HemophiliaNCT00640289
Clinical Trial of Factor XIII (FXIII) Concentrate
A study testing Fibrogammin P in people with Hemophilia and Factor XIII Deficiency, run by Children's Hospital of Orange County at 1 site in United States.
Trial 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. Read more about this study
- Who is it for?
- Any age
- Conditions the study is for
- Hemophilia, Factor XIII Deficiency
- What is being tested?
- Fibrogammin P
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 72 participants
- Where
- 1 siteUnited States
- Study dates
- Jan 2000to Dec 2011
- Phase
- Interventional
- 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
- 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.
Who cannot join
- Patient has acquired Factor XIII deficiency
Trial locations and contacts
1 site in 1 country.
United States1 site
Children's Hospital of Orange Co.
Orange, California, 92868
About this study
Official title: Clinical Research Study of Factor XIII Concentrate From Human Plasma Fibrogammin P in Patients With Factor XIII Deficiency
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.
- Conditions
- Hemophilia, Factor XIII Deficiency
- Being tested
- Fibrogammin P
- Keywords
- Factor XIII Deficiency, Rare Bleeding Disorder, Hemophilia, Fibrogammin P
- Medical subject headings
- Hemophilia A, Factor XIII Deficiency
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Fibrogammin PDrug
Prophylaxis treatment
| Study group | Role | What they receive |
|---|---|---|
| A | Experimental | Treatment Receives: Drug: Fibrogammin P |
What is being measured
The primary outcome is the main question the study is designed to answer.
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.
Measured: Within 12 hours of FXIII infusion
Secondary outcomes (1)
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.
Measured: During surgical procedure
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted21 Mar 2008 (estimated)
- Study startJan 2000
- Primary completionDec 2011
- Study completionDec 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 HemophiliaStudies of the same condition, open and under way.
- 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 N/A
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 72 participants (actual)
Sponsor and investigators
- Lead sponsor
- Children's Hospital of Orange County
- Sponsor type
- University, hospital or other
- Collaborators
- CSL Behring
- Responsible party
- Sponsor
- Investigators
- Diane J. Nugent, MD, Principal investigator, Children's Hospital of Orange Co.
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00640289
- Sponsor's protocol ID
- BB-IND5986
- Status last verified
- Mar 2008
- First submitted
- 17 Mar 2008
- Last updated
- 7 Nov 2018
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- No
Study at a glance
From the registry record, last updated by the sponsor on 7 Nov 2018. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Clinical Research Study of Factor XIII Concentrate From Human Plasma Fibrogammin P in Patients With Factor XIII Deficiency
- ClinicalTrials.gov ID
- NCT00640289
- Acronym / sponsor ID
- BB-IND5986
- Conditions
- Hemophilia, Factor XIII Deficiency
- Keywords
- Factor XIII Deficiency, Rare Bleeding Disorder, Hemophilia, Fibrogammin P
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 72 participants (actual)
- Lead sponsor
- Children's Hospital of Orange CountyUniversity, hospital or other
- Collaborators
- CSL Behring
- Study start
- Jan 2000
- Primary completion
- Dec 2011 (actual)
- Study completion
- Dec 2011 (actual)
- First posted
- 21 Mar 2008 (estimated)
- Results
- Posted 7 Nov 2018
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 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.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| A | Experimental | Drug: Fibrogammin PTreatment |
Who may qualify
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (4 listed)
- 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.
Key exclusion criteria (1 listed)
- Patient has acquired Factor XIII deficiency
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 |
|---|---|---|
| Fibrogammin PProphylaxis treatment | Drug | A |
Primary outcome
Response to Treatment of Bleeding Events Requiring Additional Factor XIII Infusions
Time frame: 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.
1 secondary outcome
Surgical Efficacy Assessments With Factor XIII
Time frame: During surgical procedure
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 7 Nov 2018.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
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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.
Diane J. Nugent, MD principal investigator
Children's Hospital of Orange Co.
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A study testing Fibrogammin P in people with Hemophilia and Factor XIII Deficiency, run by Children's Hospital of Orange County at 1 site 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 4 inclusion and 1 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 7 Nov 2018. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site 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.
Are results available for this study?
Yes. Results are posted in the registry (first posted 7 Nov 2018). 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?
Response to Treatment of Bleeding Events Requiring Additional Factor XIII Infusions (time frame: Within 12 hours of FXIII infusion). 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 7 Nov 2018.