DermatomyositisPhase 2NCT00001261
Intravenousimmunoglobulin (IVIg) for the Treatment of Inflammatory Myopathies
A Phase 2 study testing Gamma Globulin in people with Dermatomyositis and Inclusion Body Myositis, run by National Institute of Neurological Disorders and Stroke (NINDS) at 1 site in United States.
Trial summary
Inflammatory myopathies are a group of muscle diseases characterized by muscle weakness, high levels of muscle enzymes in the blood, and inflammation of the tissue surrounding muscle fibers (endomysium). Read more about this study
- Who is it for?
- Any age
- Conditions the study is for
- Dermatomyositis, Inclusion Body Myositis, Polymyositis
- What is being tested?
- Gamma Globulin
- How many people
- 120 participants
- Where
- 1 siteUnited States
- Study dates
- May 1990to Jul 2002
- 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
Who may be able to join
- Selected patients should have PM, IBM or DM.
- Specifically they should have a) proximal muscle weakness; b) no evidence of clinical, histological or family history of another neuromuscular illness; c) elevation of muscle enzymes during the course of the disease; d) typical skin rash in case of DM; and e) diagnostic muscle biopsy.
- Suitable candidates for IVIg should be patients with active, bonefide disease who:
- have been treated with steroids but had: a) no response or incomplete response (as defined by continued muscle weakness) to high-dose therapy or b) a good response to steroids but inability to taper the dose without a flare of disease activity or c) unacceptable steroid side effects such as gastrointestinal hemorrhages, osteonecrosis, hyperglycemia, extreme weight gain etc., and
- have been treated with one immunosuppressive drug (such as azathioprine, Methotrexate, Cyclophosphamide, Cyclosporine) but without benefit or with unacceptable side effects.
Who cannot join
- Pregnant or nursing women (confirmed by a screening pregnancy test).
- Critically ill patients such as those requiring intravenous pressors for maintenance of cardiac output due to severe cardiomyopathy, patients with respiratory insufficiency and patients with severe muscle weakness requiring help for basic self care.
- Children below age 18.
- Patients with severe renal or hepatic disease, severe COPD or coronary artery disease or other systemic medical problems often seen when PM or DM is associated with severe cases of lupus, rheumatoid arthritis or scleroderma.
- Patients with known allergic reaction to IVIg.
- Serum IgA less than 11mg/dl.
Trial locations and contacts
1 site in 1 country.
United States1 site
National Institute of Neurological Disorders and Stroke (NINDS)
Bethesda, Maryland, 20892
About this study
Official title: The Efficacy of High-Dose Intravenous Immunoglobulin in Patients With Inflammatory Myopathies: A Three Month Randomized Trial With Option for Cross-Over
Inflammatory myopathies are a group of muscle diseases characterized by muscle weakness, high levels of muscle enzymes in the blood, and inflammation of the tissue surrounding muscle fibers (endomysium).
The diseases making up the inflammatory myopathies are grouped into three subsets:
Read more
- Polymyositis (PM)
II) Dermatomyositis (DM)
III) Inclusion Body Myositis (IBM)
Inflammatory myopathies are thought to be autoimmune processes and are treated with steroids and immunosuppressive drugs. However, many patients who initially respond to these treatments develop resistance to the therapy or experience side effects causing the treatments to be stopped.
Researchers believe that intravenous immunoglobulin (IVIg) may provide patients with PM, DM, and IBM a safer and more effective alternative to standard therapies for the diseases. IVIg is a drug that has been used successfully to treat other immune-related diseases of the nervous system.
The study will take 60 patients and divide them into two groups. Group one will receive 2 injections of IVIg once a month for three months. Group two will receive 2 injections of placebo "inactive injection of sterile water" once a month for three months. Following the three months of treatment, group one will begin taking the placebo and group two will begin taking IVIg for an additional 3 months. The drug will be considered effective if patients receiving it experience a significant improvement (>15%) in muscle strength.
Read the detailed description
The inflammatory myopathies are a group of acquired muscle diseases characterized by subacute onset of progressive proximal muscle weakness, elevated serum muscle enzymes and endomysial inflammation. They comprise 3 clinically distinct subsets: polymyositis (PM), dermatomyositis (DM) and Inclusion Body Myositis (IBM). Because immune-mediated mechanisms are primarily responsible for the clinical manifestations of these conditions, the treatment of choice is with corticosteroids or immunotherapy drugs. Although most of the patients initially respond to these drugs, a number of them become resistant or develop unacceptable side effects that necessitate their discontinuation. The need for a more effective and safe immunotherapy in patients with PM, DM or IBM prompted the present study using high dose intravenous immunoglobulin (IVIg). IVIg is an immunomodulating agent which has been shown to be effective and safe in the treatment of a number of patients with immune-related neuromuscular diseases.
This is a double-blind, randomized, placebo-controlled study involving 30 patients, who will receive IVIg or placebo for 3 months and then will cross-over to the alternate therapy for another period of 3 monthly infusions. The monthly dose of IVIg is 2 GM/Kg divided into two daily doses. The drug will be considered effective if patients experience an increase of more than 15% in their baseline muscle strength. Muscle strength will be assessed with a series of objective dynamometric measurements performed before and at the end of each monthly infusion.
- Conditions
- Dermatomyositis, Inclusion Body Myositis, Polymyositis
- Being tested
- Gamma Globulin
- Keywords
- Polymyositis/Dermatomyositis, Muscle Immunopathology, Intravenous High-Dose Immunoglobulin, Inclusion Body Myositis
- Medical subject headings
- Dermatomyositis, Myositis, Inclusion Body, Polymyositis
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Gamma GlobulinDrug
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 startMay 1990
- Study completionJul 2002
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.
- More trials run by National Institute of Neurological Disorders and Stroke (NINDS)The 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
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Enrolment
- 120 participants
Sponsor and investigators
- Sponsor type
- US National Institutes of Health
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00001261
- Sponsor's protocol ID
- 900139
- Other IDs
- 90-N-0139
- Status last verified
- Jul 2002
- First submitted
- 3 Nov 1999
- Last updated
- 4 Mar 2008
- Results posted
- No
Publications
Dalakas MC. Polymyositis, dermatomyositis and inclusion-body myositis. N Engl J Med. 1991 Nov 21;325(21):1487-98. doi: 10.1056/NEJM199111213252107. No abstract available.
PubMed 1658649Dalakas M. Treatment of polymyositis and dermatomyositis. Curr Opin Rheumatol. 1989 Dec;1(4):443-9. doi: 10.1097/00002281-198901040-00005. No abstract available.
PubMed 2702045Roifman CM, Schaffer FM, Wachsmuth SE, Murphy G, Gelfand EW. Reversal of chronic polymyositis following intravenous immune serum globulin therapy. JAMA. 1987 Jul 24-31;258(4):513-5. doi: 10.1001/jama.1987.03400040111034. No abstract available.
PubMed 3599350
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
- The Efficacy of High-Dose Intravenous Immunoglobulin in Patients With Inflammatory Myopathies: A Three Month Randomized Trial With Option for Cross-Over
- ClinicalTrials.gov ID
- NCT00001261
- Acronym / sponsor ID
- 900139
- Conditions
- Dermatomyositis, Inclusion Body Myositis, Polymyositis
- Keywords
- Polymyositis/Dermatomyositis, Muscle Immunopathology, Intravenous High-Dose Immunoglobulin, Inclusion Body Myositis
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 120 participants
- Lead sponsor
- National Institute of Neurological Disorders and Stroke (NINDS)US National Institutes of Health
- Study start
- May 1990
- Study completion
- Jul 2002
- 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.
Inflammatory myopathies are a group of muscle diseases characterized by muscle weakness, high levels of muscle enzymes in the blood, and inflammation of the tissue surrounding muscle fibers (endomysium).
The diseases making up the inflammatory myopathies are grouped into three subsets:
- Polymyositis (PM)
II) Dermatomyositis (DM)
III) Inclusion Body Myositis (IBM)
Inflammatory myopathies are thought to be autoimmune processes and are treated with steroids and immunosuppressive drugs. However, many patients who initially respond to these treatments develop resistance to the therapy or experience side effects causing the treatments to be stopped.
Researchers believe that intravenous immunoglobulin (IVIg) may provide patients with PM, DM, and IBM a safer and more effective alternative to standard therapies for the diseases. IVIg is a drug that has been used successfully to treat other immune-related diseases of the nervous system.
The study will take 60 patients and divide them into two groups. Group one will receive 2 injections of IVIg once a month for three months. Group two will receive 2 injections of placebo "inactive injection of sterile water" once a month for three months. Following the three months of treatment, group one will begin taking the placebo and group two will begin taking IVIg for an additional 3 months. The drug will be considered effective if patients receiving it experience a significant improvement (>15%) in muscle strength.
Detailed description
The inflammatory myopathies are a group of acquired muscle diseases characterized by subacute onset of progressive proximal muscle weakness, elevated serum muscle enzymes and endomysial inflammation. They comprise 3 clinically distinct subsets: polymyositis (PM), dermatomyositis (DM) and Inclusion Body Myositis (IBM). Because immune-mediated mechanisms are primarily responsible for the clinical manifestations of these conditions, the treatment of choice is with corticosteroids or immunotherapy drugs. Although most of the patients initially respond to these drugs, a number of them become resistant or develop unacceptable side effects that necessitate their discontinuation. The need for a more effective and safe immunotherapy in patients with PM, DM or IBM prompted the present study using high dose intravenous immunoglobulin (IVIg). IVIg is an immunomodulating agent which has been shown to be effective and safe in the treatment of a number of patients with immune-related neuromuscular diseases.
This is a double-blind, randomized, placebo-controlled study involving 30 patients, who will receive IVIg or placebo for 3 months and then will cross-over to the alternate therapy for another period of 3 monthly infusions. The monthly dose of IVIg is 2 GM/Kg divided into two daily doses. The drug will be considered effective if patients experience an increase of more than 15% in their baseline muscle strength. Muscle strength will be assessed with a series of objective dynamometric measurements performed before and at the end of each monthly infusion.
Study design
- Primary purpose
- Treatment
Who may qualify
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (5 listed)
- Selected patients should have PM, IBM or DM.
- Specifically they should have a) proximal muscle weakness; b) no evidence of clinical, histological or family history of another neuromuscular illness; c) elevation of muscle enzymes during the course of the disease; d) typical skin rash in case of DM; and e) diagnostic muscle biopsy.
- have been treated with steroids but had: a) no response or incomplete response (as defined by continued muscle weakness) to high-dose therapy or b) a good response to steroids but inability to taper the dose without a flare of disease activity or c) unacceptable steroid side effects such as gastrointestinal hemorrhages, osteonecrosis, hyperglycemia, extreme weight gain etc., and
- have been treated with one immunosuppressive drug (such as azathioprine, Methotrexate, Cyclophosphamide, Cyclosporine) but without benefit or with unacceptable side effects.
Key exclusion criteria (6 listed)
- Pregnant or nursing women (confirmed by a screening pregnancy test).
- Critically ill patients such as those requiring intravenous pressors for maintenance of cardiac output due to severe cardiomyopathy, patients with respiratory insufficiency and patients with severe muscle weakness requiring help for basic self care.
- Children below age 18.
- Patients with severe renal or hepatic disease, severe COPD or coronary artery disease or other systemic medical problems often seen when PM or DM is associated with severe cases of lupus, rheumatoid arthritis or scleroderma.
- Patients with known allergic reaction to IVIg.
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 |
|---|---|---|
| Gamma Globulin | 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 1658649Dalakas MC. Polymyositis, dermatomyositis and inclusion-body myositis. N Engl J Med. 1991 Nov 21;325(21):1487-98. doi: 10.1056/NEJM199111213252107. No abstract available.
- Background: PubMed 2702045Dalakas M. Treatment of polymyositis and dermatomyositis. Curr Opin Rheumatol. 1989 Dec;1(4):443-9. doi: 10.1097/00002281-198901040-00005. No abstract available.
- Background: PubMed 3599350Roifman CM, Schaffer FM, Wachsmuth SE, Murphy G, Gelfand EW. Reversal of chronic polymyositis following intravenous immune serum globulin therapy. JAMA. 1987 Jul 24-31;258(4):513-5. doi: 10.1001/jama.1987.03400040111034. 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 Gamma Globulin in people with Dermatomyositis and Inclusion Body Myositis, run by National Institute of Neurological Disorders and Stroke (NINDS) 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 5 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: 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 NCT00001261, 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.