The Dynamic Interplay Between Bleeding Phenotype and Baseline Factor Level in Moderate and Mild Hemophilia A and B (DYNAMO)

August 6, 2018 updated by: Karin Fijnvandraat, Academisch Medisch Centrum - Universiteit van Amsterdam (AMC-UvA)

There are large inter-individual differences in the bleeding pattern of patients with moderate or mild hemophilia. The major determinant of bleeding phenotype is the level of coagulant factor VIII or IX. In hemophilia A, studies addressing the association between factor VIII level and the clinical bleeding pattern yield conflicting results. In hemophilia B such studies have not yet been performed.

The primary aim of this project is to analyze the association between factor VIII and factor IX levels and the bleeding phenotype. The secondary aim is to analyze potential differences in phenotype between hemophilia A and B.

The project is a multicentre observational cohort study. We will include 500 patients with moderate or mild hemophilia A (FVIII 0.02-0.35 IU/mL) and 500 patients with moderate or mild hemophilia B (FIX 0.02-0.35 IU/mL) who are 12 to 55 years old. The main cohort study consists of clinical data collection, one blood sample and an online questionnaire for patients. Data will be collected on the nature and duration of all bleeding episodes, disease and treatment characteristics, physical activity level and musculoskeletal status. One blood withdrawal will be performed for centralized laboratory assays for FVIII or FIX levels (both one-stage and chromogenic assays) and genetic analysis for the most prevalent prothrombotic mutations. The online questionnaire for patients focuses on bleeds experienced in the past.

A subset of 200 patients aged 24 years or older (100 with moderate or mild hemophilia A and 100 with moderate or mild hemophilia B) will be investigated in more detail by longitudinal data collection including analysis of physical joint status, MRI imaging of joints and biomarkers for joint damage. This longitudinal observation will consist of two time points that lie two years apart, allowing us to identify any changes that occur over the observed time period with respect to joint status.

Study Overview

Status

Unknown

Conditions

Intervention / Treatment

Study Type

Observational

Enrollment (Anticipated)

1000

Contacts and Locations

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

Study Contact

Study Contact Backup

Study Locations

  • Australia
      • Adelaide, Australia
        • Not yet recruiting
        • Royal Adelaide Hospital
        • Contact:
  • Austria
      • Vienna, Austria
        • Not yet recruiting
        • Medical University of Vienna
        • Contact:
  • Belgium
      • Multiple Locations, Belgium
        • Not yet recruiting
        • Multicentre: Leuven, Brussels
        • Contact:
  • Canada
      • Multiple Locations, Canada
        • Not yet recruiting
        • Multicentre: Vancouver, Toronto, Hamilton
        • Contact:
  • Finland
      • Helsinki, Finland
        • Not yet recruiting
        • Helsinki University Central Hospital
        • Contact:
  • Germany
      • Multiple Locations, Germany
        • Not yet recruiting
        • Multicentre: Bonn, Berlin, Frankfurt, München, Hamburg
        • Contact:
  • Italy
      • Multiple Locations, Italy
        • Not yet recruiting
        • Multicentre: Florence, Rome, Parma, Milan, Turin
        • Contact:
  • Netherlands
      • Amsterdam, Netherlands
        • Recruiting
        • Academic Medical Center
        • Contact:
      • Groningen, Netherlands
        • Recruiting
        • University Medical Center Groningen
        • Contact:
      • Leiden, Netherlands
        • Not yet recruiting
        • Leiden University Medical Center
        • Contact:
      • Maastricht, Netherlands
        • Not yet recruiting
        • Maastricht University Medical Center
        • Contact:
      • Nijmegen, Netherlands
        • Not yet recruiting
        • Radboud University Medical Center
        • Contact:
      • Rotterdam, Netherlands
        • Not yet recruiting
        • Erasmus Medical Center
        • Contact:
      • Utrecht, Netherlands
        • Not yet recruiting
        • Utrecht University Medical Center
        • Contact:
      • Veldhoven, Netherlands
        • Not yet recruiting
        • Maxima Medical Center
        • Contact:
  • Spain
      • Multiple Locations, Spain
        • Not yet recruiting
        • Multicentre: Valencia, Madrid, Barcelona
        • Contact:
  • United Kingdom
      • Multiple Locations, United Kingdom
        • Not yet recruiting
        • Multicentre: Manchester, London, Liverpool, Glasgow, Cardiff, Sheffield
        • Contact:

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

12 years to 55 years (ADULT, CHILD)

Accepts Healthy Volunteers

No

Genders Eligible for Study

Male

Sampling Method

Probability Sample

Study Population

Patients will be recruited from Hemophilia Treatment Centers participating in the INSIGHT consortium, a well-established and productive group of investigators from European countries, Canada and Australia.

Description

Inclusion Criteria:

  • Moderate or mild hemophilia A (FVIII:C 0.02-0.35 IU/mL) or hemophilia B (FIX:C 0.02-0.35 IU/mL)
  • Age from 12 up to and including 55 years

Exclusion Criteria:

  • Other clotting disorder
  • Participation in another trial with an investigational product
  • Comorbidity affecting the musculoskeletal status
  • Clinically relevant inhibitor status at present or in the past
  • Hemophilia B Leyden
  • Use of anticoagulants

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?

Design Details

Cohorts and Interventions

Group / Cohort
Intervention / Treatment
Cohort study population
For the main cohort study, we will include 500 patients with moderate or mild hemophilia A and 500 patients with moderate or mild hemophilia B.
Other: Blood sample
Blood withdrawal.
Other: Questionnaire
Online questionnaire about the bleeds that patients experienced in the past.
Sub study population
A subset of 200 patients of the cohort study population will be investigated in more detail by longitudinal data collection.
Other: Blood sample
Blood withdrawal.
Other: MRI-imaging
MRI imaging of joints.
Other: Physical examination
Physical examination of joint status.

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Bleeding phenotype
Time Frame: Retrospective 10 years
Annual bleeding rate, annual major bleeding rate, annual spontaneous joint bleeding rate, annual joint bleeding rate
Retrospective 10 years

Collaborators and Investigators

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

Sponsor

Academisch Medisch Centrum - Universiteit van Amsterdam (AMC-UvA)

Investigators

  • Principal Investigator: Karin Fijnvandraat, Academisch Medisch Centrum - Universiteit van Amsterdam (AMC-UvA)

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 (ACTUAL)

January 1, 2018

Primary Completion (ANTICIPATED)

April 1, 2020

Study Completion (ANTICIPATED)

April 1, 2022

Study Registration Dates

First Submitted

August 6, 2018

First Submitted That Met QC Criteria

August 6, 2018

First Posted (ACTUAL)

August 9, 2018

Study Record Updates

Last Update Posted (ACTUAL)

August 9, 2018

Last Update Submitted That Met QC Criteria

August 6, 2018

Last Verified

August 1, 2018

More Information

Terms related to this study

Additional Relevant MeSH Terms

Other Study ID Numbers

  • NL61564.018.17

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

NO

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

No

Studies a U.S. FDA-regulated device product

No

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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