Factor XIII DeficiencyPhase 2NCT00883090
A Study of the Use of Factor XIII Concentrate in Patients With Inherited FXIII Deficiency
A Phase 2 study testing FXIII Concentrate (Human) in people with Factor XIII Deficiency, run by CSL Behring at 6 sites in 2 countries.
Trial summary
Congenital deficiency of Factor XIII is an extremely rare hereditary disorder associated with potentially life-threatening bleeding. This study will evaluate the safety and recommended (best) amount or level of Factor XIII in a patient's blood. 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
- 15 participants
- Where
- 6 sitesin 2 countries
- Study dates
- May 2009to Apr 2010
- Phase
- Phase 2
- 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 that 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)
- Positive result at screening for human immunodeficiency virus (HIV)
Show all 14Show fewer
- Serum aspartate transaminase (AST) or serum alanine transaminase (ALT) concentration >2.5 times the upper limit of normal
- Fibrinogen < lower limit of normal
- Active bleeding
- 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
- Surgical procedure anticipated during the study period
- Suspected inability (e.g., language problems) or unwillingness to comply with study procedures or history of noncompliance
Trial locations and contacts
6 sites in 2 countries.
6 sites in 2 countries
United States5 sites
Study Site
Boston, Massachusetts, 02115
Study Site
Dothan, Alabama, 36305
Study Site
Orange, California, 92868
Study Site
San Francisco, California, 94115
Study Site
Stockton, California, 95204
About this study
Official title: A 12 Week, Multicenter, Pharmacokinetic and Safety Study of Human Plasma-Derived Factor XIII Concentrate in Subjects With Congenital Factor XIII Deficiency
Congenital deficiency of Factor XIII is an extremely rare hereditary disorder associated with potentially life-threatening bleeding. This study will evaluate the safety and recommended (best) amount or level of Factor XIII in a patient's blood. Factor XIII Concentrate (Human) is given to people whose blood is lacking Factor XIII. Factor XIII Concentrate (Human) works by assisting your blood in the usual clotting process, thereby preventing bleeding.
- Conditions
- Factor XIII Deficiency
- Being tested
- FXIII Concentrate (Human)
- Keywords
- 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
Subjects will receive approximately 40 U/kg of FXIII every 28 days for 3 doses administered as a bolus intravenous (IV) injection at approximately 250 U/minute.
| Study group | Role | What they receive |
|---|---|---|
| FXIII | Experimental | All subjects treated with Factor XIII Concentrate (Human) (FXIII) Receives: Biological: FXIII Concentrate (Human) |
What is being measured
The primary outcome is the main question the study is designed to answer.
Peak FXIII Concentration at Steady State
Measured: 12 weeks
Trough FXIII Concentration at Steady State
Measured: 12 weeks
Time to Peak Concentration
Measured: 12 weeks
Incremental Recovery
Incremental recovery (U/mL/U/kg) is defined as the maximum (peak) FXIII activity (U/mL) obtained after infusion, per dose of FXIII (U/kg) administered.
Measured: 12 weeks
Terminal Half-life
Measured: 12 weeks
Area Under the Curve at Steady State
Measured: 12 weeks
Clearance
Measured: 12 weeks
Volume of Distribution at Steady State
Measured: 12 weeks
Mean Residence Time
Measured: 12 weeks
Secondary outcomes (3)
Adverse Events
Number of participants with an adverse event
Measured: 16 weeks
Laboratory Safety Parameters
Number of participants with clinically significant laboratory safety parameter values. The laboratory safety parameters measured included serum chemistries, hematology and urinalysis.
Measured: 16 weeks
Vital Signs
Number of participants with clinically significant vital signs. The vital signs measured included blood pressure, pulse rate and temperature. Clinically significant changes in vital signs were to be reported as adverse events.
Measured: 16 weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted17 Apr 2009 (estimated)
- Study startMay 2009
- Primary completionApr 2010
- Study completionApr 2010
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 2
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 15 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
- NCT00883090
- Sponsor's protocol ID
- BI71023_2002
- Other IDs
- 2009-010387-41, 1479, 2009
- Status last verified
- Dec 2011
- First submitted
- 16 Apr 2009
- Last updated
- 16 Jan 2012
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- No
Publications
Nugent DJ, Ashley C, Garcia-Talavera J, Lo LC, Mehdi AS, Mangione A. Pharmacokinetics and safety of plasma-derived factor XIII concentrate (human) in patients with congenital factor XIII deficiency. Haemophilia. 2015 Jan;21(1):95-101. doi: 10.1111/hae.12505. Epub 2014 Dec 2.
PubMed 25458735
Study at a glance
From the registry record, last updated by the sponsor on 16 Jan 2012. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A 12 Week, Multicenter, Pharmacokinetic and Safety Study of Human Plasma-Derived Factor XIII Concentrate in Subjects With Congenital Factor XIII Deficiency
- ClinicalTrials.gov ID
- NCT00883090
- Acronym / sponsor ID
- BI71023_2002
- Conditions
- Factor XIII Deficiency
- Keywords
- Factor XIII
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 15 participants (actual)
- Lead sponsor
- CSL BehringIndustry (pharma, biotech, devices)
- Study start
- May 2009
- Primary completion
- Apr 2010 (actual)
- Study completion
- Apr 2010 (actual)
- First posted
- 17 Apr 2009 (estimated)
- Results
- Posted 16 Jan 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 hereditary disorder associated with potentially life-threatening bleeding. This study will evaluate the safety and recommended (best) amount or level of Factor XIII in a patient's blood. Factor XIII Concentrate (Human) is given to people whose blood is lacking Factor XIII. Factor XIII Concentrate (Human) works by assisting your blood in the usual clotting process, thereby preventing 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 treated with Factor XIII Concentrate (Human) (FXIII) |
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 that 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 (14 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®-PSubjects will receive approximately 40 U/kg of FXIII every 28 days for 3 doses administered as a bolus intravenous (IV) injection at approximately 250 U/minute. | Biological | FXIII |
Primary outcome
Peak FXIII Concentration at Steady State
Time frame: 12 weeks
Trough FXIII Concentration at Steady State
Time frame: 12 weeks
Time to Peak Concentration
Time frame: 12 weeks
Incremental Recovery
Time frame: 12 weeks
Incremental recovery (U/mL/U/kg) is defined as the maximum (peak) FXIII activity (U/mL) obtained after infusion, per dose of FXIII (U/kg) administered.
Terminal Half-life
Time frame: 12 weeks
Area Under the Curve at Steady State
Time frame: 12 weeks
Clearance
Time frame: 12 weeks
Volume of Distribution at Steady State
Time frame: 12 weeks
Mean Residence Time
Time frame: 12 weeks
3 secondary outcomes
Adverse Events
Time frame: 16 weeks
Laboratory Safety Parameters
Time frame: 16 weeks
Vital Signs
Time frame: 16 weeks
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 16 Jan 2012.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Derived from this study: PubMed 25458735Nugent DJ, Ashley C, Garcia-Talavera J, Lo LC, Mehdi AS, Mangione A. Pharmacokinetics and safety of plasma-derived factor XIII concentrate (human) in patients with congenital factor XIII deficiency. Haemophilia. 2015 Jan;21(1):95-101. doi: 10.1111/hae.12505. Epub 2014 Dec 2.
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
6 sites in 2 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing FXIII Concentrate (Human) in people with Factor XIII Deficiency, run by CSL Behring at 6 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 14 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 Jan 2012. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 6 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 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
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 16 Jan 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?
Peak FXIII Concentration at Steady State (time frame: 12 weeks). The study lists 9 primary outcomes in all. 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 NCT00883090, 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 Jan 2012.