HemophiliaNCT01232634
Validation of Ultrasound as a Diagnostic Tool for Assessment of Hemophilic Arthropathy of Knees and Ankles
An observational study of Hemophilia and Hemophilic Arthropathy, run by The Hospital for Sick Children at 4 sites in 2 countries.
Trial summary
The purpose of this study is to test the external validity of the systemic ultrasound protocol for data acquisition and interpretation, in order to diagnose soft tissue and osteochondral abnormalities in hemophilic children. Read more about this study
- Who is it for?
- Ages 7 to 18 years, men only
- Conditions the study is for
- Hemophilia, Hemophilic Arthropathy
- How many people
- 57 participants
- Where
- 4 sitesin 2 countries
- Study dates
- Sep 2010to Mar 2014
- 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 7 to 18 years
- Men only
- No healthy volunteers
- Child, Adult
Study population: Children attending the Hemophilia clinics at the participating institutions (Universidade de São Paulo, Universidade Federal de São Paulo and Universidade Estadual de Campinas), who have a history of ankle or knee bleed(s), will be recruited to take part in this study.
Who may be able to join
- Diagnosis of hemophilia A (factor VIII deficiency ≤1%) or B (factor IX deficiency ≤1.0%)
- Cooperative patients (age of 7 to 18 years old)
- Patients under 7 years old who have clinical evidence of arthropathy and imaging studies are required for clinical reasons. In these cases, the procedures may be done under sedation.
- Male sex (higher prevalence in males)
- History of previous ankle or knee bleed
Who cannot join
- Co-morbid illness such as juvenile idiopathic arthritis, muscular dystrophy, neuropathic arthropathy that cause osteoarticular findings that may obscure or confound the hemophilia-based joint findings
- Non-cooperative patients
- Active bleed (defined as evidence of a recent bleed with one week of the proposing imaging studies and/or musculoskeletal changes on physical examination suggestive of a bleed within one week of the proposed imaging studies).
- Prior synovectomy
Trial locations and contacts
4 sites in 2 countries.
4 sites in 2 countries
Brazil3 sites
Universidade de Sao Paulo
São Paulo
Universidade Estaduale de Campinas
São Paulo
Universidade Federal de Sao Paulo
São Paulo
About this study
Official title: External Validation of Ultrasound as a Diagnostic Tool for Assessment of Hemophilic Arthropathy of Knees and Ankles. MRI Correlation.
The purpose of this study is to test the external validity of the systemic ultrasound protocol for data acquisition and interpretation, in order to diagnose soft tissue and osteochondral abnormalities in hemophilic children.
Read the detailed description
Ultrasound has advantages over MRI as it is less expensive, does not require sedation and is more readily available in centres around the world. Given these characteristics of ultrasound it is an ideal imaging tool for early assessment of hemophilic joints in countries whose access to MRI is limited. Early evaluation of soft tissue changes in young hemophilic patients may lead to earlier interventions and better mid-term and long-term clinical outcomes. By this way the joint function of hemophilic patients can be preserved, saving their joints from severe arthropathy and offering them the possibility of a normal professional life in the society.
- Conditions
- Hemophilia, Hemophilic Arthropathy
- Keywords
- pediatrics, haemophilia, ultrasound, MRI, arthropathy
- Medical subject headings
- Hemophilia A, Joint Diseases
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 |
|---|---|---|
| All Subjects | Not described |
What is being measured
The primary outcome is the main question the study is designed to answer.
Ultrasound findings according to the International Prophylaxis Study Group (IPSG) scale
The readers will mark all the positive findings on a standardized spreadsheet and providing a final score for each component of the scale. Color Doppler findings will be rated as normal (grade 0), mild/moderate (grade 1) or severe (grade 2) synovial hyperemia according to an atlas that shows the intensity of hyperemia, which has been prepared by our group.
Measured: Day 1
Secondary outcomes (3)
MRI findings according to the International Prophylaxis Study Group (IPSG) scale
The readers will interpret the MRI findings in an additive way, i.e., marking all positive findings on a standardized spreadsheet and providing a final score for the soft tissue and osteochondral components of the MRI scale.
Measured: Day 1
X-ray findings according to the Pettersson radiographic system
Each joint will be rated on a 13-point score after the Pettersson system.
Measured: Day 1
Physical Assessment according to the HJHS and FISH scores
Each knee, elbow and ankle joint receives a numeric score, which can be compared to itself over time to determine whether it is showing degeneration due to bleeding. FISH score is a seven item measure of disability used to evaluate change in functional independence over time.
Measured: Day 1
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted2 Nov 2010 (estimated)
- Study startSep 2010
- Primary completionNov 2013
- Study completionMar 2014
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.
- More trials run by The Hospital for Sick ChildrenThe sponsor's other registered studies.
- 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
- Case only
- Time perspective
- Prospective
- Enrolment
- 57 participants (actual)
Sponsor and investigators
- Lead sponsor
- The Hospital for Sick Children
- Sponsor type
- University, hospital or other
- Collaborators
- University of Sao Paulo, Federal University of São Paulo, University of Campinas, Brazil, Baxter Healthcare Corporation
- Responsible party
- Principal investigator, Andrea Doria, The Hospital for Sick Children
- Investigators
- Andrea Doria, MD, Principal investigator, The Hospital for Sick Children, Toronto Canada
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01232634
- Sponsor's protocol ID
- 1000010841
- Status last verified
- Nov 2016
- First submitted
- 29 Oct 2010
- Last updated
- 9 Nov 2016
- Results posted
- No
- Data monitoring committee
- No
Study at a glance
From the registry record, last updated by the sponsor on 9 Nov 2016. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- External Validation of Ultrasound as a Diagnostic Tool for Assessment of Hemophilic Arthropathy of Knees and Ankles. MRI Correlation.
- ClinicalTrials.gov ID
- NCT01232634
- Acronym / sponsor ID
- 1000010841
- Conditions
- Hemophilia, Hemophilic Arthropathy
- Keywords
- pediatrics, haemophilia, ultrasound, MRI, arthropathy
- Study type
- Observational
- Recruitment status
- Completed
- Enrolment
- 57 participants (actual)
- Lead sponsor
- The Hospital for Sick ChildrenUniversity, hospital or other
- Collaborators
- University of Sao Paulo, Federal University of São Paulo, University of Campinas, Brazil, Baxter Healthcare Corporation
- Study start
- Sep 2010
- Primary completion
- Nov 2013 (actual)
- Study completion
- Mar 2014 (actual)
- First posted
- 2 Nov 2010 (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.
The purpose of this study is to test the external validity of the systemic ultrasound protocol for data acquisition and interpretation, in order to diagnose soft tissue and osteochondral abnormalities in hemophilic children.
Detailed description
Ultrasound has advantages over MRI as it is less expensive, does not require sedation and is more readily available in centres around the world. Given these characteristics of ultrasound it is an ideal imaging tool for early assessment of hemophilic joints in countries whose access to MRI is limited. Early evaluation of soft tissue changes in young hemophilic patients may lead to earlier interventions and better mid-term and long-term clinical outcomes. By this way the joint function of hemophilic patients can be preserved, saving their joints from severe arthropathy and offering them the possibility of a normal professional life in the society.
Study design
- Observational model
- Case only
- Time perspective
- Prospective
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| All Subjects | – | – |
Who may qualify
- Ages
- 7 to 18 years
- Sex
- Male only
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
- Study population
- Children attending the Hemophilia clinics at the participating institutions (Universidade de São Paulo, Universidade Federal de São Paulo and Universidade Estadual de Campinas), who have a history of ankle or knee bleed(s), will be recruited to take part in this study.
Key inclusion criteria (5 listed)
- Diagnosis of hemophilia A (factor VIII deficiency ≤1%) or B (factor IX deficiency ≤1.0%)
- Cooperative patients (age of 7 to 18 years old)
- Patients under 7 years old who have clinical evidence of arthropathy and imaging studies are required for clinical reasons. In these cases, the procedures may be done under sedation.
- Male sex (higher prevalence in males)
- History of previous ankle or knee bleed
Key exclusion criteria (4 listed)
- Co-morbid illness such as juvenile idiopathic arthritis, muscular dystrophy, neuropathic arthropathy that cause osteoarticular findings that may obscure or confound the hemophilia-based joint findings
- Non-cooperative patients
- Active bleed (defined as evidence of a recent bleed with one week of the proposing imaging studies and/or musculoskeletal changes on physical examination suggestive of a bleed within one week of the proposed imaging studies).
- Prior synovectomy
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
Ultrasound findings according to the International Prophylaxis Study Group (IPSG) scale
Time frame: Day 1
The readers will mark all the positive findings on a standardized spreadsheet and providing a final score for each component of the scale. Color Doppler findings will be rated as normal (grade 0), mild/moderate (grade 1) or severe (grade 2) synovial hyperemia according to an atlas that shows the intensity of hyperemia, which has been prepared by our group.
3 secondary outcomes
MRI findings according to the International Prophylaxis Study Group (IPSG) scale
Time frame: Day 1
X-ray findings according to the Pettersson radiographic system
Time frame: Day 1
Physical Assessment according to the HJHS and FISH scores
Time frame: Day 1
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 9 Nov 2016.
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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.
Andrea Doria, MD principal investigator
The Hospital for Sick Children, Toronto Canada
4 sites in 2 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Hemophilia and Hemophilic Arthropathy, run by The Hospital for Sick Children at 4 sites in 2 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 5 inclusion and 4 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 9 Nov 2016. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 4 sites in Brazil, Canada. 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?
Ultrasound findings according to the International Prophylaxis Study Group (IPSG) scale (time frame: Day 1). 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 9 Nov 2016.