HemophiliaDYNAMONCT03623295
The Dynamic Interplay Between Bleeding Phenotype and Baseline Factor Level in Moderate and Mild Hemophilia A and B
An observational study of Hemophilia, run by Academisch Medisch Centrum - Universiteit van Amsterdam (AMC-UvA) at 17 sites in 10 countries.
Trial 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. Read more about this study
- Who is it for?
- Ages 12 to 55 years, men only
- Conditions the study is for
- Hemophilia
- What is being tested?
- Blood sample, Questionnaire and MRI-imaging
- How many people
- 304 participants
- Where
- 17 sitesin 10 countries
- Study dates
- 1 Jan 2018to 1 Oct 2021
- 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 12 to 55 years
- Men only
- No healthy volunteers
- Child, Adult
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.
Who may be able to join
- 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
Who cannot join
- 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
Trial locations and contacts
17 sites in 10 countries.
17 sites in 10 countries
Netherlands8 sites
Academic Medical Center
Amsterdam
University Medical Center Groningen
Groningen
Leiden University Medical Center
Leiden
Maastricht University Medical Center
Maastricht
Radboud University Medical Center
Nijmegen
Erasmus Medical Center
Rotterdam
Utrecht University Medical Center
Utrecht
Máxima Medical Center
Veldhoven
Germany1 site
Multicentre: Bonn, Berlin, Frankfurt, München, Hamburg
Multiple Locations
United Kingdom1 site
Multicentre: Manchester, London, Liverpool, Glasgow, Cardiff, Sheffield
Multiple Locations
About this study
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.
Read more
The project is a multicentre observational cohort study. We will include 230 patients with moderate or mild hemophilia A or B (FVIII/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 50 patients aged 24 years or older with mild and moderate hemophilia A 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.
- Conditions
- Hemophilia
- Being tested
- Blood sample, Questionnaire, MRI-imaging, Physical examination
- Medical subject headings
- Hemophilia A
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Blood sampleOther
Blood withdrawal.
QuestionnaireOther
Online questionnaire about the bleeds that patients experienced in the past.
MRI-imagingOther
MRI imaging of joints.
Physical examinationOther
Physical examination of joint status.
| Study group | Role | What they receive |
|---|---|---|
| Cohort study population | For the main cohort study, we will include 230 patients with moderate or mild hemophilia A or B. Receives: Other: Blood sample; Other: Questionnaire | |
| Sub study population | A subset of 50 patients of the cohort study population will be investigated in more detail by longitudinal data collection. Receives: Other: Blood sample; Other: MRI-imaging; Other: Physical examination |
What is being measured
The primary outcome is the main question the study is designed to answer.
Bleeding phenotype
Annual bleeding rate, annual major bleeding rate, annual spontaneous joint bleeding rate, annual joint bleeding rate
Measured: Retrospective 10 years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted9 Aug 2018
- Study start1 Jan 2018
- Primary completion1 Oct 2021
- Study completion1 Oct 2021
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for HemophiliaStudies 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
- Observational model
- Cohort
- Time perspective
- Other
- Enrolment
- 304 participants (actual)
Sponsor and investigators
- Lead sponsor
- Academisch Medisch Centrum - Universiteit van Amsterdam (AMC-UvA)
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Karin Fijnvandraat, Academisch Medisch Centrum - Universiteit van Amsterdam (AMC-UvA)
- Investigators
- Karin Fijnvandraat, Principal investigator, Academisch Medisch Centrum - Universiteit van Amsterdam (AMC-UvA)
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03623295
- Sponsor's protocol ID
- NL61564.018.17
- Acronym
- DYNAMO
- Status last verified
- Oct 2021
- First submitted
- 6 Aug 2018
- Last updated
- 30 Apr 2025
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 30 Apr 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- The Dynamic Interplay Between Bleeding Phenotype and Baseline Factor Level in Moderate and Mild Hemophilia A and B
- ClinicalTrials.gov ID
- NCT03623295
- Acronym / sponsor ID
- DYNAMO / NL61564.018.17
- Conditions
- Hemophilia
- Study type
- Observational
- Recruitment status
- Completed
- Enrolment
- 304 participants (actual)
- Lead sponsor
- Academisch Medisch Centrum - Universiteit van Amsterdam (AMC-UvA)University, hospital or other
- Study start
- 1 Jan 2018 (actual)
- Primary completion
- 1 Oct 2021 (actual)
- Study completion
- 1 Oct 2021 (actual)
- First posted
- 9 Aug 2018 (actual)
- 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.
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 230 patients with moderate or mild hemophilia A or B (FVIII/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 50 patients aged 24 years or older with mild and moderate hemophilia A 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 design
- Observational model
- Cohort
- Time perspective
- Other
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Cohort study population | – | Other: Blood sample, Other: QuestionnaireFor the main cohort study, we will include 230 patients with moderate or mild hemophilia A or B. |
| Sub study population | – | Other: Blood sample, Other: MRI-imaging, Other: Physical examinationA subset of 50 patients of the cohort study population will be investigated in more detail by longitudinal data collection. |
Who may qualify
- Ages
- 12 to 55 years
- Sex
- Male only
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
- 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.
Key inclusion criteria (2 listed)
- 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
Key exclusion criteria (6 listed)
- 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
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 |
|---|---|---|
| Blood sampleBlood withdrawal. | Other | Cohort study population, Sub study population |
| QuestionnaireOnline questionnaire about the bleeds that patients experienced in the past. | Other | Cohort study population |
| MRI-imagingMRI imaging of joints. | Other | Sub study population |
| Physical examinationPhysical examination of joint status. | Other | Sub study population |
Primary outcome
Bleeding phenotype
Time frame: Retrospective 10 years
Annual bleeding rate, annual major bleeding rate, annual spontaneous joint bleeding rate, annual joint bleeding rate
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 30 Apr 2025.
- Individual participant dataSharing planned: No
Related trials for Hemophilia
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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.
Karin Fijnvandraat principal investigator
Academisch Medisch Centrum - Universiteit van Amsterdam (AMC-UvA)
17 sites in 10 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Hemophilia, run by Academisch Medisch Centrum - Universiteit van Amsterdam (AMC-UvA) at 17 sites in 10 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 2 inclusion and 6 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 30 Apr 2025. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 17 sites in Netherlands, Australia, Austria, Belgium, Canada and 5 more countries. 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?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
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?
Bleeding phenotype (time frame: Retrospective 10 years). 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 30 Apr 2025.