MyositisUKMYONETNCT01171573
Investigating Genes in Patients With Polymyositis and Dermatomyositis
An observational study of Myositis, run by Northern Care Alliance NHS Foundation Trust at 1 site in United Kingdom.
Trial summary
Polymyositis (PM), dermatomyositis (DM) and inclusion body myositis (IBM)belong to a group of inflammatory muscle disorders, of unknown cause, that are characterised by skeletal muscle inflammation and progressive muscular weakness, which can be debilitating and chronic in nature (occasionally fatal). Read more about this study
- Who is it for?
- Ages 18 to 70 yearsHealthy volunteers can take part
- Conditions the study is for
- Myositis
- What is being tested?
- Venepuncture
- Type of treatment
- Surgery or procedure
- How many people
- 1,000 participants
- Where
- 1 siteUnited Kingdom
- Study dates
- Jan 2001to Dec 2030
- Phase
- Observational
- Are there results?
- Not yet: the study is under way
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 18 to 70 years
- All sexes
- Healthy volunteers accepted
- Adult, Older adult
Study population: Potential participants will be identified at their routine attendance of myositis clinics at participating centres, by the study PI. Before recruitment, each prospective candidate will be given a full explanation of the study, provided with a patient information sheet (PIS) and consent form to read, and given the opportunity to ask any questions that may arise. Once all questions have been answered and the Principal Investigator is assured that the individual understands what is required, informed consent can then be sought.
Who may be able to join
- Cases must fulfil Bohan and Peter Diagnostic Criteria for Adult PM/DM
- Symmetrical weakness of limb-girdle muscles and/or anterior neck flexors.
- Muscle biopsy evidence typical of myositis.
- Elevation of serum skeletal muscle enzymes.
- Typical EMG features of myositis.
- Typical DM rash, including:
- Probable / definite PM: at least 3 of items 1-4 +ive. Probable / definite DM: item 5 \& at least 2 of items 1-4 +ive.
Who cannot join
- Patients below the age of 18 years
- Patients with myositis secondary to alcohol or drug abuse, non abusive drug ingestion (e.g with statins, fibrates etc)
- Patients with myositis following a recent viral illnesses.
- Patients unable to consent for themselves due to diminished mental capacity
- Patients that can not speak sufficiently good English.
- Patients unwilling to give blood sample.
Trial locations and contacts
1 site in 1 country.
United Kingdom1 site
Salford Royal NHS Foundation Trust
Manchester, M6 8HD
About this study
Official title: Identification of Disease Susceptibility Genes Associated With Development and Clinical Characteristics of Primary Inflammatory Muscle Diseases, PM, DM and IBM.
Polymyositis (PM), dermatomyositis (DM) and inclusion body myositis (IBM)belong to a group of inflammatory muscle disorders, of unknown cause, that are characterised by skeletal muscle inflammation and progressive muscular weakness, which can be debilitating and chronic in nature (occasionally fatal).
The current treatment options for these conditions are steroids and various other immunosuppressive drugs. However, these are usually only partially effective at reducing symptoms, and their toxic side effects also limit their usefulness. In order to develop more specific treatments for myositis in the future (and therefore more effective), it is important to understand the exact mechanisms that cause the disease in the first instance. In other similar inflammatory diseases such as rheumatoid arthritis (RA) and systemic lupus (SLE), it is known that changes to the Human Leukocyte Antigen(HLA), as well as certain inflammatory cytokines, are involved in both the development and expression of the disease.
Read more
As many of the inflammatory mechanisms that cause damage in PM, DM and IBM are similar to those in RA and SLE, it seems likely that similar genetic factors will also be involved in the development and expression of PM, DM and IBM.
In order to understand the genetic aspects / causes of myositis, and ultimately develop more effective treatment therapies in the future, patients with PM, DM or IBM, will be asked to give 20 mls of blood. These blood samples, along with the patient's clinical details, will then be sent to the Centre for Integrated Genomic Medical Research (CIGMR), at The University of Manchester, where all of the genetic analyses will take place. By understanding the genetic cause of the disease, it should be possible to design specific drugs for treating the condition in the future.
Read the detailed description
In order to understand the genetic aspects / causes of myositis, and ultimately develop more effective treatment therapies, it will be necessary to collect blood samples and clinical details from around 1000 patients with myositis. As the condition is very rare, it will not be possible to get the required number of patients from one centre alone, and therefore, a collaborative, multicentred approach will be needed. Consultant rheumatologists and neurologists throughout the UK, who have patients with PM, DM or IBM, will be approached, and asked if they would like to officially enter into the myositis genomic multicentred study. Once ethical and R\&D approval has been granted, consultants will be named as the 'Principal Investigators' at each of these 'research sites' and could then start to recruit suitable patients into the study.
Patients from each research site, with definite PM, DM or IBM, will be asked by their own consultants (Principal Investigator) to give 20 mls of blood via venepuncture for genetic and antibody analysis. The PI will also complete a clinical/laboratory proforma, for each patient, regarding their disease characteristics, to allow subsequent confirmation that patients' disease is indeed definite, and this proforma and the blood sample will be sent to CIGMR. All of the genetic analyses, will take place at CIGMR, but storage of clinical details and certain patient identifiers will be stored at SRFT on a password protected NHS tust computer.
This study will be co-ordinated by Dr RG Cooper through the Rheumatic Diseases Centre, Salford Royal NHS Foundation Trust (SRFT), and Professor Bill Ollier from the Centre for Integrated Genomic Medical Research unit (CIGMR), Stopford Building, Manchester University.
- Conditions
- Myositis
- Being tested
- Venepuncture
- Keywords
- Polymyositis, Dermatomyositis, Inclusion Body Myositis
- Medical subject headings
- Myositis, Polymyositis, Dermatomyositis, Myositis, Inclusion Body
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
VenepunctureProcedure or surgery
Venepuncture - Taking blood
| Study group | Role | What they receive |
|---|---|---|
| Myositis Patients | Cases with myositis PM DM IBM Venepuncture Receives: Procedure: Venepuncture | |
| Healthy controls | Control Receives: Procedure: Venepuncture |
What is being measured
The primary outcome is the main question the study is designed to answer.
To identify any disease susceptibility genes associated with development and clinical characteristics of primary inflammatory muscle diseases, PM, DM and IBM.
Blood sample, DNA analysis, along with clinical data, matching genotype with phenotype
Measured: 20 years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted28 Jul 2010 (estimated)
- Study startJan 2001
- Primary completionDec 2030 (estimated)
- Study completionDec 2030 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for MyositisStudies 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
- Case-control
- Time perspective
- Prospective
- Enrolment
- 1,000 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Northern Care Alliance NHS Foundation Trust
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Hector Chinoy, University of Manchester
- Investigators
- Robert Dr Cooper, Principal investigator, SRFT
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01171573
- Sponsor's protocol ID
- Ollier-002
- Acronym
- UKMYONET
- Status last verified
- Apr 2023
- First submitted
- 27 Jul 2010
- Last updated
- 5 Apr 2023
- Results posted
- No
- Data monitoring committee
- Yes
Study at a glance
From the registry record, last updated by the sponsor on 5 Apr 2023. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Identification of Disease Susceptibility Genes Associated With Development and Clinical Characteristics of Primary Inflammatory Muscle Diseases, PM, DM and IBM.
- ClinicalTrials.gov ID
- NCT01171573
- Acronym / sponsor ID
- UKMYONET / Ollier-002
- Conditions
- Myositis
- Keywords
- Polymyositis, Dermatomyositis, Inclusion Body Myositis
- Study type
- Observational
- Recruitment status
- Active, not recruiting
- Enrolment
- 1,000 participants (estimated)
- Lead sponsor
- Northern Care Alliance NHS Foundation TrustUniversity, hospital or other
- Study start
- Jan 2001
- Primary completion
- Dec 2030 (estimated)
- Study completion
- Dec 2030 (estimated)
- First posted
- 28 Jul 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.
Polymyositis (PM), dermatomyositis (DM) and inclusion body myositis (IBM)belong to a group of inflammatory muscle disorders, of unknown cause, that are characterised by skeletal muscle inflammation and progressive muscular weakness, which can be debilitating and chronic in nature (occasionally fatal).
The current treatment options for these conditions are steroids and various other immunosuppressive drugs. However, these are usually only partially effective at reducing symptoms, and their toxic side effects also limit their usefulness. In order to develop more specific treatments for myositis in the future (and therefore more effective), it is important to understand the exact mechanisms that cause the disease in the first instance. In other similar inflammatory diseases such as rheumatoid arthritis (RA) and systemic lupus (SLE), it is known that changes to the Human Leukocyte Antigen(HLA), as well as certain inflammatory cytokines, are involved in both the development and expression of the disease.
As many of the inflammatory mechanisms that cause damage in PM, DM and IBM are similar to those in RA and SLE, it seems likely that similar genetic factors will also be involved in the development and expression of PM, DM and IBM.
In order to understand the genetic aspects / causes of myositis, and ultimately develop more effective treatment therapies in the future, patients with PM, DM or IBM, will be asked to give 20 mls of blood. These blood samples, along with the patient's clinical details, will then be sent to the Centre for Integrated Genomic Medical Research (CIGMR), at The University of Manchester, where all of the genetic analyses will take place. By understanding the genetic cause of the disease, it should be possible to design specific drugs for treating the condition in the future.
Detailed description
In order to understand the genetic aspects / causes of myositis, and ultimately develop more effective treatment therapies, it will be necessary to collect blood samples and clinical details from around 1000 patients with myositis. As the condition is very rare, it will not be possible to get the required number of patients from one centre alone, and therefore, a collaborative, multicentred approach will be needed. Consultant rheumatologists and neurologists throughout the UK, who have patients with PM, DM or IBM, will be approached, and asked if they would like to officially enter into the myositis genomic multicentred study. Once ethical and R\&D approval has been granted, consultants will be named as the 'Principal Investigators' at each of these 'research sites' and could then start to recruit suitable patients into the study.
Patients from each research site, with definite PM, DM or IBM, will be asked by their own consultants (Principal Investigator) to give 20 mls of blood via venepuncture for genetic and antibody analysis. The PI will also complete a clinical/laboratory proforma, for each patient, regarding their disease characteristics, to allow subsequent confirmation that patients' disease is indeed definite, and this proforma and the blood sample will be sent to CIGMR. All of the genetic analyses, will take place at CIGMR, but storage of clinical details and certain patient identifiers will be stored at SRFT on a password protected NHS tust computer.
This study will be co-ordinated by Dr RG Cooper through the Rheumatic Diseases Centre, Salford Royal NHS Foundation Trust (SRFT), and Professor Bill Ollier from the Centre for Integrated Genomic Medical Research unit (CIGMR), Stopford Building, Manchester University.
Study design
- Observational model
- Case-control
- Time perspective
- Prospective
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Myositis Patients | – | Procedure: VenepunctureCases with myositis PM DM IBM Venepuncture |
| Healthy controls | – | Procedure: VenepunctureControl |
Who may qualify
- Ages
- 18 to 70 years
- Sex
- All
- Healthy volunteers
- Accepted
- Age groups
- Adult, Older adult
- Study population
- Potential participants will be identified at their routine attendance of myositis clinics at participating centres, by the study PI. Before recruitment, each prospective candidate will be given a full explanation of the study, provided with a patient information sheet (PIS) and consent form to read, and given the opportunity to ask any questions that may arise. Once all questions have been answered and the Principal Investigator is assured that the individual understands what is required, informed consent can then be sought.
Key inclusion criteria (7 listed)
- Cases must fulfil Bohan and Peter Diagnostic Criteria for Adult PM/DM
- Symmetrical weakness of limb-girdle muscles and/or anterior neck flexors.
- Muscle biopsy evidence typical of myositis.
- Elevation of serum skeletal muscle enzymes.
- Typical EMG features of myositis.
Key exclusion criteria (6 listed)
- Patients below the age of 18 years
- Patients with myositis secondary to alcohol or drug abuse, non abusive drug ingestion (e.g with statins, fibrates etc)
- Patients with myositis following a recent viral illnesses.
- Patients unable to consent for themselves due to diminished mental capacity
- Patients that can not speak sufficiently good English.
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 |
|---|---|---|
| VenepunctureVenepuncture - Taking blood | Procedure or surgery | Healthy controls, Myositis Patients |
Primary outcome
To identify any disease susceptibility genes associated with development and clinical characteristics of primary inflammatory muscle diseases, PM, DM and IBM.
Time frame: 20 years
Blood sample, DNA analysis, along with clinical data, matching genotype with phenotype
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 5 Apr 2023.
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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.
Robert Dr Cooper principal investigator
SRFT
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Myositis, run by Northern Care Alliance NHS Foundation Trust at 1 site in United Kingdom.
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 7 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: under way, not looking for participants, as of the sponsor's last update on 5 Apr 2023. 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 United Kingdom. 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?
This study is still under way. Results are not available in the source record yet.
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?
To identify any disease susceptibility genes associated with development and clinical characteristics of primary inflammatory muscle diseases, PM, DM and IBM. (time frame: 20 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 5 Apr 2023.