Factor XIII DeficiencyPhase 3NCT00945906
An Open Enrollment Study of Factor XIII Concentrate in Subjects With Congenital Factor XIII Deficiency
A Phase 3 study testing FXIII Concentrate (Human) (FXIII) in people with Factor XIII Deficiency, run by CSL Behring at 25 sites in United States.
Trial summary
Congenital deficiency of factor XIII 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) (FXIII)
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 61 participants
- Where
- 25 sitesUnited States
- Study dates
- Sep 2009to Aug 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
- Diagnosed with congenital FXIII deficiency requiring prophylactic treatment
- Males and females of any age
Who cannot join
- Diagnosis of acquired FXIII deficiency
- Administration of a FXIII-containing product, including blood transfusions or other blood products, within 3 weeks prior to the Baseline/Day 0 Visit
- Any known congenital or acquired coagulation disorder other than congenital FXIII deficiency
- Use of any other IMP within 4 weeks prior to Baseline/Day 0 Visit
- 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
- Any laboratory finding or medical condition which, in the opinion of the Investigator, would put the subject or subject's disease management at risk
Trial locations and contacts
25 sites in 1 country.
25 sites in 1 country
United States25 sites
Study Site
Albany, New York, 12208
Study Site
Ann Arbor, Michigan, 48109
Study Site
Boston, Massachusetts, 02115
Study Site
Chapel Hill, North Carolina, 27599
Study Site
Columbus, Ohio, 43205
Study Site
Dallas, Texas, 75390
Study Site
Detroit, Michigan, 48201
Study Site
Dothan, Alabama, 36301
Study Site
Fort Myers, Florida, 33908
Study Site
Hershey, Pennsylvania, 17033
Study Site
Houston, Texas, 77030
Study Site
Kansas City, Missouri, 64108
Study Site
Las Vegas, Nevada, 89109
Study Site
Lebanon, New Hampshire, 03756
Study Site
Miami, Florida, 33136
Study site
Milwaukee, Wisconsin, 53223
Study Site
Nashville, Tennessee, 37232
Study Site
New Orleans, Louisiana, 70121
Study Site
New York, New York, 10065
Study Site
Oakland, California, 94609
Study Site
Orange, California, 92868
Study Site
San Francisco, California, 94118
Study Site
Sioux Falls, South Dakota, 57105
Study Site
St. Petersburg, Florida, 33701
Study Site
Stockton, California, 95204
About this study
Official title: A Prospective, Multicenter, Open Enrollment Study of Human Plasma-Derived Factor XIII Concentrate in Subjects With Congenital Factor XIII Deficiency
Congenital deficiency of factor XIII 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 of FXIII Concentrate (Human) that will best minimize the chance of bruising and bleeding. The purpose of the study is to provide FXIII Concentrate (Human) to patients until the product becomes commercially available in the United States.
- Conditions
- Factor XIII Deficiency
- Being tested
- FXIII Concentrate (Human) (FXIII)
- 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) (FXIII)Biological
Also called Fibrogammin-P®, Corifact®
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 have not received at least 3 doses of FXIII Concentrate in a previous study of this product (i.e., NCT00640289, NCT00885742, or NCT00883090) will initially receive a dose of 40 U/kg by intravenous (IV) infusion.
| Study group | Role | What they receive |
|---|---|---|
| FXIII | Experimental | Subjects were administered FXIII Concentrate (Human) by intravenous (IV) infusion approximately every 28 days to maintain a trough FXIII level of approximately 5 to 20%. Receives: Biological: FXIII Concentrate (Human) (FXIII) |
What is being measured
The primary outcome is the main question the study is designed to answer.
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 treatment is related, or possibly related and AEs with missing relationship.
Measured: After the first infusion until study completion. Study completion is up to 2 years or until Factor XIII Concentrate (Human) is commercially available in the USA.
Secondary outcomes (5)
Hematology and Chemistry Testing
Number of participants with treatment-emergent clinically significant hematology and/or chemistry laboratory parameter values.
Measured: After the first infusion and at the end-of-study (or withdrawal) visit.
FXIII Antibody Testing
Number of participants with serum Factor XIII antibodies.
Measured: Before the first infusion, then every 48 weeks, at the end-of-study (or withdrawal) visit and after a bleeding episode requiring treatment with a Factor XIII -containing product.
FXIII Concentration
Trough Factor XIII concentration.
Measured: Before the first infusion, at 24 and 48 weeks after the first infusion, and at the end-of-study (or withdrawal) visit.
Number of Subjects With at Least One Bleeding Episode
Number of subjects with at least one bleeding episode at any time after the first infusion in the study, and the number of subjects with at least one bleeding episode requiring Factor XIII treatment.
Measured: After the first infusion until study completion. Study completion is up to 2 years or until Factor XIII Concentrate (Human) is commercially available in the USA.
Number of Bleeding Episodes
Number of bleeding episodes at any time after the first infusion in the study.
Measured: After the first infusion until study completion. Study completion is up to 2 years or until Factor XIII Concentrate (Human) is commercially available in the USA.
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted24 Jul 2009 (estimated)
- Study startSep 2009
- Primary completionAug 2011
- Study completionAug 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.
- 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
- 61 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
- NCT00945906
- Sponsor's protocol ID
- BI71023_3002
- Other IDs
- 1488
- Status last verified
- Sep 2012
- First submitted
- 23 Jul 2009
- Last updated
- 12 Oct 2012
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- No
Study at a glance
From the registry record, last updated by the sponsor on 12 Oct 2012. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Prospective, Multicenter, Open Enrollment Study of Human Plasma-Derived Factor XIII Concentrate in Subjects With Congenital Factor XIII Deficiency
- ClinicalTrials.gov ID
- NCT00945906
- Acronym / sponsor ID
- BI71023_3002
- Conditions
- Factor XIII Deficiency
- Keywords
- Hereditary Factor XIII deficiency, Factor XIII
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 61 participants (actual)
- Lead sponsor
- CSL BehringIndustry (pharma, biotech, devices)
- Study start
- Sep 2009
- Primary completion
- Aug 2011 (actual)
- Study completion
- Aug 2011 (actual)
- First posted
- 24 Jul 2009 (estimated)
- Results
- Posted 12 Oct 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 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 of FXIII Concentrate (Human) that will best minimize the chance of bruising and bleeding. The purpose of the study is to provide FXIII Concentrate (Human) to patients until the product becomes commercially available in the United States.
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) (FXIII)Subjects were administered FXIII Concentrate (Human) by intravenous (IV) infusion approximately every 28 days to maintain a trough FXIII level of approximately 5 to 20%. |
Who may qualify
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (3 listed)
- Written informed consent/assent for study participation obtained before undergoing any study specific procedures
- Diagnosed with congenital FXIII deficiency requiring prophylactic treatment
- Males and females of any age
Key exclusion criteria (7 listed)
- Diagnosis of acquired FXIII deficiency
- Administration of a FXIII-containing product, including blood transfusions or other blood products, within 3 weeks prior to the Baseline/Day 0 Visit
- Any known congenital or acquired coagulation disorder other than congenital FXIII deficiency
- Use of any other IMP within 4 weeks prior to Baseline/Day 0 Visit
- 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
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) (FXIII)Also: Fibrogammin-P®, Corifact®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 have not received at least 3 doses of FXIII Concentrate in a previous study of this product (i.e., NCT00640289, NCT00885742, or NCT00883090) will initially receive a dose of 40 U/kg by intravenous (IV) infusion. | Biological | FXIII |
Primary outcome
Adverse Events
Time frame: After the first infusion until study completion. Study completion is up to 2 years or until Factor XIII Concentrate (Human) is commercially available in the USA.
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 treatment is related, or possibly related and AEs with missing relationship.
5 secondary outcomes
Hematology and Chemistry Testing
Time frame: After the first infusion and at the end-of-study (or withdrawal) visit.
FXIII Antibody Testing
Time frame: Before the first infusion, then every 48 weeks, at the end-of-study (or withdrawal) visit and after a bleeding episode requiring treatment with a Factor XIII -containing product.
FXIII Concentration
Time frame: Before the first infusion, at 24 and 48 weeks after the first infusion, and at the end-of-study (or withdrawal) visit.
Number of Subjects With at Least One Bleeding Episode
Time frame: After the first infusion until study completion. Study completion is up to 2 years or until Factor XIII Concentrate (Human) is commercially available in the USA.
Number of Bleeding Episodes
Time frame: After the first infusion until study completion. Study completion is up to 2 years or until Factor XIII Concentrate (Human) is commercially available in the USA.
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 12 Oct 2012.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
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
25 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing FXIII Concentrate (Human) (FXIII) in people with Factor XIII Deficiency, run by CSL Behring at 25 sites in United States.
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 3 inclusion and 7 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 12 Oct 2012. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 25 sites in United States. 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 12 Oct 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?
Adverse Events (time frame: After the first infusion until study completion. Study completion is up to 2 years or until Factor XIII Concentrate (Human) is commercially available in the USA.). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
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 12 Oct 2012.