Factor XIII DeficiencyPhase 3NCT00885742
A Study of Factor XIII Concentrate in Subjects With Congenital Factor XIII Deficiency
A Phase 3 study testing FXIII Concentrate (Human) in people with Factor XIII Deficiency, run by CSL Behring at 23 sites in 2 countries.
Trial summary
Congenital deficiency of factor XIII (FXIII) is an extremely rare inherited disorder associated with potentially life-threatening bleeding. Factor XIII Concentrate is given to patients whose blood is lacking factor XIII. Read more about this study
- Who is it for?
- Any age
- Conditions the study is for
- Factor XIII Deficiency
- What is being tested?
- FXIII Concentrate (Human)
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 41 participants
- Where
- 23 sitesin 2 countries
- Study dates
- Aug 2009to Apr 2011
- Phase
- Phase 3
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages Any age
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- Written informed consent/assent for study participation obtained before undergoing any study-specific procedures
- Documented congenital FXIII deficiency which requires prophylactic treatment with a FXIII containing product.
- Males and females of any age with congenital FXIII deficiency
- Received full hepatitis B vaccination and/or is hepatitis B surface antibody positive
Who cannot join
- Diagnosis of acquired FXIII deficiency
- Administration of a FXIII-containing product, including blood transfusions or other blood products within 4 weeks prior to the planned Day 0
- Any known congenital or acquired coagulation disorder other than congenital FXIII deficiency
- Known or suspected to have antibodies towards FXIII
- Use of any other investigational medicinal product within 4 weeks prior to the Baseline Visit (Day 0)
- Known Positivity for human immunodeficiency virus (HIV) or a positive result for HIV at the Screening Visit of this study or the FXIII study 2002 (NCT00883090).
Show all 13Show fewer
- Serum aspartate transaminase (AST) or serum alanine transaminase (ALT) concentration >2.5 times the upper limit of normal at the Screening Visit of this study or at the Day 56 Visit of Factor XIII Study BI71023_2002 (NCT00883090)
- Fibrinogen level less than 85% of the lower limit of normal at the Screening Visit of this study or the Factor XIII Study BI71023_2002 (NCT00883090)
- Active bleeding ≥ Common Terminology Criteria for Adverse Events (CTCAE) Grade 2 and/or ≥ moderate between the Screening and Baseline Visits
- Pregnant or breast-feeding
- Intention to become pregnant during the course of the study
- Female subjects of childbearing potential not using, or not willing to use, a medically reliable method of contraception for the entire duration of the study
- Suspected inability (e.g., language problems) or unwillingness to comply with study procedures or history of noncompliance
Trial locations and contacts
23 sites in 2 countries.
23 sites in 2 countries
United States22 sites
Study Site
Albany, New York, 12208
Study Site
Boise, Idaho, 83712
Study Site
Boston, Massachusetts, 02115
Study Site
Chapel Hill, North Carolina, 27599
Study Site
Dallas, Texas, 75390
Study Site
Dothan, Alabama, 36305
Study Site
Fort Meyers, Florida, 33908
Study Site
Hartford, Connecticut, 06106
Study Site
Hershey, Pennsylvania, 17033
Study Site
Kansas City, Missouri, 64108
Study Site
Las Vegas, Nevada, 89015
Study Site
Lebanon, New Hampshire, 03756
Study
Miami, Florida, 33136
Study Site
Milwaukee, Wisconsin, 53233
Study Site
New York, New York, 10021
Study Site
Newark, New Jersey, 07102
Study Site
Oakland, California, 94610
Study Site
Orange, California, 92868
Study Site
Saint Paul, Minnesota, 55102
Study Site
San Francisco, California, 94115
Study Site
South Bend, Indiana, 46601
Study Site
Stockton, California, 95204
About this study
Official title: A Prospective, Multicenter, Open-label, Phase 3b Study of Human Plasma-Derived Factor XIII Concentrate in Subjects With Congenital Factor XIII Deficiency
Congenital deficiency of factor XIII (FXIII) is an extremely rare inherited disorder associated with potentially life-threatening bleeding. Factor XIII Concentrate is given to patients whose blood is lacking factor XIII. Factor XIII Concentrate works by assisting blood in the usual clotting process, thereby preventing bleeding.
In this study, patients will be treated with FXIII Concentrate (Human) and followed closely to determine that they receive the dose that will best minimize the chance of bruising and bleeding.
- Conditions
- Factor XIII Deficiency
- Being tested
- FXIII Concentrate (Human)
- Keywords
- Hereditary Factor XIII deficiency, Factor XIII
- Medical subject headings
- Factor XIII Deficiency
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
FXIII Concentrate (Human)Biological
Also called Fibrogammin-P
Doses will be guided by the individual subject's most recent FXIII activity levels, with the objective of dosing every 28 days to maintain a trough FXIII activity level of approximately 5 to 20%.
Subjects enrolled in this study who did not complete the pharmacokinetic study (Factor XIII Study BI71023_2002 [NCT00883090]) will initially receive FXIII Concentrate (Human) at a dose of 40 U/kg by intravenous (IV) infusion.
| Study group | Role | What they receive |
|---|---|---|
| FXIII | Experimental | All subjects who received a dose of Factor XIII (FXIII) Concentrate (Human). Receives: Biological: FXIII Concentrate (Human) |
What is being measured
The primary outcome is the main question the study is designed to answer.
The Incidence of Spontaneous Bleeding Events Requiring Treatment (Treatment is Defined as Administration of a FXIII-Containing Product to Treat the Bleeding Event)
The number of subjects requiring treatment with a Factor XIII-containing product to treat a spontaneous bleeding event.
Measured: Up to week 52
Secondary outcomes (7)
Association of the Incidence of Spontaneous Bleeding Events Requiring Treatment and FXIII Activity Trough Levels
P-value determined from Generalized Estimating Equation (GEE) model parameter estimates with bleeding as the response variable and FXIII activity trough level as the explanatory variable.
Measured: 12 months
Adverse Events
Number of subjects with any treatment-emergent adverse event (AE), treatment-related AE or serious AE (SAE). Treatment related AEs are defined as AEs whose relationship to study treatment is related, or possibly related, and AEs with missing relationship.
Measured: 12 months
Peak FXIII Concentration at Steady State
Measured: At 12, 24, 36 and 48 weeks: at 30 and 60 minutes after the end of the infusion.
Trough FXIII Concentration at Steady State
Measured: At 12, 24, 36 and 48 weeks: immediately before infusion.
Time to Peak Concentration
Measured: At 12, 24, 36 and 48 weeks: immediately before infusion, then at 30 and 60 minutes after the end of the infusion.
Incremental Recovery
Incremental recovery (U/mL/U/kg) is defined as maximum (peak) FXIII activity (U/mL) obtained after infusion, per dose of (U/kg) infusion.
Measured: At 12, 24, 36 and 48 weeks: immediately before infusion, then at 30 and 60 minutes after the end of the infusion.
Achievement of Trough Factor XIII Levels of 5% or Higher.
Number of subjects with Factor XIII level ≥ 5% before infusion at Week 12, Week 24, Week 36 and Week 48.
Measured: At 12, 24, 36 and 48 weeks: immediately before infusion.
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted22 Apr 2009 (estimated)
- Study startAug 2009
- Primary completionApr 2011
- Study completionApr 2011
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Factor XIII DeficiencyStudies of the same condition, open and under way.
- More trials run by CSL BehringThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 3
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 41 participants (actual)
Sponsor and investigators
- Lead sponsor
- CSL Behring
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Program Director, Clinical R&D, Study director, CSL Behring
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00885742
- Sponsor's protocol ID
- BI71023_3001
- Other IDs
- 1482, 2009-010722-19, 2009
- Status last verified
- Jun 2012
- First submitted
- 21 Apr 2009
- Last updated
- 16 Jul 2012
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- No
Publications
Ashley C, Chang E, Davis J, Mangione A, Frame V, Nugent DJ. Efficacy and safety of prophylactic treatment with plasma-derived factor XIII concentrate (human) in patients with congenital factor XIII deficiency. Haemophilia. 2015 Jan;21(1):102-8. doi: 10.1111/hae.12524. Epub 2014 Nov 7.
PubMed 25377187
Study at a glance
From the registry record, last updated by the sponsor on 16 Jul 2012. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Prospective, Multicenter, Open-label, Phase 3b Study of Human Plasma-Derived Factor XIII Concentrate in Subjects With Congenital Factor XIII Deficiency
- ClinicalTrials.gov ID
- NCT00885742
- Acronym / sponsor ID
- BI71023_3001
- Conditions
- Factor XIII Deficiency
- Keywords
- Hereditary Factor XIII deficiency, Factor XIII
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 41 participants (actual)
- Lead sponsor
- CSL BehringIndustry (pharma, biotech, devices)
- Study start
- Aug 2009
- Primary completion
- Apr 2011 (actual)
- Study completion
- Apr 2011 (actual)
- First posted
- 22 Apr 2009 (estimated)
- Results
- Posted 4 Jul 2012
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
Congenital deficiency of factor XIII (FXIII) is an extremely rare inherited disorder associated with potentially life-threatening bleeding. Factor XIII Concentrate is given to patients whose blood is lacking factor XIII. Factor XIII Concentrate works by assisting blood in the usual clotting process, thereby preventing bleeding.
In this study, patients will be treated with FXIII Concentrate (Human) and followed closely to determine that they receive the dose that will best minimize the chance of bruising and bleeding.
Study design
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| FXIII | Experimental | Biological: FXIII Concentrate (Human)All subjects who received a dose of Factor XIII (FXIII) Concentrate (Human). |
Who may qualify
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (4 listed)
- Written informed consent/assent for study participation obtained before undergoing any study-specific procedures
- Documented congenital FXIII deficiency which requires prophylactic treatment with a FXIII containing product.
- Males and females of any age with congenital FXIII deficiency
- Received full hepatitis B vaccination and/or is hepatitis B surface antibody positive
Key exclusion criteria (13 listed)
- Diagnosis of acquired FXIII deficiency
- Administration of a FXIII-containing product, including blood transfusions or other blood products within 4 weeks prior to the planned Day 0
- Any known congenital or acquired coagulation disorder other than congenital FXIII deficiency
- Known or suspected to have antibodies towards FXIII
- Use of any other investigational medicinal product within 4 weeks prior to the Baseline Visit (Day 0)
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| FXIII Concentrate (Human)Also: Fibrogammin-PDoses will be guided by the individual subject's most recent FXIII activity levels, with the objective of dosing every 28 days to maintain a trough FXIII activity level of approximately 5 to 20%. Subjects enrolled in this study who did not complete the pharmacokinetic study (Factor XIII Study BI71023_2002 [NCT00883090]) will initially receive FXIII Concentrate (Human) at a dose of 40 U/kg by intravenous (IV) infusion. | Biological | FXIII |
Primary outcome
The Incidence of Spontaneous Bleeding Events Requiring Treatment (Treatment is Defined as Administration of a FXIII-Containing Product to Treat the Bleeding Event)
Time frame: Up to week 52
The number of subjects requiring treatment with a Factor XIII-containing product to treat a spontaneous bleeding event.
7 secondary outcomes
Association of the Incidence of Spontaneous Bleeding Events Requiring Treatment and FXIII Activity Trough Levels
Time frame: 12 months
Adverse Events
Time frame: 12 months
Peak FXIII Concentration at Steady State
Time frame: At 12, 24, 36 and 48 weeks: at 30 and 60 minutes after the end of the infusion.
Trough FXIII Concentration at Steady State
Time frame: At 12, 24, 36 and 48 weeks: immediately before infusion.
Time to Peak Concentration
Time frame: At 12, 24, 36 and 48 weeks: immediately before infusion, then at 30 and 60 minutes after the end of the infusion.
Incremental Recovery
Time frame: At 12, 24, 36 and 48 weeks: immediately before infusion, then at 30 and 60 minutes after the end of the infusion.
Achievement of Trough Factor XIII Levels of 5% or Higher.
Time frame: At 12, 24, 36 and 48 weeks: immediately before infusion.
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 16 Jul 2012.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Derived from this study: PubMed 25377187Ashley C, Chang E, Davis J, Mangione A, Frame V, Nugent DJ. Efficacy and safety of prophylactic treatment with plasma-derived factor XIII concentrate (human) in patients with congenital factor XIII deficiency. Haemophilia. 2015 Jan;21(1):102-8. doi: 10.1111/hae.12524. Epub 2014 Nov 7.
- Factor XIII Study BI71023\_2002 (NCT00883090)Link provided by the sponsor.
- Factor XIII Study BI71023\_3002 (NCT00945906)Link provided by the sponsor.
Related trials for Factor XIII Deficiency
RecruitingPhase 4
SWIFT - SWIss Factor XIII Trial in PPH
Postpartum Hemorrhage, Coagulation Disorder
Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Program Director, Clinical R&D study director
CSL Behring
23 sites in 2 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing FXIII Concentrate (Human) in people with Factor XIII Deficiency, run by CSL Behring at 23 sites in 2 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 4 inclusion and 13 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 16 Jul 2012. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 23 sites in United States, Spain. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Yes. Results are posted in the registry (first posted 4 Jul 2012). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
The Incidence of Spontaneous Bleeding Events Requiring Treatment (Treatment is Defined as Administration of a FXIII-Containing Product to Treat the Bleeding Event) (time frame: Up to week 52). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT00885742, which also lists 1 linked publication.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 16 Jul 2012.