Trial Outcomes & Findings for A Study to Evaluate the Safety, Efficacy, Pharmacokinetics and Pharmacodynamics of Emicizumab in Participants With Mild or Moderate Hemophilia A Without FVIII Inhibitors (NCT NCT04158648)

NCT ID: NCT04158648

Last Updated: 2026-01-23

Results Overview

The number of treated bleeds over the efficacy period was estimated as an annualized bleed rate (ABR) using a negative binomial regression model, which accounts for different follow-up times. A treated bleed was defined as a bleed that was directly followed by a hemophilia medication reported to be a "treatment for bleed". The 72-hour rule was implemented: two bleeds of the same type and at the same anatomical location were counted as one bleed if the second bleed occurred within 72 hours from the last treatment for the first bleed. Bleeds due to surgery/procedure were excluded.

• Recruitment status: COMPLETED
• Study phase: PHASE3
• Target enrollment: 73 participants
• Primary outcome timeframe: From the day of first emicizumab dose to at least 52 weeks of emicizumab treatment (median [range, min-max] efficacy period: 55.64 [8.6-89.9] weeks)
• Results posted on: 2026-01-23

Participant Flow

Participant milestones

Measure	Emicizumab Participants with mild and moderate hemophilia A without factor VIII (FVIII) inhibitors were enrolled to receive the emicizumab loading dose regimen (emicizumab 3 mg/kg administered subcutaneously \[SC\] QW for 4 weeks) followed by participant preference of one of the following 3 emicizumab SC maintenance dose regimens: 1.5 mg/kg QW, 3 mg/kg Q2W, or 6 mg/kg Q4W.
Overall Study STARTED	73
Overall Study Received at Least One Dose of Study Drug	72
Overall Study Completed 24 Weeks in the Study	71
Overall Study Completed 52 Weeks in the Study	57
Overall Study COMPLETED	0
Overall Study NOT COMPLETED	73

Reasons for withdrawal

Measure	Emicizumab Participants with mild and moderate hemophilia A without factor VIII (FVIII) inhibitors were enrolled to receive the emicizumab loading dose regimen (emicizumab 3 mg/kg administered subcutaneously \[SC\] QW for 4 weeks) followed by participant preference of one of the following 3 emicizumab SC maintenance dose regimens: 1.5 mg/kg QW, 3 mg/kg Q2W, or 6 mg/kg Q4W.
Overall Study Ongoing in the Study	69
Overall Study Withdrawal by Subject	4

Baseline Characteristics

A Study to Evaluate the Safety, Efficacy, Pharmacokinetics and Pharmacodynamics of Emicizumab in Participants With Mild or Moderate Hemophilia A Without FVIII Inhibitors

Baseline characteristics by cohort

Measure	Emicizumab n=73 Participants Participants with mild and moderate hemophilia A without factor VIII (FVIII) inhibitors were enrolled to receive the emicizumab loading dose regimen (emicizumab 3 mg/kg administered subcutaneously \[SC\] QW for 4 weeks) followed by participant preference of one of the following 3 emicizumab SC maintenance dose regimens: 1.5 mg/kg QW, 3 mg/kg Q2W, or 6 mg/kg Q4W.
Age, Continuous	25.9 Years STANDARD_DEVIATION 17.0 • n=270 Participants
Age, Customized 2 to <6 Years Old	5 Participants n=270 Participants
Age, Customized 6 to <12 Years Old	11 Participants n=270 Participants
Age, Customized 12 to <18 Years Old	14 Participants n=270 Participants
Age, Customized 18 to <65 Years Old	41 Participants n=270 Participants
Age, Customized ≥65 Years Old	2 Participants n=270 Participants
Sex: Female, Male Female	3 Participants n=270 Participants
Sex: Female, Male Male	70 Participants n=270 Participants
Ethnicity (NIH/OMB) Hispanic or Latino	2 Participants n=270 Participants
Ethnicity (NIH/OMB) Not Hispanic or Latino	61 Participants n=270 Participants
Ethnicity (NIH/OMB) Unknown or Not Reported	10 Participants n=270 Participants
Race/Ethnicity, Customized Asian	3 Participants n=270 Participants
Race/Ethnicity, Customized Black or African American	6 Participants n=270 Participants
Race/Ethnicity, Customized White	62 Participants n=270 Participants
Race/Ethnicity, Customized Unknown	2 Participants n=270 Participants
Hemophilia A Severity Mild	21 Participants n=270 Participants
Hemophilia A Severity Moderate	52 Participants n=270 Participants
Emicizumab Maintenance Dosing Regimen Chosen at Enrollment 1.5 mg/kg Once Every Week (QW)	25 Participants n=270 Participants
Emicizumab Maintenance Dosing Regimen Chosen at Enrollment 3 mg/kg Once Every 2 Weeks (Q2W)	39 Participants n=270 Participants
Emicizumab Maintenance Dosing Regimen Chosen at Enrollment 6 mg/kg Once Every 4 Weeks (Q4W)	8 Participants n=270 Participants
Emicizumab Maintenance Dosing Regimen Chosen at Enrollment Not Assigned (Not Treated)	1 Participants n=270 Participants

PRIMARY outcome

Timeframe: From the day of first emicizumab dose to at least 52 weeks of emicizumab treatment (median [range, min-max] efficacy period: 55.64 [8.6-89.9] weeks)

Population: The Treated Population comprises all participants who received at least one dose of emicizumab.

The number of treated bleeds over the efficacy period was estimated as an annualized bleed rate (ABR) using a negative binomial regression model, which accounts for different follow-up times. A treated bleed was defined as a bleed that was directly followed by a hemophilia medication reported to be a "treatment for bleed". The 72-hour rule was implemented: two bleeds of the same type and at the same anatomical location were counted as one bleed if the second bleed occurred within 72 hours from the last treatment for the first bleed. Bleeds due to surgery/procedure were excluded.

Outcome measures

Measure	Emicizumab n=72 Participants Participants with mild and moderate hemophilia A without factor VIII (FVIII) inhibitors were enrolled to receive the emicizumab loading dose regimen (emicizumab 3 mg/kg administered subcutaneously \[SC\] QW for 4 weeks) followed by participant preference of one of the following 3 emicizumab SC maintenance dose regimens: 1.5 mg/kg QW, 3 mg/kg Q2W, or 6 mg/kg Q4W.	Participants ≥5 to <18 Years Old: Emicizumab Participants with mild and moderate hemophilia A without factor VIII (FVIII) inhibitors were enrolled and received the emicizumab loading dose regimen (emicizumab 3 mg/kg administered subcutaneously QW for 4 weeks) followed by participant preference of one of the following 3 emicizumab maintenance dose regimens: 1.5 mg/kg QW, 3 mg/kg Q2W, or 6 mg/kg Q4W.	Participants ≥18 Years Old: Emicizumab Participants with mild and moderate hemophilia A without factor VIII (FVIII) inhibitors were enrolled and received the emicizumab loading dose regimen (emicizumab 3 mg/kg administered subcutaneously QW for 4 weeks) followed by participant preference of one of the following 3 emicizumab maintenance dose regimens: 1.5 mg/kg QW, 3 mg/kg Q2W, or 6 mg/kg Q4W.
Model-Based Annualized Bleed Rate for Treated Bleeds	0.9 Treated bleeds per year Interval 0.55 to 1.52	—	—

PRIMARY outcome

Timeframe: From the day of first emicizumab dose to at least 52 weeks of emicizumab treatment (median [range, min-max] efficacy period: 55.64 [8.6-89.9] weeks)

Population: The Treated Population comprises all participants who received at least one dose of emicizumab.

The number of treated bleeds over the efficacy period is presented here as a calculated annualized bleed rate (ABR) that was annualized for each participant using the following formula: ABR = (number of bleeds/number of days during the efficacy period) x 365.25. A treated bleed was defined as a bleed that was directly followed by a hemophilia medication reported to be a "treatment for bleed". The 72-hour rule was implemented: two bleeds of the same type and at the same anatomical location were counted as one bleed if the second bleed occurred within 72 hours from the last treatment for the first bleed. Bleeds due to surgery/procedure were excluded.

Outcome measures

Measure	Emicizumab n=72 Participants Participants with mild and moderate hemophilia A without factor VIII (FVIII) inhibitors were enrolled to receive the emicizumab loading dose regimen (emicizumab 3 mg/kg administered subcutaneously \[SC\] QW for 4 weeks) followed by participant preference of one of the following 3 emicizumab SC maintenance dose regimens: 1.5 mg/kg QW, 3 mg/kg Q2W, or 6 mg/kg Q4W.	Participants ≥5 to <18 Years Old: Emicizumab Participants with mild and moderate hemophilia A without factor VIII (FVIII) inhibitors were enrolled and received the emicizumab loading dose regimen (emicizumab 3 mg/kg administered subcutaneously QW for 4 weeks) followed by participant preference of one of the following 3 emicizumab maintenance dose regimens: 1.5 mg/kg QW, 3 mg/kg Q2W, or 6 mg/kg Q4W.	Participants ≥18 Years Old: Emicizumab Participants with mild and moderate hemophilia A without factor VIII (FVIII) inhibitors were enrolled and received the emicizumab loading dose regimen (emicizumab 3 mg/kg administered subcutaneously QW for 4 weeks) followed by participant preference of one of the following 3 emicizumab maintenance dose regimens: 1.5 mg/kg QW, 3 mg/kg Q2W, or 6 mg/kg Q4W.
Mean Calculated Annualized Bleed Rate for Treated Bleeds	0.9 Treated bleeds per year Interval 0.02 to 5.48	—	—

PRIMARY outcome

Timeframe: From the day of first emicizumab dose to at least 52 weeks of emicizumab treatment (median [range, min-max] efficacy period: 55.64 [8.6-89.9] weeks)

Population: The Treated Population comprises all participants who received at least one dose of emicizumab.

The number of treated bleeds over the efficacy period is presented here as a calculated annualized bleed rate (ABR) that was annualized for each participant using the following formula: ABR = (number of bleeds/number of days during the efficacy period) x 365.25. A treated bleed was defined as a bleed that was directly followed by a hemophilia medication reported to be a "treatment for bleed". The 72-hour rule was implemented: two bleeds of the same type and at the same anatomical location were counted as one bleed if the second bleed occurred within 72 hours from the last treatment for the first bleed. Bleeds due to surgery/procedure were excluded.

Outcome measures

Measure	Emicizumab n=72 Participants Participants with mild and moderate hemophilia A without factor VIII (FVIII) inhibitors were enrolled to receive the emicizumab loading dose regimen (emicizumab 3 mg/kg administered subcutaneously \[SC\] QW for 4 weeks) followed by participant preference of one of the following 3 emicizumab SC maintenance dose regimens: 1.5 mg/kg QW, 3 mg/kg Q2W, or 6 mg/kg Q4W.	Participants ≥5 to <18 Years Old: Emicizumab Participants with mild and moderate hemophilia A without factor VIII (FVIII) inhibitors were enrolled and received the emicizumab loading dose regimen (emicizumab 3 mg/kg administered subcutaneously QW for 4 weeks) followed by participant preference of one of the following 3 emicizumab maintenance dose regimens: 1.5 mg/kg QW, 3 mg/kg Q2W, or 6 mg/kg Q4W.	Participants ≥18 Years Old: Emicizumab Participants with mild and moderate hemophilia A without factor VIII (FVIII) inhibitors were enrolled and received the emicizumab loading dose regimen (emicizumab 3 mg/kg administered subcutaneously QW for 4 weeks) followed by participant preference of one of the following 3 emicizumab maintenance dose regimens: 1.5 mg/kg QW, 3 mg/kg Q2W, or 6 mg/kg Q4W.
Median Calculated Annualized Bleed Rate for Treated Bleeds	0.0 Treated bleeds per year Interval 0.0 to 0.98	—	—

SECONDARY outcome

Timeframe: From the day of first emicizumab dose to at least 52 weeks of emicizumab treatment (median [range, min-max] efficacy period: 55.64 [8.6-89.9] weeks)

Population: The Treated Population comprises all participants who received at least one dose of emicizumab.

The number of all bleeds over the efficacy period was estimated as an annualized bleed rate (ABR) using a negative binomial regression model, which accounts for different follow-up times. In this outcome measure, all bleeds are included, irrespective of treatment with coagulation factors, with the following exception: bleeds due to surgery/procedure are excluded. As "all bleeds" comprises both treated and non-treated bleeds, the 72-hour rule was implemented separately for treated and non-treated bleeds. For treated bleeds, the 72-hour rule meant that two bleeds of the same type and at the same anatomical location were counted as one bleed if the second bleed occurred within 72 hours from the last treatment for the first bleed. For non-treated bleeds, the 72-hour rule was implemented by calculating a treatment-free period of 72 hours from the bleed itself.

Outcome measures

Measure	Emicizumab n=72 Participants Participants with mild and moderate hemophilia A without factor VIII (FVIII) inhibitors were enrolled to receive the emicizumab loading dose regimen (emicizumab 3 mg/kg administered subcutaneously \[SC\] QW for 4 weeks) followed by participant preference of one of the following 3 emicizumab SC maintenance dose regimens: 1.5 mg/kg QW, 3 mg/kg Q2W, or 6 mg/kg Q4W.	Participants ≥5 to <18 Years Old: Emicizumab Participants with mild and moderate hemophilia A without factor VIII (FVIII) inhibitors were enrolled and received the emicizumab loading dose regimen (emicizumab 3 mg/kg administered subcutaneously QW for 4 weeks) followed by participant preference of one of the following 3 emicizumab maintenance dose regimens: 1.5 mg/kg QW, 3 mg/kg Q2W, or 6 mg/kg Q4W.	Participants ≥18 Years Old: Emicizumab Participants with mild and moderate hemophilia A without factor VIII (FVIII) inhibitors were enrolled and received the emicizumab loading dose regimen (emicizumab 3 mg/kg administered subcutaneously QW for 4 weeks) followed by participant preference of one of the following 3 emicizumab maintenance dose regimens: 1.5 mg/kg QW, 3 mg/kg Q2W, or 6 mg/kg Q4W.
Model-Based Annualized Bleed Rate for All Bleeds	2.3 All bleeds per year Interval 1.67 to 3.12	—	—

SECONDARY outcome

Timeframe: From the day of first emicizumab dose to at least 52 weeks of emicizumab treatment (median [range, min-max] efficacy period: 55.64 [8.6-89.9] weeks)

Population: The Treated Population comprises all participants who received at least one dose of emicizumab.

The number of all bleeds over the efficacy period is presented here as a calculated annualized bleed rate (ABR) that was annualized for each participant using the following formula: ABR = (number of bleeds/number of days during the efficacy period) x 365.25. In this outcome measure, all bleeds are included, irrespective of treatment with coagulation factors, with the following exception: bleeds due to surgery/procedure are excluded. As "all bleeds" comprises both treated and non-treated bleeds, the 72-hour rule was implemented separately for treated and non-treated bleeds. For treated bleeds, the 72-hour rule meant that two bleeds of the same type and at the same anatomical location were counted as one bleed if the second bleed occurred within 72 hours from the last treatment for the first bleed. For non-treated bleeds, the 72-hour rule was implemented by calculating a treatment-free period of 72 hours from the bleed itself.

Outcome measures

Measure	Emicizumab n=72 Participants Participants with mild and moderate hemophilia A without factor VIII (FVIII) inhibitors were enrolled to receive the emicizumab loading dose regimen (emicizumab 3 mg/kg administered subcutaneously \[SC\] QW for 4 weeks) followed by participant preference of one of the following 3 emicizumab SC maintenance dose regimens: 1.5 mg/kg QW, 3 mg/kg Q2W, or 6 mg/kg Q4W.	Participants ≥5 to <18 Years Old: Emicizumab Participants with mild and moderate hemophilia A without factor VIII (FVIII) inhibitors were enrolled and received the emicizumab loading dose regimen (emicizumab 3 mg/kg administered subcutaneously QW for 4 weeks) followed by participant preference of one of the following 3 emicizumab maintenance dose regimens: 1.5 mg/kg QW, 3 mg/kg Q2W, or 6 mg/kg Q4W.	Participants ≥18 Years Old: Emicizumab Participants with mild and moderate hemophilia A without factor VIII (FVIII) inhibitors were enrolled and received the emicizumab loading dose regimen (emicizumab 3 mg/kg administered subcutaneously QW for 4 weeks) followed by participant preference of one of the following 3 emicizumab maintenance dose regimens: 1.5 mg/kg QW, 3 mg/kg Q2W, or 6 mg/kg Q4W.
Mean Calculated Annualized Bleed Rate for All Bleeds	2.3 All bleeds per year Interval 0.35 to 7.75	—	—

SECONDARY outcome

Timeframe: From the day of first emicizumab dose to at least 52 weeks of emicizumab treatment (median [range, min-max] efficacy period: 55.64 [8.6-89.9] weeks)

Population: The Treated Population comprises all participants who received at least one dose of emicizumab.

The number of all bleeds over the efficacy period is presented here as a calculated annualized bleed rate (ABR) that was annualized for each participant using the following formula: ABR = (number of bleeds/number of days during the efficacy period) x 365.25. In this outcome measure, all bleeds are included, irrespective of treatment with coagulation factors, with the following exception: bleeds due to surgery/procedure are excluded. As "all bleeds" comprises both treated and non-treated bleeds, the 72-hour rule was implemented separately for treated and non-treated bleeds. For treated bleeds, the 72-hour rule meant that two bleeds of the same type and at the same anatomical location were counted as one bleed if the second bleed occurred within 72 hours from the last treatment for the first bleed. For non-treated bleeds, the 72-hour rule was implemented by calculating a treatment-free period of 72 hours from the bleed itself.

Outcome measures

Measure	Emicizumab n=72 Participants Participants with mild and moderate hemophilia A without factor VIII (FVIII) inhibitors were enrolled to receive the emicizumab loading dose regimen (emicizumab 3 mg/kg administered subcutaneously \[SC\] QW for 4 weeks) followed by participant preference of one of the following 3 emicizumab SC maintenance dose regimens: 1.5 mg/kg QW, 3 mg/kg Q2W, or 6 mg/kg Q4W.	Participants ≥5 to <18 Years Old: Emicizumab Participants with mild and moderate hemophilia A without factor VIII (FVIII) inhibitors were enrolled and received the emicizumab loading dose regimen (emicizumab 3 mg/kg administered subcutaneously QW for 4 weeks) followed by participant preference of one of the following 3 emicizumab maintenance dose regimens: 1.5 mg/kg QW, 3 mg/kg Q2W, or 6 mg/kg Q4W.	Participants ≥18 Years Old: Emicizumab Participants with mild and moderate hemophilia A without factor VIII (FVIII) inhibitors were enrolled and received the emicizumab loading dose regimen (emicizumab 3 mg/kg administered subcutaneously QW for 4 weeks) followed by participant preference of one of the following 3 emicizumab maintenance dose regimens: 1.5 mg/kg QW, 3 mg/kg Q2W, or 6 mg/kg Q4W.
Median Calculated Annualized Bleed Rate for All Bleeds	1.0 All bleeds per year Interval 0.0 to 3.11	—	—

SECONDARY outcome

Timeframe: From the day of first emicizumab dose to at least 52 weeks of emicizumab treatment (median [range, min-max] efficacy period: 55.64 [8.6-89.9] weeks)

Population: The Treated Population comprises all participants who received at least one dose of emicizumab.

The number of treated joint bleeds over the efficacy period was estimated as an annualized bleed rate (ABR) using a negative binomial regression model, which accounts for different follow-up times. A treated joint bleed was defined as a bleed with type reported as "joint" based on at least one of the following symptoms: increasing swelling or warmth of the skin over the joint and/or increasing pain, decreased range of motion, or difficulty using the joint compared with baseline, and the bleed was directly followed by a hemophilia medication reported to be a "treatment for bleed". The 72-hour rule was implemented: two bleeds of the same type and at the same anatomical location were counted as one bleed if the second bleed occurred within 72 hours from the last treatment for the first bleed. Bleeds due to surgery/procedure were excluded.

Outcome measures

Measure	Emicizumab n=72 Participants Participants with mild and moderate hemophilia A without factor VIII (FVIII) inhibitors were enrolled to receive the emicizumab loading dose regimen (emicizumab 3 mg/kg administered subcutaneously \[SC\] QW for 4 weeks) followed by participant preference of one of the following 3 emicizumab SC maintenance dose regimens: 1.5 mg/kg QW, 3 mg/kg Q2W, or 6 mg/kg Q4W.	Participants ≥5 to <18 Years Old: Emicizumab Participants with mild and moderate hemophilia A without factor VIII (FVIII) inhibitors were enrolled and received the emicizumab loading dose regimen (emicizumab 3 mg/kg administered subcutaneously QW for 4 weeks) followed by participant preference of one of the following 3 emicizumab maintenance dose regimens: 1.5 mg/kg QW, 3 mg/kg Q2W, or 6 mg/kg Q4W.	Participants ≥18 Years Old: Emicizumab Participants with mild and moderate hemophilia A without factor VIII (FVIII) inhibitors were enrolled and received the emicizumab loading dose regimen (emicizumab 3 mg/kg administered subcutaneously QW for 4 weeks) followed by participant preference of one of the following 3 emicizumab maintenance dose regimens: 1.5 mg/kg QW, 3 mg/kg Q2W, or 6 mg/kg Q4W.
Model-Based Annualized Bleed Rate for Treated Joint Bleeds	0.2 Treated joint bleeds per year Interval 0.09 to 0.57	—	—

SECONDARY outcome

Timeframe: From the day of first emicizumab dose to at least 52 weeks of emicizumab treatment (median [range, min-max] efficacy period: 55.64 [8.6-89.9] weeks)

Population: The Treated Population comprises all participants who received at least one dose of emicizumab.

The number of treated joint bleeds over the efficacy period is presented here as a calculated annualized bleed rate (ABR) that was annualized for each participant using the following formula: ABR = (number of bleeds/number of days during the efficacy period) x 365.25. A treated joint bleed was defined as a bleed with type reported as "joint" based on at least one of the following symptoms: increasing swelling or warmth of the skin over the joint and/or increasing pain, decreased range of motion, or difficulty using the joint compared with baseline, and the bleed was directly followed by a hemophilia medication reported to be a "treatment for bleed". The 72-hour rule was implemented: two bleeds of the same type and at the same anatomical location were counted as one bleed if the second bleed occurred within 72 hours from the last treatment for the first bleed. Bleeds due to surgery/procedure were excluded.

Outcome measures

Measure	Emicizumab n=72 Participants Participants with mild and moderate hemophilia A without factor VIII (FVIII) inhibitors were enrolled to receive the emicizumab loading dose regimen (emicizumab 3 mg/kg administered subcutaneously \[SC\] QW for 4 weeks) followed by participant preference of one of the following 3 emicizumab SC maintenance dose regimens: 1.5 mg/kg QW, 3 mg/kg Q2W, or 6 mg/kg Q4W.	Participants ≥5 to <18 Years Old: Emicizumab Participants with mild and moderate hemophilia A without factor VIII (FVIII) inhibitors were enrolled and received the emicizumab loading dose regimen (emicizumab 3 mg/kg administered subcutaneously QW for 4 weeks) followed by participant preference of one of the following 3 emicizumab maintenance dose regimens: 1.5 mg/kg QW, 3 mg/kg Q2W, or 6 mg/kg Q4W.	Participants ≥18 Years Old: Emicizumab Participants with mild and moderate hemophilia A without factor VIII (FVIII) inhibitors were enrolled and received the emicizumab loading dose regimen (emicizumab 3 mg/kg administered subcutaneously QW for 4 weeks) followed by participant preference of one of the following 3 emicizumab maintenance dose regimens: 1.5 mg/kg QW, 3 mg/kg Q2W, or 6 mg/kg Q4W.
Mean Calculated Annualized Bleed Rate for Treated Joint Bleeds	0.2 Treated joint bleeds per year Interval 0.0 to 4.15	—	—

SECONDARY outcome

Timeframe: From the day of first emicizumab dose to at least 52 weeks of emicizumab treatment (median [range, min-max] efficacy period: 55.64 [8.6-89.9] weeks)

Population: The Treated Population comprises all participants who received at least one dose of emicizumab.

The number of treated joint bleeds over the efficacy period is presented here as a calculated annualized bleed rate (ABR) that was annualized for each participant using the following formula: ABR = (number of bleeds/number of days during the efficacy period) x 365.25. A treated joint bleed was defined as a bleed with type reported as "joint" based on at least one of the following symptoms: increasing swelling or warmth of the skin over the joint and/or increasing pain, decreased range of motion, or difficulty using the joint compared with baseline, and the bleed was directly followed by a hemophilia medication reported to be a "treatment for bleed". The 72-hour rule was implemented: two bleeds of the same type and at the same anatomical location were counted as one bleed if the second bleed occurred within 72 hours from the last treatment for the first bleed. Bleeds due to surgery/procedure were excluded.

Outcome measures

Measure	Emicizumab n=72 Participants Participants with mild and moderate hemophilia A without factor VIII (FVIII) inhibitors were enrolled to receive the emicizumab loading dose regimen (emicizumab 3 mg/kg administered subcutaneously \[SC\] QW for 4 weeks) followed by participant preference of one of the following 3 emicizumab SC maintenance dose regimens: 1.5 mg/kg QW, 3 mg/kg Q2W, or 6 mg/kg Q4W.	Participants ≥5 to <18 Years Old: Emicizumab Participants with mild and moderate hemophilia A without factor VIII (FVIII) inhibitors were enrolled and received the emicizumab loading dose regimen (emicizumab 3 mg/kg administered subcutaneously QW for 4 weeks) followed by participant preference of one of the following 3 emicizumab maintenance dose regimens: 1.5 mg/kg QW, 3 mg/kg Q2W, or 6 mg/kg Q4W.	Participants ≥18 Years Old: Emicizumab Participants with mild and moderate hemophilia A without factor VIII (FVIII) inhibitors were enrolled and received the emicizumab loading dose regimen (emicizumab 3 mg/kg administered subcutaneously QW for 4 weeks) followed by participant preference of one of the following 3 emicizumab maintenance dose regimens: 1.5 mg/kg QW, 3 mg/kg Q2W, or 6 mg/kg Q4W.
Median Calculated Annualized Bleed Rate for Treated Joint Bleeds	0.0 Treated joint bleeds per year Interval 0.0 to 0.0	—	—

SECONDARY outcome

Timeframe: From the day of first emicizumab dose to at least 52 weeks of emicizumab treatment (median [range, min-max] efficacy period: 55.64 [8.6-89.9] weeks)

Population: The Treated Population comprises all participants who received at least one dose of emicizumab.

The number of treated target joint bleeds over the efficacy period was estimated as an annualized bleed rate (ABR) using a negative binomial regression model, which accounts for different follow-up times. A treated target joint bleed was defined as a joint bleed in a target joint, which is a joint location where at least 3 bleeds have occurred over the last 24 weeks prior to study entry or an unresolved target joint (target joint that does not fulfil ≤2 bleeds into this joint within a consecutive 12-month period), and the bleed was directly followed by a hemophilia medication reported to be a "treatment for bleed". The 72-hour rule was implemented: two bleeds of the same type and at the same anatomical location were counted as one bleed if the second bleed occurred within 72 hours from the last treatment for the first bleed. Bleeds due to surgery/procedure were excluded.

Outcome measures

Measure	Emicizumab n=72 Participants Participants with mild and moderate hemophilia A without factor VIII (FVIII) inhibitors were enrolled to receive the emicizumab loading dose regimen (emicizumab 3 mg/kg administered subcutaneously \[SC\] QW for 4 weeks) followed by participant preference of one of the following 3 emicizumab SC maintenance dose regimens: 1.5 mg/kg QW, 3 mg/kg Q2W, or 6 mg/kg Q4W.	Participants ≥5 to <18 Years Old: Emicizumab Participants with mild and moderate hemophilia A without factor VIII (FVIII) inhibitors were enrolled and received the emicizumab loading dose regimen (emicizumab 3 mg/kg administered subcutaneously QW for 4 weeks) followed by participant preference of one of the following 3 emicizumab maintenance dose regimens: 1.5 mg/kg QW, 3 mg/kg Q2W, or 6 mg/kg Q4W.	Participants ≥18 Years Old: Emicizumab Participants with mild and moderate hemophilia A without factor VIII (FVIII) inhibitors were enrolled and received the emicizumab loading dose regimen (emicizumab 3 mg/kg administered subcutaneously QW for 4 weeks) followed by participant preference of one of the following 3 emicizumab maintenance dose regimens: 1.5 mg/kg QW, 3 mg/kg Q2W, or 6 mg/kg Q4W.
Model-Based Annualized Bleed Rate for Treated Target Joint Bleeds	0.1 Treated target joint bleeds per year Interval 0.03 to 0.4	—	—

SECONDARY outcome

Timeframe: From the day of first emicizumab dose to at least 52 weeks of emicizumab treatment (median [range, min-max] efficacy period: 55.64 [8.6-89.9] weeks)

Population: The Treated Population comprises all participants who received at least one dose of emicizumab.

The number of treated target joint bleeds over the efficacy period is presented here as a calculated annualized bleed rate (ABR) that was annualized for each participant using the following formula: ABR = (number of bleeds/number of days during the efficacy period) x 365.25. A treated target joint bleed was defined as a joint bleed in a target joint, which is a joint location where at least 3 bleeds have occurred over the last 24 weeks prior to study entry or an unresolved target joint (target joint that does not fulfil ≤2 bleeds into this joint within a consecutive 12-month period), and the bleed was directly followed by a hemophilia medication reported to be a "treatment for bleed". The 72-hour rule was implemented: two bleeds of the same type and at the same anatomical location were counted as one bleed if the second bleed occurred within 72 hours from the last treatment for the first bleed. Bleeds due to surgery/procedure were excluded.

Outcome measures

Measure	Emicizumab n=72 Participants Participants with mild and moderate hemophilia A without factor VIII (FVIII) inhibitors were enrolled to receive the emicizumab loading dose regimen (emicizumab 3 mg/kg administered subcutaneously \[SC\] QW for 4 weeks) followed by participant preference of one of the following 3 emicizumab SC maintenance dose regimens: 1.5 mg/kg QW, 3 mg/kg Q2W, or 6 mg/kg Q4W.	Participants ≥5 to <18 Years Old: Emicizumab Participants with mild and moderate hemophilia A without factor VIII (FVIII) inhibitors were enrolled and received the emicizumab loading dose regimen (emicizumab 3 mg/kg administered subcutaneously QW for 4 weeks) followed by participant preference of one of the following 3 emicizumab maintenance dose regimens: 1.5 mg/kg QW, 3 mg/kg Q2W, or 6 mg/kg Q4W.	Participants ≥18 Years Old: Emicizumab Participants with mild and moderate hemophilia A without factor VIII (FVIII) inhibitors were enrolled and received the emicizumab loading dose regimen (emicizumab 3 mg/kg administered subcutaneously QW for 4 weeks) followed by participant preference of one of the following 3 emicizumab maintenance dose regimens: 1.5 mg/kg QW, 3 mg/kg Q2W, or 6 mg/kg Q4W.
Mean Calculated Annualized Bleed Rate for Treated Target Joint Bleeds	0.1 Treated target joint bleeds per year Interval 0.0 to 3.92	—	—

SECONDARY outcome

Timeframe: From the day of first emicizumab dose to at least 52 weeks of emicizumab treatment (median [range, min-max] efficacy period: 55.64 [8.6-89.9] weeks)

Population: The Treated Population comprises all participants who received at least one dose of emicizumab.

The number of treated target joint bleeds over the efficacy period is presented here as a calculated annualized bleed rate (ABR) that was annualized for each participant using the following formula: ABR = (number of bleeds/number of days during the efficacy period) x 365.25. A treated target joint bleed was defined as a joint bleed in a target joint, which is a joint location where at least 3 bleeds have occurred over the last 24 weeks prior to study entry or an unresolved target joint (target joint that does not fulfil ≤2 bleeds into this joint within a consecutive 12-month period), and the bleed was directly followed by a hemophilia medication reported to be a "treatment for bleed". The 72-hour rule was implemented: two bleeds of the same type and at the same anatomical location were counted as one bleed if the second bleed occurred within 72 hours from the last treatment for the first bleed. Bleeds due to surgery/procedure were excluded.

Outcome measures

Measure	Emicizumab n=72 Participants Participants with mild and moderate hemophilia A without factor VIII (FVIII) inhibitors were enrolled to receive the emicizumab loading dose regimen (emicizumab 3 mg/kg administered subcutaneously \[SC\] QW for 4 weeks) followed by participant preference of one of the following 3 emicizumab SC maintenance dose regimens: 1.5 mg/kg QW, 3 mg/kg Q2W, or 6 mg/kg Q4W.	Participants ≥5 to <18 Years Old: Emicizumab Participants with mild and moderate hemophilia A without factor VIII (FVIII) inhibitors were enrolled and received the emicizumab loading dose regimen (emicizumab 3 mg/kg administered subcutaneously QW for 4 weeks) followed by participant preference of one of the following 3 emicizumab maintenance dose regimens: 1.5 mg/kg QW, 3 mg/kg Q2W, or 6 mg/kg Q4W.	Participants ≥18 Years Old: Emicizumab Participants with mild and moderate hemophilia A without factor VIII (FVIII) inhibitors were enrolled and received the emicizumab loading dose regimen (emicizumab 3 mg/kg administered subcutaneously QW for 4 weeks) followed by participant preference of one of the following 3 emicizumab maintenance dose regimens: 1.5 mg/kg QW, 3 mg/kg Q2W, or 6 mg/kg Q4W.
Median Calculated Annualized Bleed Rate for Treated Target Joint Bleeds	0.0 Treated target joint bleeds per year Interval 0.0 to 0.0	—	—

SECONDARY outcome

Timeframe: From the day of first emicizumab dose to at least 52 weeks of emicizumab treatment (median [range, min-max] efficacy period: 55.64 [8.6-89.9] weeks)

Population: The Treated Population comprises all participants who received at least one dose of emicizumab.

The number of treated spontaneous bleeds over the efficacy period was estimated as an annualized bleed rate (ABR) using a negative binomial regression model, which accounts for different follow-up times. A treated spontaneous bleed was defined as a treated bleed (bleed directly followed by a hemophilia medication reported to be a "treatment for bleed") with no other known contributing factor such as trauma or procedure/surgery. The 72-hour rule was implemented: two bleeds of the same type and at the same anatomical location were counted as one bleed if the second bleed occurred within 72 hours from the last treatment for the first bleed. Bleeds due to surgery/procedure were excluded.

Outcome measures

Measure	Emicizumab n=72 Participants Participants with mild and moderate hemophilia A without factor VIII (FVIII) inhibitors were enrolled to receive the emicizumab loading dose regimen (emicizumab 3 mg/kg administered subcutaneously \[SC\] QW for 4 weeks) followed by participant preference of one of the following 3 emicizumab SC maintenance dose regimens: 1.5 mg/kg QW, 3 mg/kg Q2W, or 6 mg/kg Q4W.	Participants ≥5 to <18 Years Old: Emicizumab Participants with mild and moderate hemophilia A without factor VIII (FVIII) inhibitors were enrolled and received the emicizumab loading dose regimen (emicizumab 3 mg/kg administered subcutaneously QW for 4 weeks) followed by participant preference of one of the following 3 emicizumab maintenance dose regimens: 1.5 mg/kg QW, 3 mg/kg Q2W, or 6 mg/kg Q4W.	Participants ≥18 Years Old: Emicizumab Participants with mild and moderate hemophilia A without factor VIII (FVIII) inhibitors were enrolled and received the emicizumab loading dose regimen (emicizumab 3 mg/kg administered subcutaneously QW for 4 weeks) followed by participant preference of one of the following 3 emicizumab maintenance dose regimens: 1.5 mg/kg QW, 3 mg/kg Q2W, or 6 mg/kg Q4W.
Model-Based Annualized Bleed Rate for Treated Spontaneous Bleeds	0.2 Treated spontaneous bleeds Interval 0.11 to 0.33	—	—

SECONDARY outcome

Timeframe: From the day of first emicizumab dose to at least 52 weeks of emicizumab treatment (median [range, min-max] efficacy period: 55.64 [8.6-89.9] weeks)

Population: The Treated Population comprises all participants who received at least one dose of emicizumab.

The number of treated spontaneous bleeds over the efficacy period is presented here as a calculated annualized bleed rate (ABR) that was annualized for each participant using the following formula: ABR = (number of bleeds/number of days during the efficacy period) x 365.25. A treated spontaneous bleed was defined as a treated bleed (bleed directly followed by a hemophilia medication reported to be a "treatment for bleed") with no other known contributing factor such as trauma or procedure/surgery. The 72-hour rule was implemented: two bleeds of the same type and at the same anatomical location were counted as one bleed if the second bleed occurred within 72 hours from the last treatment for the first bleed. Bleeds due to surgery/procedure were excluded.

Outcome measures

Measure	Emicizumab n=72 Participants Participants with mild and moderate hemophilia A without factor VIII (FVIII) inhibitors were enrolled to receive the emicizumab loading dose regimen (emicizumab 3 mg/kg administered subcutaneously \[SC\] QW for 4 weeks) followed by participant preference of one of the following 3 emicizumab SC maintenance dose regimens: 1.5 mg/kg QW, 3 mg/kg Q2W, or 6 mg/kg Q4W.	Participants ≥5 to <18 Years Old: Emicizumab Participants with mild and moderate hemophilia A without factor VIII (FVIII) inhibitors were enrolled and received the emicizumab loading dose regimen (emicizumab 3 mg/kg administered subcutaneously QW for 4 weeks) followed by participant preference of one of the following 3 emicizumab maintenance dose regimens: 1.5 mg/kg QW, 3 mg/kg Q2W, or 6 mg/kg Q4W.	Participants ≥18 Years Old: Emicizumab Participants with mild and moderate hemophilia A without factor VIII (FVIII) inhibitors were enrolled and received the emicizumab loading dose regimen (emicizumab 3 mg/kg administered subcutaneously QW for 4 weeks) followed by participant preference of one of the following 3 emicizumab maintenance dose regimens: 1.5 mg/kg QW, 3 mg/kg Q2W, or 6 mg/kg Q4W.
Mean Calculated Annualized Bleed Rate for Treated Spontaneous Bleeds	0.3 Treated spontaneous bleeds Interval 0.0 to 4.23	—	—

SECONDARY outcome

Timeframe: From the day of first emicizumab dose to at least 52 weeks of emicizumab treatment (median [range, min-max] efficacy period: 55.64 [8.6-89.9] weeks)

Population: The Treated Population comprises all participants who received at least one dose of emicizumab.

The number of treated spontaneous bleeds over the efficacy period is presented here as a calculated annualized bleed rate (ABR) that was annualized for each participant using the following formula: ABR = (number of bleeds/number of days during the efficacy period) x 365.25. A treated spontaneous bleed was defined as a treated bleed (bleed directly followed by a hemophilia medication reported to be a "treatment for bleed") with no other known contributing factor such as trauma or procedure/surgery. The 72-hour rule was implemented: two bleeds of the same type and at the same anatomical location were counted as one bleed if the second bleed occurred within 72 hours from the last treatment for the first bleed. Bleeds due to surgery/procedure were excluded.

Outcome measures

Measure	Emicizumab n=72 Participants Participants with mild and moderate hemophilia A without factor VIII (FVIII) inhibitors were enrolled to receive the emicizumab loading dose regimen (emicizumab 3 mg/kg administered subcutaneously \[SC\] QW for 4 weeks) followed by participant preference of one of the following 3 emicizumab SC maintenance dose regimens: 1.5 mg/kg QW, 3 mg/kg Q2W, or 6 mg/kg Q4W.	Participants ≥5 to <18 Years Old: Emicizumab Participants with mild and moderate hemophilia A without factor VIII (FVIII) inhibitors were enrolled and received the emicizumab loading dose regimen (emicizumab 3 mg/kg administered subcutaneously QW for 4 weeks) followed by participant preference of one of the following 3 emicizumab maintenance dose regimens: 1.5 mg/kg QW, 3 mg/kg Q2W, or 6 mg/kg Q4W.	Participants ≥18 Years Old: Emicizumab Participants with mild and moderate hemophilia A without factor VIII (FVIII) inhibitors were enrolled and received the emicizumab loading dose regimen (emicizumab 3 mg/kg administered subcutaneously QW for 4 weeks) followed by participant preference of one of the following 3 emicizumab maintenance dose regimens: 1.5 mg/kg QW, 3 mg/kg Q2W, or 6 mg/kg Q4W.
Median Calculated Annualized Bleed Rate for Treated Spontaneous Bleeds	0.0 Treated spontaneous bleeds Interval 0.0 to 0.0	—	—

SECONDARY outcome

Timeframe: Baseline (Week 1), Weeks 13, 25, 37 and 49, and every 12 weeks thereafter until Study Completion/Discontinuation Visit (up to approximately 48 months)

CATCH = Comprehensive Assessment Tool of Challenges in Hemophilia

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: Baseline (Week 1), Weeks 13, 25, 37 and 49, and every 12 weeks thereafter until Study Completion/Discontinuation Visit (up to approximately 48 months)

CATCH = Comprehensive Assessment Tool of Challenges in Hemophilia

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: Baseline (Week 1), Weeks 13, 25, 37 and 49, and every 12 weeks thereafter until Study Completion/Discontinuation Visit (up to approximately 48 months)

CATCH = Comprehensive Assessment Tool of Challenges in Hemophilia

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: Baseline (Week 1), Weeks 13, 25, 37 and 49, and every 12 weeks thereafter until Study Completion/Discontinuation Visit (up to approximately 48 months)

CATCH = Comprehensive Assessment Tool of Challenges in Hemophilia

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: Baseline (Week 1), Weeks 13, 25, 37 and 49, and every 12 weeks thereafter until Study Completion/Discontinuation Visit (up to approximately 48 months)

CATCH = Comprehensive Assessment Tool of Challenges in Hemophilia

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: Baseline (Week 1), Weeks 13, 25, 37 and 49, and every 12 weeks thereafter until Study Completion/Discontinuation Visit (up to approximately 48 months)

CATCH = Comprehensive Assessment Tool of Challenges in Hemophilia

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: Baseline (Week 1), Weeks 13, 25, 37 and 49, and every 12 weeks thereafter until Study Completion/Discontinuation Visit (up to approximately 48 months)

CATCH = Comprehensive Assessment Tool of Challenges in Hemophilia

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: Baseline (Week 1), Weeks 13, 25, 37 and 49, and every 12 weeks thereafter until Study Completion/Discontinuation Visit (up to approximately 48 months)

CATCH = Comprehensive Assessment Tool of Challenges in Hemophilia

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: Baseline (Week 1), Weeks 13, 25, 37 and 49, and every 12 weeks thereafter until Study Completion/Discontinuation Visit (up to approximately 48 months)

CATCH = Comprehensive Assessment Tool of Challenges in Hemophilia

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: Baseline (Week 1), Weeks 13, 25, 37 and 49, and every 12 weeks thereafter until Study Completion/Discontinuation Visit (up to approximately 48 months)

CATCH = Comprehensive Assessment Tool of Challenges in Hemophilia

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: Baseline (Week 1), Weeks 13, 25, 37 and 49, and every 12 weeks thereafter until Study Completion/Discontinuation Visit (up to approximately 48 months)

CATCH = Comprehensive Assessment Tool of Challenges in Hemophilia

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: Baseline (Week 1), Weeks 13, 25, 37 and 49, and every 12 weeks thereafter until Study Completion/Discontinuation Visit (up to approximately 48 months)

CATCH = Comprehensive Assessment Tool of Challenges in Hemophilia

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: Baseline (Week 1), Weeks 13, 25, 37 and 49, and every 12 weeks thereafter until Study Completion/Discontinuation Visit (up to approximately 48 months)

CATCH = Comprehensive Assessment Tool of Challenges in Hemophilia

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: Baseline (Week 1), Weeks 13, 25, 37 and 49, and every 12 weeks thereafter until Study Completion/Discontinuation Visit (up to approximately 48 months)

CATCH = Comprehensive Assessment Tool of Challenges in Hemophilia

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: Baseline (Week 1), Weeks 13, 25, 37 and 49, and every 12 weeks thereafter until Study Completion/Discontinuation Visit (up to approximately 48 months)

CATCH = Comprehensive Assessment Tool of Challenges in Hemophilia

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: Baseline (Week 1), Weeks 13, 25, 37 and 49, and every 12 weeks thereafter until Study Completion/Discontinuation Visit (up to approximately 48 months)

CATCH = Comprehensive Assessment Tool of Challenges in Hemophilia

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: Baseline (Week 1), Weeks 13, 25, 37 and 49, and every 12 weeks thereafter until Study Completion/Discontinuation Visit (up to approximately 48 months)

CATCH = Comprehensive Assessment Tool of Challenges in Hemophilia

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: Baseline (Week 1), Weeks 13, 25, 37 and 49, and every 12 weeks thereafter until Study Completion/Discontinuation Visit (up to approximately 48 months)

CATCH = Comprehensive Assessment Tool of Challenges in Hemophilia

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: Baseline (Week 1), Weeks 13, 25, 37 and 49, and every 12 weeks thereafter until Study Completion/Discontinuation Visit (up to approximately 48 months)

CATCH = Comprehensive Assessment Tool of Challenges in Hemophilia

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: Baseline (Week 1), Weeks 13, 25, 37 and 49, and every 12 weeks thereafter until Study Completion/Discontinuation Visit (up to approximately 48 months)

CATCH = Comprehensive Assessment Tool of Challenges in Hemophilia

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: Baseline (Week 1), Weeks 13, 25, 37 and 49, and every 12 weeks thereafter until Study Completion/Discontinuation Visit (up to approximately 48 months)

CATCH = Comprehensive Assessment Tool of Challenges in Hemophilia

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: Week 17

Population: The analysis population included all treated participants 12 years of age or older who provided a response to the survey at Week 17.

The Emicizumab Preference Survey is a fit-for-purpose questionnaire developed by the sponsor to record the participant's preference for treatment with subcutaneous (SC) emicizumab, intravenous (IV) factor VIIII (FVIII), or no preference. The 95% confidence intervals were calculated using the Pearson-Clopper method.

Outcome measures

Measure	Emicizumab n=52 Participants Participants with mild and moderate hemophilia A without factor VIII (FVIII) inhibitors were enrolled to receive the emicizumab loading dose regimen (emicizumab 3 mg/kg administered subcutaneously \[SC\] QW for 4 weeks) followed by participant preference of one of the following 3 emicizumab SC maintenance dose regimens: 1.5 mg/kg QW, 3 mg/kg Q2W, or 6 mg/kg Q4W.	Participants ≥5 to <18 Years Old: Emicizumab Participants with mild and moderate hemophilia A without factor VIII (FVIII) inhibitors were enrolled and received the emicizumab loading dose regimen (emicizumab 3 mg/kg administered subcutaneously QW for 4 weeks) followed by participant preference of one of the following 3 emicizumab maintenance dose regimens: 1.5 mg/kg QW, 3 mg/kg Q2W, or 6 mg/kg Q4W.	Participants ≥18 Years Old: Emicizumab Participants with mild and moderate hemophilia A without factor VIII (FVIII) inhibitors were enrolled and received the emicizumab loading dose regimen (emicizumab 3 mg/kg administered subcutaneously QW for 4 weeks) followed by participant preference of one of the following 3 emicizumab maintenance dose regimens: 1.5 mg/kg QW, 3 mg/kg Q2W, or 6 mg/kg Q4W.
Percentage of Participants Who Prefer Emicizumab SC Treatment, Their Previous Hemophilia IV Treatment, or Have No Preference, as Assessed Through Use of the Emicizumab Preference Survey at Week 17 Prefer the New Study Drug Treatment (Emicizumab SC)	96.2 Percentage of participants Interval 86.79 to 99.53	—	—
Percentage of Participants Who Prefer Emicizumab SC Treatment, Their Previous Hemophilia IV Treatment, or Have No Preference, as Assessed Through Use of the Emicizumab Preference Survey at Week 17 Prefer My Old Hemophilia Treatment (IV)	1.9 Percentage of participants Interval 0.05 to 10.26	—	—
Percentage of Participants Who Prefer Emicizumab SC Treatment, Their Previous Hemophilia IV Treatment, or Have No Preference, as Assessed Through Use of the Emicizumab Preference Survey at Week 17 Have No Preference	1.9 Percentage of participants Interval 0.05 to 10.26	—	—

SECONDARY outcome

Timeframe: Week 17

Population: The analysis population included all caregivers of treated participants less than 18 years of age and who provided a response to the survey at Week 17.

The Emicizumab Preference Survey is a fit-for-purpose questionnaire developed by the sponsor to record the caregiver's preference for their child's treatment with subcutaneous (SC) emicizumab, intravenous (IV) factor VIIII (FVIII), or no preference. The 95% confidence intervals were calculated using the Pearson-Clopper method.

Outcome measures

Measure	Emicizumab n=28 Participants Participants with mild and moderate hemophilia A without factor VIII (FVIII) inhibitors were enrolled to receive the emicizumab loading dose regimen (emicizumab 3 mg/kg administered subcutaneously \[SC\] QW for 4 weeks) followed by participant preference of one of the following 3 emicizumab SC maintenance dose regimens: 1.5 mg/kg QW, 3 mg/kg Q2W, or 6 mg/kg Q4W.	Participants ≥5 to <18 Years Old: Emicizumab Participants with mild and moderate hemophilia A without factor VIII (FVIII) inhibitors were enrolled and received the emicizumab loading dose regimen (emicizumab 3 mg/kg administered subcutaneously QW for 4 weeks) followed by participant preference of one of the following 3 emicizumab maintenance dose regimens: 1.5 mg/kg QW, 3 mg/kg Q2W, or 6 mg/kg Q4W.	Participants ≥18 Years Old: Emicizumab Participants with mild and moderate hemophilia A without factor VIII (FVIII) inhibitors were enrolled and received the emicizumab loading dose regimen (emicizumab 3 mg/kg administered subcutaneously QW for 4 weeks) followed by participant preference of one of the following 3 emicizumab maintenance dose regimens: 1.5 mg/kg QW, 3 mg/kg Q2W, or 6 mg/kg Q4W.
Percentage of Caregivers Who Prefer Emicizumab SC Treatment, Their Child's Previous Hemophilia IV Treatment, or Have No Preference, as Assessed Through Use of the Emicizumab Preference Survey at Week 17 Prefer My Child's Old Hemophilia Treatment (IV)	3.6 Percentage of caregivers Interval 0.09 to 18.35	—	—
Percentage of Caregivers Who Prefer Emicizumab SC Treatment, Their Child's Previous Hemophilia IV Treatment, or Have No Preference, as Assessed Through Use of the Emicizumab Preference Survey at Week 17 Prefer the New Study Drug Treatment (Emicizumab SC)	85.7 Percentage of caregivers Interval 67.33 to 95.97	—	—
Percentage of Caregivers Who Prefer Emicizumab SC Treatment, Their Child's Previous Hemophilia IV Treatment, or Have No Preference, as Assessed Through Use of the Emicizumab Preference Survey at Week 17 Have No Preference	10.7 Percentage of caregivers Interval 2.27 to 28.23	—	—

SECONDARY outcome

Timeframe: Days -7 to -1, Weeks 25, 49, and every 24 weeks thereafter until Study Completion/Discontinuation Visit (up to approximately 48 months)

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: Baseline (Weeks 1-2) and Weeks 13 (Weeks 12-13 period), 25 (Weeks 24-25 period), 37 (Weeks 36-37 period), and 49 (Weeks 48-49 period)

Population: The analysis population is treated participants ≥5 years old, further subdivided into analysis groups of ≥5 to \<18 years old and ≥18 years old. The number analyzed for change from baseline (BL) are participants with data that met the compliance criterion at BL and the specified timepoint (i.e., wearing device ≥8 hours/day for ≥8 days per period); if it was not met, they were excluded for each noncompliant timepoint. The overall number analyzed had valid data for at least the BL timepoint.

For physical activity assessment, participants ≥5 years of age were instructed to wear the study accelerometry device on the wrist continuously (24 hours/day) every day for the designated 2-week periods during the study. A participant was considered to be compliant with the physical activity assessments if they wore the study device continuously (≥8 hours/day) every day for at least 8 days of each of the designated 2-week periods during the study. If this compliance criterion was not reached at a specific timepoint the participant was not included in the analysis of the designated 2-week periods where compliance was not reached. Daily measurements were averaged over the 14-days timepoint. Activity count was a measure of the acceleration measured by the device. The daily peak activity duration was defined as the sum of moderate to vigorous activity per day (in minutes).

Outcome measures

Measure	Emicizumab n=61 Participants Participants with mild and moderate hemophilia A without factor VIII (FVIII) inhibitors were enrolled to receive the emicizumab loading dose regimen (emicizumab 3 mg/kg administered subcutaneously \[SC\] QW for 4 weeks) followed by participant preference of one of the following 3 emicizumab SC maintenance dose regimens: 1.5 mg/kg QW, 3 mg/kg Q2W, or 6 mg/kg Q4W.	Participants ≥5 to <18 Years Old: Emicizumab n=22 Participants Participants with mild and moderate hemophilia A without factor VIII (FVIII) inhibitors were enrolled and received the emicizumab loading dose regimen (emicizumab 3 mg/kg administered subcutaneously QW for 4 weeks) followed by participant preference of one of the following 3 emicizumab maintenance dose regimens: 1.5 mg/kg QW, 3 mg/kg Q2W, or 6 mg/kg Q4W.	Participants ≥18 Years Old: Emicizumab n=39 Participants Participants with mild and moderate hemophilia A without factor VIII (FVIII) inhibitors were enrolled and received the emicizumab loading dose regimen (emicizumab 3 mg/kg administered subcutaneously QW for 4 weeks) followed by participant preference of one of the following 3 emicizumab maintenance dose regimens: 1.5 mg/kg QW, 3 mg/kg Q2W, or 6 mg/kg Q4W.
Change From Baseline in Mean Daily Peak Activity Duration Over Time Baseline (BL) - Value at Visit	130 Minutes per day Standard Deviation 56	168 Minutes per day Standard Deviation 66	108 Minutes per day Standard Deviation 35
Change From Baseline in Mean Daily Peak Activity Duration Over Time Change from BL at Week 13	-2 Minutes per day Standard Deviation 34	-4 Minutes per day Standard Deviation 42	-1 Minutes per day Standard Deviation 29
Change From Baseline in Mean Daily Peak Activity Duration Over Time Change from BL at Week 25	4 Minutes per day Standard Deviation 53	-4 Minutes per day Standard Deviation 74	8 Minutes per day Standard Deviation 37
Change From Baseline in Mean Daily Peak Activity Duration Over Time Change from BL at Week 37	14 Minutes per day Standard Deviation 55	17 Minutes per day Standard Deviation 73	12 Minutes per day Standard Deviation 40
Change From Baseline in Mean Daily Peak Activity Duration Over Time Change from BL at Week 49	17 Minutes per day Standard Deviation 40	19 Minutes per day Standard Deviation 51	16 Minutes per day Standard Deviation 34

SECONDARY outcome

Timeframe: Baseline (Weeks 1-2) and Weeks 13 (Weeks 12-13), 25 (Weeks 24-25), 37 (Weeks 36-37), and 49 (Weeks 48-49)

Population: The analysis population is treated participants ≥5 years old, further subdivided into analysis groups of ≥5 to \<18 years old and ≥18 years old. The number analyzed for change from baseline (BL) are participants with data that met the compliance criterion at BL and the specified timepoint (i.e., wearing device ≥8 hours/day for ≥8 days per period); if it was not met, they were excluded for each noncompliant timepoint. The overall number analyzed had valid data for at least the BL timepoint.

For physical activity assessment, participants ≥5 years of age were instructed to wear the study accelerometry device on the wrist continuously (24 hours/day) every day for the designated 2-week periods during the study. A participant was considered to be compliant with the physical activity assessments if they wore the study device continuously (≥8 hours/day) every day for at least 8 days of each of the designated 2-week periods during the study. If this compliance criterion was not reached at a specific timepoint the participant was not included in the analysis of the designated 2-week periods where compliance was not reached. Daily measurements were averaged over the 14-days timepoint. Activity count was a measure of the acceleration measured by the device.

Outcome measures

Measure	Emicizumab n=61 Participants Participants with mild and moderate hemophilia A without factor VIII (FVIII) inhibitors were enrolled to receive the emicizumab loading dose regimen (emicizumab 3 mg/kg administered subcutaneously \[SC\] QW for 4 weeks) followed by participant preference of one of the following 3 emicizumab SC maintenance dose regimens: 1.5 mg/kg QW, 3 mg/kg Q2W, or 6 mg/kg Q4W.	Participants ≥5 to <18 Years Old: Emicizumab n=22 Participants Participants with mild and moderate hemophilia A without factor VIII (FVIII) inhibitors were enrolled and received the emicizumab loading dose regimen (emicizumab 3 mg/kg administered subcutaneously QW for 4 weeks) followed by participant preference of one of the following 3 emicizumab maintenance dose regimens: 1.5 mg/kg QW, 3 mg/kg Q2W, or 6 mg/kg Q4W.	Participants ≥18 Years Old: Emicizumab n=39 Participants Participants with mild and moderate hemophilia A without factor VIII (FVIII) inhibitors were enrolled and received the emicizumab loading dose regimen (emicizumab 3 mg/kg administered subcutaneously QW for 4 weeks) followed by participant preference of one of the following 3 emicizumab maintenance dose regimens: 1.5 mg/kg QW, 3 mg/kg Q2W, or 6 mg/kg Q4W.
Change From Baseline in Mean Daily Step Count Over Time Baseline (BL) - Value at Visit	6441 Step count per day Standard Deviation 2617	6755 Step count per day Standard Deviation 3168	6263 Step count per day Standard Deviation 2276
Change From Baseline in Mean Daily Step Count Over Time Change from BL at Week 13	44 Step count per day Standard Deviation 1846	-18 Step count per day Standard Deviation 2361	87 Step count per day Standard Deviation 1423
Change From Baseline in Mean Daily Step Count Over Time Change from BL at Week 25	119 Step count per day Standard Deviation 2977	-69 Step count per day Standard Deviation 4348	225 Step count per day Standard Deviation 1901
Change From Baseline in Mean Daily Step Count Over Time Change from BL at Week 37	767 Step count per day Standard Deviation 3189	1403 Step count per day Standard Deviation 4544	356 Step count per day Standard Deviation 1852
Change From Baseline in Mean Daily Step Count Over Time Change from BL at Week 49	1216 Step count per day Standard Deviation 2440	1740 Step count per day Standard Deviation 3130	919 Step count per day Standard Deviation 1945

SECONDARY outcome

Timeframe: Baseline (Week 1), Weeks 5, 9, 13, 17, 21, 25, 29, 33, 37, 41, 45, 49, and every 4 weeks thereafter until Study Completion (up to approximately 48 months)

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: Baseline (Week 1), Weeks 5, 9, 13, 17, 21, 25, 29, 33, 37, 41, 45, 49, and every 4 weeks thereafter until Study Completion (up to approximately 48 months)

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: Baseline (Week 1), Weeks 5, 9, 13, 17, 21, 25, 29, 33, 37, 41, 45, 49, and every 4 weeks thereafter until Study Completion (up to approximately 48 months)

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: Baseline (Week 1), Weeks 5, 9, 13, 17, 21, 25, 29, 33, 37, 41, 45, 49, and every 4 weeks thereafter until Study Completion (up to approximately 48 months)

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: Baseline (Week 1), Weeks 5, 9, 13, 17, 21, 25, 29, 33, 37, 41, 45, 49, and every 4 weeks thereafter until Study Completion (up to approximately 48 months)

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: Baseline (Day 1) and monthly (on days of menstruation) until Study Completion (up to approximately 48 months)

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: From Screening (Day -28 to -1) to Study Completion/Discontinuation Visit (up to approximately 48 months)

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: From Screening (Day -28 to -1) to Study Completion/Discontinuation Visit (up to approximately 48 months)

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: From Screening (Day -28 to -1) to Study Completion/Discontinuation Visit (up to approximately 48 months)

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: From Screening (Day -28 to -1) to Study Completion/Discontinuation Visit (up to approximately 48 months)

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: From Screening (Day -28 to -1) to Study Completion/Discontinuation Visit (up to approximately 48 months)

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: From Screening (Day -28 to -1) to Study Completion/Discontinuation Visit (up to approximately 48 months)

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: From Screening (Day -28 to -1) to Study Completion/Discontinuation Visit (up to approximately 48 months)

Laboratory parameters for hematology and blood chemistry will be measured and compared with a standard reference range. Values outside the standard reference range are considered abnormalities. Not every laboratory abnormality qualifies as an adverse event. A laboratory test result will be reported as an adverse event if it meets any of the following criteria: is accompanied by clinical symptoms; results in a change in study treatment or a medical intervention; or is clinically significant in the investigator's judgment.

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: Baseline, Weeks 1, 2, 3, 4, 5, 9, 13, 17, 21, 25, 29, 33, 37, 41, 45, and 49, and every 12 weeks thereafter until Study Completion/Discontinuation Visit (up to approximately 48 months)

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: Baseline, Weeks 1, 2, 3, 4, 5, 9, 13, 17, 21, 25, 29, 33, 37, 41, 45, and 49, and every 12 weeks thereafter until Study Completion/Discontinuation Visit (up to approximately 48 months)

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: Baseline, Weeks 1, 2, 3, 4, 5, 9, 13, 17, 21, 25, 29, 33, 37, 41, 45, and 49, and every 12 weeks thereafter until Study Completion/Discontinuation Visit (up to approximately 48 months)

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: Baseline, Weeks 1, 2, 3, 4, 5, 9, 13, 17, 21, 25, 29, 33, 37, 41, 45, and 49, and every 12 weeks thereafter until Study Completion/Discontinuation Visit (up to approximately 48 months)

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: Baseline, Weeks 1, 2, 3, 4, 5, 9, 13, 17, 21, 25, 29, 33, 37, 41, 45, and 49, and every 12 weeks thereafter until Study Completion/Discontinuation Visit (up to approximately 48 months)

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: Baseline, Weeks 5, 25, and 49, and every 12 weeks thereafter until Study Completion/Discontinuation Visit (up to approximately 48 months)

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: Baseline, Weeks 5, 25, and 49, and every 12 weeks thereafter until Study Completion/Discontinuation Visit (up to approximately 48 months)

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: Pre-dose at Weeks 1, 2, 3, 4, 5, 9, 13, 17, 21, 25, 29, 33, 37, 41, 45, and 49, and every 12 weeks thereafter until study completion/discontinuation (up to approximately 48 months)

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: Pre-dose at Baseline (Week 1) and Weeks 5, 13, 25, 33, 41, and 49, and every 12 weeks thereafter until study completion/discontinuation (up to approximately 48 months)

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: Screening (Day -28 to -1) and Weeks 1, 13, 25, 37, and 49, and every 12 weeks thereafter until study completion/discontinuation (up to approximately 48 months)

Outcome measures

Outcome data not reported

Adverse Events

Emicizumab

Serious events: 8 serious events

Other events: 37 other events

Deaths: 0 deaths

Serious adverse events

Measure	Emicizumab n=72 participants at risk Participants with mild and moderate hemophilia A without factor VIII (FVIII) inhibitors were enrolled to receive the emicizumab loading dose regimen (emicizumab 3 mg/kg administered subcutaneously \[SC\] QW for 4 weeks) followed by participant preference of one of the following 3 emicizumab SC maintenance dose regimens: 1.5 mg/kg QW, 3 mg/kg Q2W, or 6 mg/kg Q4W.
Gastrointestinal disorders Abdominal pain	1.4% 1/72 • Number of events 1 • From enrollment until primary completion date cutoff (median [range, min-max] observation period: 55.64 [8.7-89.9] weeks) Adverse events (AEs) are reported in the Treated Population, comprising all participants who received at least one dose of emicizumab. AEs are still being collected until the end of the study and the results will be updated within 1 year of the date of final data collection.
Gastrointestinal disorders Mallory-Weiss syndrome	1.4% 1/72 • Number of events 1 • From enrollment until primary completion date cutoff (median [range, min-max] observation period: 55.64 [8.7-89.9] weeks) Adverse events (AEs) are reported in the Treated Population, comprising all participants who received at least one dose of emicizumab. AEs are still being collected until the end of the study and the results will be updated within 1 year of the date of final data collection.
Infections and infestations COVID-19	1.4% 1/72 • Number of events 1 • From enrollment until primary completion date cutoff (median [range, min-max] observation period: 55.64 [8.7-89.9] weeks) Adverse events (AEs) are reported in the Treated Population, comprising all participants who received at least one dose of emicizumab. AEs are still being collected until the end of the study and the results will be updated within 1 year of the date of final data collection.
Infections and infestations Diverticulitis	1.4% 1/72 • Number of events 1 • From enrollment until primary completion date cutoff (median [range, min-max] observation period: 55.64 [8.7-89.9] weeks) Adverse events (AEs) are reported in the Treated Population, comprising all participants who received at least one dose of emicizumab. AEs are still being collected until the end of the study and the results will be updated within 1 year of the date of final data collection.
Infections and infestations Erysipelas	1.4% 1/72 • Number of events 1 • From enrollment until primary completion date cutoff (median [range, min-max] observation period: 55.64 [8.7-89.9] weeks) Adverse events (AEs) are reported in the Treated Population, comprising all participants who received at least one dose of emicizumab. AEs are still being collected until the end of the study and the results will be updated within 1 year of the date of final data collection.
Injury, poisoning and procedural complications Concussion	1.4% 1/72 • Number of events 1 • From enrollment until primary completion date cutoff (median [range, min-max] observation period: 55.64 [8.7-89.9] weeks) Adverse events (AEs) are reported in the Treated Population, comprising all participants who received at least one dose of emicizumab. AEs are still being collected until the end of the study and the results will be updated within 1 year of the date of final data collection.
Injury, poisoning and procedural complications Contusion	1.4% 1/72 • Number of events 1 • From enrollment until primary completion date cutoff (median [range, min-max] observation period: 55.64 [8.7-89.9] weeks) Adverse events (AEs) are reported in the Treated Population, comprising all participants who received at least one dose of emicizumab. AEs are still being collected until the end of the study and the results will be updated within 1 year of the date of final data collection.
Injury, poisoning and procedural complications Foreign body in gastrointestinal tract	1.4% 1/72 • Number of events 1 • From enrollment until primary completion date cutoff (median [range, min-max] observation period: 55.64 [8.7-89.9] weeks) Adverse events (AEs) are reported in the Treated Population, comprising all participants who received at least one dose of emicizumab. AEs are still being collected until the end of the study and the results will be updated within 1 year of the date of final data collection.
Metabolism and nutrition disorders Hyperglycaemia	1.4% 1/72 • Number of events 1 • From enrollment until primary completion date cutoff (median [range, min-max] observation period: 55.64 [8.7-89.9] weeks) Adverse events (AEs) are reported in the Treated Population, comprising all participants who received at least one dose of emicizumab. AEs are still being collected until the end of the study and the results will be updated within 1 year of the date of final data collection.
Renal and urinary disorders Renal colic	1.4% 1/72 • Number of events 1 • From enrollment until primary completion date cutoff (median [range, min-max] observation period: 55.64 [8.7-89.9] weeks) Adverse events (AEs) are reported in the Treated Population, comprising all participants who received at least one dose of emicizumab. AEs are still being collected until the end of the study and the results will be updated within 1 year of the date of final data collection.

Other adverse events

Measure	Emicizumab n=72 participants at risk Participants with mild and moderate hemophilia A without factor VIII (FVIII) inhibitors were enrolled to receive the emicizumab loading dose regimen (emicizumab 3 mg/kg administered subcutaneously \[SC\] QW for 4 weeks) followed by participant preference of one of the following 3 emicizumab SC maintenance dose regimens: 1.5 mg/kg QW, 3 mg/kg Q2W, or 6 mg/kg Q4W.
General disorders Injection site reaction	16.7% 12/72 • Number of events 29 • From enrollment until primary completion date cutoff (median [range, min-max] observation period: 55.64 [8.7-89.9] weeks) Adverse events (AEs) are reported in the Treated Population, comprising all participants who received at least one dose of emicizumab. AEs are still being collected until the end of the study and the results will be updated within 1 year of the date of final data collection.
Musculoskeletal and connective tissue disorders Arthralgia	15.3% 11/72 • Number of events 11 • From enrollment until primary completion date cutoff (median [range, min-max] observation period: 55.64 [8.7-89.9] weeks) Adverse events (AEs) are reported in the Treated Population, comprising all participants who received at least one dose of emicizumab. AEs are still being collected until the end of the study and the results will be updated within 1 year of the date of final data collection.
Musculoskeletal and connective tissue disorders Pain in extremity	6.9% 5/72 • Number of events 5 • From enrollment until primary completion date cutoff (median [range, min-max] observation period: 55.64 [8.7-89.9] weeks) Adverse events (AEs) are reported in the Treated Population, comprising all participants who received at least one dose of emicizumab. AEs are still being collected until the end of the study and the results will be updated within 1 year of the date of final data collection.
Nervous system disorders Headache	16.7% 12/72 • Number of events 16 • From enrollment until primary completion date cutoff (median [range, min-max] observation period: 55.64 [8.7-89.9] weeks) Adverse events (AEs) are reported in the Treated Population, comprising all participants who received at least one dose of emicizumab. AEs are still being collected until the end of the study and the results will be updated within 1 year of the date of final data collection.
Infections and infestations COVID-19	5.6% 4/72 • Number of events 4 • From enrollment until primary completion date cutoff (median [range, min-max] observation period: 55.64 [8.7-89.9] weeks) Adverse events (AEs) are reported in the Treated Population, comprising all participants who received at least one dose of emicizumab. AEs are still being collected until the end of the study and the results will be updated within 1 year of the date of final data collection.
Infections and infestations Nasopharyngitis	6.9% 5/72 • Number of events 8 • From enrollment until primary completion date cutoff (median [range, min-max] observation period: 55.64 [8.7-89.9] weeks) Adverse events (AEs) are reported in the Treated Population, comprising all participants who received at least one dose of emicizumab. AEs are still being collected until the end of the study and the results will be updated within 1 year of the date of final data collection.
Injury, poisoning and procedural complications Fall	5.6% 4/72 • Number of events 4 • From enrollment until primary completion date cutoff (median [range, min-max] observation period: 55.64 [8.7-89.9] weeks) Adverse events (AEs) are reported in the Treated Population, comprising all participants who received at least one dose of emicizumab. AEs are still being collected until the end of the study and the results will be updated within 1 year of the date of final data collection.

Additional Information

Medical Communications

Hoffmann-La Roche

Phone: 800-821-8590

Email: genentech@druginfo.com

Results disclosure agreements

• Principal investigator is a sponsor employee The Study being conducted under this Agreement is part of the Overall Study. Investigator is free to publish in reputable journals or to present at professional conferences the results of the Study, but only after the first publication or presentation that involves the Overall Study. The Sponsor may request that Confidential Information be deleted and/or the publication be postponed in order to protect the Sponsor's intellectual property rights.
• Publication restrictions are in place

Restriction type: OTHER