Trial Outcomes & Findings for BAX 326 Pediatric Study (NCT NCT01488994)

NCT ID: NCT01488994

Last Updated: 2021-05-20

Results Overview

• Recruitment status: COMPLETED
• Study phase: PHASE2/PHASE3
• Target enrollment: 23 participants
• Primary outcome timeframe: Throughout study period (approximately 17 months)
• Results posted on: 2021-05-20

Participant Flow

Enrollment was conducted at 11 clinical sites in 6 countries (United Kingdom, Poland, Romania, Russian Federation, Ukraine, India). A total of 23 participants were enrolled in the study. Of these, 11 were < 6 years of age and 12 were 6 to <12 years of age.

Participant milestones

Measure	Pediatric Participants < 6 Years of Age Pediatric participants \< 6 years of age. All pediatric participants underwent pharmacokinetic (PK) assessment followed by prophylactic treatment with the study product. All participants received the same dosing schedule of study product during the study. After a washout period of 5-7 days, participants received an initial infusion of study product at a dose of 75±5 IU/kg for the PK assessment. For the prophylactic regimen, participants were treated with the recommended dose of 50 IU/kg of study product twice weekly ranging from 40-80 IU/kg for 26±1 weeks or for at least 50 EDs to study product, whichever occurred last.	Pediatric Participants 6 to <12 Years of Age Pediatric participants 6 to \< 12 years of age. All pediatric participants underwent pharmacokinetic (PK) assessment followed by prophylactic treatment with the study product. All participants received the same dosing schedule of study product during the study. After a washout period of 5-7 days, participants received an initial infusion of study product at a dose of 75±5 IU/kg for the PK assessment. For the prophylactic regimen, participants were treated with the recommended dose of 50 IU/kg of study product twice weekly ranging from 40-80 IU/kg for 26±1 weeks or for at least 50 EDs to study product, whichever occurred last.
Overall Study STARTED	11	12
Overall Study COMPLETED	11	11
Overall Study NOT COMPLETED	0	1

Reasons for withdrawal

Measure	Pediatric Participants < 6 Years of Age Pediatric participants \< 6 years of age. All pediatric participants underwent pharmacokinetic (PK) assessment followed by prophylactic treatment with the study product. All participants received the same dosing schedule of study product during the study. After a washout period of 5-7 days, participants received an initial infusion of study product at a dose of 75±5 IU/kg for the PK assessment. For the prophylactic regimen, participants were treated with the recommended dose of 50 IU/kg of study product twice weekly ranging from 40-80 IU/kg for 26±1 weeks or for at least 50 EDs to study product, whichever occurred last.	Pediatric Participants 6 to <12 Years of Age Pediatric participants 6 to \< 12 years of age. All pediatric participants underwent pharmacokinetic (PK) assessment followed by prophylactic treatment with the study product. All participants received the same dosing schedule of study product during the study. After a washout period of 5-7 days, participants received an initial infusion of study product at a dose of 75±5 IU/kg for the PK assessment. For the prophylactic regimen, participants were treated with the recommended dose of 50 IU/kg of study product twice weekly ranging from 40-80 IU/kg for 26±1 weeks or for at least 50 EDs to study product, whichever occurred last.
Overall Study Withdrawal by Subject	0	1

Baseline Characteristics

BAX 326 Pediatric Study

Baseline characteristics by cohort

Measure	Pediatric Participants < 6 Years of Age n=11 Participants Pediatric participants \< 6 years of age. All pediatric participants underwent pharmacokinetic (PK) assessment followed by prophylactic treatment with the study product. All participants received the same dosing schedule of study product during the study. After a washout period of 5-7 days, participants received an initial infusion of study product at a dose of 75±5 IU/kg for the PK assessment. For the prophylactic regimen, participants were treated with the recommended dose of 50 IU/kg of study product twice weekly ranging from 40-80 IU/kg for 26±1 weeks or for at least 50 EDs to study product, whichever occurred last.	Pediatric Participants 6 to <12 Years of Age n=12 Participants Pediatric participants 6 to \<12 years of age. All pediatric participants underwent pharmacokinetic (PK) assessment followed by prophylactic treatment with the study product. All participants received the same dosing schedule of study product during the study. After a washout period of 5-7 days, participants received an initial infusion of study product at a dose of 75±5 IU/kg for the PK assessment. For the prophylactic regimen, participants were treated with the recommended dose of 50 IU/kg of study product twice weekly ranging from 40-80 IU/kg for 26±1 weeks or for at least 50 EDs to study product, whichever occurred last.	Total n=23 Participants Total of all reporting groups
Age, Continuous	3.83 years n=5 Participants	9.8 years n=7 Participants	6.94 years n=5 Participants
Sex: Female, Male Female	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Sex: Female, Male Male	11 Participants n=5 Participants	12 Participants n=7 Participants	23 Participants n=5 Participants

PRIMARY outcome

Timeframe: Throughout study period (approximately 17 months)

Population: Full analysis set

Outcome measures

Measure	Pediatric Participants < 6 Years of Age n=11 Participants Pediatric participants \< 6 years of age. All pediatric participants underwent pharmacokinetic (PK) assessment followed by prophylactic treatment with the study product. All participants received the same dosing schedule of study product during the study. After a washout period of 5-7 days, participants received an initial infusion of study product at a dose of 75±5 IU/kg for the PK assessment. For the prophylactic regimen, participants were treated with the recommended dose of 50 IU/kg of study product twice weekly ranging from 40-80 IU/kg for 26±1 weeks or for at least 50 EDs to study product, whichever occurred last.	Pediatric Participants 6 to < 12 Years of Age n=12 Participants Pediatric participants 6 to \< 12 years of age. All pediatric participants underwent pharmacokinetic (PK) assessment followed by prophylactic treatment with the study product. All participants received the same dosing schedule of study product during the study. After a washout period of 5-7 days, participants received an initial infusion of study product at a dose of 75±5 IU/kg for the PK assessment. For the prophylactic regimen, participants were treated with the recommended dose of 50 IU/kg of study product twice weekly ranging from 40-80 IU/kg for 26±1 weeks or for at least 50 EDs to study product, whichever occurred last.	Full Analysis Set n=23 Participants Comprised of all participants who received at least one infusion of study product
Adverse Events (AEs) Possibly or Probably Related to BAX326	0 AEs considered related to BAX326	0 AEs considered related to BAX326	0 AEs considered related to BAX326

SECONDARY outcome

Timeframe: Within 30 mins pre-infusion and 4 post-infusion timepoints

Population: On completion of the study, prospective changes to the planned statistical analysis were made not to analyze AUC 0-72h due to the different time points for the last PK blood sample. Only total AUC \[i.e. AUC 0-infinity\] was included in the PK analysis.

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: Within 30 mins pre-infusion and 4 post-infusion timepoints. Refer to Population Description below for more details.

Population: All participants randomized to 2 groups: Group 1 - BAX326 infusion in the morning PK timepoints: Pre-infusion and post-infusion at 15-30 minutes, then 7, 28 and 52 hours Group 2 - BAX326 infusion in the afternoon PK timepoints: Pre-infusion and post-infusion of 15-30 minutes, then 4, 24 and 69 hours

Outcome measures

Measure	Pediatric Participants < 6 Years of Age n=11 Participants Pediatric participants \< 6 years of age. All pediatric participants underwent pharmacokinetic (PK) assessment followed by prophylactic treatment with the study product. All participants received the same dosing schedule of study product during the study. After a washout period of 5-7 days, participants received an initial infusion of study product at a dose of 75±5 IU/kg for the PK assessment. For the prophylactic regimen, participants were treated with the recommended dose of 50 IU/kg of study product twice weekly ranging from 40-80 IU/kg for 26±1 weeks or for at least 50 EDs to study product, whichever occurred last.	Pediatric Participants 6 to < 12 Years of Age n=12 Participants Pediatric participants 6 to \< 12 years of age. All pediatric participants underwent pharmacokinetic (PK) assessment followed by prophylactic treatment with the study product. All participants received the same dosing schedule of study product during the study. After a washout period of 5-7 days, participants received an initial infusion of study product at a dose of 75±5 IU/kg for the PK assessment. For the prophylactic regimen, participants were treated with the recommended dose of 50 IU/kg of study product twice weekly ranging from 40-80 IU/kg for 26±1 weeks or for at least 50 EDs to study product, whichever occurred last.	Full Analysis Set n=23 Participants Comprised of all participants who received at least one infusion of study product
Pharmacokinetics (PK): Total Area Under the Plasma Concentration Versus Time Curve From 0 to Infinity Post-infusion Per Dose (Total AUC/Dose)	723.7 IU*hour (hr)/dL Standard Deviation 119	886 IU*hour (hr)/dL Standard Deviation 133.66	808.4 IU*hour (hr)/dL Standard Deviation 149.14

SECONDARY outcome

Timeframe: Within 30 mins pre-infusion and 4 post-infusion timepoints. Refer to Population Description below for more details.

Population: All participants randomized to 2 groups: Group 1 - BAX326 infusion in the morning PK timepoints: Pre-infusion and post-infusion at 15-30 minutes, then 7, 28 and 52 hours Group 2 - BAX326 infusion in the afternoon PK timepoints: Pre-infusion and post-infusion of 15-30 minutes, then 4, 24 and 69 hours

Computed as total area under the first moment curve (total AUMC) divided by the total area under the concentration versus time curve (total AUC)

Outcome measures

Measure	Pediatric Participants < 6 Years of Age n=11 Participants Pediatric participants \< 6 years of age. All pediatric participants underwent pharmacokinetic (PK) assessment followed by prophylactic treatment with the study product. All participants received the same dosing schedule of study product during the study. After a washout period of 5-7 days, participants received an initial infusion of study product at a dose of 75±5 IU/kg for the PK assessment. For the prophylactic regimen, participants were treated with the recommended dose of 50 IU/kg of study product twice weekly ranging from 40-80 IU/kg for 26±1 weeks or for at least 50 EDs to study product, whichever occurred last.	Pediatric Participants 6 to < 12 Years of Age n=12 Participants Pediatric participants 6 to \< 12 years of age. All pediatric participants underwent pharmacokinetic (PK) assessment followed by prophylactic treatment with the study product. All participants received the same dosing schedule of study product during the study. After a washout period of 5-7 days, participants received an initial infusion of study product at a dose of 75±5 IU/kg for the PK assessment. For the prophylactic regimen, participants were treated with the recommended dose of 50 IU/kg of study product twice weekly ranging from 40-80 IU/kg for 26±1 weeks or for at least 50 EDs to study product, whichever occurred last.	Full Analysis Set n=23 Participants Comprised of all participants who received at least one infusion of study product
Pharmacokinetics (PK): Mean Residence Time (MRT)	30.62 hours (hr) Standard Deviation 3.266	25.31 hours (hr) Standard Deviation 1.830	27.85 hours (hr) Standard Deviation 3.726

SECONDARY outcome

Timeframe: Within 30 mins pre-infusion and 4 post-infusion timepoints. Refer to Population Description below for more details.

Population: All participants randomized to 2 groups: Group 1 - BAX326 infusion in the morning PK timepoints: Pre-infusion and post-infusion at 15-30 minutes, then 7, 28 and 52 hours Group 2 - BAX326 infusion in the afternoon PK timepoints: Pre-infusion and post-infusion of 15-30 minutes, then 4, 24 and 69 hours

Computed as the dose divided by total Area under the curve (AUC)

Outcome measures

Measure	Pediatric Participants < 6 Years of Age n=11 Participants Pediatric participants \< 6 years of age. All pediatric participants underwent pharmacokinetic (PK) assessment followed by prophylactic treatment with the study product. All participants received the same dosing schedule of study product during the study. After a washout period of 5-7 days, participants received an initial infusion of study product at a dose of 75±5 IU/kg for the PK assessment. For the prophylactic regimen, participants were treated with the recommended dose of 50 IU/kg of study product twice weekly ranging from 40-80 IU/kg for 26±1 weeks or for at least 50 EDs to study product, whichever occurred last.	Pediatric Participants 6 to < 12 Years of Age n=12 Participants Pediatric participants 6 to \< 12 years of age. All pediatric participants underwent pharmacokinetic (PK) assessment followed by prophylactic treatment with the study product. All participants received the same dosing schedule of study product during the study. After a washout period of 5-7 days, participants received an initial infusion of study product at a dose of 75±5 IU/kg for the PK assessment. For the prophylactic regimen, participants were treated with the recommended dose of 50 IU/kg of study product twice weekly ranging from 40-80 IU/kg for 26±1 weeks or for at least 50 EDs to study product, whichever occurred last.	Full Analysis Set n=23 Participants Comprised of all participants who received at least one infusion of study product
Pharmacokinetics (PK): Factor IX (FIX) Clearance (CL)	0.1058 dL/(kg*hr) Standard Deviation 0.01650	0.0874 dL/(kg*hr) Standard Deviation 0.01213	0.0962 dL/(kg*hr) Standard Deviation 0.01689

SECONDARY outcome

Timeframe: Within 30 mins pre-infusion and 30 mins post-infusion

The rise in FIX activity in IU/dL per unit dose administered in IU/kg. Calculated as follows: (FIX activity at post-infusion minus FIX activity at pre-infusion) divided by weight-adjusted dose

Outcome measures

Measure	Pediatric Participants < 6 Years of Age n=10 Participants Pediatric participants \< 6 years of age. All pediatric participants underwent pharmacokinetic (PK) assessment followed by prophylactic treatment with the study product. All participants received the same dosing schedule of study product during the study. After a washout period of 5-7 days, participants received an initial infusion of study product at a dose of 75±5 IU/kg for the PK assessment. For the prophylactic regimen, participants were treated with the recommended dose of 50 IU/kg of study product twice weekly ranging from 40-80 IU/kg for 26±1 weeks or for at least 50 EDs to study product, whichever occurred last.	Pediatric Participants 6 to < 12 Years of Age n=12 Participants Pediatric participants 6 to \< 12 years of age. All pediatric participants underwent pharmacokinetic (PK) assessment followed by prophylactic treatment with the study product. All participants received the same dosing schedule of study product during the study. After a washout period of 5-7 days, participants received an initial infusion of study product at a dose of 75±5 IU/kg for the PK assessment. For the prophylactic regimen, participants were treated with the recommended dose of 50 IU/kg of study product twice weekly ranging from 40-80 IU/kg for 26±1 weeks or for at least 50 EDs to study product, whichever occurred last.	Full Analysis Set n=23 Participants Comprised of all participants who received at least one infusion of study product
Pharmacokinetics (PK): Incremental Recovery (IR)	0.586 IU/dL : IU/kg Standard Deviation 0.1320	0.731 IU/dL : IU/kg Standard Deviation 0.1615	0.665 IU/dL : IU/kg Standard Deviation 0.1632

SECONDARY outcome

Timeframe: Within 30 mins pre-infusion and 4 post-infusion timepoints. Refer to Population Description below for more details.

Population: All participants randomized to 2 groups: Group 1 - BAX326 infusion in the morning PK timepoints: Pre-infusion and post-infusion at 15-30 minutes, then 7, 28 and 52 hours Group 2 - BAX326 infusion in the afternoon PK timepoints: Pre-infusion and post-infusion of 15-30 minutes, then 4, 24 and 69 hours

Calculated as log_e2/λ, where λ is the regression slope in the terminal phase of the least absolute deviations regression model

Outcome measures

Measure	Pediatric Participants < 6 Years of Age n=11 Participants Pediatric participants \< 6 years of age. All pediatric participants underwent pharmacokinetic (PK) assessment followed by prophylactic treatment with the study product. All participants received the same dosing schedule of study product during the study. After a washout period of 5-7 days, participants received an initial infusion of study product at a dose of 75±5 IU/kg for the PK assessment. For the prophylactic regimen, participants were treated with the recommended dose of 50 IU/kg of study product twice weekly ranging from 40-80 IU/kg for 26±1 weeks or for at least 50 EDs to study product, whichever occurred last.	Pediatric Participants 6 to < 12 Years of Age n=12 Participants Pediatric participants 6 to \< 12 years of age. All pediatric participants underwent pharmacokinetic (PK) assessment followed by prophylactic treatment with the study product. All participants received the same dosing schedule of study product during the study. After a washout period of 5-7 days, participants received an initial infusion of study product at a dose of 75±5 IU/kg for the PK assessment. For the prophylactic regimen, participants were treated with the recommended dose of 50 IU/kg of study product twice weekly ranging from 40-80 IU/kg for 26±1 weeks or for at least 50 EDs to study product, whichever occurred last.	Full Analysis Set n=23 Participants Comprised of all participants who received at least one infusion of study product
Pharmacokinetics (PK): Elimination Phase Half-life (T 1/2)	27.67 hours (hr) Standard Deviation 2.658	23.15 hours (hr) Standard Deviation 1.582	25.31 hours (hr) Standard Deviation 3.130

SECONDARY outcome

Timeframe: Within 30 mins pre-infusion and 4 post-infusion timepoints. Refer to Population Description below for more details.

Population: All participants randomized to 2 groups: Group 1 - BAX326 infusion in the morning PK timepoints: Pre-infusion and post-infusion at 15-30 minutes, then 7, 28 and 52 hours Group 2 - BAX326 infusion in the afternoon PK timepoints: Pre-infusion and post-infusion of 15-30 minutes, then 4, 24 and 69 hours

Computed as Clearance (CL) * Mean residence time (MRT)

Outcome measures

Measure	Pediatric Participants < 6 Years of Age n=11 Participants Pediatric participants \< 6 years of age. All pediatric participants underwent pharmacokinetic (PK) assessment followed by prophylactic treatment with the study product. All participants received the same dosing schedule of study product during the study. After a washout period of 5-7 days, participants received an initial infusion of study product at a dose of 75±5 IU/kg for the PK assessment. For the prophylactic regimen, participants were treated with the recommended dose of 50 IU/kg of study product twice weekly ranging from 40-80 IU/kg for 26±1 weeks or for at least 50 EDs to study product, whichever occurred last.	Pediatric Participants 6 to < 12 Years of Age n=12 Participants Pediatric participants 6 to \< 12 years of age. All pediatric participants underwent pharmacokinetic (PK) assessment followed by prophylactic treatment with the study product. All participants received the same dosing schedule of study product during the study. After a washout period of 5-7 days, participants received an initial infusion of study product at a dose of 75±5 IU/kg for the PK assessment. For the prophylactic regimen, participants were treated with the recommended dose of 50 IU/kg of study product twice weekly ranging from 40-80 IU/kg for 26±1 weeks or for at least 50 EDs to study product, whichever occurred last.	Full Analysis Set n=23 Participants Comprised of all participants who received at least one infusion of study product
Pharmacokinetics (PK): Volume of Distribution at Steady State (Vss)	3.225 dL/kg Standard Deviation 0.5233	2.209 dL/kg Standard Deviation 0.3165	2.695 dL/kg Standard Deviation 0.6662

SECONDARY outcome

Timeframe: Within 30 mins pre-infusion and 30 mins post-infusion at baseline, Week 5, Week 13 and Week 26.

IR calculated as follows: (FIX activity at post-infusion minus FIX activity at pre-infusion) divided by weight-adjusted dose. IR is determined at baseline (PK analysis), Week 5, Week 13 and Week 26 timepoints. Number of participants contributing data (N) for this outcome measure is included in the category title in the order: pediatric participants > 6 years of age; pediatric participants 6 to <12 years of age; pharmacokinetic Full Analysis Set (PKFAS).

Outcome measures

Measure	Pediatric Participants < 6 Years of Age n=11 Participants Pediatric participants \< 6 years of age. All pediatric participants underwent pharmacokinetic (PK) assessment followed by prophylactic treatment with the study product. All participants received the same dosing schedule of study product during the study. After a washout period of 5-7 days, participants received an initial infusion of study product at a dose of 75±5 IU/kg for the PK assessment. For the prophylactic regimen, participants were treated with the recommended dose of 50 IU/kg of study product twice weekly ranging from 40-80 IU/kg for 26±1 weeks or for at least 50 EDs to study product, whichever occurred last.	Pediatric Participants 6 to < 12 Years of Age n=12 Participants Pediatric participants 6 to \< 12 years of age. All pediatric participants underwent pharmacokinetic (PK) assessment followed by prophylactic treatment with the study product. All participants received the same dosing schedule of study product during the study. After a washout period of 5-7 days, participants received an initial infusion of study product at a dose of 75±5 IU/kg for the PK assessment. For the prophylactic regimen, participants were treated with the recommended dose of 50 IU/kg of study product twice weekly ranging from 40-80 IU/kg for 26±1 weeks or for at least 50 EDs to study product, whichever occurred last.	Full Analysis Set n=23 Participants Comprised of all participants who received at least one infusion of study product
Pharmacokinetics (PK): Incremental Recovery (IR) Over Time Baseline (N=10, 12, 22)	0.586 IU/dL : IU/kg Standard Deviation 0.1320	0.731 IU/dL : IU/kg Standard Deviation 0.1615	0.665 IU/dL : IU/kg Standard Deviation 0.1632
Pharmacokinetics (PK): Incremental Recovery (IR) Over Time Week 5 (N=11, 12, 23)	0.630 IU/dL : IU/kg Standard Deviation 0.1028	0.726 IU/dL : IU/kg Standard Deviation 0.1291	0.680 IU/dL : IU/kg Standard Deviation 0.1245
Pharmacokinetics (PK): Incremental Recovery (IR) Over Time Week 13 (N=10, 11, 21)	0.676 IU/dL : IU/kg Standard Deviation 0.1211	0.733 IU/dL : IU/kg Standard Deviation 0.1400	0.706 IU/dL : IU/kg Standard Deviation 0.1313
Pharmacokinetics (PK): Incremental Recovery (IR) Over Time Week 26 (N=10, 11, 21)	0.647 IU/dL : IU/kg Standard Deviation 0.1274	0.795 IU/dL : IU/kg Standard Deviation 0.1445	0.724 IU/dL : IU/kg Standard Deviation 0.1533

SECONDARY outcome

Timeframe: Throughout study period (approximately 17 months)

Population: Participants in the Full Analysis Set who had bleeding episodes

Outcome measures

Measure	Pediatric Participants < 6 Years of Age n=7 Participants Pediatric participants \< 6 years of age. All pediatric participants underwent pharmacokinetic (PK) assessment followed by prophylactic treatment with the study product. All participants received the same dosing schedule of study product during the study. After a washout period of 5-7 days, participants received an initial infusion of study product at a dose of 75±5 IU/kg for the PK assessment. For the prophylactic regimen, participants were treated with the recommended dose of 50 IU/kg of study product twice weekly ranging from 40-80 IU/kg for 26±1 weeks or for at least 50 EDs to study product, whichever occurred last.	Pediatric Participants 6 to < 12 Years of Age n=7 Participants Pediatric participants 6 to \< 12 years of age. All pediatric participants underwent pharmacokinetic (PK) assessment followed by prophylactic treatment with the study product. All participants received the same dosing schedule of study product during the study. After a washout period of 5-7 days, participants received an initial infusion of study product at a dose of 75±5 IU/kg for the PK assessment. For the prophylactic regimen, participants were treated with the recommended dose of 50 IU/kg of study product twice weekly ranging from 40-80 IU/kg for 26±1 weeks or for at least 50 EDs to study product, whichever occurred last.	Full Analysis Set n=14 Participants Comprised of all participants who received at least one infusion of study product
Hemostatic Efficacy: Treatment of Bleeding Episodes: Number of Infusions Per Bleeding Episode Bleeding Episodes controlled with 1 infusion	9 Bleeding Episodes	6 Bleeding Episodes	15 Bleeding Episodes
Hemostatic Efficacy: Treatment of Bleeding Episodes: Number of Infusions Per Bleeding Episode Bleeding Episodes controlled with 2 infusions	1 Bleeding Episodes	7 Bleeding Episodes	8 Bleeding Episodes
Hemostatic Efficacy: Treatment of Bleeding Episodes: Number of Infusions Per Bleeding Episode Bleeding episodes controlled with ≥ 3 infusions	1 Bleeding Episodes	2 Bleeding Episodes	3 Bleeding Episodes

SECONDARY outcome

Timeframe: Throughout study period (approximately 17 months)

Population: Participants in the Full Analysis Set who had bleeding episodes

Rating Scale for Treatment of bleeding episodes (4-point ordinal scale): - Excellent: Full relief of pain and cessation of objective signs of bleeding (eg, swelling, tenderness, and decreased range of motion in the case of musculoskeletal hemorrhage) after a single infusion. No additional infusion required for the control of bleeding. Administration of further infusions to maintain hemostasis did not affect this scoring. - Good: Definite pain relief and/or improvement in signs of bleeding after a single infusion. Possibly requires more than 1 infusion for complete resolution. - Fair: Probable and/or slight relief of pain and slight improvement in signs of bleeding after single infusion. Required more than 1 infusion for complete resolution. - None: No improvement or condition worsens.

Outcome measures

Measure	Pediatric Participants < 6 Years of Age n=11 Bleeding Episodes Pediatric participants \< 6 years of age. All pediatric participants underwent pharmacokinetic (PK) assessment followed by prophylactic treatment with the study product. All participants received the same dosing schedule of study product during the study. After a washout period of 5-7 days, participants received an initial infusion of study product at a dose of 75±5 IU/kg for the PK assessment. For the prophylactic regimen, participants were treated with the recommended dose of 50 IU/kg of study product twice weekly ranging from 40-80 IU/kg for 26±1 weeks or for at least 50 EDs to study product, whichever occurred last.	Pediatric Participants 6 to < 12 Years of Age n=15 Bleeding Episodes Pediatric participants 6 to \< 12 years of age. All pediatric participants underwent pharmacokinetic (PK) assessment followed by prophylactic treatment with the study product. All participants received the same dosing schedule of study product during the study. After a washout period of 5-7 days, participants received an initial infusion of study product at a dose of 75±5 IU/kg for the PK assessment. For the prophylactic regimen, participants were treated with the recommended dose of 50 IU/kg of study product twice weekly ranging from 40-80 IU/kg for 26±1 weeks or for at least 50 EDs to study product, whichever occurred last.	Full Analysis Set n=26 Bleeding Episodes Comprised of all participants who received at least one infusion of study product
Hemostatic Efficacy: Treatment of Bleeding Episodes: Overall Hemostatic Efficacy Rating at Resolution of Bleed Excellent	9 Bleeding Episodes	4 Bleeding Episodes	13 Bleeding Episodes
Hemostatic Efficacy: Treatment of Bleeding Episodes: Overall Hemostatic Efficacy Rating at Resolution of Bleed Good	2 Bleeding Episodes	10 Bleeding Episodes	12 Bleeding Episodes
Hemostatic Efficacy: Treatment of Bleeding Episodes: Overall Hemostatic Efficacy Rating at Resolution of Bleed Fair	0 Bleeding Episodes	1 Bleeding Episodes	1 Bleeding Episodes
Hemostatic Efficacy: Treatment of Bleeding Episodes: Overall Hemostatic Efficacy Rating at Resolution of Bleed None	0 Bleeding Episodes	0 Bleeding Episodes	0 Bleeding Episodes

SECONDARY outcome

Timeframe: Throughout study period (approximately 17 months)

The annualized bleeding rate (ABR) during prophylaxis was calculated only for participants who had adequate treatment time for bleeding rate assessment (i.e., more than 3 months of prophylaxis treatment). The observation period for prophylaxis was to be the time between the first and the last prophylactic infusions. The treatment period for surgery was to be excluded from the bleed rate calculation. ABR calculated as (Number of bleeding episodes/observed treatment period in days) * 365.25.

Outcome measures

Measure	Pediatric Participants < 6 Years of Age n=11 Participants Pediatric participants \< 6 years of age. All pediatric participants underwent pharmacokinetic (PK) assessment followed by prophylactic treatment with the study product. All participants received the same dosing schedule of study product during the study. After a washout period of 5-7 days, participants received an initial infusion of study product at a dose of 75±5 IU/kg for the PK assessment. For the prophylactic regimen, participants were treated with the recommended dose of 50 IU/kg of study product twice weekly ranging from 40-80 IU/kg for 26±1 weeks or for at least 50 EDs to study product, whichever occurred last.	Pediatric Participants 6 to < 12 Years of Age n=12 Participants Pediatric participants 6 to \< 12 years of age. All pediatric participants underwent pharmacokinetic (PK) assessment followed by prophylactic treatment with the study product. All participants received the same dosing schedule of study product during the study. After a washout period of 5-7 days, participants received an initial infusion of study product at a dose of 75±5 IU/kg for the PK assessment. For the prophylactic regimen, participants were treated with the recommended dose of 50 IU/kg of study product twice weekly ranging from 40-80 IU/kg for 26±1 weeks or for at least 50 EDs to study product, whichever occurred last.	Full Analysis Set n=23 Participants Comprised of all participants who received at least one infusion of study product
Hemostatic Efficacy: Prophylaxis: Annualized Bleeding Rate (ABR)	1.9 Bleeding episodes per year Standard Deviation 1.89	3.4 Bleeding episodes per year Standard Deviation 3.93	2.7 Bleeding episodes per year Standard Deviation 3.14

SECONDARY outcome

Timeframe: Throughout study period (approximately 17 months)

Outcome measures

Measure	Pediatric Participants < 6 Years of Age n=11 Participants Pediatric participants \< 6 years of age. All pediatric participants underwent pharmacokinetic (PK) assessment followed by prophylactic treatment with the study product. All participants received the same dosing schedule of study product during the study. After a washout period of 5-7 days, participants received an initial infusion of study product at a dose of 75±5 IU/kg for the PK assessment. For the prophylactic regimen, participants were treated with the recommended dose of 50 IU/kg of study product twice weekly ranging from 40-80 IU/kg for 26±1 weeks or for at least 50 EDs to study product, whichever occurred last.	Pediatric Participants 6 to < 12 Years of Age n=12 Participants Pediatric participants 6 to \< 12 years of age. All pediatric participants underwent pharmacokinetic (PK) assessment followed by prophylactic treatment with the study product. All participants received the same dosing schedule of study product during the study. After a washout period of 5-7 days, participants received an initial infusion of study product at a dose of 75±5 IU/kg for the PK assessment. For the prophylactic regimen, participants were treated with the recommended dose of 50 IU/kg of study product twice weekly ranging from 40-80 IU/kg for 26±1 weeks or for at least 50 EDs to study product, whichever occurred last.	Full Analysis Set n=23 Participants Comprised of all participants who received at least one infusion of study product
Consumption of BAX326: Number of Infusions Per Month	6.8 Infusions per month Standard Deviation 0.44	7.2 Infusions per month Standard Deviation 0.40	7.0 Infusions per month Standard Deviation 0.44

SECONDARY outcome

Timeframe: Throughout study period (approximately 17 months)

Outcome measures

Measure	Pediatric Participants < 6 Years of Age n=11 Participants Pediatric participants \< 6 years of age. All pediatric participants underwent pharmacokinetic (PK) assessment followed by prophylactic treatment with the study product. All participants received the same dosing schedule of study product during the study. After a washout period of 5-7 days, participants received an initial infusion of study product at a dose of 75±5 IU/kg for the PK assessment. For the prophylactic regimen, participants were treated with the recommended dose of 50 IU/kg of study product twice weekly ranging from 40-80 IU/kg for 26±1 weeks or for at least 50 EDs to study product, whichever occurred last.	Pediatric Participants 6 to < 12 Years of Age n=12 Participants Pediatric participants 6 to \< 12 years of age. All pediatric participants underwent pharmacokinetic (PK) assessment followed by prophylactic treatment with the study product. All participants received the same dosing schedule of study product during the study. After a washout period of 5-7 days, participants received an initial infusion of study product at a dose of 75±5 IU/kg for the PK assessment. For the prophylactic regimen, participants were treated with the recommended dose of 50 IU/kg of study product twice weekly ranging from 40-80 IU/kg for 26±1 weeks or for at least 50 EDs to study product, whichever occurred last.	Full Analysis Set n=23 Participants Comprised of all participants who received at least one infusion of study product
Consumption of BAX326: Number of Infusions Per Year	82.1 Infusions per year Standard Deviation 5.27	85.9 Infusions per year Standard Deviation 4.79	84.1 Infusions per year Standard Deviation 5.27

SECONDARY outcome

Timeframe: Throughout study period (approximately 17 months)

Outcome measures

Measure	Pediatric Participants < 6 Years of Age n=11 Participants Pediatric participants \< 6 years of age. All pediatric participants underwent pharmacokinetic (PK) assessment followed by prophylactic treatment with the study product. All participants received the same dosing schedule of study product during the study. After a washout period of 5-7 days, participants received an initial infusion of study product at a dose of 75±5 IU/kg for the PK assessment. For the prophylactic regimen, participants were treated with the recommended dose of 50 IU/kg of study product twice weekly ranging from 40-80 IU/kg for 26±1 weeks or for at least 50 EDs to study product, whichever occurred last.	Pediatric Participants 6 to < 12 Years of Age n=12 Participants Pediatric participants 6 to \< 12 years of age. All pediatric participants underwent pharmacokinetic (PK) assessment followed by prophylactic treatment with the study product. All participants received the same dosing schedule of study product during the study. After a washout period of 5-7 days, participants received an initial infusion of study product at a dose of 75±5 IU/kg for the PK assessment. For the prophylactic regimen, participants were treated with the recommended dose of 50 IU/kg of study product twice weekly ranging from 40-80 IU/kg for 26±1 weeks or for at least 50 EDs to study product, whichever occurred last.	Full Analysis Set n=23 Participants Comprised of all participants who received at least one infusion of study product
Consumption of BAX326: Weight-adjusted Consumption Per Month	393.4 IU/kg per month Standard Deviation 50.53	414.8 IU/kg per month Standard Deviation 58.44	404.6 IU/kg per month Standard Deviation 54.66

SECONDARY outcome

Timeframe: Throughout study period (approximately 17 months)

Outcome measures

Measure	Pediatric Participants < 6 Years of Age n=11 Participants Pediatric participants \< 6 years of age. All pediatric participants underwent pharmacokinetic (PK) assessment followed by prophylactic treatment with the study product. All participants received the same dosing schedule of study product during the study. After a washout period of 5-7 days, participants received an initial infusion of study product at a dose of 75±5 IU/kg for the PK assessment. For the prophylactic regimen, participants were treated with the recommended dose of 50 IU/kg of study product twice weekly ranging from 40-80 IU/kg for 26±1 weeks or for at least 50 EDs to study product, whichever occurred last.	Pediatric Participants 6 to < 12 Years of Age n=12 Participants Pediatric participants 6 to \< 12 years of age. All pediatric participants underwent pharmacokinetic (PK) assessment followed by prophylactic treatment with the study product. All participants received the same dosing schedule of study product during the study. After a washout period of 5-7 days, participants received an initial infusion of study product at a dose of 75±5 IU/kg for the PK assessment. For the prophylactic regimen, participants were treated with the recommended dose of 50 IU/kg of study product twice weekly ranging from 40-80 IU/kg for 26±1 weeks or for at least 50 EDs to study product, whichever occurred last.	Full Analysis Set n=23 Participants Comprised of all participants who received at least one infusion of study product
Consumption of BAX326: Weight-adjusted Consumption Per Year (Annualized)	4720.9 IU/kg per year Standard Deviation 606.31	4978.2 IU/kg per year Standard Deviation 701.26	4855.1 IU/kg per year Standard Deviation 655.93

SECONDARY outcome

Timeframe: Throughout study period (approximately 17 months)

Event includes prophylactic infusions of study product and infusions of study product for treatment of bleeding episodes (BEs).

Outcome measures

Measure	Pediatric Participants < 6 Years of Age n=11 Participants Pediatric participants \< 6 years of age. All pediatric participants underwent pharmacokinetic (PK) assessment followed by prophylactic treatment with the study product. All participants received the same dosing schedule of study product during the study. After a washout period of 5-7 days, participants received an initial infusion of study product at a dose of 75±5 IU/kg for the PK assessment. For the prophylactic regimen, participants were treated with the recommended dose of 50 IU/kg of study product twice weekly ranging from 40-80 IU/kg for 26±1 weeks or for at least 50 EDs to study product, whichever occurred last.	Pediatric Participants 6 to < 12 Years of Age n=12 Participants Pediatric participants 6 to \< 12 years of age. All pediatric participants underwent pharmacokinetic (PK) assessment followed by prophylactic treatment with the study product. All participants received the same dosing schedule of study product during the study. After a washout period of 5-7 days, participants received an initial infusion of study product at a dose of 75±5 IU/kg for the PK assessment. For the prophylactic regimen, participants were treated with the recommended dose of 50 IU/kg of study product twice weekly ranging from 40-80 IU/kg for 26±1 weeks or for at least 50 EDs to study product, whichever occurred last.	Full Analysis Set n=23 Participants Comprised of all participants who received at least one infusion of study product
Consumption of BAX326: Weight-adjusted Consumption Per Event Consumption of BAX326 for prophylactic infusions	56.3 IU/kg Standard Deviation 10.29	56.2 IU/kg Standard Deviation 6.55	56.2 IU/kg Standard Deviation 8.34
Consumption of BAX326: Weight-adjusted Consumption Per Event Consumption of BAX326 for infusions to treat BEs	57.6 IU/kg Standard Deviation 11.87	62.1 IU/kg Standard Deviation 16.00	59.9 IU/kg Standard Deviation 13.74

SECONDARY outcome

Timeframe: Throughout study period (approximately 17 months)

Outcome measures

Measure	Pediatric Participants < 6 Years of Age n=11 Participants Pediatric participants \< 6 years of age. All pediatric participants underwent pharmacokinetic (PK) assessment followed by prophylactic treatment with the study product. All participants received the same dosing schedule of study product during the study. After a washout period of 5-7 days, participants received an initial infusion of study product at a dose of 75±5 IU/kg for the PK assessment. For the prophylactic regimen, participants were treated with the recommended dose of 50 IU/kg of study product twice weekly ranging from 40-80 IU/kg for 26±1 weeks or for at least 50 EDs to study product, whichever occurred last.	Pediatric Participants 6 to < 12 Years of Age n=12 Participants Pediatric participants 6 to \< 12 years of age. All pediatric participants underwent pharmacokinetic (PK) assessment followed by prophylactic treatment with the study product. All participants received the same dosing schedule of study product during the study. After a washout period of 5-7 days, participants received an initial infusion of study product at a dose of 75±5 IU/kg for the PK assessment. For the prophylactic regimen, participants were treated with the recommended dose of 50 IU/kg of study product twice weekly ranging from 40-80 IU/kg for 26±1 weeks or for at least 50 EDs to study product, whichever occurred last.	Full Analysis Set n=23 Participants Comprised of all participants who received at least one infusion of study product
Safety and Immunogenicity: Number of Participants Who Developed Inhibitory Antibodies to Factor IX (FIX)	0 Participants	0 Participants	0 Participants

SECONDARY outcome

Timeframe: Throughout study period (approximately 17 months)

If more than 2-dilution increase as compared to pre-study level at screening and titers verified for specificity in the confirmatory assay. AB=antibodies in category for outcome measure data.

Outcome measures

Measure	Pediatric Participants < 6 Years of Age n=11 Participants Pediatric participants \< 6 years of age. All pediatric participants underwent pharmacokinetic (PK) assessment followed by prophylactic treatment with the study product. All participants received the same dosing schedule of study product during the study. After a washout period of 5-7 days, participants received an initial infusion of study product at a dose of 75±5 IU/kg for the PK assessment. For the prophylactic regimen, participants were treated with the recommended dose of 50 IU/kg of study product twice weekly ranging from 40-80 IU/kg for 26±1 weeks or for at least 50 EDs to study product, whichever occurred last.	Pediatric Participants 6 to < 12 Years of Age n=12 Participants Pediatric participants 6 to \< 12 years of age. All pediatric participants underwent pharmacokinetic (PK) assessment followed by prophylactic treatment with the study product. All participants received the same dosing schedule of study product during the study. After a washout period of 5-7 days, participants received an initial infusion of study product at a dose of 75±5 IU/kg for the PK assessment. For the prophylactic regimen, participants were treated with the recommended dose of 50 IU/kg of study product twice weekly ranging from 40-80 IU/kg for 26±1 weeks or for at least 50 EDs to study product, whichever occurred last.	Full Analysis Set n=23 Participants Comprised of all participants who received at least one infusion of study product
Safety and Immunogenicity: Number of Participants Who Developed Total Binding Antibodies to Factor IX (FIX)	0 Participants	0 Participants	0 Participants

SECONDARY outcome

Timeframe: Throughout study period (approximately 17 months)

Outcome measures

Measure	Pediatric Participants < 6 Years of Age n=11 Participants Pediatric participants \< 6 years of age. All pediatric participants underwent pharmacokinetic (PK) assessment followed by prophylactic treatment with the study product. All participants received the same dosing schedule of study product during the study. After a washout period of 5-7 days, participants received an initial infusion of study product at a dose of 75±5 IU/kg for the PK assessment. For the prophylactic regimen, participants were treated with the recommended dose of 50 IU/kg of study product twice weekly ranging from 40-80 IU/kg for 26±1 weeks or for at least 50 EDs to study product, whichever occurred last.	Pediatric Participants 6 to < 12 Years of Age n=12 Participants Pediatric participants 6 to \< 12 years of age. All pediatric participants underwent pharmacokinetic (PK) assessment followed by prophylactic treatment with the study product. All participants received the same dosing schedule of study product during the study. After a washout period of 5-7 days, participants received an initial infusion of study product at a dose of 75±5 IU/kg for the PK assessment. For the prophylactic regimen, participants were treated with the recommended dose of 50 IU/kg of study product twice weekly ranging from 40-80 IU/kg for 26±1 weeks or for at least 50 EDs to study product, whichever occurred last.	Full Analysis Set n=23 Participants Comprised of all participants who received at least one infusion of study product
Safety: Number of Participants With Severe Allergic Reactions, e.g. Anaphylaxis	0 Participants	0 Participants	0 Participants

SECONDARY outcome

Timeframe: Throughout study period (approximately 17 months)

Outcome measures

Measure	Pediatric Participants < 6 Years of Age n=11 Participants Pediatric participants \< 6 years of age. All pediatric participants underwent pharmacokinetic (PK) assessment followed by prophylactic treatment with the study product. All participants received the same dosing schedule of study product during the study. After a washout period of 5-7 days, participants received an initial infusion of study product at a dose of 75±5 IU/kg for the PK assessment. For the prophylactic regimen, participants were treated with the recommended dose of 50 IU/kg of study product twice weekly ranging from 40-80 IU/kg for 26±1 weeks or for at least 50 EDs to study product, whichever occurred last.	Pediatric Participants 6 to < 12 Years of Age n=12 Participants Pediatric participants 6 to \< 12 years of age. All pediatric participants underwent pharmacokinetic (PK) assessment followed by prophylactic treatment with the study product. All participants received the same dosing schedule of study product during the study. After a washout period of 5-7 days, participants received an initial infusion of study product at a dose of 75±5 IU/kg for the PK assessment. For the prophylactic regimen, participants were treated with the recommended dose of 50 IU/kg of study product twice weekly ranging from 40-80 IU/kg for 26±1 weeks or for at least 50 EDs to study product, whichever occurred last.	Full Analysis Set n=23 Participants Comprised of all participants who received at least one infusion of study product
Safety: Number of Participants With Thrombotic Events	0 Participants	0 Participants	0 Participants

SECONDARY outcome

Timeframe: Throughout study period (approximately 17 months)

Categories consist of Clinically Significant (CS) changes in haemaotology parameters, clinical chemistry parameters and vital signs. Abbreviations in categories; Clin=clinical; params=parameters

Outcome measures

Measure	Pediatric Participants < 6 Years of Age n=11 Participants Pediatric participants \< 6 years of age. All pediatric participants underwent pharmacokinetic (PK) assessment followed by prophylactic treatment with the study product. All participants received the same dosing schedule of study product during the study. After a washout period of 5-7 days, participants received an initial infusion of study product at a dose of 75±5 IU/kg for the PK assessment. For the prophylactic regimen, participants were treated with the recommended dose of 50 IU/kg of study product twice weekly ranging from 40-80 IU/kg for 26±1 weeks or for at least 50 EDs to study product, whichever occurred last.	Pediatric Participants 6 to < 12 Years of Age n=12 Participants Pediatric participants 6 to \< 12 years of age. All pediatric participants underwent pharmacokinetic (PK) assessment followed by prophylactic treatment with the study product. All participants received the same dosing schedule of study product during the study. After a washout period of 5-7 days, participants received an initial infusion of study product at a dose of 75±5 IU/kg for the PK assessment. For the prophylactic regimen, participants were treated with the recommended dose of 50 IU/kg of study product twice weekly ranging from 40-80 IU/kg for 26±1 weeks or for at least 50 EDs to study product, whichever occurred last.	Full Analysis Set n=23 Participants Comprised of all participants who received at least one infusion of study product
Safety: Number of Participants With Clinically Significant Changes in Routine Laboratory Parameters (Haematology and Clinical Chemistry), and Vital Signs Number with CS changes in haematology params	0 Participants	2 Participants	2 Participants
Safety: Number of Participants With Clinically Significant Changes in Routine Laboratory Parameters (Haematology and Clinical Chemistry), and Vital Signs Number with CS changes in clin. chemistry params	0 Participants	0 Participants	0 Participants
Safety: Number of Participants With Clinically Significant Changes in Routine Laboratory Parameters (Haematology and Clinical Chemistry), and Vital Signs Number with CS changes in vital signs	0 Participants	0 Participants	0 Participants

SECONDARY outcome

Timeframe: Throughout study period (approximately 17 months)

If more than 2-dilution increase as compared to pre-study level at screening and titers verified for specificity in the confirmatory assay.

Outcome measures

Measure	Pediatric Participants < 6 Years of Age n=11 Participants Pediatric participants \< 6 years of age. All pediatric participants underwent pharmacokinetic (PK) assessment followed by prophylactic treatment with the study product. All participants received the same dosing schedule of study product during the study. After a washout period of 5-7 days, participants received an initial infusion of study product at a dose of 75±5 IU/kg for the PK assessment. For the prophylactic regimen, participants were treated with the recommended dose of 50 IU/kg of study product twice weekly ranging from 40-80 IU/kg for 26±1 weeks or for at least 50 EDs to study product, whichever occurred last.	Pediatric Participants 6 to < 12 Years of Age n=12 Participants Pediatric participants 6 to \< 12 years of age. All pediatric participants underwent pharmacokinetic (PK) assessment followed by prophylactic treatment with the study product. All participants received the same dosing schedule of study product during the study. After a washout period of 5-7 days, participants received an initial infusion of study product at a dose of 75±5 IU/kg for the PK assessment. For the prophylactic regimen, participants were treated with the recommended dose of 50 IU/kg of study product twice weekly ranging from 40-80 IU/kg for 26±1 weeks or for at least 50 EDs to study product, whichever occurred last.	Full Analysis Set n=23 Participants Comprised of all participants who received at least one infusion of study product
Safety: Number of Participants Who Developed Antibodies to Chinese Hamster Ovary (CHO) Proteins and Recombinant Furin (rFurin) Number who developed antibodies to CHO proteins	0 Participants	0 Participants	0 Participants
Safety: Number of Participants Who Developed Antibodies to Chinese Hamster Ovary (CHO) Proteins and Recombinant Furin (rFurin) Number who developed antibodies to rFurin	0 Participants	0 Participants	0 Participants

SECONDARY outcome

Timeframe: Baseline and 6 months

Population: Participants in the Full Analysis Set who had PedsQL™ data for baseline and 6 months

For this study, the PedsQL™ questionnaires for participants 2 to 7 years of age (parent-proxy versions for age groups 2-4 years and 5-7 years) and PedsQL™ Child version for participants 8 to 12 years of age were used. The Peds-QL is a generic Health-Related Quality of Life (HR QoL) instrument designed specifically for a pediatric population. It captures the following domains: general health/activities, feelings/emotional, social functioning, school functioning. A 5-point score is used for each domain: from 0 (never) to 4 (almost always). Items are reversed scored and linearly transformed to a 0-100 scale as follows: 0=100, 1=75, 2=50, 3=25, 4=0 so that higher scores indicate better quality of life (QoL). The total score is the mean (average) of all scores from the 4 domains. The change from baseline in total score is reported- a positive score indicates a better QoL compared to baseline and a negative score indicates a poorer QoL compared to baseline.

Outcome measures

Measure	Pediatric Participants < 6 Years of Age n=16 Participants Pediatric participants \< 6 years of age. All pediatric participants underwent pharmacokinetic (PK) assessment followed by prophylactic treatment with the study product. All participants received the same dosing schedule of study product during the study. After a washout period of 5-7 days, participants received an initial infusion of study product at a dose of 75±5 IU/kg for the PK assessment. For the prophylactic regimen, participants were treated with the recommended dose of 50 IU/kg of study product twice weekly ranging from 40-80 IU/kg for 26±1 weeks or for at least 50 EDs to study product, whichever occurred last.	Pediatric Participants 6 to < 12 Years of Age Pediatric participants 6 to \< 12 years of age. All pediatric participants underwent pharmacokinetic (PK) assessment followed by prophylactic treatment with the study product. All participants received the same dosing schedule of study product during the study. After a washout period of 5-7 days, participants received an initial infusion of study product at a dose of 75±5 IU/kg for the PK assessment. For the prophylactic regimen, participants were treated with the recommended dose of 50 IU/kg of study product twice weekly ranging from 40-80 IU/kg for 26±1 weeks or for at least 50 EDs to study product, whichever occurred last.	Full Analysis Set Comprised of all participants who received at least one infusion of study product
Health-related Quality of Life (HRQoL): PedsQL™ Change From Baseline in Total Score Peds-QL 2-4 (N=4)	3.27 Scores on a scale Standard Deviation 10.119	—	—
Health-related Quality of Life (HRQoL): PedsQL™ Change From Baseline in Total Score Peds-QL 5-7 (N=2)	-7.07 Scores on a scale Standard Deviation 6.917	—	—
Health-related Quality of Life (HRQoL): PedsQL™ Change From Baseline in Total Score Peds-QL 8-12 (N=10)	4.02 Scores on a scale Standard Deviation 11.038	—	—

SECONDARY outcome

Timeframe: Baseline and 6 months

Population: Participants in the Full Analysis Set who had Haemo-QoL data for baseline and 6 months

The Haemo-QoL is a quality of life (QoL) assessment instrument for children and adolescents with haemophilia. As a hemophilia-specific instrument, this measure assesses very specific aspects of dealing with hemophilia. For the Haemo-QoL, higher scores indicate a worse quality of life. Scores on a scale range between 0 and 100.

Outcome measures

Measure	Pediatric Participants < 6 Years of Age n=10 Participants Pediatric participants \< 6 years of age. All pediatric participants underwent pharmacokinetic (PK) assessment followed by prophylactic treatment with the study product. All participants received the same dosing schedule of study product during the study. After a washout period of 5-7 days, participants received an initial infusion of study product at a dose of 75±5 IU/kg for the PK assessment. For the prophylactic regimen, participants were treated with the recommended dose of 50 IU/kg of study product twice weekly ranging from 40-80 IU/kg for 26±1 weeks or for at least 50 EDs to study product, whichever occurred last.	Pediatric Participants 6 to < 12 Years of Age Pediatric participants 6 to \< 12 years of age. All pediatric participants underwent pharmacokinetic (PK) assessment followed by prophylactic treatment with the study product. All participants received the same dosing schedule of study product during the study. After a washout period of 5-7 days, participants received an initial infusion of study product at a dose of 75±5 IU/kg for the PK assessment. For the prophylactic regimen, participants were treated with the recommended dose of 50 IU/kg of study product twice weekly ranging from 40-80 IU/kg for 26±1 weeks or for at least 50 EDs to study product, whichever occurred last.	Full Analysis Set Comprised of all participants who received at least one infusion of study product
Health-related Quality of Life (HRQoL): Haemo-QoL, Change From Baseline in Total Score	-0.18 Scores on a scale Standard Deviation 0.456	—	—

SECONDARY outcome

Timeframe: Baseline (Pharmacokinetic [PK] assessment), Week 5, Week 13 and Week 26

The number of hospitalizations per participant. Number of participants contributing data (N) for this outcome measure is included in the category title in the order: pediatric participants < 6 years of age; pediatric participants 6 to <12 years of age; Full Analysis Set.

Outcome measures

Measure	Pediatric Participants < 6 Years of Age n=11 Participants Pediatric participants \< 6 years of age. All pediatric participants underwent pharmacokinetic (PK) assessment followed by prophylactic treatment with the study product. All participants received the same dosing schedule of study product during the study. After a washout period of 5-7 days, participants received an initial infusion of study product at a dose of 75±5 IU/kg for the PK assessment. For the prophylactic regimen, participants were treated with the recommended dose of 50 IU/kg of study product twice weekly ranging from 40-80 IU/kg for 26±1 weeks or for at least 50 EDs to study product, whichever occurred last.	Pediatric Participants 6 to < 12 Years of Age n=12 Participants Pediatric participants 6 to \< 12 years of age. All pediatric participants underwent pharmacokinetic (PK) assessment followed by prophylactic treatment with the study product. All participants received the same dosing schedule of study product during the study. After a washout period of 5-7 days, participants received an initial infusion of study product at a dose of 75±5 IU/kg for the PK assessment. For the prophylactic regimen, participants were treated with the recommended dose of 50 IU/kg of study product twice weekly ranging from 40-80 IU/kg for 26±1 weeks or for at least 50 EDs to study product, whichever occurred last.	Full Analysis Set n=23 Participants Comprised of all participants who received at least one infusion of study product
Health Resource Use: Number of Hospitalizations Week 5 (N=11, 12, 23)	0.0 Hospitalizations Interval 0.0 to 0.0	0.0 Hospitalizations Interval 0.0 to 0.0	0.0 Hospitalizations Interval 0.0 to 0.0
Health Resource Use: Number of Hospitalizations Week 13 (N=11, 11, 22)	0.0 Hospitalizations Interval 0.0 to 1.0	0.0 Hospitalizations Interval 0.0 to 1.0	0.0 Hospitalizations Interval 0.0 to 1.0
Health Resource Use: Number of Hospitalizations Week 26 (N=11, 11, 22)	0.0 Hospitalizations Interval 0.0 to 0.0	0.0 Hospitalizations Interval 0.0 to 1.0	0.0 Hospitalizations Interval 0.0 to 1.0

SECONDARY outcome

Timeframe: Baseline (Pharmacokinetic [PK] assessment), Week 5, Week 13 and Week 26

The length of hospitalization per participant. Number of participants contributing data (N) for this outcome measure is included in the category title in the order: pediatric participants < 6 years of age; pediatric participants 6 to <12 years of age; Full Analysis Set.

Outcome measures

Measure	Pediatric Participants < 6 Years of Age n=1 Participants Pediatric participants \< 6 years of age. All pediatric participants underwent pharmacokinetic (PK) assessment followed by prophylactic treatment with the study product. All participants received the same dosing schedule of study product during the study. After a washout period of 5-7 days, participants received an initial infusion of study product at a dose of 75±5 IU/kg for the PK assessment. For the prophylactic regimen, participants were treated with the recommended dose of 50 IU/kg of study product twice weekly ranging from 40-80 IU/kg for 26±1 weeks or for at least 50 EDs to study product, whichever occurred last.	Pediatric Participants 6 to < 12 Years of Age n=2 Participants Pediatric participants 6 to \< 12 years of age. All pediatric participants underwent pharmacokinetic (PK) assessment followed by prophylactic treatment with the study product. All participants received the same dosing schedule of study product during the study. After a washout period of 5-7 days, participants received an initial infusion of study product at a dose of 75±5 IU/kg for the PK assessment. For the prophylactic regimen, participants were treated with the recommended dose of 50 IU/kg of study product twice weekly ranging from 40-80 IU/kg for 26±1 weeks or for at least 50 EDs to study product, whichever occurred last.	Full Analysis Set n=3 Participants Comprised of all participants who received at least one infusion of study product
Health Resource Use: Length of Hospitalization PK assessment	NA Days No participants were hospitalized during this timepoint.	NA Days No participants were hospitalized during this timepoint.	NA Days No participants were hospitalized during this timepoint.
Health Resource Use: Length of Hospitalization Week 5	NA Days No participants were hospitalized during this timepoint.	NA Days No participants were hospitalized during this timepoint.	NA Days No participants were hospitalized during this timepoint.
Health Resource Use: Length of Hospitalization Week 13 (N=1, 1, 2)	2.0 Days Interval 2.0 to 2.0	4.0 Days Interval 4.0 to 4.0	3.0 Days Interval 2.0 to 4.0
Health Resource Use: Length of Hospitalization Week 26 (N=NA, 1, 1)	NA Days No participants were hospitalized during this timepoint.	13.0 Days Interval 13.0 to 13.0	13.0 Days Interval 13.0 to 13.0

SECONDARY outcome

Timeframe: Baseline (Pharmacokinetic [PK] assessment), Week 5, Week 13 and Week 26

Population: Participants who had unscheduled visits to a doctor's office

The number of unscheduled doctor's Office visits per participant. Number of participants contributing data (N) for this outcome measure is included in the category title in the order: pediatric participants < 6 years of age; pediatric participants 6 to <12 years of age; Full Analysis Set.

Outcome measures

Measure	Pediatric Participants < 6 Years of Age n=11 Participants Pediatric participants \< 6 years of age. All pediatric participants underwent pharmacokinetic (PK) assessment followed by prophylactic treatment with the study product. All participants received the same dosing schedule of study product during the study. After a washout period of 5-7 days, participants received an initial infusion of study product at a dose of 75±5 IU/kg for the PK assessment. For the prophylactic regimen, participants were treated with the recommended dose of 50 IU/kg of study product twice weekly ranging from 40-80 IU/kg for 26±1 weeks or for at least 50 EDs to study product, whichever occurred last.	Pediatric Participants 6 to < 12 Years of Age n=12 Participants Pediatric participants 6 to \< 12 years of age. All pediatric participants underwent pharmacokinetic (PK) assessment followed by prophylactic treatment with the study product. All participants received the same dosing schedule of study product during the study. After a washout period of 5-7 days, participants received an initial infusion of study product at a dose of 75±5 IU/kg for the PK assessment. For the prophylactic regimen, participants were treated with the recommended dose of 50 IU/kg of study product twice weekly ranging from 40-80 IU/kg for 26±1 weeks or for at least 50 EDs to study product, whichever occurred last.	Full Analysis Set n=23 Participants Comprised of all participants who received at least one infusion of study product
Health Resource Use: Unscheduled Doctor's Office Visits PK assessment (N=11, 12, 23)	0.0 Visits Interval 0.0 to 0.0	0.0 Visits Interval 0.0 to 0.0	0.0 Visits Interval 0.0 to 0.0
Health Resource Use: Unscheduled Doctor's Office Visits Week 5 (N=11, 12, 23)	0.0 Visits Interval 0.0 to 1.0	0.0 Visits Interval 0.0 to 0.0	0.0 Visits Interval 0.0 to 1.0
Health Resource Use: Unscheduled Doctor's Office Visits Week 13 (N=11, 11, 22)	0.0 Visits Interval 0.0 to 1.0	0.0 Visits Interval 0.0 to 2.0	0.0 Visits Interval 0.0 to 2.0
Health Resource Use: Unscheduled Doctor's Office Visits Week 26 (N=11, 11, 22)	0.0 Visits Interval 0.0 to 6.0	0.0 Visits Interval 0.0 to 0.0	0.0 Visits Interval 0.0 to 6.0

SECONDARY outcome

Timeframe: Baseline (Pharmacokinetic [PK] assessment), Week 5, Week 13 and Week 26

The number of Emergency Room visits per participant. Number of participants contributing data (N) for this outcome measure is included in the category title in the order: pediatric participants < 6 years of age; pediatric participants 6 to <12 years of age; Full Analysis Set.

Outcome measures

Measure	Pediatric Participants < 6 Years of Age n=11 Participants Pediatric participants \< 6 years of age. All pediatric participants underwent pharmacokinetic (PK) assessment followed by prophylactic treatment with the study product. All participants received the same dosing schedule of study product during the study. After a washout period of 5-7 days, participants received an initial infusion of study product at a dose of 75±5 IU/kg for the PK assessment. For the prophylactic regimen, participants were treated with the recommended dose of 50 IU/kg of study product twice weekly ranging from 40-80 IU/kg for 26±1 weeks or for at least 50 EDs to study product, whichever occurred last.	Pediatric Participants 6 to < 12 Years of Age n=12 Participants Pediatric participants 6 to \< 12 years of age. All pediatric participants underwent pharmacokinetic (PK) assessment followed by prophylactic treatment with the study product. All participants received the same dosing schedule of study product during the study. After a washout period of 5-7 days, participants received an initial infusion of study product at a dose of 75±5 IU/kg for the PK assessment. For the prophylactic regimen, participants were treated with the recommended dose of 50 IU/kg of study product twice weekly ranging from 40-80 IU/kg for 26±1 weeks or for at least 50 EDs to study product, whichever occurred last.	Full Analysis Set n=23 Participants Comprised of all participants who received at least one infusion of study product
Health Resource Use: Emergency Room Visits PK assessment (N=11, 12, 23)	0.0 Visits Interval 0.0 to 0.0	0.0 Visits Interval 0.0 to 0.0	0.0 Visits Interval 0.0 to 0.0
Health Resource Use: Emergency Room Visits Week 5 (N=11, 12, 23)	0.0 Visits Interval 0.0 to 0.0	0.0 Visits Interval 0.0 to 0.0	0.0 Visits Interval 0.0 to 0.0
Health Resource Use: Emergency Room Visits Week 13 (N=11, 11, 22)	0.0 Visits Interval 0.0 to 2.0	0.0 Visits Interval 0.0 to 0.0	0.0 Visits Interval 0.0 to 2.0
Health Resource Use: Emergency Room Visits Week 26 (N=11, 11, 22)	0.0 Visits Interval 0.0 to 4.0	0.0 Visits Interval 0.0 to 1.0	0.0 Visits Interval 0.0 to 4.0

SECONDARY outcome

Timeframe: Baseline (Pharmacokinetic [PK] assessment), Week 5, Week 13 and Week 26

Population: Participants who missed days from school

The number of days lost from school per participant. Number of participants contributing data (N) for this outcome measure is included in the category title in the order: pediatric participants < 6 years of age; pediatric participants 6 to <12 years of age; Full Analysis Set.

Outcome measures

Measure	Pediatric Participants < 6 Years of Age n=11 Participants Pediatric participants \< 6 years of age. All pediatric participants underwent pharmacokinetic (PK) assessment followed by prophylactic treatment with the study product. All participants received the same dosing schedule of study product during the study. After a washout period of 5-7 days, participants received an initial infusion of study product at a dose of 75±5 IU/kg for the PK assessment. For the prophylactic regimen, participants were treated with the recommended dose of 50 IU/kg of study product twice weekly ranging from 40-80 IU/kg for 26±1 weeks or for at least 50 EDs to study product, whichever occurred last.	Pediatric Participants 6 to < 12 Years of Age n=12 Participants Pediatric participants 6 to \< 12 years of age. All pediatric participants underwent pharmacokinetic (PK) assessment followed by prophylactic treatment with the study product. All participants received the same dosing schedule of study product during the study. After a washout period of 5-7 days, participants received an initial infusion of study product at a dose of 75±5 IU/kg for the PK assessment. For the prophylactic regimen, participants were treated with the recommended dose of 50 IU/kg of study product twice weekly ranging from 40-80 IU/kg for 26±1 weeks or for at least 50 EDs to study product, whichever occurred last.	Full Analysis Set n=23 Participants Comprised of all participants who received at least one infusion of study product
Health Resource Use: Days Lost From School PK assessment (N=11, 12, 23)	0.0 Days Interval 0.0 to 0.0	0.0 Days Interval 0.0 to 6.0	0.0 Days Interval 0.0 to 6.0
Health Resource Use: Days Lost From School Week 5 (N=11, 12, 23)	0.0 Days Interval 0.0 to 9.0	0.0 Days Interval 0.0 to 0.0	0.0 Days Interval 0.0 to 9.0
Health Resource Use: Days Lost From School Week 13 (N=11, 11, 22)	0.0 Days Interval 0.0 to 14.0	0.0 Days Interval 0.0 to 4.0	0.0 Days Interval 0.0 to 14.0
Health Resource Use: Days Lost From School Week 26 (N=11, 11, 22)	0.0 Days Interval 0.0 to 6.0	0.0 Days Interval 0.0 to 11.0	0.0 Days Interval 0.0 to 11.0

Adverse Events

Full Analysis Set

Serious events: 3 serious events

Other events: 15 other events

Deaths: 0 deaths

Serious adverse events

Measure	Full Analysis Set n=23 participants at risk Comprised of all participants who received at least one infusion of study product
Infections and infestations Device related infection	4.3% 1/23 • Number of events 1 • Throughout study period (approximately 17 months) AEs were monitored from the screening visit until the study completion/termination visit.
Injury, poisoning and procedural complications Humerus fracture	4.3% 1/23 • Number of events 1 • Throughout study period (approximately 17 months) AEs were monitored from the screening visit until the study completion/termination visit.
Musculoskeletal and connective tissue disorders Hemarthrosis	4.3% 1/23 • Number of events 1 • Throughout study period (approximately 17 months) AEs were monitored from the screening visit until the study completion/termination visit.
Skin and subcutaneous tissue disorders Subcutaneous hemorrhage	4.3% 1/23 • Number of events 1 • Throughout study period (approximately 17 months) AEs were monitored from the screening visit until the study completion/termination visit.

Other adverse events

Measure	Full Analysis Set n=23 participants at risk Comprised of all participants who received at least one infusion of study product
Gastrointestinal disorders Abdominal pain	8.7% 2/23 • Number of events 2 • Throughout study period (approximately 17 months) AEs were monitored from the screening visit until the study completion/termination visit.
Gastrointestinal disorders Toothache	8.7% 2/23 • Number of events 2 • Throughout study period (approximately 17 months) AEs were monitored from the screening visit until the study completion/termination visit.
Infections and infestations Bronchitis	8.7% 2/23 • Number of events 2 • Throughout study period (approximately 17 months) AEs were monitored from the screening visit until the study completion/termination visit.
Infections and infestations Nasopharyngitis	8.7% 2/23 • Number of events 3 • Throughout study period (approximately 17 months) AEs were monitored from the screening visit until the study completion/termination visit.
Infections and infestations Respiratory tract infection viral	8.7% 2/23 • Number of events 2 • Throughout study period (approximately 17 months) AEs were monitored from the screening visit until the study completion/termination visit.
Infections and infestations Rhinitis	8.7% 2/23 • Number of events 4 • Throughout study period (approximately 17 months) AEs were monitored from the screening visit until the study completion/termination visit.
Infections and infestations Upper respiratory tract infection	13.0% 3/23 • Number of events 3 • Throughout study period (approximately 17 months) AEs were monitored from the screening visit until the study completion/termination visit.
Investigations Immunology test abnormal	26.1% 6/23 • Number of events 8 • Throughout study period (approximately 17 months) AEs were monitored from the screening visit until the study completion/termination visit.
Nervous system disorders Headache	8.7% 2/23 • Number of events 2 • Throughout study period (approximately 17 months) AEs were monitored from the screening visit until the study completion/termination visit.

Additional Information

Study Director

Shire

Phone: +1 866 842 5335

Email: ClinicalTransparency@shire.com

Results disclosure agreements

• Principal investigator is a sponsor employee Baxalta's agreements with PIs may vary per individual PI, but contain common elements. For this study, PIs may be restricted from independently publishing results without prior approval.
• Publication restrictions are in place

Restriction type: OTHER