Trial Outcomes & Findings for A Safety, Efficacy and Pharmacokinetics Study of a Recombinant Fusion Protein Linking Coagulation Factor IX With Albumin (rIX-FP) in Children With Hemophilia B (NCT NCT01662531)

Last Updated: 2016-05-09

Results Overview

Incremental recovery (IU/dL/IU/kg) is defined as the FIX activity (IU/dL) obtained 30 minutes following infusion, per dose of (IU/kg) infusion. FIX activity was measured at a central laboratory using validated one-stage clotting method. Recovery values were baseline-corrected for pre-infusion plasma FIX activity. Incremental recovery was measured following a single intravenous dose of 50 IU/kg rIX-FP on Day 1. Analysis of previous FIX product was conducted at the beginning of the study in a subset of subjects who had no historical pharmacokinetic (PK) data of their previous FIX product. For the PK assessment, the previous FIX product was administered by IV infusion after approximately 4 days following the last FIX treatment, prior to any dosing of rIX-FP. The formal PK population consisted of subjects who received at least 1 dose of rIX-FP for PK assessment and for whom a sufficient number of analyzable PK samples had been obtained to permit the evaluation of the PK profile of rIX-FP.

Recruitment status

COMPLETED

Study phase

PHASE3

Target enrollment

27 participants

Primary outcome timeframe

30 minutes after infusion

Results posted on

2016-05-09

Participant Flow

Of the 18 sites that were activated, subjects were enrolled from 17 sites in 10 countries.

Subjects \<12 years of age with severe hemophilia B (FIX activity of ≤2%) were planned to be enrolled in the study, including 11 to 12 subjects in each age group (6 to \<12 years and \<6 years of age). Of 29 subjects screened, 27 subjects were enrolled and treated with rIX-FP.

Participant milestones

Participant milestones
Measure	rIX-FP Recombinant Fusion Protein Linking Coagulation Factor IX with Albumin (rIX-FP) will be administered by IV infusion as routine weekly prophylaxis and episodic treatment for bleeding episodes. rIX-FP: Recombinant Fusion Protein Linking Coagulation Factor IX with Albumin (rIX-FP)
Overall Study STARTED	27
Overall Study COMPLETED	27
Overall Study NOT COMPLETED	0

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

A Safety, Efficacy and Pharmacokinetics Study of a Recombinant Fusion Protein Linking Coagulation Factor IX With Albumin (rIX-FP) in Children With Hemophilia B

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Age < 6 Years n=12 Participants Subjects less than 6 years of age who received a single dose of 50 IU/kg rIX-FP on Day 1 during the pharmacokinetic phase of the study. Subjects received weekly (7-day) routine prophylaxis treatment with an initial weekly dose of 35 to 50 IU/kg rIX-FP, which may have been adjusted based on protocol-specified criteria.	Age 6 to <12 Years n=15 Participants Subjects between 6 and less than 12 years of age who received a single dose of 50 IU/kg rIX-FP on Day 1 during the pharmacokinetic phase of the study. Subjects received weekly (7-day) routine prophylaxis treatment with an initial weekly dose of 35 to 50 IU/kg rIX-FP, which may have been adjusted based on protocol-specified criteria.	Total n=27 Participants Total of all reporting groups
Age, Continuous	3.2 years STANDARD_DEVIATION 1.70 • n=5 Participants	8.1 years STANDARD_DEVIATION 1.41 • n=7 Participants	5.9 years STANDARD_DEVIATION 2.93 • 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	12 Participants n=5 Participants	15 Participants n=7 Participants	27 Participants n=5 Participants

PRIMARY outcome

Timeframe: 30 minutes after infusion

Population: PK Population

Outcome measures

Outcome measures
Measure	rIX-FP n=27 Participants All subjects received a single dose of 50 IU/kg rIX-FP on Day 1 during the pharmacokinetic phase of the study. Subjects received weekly (7-day) routine prophylaxis treatment with an initial weekly dose of 35 to 50 IU/kg rIX-FP, which may have been adjusted based on protocol-specified criteria.	Age < 6 Years n=12 Participants Subjects less than 6 years of age who received a single dose of 50 IU/kg rIX-FP on Day 1 during the pharmacokinetic phase of the study. Subjects received weekly (7-day) routine prophylaxis treatment with an initial weekly dose of 35 to 50 IU/kg rIX-FP, which may have been adjusted based on protocol-specified criteria.	Age 6 to <12 Years n=15 Participants Subjects between 6 and less than 12 years of age who received a single dose of 50 IU/kg rIX-FP on Day 1 during the pharmacokinetic phase of the study. Subjects received weekly (7-day) routine prophylaxis treatment with an initial weekly dose of 35 to 50 IU/kg rIX-FP, which may have been adjusted based on protocol-specified criteria.
Incremental Recovery Following a Single Intravenous Dose of 50 IU/kg rIX-FP or Previous FIX Product rIX-FP Assessment (n = 27, 12, 15)	1.0114 (IU/dL)/(IU/kg) Standard Deviation 0.22711	0.9506 (IU/dL)/(IU/kg) Standard Deviation 0.20432	1.0600 (IU/dL)/(IU/kg) Standard Deviation 0.23934
Incremental Recovery Following a Single Intravenous Dose of 50 IU/kg rIX-FP or Previous FIX Product Previous FIX Assessment (n = 17, 8, 9)	0.7379 (IU/dL)/(IU/kg) Standard Deviation 0.19768	0.6764 (IU/dL)/(IU/kg) Standard Deviation 0.13980	0.7925 (IU/dL)/(IU/kg) Standard Deviation 0.23219

PRIMARY outcome

Timeframe: Pre-dose, 30 minutes, 3, 24, 48, 72 120, 168, 240 and 336 hours post-dose

Population: PK Population

FIX activity was measured at a central laboratory using validated one-stage clotting method. FIX levels were not corrected for baseline values.

Outcome measures

Outcome measures
Measure	rIX-FP n=27 Participants All subjects received a single dose of 50 IU/kg rIX-FP on Day 1 during the pharmacokinetic phase of the study. Subjects received weekly (7-day) routine prophylaxis treatment with an initial weekly dose of 35 to 50 IU/kg rIX-FP, which may have been adjusted based on protocol-specified criteria.	Age < 6 Years n=12 Participants Subjects less than 6 years of age who received a single dose of 50 IU/kg rIX-FP on Day 1 during the pharmacokinetic phase of the study. Subjects received weekly (7-day) routine prophylaxis treatment with an initial weekly dose of 35 to 50 IU/kg rIX-FP, which may have been adjusted based on protocol-specified criteria.	Age 6 to <12 Years n=15 Participants Subjects between 6 and less than 12 years of age who received a single dose of 50 IU/kg rIX-FP on Day 1 during the pharmacokinetic phase of the study. Subjects received weekly (7-day) routine prophylaxis treatment with an initial weekly dose of 35 to 50 IU/kg rIX-FP, which may have been adjusted based on protocol-specified criteria.
Half-life (t1/2) Following a Single Intravenous Dose of 50 IU/kg rIX-FP or Previous FIX Product rIX-FP Assessment (n = 26, 11, 15)	91.4492 hours Standard Deviation 15.9754	89.6124 hours Standard Deviation 11.17364	92.7962 hours Standard Deviation 19.02537
Half-life (t1/2) Following a Single Intravenous Dose of 50 IU/kg rIX-FP or Previous FIX Product Previous FIX Assessment (n = 16, 7, 9)	18.6291 hours Standard Deviation 6.15551	19.8816 hours Standard Deviation 8.01073	17.6550 hours Standard Deviation 4.52497

PRIMARY outcome

Timeframe: Pre-dose, 30 minutes, 3, 24, 48, 72 120, 168, 240 and 336 hours post-dose

Population: PK Population

AUClast following a single intravenous dose of 50 IU/kg rIX-FP or previous FIX product. FIX activity was measured at a central laboratory using validated one-stage clotting method. FIX levels were not corrected for baseline values.

Outcome measures

Outcome measures
Measure	rIX-FP n=27 Participants All subjects received a single dose of 50 IU/kg rIX-FP on Day 1 during the pharmacokinetic phase of the study. Subjects received weekly (7-day) routine prophylaxis treatment with an initial weekly dose of 35 to 50 IU/kg rIX-FP, which may have been adjusted based on protocol-specified criteria.	Age < 6 Years n=12 Participants Subjects less than 6 years of age who received a single dose of 50 IU/kg rIX-FP on Day 1 during the pharmacokinetic phase of the study. Subjects received weekly (7-day) routine prophylaxis treatment with an initial weekly dose of 35 to 50 IU/kg rIX-FP, which may have been adjusted based on protocol-specified criteria.	Age 6 to <12 Years n=15 Participants Subjects between 6 and less than 12 years of age who received a single dose of 50 IU/kg rIX-FP on Day 1 during the pharmacokinetic phase of the study. Subjects received weekly (7-day) routine prophylaxis treatment with an initial weekly dose of 35 to 50 IU/kg rIX-FP, which may have been adjusted based on protocol-specified criteria.
Area Under the Concentration Versus Time Curve From Time Point Zero to the Last Sample With Quantifiable Drug Concentration (AUClast) rIX-FP Assessment (n = 27, 12, 15)	4156.7037 IU*hr/dL Standard Deviation 1204.09549	3891.4820 IU*hr/dL Standard Deviation 1252.99415	4368.8810 IU*hr/dL Standard Deviation 1162.09967
Area Under the Concentration Versus Time Curve From Time Point Zero to the Last Sample With Quantifiable Drug Concentration (AUClast) Previous FIX Assessment (n = 16, 7, 9)	718.9386 IU*hr/dL Standard Deviation 230.52884	676.5414 IU*hr/dL Standard Deviation 316.91381	751.9143 IU*hr/dL Standard Deviation 146.70454

PRIMARY outcome

Timeframe: Pre-dose, 30 minutes, 3, 24, 48, 72 120, 168, 240 and 336 hours post-dose

Population: PK Population

FIX activity was measured at a central laboratory using validated one-stage clotting method. FIX levels were not corrected for baseline values. Clearance is normalized for body weight.

Outcome measures

Outcome measures
Measure	rIX-FP n=27 Participants All subjects received a single dose of 50 IU/kg rIX-FP on Day 1 during the pharmacokinetic phase of the study. Subjects received weekly (7-day) routine prophylaxis treatment with an initial weekly dose of 35 to 50 IU/kg rIX-FP, which may have been adjusted based on protocol-specified criteria.	Age < 6 Years n=12 Participants Subjects less than 6 years of age who received a single dose of 50 IU/kg rIX-FP on Day 1 during the pharmacokinetic phase of the study. Subjects received weekly (7-day) routine prophylaxis treatment with an initial weekly dose of 35 to 50 IU/kg rIX-FP, which may have been adjusted based on protocol-specified criteria.	Age 6 to <12 Years n=15 Participants Subjects between 6 and less than 12 years of age who received a single dose of 50 IU/kg rIX-FP on Day 1 during the pharmacokinetic phase of the study. Subjects received weekly (7-day) routine prophylaxis treatment with an initial weekly dose of 35 to 50 IU/kg rIX-FP, which may have been adjusted based on protocol-specified criteria.
Clearance for FIX Activity Following a Single Intravenous Dose of 50 IU/kg rIX-FP or Previous FIX Product rIX-FP Assessment (n = 26, 11, 15)	1.1119 mL/hr/kg Standard Deviation 0.31373	1.1841 mL/hr/kg Standard Deviation 0.32924	1.0589 mL/hr/kg Standard Deviation 0.30203
Clearance for FIX Activity Following a Single Intravenous Dose of 50 IU/kg rIX-FP or Previous FIX Product Previous FIX Assessment (n = 16, 7, 9)	6.4007 mL/hr/kg Standard Deviation 2.14434	7.1576 mL/hr/kg Standard Deviation 2.78944	5.8119 mL/hr/kg Standard Deviation 1.37641

PRIMARY outcome

Timeframe: 12 months

Population: Safety Population

Inhibitor formation was defined as any inhibitor (≥0.6 BU \[Bethesda Units\]/mL) identified and confirmed by retesting.

Outcome measures

Outcome measures
Measure	rIX-FP n=27 Participants All subjects received a single dose of 50 IU/kg rIX-FP on Day 1 during the pharmacokinetic phase of the study. Subjects received weekly (7-day) routine prophylaxis treatment with an initial weekly dose of 35 to 50 IU/kg rIX-FP, which may have been adjusted based on protocol-specified criteria.	Age < 6 Years Subjects less than 6 years of age who received a single dose of 50 IU/kg rIX-FP on Day 1 during the pharmacokinetic phase of the study. Subjects received weekly (7-day) routine prophylaxis treatment with an initial weekly dose of 35 to 50 IU/kg rIX-FP, which may have been adjusted based on protocol-specified criteria.	Age 6 to <12 Years Subjects between 6 and less than 12 years of age who received a single dose of 50 IU/kg rIX-FP on Day 1 during the pharmacokinetic phase of the study. Subjects received weekly (7-day) routine prophylaxis treatment with an initial weekly dose of 35 to 50 IU/kg rIX-FP, which may have been adjusted based on protocol-specified criteria.
Number of Subjects Developing Inhibitors to Factor IX (FIX)	0 participants	—	—

SECONDARY outcome

Timeframe: 12 months

Population: Safety Population

Outcome measures

Outcome measures
Measure	rIX-FP n=27 Participants All subjects received a single dose of 50 IU/kg rIX-FP on Day 1 during the pharmacokinetic phase of the study. Subjects received weekly (7-day) routine prophylaxis treatment with an initial weekly dose of 35 to 50 IU/kg rIX-FP, which may have been adjusted based on protocol-specified criteria.	Age < 6 Years Subjects less than 6 years of age who received a single dose of 50 IU/kg rIX-FP on Day 1 during the pharmacokinetic phase of the study. Subjects received weekly (7-day) routine prophylaxis treatment with an initial weekly dose of 35 to 50 IU/kg rIX-FP, which may have been adjusted based on protocol-specified criteria.	Age 6 to <12 Years Subjects between 6 and less than 12 years of age who received a single dose of 50 IU/kg rIX-FP on Day 1 during the pharmacokinetic phase of the study. Subjects received weekly (7-day) routine prophylaxis treatment with an initial weekly dose of 35 to 50 IU/kg rIX-FP, which may have been adjusted based on protocol-specified criteria.
Number of Subjects With Treatment-related Adverse Events Any adverse event	26 participants	—	—
Number of Subjects With Treatment-related Adverse Events Treatment-related adverse event	0 participants	—	—

SECONDARY outcome

Timeframe: 12 months

Population: Safety Population

Antibodies to rIX-FP were measured using a direct-binding enzyme-linked immunosorbent assay (ELISA).

Outcome measures

Outcome measures
Measure	rIX-FP n=27 Participants All subjects received a single dose of 50 IU/kg rIX-FP on Day 1 during the pharmacokinetic phase of the study. Subjects received weekly (7-day) routine prophylaxis treatment with an initial weekly dose of 35 to 50 IU/kg rIX-FP, which may have been adjusted based on protocol-specified criteria.	Age < 6 Years Subjects less than 6 years of age who received a single dose of 50 IU/kg rIX-FP on Day 1 during the pharmacokinetic phase of the study. Subjects received weekly (7-day) routine prophylaxis treatment with an initial weekly dose of 35 to 50 IU/kg rIX-FP, which may have been adjusted based on protocol-specified criteria.	Age 6 to <12 Years Subjects between 6 and less than 12 years of age who received a single dose of 50 IU/kg rIX-FP on Day 1 during the pharmacokinetic phase of the study. Subjects received weekly (7-day) routine prophylaxis treatment with an initial weekly dose of 35 to 50 IU/kg rIX-FP, which may have been adjusted based on protocol-specified criteria.
Number of Subjects Developing Antibodies Against rIX-FP	0 participants	—	—

SECONDARY outcome

Timeframe: Approximately 12 months

Population: Efficacy Population

For each bleeding episode that required treatment, the number of episodes that required one, two or more than two infusions of rIX-FP to achieve hemostasis

Outcome measures

Outcome measures
Measure	rIX-FP n=27 Participants All subjects received a single dose of 50 IU/kg rIX-FP on Day 1 during the pharmacokinetic phase of the study. Subjects received weekly (7-day) routine prophylaxis treatment with an initial weekly dose of 35 to 50 IU/kg rIX-FP, which may have been adjusted based on protocol-specified criteria.	Age < 6 Years n=12 Participants Subjects less than 6 years of age who received a single dose of 50 IU/kg rIX-FP on Day 1 during the pharmacokinetic phase of the study. Subjects received weekly (7-day) routine prophylaxis treatment with an initial weekly dose of 35 to 50 IU/kg rIX-FP, which may have been adjusted based on protocol-specified criteria.	Age 6 to <12 Years n=15 Participants Subjects between 6 and less than 12 years of age who received a single dose of 50 IU/kg rIX-FP on Day 1 during the pharmacokinetic phase of the study. Subjects received weekly (7-day) routine prophylaxis treatment with an initial weekly dose of 35 to 50 IU/kg rIX-FP, which may have been adjusted based on protocol-specified criteria.
Number of Bleeding Episodes Requiring One, Two or More Than Two Infusions of rIX-FP to Achieve Hemostasis 1 infusion	94 bleeding episodes	40 bleeding episodes	54 bleeding episodes
Number of Bleeding Episodes Requiring One, Two or More Than Two Infusions of rIX-FP to Achieve Hemostasis 1 or 2 infusions	103 bleeding episodes	45 bleeding episodes	58 bleeding episodes
Number of Bleeding Episodes Requiring One, Two or More Than Two Infusions of rIX-FP to Achieve Hemostasis 2 infusions	9 bleeding episodes	5 bleeding episodes	4 bleeding episodes
Number of Bleeding Episodes Requiring One, Two or More Than Two Infusions of rIX-FP to Achieve Hemostasis > 2 infusions	3 bleeding episodes	0 bleeding episodes	3 bleeding episodes

SECONDARY outcome

Timeframe: 12 months

Population: Efficacy Population

Consumption of rIX-FP during routine prophylaxis is expressed as the total prophylaxis dose per month.

Outcome measures

Outcome measures
Measure	rIX-FP n=27 Participants All subjects received a single dose of 50 IU/kg rIX-FP on Day 1 during the pharmacokinetic phase of the study. Subjects received weekly (7-day) routine prophylaxis treatment with an initial weekly dose of 35 to 50 IU/kg rIX-FP, which may have been adjusted based on protocol-specified criteria.	Age < 6 Years n=12 Participants Subjects less than 6 years of age who received a single dose of 50 IU/kg rIX-FP on Day 1 during the pharmacokinetic phase of the study. Subjects received weekly (7-day) routine prophylaxis treatment with an initial weekly dose of 35 to 50 IU/kg rIX-FP, which may have been adjusted based on protocol-specified criteria.	Age 6 to <12 Years n=15 Participants Subjects between 6 and less than 12 years of age who received a single dose of 50 IU/kg rIX-FP on Day 1 during the pharmacokinetic phase of the study. Subjects received weekly (7-day) routine prophylaxis treatment with an initial weekly dose of 35 to 50 IU/kg rIX-FP, which may have been adjusted based on protocol-specified criteria.
Consumption of rIX-FP During Routine Prophylaxis	205.071 IU/kg/month Standard Deviation 41.1550	213.517 IU/kg/month Standard Deviation 44.3848	198.314 IU/kg/month Standard Deviation 38.5693

Adverse Events

rIX-FP

Serious events: 4 serious events

Other events: 23 other events

Deaths: 0 deaths

Serious adverse events

Serious adverse events
Measure	rIX-FP n=27 participants at risk All subjects received a single dose of 50 IU/kg rIX-FP on Day 1 during the pharmacokinetic phase of the study. Subjects received weekly (7-day) routine prophylaxis treatment with an initial weekly dose of 35 to 50 IU/kg rIX-FP, which may have been adjusted based on protocol-specified criteria.
Injury, poisoning and procedural complications Forearm fracture	3.7% 1/27 • Number of events 1 • 12 months Treatment-emergent adverse events, defined as adverse events present prior to the first dose of rIX-FP that subsequently worsened in severity or those that were not present prior to the first dose but subsequently appeared are summarized.
Injury, poisoning and procedural complications Head injury	3.7% 1/27 • Number of events 1 • 12 months Treatment-emergent adverse events, defined as adverse events present prior to the first dose of rIX-FP that subsequently worsened in severity or those that were not present prior to the first dose but subsequently appeared are summarized.
Injury, poisoning and procedural complications Tongue injury	3.7% 1/27 • Number of events 1 • 12 months Treatment-emergent adverse events, defined as adverse events present prior to the first dose of rIX-FP that subsequently worsened in severity or those that were not present prior to the first dose but subsequently appeared are summarized.
Musculoskeletal and connective tissue disorders Arthralgia	3.7% 1/27 • Number of events 2 • 12 months Treatment-emergent adverse events, defined as adverse events present prior to the first dose of rIX-FP that subsequently worsened in severity or those that were not present prior to the first dose but subsequently appeared are summarized.
Musculoskeletal and connective tissue disorders Groin pain	3.7% 1/27 • Number of events 1 • 12 months Treatment-emergent adverse events, defined as adverse events present prior to the first dose of rIX-FP that subsequently worsened in severity or those that were not present prior to the first dose but subsequently appeared are summarized.

Other adverse events

Other adverse events
Measure	rIX-FP n=27 participants at risk All subjects received a single dose of 50 IU/kg rIX-FP on Day 1 during the pharmacokinetic phase of the study. Subjects received weekly (7-day) routine prophylaxis treatment with an initial weekly dose of 35 to 50 IU/kg rIX-FP, which may have been adjusted based on protocol-specified criteria.
Injury, poisoning and procedural complications Contusion	18.5% 5/27 • Number of events 9 • 12 months Treatment-emergent adverse events, defined as adverse events present prior to the first dose of rIX-FP that subsequently worsened in severity or those that were not present prior to the first dose but subsequently appeared are summarized.
Injury, poisoning and procedural complications Injury	7.4% 2/27 • Number of events 4 • 12 months Treatment-emergent adverse events, defined as adverse events present prior to the first dose of rIX-FP that subsequently worsened in severity or those that were not present prior to the first dose but subsequently appeared are summarized.
Injury, poisoning and procedural complications Head injury	7.4% 2/27 • Number of events 2 • 12 months Treatment-emergent adverse events, defined as adverse events present prior to the first dose of rIX-FP that subsequently worsened in severity or those that were not present prior to the first dose but subsequently appeared are summarized.
Blood and lymphatic system disorders Anaemia	7.4% 2/27 • Number of events 2 • 12 months Treatment-emergent adverse events, defined as adverse events present prior to the first dose of rIX-FP that subsequently worsened in severity or those that were not present prior to the first dose but subsequently appeared are summarized.
Respiratory, thoracic and mediastinal disorders Cough	14.8% 4/27 • Number of events 4 • 12 months Treatment-emergent adverse events, defined as adverse events present prior to the first dose of rIX-FP that subsequently worsened in severity or those that were not present prior to the first dose but subsequently appeared are summarized.
Respiratory, thoracic and mediastinal disorders Oropharyngeal pain	7.4% 2/27 • Number of events 2 • 12 months Treatment-emergent adverse events, defined as adverse events present prior to the first dose of rIX-FP that subsequently worsened in severity or those that were not present prior to the first dose but subsequently appeared are summarized.
Nervous system disorders Headache	7.4% 2/27 • Number of events 4 • 12 months Treatment-emergent adverse events, defined as adverse events present prior to the first dose of rIX-FP that subsequently worsened in severity or those that were not present prior to the first dose but subsequently appeared are summarized.
General disorders Pyrexia	33.3% 9/27 • Number of events 14 • 12 months Treatment-emergent adverse events, defined as adverse events present prior to the first dose of rIX-FP that subsequently worsened in severity or those that were not present prior to the first dose but subsequently appeared are summarized.
Gastrointestinal disorders Dental discomfort	7.4% 2/27 • Number of events 2 • 12 months Treatment-emergent adverse events, defined as adverse events present prior to the first dose of rIX-FP that subsequently worsened in severity or those that were not present prior to the first dose but subsequently appeared are summarized.
Gastrointestinal disorders Diarrhoea	7.4% 2/27 • Number of events 2 • 12 months Treatment-emergent adverse events, defined as adverse events present prior to the first dose of rIX-FP that subsequently worsened in severity or those that were not present prior to the first dose but subsequently appeared are summarized.
Gastrointestinal disorders Toothache	7.4% 2/27 • Number of events 2 • 12 months Treatment-emergent adverse events, defined as adverse events present prior to the first dose of rIX-FP that subsequently worsened in severity or those that were not present prior to the first dose but subsequently appeared are summarized.
Gastrointestinal disorders Vomiting	7.4% 2/27 • Number of events 2 • 12 months Treatment-emergent adverse events, defined as adverse events present prior to the first dose of rIX-FP that subsequently worsened in severity or those that were not present prior to the first dose but subsequently appeared are summarized.
Musculoskeletal and connective tissue disorders Arthralgia	11.1% 3/27 • Number of events 3 • 12 months Treatment-emergent adverse events, defined as adverse events present prior to the first dose of rIX-FP that subsequently worsened in severity or those that were not present prior to the first dose but subsequently appeared are summarized.
Musculoskeletal and connective tissue disorders Pain in extremity	7.4% 2/27 • Number of events 2 • 12 months Treatment-emergent adverse events, defined as adverse events present prior to the first dose of rIX-FP that subsequently worsened in severity or those that were not present prior to the first dose but subsequently appeared are summarized.
Infections and infestations Nasopharyngitis	14.8% 4/27 • Number of events 6 • 12 months Treatment-emergent adverse events, defined as adverse events present prior to the first dose of rIX-FP that subsequently worsened in severity or those that were not present prior to the first dose but subsequently appeared are summarized.
Infections and infestations Bronchitis	11.1% 3/27 • Number of events 4 • 12 months Treatment-emergent adverse events, defined as adverse events present prior to the first dose of rIX-FP that subsequently worsened in severity or those that were not present prior to the first dose but subsequently appeared are summarized.
Infections and infestations Ear infection	11.1% 3/27 • Number of events 4 • 12 months Treatment-emergent adverse events, defined as adverse events present prior to the first dose of rIX-FP that subsequently worsened in severity or those that were not present prior to the first dose but subsequently appeared are summarized.
Infections and infestations Gastroenteritis	11.1% 3/27 • Number of events 3 • 12 months Treatment-emergent adverse events, defined as adverse events present prior to the first dose of rIX-FP that subsequently worsened in severity or those that were not present prior to the first dose but subsequently appeared are summarized.
Infections and infestations Pharyngitis	7.4% 2/27 • Number of events 3 • 12 months Treatment-emergent adverse events, defined as adverse events present prior to the first dose of rIX-FP that subsequently worsened in severity or those that were not present prior to the first dose but subsequently appeared are summarized.
Infections and infestations Viral infection	7.4% 2/27 • Number of events 3 • 12 months Treatment-emergent adverse events, defined as adverse events present prior to the first dose of rIX-FP that subsequently worsened in severity or those that were not present prior to the first dose but subsequently appeared are summarized.
Infections and infestations Molluscum contagiosum	7.4% 2/27 • Number of events 2 • 12 months Treatment-emergent adverse events, defined as adverse events present prior to the first dose of rIX-FP that subsequently worsened in severity or those that were not present prior to the first dose but subsequently appeared are summarized.
Infections and infestations Upper respiratory tract infection	7.4% 2/27 • Number of events 2 • 12 months Treatment-emergent adverse events, defined as adverse events present prior to the first dose of rIX-FP that subsequently worsened in severity or those that were not present prior to the first dose but subsequently appeared are summarized.

Additional Information

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Principal investigator is a sponsor employee CSL agreements and restrictions on publishing may vary with individual investigators; however, CSL will not prohibit any investigator from publishing. CSL supports the publication of results from all centers of a multi-center trial and generally requires that reports based on single-site data not precede the primary publication of the entire clinical trial.
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