HemophiliaNCT03914716
Imaging of Arthropathy in Boys With Hemophilia in China
A study testing Study group in people with Hemophilia, run by Andrea Doria at 1 site in China.
Trial summary
Hemophilia is a genetic condition characterized by marked phenotypic heterogeneity. Bleeding into a joint is the single most important risk factor for the development of hemophilic arthropathy (HA). Read more about this study
- Who is it for?
- Ages 4 to 11 years, men only
- Conditions the study is for
- Hemophilia
- What is being tested?
- Study group
- How many people
- 49 participants
- Where
- 1 siteChina
- Study dates
- 21 Mar 2018to 30 Nov 2023
- Phase
- Interventional
- 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 4 to 11 years
- Men only
- No healthy volunteers
- Child
Who may be able to join
- Hemophilia A with baseline FVIII levels of <2%
- Clinical history of ≥ 50 exposure days to FVIII prior to the study start.
- On-demand treatment, prophylaxis FVIII infusions or treatment with plasma-derived products for >3 months prior to enrollment into the study.
Who cannot join
- History of FVIII inhibitor (titer >0.6 Bethesda Units [BU])
- Chronic renal failure (serum creatinine >2.0 mg /dL).
- Chronic liver disease (alanine aminotransferase [ALT] >200 U/L).
- Clinically documented immunodeficiency.
- Anticipation of need for major surgery during the study period.
- Association of diseases known to mimic or cause joint diseases such as symptomatic human immunodeficiency virus (HIV) infection, juvenile idiopathic arthritis, and metabolic bone diseases.
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- Social barriers for participation in the study such as long distance between home and the comprehensive care centre, and documented track record of non-compliance to therapies or participation in clinical studies.
- Neuro-developmental/behavioral problems.
- Contraindications to MR imaging (presence of heart pacemakers, metallic foreign bodies in the eye, aneurysm clips, severe claustrophobia).
Trial locations and contacts
1 site in 1 country.
About this study
Official title: Radiogenomics: Personalized Imaging of Arthropathy in Boys With Hemophilia in China
Hemophilia is a genetic condition characterized by marked phenotypic heterogeneity. Bleeding into a joint is the single most important risk factor for the development of hemophilic arthropathy (HA). It is thought that clinical and imaging manifestations of HA are at least partially attributable to genetic polymorphisms unrelated to the hemophilia genotype. Identifying and characterizing biologic factors that could explain differences in susceptibility to joint degeneration of patients with hemophilia would help stratify patients according to the risk of degeneration of their joints and develop personalized therapeutic and prophylactic strategies. This study is conducted in China.
Read the detailed description
This will be a 3-year prospective cohort study conducted in a single centre (Beijing Children's Hospital, BCH, China) with a 2-year follow-up of patients Index joints (ankles, elbows and knees) of young Chinese boys with hemophilia A will be evaluated as follows: physical examination every 6 months using the Hemophilia Joint Health Score [HJHS], ultrasound imaging (gray-scale and color Doppler ultrasound [US]), and by laboratory (serum) at baseline, at 6, and 24 months. Magnetic resonance imaging (MRI) scans of index joints will be obtained at baseline, and 24 months. Features that will be captured either quantitatively or semantically in the imaging scans will be aggregated to generate "imaging phenotypes" which will be associated with clusters of co-expressed genes (metagenes) and clinical data.
- Conditions
- Hemophilia
- Being tested
- Study group
- Keywords
- Hemophilia, Radiogenomics, 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 groupDiagnostic test
Subjects will have physical, imaging examinations and provide samples for biological markers
What is being measured
The primary outcome is the main question the study is designed to answer.
Annualized total index joint bleeding rates (AJBRs)
AJBRs will be calculated from prospectively collected joint bleeding logs and clinic records.
Measured: Between baseline and 24 months
Secondary outcomes (8)
Number of participants with joint inflammation
Assessed by ultrasound
Measured: At baseline, 6 months and 24 months
Number of participants with joint inflammation
Assessed by MRI of the joints
Measured: Between baseline and 24 months
Number of participants with joint damage
Assessed by ultrasound
Measured: At baseline, 6 months and 24 months
Number of participants with joint damage
Assessed by MRI of the joints
Measured: Between baseline and 24 months
Number of participants with clinical arthropathy
Assessed by the Hemophilia Joint Health Score tool (HJHS), version 2.1 - Joint score range: 0 (normal) to 16 (worse outcome).
Measured: Every 6 months
Presence of inflammatory biomarkers in plasma
Measured by ELISA (enzyme-linked immunosorbent assay)
Measured: At baseline, 6 months and 24 months
Internal MRI-based soft tissue score change
Assessed by the 17-point International Prophylaxis Study Group (IPSG) MRI scale. Score range: 0 to 17 (worse outcome)
Measured: Between baseline and 24 months
Internal MRI-based osteochondral tissue score change
Assessed by the 17-point International Prophylaxis Study Group (IPSG) MRI scale. Score range: 0 to 17 (worse outcome)
Measured: Between baseline and 24 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted16 Apr 2019
- Study start21 Mar 2018
- Primary completion2 Feb 2022
- Study completion30 Nov 2023
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
- Interventional
- Phase
- Phase N/A
- Primary purpose
- Diagnosis
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 49 participants (actual)
Sponsor and investigators
- Lead sponsor
- Andrea Doria
- Sponsor type
- University, hospital or other
- Collaborators
- Beijing Children's Hospital, Baxalta US Inc.
- Responsible party
- Sponsor-investigator, Andrea Doria, The Hospital for Sick Children
- Investigators
- Andrea Doria, MD, Principal investigator, The Hospital for Sick Children
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03914716
- Sponsor's protocol ID
- 1000058500
- Status last verified
- Apr 2024
- First submitted
- 19 Feb 2019
- Last updated
- 19 Apr 2024
- 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 19 Apr 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Radiogenomics: Personalized Imaging of Arthropathy in Boys With Hemophilia in China
- ClinicalTrials.gov ID
- NCT03914716
- Acronym / sponsor ID
- 1000058500
- Conditions
- Hemophilia
- Keywords
- Hemophilia, Radiogenomics, Arthropathy
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 49 participants (actual)
- Lead sponsor
- Andrea DoriaUniversity, hospital or other
- Collaborators
- Beijing Children's Hospital, Baxalta US Inc.
- Study start
- 21 Mar 2018 (actual)
- Primary completion
- 2 Feb 2022 (actual)
- Study completion
- 30 Nov 2023 (actual)
- First posted
- 16 Apr 2019 (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.
Hemophilia is a genetic condition characterized by marked phenotypic heterogeneity. Bleeding into a joint is the single most important risk factor for the development of hemophilic arthropathy (HA). It is thought that clinical and imaging manifestations of HA are at least partially attributable to genetic polymorphisms unrelated to the hemophilia genotype. Identifying and characterizing biologic factors that could explain differences in susceptibility to joint degeneration of patients with hemophilia would help stratify patients according to the risk of degeneration of their joints and develop personalized therapeutic and prophylactic strategies. This study is conducted in China.
Detailed description
This will be a 3-year prospective cohort study conducted in a single centre (Beijing Children's Hospital, BCH, China) with a 2-year follow-up of patients Index joints (ankles, elbows and knees) of young Chinese boys with hemophilia A will be evaluated as follows: physical examination every 6 months using the Hemophilia Joint Health Score [HJHS], ultrasound imaging (gray-scale and color Doppler ultrasound [US]), and by laboratory (serum) at baseline, at 6, and 24 months. Magnetic resonance imaging (MRI) scans of index joints will be obtained at baseline, and 24 months. Features that will be captured either quantitatively or semantically in the imaging scans will be aggregated to generate "imaging phenotypes" which will be associated with clusters of co-expressed genes (metagenes) and clinical data.
Study design
- Primary purpose
- Diagnosis
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
Who may qualify
- Ages
- 4 to 11 years
- Sex
- Male only
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (3 listed)
- Hemophilia A with baseline FVIII levels of <2%
- Clinical history of ≥ 50 exposure days to FVIII prior to the study start.
- On-demand treatment, prophylaxis FVIII infusions or treatment with plasma-derived products for >3 months prior to enrollment into the study.
Key exclusion criteria (9 listed)
- History of FVIII inhibitor (titer >0.6 Bethesda Units [BU])
- Chronic renal failure (serum creatinine >2.0 mg /dL).
- Chronic liver disease (alanine aminotransferase [ALT] >200 U/L).
- Clinically documented immunodeficiency.
- Anticipation of need for major surgery during the study period.
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 |
|---|---|---|
| Study groupSubjects will have physical, imaging examinations and provide samples for biological markers | Diagnostic test | – |
Primary outcome
Annualized total index joint bleeding rates (AJBRs)
Time frame: Between baseline and 24 months
AJBRs will be calculated from prospectively collected joint bleeding logs and clinic records.
8 secondary outcomes
Number of participants with joint inflammation
Time frame: At baseline, 6 months and 24 months
Number of participants with joint inflammation
Time frame: Between baseline and 24 months
Number of participants with joint damage
Time frame: At baseline, 6 months and 24 months
Number of participants with joint damage
Time frame: Between baseline and 24 months
Number of participants with clinical arthropathy
Time frame: Every 6 months
Presence of inflammatory biomarkers in plasma
Time frame: At baseline, 6 months and 24 months
Internal MRI-based soft tissue score change
Time frame: Between baseline and 24 months
Internal MRI-based osteochondral tissue score change
Time frame: Between baseline and 24 months
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 19 Apr 2024.
- Individual participant dataSharing planned: No
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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
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A study testing Study group in people with Hemophilia, run by Andrea Doria at 1 site in China.
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 3 inclusion and 9 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 19 Apr 2024. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in China. 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?
Annualized total index joint bleeding rates (AJBRs) (time frame: Between baseline and 24 months). 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 19 Apr 2024.