Myasthenia GravisPhase 3NCT00285350
Mycophenolate Mofetil in Myasthenia Gravis
A Phase 3 study testing mycophenolate mofetil in people with Myasthenia Gravis, run by FDA Office of Orphan Products Development.
Trial summary
This is a prospective, multi-center, double-blind, placebo-controlled trial to determine the efficacy and safety of mycophenolate mofetil (MM) in combination with prednisone as the initial form of immunosuppression in patients with acquired myasthenia gravis (MG). Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Myasthenia Gravis
- What is being tested?
- mycophenolate mofetil
- How many people
- 80 participants
- Where
- No sites listed yet
- Study dates
- Sep 2002to Mar 2007
- Phase
- Phase 3
- 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 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Acquired generalized MG diagnosed by one of the Principal Investigators based on:
- Examination by site PI showing myasthenic weakness that is not limited to the ocular or peri-ocular muscles.
- Elevated acetylcholine receptor antibodies.
- Positive edrophonium chloride test or abnormal neuromuscular transmission demonstrated by single fiber EMG or repetitive nerve stimulation.
- Aged at least 18.
- Able to give informed consent.
Show all 9Show fewer
- Taking a constant dose of Mestinon for at least 2 weeks.
- Symptom severity that would, in the judgment of the site investigator, justify initiation of immunosuppressive treatment.
- Able and willing to comply with study requirements.
Who cannot join
- Thymoma now or in the past.
- Plasma exchange or IVIG treatment within 90 days of randomization.
- Treatment with azathioprine, cyclosporine, mycophenolate mofetil, or other immunosuppressive medication since onset of MG. Treatment with prednisone or other corticosteroids within the previous 90 days.
- Exception: patients may have taken doses of these immunosuppressant medications that are judged by the Principal Investigator to have been clinically insignificant, i.e. unlikely to produce improvement in MG.
- Women of childbearing potential who are pregnant, breast-feeding or not practicing effective contraception.
- Renal failure, active thyroid or hepatocellular disease, chronic infection, poorly controlled cardiac disease, or any other illness, including psychiatric disease, that would, in the opinion of the treating physician, make it unsafe for the patient to participate or would interfere with the interpretation of study results.
Show all 13Show fewer
- Weakness affecting only ocular or peri-ocular muscles (Myasthenia Gravis Foundation of America Class I).
- Severe weakness predominantly affecting oropharyngeal, respiratory muscles or both (MGFA Class IVB).
- Crisis or impending crisis (defined as FVC <10ml/Kg or bulbar weakness severe enough to compromise airway protection.)
- Hemoglobin <10mg/dl; WBC <3,500.
- History of non-compliance with treatment and office visits.
- Thymectomy within 12 months before randomization.
- Concurrent medical condition that would pose an unacceptable risk from immunosuppression, including a positive skin test for tuberculosis (PPD), unless the patient has previously received appropriate treatment.
About this study
Official title: A Trial of Mycophenolate Mofetil in Myasthenia Gravis
This is a prospective, multi-center, double-blind, placebo-controlled trial to determine the efficacy and safety of mycophenolate mofetil (MM) in combination with prednisone as the initial form of immunosuppression in patients with acquired myasthenia gravis (MG).
Read the detailed description
80 patients with seropositive MG at 18 academic centers will be randomized to 3 months of treatment with 2.5 gm MM/day (1,250 mg q 12 hours, +/- 2 hours) plus 20 mg prednisone/day versus placebo plus 20 mg/day prednisone. The primary measure of efficacy will be the change from baseline in Quantitative MG (QMG) score at the end of 3 months. Secondary outcome measures include survival analysis for treatment failure, MG-related impairment of daily activities, functional assessment, manual muscle testing, SF-36 Health Status, and serum concentration of antibodies to the acetylcholine receptor. Study completers will have the option of taking open-label MM for an additional 6 months, during which prednisone will be reduced to the lowest dose necessary to maintain the optimum clinical response.
- Conditions
- Myasthenia Gravis
- Being tested
- mycophenolate mofetil
- Keywords
- myasthenia gravis
- Medical subject headings
- Myasthenia Gravis
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
What is being measured
The primary outcome is the main question the study is designed to answer.
QMGS
Secondary outcomes (1)
Multiple
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted2 Feb 2006 (estimated)
- Study startSep 2002
- Study completionMar 2007
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Myasthenia GravisStudies 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 3
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Double blind
- Enrolment
- 80 participants
Sponsor and investigators
- Lead sponsor
- FDA Office of Orphan Products Development
- Sponsor type
- US federal agency
- Collaborators
- Duke University
- Investigators
- Donald B Sanders, MD, Principal investigator, Duke University
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00285350
- Sponsor's protocol ID
- 2154
- Status last verified
- Mar 2007
- First submitted
- 31 Jan 2006
- Last updated
- 25 Mar 2015
- Results posted
- No
Study at a glance
From the registry record, last updated by the sponsor on 25 Mar 2015. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Trial of Mycophenolate Mofetil in Myasthenia Gravis
- ClinicalTrials.gov ID
- NCT00285350
- Acronym / sponsor ID
- 2154
- Conditions
- Myasthenia Gravis
- Keywords
- myasthenia gravis
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 80 participants
- Lead sponsor
- FDA Office of Orphan Products DevelopmentUS federal agency
- Collaborators
- Duke University
- Study start
- Sep 2002
- Study completion
- Mar 2007 (actual)
- First posted
- 2 Feb 2006 (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 is a prospective, multi-center, double-blind, placebo-controlled trial to determine the efficacy and safety of mycophenolate mofetil (MM) in combination with prednisone as the initial form of immunosuppression in patients with acquired myasthenia gravis (MG).
Detailed description
80 patients with seropositive MG at 18 academic centers will be randomized to 3 months of treatment with 2.5 gm MM/day (1,250 mg q 12 hours, +/- 2 hours) plus 20 mg prednisone/day versus placebo plus 20 mg/day prednisone. The primary measure of efficacy will be the change from baseline in Quantitative MG (QMG) score at the end of 3 months. Secondary outcome measures include survival analysis for treatment failure, MG-related impairment of daily activities, functional assessment, manual muscle testing, SF-36 Health Status, and serum concentration of antibodies to the acetylcholine receptor. Study completers will have the option of taking open-label MM for an additional 6 months, during which prednisone will be reduced to the lowest dose necessary to maintain the optimum clinical response.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Double blind
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (9 listed)
- Acquired generalized MG diagnosed by one of the Principal Investigators based on:
- Aged at least 18.
- Able to give informed consent.
- Taking a constant dose of Mestinon for at least 2 weeks.
- Symptom severity that would, in the judgment of the site investigator, justify initiation of immunosuppressive treatment.
Key exclusion criteria (13 listed)
- Thymoma now or in the past.
- Plasma exchange or IVIG treatment within 90 days of randomization.
- Treatment with azathioprine, cyclosporine, mycophenolate mofetil, or other immunosuppressive medication since onset of MG. Treatment with prednisone or other corticosteroids within the previous 90 days.
- Women of childbearing potential who are pregnant, breast-feeding or not practicing effective contraception.
- Renal failure, active thyroid or hepatocellular disease, chronic infection, poorly controlled cardiac disease, or any other illness, including psychiatric disease, that would, in the opinion of the treating physician, make it unsafe for the patient to participate or would interfere with the interpretation of study results.
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 |
|---|---|---|
| mycophenolate mofetil | Drug | – |
Primary outcome
QMGS
1 secondary outcome
Multiple
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 25 Mar 2015.
Related trials for Myasthenia Gravis
RecruitingPhase 3
A Phase 3 Study to Evaluate Claseprubart in Adults With Generalized Myasthenia Gravis (EMERGE)
Myasthenia Gravis, Generalized
Active, not recruitingPhase 2
A Phase 2 Study to Evaluate Povetacicept in Adults With Generalized Myasthenia Gravis
Myasthenia Gravis, Generalized
RecruitingPhase 2
A Study to Evaluate the Efficacy and Safety of Maintenance Ublituximab Following Induction With Efgartigimod Administration in Participants With Myasthenia Gravis (MG)
Myasthenia Gravis
Active, not recruitingPhase 4
Phase 4 Study Evaluating Efficacy and Safety of Rozanolixizumab in Adult Chinese Participants With Generalized Myasthenia Gravis
Myasthenia Gravis
Active, not recruitingPhase 2/3
Evaluating the Pharmacokinetics, Pharmacodynamics, and Safety of Efgartigimod Administered Intravenously in Children With Generalized Myasthenia Gravis
Generalized Myasthenia Gravis
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.
Donald B Sanders, MD principal investigator
Duke University
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing mycophenolate mofetil in people with Myasthenia Gravis, run by FDA Office of Orphan Products Development.
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 9 inclusion and 13 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 25 Mar 2015. Status can change between updates, so confirm with the study contact before making plans.
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 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
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.
What does the primary outcome measure?
QMGS. 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 25 Mar 2015.