calculated as the number of infusions received over number of days from baseline visit (Day 1) to end of study \* 365.25 days.

Outcome measures

Outcome measures
Measure	Standard of Care FIX Replacement Therapy (Hemophilia B) n=110 Participants Participants with moderately severe to severe hemophilia B who administered their own current FIX replacement therapy in the usual healthcare setting were included. No investigational product was administered as a part of this study.	Standard of Care FVIII Replacement Therapy (Hemophilia A) Participants with moderately severe to severe hemophilia A who administered their own current FVIII replacement therapy in the usual healthcare setting were included. No investigational product was administered as a part of this study.
Annualized Infusion Rate (AIR) During Prospective Data Collection Period in Hemophilia B Participants: Efficacy Analysis Set	62.81 Infusions per year Standard Deviation 33.250	—

SECONDARY outcome

Timeframe: During prospective data collection period (from baseline visit of the study [Day 1] up to end of study follow-up [maximum follow-up: 1269 days])

Population: Per-Protocol analysis set: all participants who signed an ICF, had their blood sample collected and assayed for AAV-Spark100 or AAV6 immunity testing, who were subsequently identified as nAb negative (negative for nAb to AAV-Spark100 for the hemophilia B cohort), met the inclusion/ exclusion criteria and who participated and completed at least 6 months of the prospective data collection phase as part of their usual healthcare setting.

AIR per participant was calculated as the number of infusions received over number of days from baseline visit (Day 1) to end of study \* 365.25 days.

Outcome measures

Outcome measures
Measure	Standard of Care FIX Replacement Therapy (Hemophilia B) n=107 Participants Participants with moderately severe to severe hemophilia B who administered their own current FIX replacement therapy in the usual healthcare setting were included. No investigational product was administered as a part of this study.	Standard of Care FVIII Replacement Therapy (Hemophilia A) Participants with moderately severe to severe hemophilia A who administered their own current FVIII replacement therapy in the usual healthcare setting were included. No investigational product was administered as a part of this study.
AIR During Prospective Data Collection Period in Hemophilia B Participants: Per-protocol Analysis Set	62.82 Infusions per year Standard Deviation 33.213	—

SECONDARY outcome

Timeframe: During prospective data collection period (from baseline visit of the study [Day 1] up to end of study follow-up [maximum follow-up: 1269 days])

Population: Protocol Amendment 5 analysis set: all participants enrolled under Protocol Amendment 5 and afterwards, who fulfilled the inclusion/exclusion criteria and had prospective data collected.

AIR per participant was calculated as the number of infusions received over number of days from baseline visit (Day 1) to end of study \* 365.25 days.

Outcome measures

Outcome measures
Measure	Standard of Care FIX Replacement Therapy (Hemophilia B) n=39 Participants Participants with moderately severe to severe hemophilia B who administered their own current FIX replacement therapy in the usual healthcare setting were included. No investigational product was administered as a part of this study.	Standard of Care FVIII Replacement Therapy (Hemophilia A) Participants with moderately severe to severe hemophilia A who administered their own current FVIII replacement therapy in the usual healthcare setting were included. No investigational product was administered as a part of this study.
AIR During Prospective Data Collection Period in Hemophilia B Participants: Protocol Amendment 5 Analysis Set	56.97 Infusions per year Standard Deviation 20.987	—

SECONDARY outcome

Timeframe: During retrospective data collection period (12 months before screening collected in the hemophilia history CRF)

Population: Protocol Amendment 5 analysis set: all participants enrolled under Protocol Amendment 5 and afterwards, who fulfilled the inclusion/exclusion criteria and had retrospective data collected.

AIR per participant was calculated as the number of infusions received over number of days from baseline visit (Day 1) to end of study \* 365.25 days.

Outcome measures

Outcome measures
Measure	Standard of Care FIX Replacement Therapy (Hemophilia B) n=39 Participants Participants with moderately severe to severe hemophilia B who administered their own current FIX replacement therapy in the usual healthcare setting were included. No investigational product was administered as a part of this study.	Standard of Care FVIII Replacement Therapy (Hemophilia A) Participants with moderately severe to severe hemophilia A who administered their own current FVIII replacement therapy in the usual healthcare setting were included. No investigational product was administered as a part of this study.
AIR During Retrospective Data Collection Period in Hemophilia B Participants: Protocol Amendment 5 Analysis Set	66.97 Infusions per year Standard Deviation 30.535	—

SECONDARY outcome

Timeframe: From start of retrospective data collection period (12 months before screening collected in hemophilia history CRF) up to end of prospective data collection follow-up of period (maximum follow-up:1269 days), for a total of approximately 4.5 years

Population: Protocol Amendment 5 analysis set: all participants enrolled under Protocol Amendment 5 and afterwards, who fulfilled the inclusion/exclusion criteria and had retrospective and prospective data collected.

AIR combining retrospective and prospective data was calculated as (number of infusions from baseline visit (Day 1) to end of study + number of infusions collected in the Hemophilia History form) / (number of days from baseline visit (Day 1) to end of study + 365.25) / 365.25.

Outcome measures

Outcome measures
Measure	Standard of Care FIX Replacement Therapy (Hemophilia B) n=39 Participants Participants with moderately severe to severe hemophilia B who administered their own current FIX replacement therapy in the usual healthcare setting were included. No investigational product was administered as a part of this study.	Standard of Care FVIII Replacement Therapy (Hemophilia A) Participants with moderately severe to severe hemophilia A who administered their own current FVIII replacement therapy in the usual healthcare setting were included. No investigational product was administered as a part of this study.
AIR From the Combined Retrospective and Prospective Data Collection Period in Hemophilia B Participants: Protocol Amendment 5 Analysis Set	60.98 Infusions per year Standard Deviation 23.130	—

SECONDARY outcome

Timeframe: During prospective data collection period (from baseline visit of the study [Day 1] up to end of study follow-up [maximum follow-up: 948 days])

Population: Efficacy analysis set: participants who signed an ICF, had their blood sample collected and assayed for AAV-Spark100 or AAV6 immunity testing, who were subsequently identified as nAb negative (negative for nAb to AAV6 for the hemophilia A cohort), met the inclusion/ exclusion criteria and who participated in the prospective data collection phase as part of their usual healthcare setting.

AIR per participant was calculated as the number of infusions received over number of days from baseline visit (Day 1) to end of study \* 365.25 days.

Outcome measures

Outcome measures
Measure	Standard of Care FIX Replacement Therapy (Hemophilia B) n=96 Participants Participants with moderately severe to severe hemophilia B who administered their own current FIX replacement therapy in the usual healthcare setting were included. No investigational product was administered as a part of this study.	Standard of Care FVIII Replacement Therapy (Hemophilia A) Participants with moderately severe to severe hemophilia A who administered their own current FVIII replacement therapy in the usual healthcare setting were included. No investigational product was administered as a part of this study.
AIR During Prospective Data Collection Period in Hemophilia A Participants: Efficacy Analysis Set	127.07 Infusions per year Standard Deviation 51.784	—

SECONDARY outcome

Timeframe: During prospective data collection period (from baseline visit of the study [Day 1] up to end of study follow-up [maximum follow-up: 948 days])

Population: Per-Protocol analysis set: all participants who signed an ICF, had their blood sample collected and assayed for AAV-Spark100 or AAV6 immunity testing, who were subsequently identified as nAb negative (negative for nAb to AAV6 for the hemophilia A cohort), met the inclusion/ exclusion criteria and who participated and completed at least 6 months of the prospective data collection phase as part of their usual healthcare setting.

AIR per participant was calculated as the number of infusions received over number of days from baseline visit (Day 1) to end of study \* 365.25 days.

Outcome measures

Outcome measures
Measure	Standard of Care FIX Replacement Therapy (Hemophilia B) n=81 Participants Participants with moderately severe to severe hemophilia B who administered their own current FIX replacement therapy in the usual healthcare setting were included. No investigational product was administered as a part of this study.	Standard of Care FVIII Replacement Therapy (Hemophilia A) Participants with moderately severe to severe hemophilia A who administered their own current FVIII replacement therapy in the usual healthcare setting were included. No investigational product was administered as a part of this study.
AIR During Prospective Data Collection Period in Hemophilia A Participants: Per-protocol Analysis Set	127.12 Infusions per year Standard Deviation 55.418	—

SECONDARY outcome

Timeframe: During prospective data collection period (from baseline visit of the study [Day 1] up to end of study follow-up [maximum follow-up: 948 days])

Population: Protocol Amendment 5 analysis set: all participants enrolled under Protocol Amendment 5 and afterwards, who fulfilled the inclusion/exclusion criteria and had prospective data collected.

AIR per participant was calculated as the number of infusions received over number of days from baseline visit (Day 1) to end of study \* 365.25 days.

Outcome measures

Outcome measures
Measure	Standard of Care FIX Replacement Therapy (Hemophilia B) n=24 Participants Participants with moderately severe to severe hemophilia B who administered their own current FIX replacement therapy in the usual healthcare setting were included. No investigational product was administered as a part of this study.	Standard of Care FVIII Replacement Therapy (Hemophilia A) Participants with moderately severe to severe hemophilia A who administered their own current FVIII replacement therapy in the usual healthcare setting were included. No investigational product was administered as a part of this study.
AIR During Prospective Data Collection Period in Hemophilia A Participants: Protocol Amendment 5 Analysis Set	133.19 Infusions per year Standard Deviation 56.725	—

SECONDARY outcome

Timeframe: During retrospective data collection period (12 months before screening collected in the hemophilia history CRF)

Population: Protocol Amendment 5 analysis set: all participants enrolled under Protocol Amendment 5 and afterwards, who fulfilled the inclusion/exclusion criteria and had retrospective data collected.

AIR per participant was calculated as the number of infusions received over number of days from baseline visit (Day 1) to end of study \* 365.25 days.

Outcome measures

Outcome measures
Measure	Standard of Care FIX Replacement Therapy (Hemophilia B) n=24 Participants Participants with moderately severe to severe hemophilia B who administered their own current FIX replacement therapy in the usual healthcare setting were included. No investigational product was administered as a part of this study.	Standard of Care FVIII Replacement Therapy (Hemophilia A) Participants with moderately severe to severe hemophilia A who administered their own current FVIII replacement therapy in the usual healthcare setting were included. No investigational product was administered as a part of this study.
AIR During Retrospective Data Collection Period in Hemophilia A Participants: Protocol Amendment 5 Analysis Set	141.88 Infusions per year Standard Deviation 50.487	—

SECONDARY outcome

Timeframe: From start of retrospective data collection period (12 months before screening collected in hemophilia history CRF) up to end of prospective data collection follow-up of period (maximum follow-up:948 days), for a total of approximately 3.6 years

Population: Protocol Amendment 5 analysis set: all participants enrolled under Protocol Amendment 5 and afterwards, who fulfilled the inclusion/exclusion criteria and had retrospective and prospective data collected.

AIR combining retrospective and prospective data was calculated as (number of infusions from baseline visit (Day 1) to end of study + number of infusions collected in the Hemophilia History form) / (number of days from baseline visit (Day 1) to end of study + 365.25) / 365.25.

Outcome measures

Outcome measures
Measure	Standard of Care FIX Replacement Therapy (Hemophilia B) n=24 Participants Participants with moderately severe to severe hemophilia B who administered their own current FIX replacement therapy in the usual healthcare setting were included. No investigational product was administered as a part of this study.	Standard of Care FVIII Replacement Therapy (Hemophilia A) Participants with moderately severe to severe hemophilia A who administered their own current FVIII replacement therapy in the usual healthcare setting were included. No investigational product was administered as a part of this study.
AIR From the Combined Retrospective and Prospective Data Collection Period in Hemophilia A Participants: Protocol Amendment 5 Analysis Set	139.88 Infusions per year Standard Deviation 52.490	—

SECONDARY outcome

Timeframe: During prospective data collection period (from baseline visit of the study [Day 1] up to end of study follow-up [maximum follow-up: 1269 days])

Population: Efficacy analysis set: participants who signed an ICF, had their blood sample collected and assayed for AAV-Spark100 or AAV6 immunity testing, who were subsequently identified as nAb negative (negative for nAb to AAV-Spark100 for the hemophilia B cohort), met the inclusion/ exclusion criteria and who participated in the prospective data collection phase as part of their usual healthcare setting.

Annualized factor consumption was calculated as the total factor replacement therapy consumption (in international unit \[IU\] and dose) \*365.25 days/number of days during the observation time period while the participant received factor prophylaxis replacement therapy in the usual care setting from baseline visit (Day 1) to end of study.

Outcome measures

Outcome measures
Measure	Standard of Care FIX Replacement Therapy (Hemophilia B) n=110 Participants Participants with moderately severe to severe hemophilia B who administered their own current FIX replacement therapy in the usual healthcare setting were included. No investigational product was administered as a part of this study.	Standard of Care FVIII Replacement Therapy (Hemophilia A) Participants with moderately severe to severe hemophilia A who administered their own current FVIII replacement therapy in the usual healthcare setting were included. No investigational product was administered as a part of this study.
Annualized Total Factor IX Replacement Therapy Consumption During Prospective Data Collection Period in Hemophilia B Participants: Efficacy Analysis Set	238312 International units per year Standard Deviation 127817	—

SECONDARY outcome

Timeframe: During prospective data collection period (from baseline visit of the study [Day 1] up to end of study follow-up [maximum follow-up: 1269 days])

Population: Per-Protocol analysis set: all participants who signed an ICF, had their blood sample collected and assayed for AAV-Spark100 or AAV6 immunity testing, who were subsequently identified as nAb negative (negative for nAb to AAV-Spark100 for the hemophilia B cohort), met the inclusion/ exclusion criteria and who participated and completed at least 6 months of the prospective data collection phase as part of their usual healthcare setting.

Annualized factor consumption was calculated as the total factor replacement therapy consumption (in IU and dose) \*365.25 days/number of days during the observation time period while the participant received factor prophylaxis replacement therapy in the usual care setting from baseline visit (Day 1) to end of study.

Outcome measures

Outcome measures
Measure	Standard of Care FIX Replacement Therapy (Hemophilia B) n=107 Participants Participants with moderately severe to severe hemophilia B who administered their own current FIX replacement therapy in the usual healthcare setting were included. No investigational product was administered as a part of this study.	Standard of Care FVIII Replacement Therapy (Hemophilia A) Participants with moderately severe to severe hemophilia A who administered their own current FVIII replacement therapy in the usual healthcare setting were included. No investigational product was administered as a part of this study.
Annualized Total Factor IX Replacement Therapy Consumption During Prospective Data Collection Period in Hemophilia B Participants: Per-protocol Analysis Set	240501 International units per year Standard Deviation 128676	—

SECONDARY outcome

Timeframe: During prospective data collection period (from baseline visit of the study [Day 1] up to end of study follow-up [maximum follow-up: 1269 days])

Population: Protocol Amendment 5 analysis set: all participants enrolled under Protocol Amendment 5 and afterwards, who fulfilled the inclusion/exclusion criteria and had prospective data collected.

Annualized factor consumption was calculated as the total factor replacement therapy consumption (in IU and dose) \*365.25 days/number of days during the observation time period while the participant received factor prophylaxis replacement therapy in the usual care setting from baseline visit (Day 1) to end of study.

Outcome measures

Outcome measures
Measure	Standard of Care FIX Replacement Therapy (Hemophilia B) n=39 Participants Participants with moderately severe to severe hemophilia B who administered their own current FIX replacement therapy in the usual healthcare setting were included. No investigational product was administered as a part of this study.	Standard of Care FVIII Replacement Therapy (Hemophilia A) Participants with moderately severe to severe hemophilia A who administered their own current FVIII replacement therapy in the usual healthcare setting were included. No investigational product was administered as a part of this study.
Annualized Total Factor IX Replacement Therapy Consumption During Prospective Data Collection Period in Hemophilia B Participants: Protocol Amendment 5 Analysis Set	223408 International units per year Standard Deviation 85995	—

SECONDARY outcome

Timeframe: During prospective data collection period (from baseline visit of the study [Day 1] up to end of study follow-up [maximum follow-up: 948 days])

Population: Efficacy analysis set: participants who signed an ICF, had their blood sample collected and assayed for AAV-Spark100 or AAV6 immunity testing, who were subsequently identified as nAb negative (negative for nAb to AAV6 for the hemophilia A cohort), met the inclusion/ exclusion criteria and who participated in the prospective data collection phase as part of their usual healthcare setting.

Annualized factor consumption was calculated as the total factor replacement therapy consumption (in IU and dose) \*365.25 days/number of days during the observation time period while the participant received factor prophylaxis replacement therapy in the usual care setting from baseline visit (Day 1) to end of study.

Outcome measures

Outcome measures
Measure	Standard of Care FIX Replacement Therapy (Hemophilia B) n=96 Participants Participants with moderately severe to severe hemophilia B who administered their own current FIX replacement therapy in the usual healthcare setting were included. No investigational product was administered as a part of this study.	Standard of Care FVIII Replacement Therapy (Hemophilia A) Participants with moderately severe to severe hemophilia A who administered their own current FVIII replacement therapy in the usual healthcare setting were included. No investigational product was administered as a part of this study.
Annualized Total Factor IX Replacement Therapy Consumption During Prospective Data Collection Period in Hemophilia A Participants: Efficacy Analysis Set	304998 International units per year Standard Deviation 153932	—

SECONDARY outcome

Timeframe: During prospective data collection period (from baseline visit of the study [Day 1] up to end of study follow-up [maximum follow-up: 948 days])

Population: Per-Protocol analysis set: all participants who signed an ICF, had their blood sample collected and assayed for AAV-Spark100 or AAV6 immunity testing, who were subsequently identified as nAb negative (negative for nAb to AAV6 for the hemophilia A cohort), met the inclusion/ exclusion criteria and who participated and completed at least 6 months of the prospective data collection phase as part of their usual healthcare setting.

Annualized factor consumption was calculated as the total factor replacement therapy consumption (in IU and dose) \*365.25 days/number of days during the observation time period while the participant received factor prophylaxis replacement therapy in the usual care setting from baseline visit (Day 1) to end of study.

Outcome measures

Outcome measures
Measure	Standard of Care FIX Replacement Therapy (Hemophilia B) n=81 Participants Participants with moderately severe to severe hemophilia B who administered their own current FIX replacement therapy in the usual healthcare setting were included. No investigational product was administered as a part of this study.	Standard of Care FVIII Replacement Therapy (Hemophilia A) Participants with moderately severe to severe hemophilia A who administered their own current FVIII replacement therapy in the usual healthcare setting were included. No investigational product was administered as a part of this study.
Annualized Total Factor IX Replacement Therapy Consumption During Prospective Data Collection Period in Hemophilia A Participants: Per-protocol Analysis Set	314195 International units per year Standard Deviation 163786	—

SECONDARY outcome

Timeframe: During prospective data collection period (from baseline visit of the study [Day 1] up to end of study follow-up [maximum follow-up: 948 days])

Population: Protocol Amendment 5 analysis set: all participants enrolled under Protocol Amendment 5 and afterwards, who fulfilled the inclusion/exclusion criteria and had prospective data collected.

Annualized factor consumption was calculated as the total factor replacement therapy consumption (in IU and dose) \*365.25 days/number of days during the observation time period while the participant received factor prophylaxis replacement therapy in the usual care setting from baseline visit (Day 1) to end of study.

Outcome measures

Outcome measures
Measure	Standard of Care FIX Replacement Therapy (Hemophilia B) n=24 Participants Participants with moderately severe to severe hemophilia B who administered their own current FIX replacement therapy in the usual healthcare setting were included. No investigational product was administered as a part of this study.	Standard of Care FVIII Replacement Therapy (Hemophilia A) Participants with moderately severe to severe hemophilia A who administered their own current FVIII replacement therapy in the usual healthcare setting were included. No investigational product was administered as a part of this study.
Annualized Total Factor IX Replacement Therapy Consumption During Prospective Data Collection Period in Hemophilia A Participants: Protocol Amendment 5 Analysis Set	235097 International units per year Standard Deviation 67977	—

SECONDARY outcome

Timeframe: During prospective data collection period (from baseline visit of the study [Day 1] up to end of study follow-up [maximum follow-up: 1269 days])

Population: Efficacy analysis set: participants who signed an ICF, had their blood sample collected and assayed for AAV-Spark100 or AAV6 immunity testing, who were subsequently identified as nAb negative (negative for nAb to AAV-Spark100 for the hemophilia B cohort), met the inclusion/ exclusion criteria and who participated in the prospective data collection phase as part of their usual healthcare setting.

ABR per participant was calculated as number of bleeds over number of days from baseline visit (Day 1 of the study) to end of study\*365.25 days. Spontaneous bleeds were defined as bleeding for no apparent or known reason particularly into the joints, muscles, and soft tissues. All bleeds included treated and untreated bleeds. A treated bleed was defined as an event necessitating administration of coagulation factor within 72 hours of signs or symptoms of bleeding. An untreated bleed was defined as an event not necessitating administration of coagulation factor within 72 hours of signs or symptoms of bleeding. In this outcome measure, ABR for spontaneous all and treated bleeds during prospective data collection period in hemophilia B participants per efficacy analysis set was reported.

Outcome measures

Outcome measures
Measure	Standard of Care FIX Replacement Therapy (Hemophilia B) n=110 Participants Participants with moderately severe to severe hemophilia B who administered their own current FIX replacement therapy in the usual healthcare setting were included. No investigational product was administered as a part of this study.	Standard of Care FVIII Replacement Therapy (Hemophilia A) Participants with moderately severe to severe hemophilia A who administered their own current FVIII replacement therapy in the usual healthcare setting were included. No investigational product was administered as a part of this study.
ABR for Spontaneous Treated Bleeds and All Bleeds During Prospective Data Collection Period in Hemophilia B Participants: Efficacy Analysis Set Treated bleeds	2.11 Bleeds per year Standard Deviation 3.835	—
ABR for Spontaneous Treated Bleeds and All Bleeds During Prospective Data Collection Period in Hemophilia B Participants: Efficacy Analysis Set All bleeds	2.79 Bleeds per year Standard Deviation 6.411	—

SECONDARY outcome

Timeframe: During prospective data collection period (from baseline visit of the study [Day 1] up to end of study follow-up [maximum follow-up: 1269 days])

Population: Efficacy analysis set: participants who signed an ICF, had their blood sample collected and assayed for AAV-Spark100 or AAV6 immunity testing, who were subsequently identified as nAb negative (negative for nAb to AAV-Spark100 for the hemophilia B cohort), met the inclusion/ exclusion criteria and who participated in the prospective data collection phase as part of their usual healthcare setting.

ABR per participant was calculated as number of bleeds over number of days from baseline visit (Day 1 of the study) to end of study\*365.25 days. Traumatic bleeds were defined as bleeding event occurring for an apparent or known reason. All bleeds included treated and untreated bleeds. A treated bleed was defined as an event necessitating administration of coagulation factor within 72 hours of signs or symptoms of bleeding. An untreated bleed was defined as an event not necessitating administration of coagulation factor within 72 hours of signs or symptoms of bleeding. In this outcome measure, ABR for traumatic all and treated bleeds during prospective data collection period in hemophilia B participants per efficacy analysis set was reported.

Outcome measures

Outcome measures
Measure	Standard of Care FIX Replacement Therapy (Hemophilia B) n=110 Participants Participants with moderately severe to severe hemophilia B who administered their own current FIX replacement therapy in the usual healthcare setting were included. No investigational product was administered as a part of this study.	Standard of Care FVIII Replacement Therapy (Hemophilia A) Participants with moderately severe to severe hemophilia A who administered their own current FVIII replacement therapy in the usual healthcare setting were included. No investigational product was administered as a part of this study.
ABR for Traumatic Treated Bleeds and All Bleeds During Prospective Data Collection Period in Hemophilia B Participants: Efficacy Analysis Set Treated bleeds	1.50 Bleeds per year Standard Deviation 6.380	—
ABR for Traumatic Treated Bleeds and All Bleeds During Prospective Data Collection Period in Hemophilia B Participants: Efficacy Analysis Set All bleeds	1.68 Bleeds per year Standard Deviation 6.623	—

SECONDARY outcome

Timeframe: During prospective data collection period (from baseline visit of the study [Day 1] up to end of study follow-up [maximum follow-up: 1269 days])

Population: Per-Protocol analysis set: all participants who signed an ICF, had their blood sample collected and assayed for AAV-Spark100 or AAV6 immunity testing, who were subsequently identified as nAb negative (negative for nAb to AAV-Spark100 for the hemophilia B cohort), met the inclusion/ exclusion criteria and who participated and completed at least 6 months of the prospective data collection phase as part of their usual healthcare setting.

ABR per participant was calculated as number of bleeds over number of days from baseline visit (Day 1 of the study) to end of study\*365.25 days. Spontaneous bleeds were defined as bleeding for no apparent or known reason particularly into the joints, muscles, and soft tissues. All bleeds included treated and untreated bleeds. A treated bleed was defined as an event necessitating administration of coagulation factor within 72 hours of signs or symptoms of bleeding. An untreated bleed was defined as an event not necessitating administration of coagulation factor within 72 hours of signs or symptoms of bleeding. In this outcome measure, ABR for spontaneous all and treated bleeds during prospective data collection period in hemophilia B participants per protocol analysis set was reported.

Outcome measures

Outcome measures
Measure	Standard of Care FIX Replacement Therapy (Hemophilia B) n=107 Participants Participants with moderately severe to severe hemophilia B who administered their own current FIX replacement therapy in the usual healthcare setting were included. No investigational product was administered as a part of this study.	Standard of Care FVIII Replacement Therapy (Hemophilia A) Participants with moderately severe to severe hemophilia A who administered their own current FVIII replacement therapy in the usual healthcare setting were included. No investigational product was administered as a part of this study.
ABR for Spontaneous Treated Bleeds and All Bleeds During Prospective Data Collection Period in Hemophilia B Participants: Per-protocol Analysis Set Treated bleeds	2.17 Bleeds per year Standard Deviation 3.872	—
ABR for Spontaneous Treated Bleeds and All Bleeds During Prospective Data Collection Period in Hemophilia B Participants: Per-protocol Analysis Set All bleeds	2.84 Bleeds per year Standard Deviation 6.490	—

SECONDARY outcome

Timeframe: During prospective data collection period (from baseline visit of the study [Day 1] up to end of study follow-up [maximum follow-up: 1269 days])

Population: Per-Protocol analysis set: all participants who signed an ICF, had their blood sample collected and assayed for AAV-Spark100 or AAV6 immunity testing, who were subsequently identified as nAb negative (negative for nAb to AAV-Spark100 for the hemophilia B cohort), met the inclusion/ exclusion criteria and who participated and completed at least 6 months of the prospective data collection phase as part of their usual healthcare setting.

ABR per participant was calculated as number of bleeds over number of days from baseline visit (Day 1 of the study) to end of study\*365.25 days. Traumatic bleeds were defined as bleeding event occurring for an apparent or known reason. All bleeds included treated and untreated bleeds. A treated bleed was defined as an event necessitating administration of coagulation factor within 72 hours of signs or symptoms of bleeding. An untreated bleed was defined as an event not necessitating administration of coagulation factor within 72 hours of signs or symptoms of bleeding. In this outcome measure, ABR for traumatic all and treated bleeds during prospective data collection period in hemophilia B participants per protocol analysis set was reported.

Outcome measures

Outcome measures
Measure	Standard of Care FIX Replacement Therapy (Hemophilia B) n=107 Participants Participants with moderately severe to severe hemophilia B who administered their own current FIX replacement therapy in the usual healthcare setting were included. No investigational product was administered as a part of this study.	Standard of Care FVIII Replacement Therapy (Hemophilia A) Participants with moderately severe to severe hemophilia A who administered their own current FVIII replacement therapy in the usual healthcare setting were included. No investigational product was administered as a part of this study.
ABR for Traumatic Treated Bleeds and All Bleeds During Prospective Data Collection Period in Hemophilia B Participants: Per-protocol Analysis Set Treated bleeds	1.54 Bleeds per year Standard Deviation 6.465	—
ABR for Traumatic Treated Bleeds and All Bleeds During Prospective Data Collection Period in Hemophilia B Participants: Per-protocol Analysis Set All bleeds	1.72 Bleeds per year Standard Deviation 6.710	—

SECONDARY outcome

Timeframe: During prospective data collection period (from baseline visit of the study [Day 1] up to end of study follow-up [maximum follow-up: 948 days])

Population: Efficacy analysis set: participants who signed an ICF, had their blood sample collected and assayed for AAV-Spark100 or AAV6 immunity testing, who were subsequently identified as nAb negative (negative for nAb to AAV6 for the hemophilia A cohort), met the inclusion/ exclusion criteria and who participated in the prospective data collection phase as part of their usual healthcare setting.

ABR per participant was calculated as number of bleeds over number of days from baseline visit (Day 1 of the study) to end of study\*365.25 days. Spontaneous bleeds were defined as bleeding for no apparent or known reason particularly into the joints, muscles, and soft tissues. All bleeds included treated and untreated bleeds. A treated bleed was defined as an event necessitating administration of coagulation factor within 72 hours of signs or symptoms of bleeding. An untreated bleed was defined as an event not necessitating administration of coagulation factor within 72 hours of signs or symptoms of bleeding. In this outcome measure, ABR for spontaneous all and treated bleeds during prospective data collection period in hemophilia A participants per efficacy analysis set was reported.

Outcome measures

Outcome measures
Measure	Standard of Care FIX Replacement Therapy (Hemophilia B) n=96 Participants Participants with moderately severe to severe hemophilia B who administered their own current FIX replacement therapy in the usual healthcare setting were included. No investigational product was administered as a part of this study.	Standard of Care FVIII Replacement Therapy (Hemophilia A) Participants with moderately severe to severe hemophilia A who administered their own current FVIII replacement therapy in the usual healthcare setting were included. No investigational product was administered as a part of this study.
ABR for Spontaneous Treated Bleeds and All Bleeds During Prospective Data Collection Period in Hemophilia A Participants: Efficacy Analysis Set Treated bleeds	3.15 Bleeds per year Standard Deviation 5.948	—
ABR for Spontaneous Treated Bleeds and All Bleeds During Prospective Data Collection Period in Hemophilia A Participants: Efficacy Analysis Set All bleeds	3.89 Bleeds per year Standard Deviation 7.552	—

SECONDARY outcome

Timeframe: During prospective data collection period (from baseline visit of the study [Day 1] up to end of study follow-up [maximum follow-up: 948 days])

Population: Efficacy analysis set: participants who signed an ICF, had their blood sample collected and assayed for AAV-Spark100 or AAV6 immunity testing, who were subsequently identified as nAb negative (negative for nAb to AAV6 for the hemophilia A cohort), met the inclusion/ exclusion criteria and who participated in the prospective data collection phase as part of their usual healthcare setting.

ABR per participant was calculated as number of bleeds over number of days from baseline visit (Day 1 of the study) to end of study\*365.25 days. Traumatic bleeds were defined as bleeding event occurring for an apparent or known reason. All bleeds included treated and untreated bleeds. A treated bleed was defined as an event necessitating administration of coagulation factor within 72 hours of signs or symptoms of bleeding. An untreated bleed was defined as an event not necessitating administration of coagulation factor within 72 hours of signs or symptoms of bleeding. In this outcome measure, ABR for traumatic all and treated bleeds during prospective data collection period in hemophilia A participants per efficacy analysis set was reported.

Outcome measures

Outcome measures
Measure	Standard of Care FIX Replacement Therapy (Hemophilia B) n=96 Participants Participants with moderately severe to severe hemophilia B who administered their own current FIX replacement therapy in the usual healthcare setting were included. No investigational product was administered as a part of this study.	Standard of Care FVIII Replacement Therapy (Hemophilia A) Participants with moderately severe to severe hemophilia A who administered their own current FVIII replacement therapy in the usual healthcare setting were included. No investigational product was administered as a part of this study.
ABR for Traumatic Treated Bleeds and All Bleeds During Prospective Data Collection Period in Hemophilia A Participants: Efficacy Analysis Set Treated bleeds	1.78 Bleeds per year Standard Deviation 3.893	—
ABR for Traumatic Treated Bleeds and All Bleeds During Prospective Data Collection Period in Hemophilia A Participants: Efficacy Analysis Set All bleeds	2.27 Bleeds per year Standard Deviation 5.503	—

SECONDARY outcome

Timeframe: During prospective data collection period (from baseline visit of the study [Day 1] up to end of study follow-up [maximum follow-up: 948 days])

Population: Per-Protocol analysis set: all participants who signed an ICF, had their blood sample collected and assayed for AAV-Spark100 or AAV6 immunity testing, who were subsequently identified as nAb negative (negative for nAb to AAV6 for the hemophilia A cohort), met the inclusion/ exclusion criteria and who participated and completed at least 6 months of the prospective data collection phase as part of their usual healthcare setting.

ABR per participant was calculated as number of bleeds over number of days from baseline visit (Day 1 of the study) to end of study\*365.25 days. Spontaneous bleeds were defined as bleeding for no apparent or known reason particularly into the joints, muscles, and soft tissues. All bleeds included treated and untreated bleeds. A treated bleed was defined as an event necessitating administration of coagulation factor within 72 hours of signs or symptoms of bleeding. An untreated bleed was defined as an event not necessitating administration of coagulation factor within 72 hours of signs or symptoms of bleeding. In this outcome measure, ABR for spontaneous all and treated bleeds during prospective data collection period in hemophilia A participants per protocol analysis set was reported.

Outcome measures

Outcome measures
Measure	Standard of Care FIX Replacement Therapy (Hemophilia B) n=81 Participants Participants with moderately severe to severe hemophilia B who administered their own current FIX replacement therapy in the usual healthcare setting were included. No investigational product was administered as a part of this study.	Standard of Care FVIII Replacement Therapy (Hemophilia A) Participants with moderately severe to severe hemophilia A who administered their own current FVIII replacement therapy in the usual healthcare setting were included. No investigational product was administered as a part of this study.
ABR for Spontaneous Treated Bleeds and All Bleeds During Prospective Data Collection Period in Hemophilia A Participants: Per-protocol Analysis Set Treated bleeds	2.37 Bleeds per year Standard Deviation 4.101	—
ABR for Spontaneous Treated Bleeds and All Bleeds During Prospective Data Collection Period in Hemophilia A Participants: Per-protocol Analysis Set All bleeds	2.73 Bleeds per year Standard Deviation 5.143	—

SECONDARY outcome

Timeframe: During prospective data collection period (from baseline visit of the study [Day 1] up to end of study follow-up [maximum follow-up: 948 days])

Population: Per-Protocol analysis set: all participants who signed an ICF, had their blood sample collected and assayed for AAV-Spark100 or AAV6 immunity testing, who were subsequently identified as nAb negative (negative for nAb to AAV6 for the hemophilia A cohort), met the inclusion/ exclusion criteria and who participated and completed at least 6 months of the prospective data collection phase as part of their usual healthcare setting.

ABR per participant was calculated as number of bleeds over number of days from baseline visit (Day 1 of the study) to end of study\*365.25 days. Traumatic bleeds were defined as bleeding event occurring for an apparent or known reason. All bleeds included treated and untreated bleeds. A treated bleed was defined as an event necessitating administration of coagulation factor within 72 hours of signs or symptoms of bleeding. An untreated bleed was defined as an event not necessitating administration of coagulation factor within 72 hours of signs or symptoms of bleeding. In this outcome measure, ABR for traumatic all and treated bleeds during prospective data collection period in hemophilia A participants per protocol analysis set was reported.

Outcome measures

Outcome measures
Measure	Standard of Care FIX Replacement Therapy (Hemophilia B) n=81 Participants Participants with moderately severe to severe hemophilia B who administered their own current FIX replacement therapy in the usual healthcare setting were included. No investigational product was administered as a part of this study.	Standard of Care FVIII Replacement Therapy (Hemophilia A) Participants with moderately severe to severe hemophilia A who administered their own current FVIII replacement therapy in the usual healthcare setting were included. No investigational product was administered as a part of this study.
ABR for Traumatic Treated Bleeds and All Bleeds During Prospective Data Collection Period in Hemophilia A Participants: Per-protocol Analysis Set Treated bleeds	1.45 Bleeds per year Standard Deviation 3.467	—
ABR for Traumatic Treated Bleeds and All Bleeds During Prospective Data Collection Period in Hemophilia A Participants: Per-protocol Analysis Set All bleeds	1.60 Bleeds per year Standard Deviation 3.608	—

OTHER_PRE_SPECIFIED outcome

Timeframe: During prospective data collection period: Day 1 through end of study visit (from baseline visit of the study [Day 1] up to end of study follow-up [maximum follow-up: 1269 days])

Population: Safety analysis set included all enrolled participants (negative for nAb to AAV-Spark100 for the hemophilia B cohort, or negative for nAb to AAV6 for the hemophilia A cohort) who entered the prospective data collection phase.

An adverse event (AE) was any untoward medical occurrence in a study participant administered a product or medical device; the event need not necessarily have a causal relationship with the treatment or usage. An SAE was any untoward medical occurrence at any dose that met 1 of the following criteria: resulted in death; was life-threatening; required inpatient hospitalization or prolongation of existing hospitalization; resulted in persistent or significant disability/ incapacity; resulted in congenital anomaly/birth defect; other important medical events per protocol of the study.

Outcome measures

Outcome measures
Measure	Standard of Care FIX Replacement Therapy (Hemophilia B) n=111 Participants Participants with moderately severe to severe hemophilia B who administered their own current FIX replacement therapy in the usual healthcare setting were included. No investigational product was administered as a part of this study.	Standard of Care FVIII Replacement Therapy (Hemophilia A) n=101 Participants Participants with moderately severe to severe hemophilia A who administered their own current FVIII replacement therapy in the usual healthcare setting were included. No investigational product was administered as a part of this study.
Number of Participants With Serious Adverse Events (SAEs)	9 Participants	4 Participants

OTHER_PRE_SPECIFIED outcome

Timeframe: During prospective data collection period: Day 1 through end of study visit (from baseline visit of the study [Day 1] up to end of study follow-up [maximum follow-up: 1269 days])

Population: Safety analysis set included all enrolled participants (negative for nAb to AAV-Spark100 for the hemophilia B cohort, or negative for nAb to AAV6 for the hemophilia A cohort) who entered the prospective data collection phase.

An AE was any untoward medical occurrence in a study participant administered a product or medical device; the event need not necessarily have a causal relationship with the treatment or usage. Adverse events of special interest are FIX/FVIII inhibitor development, thrombotic events, and FIX hypersensitivity events for this study.

Outcome measures

Outcome measures
Measure	Standard of Care FIX Replacement Therapy (Hemophilia B) n=111 Participants Participants with moderately severe to severe hemophilia B who administered their own current FIX replacement therapy in the usual healthcare setting were included. No investigational product was administered as a part of this study.	Standard of Care FVIII Replacement Therapy (Hemophilia A) n=101 Participants Participants with moderately severe to severe hemophilia A who administered their own current FVIII replacement therapy in the usual healthcare setting were included. No investigational product was administered as a part of this study.
Number of Participants With Adverse Events of Special Interest (AESIs)	2 Participants	0 Participants

Adverse Events

Standard of Care FIX Replacement Therapy (Hemophilia B)

Serious events: 9 serious events

Other events: 4 other events

Deaths: 0 deaths

Standard of Care FVIII Replacement Therapy (Hemophilia A)

Serious events: 4 serious events

Other events: 0 other events

Deaths: 0 deaths

Serious adverse events

Serious adverse events
Measure	Standard of Care FIX Replacement Therapy (Hemophilia B) n=111 participants at risk Participants with moderately severe to severe hemophilia B who administered their own current FIX replacement therapy in the usual healthcare setting were included. No investigational product was administered as a part of this study.	Standard of Care FVIII Replacement Therapy (Hemophilia A) n=101 participants at risk Participants with moderately severe to severe hemophilia A who administered their own current FVIII replacement therapy in the usual healthcare setting were included. No investigational product was administered as a part of this study.
Gastrointestinal disorders Enteritis	0.90% 1/111 • During prospective data collection period (from baseline visit of the study [Day 1] up to end of study follow-up [maximum follow-up: 1269 days]) Same event may occur as both non-SAE and SAE but are distinct events. An event may be categorised as serious in 1 participant and non-serious in another, or a participant may have experienced both SAE and non-SAE. Safety analysis set was evaluated.	0.00% 0/101 • During prospective data collection period (from baseline visit of the study [Day 1] up to end of study follow-up [maximum follow-up: 1269 days]) Same event may occur as both non-SAE and SAE but are distinct events. An event may be categorised as serious in 1 participant and non-serious in another, or a participant may have experienced both SAE and non-SAE. Safety analysis set was evaluated.
Gastrointestinal disorders Gastrointestinal haemorrhage	1.8% 2/111 • During prospective data collection period (from baseline visit of the study [Day 1] up to end of study follow-up [maximum follow-up: 1269 days]) Same event may occur as both non-SAE and SAE but are distinct events. An event may be categorised as serious in 1 participant and non-serious in another, or a participant may have experienced both SAE and non-SAE. Safety analysis set was evaluated.	0.00% 0/101 • During prospective data collection period (from baseline visit of the study [Day 1] up to end of study follow-up [maximum follow-up: 1269 days]) Same event may occur as both non-SAE and SAE but are distinct events. An event may be categorised as serious in 1 participant and non-serious in another, or a participant may have experienced both SAE and non-SAE. Safety analysis set was evaluated.
Infections and infestations Appendicitis	0.90% 1/111 • During prospective data collection period (from baseline visit of the study [Day 1] up to end of study follow-up [maximum follow-up: 1269 days]) Same event may occur as both non-SAE and SAE but are distinct events. An event may be categorised as serious in 1 participant and non-serious in another, or a participant may have experienced both SAE and non-SAE. Safety analysis set was evaluated.	0.00% 0/101 • During prospective data collection period (from baseline visit of the study [Day 1] up to end of study follow-up [maximum follow-up: 1269 days]) Same event may occur as both non-SAE and SAE but are distinct events. An event may be categorised as serious in 1 participant and non-serious in another, or a participant may have experienced both SAE and non-SAE. Safety analysis set was evaluated.
Injury, poisoning and procedural complications Joint injury	0.90% 1/111 • During prospective data collection period (from baseline visit of the study [Day 1] up to end of study follow-up [maximum follow-up: 1269 days]) Same event may occur as both non-SAE and SAE but are distinct events. An event may be categorised as serious in 1 participant and non-serious in another, or a participant may have experienced both SAE and non-SAE. Safety analysis set was evaluated.	0.00% 0/101 • During prospective data collection period (from baseline visit of the study [Day 1] up to end of study follow-up [maximum follow-up: 1269 days]) Same event may occur as both non-SAE and SAE but are distinct events. An event may be categorised as serious in 1 participant and non-serious in another, or a participant may have experienced both SAE and non-SAE. Safety analysis set was evaluated.
Injury, poisoning and procedural complications Ligament rupture	0.90% 1/111 • During prospective data collection period (from baseline visit of the study [Day 1] up to end of study follow-up [maximum follow-up: 1269 days]) Same event may occur as both non-SAE and SAE but are distinct events. An event may be categorised as serious in 1 participant and non-serious in another, or a participant may have experienced both SAE and non-SAE. Safety analysis set was evaluated.	0.00% 0/101 • During prospective data collection period (from baseline visit of the study [Day 1] up to end of study follow-up [maximum follow-up: 1269 days]) Same event may occur as both non-SAE and SAE but are distinct events. An event may be categorised as serious in 1 participant and non-serious in another, or a participant may have experienced both SAE and non-SAE. Safety analysis set was evaluated.
Injury, poisoning and procedural complications Ligament sprain	0.90% 1/111 • During prospective data collection period (from baseline visit of the study [Day 1] up to end of study follow-up [maximum follow-up: 1269 days]) Same event may occur as both non-SAE and SAE but are distinct events. An event may be categorised as serious in 1 participant and non-serious in another, or a participant may have experienced both SAE and non-SAE. Safety analysis set was evaluated.	0.00% 0/101 • During prospective data collection period (from baseline visit of the study [Day 1] up to end of study follow-up [maximum follow-up: 1269 days]) Same event may occur as both non-SAE and SAE but are distinct events. An event may be categorised as serious in 1 participant and non-serious in another, or a participant may have experienced both SAE and non-SAE. Safety analysis set was evaluated.
Injury, poisoning and procedural complications Tooth fracture	0.90% 1/111 • During prospective data collection period (from baseline visit of the study [Day 1] up to end of study follow-up [maximum follow-up: 1269 days]) Same event may occur as both non-SAE and SAE but are distinct events. An event may be categorised as serious in 1 participant and non-serious in another, or a participant may have experienced both SAE and non-SAE. Safety analysis set was evaluated.	0.00% 0/101 • During prospective data collection period (from baseline visit of the study [Day 1] up to end of study follow-up [maximum follow-up: 1269 days]) Same event may occur as both non-SAE and SAE but are distinct events. An event may be categorised as serious in 1 participant and non-serious in another, or a participant may have experienced both SAE and non-SAE. Safety analysis set was evaluated.
Musculoskeletal and connective tissue disorders Arthropathy	0.90% 1/111 • During prospective data collection period (from baseline visit of the study [Day 1] up to end of study follow-up [maximum follow-up: 1269 days]) Same event may occur as both non-SAE and SAE but are distinct events. An event may be categorised as serious in 1 participant and non-serious in another, or a participant may have experienced both SAE and non-SAE. Safety analysis set was evaluated.	0.00% 0/101 • During prospective data collection period (from baseline visit of the study [Day 1] up to end of study follow-up [maximum follow-up: 1269 days]) Same event may occur as both non-SAE and SAE but are distinct events. An event may be categorised as serious in 1 participant and non-serious in another, or a participant may have experienced both SAE and non-SAE. Safety analysis set was evaluated.
Musculoskeletal and connective tissue disorders Haemophilic arthropathy	0.90% 1/111 • During prospective data collection period (from baseline visit of the study [Day 1] up to end of study follow-up [maximum follow-up: 1269 days]) Same event may occur as both non-SAE and SAE but are distinct events. An event may be categorised as serious in 1 participant and non-serious in another, or a participant may have experienced both SAE and non-SAE. Safety analysis set was evaluated.	0.00% 0/101 • During prospective data collection period (from baseline visit of the study [Day 1] up to end of study follow-up [maximum follow-up: 1269 days]) Same event may occur as both non-SAE and SAE but are distinct events. An event may be categorised as serious in 1 participant and non-serious in another, or a participant may have experienced both SAE and non-SAE. Safety analysis set was evaluated.
Respiratory, thoracic and mediastinal disorders Pneumonitis	0.90% 1/111 • During prospective data collection period (from baseline visit of the study [Day 1] up to end of study follow-up [maximum follow-up: 1269 days]) Same event may occur as both non-SAE and SAE but are distinct events. An event may be categorised as serious in 1 participant and non-serious in another, or a participant may have experienced both SAE and non-SAE. Safety analysis set was evaluated.	0.00% 0/101 • During prospective data collection period (from baseline visit of the study [Day 1] up to end of study follow-up [maximum follow-up: 1269 days]) Same event may occur as both non-SAE and SAE but are distinct events. An event may be categorised as serious in 1 participant and non-serious in another, or a participant may have experienced both SAE and non-SAE. Safety analysis set was evaluated.
Gastrointestinal disorders Haemorrhoidal haemorrhage	0.00% 0/111 • During prospective data collection period (from baseline visit of the study [Day 1] up to end of study follow-up [maximum follow-up: 1269 days]) Same event may occur as both non-SAE and SAE but are distinct events. An event may be categorised as serious in 1 participant and non-serious in another, or a participant may have experienced both SAE and non-SAE. Safety analysis set was evaluated.	0.99% 1/101 • During prospective data collection period (from baseline visit of the study [Day 1] up to end of study follow-up [maximum follow-up: 1269 days]) Same event may occur as both non-SAE and SAE but are distinct events. An event may be categorised as serious in 1 participant and non-serious in another, or a participant may have experienced both SAE and non-SAE. Safety analysis set was evaluated.
Gastrointestinal disorders Upper gastrointestinal haemorrhage	0.00% 0/111 • During prospective data collection period (from baseline visit of the study [Day 1] up to end of study follow-up [maximum follow-up: 1269 days]) Same event may occur as both non-SAE and SAE but are distinct events. An event may be categorised as serious in 1 participant and non-serious in another, or a participant may have experienced both SAE and non-SAE. Safety analysis set was evaluated.	0.99% 1/101 • During prospective data collection period (from baseline visit of the study [Day 1] up to end of study follow-up [maximum follow-up: 1269 days]) Same event may occur as both non-SAE and SAE but are distinct events. An event may be categorised as serious in 1 participant and non-serious in another, or a participant may have experienced both SAE and non-SAE. Safety analysis set was evaluated.
Infections and infestations Wound infection	0.00% 0/111 • During prospective data collection period (from baseline visit of the study [Day 1] up to end of study follow-up [maximum follow-up: 1269 days]) Same event may occur as both non-SAE and SAE but are distinct events. An event may be categorised as serious in 1 participant and non-serious in another, or a participant may have experienced both SAE and non-SAE. Safety analysis set was evaluated.	0.99% 1/101 • During prospective data collection period (from baseline visit of the study [Day 1] up to end of study follow-up [maximum follow-up: 1269 days]) Same event may occur as both non-SAE and SAE but are distinct events. An event may be categorised as serious in 1 participant and non-serious in another, or a participant may have experienced both SAE and non-SAE. Safety analysis set was evaluated.
Neoplasms benign, malignant and unspecified (incl cysts and polyps) B-cell lymphoma	0.00% 0/111 • During prospective data collection period (from baseline visit of the study [Day 1] up to end of study follow-up [maximum follow-up: 1269 days]) Same event may occur as both non-SAE and SAE but are distinct events. An event may be categorised as serious in 1 participant and non-serious in another, or a participant may have experienced both SAE and non-SAE. Safety analysis set was evaluated.	0.99% 1/101 • During prospective data collection period (from baseline visit of the study [Day 1] up to end of study follow-up [maximum follow-up: 1269 days]) Same event may occur as both non-SAE and SAE but are distinct events. An event may be categorised as serious in 1 participant and non-serious in another, or a participant may have experienced both SAE and non-SAE. Safety analysis set was evaluated.

Other adverse events

Other adverse events
Measure	Standard of Care FIX Replacement Therapy (Hemophilia B) n=111 participants at risk Participants with moderately severe to severe hemophilia B who administered their own current FIX replacement therapy in the usual healthcare setting were included. No investigational product was administered as a part of this study.	Standard of Care FVIII Replacement Therapy (Hemophilia A) n=101 participants at risk Participants with moderately severe to severe hemophilia A who administered their own current FVIII replacement therapy in the usual healthcare setting were included. No investigational product was administered as a part of this study.
Vascular disorders Haematoma	1.8% 2/111 • During prospective data collection period (from baseline visit of the study [Day 1] up to end of study follow-up [maximum follow-up: 1269 days]) Same event may occur as both non-SAE and SAE but are distinct events. An event may be categorised as serious in 1 participant and non-serious in another, or a participant may have experienced both SAE and non-SAE. Safety analysis set was evaluated.	0.00% 0/101 • During prospective data collection period (from baseline visit of the study [Day 1] up to end of study follow-up [maximum follow-up: 1269 days]) Same event may occur as both non-SAE and SAE but are distinct events. An event may be categorised as serious in 1 participant and non-serious in another, or a participant may have experienced both SAE and non-SAE. Safety analysis set was evaluated.
Musculoskeletal and connective tissue disorders Arthralgia	1.8% 2/111 • During prospective data collection period (from baseline visit of the study [Day 1] up to end of study follow-up [maximum follow-up: 1269 days]) Same event may occur as both non-SAE and SAE but are distinct events. An event may be categorised as serious in 1 participant and non-serious in another, or a participant may have experienced both SAE and non-SAE. Safety analysis set was evaluated.	0.00% 0/101 • During prospective data collection period (from baseline visit of the study [Day 1] up to end of study follow-up [maximum follow-up: 1269 days]) Same event may occur as both non-SAE and SAE but are distinct events. An event may be categorised as serious in 1 participant and non-serious in another, or a participant may have experienced both SAE and non-SAE. Safety analysis set was evaluated.

Additional Information

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