Hemophilia A With InhibitorNCT02554526
Basic Study of Combination Therapy Based on APCC-induced FVllI Activation in Hemophilia A Patients With Inhibitors
An observational study of Hemophilia A With Inhibitor, run by Nara Medical University at 1 site in Japan.
Trial summary
Using whole blood samples and plasma samples obtained from some hemophilia A patients with inhibitors, the investigators will perform the coagulation assessment in the co-presence of aPCC and factor VIII by comprehensive coagulation assays and flow chamber analysis under blood flow conditions. Read more about this study
- Who is it for?
- Any age, men only
- Conditions the study is for
- Hemophilia A With Inhibitor
- How many people
- 10 participants
- Where
- 1 siteJapan
- Study dates
- Sep 2015to Dec 2017
- Phase
- Observational
- Are there results?
- None posted in the registry
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
- Men only
- No healthy volunteers
- Child, Adult, Older adult
Study population: We guess that there are approximately 200-300 hemophilia A patients with inhibitors in our country. We have approximately 20 out-patients in our hospital. Based on this information, we set up the patients subjects (n=10).
Who may be able to join
- Patients with hemophilia A with inhibitors (Inhibitor titer; >0.5 Bethesda U/ml)
- Patients with agreement consents with the patients signatures
Who cannot join
- Patients that do not fill the conditions described above
Trial locations and contacts
1 site in 1 country.
Japan1 site
Department of Pediatrics
Kashihara, Nara, 634-8522
Keiji Nogami, MD, PhD, +81-744-29-8881, roc-noga@naramed-u.ac.jp
Tomoko Matsumoto, PhD
About this study
Official title: Basic Study on Novel Therapeutic Strategy With Bypassing Agent Therapy in Hemophilia A Patients With Inhibitors
Using whole blood samples and plasma samples obtained from some hemophilia A patients with inhibitors, the investigators will perform the coagulation assessment in the co-presence of aPCC and factor VIII by comprehensive coagulation assays and flow chamber analysis under blood flow conditions.
Read the detailed description
Comprehensive coagulation assays using recently popular rotational thromboelastometry (ROTEM), factor Xa/thrombin/plasmin generation test, and coagulation wave analysis and recently developed flow chamber system under blood flow conditions (T-TAS®) will be used. A bypassing agent, aPCC (Feiba®) and recombinant factor VIII (Advate®) will be added ex vivo to whole blood and plasma samples from patients with hemophilia A with inhibitors to compare with the coagulation effects of aPCC in the presence of factor VIII and those of aPCC in its absence under the conditions close to the physiological condition. Furthermore, the difference in the coagulation effects by the difference in inhibitor epitopes will be examined.
- Conditions
- Hemophilia A With Inhibitor
- Keywords
- hemophilia A, inhibitor, bypassing therapy, factor VIII
- Medical subject headings
- Hemophilia A
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
| Study group | Role | What they receive |
|---|---|---|
| Combination therapy | Effects of aPCC reaction in the presence of FVIII |
What is being measured
The primary outcome is the main question the study is designed to answer.
Evaluation of coagulation effects using patients' plasmas by FXa/thrombin/plasmin generation tests and clot waveform analysis
Measured: up to 10 months
Evaluation of coagulation effects using patients' whole bloods by ROTEM and T-TAS
Measured: up to 15 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted18 Sep 2015 (estimated)
- Study startSep 2015
- Primary completionMar 2017 (estimated)
- Study completionDec 2017 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Hemophilia A With InhibitorStudies 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
- Observational
- Enrolment
- 10 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Nara Medical University
- Sponsor type
- University, hospital or other
- Collaborators
- Shire
- Responsible party
- Principal investigator, Keiji Nogami, MD,PhD, Nara Medical University
- Investigators
- Midori Shima, MD, PhD, Study director, Nara Medical University
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02554526
- Sponsor's protocol ID
- J150100036
- Status last verified
- Sep 2015
- First submitted
- 15 Sep 2015
- Last updated
- 18 Sep 2015
- Results posted
- No
- Data monitoring committee
- No
Study at a glance
From the registry record, last updated by the sponsor on 18 Sep 2015. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Basic Study on Novel Therapeutic Strategy With Bypassing Agent Therapy in Hemophilia A Patients With Inhibitors
- ClinicalTrials.gov ID
- NCT02554526
- Acronym / sponsor ID
- J150100036
- Conditions
- Hemophilia A With Inhibitor
- Keywords
- hemophilia A, inhibitor, bypassing therapy, factor VIII
- Study type
- Observational
- Recruitment status
- Unknown status
- Enrolment
- 10 participants (estimated)
- Lead sponsor
- Nara Medical UniversityUniversity, hospital or other
- Collaborators
- Shire
- Study start
- Sep 2015
- Primary completion
- Mar 2017 (estimated)
- Study completion
- Dec 2017 (estimated)
- First posted
- 18 Sep 2015 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
Using whole blood samples and plasma samples obtained from some hemophilia A patients with inhibitors, the investigators will perform the coagulation assessment in the co-presence of aPCC and factor VIII by comprehensive coagulation assays and flow chamber analysis under blood flow conditions.
Detailed description
Comprehensive coagulation assays using recently popular rotational thromboelastometry (ROTEM), factor Xa/thrombin/plasmin generation test, and coagulation wave analysis and recently developed flow chamber system under blood flow conditions (T-TAS®) will be used. A bypassing agent, aPCC (Feiba®) and recombinant factor VIII (Advate®) will be added ex vivo to whole blood and plasma samples from patients with hemophilia A with inhibitors to compare with the coagulation effects of aPCC in the presence of factor VIII and those of aPCC in its absence under the conditions close to the physiological condition. Furthermore, the difference in the coagulation effects by the difference in inhibitor epitopes will be examined.
Study design
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Combination therapy | – | –Effects of aPCC reaction in the presence of FVIII |
Who may qualify
- Sex
- Male only
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
- Study population
- We guess that there are approximately 200-300 hemophilia A patients with inhibitors in our country. We have approximately 20 out-patients in our hospital. Based on this information, we set up the patients subjects (n=10).
Key inclusion criteria (2 listed)
- Patients with hemophilia A with inhibitors (Inhibitor titer; >0.5 Bethesda U/ml)
- Patients with agreement consents with the patients signatures
Key exclusion criteria (1 listed)
- Patients that do not fill the conditions described above
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Primary outcome
Evaluation of coagulation effects using patients' plasmas by FXa/thrombin/plasmin generation tests and clot waveform analysis
Time frame: up to 10 months
Evaluation of coagulation effects using patients' whole bloods by ROTEM and T-TAS
Time frame: up to 15 months
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 18 Sep 2015.
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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.
Midori Shima, MD, PhD study director
Nara Medical University
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Hemophilia A With Inhibitor, run by Nara Medical University at 1 site in Japan.
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 2 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: status not confirmed, as of the sponsor's last update on 18 Sep 2015. 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 Japan. 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?
Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
Contact Keiji Nogami, MD, PhD (+81-744-29-8881, roc-noga@naramed-u.ac.jp), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Evaluation of coagulation effects using patients' plasmas by FXa/thrombin/plasmin generation tests and clot waveform analysis (time frame: up to 10 months). 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?
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 18 Sep 2015.