Trial Outcomes & Findings for Study Evaluating On-Demand Treatment With BeneFIX In Chinese Subjects (NCT NCT00866606)

Last Updated: 2011-04-05

Results Overview

Investigator Hemostatic Efficacy Assessment was based on response of bleeding episodes to BeneFIX treatment on 4-point rating scale: Excellent(1): definite pain relief or improvement in signs of bleeding starting within 8 hrs after infusion, with no additional infusion; Good(2): definite pain relief or improvement in signs of bleeding starting within 8 hrs or following infusion; Moderate(3): probable or slight improvement starting after 8 hours following infusion; No Response(4): no improvement at all between infusions or during 24 hour interval following an infusion, or condition worsens.

Recruitment status

COMPLETED

Study phase

PHASE3

Target enrollment

35 participants

Primary outcome timeframe

8 hours post infusion

Results posted on

2011-04-05

Participant Flow

Participants were enrolled in six centers in China.

Participant milestones

Participant milestones
Measure	BeneFactor IX (BeneFIX) Participants received on-demand treatments with BeneFIX according to investigator's prescription over a 6-month (calendar day) period. A single 75 International Unit (IU)/kg (±5 IU/kg) intravenous (IV) bolus infusion of BeneFIX was given for recovery assessments. All BeneFIX administrations occurred in the clinic (hospital).
Overall Study STARTED	35
Overall Study COMPLETED	31
Overall Study NOT COMPLETED	4

Reasons for withdrawal

Reasons for withdrawal
Measure	BeneFactor IX (BeneFIX) Participants received on-demand treatments with BeneFIX according to investigator's prescription over a 6-month (calendar day) period. A single 75 International Unit (IU)/kg (±5 IU/kg) intravenous (IV) bolus infusion of BeneFIX was given for recovery assessments. All BeneFIX administrations occurred in the clinic (hospital).
Overall Study Lost to Follow-up	3
Overall Study Adverse Event	1

Baseline Characteristics

Study Evaluating On-Demand Treatment With BeneFIX In Chinese Subjects

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	BeneFactor IX (BeneFIX) n=35 Participants Participants received on-demand treatments with BeneFIX according to investigator's prescription over a 6-month (calendar day) period. A single 75 International Unit (IU)/kg (±5 IU/kg) intravenous (IV) bolus infusion of BeneFIX was given for recovery assessments. All BeneFIX administrations occurred in the clinic (hospital).
Age Continuous	25.7 Years STANDARD_DEVIATION 13.6 • n=5 Participants
Sex: Female, Male Female	0 Participants n=5 Participants
Sex: Female, Male Male	35 Participants n=5 Participants

PRIMARY outcome

Timeframe: 8 hours post infusion

Population: FAS population included all participants who were treated and had at least 1 evaluable efficacy assessment after treatment.

Outcome measures

Outcome measures
Measure	BeneFactor IX (BeneFIX) n=34 Participants Participants received on-demand treatments with BeneFIX according to investigator's prescription over a 6-month (calendar day) period. A single 75 International Unit (IU)/kg (±5 IU/kg) intravenous (IV) bolus infusion of BeneFIX was given for recovery assessments. All BeneFIX administrations occurred in the clinic (hospital).
Investigator Hemostatic Efficacy Assessment of Participants After 8 Hours Post Infusion	1.70 Units on a scale Standard Deviation 0.72

PRIMARY outcome

Timeframe: 24 hours post infusion

Population: FAS population included all participants who were treated and had at least 1 evaluable efficacy assessment after treatment.

Outcome measures

Outcome measures
Measure	BeneFactor IX (BeneFIX) n=34 Participants Participants received on-demand treatments with BeneFIX according to investigator's prescription over a 6-month (calendar day) period. A single 75 International Unit (IU)/kg (±5 IU/kg) intravenous (IV) bolus infusion of BeneFIX was given for recovery assessments. All BeneFIX administrations occurred in the clinic (hospital).
Investigator Hemostatic Efficacy Assessment of Participants After 24 Hours Post Infusion	1.58 Units on a scale Standard Deviation 0.71

PRIMARY outcome

Timeframe: Baseline up to 6 months

Population: Safety Set (SS) population included all enrolled participants who had taken at least 1 dose of drug.The 'n' is signifying those participants who received study drug and were evaluated for this measure at the timepoint for each visit respectively.

Incidence of FIX inhibitor was defined as any result determined as positive at local laboratory, and confirmed at central laboratory with Nijmegen assay result \>=0.6 Bethesda Unit (BU). Incidence was stratified by participant exposure history - Minimally Treated Patients (MTPs): those who had received at least one prior FIX 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

Outcome measures
Measure	BeneFactor IX (BeneFIX) n=35 Participants Participants received on-demand treatments with BeneFIX according to investigator's prescription over a 6-month (calendar day) period. A single 75 International Unit (IU)/kg (±5 IU/kg) intravenous (IV) bolus infusion of BeneFIX was given for recovery assessments. All BeneFIX administrations occurred in the clinic (hospital).
Percentage of Participants With FIX Inhibitor Development Total (n=35)	2.86 Percentage of Participants
Percentage of Participants With FIX Inhibitor Development MTP (n=24)	4.17 Percentage of Participants
Percentage of Participants With FIX Inhibitor Development PTP (n=11)	0 Percentage of Participants

SECONDARY outcome

Timeframe: Baseline up to 6 months

Population: FAS population included all participants who were treated and had at least 1 evaluable efficacy assessment after treatment.

The number of BeneFIX infusions required to treat each bleeding episode were analyzed. The average frequency of BeneFIX infusions per hemorrhage incidence to treat every hemorrhage was equal to the total number of injections throughout the study divided by total number of hemorrhagic events.

Outcome measures

Outcome measures
Measure	BeneFactor IX (BeneFIX) n=34 Participants Participants received on-demand treatments with BeneFIX according to investigator's prescription over a 6-month (calendar day) period. A single 75 International Unit (IU)/kg (±5 IU/kg) intravenous (IV) bolus infusion of BeneFIX was given for recovery assessments. All BeneFIX administrations occurred in the clinic (hospital).
Number of Infusions Required to Treat Each Bleed	1.20 Infusions Standard Deviation 0.86

SECONDARY outcome

Timeframe: Baseline (Visit 2) up to 6 months (Visit 4)

Population: FAS population included all participants who were treated and had at least 1 evaluable efficacy assessment after treatment. The 'n' is signifying those participants who received study drug and were evaluated for this measure at the timepoint for each visit respectively.

FIX recovery was assessed by evaluating FIX:C after initial exposure and following 6 months of repeated exposures to BeneFIX. A modified FIX recovery study was performed at Day 1 (Visit 2) and Month 6/Final/Early Termination visits (Visit 4) and when clinically indicated at the applicable on-demand visits. Blood samples for determination of FIX:C were collected immediately before BeneFIX infusion and at 30 minutes (±5 minutes) after the start of infusion. Post-infusion blood samples were collected via venipuncture in arm contralateral to arm used for infusion.

Outcome measures

Outcome measures
Measure	BeneFactor IX (BeneFIX) n=34 Participants Participants received on-demand treatments with BeneFIX according to investigator's prescription over a 6-month (calendar day) period. A single 75 International Unit (IU)/kg (±5 IU/kg) intravenous (IV) bolus infusion of BeneFIX was given for recovery assessments. All BeneFIX administrations occurred in the clinic (hospital).
FIX Incremental Recovery Visit 2 (n=30)	0.760 IU/dL per IU/kg Standard Deviation 0.222
FIX Incremental Recovery Visit 4 (n=31)	0.727 IU/dL per IU/kg Standard Deviation 0.275

SECONDARY outcome

Timeframe: Baseline up to 6 months

Population: FAS population included all participants who were treated and had at least 1 evaluable efficacy assessment after treatment.

The incidence of LETE for on-demand treatment was defined 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

Outcome measures
Measure	BeneFactor IX (BeneFIX) n=34 Participants Participants received on-demand treatments with BeneFIX according to investigator's prescription over a 6-month (calendar day) period. A single 75 International Unit (IU)/kg (±5 IU/kg) intravenous (IV) bolus infusion of BeneFIX was given for recovery assessments. All BeneFIX administrations occurred in the clinic (hospital).
Percentage of Participants With Less Than Expected Therapeutic Effect (LETE)	0 Percentage of Participants

SECONDARY outcome

Timeframe: Baseline up to 6 months

Population: SS population included all enrolled participants who had taken at least 1 dose of drug.

Hypersensitivity to undesirable (damaging, discomfort-producing and sometimes fatal) reactions produced by the normal immune system. Hypersensitivity reactions require a pre-sensitized (immune) state of the host.

Outcome measures

Outcome measures
Measure	BeneFactor IX (BeneFIX) n=35 Participants Participants received on-demand treatments with BeneFIX according to investigator's prescription over a 6-month (calendar day) period. A single 75 International Unit (IU)/kg (±5 IU/kg) intravenous (IV) bolus infusion of BeneFIX was given for recovery assessments. All BeneFIX administrations occurred in the clinic (hospital).
Percentage of Participants With Allergic-Type Allergic Reactions	0 Percentage of Participants

SECONDARY outcome

Timeframe: Baseline up to 6 months

Population: SS population included all enrolled participants who had taken at least 1 dose of drug.

Thrombosis is the formation of a blood clot (thrombus) inside a blood vessel, obstructing the flow of blood through the circulatory system. When a blood vessel is injured, the body uses platelets and fibrin to form a blood clot to prevent blood loss.

Outcome measures

Outcome measures
Measure	BeneFactor IX (BeneFIX) n=35 Participants Participants received on-demand treatments with BeneFIX according to investigator's prescription over a 6-month (calendar day) period. A single 75 International Unit (IU)/kg (±5 IU/kg) intravenous (IV) bolus infusion of BeneFIX was given for recovery assessments. All BeneFIX administrations occurred in the clinic (hospital).
Percentage of Participants With Thrombosis	0 Percentage of Participants

SECONDARY outcome

Timeframe: Baseline up to 6 months

Population: SS population included all enrolled participants who had taken at least 1 dose of drug.

RBC Agglutination is the clumping of red blood cells in the presence of an antibody. The antibody or other molecule bonded multiple particles and joined them, creating a large complex.

Outcome measures

Outcome measures
Measure	BeneFactor IX (BeneFIX) n=35 Participants Participants received on-demand treatments with BeneFIX according to investigator's prescription over a 6-month (calendar day) period. A single 75 International Unit (IU)/kg (±5 IU/kg) intravenous (IV) bolus infusion of BeneFIX was given for recovery assessments. All BeneFIX administrations occurred in the clinic (hospital).
Percentage of Participants With Red Blood Cell (RBC) Agglutination	0 Percentage of Participants

Adverse Events

BeneFactor IX (BeneFIX)

Serious events: 2 serious events

Other events: 19 other events

Deaths: 0 deaths

Serious adverse events

Serious adverse events
Measure	BeneFactor IX (BeneFIX) n=35 participants at risk Participants received on-demand treatments with BeneFIX according to investigator's prescription over a 6-month (calendar day) period. A single 75 International Unit (IU)/kg (±5 IU/kg) intravenous (IV) bolus infusion of BeneFIX was given for recovery assessments. All BeneFIX administrations occurred in the clinic (hospital).
Nervous system disorders Hemorrhage intracranial	2.9% 1/35 • Number of events 1 • From screening until 30 days after the last visit (Month 6/Final/Early Termination visit 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 participant and as non-serious in another participant, or one participant may have experienced both a serious and non-serious event during the study.
Congenital, familial and genetic disorders Hemophilia B with anti factor IX	2.9% 1/35 • Number of events 1 • From screening until 30 days after the last visit (Month 6/Final/Early Termination visit 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 participant and as non-serious in another participant, or one participant may have experienced both a serious and non-serious event during the study.

Other adverse events

Other adverse events
Measure	BeneFactor IX (BeneFIX) n=35 participants at risk Participants received on-demand treatments with BeneFIX according to investigator's prescription over a 6-month (calendar day) period. A single 75 International Unit (IU)/kg (±5 IU/kg) intravenous (IV) bolus infusion of BeneFIX was given for recovery assessments. All BeneFIX administrations occurred in the clinic (hospital).
Blood and lymphatic system disorders Anemia	5.7% 2/35 • Number of events 2 • From screening until 30 days after the last visit (Month 6/Final/Early Termination visit 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 participant and as non-serious in another participant, or one participant may have experienced both a serious and non-serious event during the study.
Gastrointestinal disorders Abdominal pain	2.9% 1/35 • Number of events 1 • From screening until 30 days after the last visit (Month 6/Final/Early Termination visit 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 participant and as non-serious in another participant, or one participant may have experienced both a serious and non-serious event during the study.
General disorders Fatigue	2.9% 1/35 • Number of events 1 • From screening until 30 days after the last visit (Month 6/Final/Early Termination visit 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 participant and as non-serious in another participant, or one participant may have experienced both a serious and non-serious event during the study.
General disorders Inflammation	5.7% 2/35 • Number of events 2 • From screening until 30 days after the last visit (Month 6/Final/Early Termination visit 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 participant and as non-serious in another participant, or one participant may have experienced both a serious and non-serious event during the study.
General disorders Edema peripheral	2.9% 1/35 • Number of events 1 • From screening until 30 days after the last visit (Month 6/Final/Early Termination visit 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 participant and as non-serious in another participant, or one participant may have experienced both a serious and non-serious event during the study.
Injury, poisoning and procedural complications Fall	2.9% 1/35 • Number of events 1 • From screening until 30 days after the last visit (Month 6/Final/Early Termination visit 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 participant and as non-serious in another participant, or one participant may have experienced both a serious and non-serious event during the study.
Injury, poisoning and procedural complications Injury	5.7% 2/35 • Number of events 2 • From screening until 30 days after the last visit (Month 6/Final/Early Termination visit 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 participant and as non-serious in another participant, or one participant may have experienced both a serious and non-serious event during the study.
Injury, poisoning and procedural complications Joint Sprain	5.7% 2/35 • Number of events 2 • From screening until 30 days after the last visit (Month 6/Final/Early Termination visit 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 participant and as non-serious in another participant, or one participant may have experienced both a serious and non-serious event during the study.
Investigations Alanine aminotransferase increased	2.9% 1/35 • Number of events 1 • From screening until 30 days after the last visit (Month 6/Final/Early Termination visit 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 participant and as non-serious in another participant, or one participant may have experienced both a serious and non-serious event during the study.
Investigations Hematocrit abnormal	2.9% 1/35 • Number of events 1 • From screening until 30 days after the last visit (Month 6/Final/Early Termination visit 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 participant and as non-serious in another participant, or one participant may have experienced both a serious and non-serious event during the study.
Investigations Hemoglobin abnormal	2.9% 1/35 • Number of events 1 • From screening until 30 days after the last visit (Month 6/Final/Early Termination visit 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 participant and as non-serious in another participant, or one participant may have experienced both a serious and non-serious event during the study.
Investigations Red blood cell count abnormal	2.9% 1/35 • Number of events 1 • From screening until 30 days after the last visit (Month 6/Final/Early Termination visit 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 participant and as non-serious in another participant, or one participant may have experienced both a serious and non-serious event during the study.
Nervous system disorders Vertigo	2.9% 1/35 • Number of events 1 • From screening until 30 days after the last visit (Month 6/Final/Early Termination visit 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 participant and as non-serious in another participant, or one participant may have experienced both a serious and non-serious event during the study.
Musculoskeletal and connective tissue disorders Synovitis	2.9% 1/35 • Number of events 1 • From screening until 30 days after the last visit (Month 6/Final/Early Termination visit 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 participant and as non-serious in another participant, or one participant may have experienced both a serious and non-serious event during the study.
Vascular disorders Hemorrhage	2.9% 1/35 • Number of events 1 • From screening until 30 days after the last visit (Month 6/Final/Early Termination visit 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 participant and as non-serious in another participant, or one participant may have experienced both a serious and non-serious event during the study.

Additional Information

Pfizer ClinicalTrials.gov Call Center

Pfizer, Inc.

Phone: 1-800-718-1021

Email: [email protected]

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