Factor XIII Deficiency, Hemophilia
Conditions
Keywords
Factor XIII Deficiency, Rare Bleeding Disorder, Hemophilia, Fibrogammin P
Brief summary
Congenital deficiency of Factor XIII is a rare but potentially life threatening disorder. It is inherited in an autosomal recessive fashion. Infusion of Factor XIII has proved to be useful for prevention and treatment of bleeding episodes, especially of spontaneous intracranial bleedings. In this study, Fibrogammin P will be given to patients with congenital Factor XIII deficiency and congenital/acquired FXIII deficiency to prevent bleeding and to treat established bleeding episodes. For Factor XIII prophylaxis to prevent hemorrhages, the dosage will depend on the weight of the subject. The frequency of Factor XIII administration will be determined by the factor's circulating half-life. During the first month only, a Factor XIII pharmacokinetic study will be determined over a 4-week period. Safety data will include accrual of information on viral safety, liver function, complete blood counts and adverse events. Historical data concerning spontaneous bleeds will be collected whenever possible two years prior to treatment with Fibrogammin P.
Interventions
DRUGFibrogammin P
Prophylaxis treatment
Sponsors
CSL Behring
Children's Hospital of Orange County
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Healthy volunteers
No
Inclusion criteria
* Patients may be of either sex or age. Children and newborn infants are specifically included in this study. * Patient must have documented congenital Factor XIII deficiency * Patient or legal guardian must sign informed consent * Patients who have negative serology for hepatitis B should receive Hepatitis B vaccination.
Exclusion criteria
* Patient has acquired Factor XIII deficiency
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Response to Treatment of Bleeding Events Requiring Additional Factor XIII Infusions | Within 12 hours of FXIII infusion | 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. |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Surgical Efficacy Assessments With Factor XIII | During surgical procedure | 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. |
Countries
United States
Participant flow
Participants by arm
| Arm | Count |
|---|---|
| Factor XIII | 72 |
| Total | 72 |
Withdrawals & dropouts
| Period | Reason | FG000 |
|---|---|---|
| Overall Study | Data collected under different amendment | 2 |
| Overall Study | Death | 3 |
| Overall Study | Lost to Follow-up | 1 |
| Overall Study | Missing status | 5 |
| Overall Study | Transferred to another center | 1 |
| Overall Study | Withdrawal by Subject | 1 |
Baseline characteristics
| Characteristic | Factor XIII |
|---|---|
| Age, Continuous | 15.0 years STANDARD_DEVIATION 14.09 |
| Age, Customized 16 to<65 years | 28 Participants |
| Age, Customized 2 to<16 years | 34 Participants |
| Age, Customized <2 years | 9 Participants |
| Age, Customized ≥65 years | 1 Participants |
| Region of Enrollment United States | 72 Participants |
| Sex: Female, Male Female | 24 Participants |
| Sex: Female, Male Male | 48 Participants |
Adverse events
| Event type | EG000 affected / at risk |
|---|---|
| deaths Total, all-cause mortality | 3 / 72 |
| other Total, other adverse events | 32 / 72 |
| serious Total, serious adverse events | 8 / 72 |
Outcome results
Primary
Response to Treatment of Bleeding Events Requiring Additional Factor XIII Infusions
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.
Time frame: 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.
| Arm | Measure | Category | Value (COUNT_OF_UNITS) |
|---|---|---|---|
| Factor XIII | Response to Treatment of Bleeding Events Requiring Additional Factor XIII Infusions | Excellent/Good | 30 Number of bleeds |
| Factor XIII | Response to Treatment of Bleeding Events Requiring Additional Factor XIII Infusions | Fair/Poor | 1 Number of bleeds |
| Factor XIII | Response to Treatment of Bleeding Events Requiring Additional Factor XIII Infusions | None | 0 Number of bleeds |
| Factor XIII | Response to Treatment of Bleeding Events Requiring Additional Factor XIII Infusions | Missing | 3 Number of bleeds |
Secondary
Surgical Efficacy Assessments With Factor XIII
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.
Time frame: During surgical procedure
Population: SP
| Arm | Measure | Category | Value (COUNT_OF_UNITS) |
|---|---|---|---|
| Factor XIII | Surgical Efficacy Assessments With Factor XIII | Excellent/Good | 15 Surgical procedures |
| Factor XIII | Surgical Efficacy Assessments With Factor XIII | Fair/Poor | 0 Surgical procedures |
| Factor XIII | Surgical Efficacy Assessments With Factor XIII | None | 0 Surgical procedures |
| Factor XIII | Surgical Efficacy Assessments With Factor XIII | Missing | 1 Surgical procedures |