Myasthenia Gravis, GeneralizedPhase 3NCT00683969
A Study to Assess the Effect of CellCept (Mycophenolate Mofetil) and Reduced Corticosteroids in Controlling Symptoms of Myasthenia Gravis
A Phase 3 study testing mycophenolate mofetil (CellCept) in people with Myasthenia Gravis, Generalized, run by Hoffmann-La Roche at 43 sites in 14 countries.
Trial summary
The efficacy and safety of CellCept (1g po, bid for 36 weeks) will be assessed in patients with myasthenia gravis receiving prednisone, or other corticosteroids. During the study, patients will undergo gradual corticosteroid dose reduction, if they respond to treatment. Read more about this study
- Who is it for?
- Ages 18 to 80 years
- Conditions the study is for
- Myasthenia Gravis, Generalized
- What is being tested?
- mycophenolate mofetil (CellCept)
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 136 participants
- Where
- 43 sitesin 14 countries
- Study dates
- Aug 2004to May 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 to 80 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- adult patients 18 to 80 years of age;
- diagnosis of myasthenia gravis;
- history of myasthenia weakness involving more than ocular (ie eye) or peri-ocular muscle;
- duration of myasthenia gravis symptoms (including ocular symptoms) <=10 years;
- prednisone dose >=20 mg/day (or equivalent alternate-day dose) for >=4 weeks.
Who cannot join
- female patients who are pregnant, breastfeeding, or lactating;
- regularly scheduled plasma exchange (PE) or intravenous immunoglobulin (IVIG) treatment, or PE or IVIG treatment within 2 weeks prior to randomization;
- any prior clinically significant use of CellCept or other immunosuppressive therapy (except corticosteroids), or within 8 weeks prior to randomization.
Trial locations and contacts
43 sites in 14 countries.
43 sites in 14 countries
United States10 sites
Study site
Chapel Hill, North Carolina, 27599-7025
Study site
Columbus, Ohio, 43210
Study site
Dallas, Texas, 75390
Study site
Galveston, Texas, 77555
Study site
Kansas City, Kansas, 66160-7314
Study site
Miami, Florida, 33136
Study site
Rochester, New York, 14607
Study site
Sacramento, California, 95817
Study site
Sun City, Arizona
Study site
Upland, Pennsylvania, 19013-3395
Germany4 sites
Study site
Düsseldorf, 40225
Study site
München, 81675
Study site
Regensburg, 93053
Study site
Tübingen, 72076
Italy3 sites
Study site
Milan, 20133
Study site
Roma, 185
Study site
San Donato Milanese, 20097
Mexico3 sites
Study site
Mexico City, 06700
Study site
Mexico City, 06726
Study site
Mexico City, 14000
Netherlands3 sites
Study site
Leiden, 2300 RC
Study site
Maastricht, 6202 AZ
Study site
Rotterdam, 3015 GD
Ukraine3 sites
Study site
Kharkiv, 61068
Study site
Kiev
Study site
Zaporizhzhya, 69035
United Kingdom3 sites
Study site
Liverpool, L9 1AE
Study site
Oxford, OX2 6HE
Study site
Salford, M6 8HD
Canada2 sites
Study site
Edmonton, Alberta, T6G 2B7
Study site
London, Ontario, N6A 5A5
About this study
Official title: A Prospective, Randomized, Double-Blind, Placebo-Controlled, Parallel Group, Multicenter, 36-Week Trial to Assess the Efficacy and Safety of Adjunct Mycophenolate Mofetil (MMF) to Maintain or Improve Symptom Control With Reduced Corticosteroid in Subjects With Myasthenia Gravis
The efficacy and safety of CellCept (1g po, bid for 36 weeks) will be assessed in patients with myasthenia gravis receiving prednisone, or other corticosteroids. During the study, patients will undergo gradual corticosteroid dose reduction, if they respond to treatment. The anticipated time on study treatment is 3-12 months, and the target sample size is 100-500 individuals.
- Conditions
- Myasthenia Gravis, Generalized
- Being tested
- mycophenolate mofetil (CellCept), placebo
- Medical subject headings
- Myasthenia Gravis
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
mycophenolate mofetil (CellCept)Drug
1g bid for 36 weeks
placeboDrug
po bid for 36 weeks
| Study group | Role | What they receive |
|---|---|---|
| 1 | Experimental | Not described Receives: Drug: mycophenolate mofetil (CellCept) |
| 2 | Placebo comparator | Not described Receives: Drug: placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
Proportion of subjects reaching responder status
Measured: Week 36
Secondary outcomes (3)
Time to start of response
Measured: Event driven
Mean and median prednisone dose and cholinesterase inhibitor dose
Measured: Week 36
Adverse events, lab parameters, vital signs
Measured: Throughout study
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted26 May 2008 (estimated)
- Study startAug 2004
- Study completionMay 2007
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Myasthenia Gravis, GeneralizedStudies of the same condition, open and under way.
- More trials run by Hoffmann-La RocheThe 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 3
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Double blind
- Who is masked
- Participant, Investigator
- Enrolment
- 136 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Hoffmann-La Roche
- Sponsor type
- Industry (pharma, biotech, devices)
- Collaborators
- Aspreva Pharmaceuticals
- Investigators
- Clinical Trials, Study director, Hoffmann-La Roche, 973-235-5000
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00683969
- Sponsor's protocol ID
- WX17798
- Status last verified
- May 2008
- First submitted
- 19 May 2008
- Last updated
- 26 May 2008
- Results posted
- No
Publications
Sengupta M, Cheema A, Kaminski HJ, Kusner LL; Muscle Study Group. Serum metabolomic response of myasthenia gravis patients to chronic prednisone treatment. PLoS One. 2014 Jul 17;9(7):e102635. doi: 10.1371/journal.pone.0102635. eCollection 2014.
PubMed 25032816
Study at a glance
From the registry record, last updated by the sponsor on 26 May 2008. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Prospective, Randomized, Double-Blind, Placebo-Controlled, Parallel Group, Multicenter, 36-Week Trial to Assess the Efficacy and Safety of Adjunct Mycophenolate Mofetil (MMF) to Maintain or Improve Symptom Control With Reduced Corticosteroid in Subjects With Myasthenia Gravis
- ClinicalTrials.gov ID
- NCT00683969
- Acronym / sponsor ID
- WX17798
- Conditions
- Myasthenia Gravis, Generalized
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 136 participants (estimated)
- Lead sponsor
- Hoffmann-La RocheIndustry (pharma, biotech, devices)
- Collaborators
- Aspreva Pharmaceuticals
- Study start
- Aug 2004
- Study completion
- May 2007 (actual)
- First posted
- 26 May 2008 (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.
The efficacy and safety of CellCept (1g po, bid for 36 weeks) will be assessed in patients with myasthenia gravis receiving prednisone, or other corticosteroids. During the study, patients will undergo gradual corticosteroid dose reduction, if they respond to treatment. The anticipated time on study treatment is 3-12 months, and the target sample size is 100-500 individuals.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Double blind (participant, investigator)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| 1 | Experimental | Drug: mycophenolate mofetil (CellCept) |
| 2 | Placebo comparator | Drug: placebo |
Who may qualify
- Ages
- 18 to 80 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (5 listed)
- adult patients 18 to 80 years of age;
- diagnosis of myasthenia gravis;
- history of myasthenia weakness involving more than ocular (ie eye) or peri-ocular muscle;
- duration of myasthenia gravis symptoms (including ocular symptoms) <=10 years;
- prednisone dose >=20 mg/day (or equivalent alternate-day dose) for >=4 weeks.
Key exclusion criteria (3 listed)
- female patients who are pregnant, breastfeeding, or lactating;
- regularly scheduled plasma exchange (PE) or intravenous immunoglobulin (IVIG) treatment, or PE or IVIG treatment within 2 weeks prior to randomization;
- any prior clinically significant use of CellCept or other immunosuppressive therapy (except corticosteroids), or within 8 weeks prior to randomization.
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 (CellCept)1g bid for 36 weeks | Drug | 1 |
| placebopo bid for 36 weeks | Drug | 2 |
Primary outcome
Proportion of subjects reaching responder status
Time frame: Week 36
3 secondary outcomes
Time to start of response
Time frame: Event driven
Mean and median prednisone dose and cholinesterase inhibitor dose
Time frame: Week 36
Adverse events, lab parameters, vital signs
Time frame: Throughout study
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 26 May 2008.
- Derived from this study: PubMed 25032816Sengupta M, Cheema A, Kaminski HJ, Kusner LL; Muscle Study Group. Serum metabolomic response of myasthenia gravis patients to chronic prednisone treatment. PLoS One. 2014 Jul 17;9(7):e102635. doi: 10.1371/journal.pone.0102635. eCollection 2014.
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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.
Clinical Trials study director
Hoffmann-La Roche, 973-235-5000
43 sites in 14 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing mycophenolate mofetil (CellCept) in people with Myasthenia Gravis, Generalized, run by Hoffmann-La Roche at 43 sites in 14 countries.
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 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 26 May 2008. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 43 sites in United States, Germany, India, Italy, Mexico and 9 more countries. 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. Some participants in this study may receive a placebo. 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?
Proportion of subjects reaching responder status (time frame: Week 36). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT00683969, which also lists 1 linked publication.
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 26 May 2008.