Trial Outcomes & Findings for Post Marketing Surveillance To Observe Safety And Efficacy Of Xyntha® In Subjects With Hemophilia A (NCT NCT01790828)

NCT ID: NCT01790828

Last Updated: 2015-08-17

Results Overview

• Recruitment status: COMPLETED
• Target enrollment: 42 participants
• Primary outcome timeframe: 4 years
• Results posted on: 2015-08-17

Participant Flow

Participant milestones

Measure	Xyntha Non-interventional, observational study of participants who were prescribed and received at least 1 dose of Xyntha 250, 500, 1000, or 2000 IU.
Overall Study STARTED	42
Overall Study COMPLETED	8
Overall Study NOT COMPLETED	34

Reasons for withdrawal

Measure	Xyntha Non-interventional, observational study of participants who were prescribed and received at least 1 dose of Xyntha 250, 500, 1000, or 2000 IU.
Overall Study Other	34

Baseline Characteristics

Post Marketing Surveillance To Observe Safety And Efficacy Of Xyntha® In Subjects With Hemophilia A

Baseline characteristics by cohort

Measure	Xyntha n=42 Participants Non-interventional, observational study of participants who were prescribed and received at least 1 dose of Xyntha 250, 500, 1000, or 2000 IU.
Age, Continuous	36.4 years STANDARD_DEVIATION 14.50 • n=5 Participants
Sex: Female, Male Female	2 Participants n=5 Participants
Sex: Female, Male Male	40 Participants n=5 Participants

PRIMARY outcome

Timeframe: 4 years

Population: All enrolled participants.

Outcome measures

Measure	Xyntha n=42 Participants Non-interventional, observational study of participants who were prescribed and received at least 1 dose of Xyntha 250, 500, 1000, or 2000 IU.
Percentage of Participants by Family History of Factor VIII Inhibitor Yes	0 percentage of participants
Percentage of Participants by Family History of Factor VIII Inhibitor No	100.0 percentage of participants

SECONDARY outcome

Timeframe: 4 years

Population: All enrolled participants; only participants who received on-demand therapy were included in the analysis.

Outcome measures

Measure	Xyntha n=5 Participants Non-interventional, observational study of participants who were prescribed and received at least 1 dose of Xyntha 250, 500, 1000, or 2000 IU.
Number of Xyntha Infusions Used to Treat Each New Bleed for On-Demand Therapy (All Participants)	1.40 infusions Standard Deviation 0.55

SECONDARY outcome

Timeframe: 4 years

Population: All enrolled participants; only participants aged \<18 years who received on-demand therapy were included in the analysis.

Outcome measures

Measure	Xyntha n=1 Participants Non-interventional, observational study of participants who were prescribed and received at least 1 dose of Xyntha 250, 500, 1000, or 2000 IU.
Number of Xyntha Infusions Used to Treat Each New Bleed for On-Demand Therapy in Participants Less Than (<)18 Years of Age	1.00 infusions Standard Deviation 0

SECONDARY outcome

Timeframe: 4 years

Population: All enrolled participants; participants aged ≥18 years who received on-demand therapy were included in the analysis.

Outcome measures

Measure	Xyntha n=4 Participants Non-interventional, observational study of participants who were prescribed and received at least 1 dose of Xyntha 250, 500, 1000, or 2000 IU.
Number of Xyntha Infusions Used to Treat Each New Bleed for On-Demand Therapy in Participants Greater Than or Equal to (≥) 18 Years of Age	1.50 infusions Standard Deviation 0.58

SECONDARY outcome

Timeframe: 4 years

Population: All enrolled participants; only participants who received on-demand therapy were included in the analysis.

Response categories were excellent, good, moderate, or no response.

Outcome measures

Measure	Xyntha n=7 infusions Non-interventional, observational study of participants who were prescribed and received at least 1 dose of Xyntha 250, 500, 1000, or 2000 IU.
Number of Responses by Type of Response for All Xyntha Infusions for Treatment of a Bleed for On-Demand Therapy (All Participants) Excellent	5 Number of responses
Number of Responses by Type of Response for All Xyntha Infusions for Treatment of a Bleed for On-Demand Therapy (All Participants) Good	2 Number of responses
Number of Responses by Type of Response for All Xyntha Infusions for Treatment of a Bleed for On-Demand Therapy (All Participants) Moderate	0 Number of responses
Number of Responses by Type of Response for All Xyntha Infusions for Treatment of a Bleed for On-Demand Therapy (All Participants) No response	0 Number of responses

SECONDARY outcome

Timeframe: 4 years

Population: All enrolled participants; only participants \<18 years who received on-demand therapy were included in the analysis.

Response categories were excellent, good, moderate, or no response.

Outcome measures

Measure	Xyntha n=1 infusions Non-interventional, observational study of participants who were prescribed and received at least 1 dose of Xyntha 250, 500, 1000, or 2000 IU.
Number of Responses by Type of Response for All Xyntha Infusions for Treatment of a Bleed for On-Demand Therapy in Participants <18 Years of Age Good	0 number of responses
Number of Responses by Type of Response for All Xyntha Infusions for Treatment of a Bleed for On-Demand Therapy in Participants <18 Years of Age Excellent	1 number of responses
Number of Responses by Type of Response for All Xyntha Infusions for Treatment of a Bleed for On-Demand Therapy in Participants <18 Years of Age Moderate	0 number of responses
Number of Responses by Type of Response for All Xyntha Infusions for Treatment of a Bleed for On-Demand Therapy in Participants <18 Years of Age No response	0 number of responses

SECONDARY outcome

Timeframe: 4 years

Population: All enrolled participants; only participants ≥18 years who receive don-demand therapy were included in the analysis.

Response categories were excellent, good, moderate, and no response.

Outcome measures

Measure	Xyntha n=6 infusions Non-interventional, observational study of participants who were prescribed and received at least 1 dose of Xyntha 250, 500, 1000, or 2000 IU.
Number of Responses by Type of Response for All Xyntha Infusions for Treatment of a Bleed for On-Demand Therapy in Participants ≥18 Years of Age Excellent	4 number of responses
Number of Responses by Type of Response for All Xyntha Infusions for Treatment of a Bleed for On-Demand Therapy in Participants ≥18 Years of Age Good	2 number of responses
Number of Responses by Type of Response for All Xyntha Infusions for Treatment of a Bleed for On-Demand Therapy in Participants ≥18 Years of Age Moderate	0 number of responses
Number of Responses by Type of Response for All Xyntha Infusions for Treatment of a Bleed for On-Demand Therapy in Participants ≥18 Years of Age No response	0 number of responses

SECONDARY outcome

Timeframe: 4 years

Population: Data were not analyzed as no participants experienced rating of 'no response'.

Less than expected therapeutic effect was defined as a 'no response' rating after each of two successive infusions less than or equl to (≤) 24 hours of on-demand therapy.

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: 4 years

Population: All enrolled participants; only participants who received on-demand therapy were included in the analysis.

Outcome measures

Measure	Xyntha n=5 Participants Non-interventional, observational study of participants who were prescribed and received at least 1 dose of Xyntha 250, 500, 1000, or 2000 IU.
Average Infusion Dose Per Bleeding for On-Demand Therapy (All Participants)	1997.00 IU Standard Deviation 892.77

SECONDARY outcome

Timeframe: 4 years

Population: All enrolled participants; only participants \<18 years who received on-demand therapy were included in the analysis.

Outcome measures

Measure	Xyntha n=1 Participants Non-interventional, observational study of participants who were prescribed and received at least 1 dose of Xyntha 250, 500, 1000, or 2000 IU.
Average Infusion Dose Per Bleeding for On-Demand Therapy in Participants <18 Years of Age	725.00 IU Full Range 892.77 • Interval 725.0 to 725.0

SECONDARY outcome

Timeframe: 4 years

Population: All enrolled participants; only participants ≥18 years who received on-demand therapy were included in the analysis.

Outcome measures

Measure	Xyntha n=4 Participants Non-interventional, observational study of participants who were prescribed and received at least 1 dose of Xyntha 250, 500, 1000, or 2000 IU.
Average Infusion Dose Per Bleeding for On-Demand Therapy in Participants ≥18 Years of Age	2315.00 IU Standard Deviation 623.00

SECONDARY outcome

Timeframe: 4 years

Population: All enrolled participants; n (number) equals (=) number of participants with nonmissing values

Outcome measures

Measure	Xyntha n=41 Participants Non-interventional, observational study of participants who were prescribed and received at least 1 dose of Xyntha 250, 500, 1000, or 2000 IU.
Percentage of Participants Experiencing Hemorrhages During Prophylaxis All participants (n=41)	0 percentage of participants
Percentage of Participants Experiencing Hemorrhages During Prophylaxis Participants <18 years (n=4)	0 percentage of participants
Percentage of Participants Experiencing Hemorrhages During Prophylaxis Participants ≥18 years (n=37)	0 percentage of participants

SECONDARY outcome

Timeframe: 4 years

Population: Data were not analyzed as information on breakthrough bleeds within 48 hours was not collected.

Annualized bleeding rate defined as total number of breakthrough bleeds within 48 hours (for prophylaxis purpose) divided by (/) [(total period of date of bleeding)/365.25)]

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: 4 years

Population: Data were not analyzed as information on breakthrough bleeds within 48 hours was not collected.

Less than expected therapeutic effect for prophylaxis therapy defined as breakthrough (spontaneous/non-traumatic) bleed wtihin 48 hours of prophylaxis infusion.

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: 4 years

Population: All enrolled participants; only participants with nonmissing data were included in the analysis.

Outcome measures

Measure	Xyntha n=41 Participants Non-interventional, observational study of participants who were prescribed and received at least 1 dose of Xyntha 250, 500, 1000, or 2000 IU.
Average Infusion Dose During Prophylaxis (All Participants)	1926.70 IU Standard Deviation 532.90

SECONDARY outcome

Timeframe: 4 years

Population: All enrolled participants; only participants \<18 years were included in the analysis.

Outcome measures

Measure	Xyntha n=4 Participants Non-interventional, observational study of participants who were prescribed and received at least 1 dose of Xyntha 250, 500, 1000, or 2000 IU.
Average Infusion Dose During Prophylaxis in Participants <18 Years of Age	1243.75 IU Standard Deviation 894.51 • Interval 725.0 to 725.0

SECONDARY outcome

Timeframe: 4 years

Population: All enrolled participants; only participants ≥18 years with nonmissing data were included in the analysis.

Outcome measures

Measure	Xyntha n=37 Participants Non-interventional, observational study of participants who were prescribed and received at least 1 dose of Xyntha 250, 500, 1000, or 2000 IU.
Average Infusion Dose During Prophylaxis in Participants ≥18 Years of Age	2000.16 IU Standard Deviation 437.59

SECONDARY outcome

Timeframe: 4 years

Population: All enrolled participants; participants were excluded from the analysis if total number of infusions for prophylaxis was unknown.

Outcome measures

Measure	Xyntha n=18 Participants Non-interventional, observational study of participants who were prescribed and received at least 1 dose of Xyntha 250, 500, 1000, or 2000 IU.
Total Factor Consumption for On-Demand Therapy and During Prophylaxis (All Participants)	66371.00 IU Standard Deviation 60985.45

SECONDARY outcome

Timeframe: 4 years

Population: All enrolled participants; only participants \<18 years were included in the analysis and participants were excluded from the analysis if total number of infusions for prophylaxis was unknown.

Outcome measures

Measure	Xyntha n=1 Participants Non-interventional, observational study of participants who were prescribed and received at least 1 dose of Xyntha 250, 500, 1000, or 2000 IU.
Total Factor Consumption for On-Demand Therapy and During Prophylaxis in Participants <18 Years of Age	2900.00 IU Full Range 894.51 • Interval 2900.0 to 2900.0

SECONDARY outcome

Timeframe: 4 years

Population: All enrolled participants; only participants ≥18 years were included in the analysis; participants were excluded from the analysis if total number of infusions for prophylaxis was unknown.

Outcome measures

Measure	Xyntha n=17 Participants Non-interventional, observational study of participants who were prescribed and received at least 1 dose of Xyntha 250, 500, 1000, or 2000 IU.
Total Factor Consumption for On-Demand Therapy and During Prophylaxis in Participants ≥18 Years of Age	70104.59 IU Standard Deviation 60704.87

Adverse Events

Xyntha

Serious events: 0 serious events

Other events: 10 other events

Deaths: 0 deaths

Serious adverse events

Adverse event data not reported

Other adverse events

Measure	Xyntha n=42 participants at risk Non-interventional, observational study of participants who were prescribed and received at least 1 dose of Xyntha 250, 500, 1000, or 2000 IU.
Nervous system disorders Headache	2.4% 1/42 • Adverse events were collected up to 6 months after the first administration of Xyntha. The same event may appear as both an adverse event (AE) and a serious adverse event (SAE). However, what is presented are distinct events. An event may be categorized as serious in one participant and as nonserious in another subject, or one participant may have experienced both a serious and nonserious event during the study.
Gastrointestinal disorders Dyspepsia	2.4% 1/42 • Adverse events were collected up to 6 months after the first administration of Xyntha. The same event may appear as both an adverse event (AE) and a serious adverse event (SAE). However, what is presented are distinct events. An event may be categorized as serious in one participant and as nonserious in another subject, or one participant may have experienced both a serious and nonserious event during the study.
Gastrointestinal disorders Gastritis	7.1% 3/42 • Adverse events were collected up to 6 months after the first administration of Xyntha. The same event may appear as both an adverse event (AE) and a serious adverse event (SAE). However, what is presented are distinct events. An event may be categorized as serious in one participant and as nonserious in another subject, or one participant may have experienced both a serious and nonserious event during the study.
Gastrointestinal disorders Irritable bowel syndrome	2.4% 1/42 • Adverse events were collected up to 6 months after the first administration of Xyntha. The same event may appear as both an adverse event (AE) and a serious adverse event (SAE). However, what is presented are distinct events. An event may be categorized as serious in one participant and as nonserious in another subject, or one participant may have experienced both a serious and nonserious event during the study.
Musculoskeletal and connective tissue disorders Arthralgia	2.4% 1/42 • Adverse events were collected up to 6 months after the first administration of Xyntha. The same event may appear as both an adverse event (AE) and a serious adverse event (SAE). However, what is presented are distinct events. An event may be categorized as serious in one participant and as nonserious in another subject, or one participant may have experienced both a serious and nonserious event during the study.
Musculoskeletal and connective tissue disorders Haemarthrosis	4.8% 2/42 • Adverse events were collected up to 6 months after the first administration of Xyntha. The same event may appear as both an adverse event (AE) and a serious adverse event (SAE). However, what is presented are distinct events. An event may be categorized as serious in one participant and as nonserious in another subject, or one participant may have experienced both a serious and nonserious event during the study.
Musculoskeletal and connective tissue disorders Ligament disorder	2.4% 1/42 • Adverse events were collected up to 6 months after the first administration of Xyntha. The same event may appear as both an adverse event (AE) and a serious adverse event (SAE). However, what is presented are distinct events. An event may be categorized as serious in one participant and as nonserious in another subject, or one participant may have experienced both a serious and nonserious event during the study.
Musculoskeletal and connective tissue disorders Muscle haemorrhage	2.4% 1/42 • Adverse events were collected up to 6 months after the first administration of Xyntha. The same event may appear as both an adverse event (AE) and a serious adverse event (SAE). However, what is presented are distinct events. An event may be categorized as serious in one participant and as nonserious in another subject, or one participant may have experienced both a serious and nonserious event during the study.
Musculoskeletal and connective tissue disorders Myalgia	4.8% 2/42 • Adverse events were collected up to 6 months after the first administration of Xyntha. The same event may appear as both an adverse event (AE) and a serious adverse event (SAE). However, what is presented are distinct events. An event may be categorized as serious in one participant and as nonserious in another subject, or one participant may have experienced both a serious and nonserious event during the study.
Musculoskeletal and connective tissue disorders Intervertebral disc disorder	2.4% 1/42 • Adverse events were collected up to 6 months after the first administration of Xyntha. The same event may appear as both an adverse event (AE) and a serious adverse event (SAE). However, what is presented are distinct events. An event may be categorized as serious in one participant and as nonserious in another subject, or one participant may have experienced both a serious and nonserious event during the study.
Skin and subcutaneous tissue disorders Dermatitis	2.4% 1/42 • Adverse events were collected up to 6 months after the first administration of Xyntha. The same event may appear as both an adverse event (AE) and a serious adverse event (SAE). However, what is presented are distinct events. An event may be categorized as serious in one participant and as nonserious in another subject, or one participant may have experienced both a serious and nonserious event during the study.
Skin and subcutaneous tissue disorders Dermatitis fungal	2.4% 1/42 • Adverse events were collected up to 6 months after the first administration of Xyntha. The same event may appear as both an adverse event (AE) and a serious adverse event (SAE). However, what is presented are distinct events. An event may be categorized as serious in one participant and as nonserious in another subject, or one participant may have experienced both a serious and nonserious event during the study.
Renal and urinary disorders Renal calculus	2.4% 1/42 • Adverse events were collected up to 6 months after the first administration of Xyntha. The same event may appear as both an adverse event (AE) and a serious adverse event (SAE). However, what is presented are distinct events. An event may be categorized as serious in one participant and as nonserious in another subject, or one participant may have experienced both a serious and nonserious event during the study.
Eye disorders Xerophthalmia	2.4% 1/42 • Adverse events were collected up to 6 months after the first administration of Xyntha. The same event may appear as both an adverse event (AE) and a serious adverse event (SAE). However, what is presented are distinct events. An event may be categorized as serious in one participant and as nonserious in another subject, or one participant may have experienced both a serious and nonserious event during the study.

Additional Information

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Phone: 1-800-718-1021

Email: ClinicalTrials.gov_Inquiries@pfizer.com

Results disclosure agreements

• Principal investigator is a sponsor employee
• Publication restrictions are in place