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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/study/NCT03623295




Registration number
NCT03623295
Ethics application status
Date submitted
6/08/2018
Date registered
9/08/2018
Date last updated
9/08/2018

Titles & IDs
Public title
The Dynamic Interplay Between Bleeding Phenotype and Baseline Factor Level in Moderate and Mild Hemophilia A and B
Scientific title
The Dynamic Interplay Between Bleeding Phenotype and Baseline Factor Level in Moderate and Mild Hemophilia A and B
Secondary ID [1] 0 0
NL61564.018.17
Universal Trial Number (UTN)
Trial acronym
DYNAMO
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Hemophilia 0 0
Condition category
Condition code
Blood 0 0 0 0
Clotting disorders
Human Genetics and Inherited Disorders 0 0 0 0
Other human genetics and inherited disorders

Intervention/exposure
Study type
Observational
Patient registry
Target follow-up duration
Target follow-up type
Description of intervention(s) / exposure
Other interventions - Blood sample
Other interventions - Questionnaire
Other interventions - MRI-imaging
Other interventions - Physical examination

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.

Sub study population - A subset of 200 patients of the cohort study population will be investigated in more detail by longitudinal data collection.


Other interventions: Blood sample
Blood withdrawal.

Other interventions: Questionnaire
Online questionnaire about the bleeds that patients experienced in the past.

Other interventions: MRI-imaging
MRI imaging of joints.

Other interventions: Physical examination
Physical examination of joint status.
Intervention code [1] 0 0
Other interventions
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Bleeding phenotype
Timepoint [1] 0 0
Retrospective 10 years

Eligibility
Key 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
Minimum age
12 Years
Maximum age
55 Years
Sex
Males
Can healthy volunteers participate?
No
Key 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 design
Purpose
Duration
Selection
Timing
Statistical methods / analysis

Recruitment
Recruitment status
UNKNOWN
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
1/01/2018
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
1/04/2022
Actual
Sample size
Target
1000
Accrual to date
Final
Recruitment in Australia
Recruitment state(s)
Recruitment hospital [1] 0 0
Royal Adelaide Hospital - Adelaide
Recruitment postcode(s) [1] 0 0
- Adelaide
Recruitment outside Australia
Country [1] 0 0
Austria
State/province [1] 0 0
Vienna
Country [2] 0 0
Belgium
State/province [2] 0 0
Multiple Locations
Country [3] 0 0
Canada
State/province [3] 0 0
Multiple Locations
Country [4] 0 0
Finland
State/province [4] 0 0
Helsinki
Country [5] 0 0
Germany
State/province [5] 0 0
Multiple Locations
Country [6] 0 0
Italy
State/province [6] 0 0
Multiple Locations
Country [7] 0 0
Netherlands
State/province [7] 0 0
Amsterdam
Country [8] 0 0
Netherlands
State/province [8] 0 0
Groningen
Country [9] 0 0
Netherlands
State/province [9] 0 0
Leiden
Country [10] 0 0
Netherlands
State/province [10] 0 0
Maastricht
Country [11] 0 0
Netherlands
State/province [11] 0 0
Nijmegen
Country [12] 0 0
Netherlands
State/province [12] 0 0
Rotterdam
Country [13] 0 0
Netherlands
State/province [13] 0 0
Utrecht
Country [14] 0 0
Netherlands
State/province [14] 0 0
Veldhoven
Country [15] 0 0
Spain
State/province [15] 0 0
Multiple Locations
Country [16] 0 0
United Kingdom
State/province [16] 0 0
Multiple Locations

Funding & Sponsors
Primary sponsor type
Other
Name
Academisch Medisch Centrum - Universiteit van Amsterdam (AMC-UvA)
Address
Country

Ethics approval
Ethics application status

Summary
Brief summary
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.
Trial website
https://clinicaltrials.gov/study/NCT03623295
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 0 0
Karin Fijnvandraat
Address 0 0
Academisch Medisch Centrum - Universiteit van Amsterdam (AMC-UvA)
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for public queries
Name 0 0
Anne-Fleur Zwagemaker
Address 0 0
Country 0 0
Phone 0 0
+31205668668
Fax 0 0
Email 0 0
[email protected]
Contact person for scientific queries



Summary Results

For IPD and results data, please see https://clinicaltrials.gov/study/NCT03623295