Factor X DeficiencyPhase 3NCT00930176
A Study Investigating Treatment Factor X in People With Factor X Deficiency
A Phase 3 study testing Human Coagulation FACTOR X in people with Factor X Deficiency, run by Bio Products Laboratory at 14 sites in 5 countries.
Trial summary
The main objective of the study is to assess the pharmacokinetics of FACTOR X after a single dose of 25IU/kg. The secondary objectives of the study are to assess efficacy and safety of FACTOR X in the treatment of bleeding episodes over at least 6 months. Read more about this study
- Who is it for?
- Ages 12 years and older
- Conditions the study is for
- Factor X Deficiency
- What is being tested?
- Human Coagulation FACTOR X
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 16 participants
- Where
- 14 sitesin 5 countries
- Study dates
- Jan 2010to Nov 2013
- 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 12 years and older
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- Written informed consent given, or for subjects aged 12-17 years, have given written assent and whose parent/guardian has given written informed consent
- At least 12 years of age at dtae of written informed consent
- Have hereditary severe or moderate FX deficiency
- Currently treated with Fresh Frozen Plasma FFP, Prothrombin Complex Concentrate PCC or factor IX/X concentrate
- Must have a minimum of one spontaneous or menorrhagic bleed in the last 12 months which required treatment of FFP, PCC or factor IX/X concentrate. Newly diagnosed subjects who present at the hospital with a bleed may be included
- Must have had at least 7 days, and ideally 10-14 days, since an infusion of either FFP, PCC or factor IX/X concentrate at Baseline Visit
- Females of child bearing potential must have a negative result on a HCG based pregnancy test. If they are or become sexually active, they must practise contraception by using a method of proven reliability for the duration of the study
Who cannot join
- Have a history of inhibitor development to FX or a positive result at the Screening Visit
- Bleeding at the appointment for the PK assessment
- Subjects who have thrombocytopenia
- Have clinically significant liver disease
- Known to have other coagulopathy or thrombophilia
- Have known or suspected hypersensitivity to the investigational medicinal product or its excipients
Show all 11Show fewer
- Have abused chemicals or drugs within the past 12 months
- Have a history of unreliability or non-cooperation
- Participating or have taken part in another trial within the last 30 days, with the exception of BPL FX surgery study - Protocol Ten03. In such cases, subjects should have completed their End of Study Visit either before or on the day of Screening Visit for this study
- Female subjects who are pregnant or lactating
- Subjects planning greater than 4 weeks absence from the locality of the Investigational site, between the screening visit and the repeat PK assessment
Trial locations and contacts
14 sites in 5 countries.
14 sites in 5 countries
Turkey (Türkiye)6 sites
Cukurova University Hospital
Balcali, Adana
Ministry of Health Istanbul Goztepe Training & Research Hospital
Göztepe, Istanbul
Istanbul University Cerrahpasa School of Medicine
Istanbul
Kanuni Sultan Suleyman Training and Research Hospital
Istanbul
Prof. Kavakli
Izmir
Prof. Oner
Van
United States3 sites
Indiana Hemophilia & Thrombosis Center
Indianapolis, Indiana, 46260
Dr. William Mitchell New York Blood Center, Weill Cornell Medical College
New York, New York, 10065
UCSF School of Medicine
San Francisco, California, 94117
About this study
Official title: A Phase III Open, Multicentre Study to Investigate the Pharmacokinetics, Safety and Efficacy of BPL's High Purity Factor X in the Treatment of Severe and Moderate Factor X Deficiency.
The main objective of the study is to assess the pharmacokinetics of FACTOR X after a single dose of 25IU/kg.
The secondary objectives of the study are to assess efficacy and safety of FACTOR X in the treatment of bleeding episodes over at least 6 months.
- Conditions
- Factor X Deficiency
- Being tested
- Human Coagulation FACTOR X
- Medical subject headings
- Factor X Deficiency
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Human Coagulation FACTOR XBiological
Standard dose 25IU/kg to be administered at the Baseline Visit (1st PK assessment) and at the repeat PK assessment. Also administered to treat a bleed or to prevent a bleed.
| Study group | Role | What they receive |
|---|---|---|
| Human Coagulation FACTOR X | Experimental | Not described Receives: Biological: Human Coagulation FACTOR X |
What is being measured
The primary outcome is the main question the study is designed to answer.
FX:C Incremental Recovery
Incremental recovery is defined as the peak rise in plasma FX levels (IU/dL), as measured at 15, 30 and 60 minutes post-dose, divided by the dose (IU/kg).
Value given is the mean of 31 results: 16 for Baseline Visit + 15 for Repeat PK assessment
Measured: At Baseline (during first 60 minutes post-dose) and at 6 months post-Baseline (during first 60 minutes post-dose)
FX:C Half-life
Value given is the mean of 31 results: 16 for Baseline Visit + 15 for Repeat PK assessment
Measured: At Baseline and at 6 months post-Baseline
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted30 Jun 2009 (estimated)
- Study startJan 2010
- Primary completionNov 2013
- Study completionNov 2013
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 X DeficiencyStudies 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 3
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 16 participants (actual)
Sponsor and investigators
- Lead sponsor
- Bio Products Laboratory
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- Amy Shapiro, Dr, Principal investigator, Co-Medical Director, Indiana Hemophilia and Thrombosis Center, 8402 Harcourt Road, Suite 420, Indianapolis, IN46260, USA
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00930176
- Sponsor's protocol ID
- Ten01
- Status last verified
- Dec 2014
- First submitted
- 10 Jun 2009
- Last updated
- 12 Dec 2014
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- No
Publications
Oner AF, Celkan T, Timur C, Norton M, Kavakli K. Use of a High-Purity Factor X Concentrate in Turkish Subjects with Hereditary Factor X Deficiency: Post Hoc Cohort Subanalysis of a Phase 3 Study. Turk J Haematol. 2018 May 25;35(2):129-133. doi: 10.4274/tjh.2017.0446. Epub 2018 Mar 16.
PubMed 29545231
Study at a glance
From the registry record, last updated by the sponsor on 12 Dec 2014. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase III Open, Multicentre Study to Investigate the Pharmacokinetics, Safety and Efficacy of BPL's High Purity Factor X in the Treatment of Severe and Moderate Factor X Deficiency.
- ClinicalTrials.gov ID
- NCT00930176
- Acronym / sponsor ID
- Ten01
- Conditions
- Factor X Deficiency
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 16 participants (actual)
- Lead sponsor
- Bio Products LaboratoryUniversity, hospital or other
- Study start
- Jan 2010
- Primary completion
- Nov 2013 (actual)
- Study completion
- Nov 2013 (actual)
- First posted
- 30 Jun 2009 (estimated)
- Results
- Posted 12 Dec 2014
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
The main objective of the study is to assess the pharmacokinetics of FACTOR X after a single dose of 25IU/kg.
The secondary objectives of the study are to assess efficacy and safety of FACTOR X in the treatment of bleeding episodes over at least 6 months.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Human Coagulation FACTOR X | Experimental | Biological: Human Coagulation FACTOR X |
Who may qualify
- Ages
- 12 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (7 listed)
- Written informed consent given, or for subjects aged 12-17 years, have given written assent and whose parent/guardian has given written informed consent
- At least 12 years of age at dtae of written informed consent
- Have hereditary severe or moderate FX deficiency
- Currently treated with Fresh Frozen Plasma FFP, Prothrombin Complex Concentrate PCC or factor IX/X concentrate
- Must have a minimum of one spontaneous or menorrhagic bleed in the last 12 months which required treatment of FFP, PCC or factor IX/X concentrate. Newly diagnosed subjects who present at the hospital with a bleed may be included
Key exclusion criteria (11 listed)
- Have a history of inhibitor development to FX or a positive result at the Screening Visit
- Bleeding at the appointment for the PK assessment
- Subjects who have thrombocytopenia
- Have clinically significant liver disease
- Known to have other coagulopathy or thrombophilia
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 |
|---|---|---|
| Human Coagulation FACTOR XStandard dose 25IU/kg to be administered at the Baseline Visit (1st PK assessment) and at the repeat PK assessment. Also administered to treat a bleed or to prevent a bleed. | Biological | Human Coagulation FACTOR X |
Primary outcome
FX:C Incremental Recovery
Time frame: At Baseline (during first 60 minutes post-dose) and at 6 months post-Baseline (during first 60 minutes post-dose)
Incremental recovery is defined as the peak rise in plasma FX levels (IU/dL), as measured at 15, 30 and 60 minutes post-dose, divided by the dose (IU/kg). Value given is the mean of 31 results: 16 for Baseline Visit + 15 for Repeat PK assessment
FX:C Half-life
Time frame: At Baseline and at 6 months post-Baseline
Value given is the mean of 31 results: 16 for Baseline Visit + 15 for Repeat PK assessment
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 12 Dec 2014.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Derived from this study: PubMed 29545231Oner AF, Celkan T, Timur C, Norton M, Kavakli K. Use of a High-Purity Factor X Concentrate in Turkish Subjects with Hereditary Factor X Deficiency: Post Hoc Cohort Subanalysis of a Phase 3 Study. Turk J Haematol. 2018 May 25;35(2):129-133. doi: 10.4274/tjh.2017.0446. Epub 2018 Mar 16.
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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.
Amy Shapiro, Dr principal investigator
Co-Medical Director, Indiana Hemophilia and Thrombosis Center, 8402 Harcourt Road, Suite 420, Indianapolis, IN46260, USA
14 sites in 5 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing Human Coagulation FACTOR X in people with Factor X Deficiency, run by Bio Products Laboratory at 14 sites in 5 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 7 inclusion and 11 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 Dec 2014. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 14 sites in Turkey (Türkiye), United States, Spain, United Kingdom, Germany. 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 Dec 2014). 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?
FX:C Incremental Recovery (time frame: At Baseline (during first 60 minutes post-dose) and at 6 months post-Baseline (during first 60 minutes post-dose)). The study lists 2 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 NCT00930176, 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 12 Dec 2014.