Trial Outcomes & Findings for Study Evaluating On-Demand Treatment Of Xyntha In Chinese Subjects (NCT NCT00868530)

NCT ID: NCT00868530

Last Updated: 2025-06-12

Results Overview

The Investigator Hemostatic Efficacy Assessment was based on a 4-point rating scale (Excellent = 1: definite pain relief or improvement in signs of bleeding, with no additional infusion, Good = 2: definite pain relief or improvement in signs of bleeding, Moderate = 3: probable or slight improvement, No Response = 4: no improvement at all between infusions).

• Recruitment status: COMPLETED
• Study phase: PHASE3
• Target enrollment: 53 participants
• Primary outcome timeframe: 8 hours post infusion
• Results posted on: 2025-06-12

Participant Flow

Participants were recruited in China from September 2008 to December 2009.

Participant milestones

Measure	Xyntha Participants received on-demand treatments with Xyntha (which occurred each time a participant experienced bleeding episode during the active phase of the study) according to investigator's prescription over a 6-month (calendar day) period. A single 50 International Unit (IU)/kg (+/-5 IU/kg) intravenous (IV) bolus infusion of Xyntha was given for recovery assessments.
Overall Study STARTED	53
Overall Study COMPLETED	49
Overall Study NOT COMPLETED	4

Reasons for withdrawal

Measure	Xyntha Participants received on-demand treatments with Xyntha (which occurred each time a participant experienced bleeding episode during the active phase of the study) according to investigator's prescription over a 6-month (calendar day) period. A single 50 International Unit (IU)/kg (+/-5 IU/kg) intravenous (IV) bolus infusion of Xyntha was given for recovery assessments.
Overall Study Adverse Event	3
Overall Study Lost to Follow-up	1

Baseline Characteristics

Study Evaluating On-Demand Treatment Of Xyntha In Chinese Subjects

Baseline characteristics by cohort

Measure	Xyntha n=53 Participants Participants received on-demand treatments with Xyntha (which occurred each time a participant experienced bleeding episode during the active phase of the study) according to investigator's prescription over a 6-month (calendar day) period. A single 50 International Unit (IU)/kg (+/-5 IU/kg) intravenous (IV) bolus infusion of Xyntha was given for recovery assessments.
Age, Continuous	23.2 Years STANDARD_DEVIATION 10.0 • n=5 Participants
Sex: Female, Male Female	0 Participants n=5 Participants
Sex: Female, Male Male	53 Participants n=5 Participants

PRIMARY outcome

Timeframe: 8 hours post infusion

Population: The Full Analysis Set (FAS) consisted of all participants who were treated and had at least 1 evaluable efficacy assessment after treatment.

The Investigator Hemostatic Efficacy Assessment was based on a 4-point rating scale (Excellent = 1: definite pain relief or improvement in signs of bleeding, with no additional infusion, Good = 2: definite pain relief or improvement in signs of bleeding, Moderate = 3: probable or slight improvement, No Response = 4: no improvement at all between infusions).

Outcome measures

Measure	Xyntha n=51 Participants Participants received on-demand treatments with Xyntha (which occurred each time a participant experienced bleeding episode during the active phase of the study) according to investigator's prescription over a 6-month (calendar day) period. A single 50 International Unit (IU)/kg (+/-5 IU/kg) intravenous (IV) bolus infusion of Xyntha was given for recovery assessments.
Investigator Hemostatic Efficacy Assessment 8 Hours Post Infusion	1.86 Units on a scale Standard Deviation 0.65

PRIMARY outcome

Timeframe: 24 hours post infusion

Population: The FAS consisted of all participants who were treated and had at least 1 evaluable efficacy assessment after treatment.

The Investigator Hemostatic Efficacy Assessment was based on a 4-point rating scale (Excellent = 1: definite pain relief or improvement in signs of bleeding, with no additional infusion, Good = 2: definite pain relief or improvement in signs of bleeding, Moderate = 3: probable or slight improvement, No Response = 4: no improvement at all between infusions).

Outcome measures

Measure	Xyntha n=51 Participants Participants received on-demand treatments with Xyntha (which occurred each time a participant experienced bleeding episode during the active phase of the study) according to investigator's prescription over a 6-month (calendar day) period. A single 50 International Unit (IU)/kg (+/-5 IU/kg) intravenous (IV) bolus infusion of Xyntha was given for recovery assessments.
Investigator Hemostatic Efficacy Assessment 24 Hours Post Infusion	1.74 Units on a scale Standard Deviation 0.61

PRIMARY outcome

Timeframe: Day 1 and Month 6 or Early Termination Visit

Population: The Safety Set (SS) consisted of all participants who had taken at least 1 dose of investigational drug.

Incidence of FVIII inhibitor was defined as any result determined as positive at local laboratory, and confirmed at central laboratory. Incidence was stratified by participant exposure history: Minimally Treated Patients (MTPs): those who had received at least 1 prior FVIII infusion, and <= 100 documented Exposure Days (EDs), while Previously Treated Patients (PTPs): those who had received >100 documented prior EDs. When number of prior EDs for an individual was not known to be at least 100, participants were included in the MTP population.

Outcome measures

Measure	Xyntha n=53 Participants Participants received on-demand treatments with Xyntha (which occurred each time a participant experienced bleeding episode during the active phase of the study) according to investigator's prescription over a 6-month (calendar day) period. A single 50 International Unit (IU)/kg (+/-5 IU/kg) intravenous (IV) bolus infusion of Xyntha was given for recovery assessments.
Number of Participants With Factor VIII (FVIII) Inhibitor Development MTP (n=36)	6 Participants
Number of Participants With Factor VIII (FVIII) Inhibitor Development PTP (n=17)	1 Participants

SECONDARY outcome

Timeframe: Day 1 and Month 6 or Early Termination Visit

Population: The FAS consisted of all participants who were treated and had at least 1 evaluable efficacy assessment after treatment. Participants with missing data were not included.

FVIII recovery was assessed by evaluating the change in FVIII concentration at 6 months compared to baseline.

Outcome measures

Measure	Xyntha n=42 Participants Participants received on-demand treatments with Xyntha (which occurred each time a participant experienced bleeding episode during the active phase of the study) according to investigator's prescription over a 6-month (calendar day) period. A single 50 International Unit (IU)/kg (+/-5 IU/kg) intravenous (IV) bolus infusion of Xyntha was given for recovery assessments.
FVIII Recovery : Change From Baseline in FVIII Concentration	-0.11 IU/dL per IU/kg Standard Deviation 0.45

SECONDARY outcome

Timeframe: 24 hours after each of 2 successive infusion, up to 6 months

Population: The FAS consisted of all participants who were treated and had at least 1 evaluable efficacy assessment after treatment.

The incidence of LETE, defined for on-demand treatment as no response after each of 2 successive infusions within 24 hours for the same bleeding event in the absence of confounding factors.

Outcome measures

Measure	Xyntha n=51 Participants Participants received on-demand treatments with Xyntha (which occurred each time a participant experienced bleeding episode during the active phase of the study) according to investigator's prescription over a 6-month (calendar day) period. A single 50 International Unit (IU)/kg (+/-5 IU/kg) intravenous (IV) bolus infusion of Xyntha was given for recovery assessments.
Number of Participants With Less Than Expected Therapeutic Effect (LETE)	0 Participants

SECONDARY outcome

Timeframe: Baseline up to 6 months

Population: The Safety Set (SS) consisted of all participants who had taken at least 1 dose of investigational drug.

Outcome measures

Measure	Xyntha n=53 Participants Participants received on-demand treatments with Xyntha (which occurred each time a participant experienced bleeding episode during the active phase of the study) according to investigator's prescription over a 6-month (calendar day) period. A single 50 International Unit (IU)/kg (+/-5 IU/kg) intravenous (IV) bolus infusion of Xyntha was given for recovery assessments.
Number of Participants With Thrombosis Allergic-Type Reactions	1 Participants

SECONDARY outcome

Timeframe: Baseline up to 6 months

Population: The SS consisted of all participants who had taken at least 1 dose of investigational drug.

Outcome measures

Measure	Xyntha n=53 Participants Participants received on-demand treatments with Xyntha (which occurred each time a participant experienced bleeding episode during the active phase of the study) according to investigator's prescription over a 6-month (calendar day) period. A single 50 International Unit (IU)/kg (+/-5 IU/kg) intravenous (IV) bolus infusion of Xyntha was given for recovery assessments.
Number of Participants With Thrombosis	0 Participants

OTHER_PRE_SPECIFIED outcome

Timeframe: Day 1 to Month 6 or Early Termination Visit

Population: The FAS consisted of all participants who were treated and had at least 1 evaluable efficacy assessment after treatment.

The mean frequency of Xyntha infusions per hemorrhage was calculated as total number of injections throughout the study divided by total number of hemorrhagic events.

Outcome measures

Measure	Xyntha n=51 Participants Participants received on-demand treatments with Xyntha (which occurred each time a participant experienced bleeding episode during the active phase of the study) according to investigator's prescription over a 6-month (calendar day) period. A single 50 International Unit (IU)/kg (+/-5 IU/kg) intravenous (IV) bolus infusion of Xyntha was given for recovery assessments.
Frequency of Xyntha Infusions Required Per Hemorrhage	1.16 Infusions Standard Deviation 0.72

OTHER_PRE_SPECIFIED outcome

Timeframe: Day 1 to Month 6 or Early Termination Visit

Population: The FAS consisted of all participants who were treated and had at least 1 evaluable efficacy assessment after treatment.

The average dose of Xyntha per hemorrhagic event was calculated as total dose of Xyntha throughout the study (in IU) divided by total number of hemorrhage incidence.

Outcome measures

Measure	Xyntha n=51 Participants Participants received on-demand treatments with Xyntha (which occurred each time a participant experienced bleeding episode during the active phase of the study) according to investigator's prescription over a 6-month (calendar day) period. A single 50 International Unit (IU)/kg (+/-5 IU/kg) intravenous (IV) bolus infusion of Xyntha was given for recovery assessments.
Average Dose of Xyntha Infusions Required Per Hemorrhage	1226.28 Dose/Bleed (IU) Standard Deviation 1208.49

Adverse Events

Xyntha

Serious events: 9 serious events

Other events: 13 other events

Deaths: 0 deaths

Serious adverse events

Measure	Xyntha n=53 participants at risk Participants received on-demand treatments with Xyntha (which occurred each time a participant experienced bleeding episode during the active phase of the study) according to investigator's prescription over a 6-month (calendar day) period. A single 50 International Unit (IU)/kg (+/-5 IU/kg) intravenous (IV) bolus infusion of Xyntha was given for recovery assessments.
Gastrointestinal disorders Upper gastrointestinal haemorrhage	1.9% 1/53 • Number of events 1 • AEs and SAEs were collected from the signing of the informed consent form to approximately 30 days following the Month 6/Final/Early Termination visit. Safety population = subjects who received at least 1 dose of Xyntha. The same event may appear as both an AE and a SAE. However, what is presented are distinct events. An event may be categorized as serious in one subject and as nonserious in another subject, or one subject may have experienced both a serious and nonserious event during the study.
Gastrointestinal disorders Gingival bleeding	1.9% 1/53 • Number of events 1 • AEs and SAEs were collected from the signing of the informed consent form to approximately 30 days following the Month 6/Final/Early Termination visit. Safety population = subjects who received at least 1 dose of Xyntha. The same event may appear as both an AE and a SAE. However, what is presented are distinct events. An event may be categorized as serious in one subject and as nonserious in another subject, or one subject may have experienced both a serious and nonserious event during the study.
Investigations Anti factor VIII antibody positive	17.0% 9/53 • Number of events 9 • AEs and SAEs were collected from the signing of the informed consent form to approximately 30 days following the Month 6/Final/Early Termination visit. Safety population = subjects who received at least 1 dose of Xyntha. The same event may appear as both an AE and a SAE. However, what is presented are distinct events. An event may be categorized as serious in one subject and as nonserious in another subject, or one subject may have experienced both a serious and nonserious event during the study.

Other adverse events

Measure	Xyntha n=53 participants at risk Participants received on-demand treatments with Xyntha (which occurred each time a participant experienced bleeding episode during the active phase of the study) according to investigator's prescription over a 6-month (calendar day) period. A single 50 International Unit (IU)/kg (+/-5 IU/kg) intravenous (IV) bolus infusion of Xyntha was given for recovery assessments.
General disorders Pyrexia	1.9% 1/53 • Number of events 1 • AEs and SAEs were collected from the signing of the informed consent form to approximately 30 days following the Month 6/Final/Early Termination visit. Safety population = subjects who received at least 1 dose of Xyntha. The same event may appear as both an AE and a SAE. However, what is presented are distinct events. An event may be categorized as serious in one subject and as nonserious in another subject, or one subject may have experienced both a serious and nonserious event during the study.
Hepatobiliary disorders Hepatic steatosis	1.9% 1/53 • Number of events 1 • AEs and SAEs were collected from the signing of the informed consent form to approximately 30 days following the Month 6/Final/Early Termination visit. Safety population = subjects who received at least 1 dose of Xyntha. The same event may appear as both an AE and a SAE. However, what is presented are distinct events. An event may be categorized as serious in one subject and as nonserious in another subject, or one subject may have experienced both a serious and nonserious event during the study.
Immune system disorders Hypersensitivity	1.9% 1/53 • Number of events 2 • AEs and SAEs were collected from the signing of the informed consent form to approximately 30 days following the Month 6/Final/Early Termination visit. Safety population = subjects who received at least 1 dose of Xyntha. The same event may appear as both an AE and a SAE. However, what is presented are distinct events. An event may be categorized as serious in one subject and as nonserious in another subject, or one subject may have experienced both a serious and nonserious event during the study.
Injury, poisoning and procedural complications Fall	1.9% 1/53 • Number of events 1 • AEs and SAEs were collected from the signing of the informed consent form to approximately 30 days following the Month 6/Final/Early Termination visit. Safety population = subjects who received at least 1 dose of Xyntha. The same event may appear as both an AE and a SAE. However, what is presented are distinct events. An event may be categorized as serious in one subject and as nonserious in another subject, or one subject may have experienced both a serious and nonserious event during the study.
Injury, poisoning and procedural complications Injury	11.3% 6/53 • Number of events 8 • AEs and SAEs were collected from the signing of the informed consent form to approximately 30 days following the Month 6/Final/Early Termination visit. Safety population = subjects who received at least 1 dose of Xyntha. The same event may appear as both an AE and a SAE. However, what is presented are distinct events. An event may be categorized as serious in one subject and as nonserious in another subject, or one subject may have experienced both a serious and nonserious event during the study.
Injury, poisoning and procedural complications Joint dislocation	1.9% 1/53 • Number of events 1 • AEs and SAEs were collected from the signing of the informed consent form to approximately 30 days following the Month 6/Final/Early Termination visit. Safety population = subjects who received at least 1 dose of Xyntha. The same event may appear as both an AE and a SAE. However, what is presented are distinct events. An event may be categorized as serious in one subject and as nonserious in another subject, or one subject may have experienced both a serious and nonserious event during the study.
Injury, poisoning and procedural complications Joint injury	1.9% 1/53 • Number of events 1 • AEs and SAEs were collected from the signing of the informed consent form to approximately 30 days following the Month 6/Final/Early Termination visit. Safety population = subjects who received at least 1 dose of Xyntha. The same event may appear as both an AE and a SAE. However, what is presented are distinct events. An event may be categorized as serious in one subject and as nonserious in another subject, or one subject may have experienced both a serious and nonserious event during the study.
Injury, poisoning and procedural complications Joint sprain	1.9% 1/53 • Number of events 2 • AEs and SAEs were collected from the signing of the informed consent form to approximately 30 days following the Month 6/Final/Early Termination visit. Safety population = subjects who received at least 1 dose of Xyntha. The same event may appear as both an AE and a SAE. However, what is presented are distinct events. An event may be categorized as serious in one subject and as nonserious in another subject, or one subject may have experienced both a serious and nonserious event during the study.
Investigations Blood potassium decreased	3.8% 2/53 • Number of events 2 • AEs and SAEs were collected from the signing of the informed consent form to approximately 30 days following the Month 6/Final/Early Termination visit. Safety population = subjects who received at least 1 dose of Xyntha. The same event may appear as both an AE and a SAE. However, what is presented are distinct events. An event may be categorized as serious in one subject and as nonserious in another subject, or one subject may have experienced both a serious and nonserious event during the study.
Musculoskeletal and connective tissue disorders Arthritis	1.9% 1/53 • Number of events 1 • AEs and SAEs were collected from the signing of the informed consent form to approximately 30 days following the Month 6/Final/Early Termination visit. Safety population = subjects who received at least 1 dose of Xyntha. The same event may appear as both an AE and a SAE. However, what is presented are distinct events. An event may be categorized as serious in one subject and as nonserious in another subject, or one subject may have experienced both a serious and nonserious event during the study.
Gastrointestinal disorders Diarrhea	1.9% 1/53 • Number of events 1 • AEs and SAEs were collected from the signing of the informed consent form to approximately 30 days following the Month 6/Final/Early Termination visit. Safety population = subjects who received at least 1 dose of Xyntha. The same event may appear as both an AE and a SAE. However, what is presented are distinct events. An event may be categorized as serious in one subject and as nonserious in another subject, or one subject may have experienced both a serious and nonserious event during the study.

Additional Information

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Results disclosure agreements

• Principal investigator is a sponsor employee Pfizer has the right to review disclosures, requesting a delay of less than 60 days. Investigator will postpone single center publications until after disclosure of pooled data (all sites), less than 12 months from study completion/termination at all participating sites. Investigator may not disclose previously undisclosed confidential information other than study results.
• Publication restrictions are in place

Restriction type: OTHER