Trial Outcomes & Findings for Clinical Trial of Factor XIII (FXIII) Concentrate (NCT NCT00640289)

Last Updated: 2018-11-07

Results Overview

Response is defined as: Excellent/Good = adequate hemostasis, similar to that expected for subjects without known bleeding disorders; Fair/Poor = hemostasis less than expected; None = severe bleeding, judged due to disease despite Factor XIII (FXIII) therapy. Only the subjects who needed additional FXIII infusions (apart from the prophylactic treatment) to control a bleed and who had investigator assessment of efficacy were counted in this outcome.

Recruitment status

COMPLETED

Study phase

Target enrollment

72 participants

Primary outcome timeframe

Within 12 hours of FXIII infusion

Results posted on

2018-11-07

Participant Flow

Participant milestones

Participant milestones
Measure	Factor XIII Prophylactic dose of Factor XIII Concentrate (Human) was to be 10 to 20 U/kg b.w. administered IV over 5 minutes once every 4 weeks, and later tailored to subjects PK profile.
Overall Study STARTED	72
Overall Study COMPLETED	59
Overall Study NOT COMPLETED	13

Reasons for withdrawal

Reasons for withdrawal
Measure	Factor XIII Prophylactic dose of Factor XIII Concentrate (Human) was to be 10 to 20 U/kg b.w. administered IV over 5 minutes once every 4 weeks, and later tailored to subjects PK profile.
Overall Study Death	3
Overall Study Withdrawal by Subject	1
Overall Study Lost to Follow-up	1
Overall Study Data collected under different amendment	2
Overall Study Transferred to another center	1
Overall Study Missing status	5

Baseline Characteristics

Clinical Trial of Factor XIII (FXIII) Concentrate

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Factor XIII n=72 Participants
Age, Continuous	15.0 years STANDARD_DEVIATION 14.09 • n=99 Participants
Age, Customized <2 years	9 Participants n=99 Participants
Age, Customized 2 to<16 years	34 Participants n=99 Participants
Age, Customized 16 to<65 years	28 Participants n=99 Participants
Age, Customized ≥65 years	1 Participants n=99 Participants
Sex: Female, Male Female	24 Participants n=99 Participants
Sex: Female, Male Male	48 Participants n=99 Participants
Region of Enrollment United States	72 Participants n=99 Participants

PRIMARY outcome

Timeframe: Within 12 hours of FXIII infusion

Population: Safety Population (SP): The safety population consists of all subjects who received a dose of FXIII during the study.

Outcome measures

Outcome measures
Measure	Factor XIII n=34 Number of bleeds
Response to Treatment of Bleeding Events Requiring Additional Factor XIII Infusions Excellent/Good	30 Number of bleeds
Response to Treatment of Bleeding Events Requiring Additional Factor XIII Infusions Fair/Poor	1 Number of bleeds
Response to Treatment of Bleeding Events Requiring Additional Factor XIII Infusions None	0 Number of bleeds
Response to Treatment of Bleeding Events Requiring Additional Factor XIII Infusions Missing	3 Number of bleeds

SECONDARY outcome

Timeframe: During surgical procedure

Population: SP

Surgical efficacy is defined as: Excellent/Good = adequate hemostasis, similar to that expected for subjects without known bleeding disorders; Fair/Poor = hemostasis less than expected; None = severe bleeding, judged due to disease despite Factor XIII (FXIII) therapy. Only the subjects who underwent a surgical procedure were counted in this outcome measure.

Outcome measures

Outcome measures
Measure	Factor XIII n=16 Surgical procedures
Surgical Efficacy Assessments With Factor XIII Excellent/Good	15 Surgical procedures
Surgical Efficacy Assessments With Factor XIII Fair/Poor	0 Surgical procedures
Surgical Efficacy Assessments With Factor XIII None	0 Surgical procedures
Surgical Efficacy Assessments With Factor XIII Missing	1 Surgical procedures

Adverse Events

Factor XIII

Serious events: 8 serious events

Other events: 32 other events

Deaths: 3 deaths

Serious adverse events

Serious adverse events
Measure	Factor XIII n=72 participants at risk
Infections and infestations Catheter related infection	1.4% 1/72 • Number of events 1 • 9 years
Infections and infestations Wound infection	1.4% 1/72 • Number of events 1 • 9 years
Injury, poisoning and procedural complications Gunshot wound	1.4% 1/72 • Number of events 1 • 9 years
Injury, poisoning and procedural complications Road traffic accident	1.4% 1/72 • Number of events 1 • 9 years
Nervous system disorders Brain mass	1.4% 1/72 • Number of events 1 • 9 years
Nervous system disorders Syncope	1.4% 1/72 • Number of events 1 • 9 years
Vascular disorders Hypertension	1.4% 1/72 • Number of events 1 • 9 years
Vascular disorders Vasculitis	1.4% 1/72 • Number of events 1 • 9 years
Psychiatric disorders Suicidal behavior	1.4% 1/72 • Number of events 1 • 9 years

Other adverse events

Other adverse events
Measure	Factor XIII n=72 participants at risk
Injury, poisoning and procedural complications Contusion	18.1% 13/72 • Number of events 32 • 9 years
Injury, poisoning and procedural complications Joint injury	9.7% 7/72 • Number of events 9 • 9 years
Injury, poisoning and procedural complications Limb injury	5.6% 4/72 • Number of events 10 • 9 years
Injury, poisoning and procedural complications Road traffic accident	5.6% 4/72 • Number of events 4 • 9 years
Musculoskeletal and connective tissue disorders Arthralgia	8.3% 6/72 • Number of events 22 • 9 years
Skin and subcutaneous tissue disorders Rash	5.6% 4/72 • Number of events 5 • 9 years

Additional Information

Clinical Trial Coordinator

Children's Hospital of Orange County

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place