Basic Study of Combination Therapy Based on APCC-induced FVllI Activation in Hemophilia A Patients With Inhibitors

September 17, 2015 updated by: Keiji Nogami, MD,PhD, Nara Medical University

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.

Study Overview

Status

Unknown

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 Type

Observational

Enrollment (Anticipated)

10

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • Japan
    • Nara
      • Kashihara, Nara, Japan, 634-8522
        • Department of Pediatrics

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

  • Child
  • Adult
  • Older Adult

Accepts Healthy Volunteers

No

Genders Eligible for Study

Male

Sampling Method

Non-Probability Sample

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).

Description

Inclusion Criteria:

  • Patients with hemophilia A with inhibitors (Inhibitor titer; >0.5 Bethesda U/ml)
  • Patients with agreement consents with the patients signatures

Exclusion Criteria:

  • Patients that do not fill the conditions described above

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Cohorts and Interventions

Group / Cohort
Combination therapy
Effects of aPCC reaction in the presence of FVIII

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Time Frame
Evaluation of coagulation effects using patients' plasmas by FXa/thrombin/plasmin generation tests and clot waveform analysis
Time Frame: up to 10 months
up to 10 months
Evaluation of coagulation effects using patients' whole bloods by ROTEM and T-TAS
Time Frame: up to 15 months
up to 15 months

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Nara Medical University

Collaborators

Shire

Investigators

  • Study Director: Midori Shima, MD, PhD, Nara Medical University

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start

September 1, 2015

Primary Completion (Anticipated)

March 1, 2017

Study Completion (Anticipated)

December 1, 2017

Study Registration Dates

First Submitted

September 15, 2015

First Submitted That Met QC Criteria

September 17, 2015

First Posted (Estimate)

September 18, 2015

Study Record Updates

Last Update Posted (Estimate)

September 18, 2015

Last Update Submitted That Met QC Criteria

September 17, 2015

Last Verified

September 1, 2015

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • J150100036

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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