DermatomyositisPhase 2NCT00001421
Methimazole to Treat Polymyositis and Dermatomyositis
A Phase 2 study testing methimazole in people with 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 will test the safety and effectiveness of the drug methimazole in treating polymyositis and dermatomyositis-inflammatory muscle diseases causing weakness and muscle wasting. Although it is not known what causes of these diseases, abnormal immune function is thought to be involved. Read more about this study
- Who is it for?
- Any age
- Conditions the study is for
- Dermatomyositis, Polymyositis
- What is being tested?
- methimazole
- How many people
- 20 participants
- Where
- 1 siteUnited States
- Study dates
- Jun 1995to Apr 2001
- Phase
- Phase 2
- 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 Any age
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Diagnosis of Polymyositis or Dermatomyositis.
Baseline muscle weakness score of less than or equal to 139 out of 160 on manual testing (MMT).
Baseline functional assessment score of less than or equal to 82 out of 91.
Ability to provide informed consent to all aspects of the study after full information is provided.
Age equal to or older than 18.
A diagnosis of classic or definite polymyositis or dermatomyositis (Critieria A and B plus at least one of the three other criteria):
- Symmetrical proximal muscle weakness;
- Muscle biopsy abnormalities at some time during their disease:
- degeneration and regeneration of muscle fibers ii. necrosis iii. phagocytosis iv. interstitial mononuclear infiltration;
- Elevation of serum creatinine phosphokinase (CPK), transaminases, lactic dehydrogenase (LDH) or aldolase activity;
- Electromyography (EMG) triad of changes
- small amplitude, short duration polyphasic motor unit potentials ii. fibrillations, positive sharp waves, increased insertional irritability iii. spontaneous bizarre high frequency discharges;
- Typical skin rash of DM.
Willingness to undergo 2 muscle biopsies.
Evidence of active disease as measured by weakness, and an elevated CK or an active MRI.
Must be tapered to a stable dose of steroid equal to or less than 0.50 mg/kg/day equivalent of prednisone for one month prior to the study.
If on additional immunosuppressive drugs, the drugs must be maintained at a stable dose for 1 month prior to the initiation of therapy and will be maintained throughout the trial.
Women of childbearing potential and men whose partners are of childbearing potential must practice an acceptable form of contraception. No pregnant females or nursing mothers.
No history of hepatitis or abnormal liver function tests.
No history of prior thyroid disease.
No active acute or chronic infections requiring antimicrobial therapy, or serious viral or fungal infections.
No preexisting or coexisting malignancy other than basal cell and localized squamous cell carcinoma of the skin.
No history of cerebrovascular accidents, seizure disorder, aseptic meningitis, transverse myelitis or central nervous system disease.
No history of documented coronary artery disease, cardiomyopathy, greater than first-degree heart block, or dysrhythmia requiring therapy.
No confounding medical illness that in the judgement of the investigators would pose added risk for study participants.
No anemia requiring maintenance blood transfusions; leukoplakia with WBC less than 3,000 micrograms or absolute neutrophil count less than 2,000 micrograms; and platelet count less than 100,000 micrograms on at least two different occasions.
No history of (or current) autoimmune hemolytic anemia.
No current anticoagulant therapy.
Trial locations and contacts
1 site in 1 country.
United States1 site
National Institute of Arthritis and Musculoskeletal and Skin Diseases (NIAMS)
Bethesda, Maryland, 20892
About this study
Official title: A Pilot Study of the Role of Methimazole in Patients With Polymyositis and Dermatomyositis
This study will test the safety and effectiveness of the drug methimazole in treating polymyositis and dermatomyositis-inflammatory muscle diseases causing weakness and muscle wasting. Although it is not known what causes of these diseases, abnormal immune function is thought to be involved. Recent studies indicate that methimazole, which has been used for many years to treat thyroid disease, may alter immune activity by affecting the interaction between white blood cells called lymphocytes and certain molecules on cell surfaces. This study will examine the effects of methimazole on immune activity and muscle strength in patients with inflammatory muscle diseases and evaluate the drug side effects.
Patients with polymyositis and dermatomyositis who have normal thyroid function may be eligible for this study [age requirement?]. Candidates will undergo a history and physical examination; blood and urine tests; chest X-ray; muscle strength testing, daily living skills questionnaire, and speech and swallowing evaluation; magnetic resonance imaging of muscles; and muscle biopsy (removal of a small piece of muscle tissue under local anesthetic). When indicated, some candidates may also have cancer screening tests (for example, mammogram, Pap smear), a lung function test to measure breathing capacity, or an electromyogram, in which small needles are inserted into a muscle to measure the electrical activity .
Participants will take 30 mg of methimazole by mouth twice a day for 6 months. They will have blood tests weekly for the first 2 weeks and then every other week for the rest of the study to measure blood counts and liver and thyroid function. Blood will also be drawn for white blood cell studies during the screening evaluation, at the beginning of therapy, 6 to 12 weeks after therapy starts, at the end of the 6-month treatment period, and 1 and 3 months after therapy ends. Muscle enzyme and urine tests will be done once a month.. During drug treatment, patients will have periodic physical examinations and blood and muscle function tests to evaluate the response to therapy.
Read the detailed description
This open label pilot study will assess the (1) activity of methimazole (MMI), a down-regulator of MHC Class I transcription used in treatment of autoimmune thyroiditis (AIT), on the tissue expression of HLA class I and its (2) efficacy as measured by serum muscle enzyme levels and manual muscle testing in up to twenty patients with dermatomyositis (DM) or polymyositis (PM). Participants will have persistent weakness, laboratory evidence of inflammation and be on a stable regimen of medication to control their myopathy. MMI will be administered orally at 30 mg twice a day for six months. Patients will be evaluated for alteration of HLA Class I expression in muscle and peripheral blood lymphocytes (PBL) and drug-related toxicities during the study and for three months after the discontinuation of treatment.
- Conditions
- Dermatomyositis, Polymyositis
- Being tested
- methimazole
- Keywords
- Cytotoxicity, HLA Class I, Lymphocytes, Muscle, Myositis, Thionamides, Dermatomyositis, Polymyositis
- Medical subject headings
- Dermatomyositis, Polymyositis, Myositis
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
methimazoleDrug
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted10 Dec 2002 (estimated)
- Study startJun 1995
- Study completionApr 2001
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (3)Papers the sponsor has linked to this study.
- 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
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Enrolment
- 20 participants
Sponsor and investigators
- Lead sponsor
- National Institute of Arthritis and Musculoskeletal and Skin Diseases (NIAMS)
- Sponsor type
- US National Institutes of Health
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00001421
- Sponsor's protocol ID
- 950139
- Other IDs
- 95-AR-0139
- Status last verified
- May 2000
- First submitted
- 3 Nov 1999
- Last updated
- 4 Mar 2008
- Results posted
- No
Publications
Miller JF, Morahan G. Peripheral T cell tolerance. Annu Rev Immunol. 1992;10:51-69. doi: 10.1146/annurev.iy.10.040192.000411.
PubMed 1590995Hammerling GJ, Schonrich G, Momburg F, Auphan N, Malissen M, Malissen B, Schmitt-Verhulst AM, Arnold B. Non-deletional mechanisms of peripheral and central tolerance: studies with transgenic mice with tissue-specific expression of a foreign MHC class I antigen. Immunol Rev. 1991 Aug;122:47-67. doi: 10.1111/j.1600-065x.1991.tb00596.x.
PubMed 1834543Ploegh HL, Orr HT, Strominger JL. Major histocompatibility antigens: the human (HLA-A, -B, -C) and murine (H-2K, H-2D) class I molecules. Cell. 1981 May;24(2):287-99. doi: 10.1016/0092-8674(81)90318-4. No abstract available.
PubMed 7016338
Study at a glance
From the registry record, last updated by the sponsor on 4 Mar 2008. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Pilot Study of the Role of Methimazole in Patients With Polymyositis and Dermatomyositis
- ClinicalTrials.gov ID
- NCT00001421
- Acronym / sponsor ID
- 950139
- Conditions
- Dermatomyositis, Polymyositis
- Keywords
- Cytotoxicity, HLA Class I, Lymphocytes, Muscle, Myositis, Thionamides, Dermatomyositis, Polymyositis
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 20 participants
- Lead sponsor
- National Institute of Arthritis and Musculoskeletal and Skin Diseases (NIAMS)US National Institutes of Health
- Study start
- Jun 1995
- Study completion
- Apr 2001
- First posted
- 10 Dec 2002 (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 will test the safety and effectiveness of the drug methimazole in treating polymyositis and dermatomyositis-inflammatory muscle diseases causing weakness and muscle wasting. Although it is not known what causes of these diseases, abnormal immune function is thought to be involved. Recent studies indicate that methimazole, which has been used for many years to treat thyroid disease, may alter immune activity by affecting the interaction between white blood cells called lymphocytes and certain molecules on cell surfaces. This study will examine the effects of methimazole on immune activity and muscle strength in patients with inflammatory muscle diseases and evaluate the drug side effects.
Patients with polymyositis and dermatomyositis who have normal thyroid function may be eligible for this study [age requirement?]. Candidates will undergo a history and physical examination; blood and urine tests; chest X-ray; muscle strength testing, daily living skills questionnaire, and speech and swallowing evaluation; magnetic resonance imaging of muscles; and muscle biopsy (removal of a small piece of muscle tissue under local anesthetic). When indicated, some candidates may also have cancer screening tests (for example, mammogram, Pap smear), a lung function test to measure breathing capacity, or an electromyogram, in which small needles are inserted into a muscle to measure the electrical activity .
Participants will take 30 mg of methimazole by mouth twice a day for 6 months. They will have blood tests weekly for the first 2 weeks and then every other week for the rest of the study to measure blood counts and liver and thyroid function. Blood will also be drawn for white blood cell studies during the screening evaluation, at the beginning of therapy, 6 to 12 weeks after therapy starts, at the end of the 6-month treatment period, and 1 and 3 months after therapy ends. Muscle enzyme and urine tests will be done once a month.. During drug treatment, patients will have periodic physical examinations and blood and muscle function tests to evaluate the response to therapy.
Detailed description
This open label pilot study will assess the (1) activity of methimazole (MMI), a down-regulator of MHC Class I transcription used in treatment of autoimmune thyroiditis (AIT), on the tissue expression of HLA class I and its (2) efficacy as measured by serum muscle enzyme levels and manual muscle testing in up to twenty patients with dermatomyositis (DM) or polymyositis (PM). Participants will have persistent weakness, laboratory evidence of inflammation and be on a stable regimen of medication to control their myopathy. MMI will be administered orally at 30 mg twice a day for six months. Patients will be evaluated for alteration of HLA Class I expression in muscle and peripheral blood lymphocytes (PBL) and drug-related toxicities during the study and for three months after the discontinuation of treatment.
Study design
- Primary purpose
- Treatment
Who may qualify
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
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 |
|---|---|---|
| methimazole | Drug | – |
Primary outcome
Not reported in the source record.
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 4 Mar 2008.
- Background: PubMed 1590995Miller JF, Morahan G. Peripheral T cell tolerance. Annu Rev Immunol. 1992;10:51-69. doi: 10.1146/annurev.iy.10.040192.000411.
- Background: PubMed 1834543Hammerling GJ, Schonrich G, Momburg F, Auphan N, Malissen M, Malissen B, Schmitt-Verhulst AM, Arnold B. Non-deletional mechanisms of peripheral and central tolerance: studies with transgenic mice with tissue-specific expression of a foreign MHC class I antigen. Immunol Rev. 1991 Aug;122:47-67. doi: 10.1111/j.1600-065x.1991.tb00596.x.
- Background: PubMed 7016338Ploegh HL, Orr HT, Strominger JL. Major histocompatibility antigens: the human (HLA-A, -B, -C) and murine (H-2K, H-2D) class I molecules. Cell. 1981 May;24(2):287-99. doi: 10.1016/0092-8674(81)90318-4. No abstract available.
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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.
The record lists no contact. Use the official record to reach the sponsor.
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing methimazole in people with 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.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 4 Mar 2008. 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.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
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.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT00001421, which also lists 3 linked publications.
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 4 Mar 2008.