DermatomyositisNCT00001265
Study and Treatment of Inflammatory Muscle Diseases
An observational study of Dermatomyositis and Polymyositis, run by National Institute of Arthritis and Musculoskeletal and Skin Diseases (NIAMS) at 1 site in United States.
Trial summary
This study of inflammatory muscle diseases-polymyositis and dermatomyositis and related disorders-will examine what causes these diseases and describe the clinical features (signs and symptoms) associated with them. Read more about this study
- Who is it for?
- Ages 16 years and olderHealthy volunteers can take part
- Conditions the study is for
- Dermatomyositis, Polymyositis
- How many people
- 1,325 participants
- Where
- 1 siteUnited States
- Study dates
- 10 Jan 1992to 5 Apr 2024
- 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 16 years and older
- All sexes
- Healthy volunteers accepted
- Child, Adult, Older adult
Study population: Healthy Volunteers and patients with known or suspected Idiopathic Inflammatory myopathies (IIM)
Who may be able to join
- All eligible patients are invited to participate in this protocol. Patients are adults and minors, aged 16 or higher, with possible idiopathic muscle disease (suspected or confirmed). Since both men and women are affected with the disease, both sexes will be studied. All ethnic and racial groups are at risk and will be included. Pregnant women and women who intend to become pregnant during the study will be eligible since this disease affects both segments of the population. This protocol proposes responsible inclusion of pregnant women and the justification for this derives in part from the fact that women (who are suffering from autoimmune and rheumatic diseases) lack data from which to make informed decisions about health care during pregnancy.
- The majority of patients with autoimmune disease are women and of these individuals, the majority are of childbearing age. Furthermore, pregnancy is a risk factor for fluctuations in autoimmune disease activity, including organ- and life- threatening flares. The majority of our study population is comprised of women. The exclusion of women who become pregnant from systemic study by way of our natural history protocol, threatens both the well-being of the individual women (owing to the standard of care provision of the protocol) and the systematic discovery of pregnancy-specific data concerning the immunopathological basis of autoimmune disease. Skewed understanding from a restricted natural history study may leave vulnerable groups open to disproportionate harm owing to inequities in research. In other words, automatic disqualification due to pregnancy denies women agency in the issue of gender equity for access to research and in addition the systematic exclusion of pregnant women prevents generalizability of information to help under-resourced and marginalized populations, nationwide.
- In summary,
- The cost of exclusion of pregnant women with myositis 1) insufficient evidence concerning how to manage such disease for the population, and 2) lack of access to the best myositis specific advice for individuals enrolled in the protocol who are unlikely to have access to specialty care outside of the NIH
- The cost of exclusion in research with regard to fetal safety derives from the fact that preeclampsia, eclampsia, preterm birth and miscarriage are higher in women who do not have access to subspecialty care. In this situation, reticence on the part of nonspecialists to treat active autoimmune disease during pregnancy or lack of access to appropriate treatment may lead to harm to women and their fetuses.
- The pregnant body possesses a unique hormonal and immunological environment. Similarly, it metabolizes medications in a manner different from non-pregnant women, let alone men. Hence, there is no reasonable path towards generating important biomedical knowledge other than to include pregnant women in this protocol.
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- The tension between protection by exclusion and the presumption of inclusion in research protocols has been specifically addressed by the US Institute of Medicine (now National Academy of Medicine) and the Task Force on Research Specific to Pregnant Women and Lactating Women to advise the Secretary of Health and Human Services regarding gaps in knowledge and research on safe and effective therapies for pregnant and lactating women, and both have both weighed in as favoring the presumption of inclusion (https://www.nichd.nih.gov/about/advisory/PRGLAC). Our study falls in line with these position papers since it is responsible (it benefits, patients and their fetuses and provides relevant data to the population at large) and because it is low risk (it is standard of care, only, and does not invoke experimental interventions).
- Healthy volunteers will be eligible only if they are adults aged 18 or older and only if they are willing to provide informed consent. There is no benefit to the healthy volunteers; however their inclusion will provide invaluable information in comparison to the patients for the purpose of research. Recruitment of healthy volunteers has been completed.
- Participation of NIH Staff or family members of study team members
- NIH staff and family members of study team members may be enrolled in this study as this population meets the study entry criteria. Neither participation nor refusal to participate as a subject in the research will have an effect, either beneficial or adverse, on the participant s employment or position at NIH.
- Every effort will be made to protect participant information, but such information may be available in medical records and may be available to authorized users outside of the study team in both an identifiable an unidentifiable manner.
- The NIH Information Sheet on NIH Staff Research Participation will be made available. Please see section for consent of NIH Staff.
Who cannot join
- Children (age less than 16 years) are excluded
- Subjects with severe active infection (such as requiring intravenous antibiotics) or other co-morbidities that in the opinion of the investigator would warrant exclusion.
- Subjects or their legal guardians who are unable or unwilling to give informed consent/assent.
Trial locations and contacts
1 site in 1 country.
United States1 site
National Institutes of Health Clinical Center
Bethesda, Maryland, 20892
About this study
Official title: Studies on the Natural History and Pathogenesis of Polymyositis, Dermatomyositis, and Related Diseases
This study of inflammatory muscle diseases-polymyositis and dermatomyositis and related disorders-will examine what causes these diseases and describe the clinical features (signs and symptoms) associated with them. Inflammation and degeneration of skeletal muscles in these disorders leads to weakness and muscle wasting. The skin, lungs and other organs may also be involved.
Patients 16 years of age and older with polymyositis, dermatomyositis, or a related disorder may be eligible for this study. Participants will undergo a complete history and physical examination, including routine blood and urine tests. Additional procedures for diagnosis, treatment or research may include:
Read more
- Blood sample for genetic studies.
- Muscle biopsy-removal of a tissue sample for microscopic examination. Under local anesthetic, a 1/2- to 1-inch long incision is made in the thigh or upper arm, and a small piece of muscle is removed.
- Electromyography-measurement of the electrical activity of a muscle. A needle is inserted through the skin into a muscle to record its electrical activity.
- Magnetic resonance imaging-visualization of organs or tissues, using a magnetic field and radio waves. The patient lies on a table inside a narrow cylinder (the MRI scanner) with a strong magnetic field for the scanning.
- Manual muscle strength testing by a physiotherapist.
- Swallowing studies using ultrasound (imaging using sound waves) and X-rays (barium swallow) to evaluate swallowing and speaking abilities.
- Questionnaires on swallowing ability and ability to perform daily living activities
- Pulmonary function tests-measurement of movement of air in and out of the lungs. The patient breathes into a machine to evaluate lung function.
- Chest X-rays to evaluate lung function.
- Electrocardiogram and, if necessary, Holter monitoring (measurement of the electrical activity of the heart) and echocardiogram (ultrasound imaging of the heart) to evaluate heart function.
- Apheresis-collection of white blood cells for research. Whole blood is collected through a needle placed in an arm vein. The blood circulates through a machine that separates it into its components. The white cells are removed and the rest of the blood is returned to the body through the same needle or through a second one placed in the other arm.
- MR guided muscle biopsy-measurement of glycogen in muscle tissue using magnetic resonance imaging. Certain patients may undergo this experimental procedure to compare MRI findings with those of muscle biopsy. The affected muscles are identified using MRI and the biopsy incision is made. MRI is then used to guide the biopsy needle to the muscle and a small piece is removed.
Patients who are eligible for experimental treatment studies will be offered the opportunity to join them. Others will be advised of treatment recommendations.
Read the detailed description
Polymyositis, dermatomyositis and related disorders, also known as the idiopathic inflammatory myopathies (IIM), are an uncommon, heterogeneous group of diseases. This is an omnibus protocol designed to continue our description of this rare group of diseases, further delineate important groups of patients, and obtain useful material for further study of immunological abnormalities, pathogenesis and etiology of IIM. Often the diagnosis of an IIM can be confused with other illness (such as adult-onset dystrophies), and therefore, we will be evaluating patients with other illnesses (who are referred with a preliminary diagnosis of an IIM) (1). Patients evaluated with IIM will receive (with informed consent) the best standard therapies available and consideration for entry into any therapeutic protocols. MRI will be performed on healthy volunteers to help develop a mathematical method for quantitating edema of muscle MRI in patients (2). MRI/PET will be performed on some patients and on healthy volunteers to evaluate its role in understanding muscle disease.
- Conditions
- Dermatomyositis, Polymyositis
- Keywords
- Autoimmune Diseases, Idiopathic Inflammatory Myopathies, Dermatomyositis, Inclusion Body Myositis, Pathogenesis, Natural History, Connective Tissue Diseases
- Medical subject headings
- Dermatomyositis, Polymyositis, Autoimmune Diseases, Myositis, Myositis, Inclusion Body, Connective Tissue 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 |
|---|---|---|
| Healthy Volunteers | Patients with no disease | |
| Patients with muscle disease | Patients with known or suspected Idiopathic Inflammatory myopathies (IIM) |
What is being measured
The primary outcome is the main question the study is designed to answer.
Muscle strength
To determine its utility in differentiating different muscle diseases, in identifying early extra muscular involvement of muscle diseases, in characterizing the pattern of muscle involvement for the diseases studied, and whether it can serve as a diagnostic tool to better identify muscle inflammation and atrophy.
Measured: End of Study
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted4 Nov 1999 (estimated)
- Study start10 Jan 1992
- Primary completion5 Apr 2024
- Study completion5 Apr 2024
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for DermatomyositisStudies 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
- Prospective
- Enrolment
- 1,325 participants (actual)
Sponsor and investigators
- Lead sponsor
- National Institute of Arthritis and Musculoskeletal and Skin Diseases (NIAMS)
- Sponsor type
- US National Institutes of Health
- Responsible party
- Sponsor
- Investigators
- Andrew L Mammen, M.D., Principal investigator, National Institute of Arthritis and Musculoskeletal and Skin Diseases (NIAMS)
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00001265
- Sponsor's protocol ID
- 910196
- Other IDs
- 91-AR-0196
- Status last verified
- Apr 2024
- First submitted
- 3 Nov 1999
- Last updated
- 17 Apr 2024
- Results posted
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- Undecided: .At this time, It is not yet known if there will be a plan to make IPD available . The protocol is silent about IPD sharing.
Study at a glance
From the registry record, last updated by the sponsor on 17 Apr 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Studies on the Natural History and Pathogenesis of Polymyositis, Dermatomyositis, and Related Diseases
- ClinicalTrials.gov ID
- NCT00001265
- Acronym / sponsor ID
- 910196
- Conditions
- Dermatomyositis, Polymyositis
- Keywords
- Autoimmune Diseases, Idiopathic Inflammatory Myopathies, Dermatomyositis, Inclusion Body Myositis, Pathogenesis, Natural History, Connective Tissue Diseases
- Study type
- Observational
- Recruitment status
- Completed
- Enrolment
- 1,325 participants (actual)
- Lead sponsor
- National Institute of Arthritis and Musculoskeletal and Skin Diseases (NIAMS)US National Institutes of Health
- Study start
- 10 Jan 1992 (actual)
- Primary completion
- 5 Apr 2024 (actual)
- Study completion
- 5 Apr 2024 (actual)
- First posted
- 4 Nov 1999 (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.
This study of inflammatory muscle diseases-polymyositis and dermatomyositis and related disorders-will examine what causes these diseases and describe the clinical features (signs and symptoms) associated with them. Inflammation and degeneration of skeletal muscles in these disorders leads to weakness and muscle wasting. The skin, lungs and other organs may also be involved.
Patients 16 years of age and older with polymyositis, dermatomyositis, or a related disorder may be eligible for this study. Participants will undergo a complete history and physical examination, including routine blood and urine tests. Additional procedures for diagnosis, treatment or research may include:
- Blood sample for genetic studies.
- Muscle biopsy-removal of a tissue sample for microscopic examination. Under local anesthetic, a 1/2- to 1-inch long incision is made in the thigh or upper arm, and a small piece of muscle is removed.
- Electromyography-measurement of the electrical activity of a muscle. A needle is inserted through the skin into a muscle to record its electrical activity.
- Magnetic resonance imaging-visualization of organs or tissues, using a magnetic field and radio waves. The patient lies on a table inside a narrow cylinder (the MRI scanner) with a strong magnetic field for the scanning.
- Manual muscle strength testing by a physiotherapist.
- Swallowing studies using ultrasound (imaging using sound waves) and X-rays (barium swallow) to evaluate swallowing and speaking abilities.
- Questionnaires on swallowing ability and ability to perform daily living activities
- Pulmonary function tests-measurement of movement of air in and out of the lungs. The patient breathes into a machine to evaluate lung function.
- Chest X-rays to evaluate lung function.
- Electrocardiogram and, if necessary, Holter monitoring (measurement of the electrical activity of the heart) and echocardiogram (ultrasound imaging of the heart) to evaluate heart function.
- Apheresis-collection of white blood cells for research. Whole blood is collected through a needle placed in an arm vein. The blood circulates through a machine that separates it into its components. The white cells are removed and the rest of the blood is returned to the body through the same needle or through a second one placed in the other arm.
- MR guided muscle biopsy-measurement of glycogen in muscle tissue using magnetic resonance imaging. Certain patients may undergo this experimental procedure to compare MRI findings with those of muscle biopsy. The affected muscles are identified using MRI and the biopsy incision is made. MRI is then used to guide the biopsy needle to the muscle and a small piece is removed.
Patients who are eligible for experimental treatment studies will be offered the opportunity to join them. Others will be advised of treatment recommendations.
Detailed description
Polymyositis, dermatomyositis and related disorders, also known as the idiopathic inflammatory myopathies (IIM), are an uncommon, heterogeneous group of diseases. This is an omnibus protocol designed to continue our description of this rare group of diseases, further delineate important groups of patients, and obtain useful material for further study of immunological abnormalities, pathogenesis and etiology of IIM. Often the diagnosis of an IIM can be confused with other illness (such as adult-onset dystrophies), and therefore, we will be evaluating patients with other illnesses (who are referred with a preliminary diagnosis of an IIM) (1). Patients evaluated with IIM will receive (with informed consent) the best standard therapies available and consideration for entry into any therapeutic protocols. MRI will be performed on healthy volunteers to help develop a mathematical method for quantitating edema of muscle MRI in patients (2). MRI/PET will be performed on some patients and on healthy volunteers to evaluate its role in understanding muscle disease.
Study design
- Observational model
- Cohort
- Time perspective
- Prospective
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Healthy Volunteers | – | –Patients with no disease |
| Patients with muscle disease | – | –Patients with known or suspected Idiopathic Inflammatory myopathies (IIM) |
Who may qualify
- Ages
- 16 years and older
- Sex
- All
- Healthy volunteers
- Accepted
- Age groups
- Child, Adult, Older adult
- Study population
- Healthy Volunteers and patients with known or suspected Idiopathic Inflammatory myopathies (IIM)
Key inclusion criteria (12 listed)
- All eligible patients are invited to participate in this protocol. Patients are adults and minors, aged 16 or higher, with possible idiopathic muscle disease (suspected or confirmed). Since both men and women are affected with the disease, both sexes will be studied. All ethnic and racial groups are at risk and will be included. Pregnant women and women who intend to become pregnant during the study will be eligible since this disease affects both segments of the population. This protocol proposes responsible inclusion of pregnant women and the justification for this derives in part from the fact that women (who are suffering from autoimmune and rheumatic diseases) lack data from which to make informed decisions about health care during pregnancy.
- The majority of patients with autoimmune disease are women and of these individuals, the majority are of childbearing age. Furthermore, pregnancy is a risk factor for fluctuations in autoimmune disease activity, including organ- and life- threatening flares. The majority of our study population is comprised of women. The exclusion of women who become pregnant from systemic study by way of our natural history protocol, threatens both the well-being of the individual women (owing to the standard of care provision of the protocol) and the systematic discovery of pregnancy-specific data concerning the immunopathological basis of autoimmune disease. Skewed understanding from a restricted natural history study may leave vulnerable groups open to disproportionate harm owing to inequities in research. In other words, automatic disqualification due to pregnancy denies women agency in the issue of gender equity for access to research and in addition the systematic exclusion of pregnant women prevents generalizability of information to help under-resourced and marginalized populations, nationwide.
- In summary,
- The cost of exclusion of pregnant women with myositis 1) insufficient evidence concerning how to manage such disease for the population, and 2) lack of access to the best myositis specific advice for individuals enrolled in the protocol who are unlikely to have access to specialty care outside of the NIH
- The cost of exclusion in research with regard to fetal safety derives from the fact that preeclampsia, eclampsia, preterm birth and miscarriage are higher in women who do not have access to subspecialty care. In this situation, reticence on the part of nonspecialists to treat active autoimmune disease during pregnancy or lack of access to appropriate treatment may lead to harm to women and their fetuses.
Key exclusion criteria (3 listed)
- Children (age less than 16 years) are excluded
- Subjects with severe active infection (such as requiring intravenous antibiotics) or other co-morbidities that in the opinion of the investigator would warrant exclusion.
- Subjects or their legal guardians who are unable or unwilling to give informed consent/assent.
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
Muscle strength
Time frame: End of Study
To determine its utility in differentiating different muscle diseases, in identifying early extra muscular involvement of muscle diseases, in characterizing the pattern of muscle involvement for the diseases studied, and whether it can serve as a diagnostic tool to better identify muscle inflammation and atrophy.
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 17 Apr 2024.
- NIH Clinical Center Detailed Web PageLink provided by the sponsor.
- Individual participant dataSharing planned: Undecided. .At this time, It is not yet known if there will be a plan to make IPD available . The protocol is silent about IPD sharing.
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Dermatomyositis
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.
Andrew L Mammen, M.D. principal investigator
National Institute of Arthritis and Musculoskeletal and Skin Diseases (NIAMS)
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 Dermatomyositis and Polymyositis, run by National Institute of Arthritis and Musculoskeletal and Skin Diseases (NIAMS) at 1 site in United States.
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 12 inclusion and 3 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 17 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 United States. 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?
Muscle strength (time frame: End of Study). 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 17 Apr 2024.