Primary Sjogren's SyndromePhase 1NCT00542763
Mycophenolate Sodium Treatment in Patients With Primary Sjogren's Syndrome
A Phase 1 study testing Mycophenolate sodium in people with Primary Sjogren's Syndrome, run by University Hospital Muenster at 1 site in Germany.
Trial summary
Primary Sjogren's syndrome (pSS) is an autoimmune disorder characterized by keratoconjunctivitis sicca and xerostomia. In addition, various extraglandular manifestations may develop. Several immunomodulating agents have been attempted in the treatment of pSS without achieving satisfactory results. Read more about this study
- Who is it for?
- Ages 18 to 75 years
- Conditions the study is for
- Primary Sjogren's Syndrome
- What is being tested?
- Mycophenolate sodium
- Type of treatment
- Pill or oral medicine
- How many people
- 12 participants
- Where
- 1 siteGermany
- Study dates
- Apr 2005to Sep 2007
- Phase
- Phase 1
- 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 75 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Diagnosis of primary Sjogren' Syndrome based on the American-European Consensus criteria
- Erythrocyte sedimentation rate >25mm/h and hypergammaglobulinemia (>1500 mg/dl)
- Presence of anti-SS-A and /or SS-B antibodies and / or rheumatoid factor
- Requirement of artificial teardrops due to symptomatic sicca syndrome
- Inadequate response or intolerance of prior treatment with hydroxychloroquine and / or azathioprine
- Adequate contraception for females of childbearing potential
Who cannot join
- Age below 18 or above 75 years
- Secondary Sjogren's syndrome
- History of cancer, severe infections or other uncontrolled diseases
- Treatment with concomitant disease modifying anti-rheumatic drugs within the least 8 weeks before baseline evaluation
- Prednisolone dose of > 5mg/d or changes of prednisolone dose within the least 4 weeks before baseline
- Use of secretagogues (e.g. pilocarpine, cevimeline) or medications that potentially diminish exocrine gland function (e.g. tricyclic antidepressants, anti-cholinergic drugs)
- Pregnant or lactating women
Trial locations and contacts
1 site in 1 country.
Germany1 site
University Hospital Muenster
Münster, North Rhine-Westphalia, 48129
About this study
Official title: Mycophenolate Sodium Treatment in Patients With Primary Sjogren's Syndrome - An Open Label Pilot Trial
Primary Sjogren's syndrome (pSS) is an autoimmune disorder characterized by keratoconjunctivitis sicca and xerostomia. In addition, various extraglandular manifestations may develop. Several immunomodulating agents have been attempted in the treatment of pSS without achieving satisfactory results. Currently, there is no approved systemic treatment for pSS.
Mycophenolic acid (MPA) is a selective inhibitor of inosine-monophosphate-dehydrogenase which leads to inhibition of the de novo pathway of nucleotide synthesis. The antiproliferative effect of MPA mainly affects activated T- and B-lymphocytes because the proliferation of these cells is critically dependent on the de novo purine synthesis compared to other eukaryotic cells. Since these lymphocytes have been suggested to play a pivotal role in the inflammation and immunopathogenesis of pSS, mycophenolate-sodium might be a promising agent in the treatment of pSS.
We perform a single-centre, open-label pilot trial with Mycophenolate sodium in pSS.
Read the detailed description
Mycophenolic acid containing compounds such as mycophenolate mofetil and enteric coated mycophenolate sodium are immunosuppressive drugs approved for the prevention of transplant rejection. Mycophenolate mofetil (MMF) is an effective treatment in systemic lupus erythematosus and other autoimmune diseases.
MMF has been used as maintenance therapy after treatment with rituximab (anti-CD20 antibody) in a pSS patient. We have reported a case of successful treatment with MMF in pSS with vasculitis.
The recent observations and the immunosuppressive effect of MPA in other autoimmune diseases led us to evaluate the efficacy and safety of MPA treatment in patients with pSS refractory to other immunosuppressive agents.
The observation period will be 6 months. At baseline, after 3, and after 6 months we examine the clinical status including glandular function tests as well as different laboratory parameters associated with pSS. In addition subjective parameters will be determined on the basis of different questionnaires.
- Conditions
- Primary Sjogren's Syndrome
- Being tested
- Mycophenolate sodium
- Keywords
- Sjogren's syndrome, Mycophenolate sodium
- Medical subject headings
- Sjogren's Syndrome
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Mycophenolate sodiumDrug
Also called Myfortic
Medical treatment is initiated with one tablet of 360 mg mycophenolate sodium orally per day for eligible patient. The dosage will be increased weekly by 360 mg up to a maximum stable dose of 1440mg daily. In patients not well tolerating the drug the dosage can be reduced to 720 mg per day.
What is being measured
The primary outcome is the main question the study is designed to answer.
Efficacy of mycophenolate sodium for sicca syndrome and changes in laboratory values associated with the disease
Measured: Basline, week 12 and week 24
Secondary outcomes (1)
Safety of mycophenolate sodium in patients with primary Sjogren's syndrome: Clinical examination, full blood count, renal function tests, liver function tests
Measured: basline, after week 4, 12, and week 24
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted11 Oct 2007 (estimated)
- Study startApr 2005
- Study completionSep 2007
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (4)Papers the sponsor has linked to this study.
- Other trials for Primary Sjogren's SyndromeStudies 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 1
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 12 participants (actual)
Sponsor and investigators
- Lead sponsor
- University Hospital Muenster
- Sponsor type
- University, hospital or other
- Collaborators
- Novartis
- Investigators
- Markus Gaubitz, MD, Principal investigator, University Hospital Muenster
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00542763
- Sponsor's protocol ID
- myf-01-049
- Status last verified
- Aug 2004
- First submitted
- 10 Oct 2007
- Last updated
- 11 Oct 2007
- Results posted
- No
- Data monitoring committee
- No
Publications
Mavragani CP, Moutsopoulos NM, Moutsopoulos HM. The management of Sjogren's syndrome. Nat Clin Pract Rheumatol. 2006 May;2(5):252-61. doi: 10.1038/ncprheum0165.
PubMed 16932698Gaubitz M, Schorat A, Schotte H, Kern P, Domschke W. Mycophenolate mofetil for the treatment of systemic lupus erythematosus: an open pilot trial. Lupus. 1999;8(9):731-6. doi: 10.1191/096120399678840927.
PubMed 10602445Vitali C, Bombardieri S, Jonsson R, Moutsopoulos HM, Alexander EL, Carsons SE, Daniels TE, Fox PC, Fox RI, Kassan SS, Pillemer SR, Talal N, Weisman MH; European Study Group on Classification Criteria for Sjogren's Syndrome. Classification criteria for Sjogren's syndrome: a revised version of the European criteria proposed by the American-European Consensus Group. Ann Rheum Dis. 2002 Jun;61(6):554-8. doi: 10.1136/ard.61.6.554.
PubMed 12006334Willeke P, Domschke W, Gaubitz M: Mycophenolate Mofetil for the treatment of primary Sjogren's Syndrome: A case report. Ann Rheum Dis, 62 (Suppl. 1):352,2003
Study at a glance
From the registry record, last updated by the sponsor on 11 Oct 2007. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Mycophenolate Sodium Treatment in Patients With Primary Sjogren's Syndrome - An Open Label Pilot Trial
- ClinicalTrials.gov ID
- NCT00542763
- Acronym / sponsor ID
- myf-01-049
- Conditions
- Primary Sjogren's Syndrome
- Keywords
- Sjogren's syndrome, Mycophenolate sodium
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 12 participants (actual)
- Lead sponsor
- University Hospital MuensterUniversity, hospital or other
- Collaborators
- Novartis
- Study start
- Apr 2005
- Study completion
- Sep 2007 (actual)
- First posted
- 11 Oct 2007 (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.
Primary Sjogren's syndrome (pSS) is an autoimmune disorder characterized by keratoconjunctivitis sicca and xerostomia. In addition, various extraglandular manifestations may develop. Several immunomodulating agents have been attempted in the treatment of pSS without achieving satisfactory results. Currently, there is no approved systemic treatment for pSS.
Mycophenolic acid (MPA) is a selective inhibitor of inosine-monophosphate-dehydrogenase which leads to inhibition of the de novo pathway of nucleotide synthesis. The antiproliferative effect of MPA mainly affects activated T- and B-lymphocytes because the proliferation of these cells is critically dependent on the de novo purine synthesis compared to other eukaryotic cells. Since these lymphocytes have been suggested to play a pivotal role in the inflammation and immunopathogenesis of pSS, mycophenolate-sodium might be a promising agent in the treatment of pSS.
We perform a single-centre, open-label pilot trial with Mycophenolate sodium in pSS.
Detailed description
Mycophenolic acid containing compounds such as mycophenolate mofetil and enteric coated mycophenolate sodium are immunosuppressive drugs approved for the prevention of transplant rejection. Mycophenolate mofetil (MMF) is an effective treatment in systemic lupus erythematosus and other autoimmune diseases.
MMF has been used as maintenance therapy after treatment with rituximab (anti-CD20 antibody) in a pSS patient. We have reported a case of successful treatment with MMF in pSS with vasculitis.
The recent observations and the immunosuppressive effect of MPA in other autoimmune diseases led us to evaluate the efficacy and safety of MPA treatment in patients with pSS refractory to other immunosuppressive agents.
The observation period will be 6 months. At baseline, after 3, and after 6 months we examine the clinical status including glandular function tests as well as different laboratory parameters associated with pSS. In addition subjective parameters will be determined on the basis of different questionnaires.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
Who may qualify
- Ages
- 18 to 75 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (6 listed)
- Diagnosis of primary Sjogren' Syndrome based on the American-European Consensus criteria
- Erythrocyte sedimentation rate >25mm/h and hypergammaglobulinemia (>1500 mg/dl)
- Presence of anti-SS-A and /or SS-B antibodies and / or rheumatoid factor
- Requirement of artificial teardrops due to symptomatic sicca syndrome
- Inadequate response or intolerance of prior treatment with hydroxychloroquine and / or azathioprine
Key exclusion criteria (7 listed)
- Age below 18 or above 75 years
- Secondary Sjogren's syndrome
- History of cancer, severe infections or other uncontrolled diseases
- Treatment with concomitant disease modifying anti-rheumatic drugs within the least 8 weeks before baseline evaluation
- Prednisolone dose of > 5mg/d or changes of prednisolone dose within the least 4 weeks before baseline
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 sodiumAlso: MyforticMedical treatment is initiated with one tablet of 360 mg mycophenolate sodium orally per day for eligible patient. The dosage will be increased weekly by 360 mg up to a maximum stable dose of 1440mg daily. In patients not well tolerating the drug the dosage can be reduced to 720 mg per day. | Drug | – |
Primary outcome
Efficacy of mycophenolate sodium for sicca syndrome and changes in laboratory values associated with the disease
Time frame: Basline, week 12 and week 24
1 secondary outcome
Safety of mycophenolate sodium in patients with primary Sjogren's syndrome: Clinical examination, full blood count, renal function tests, liver function tests
Time frame: basline, after week 4, 12, and week 24
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 11 Oct 2007.
- Background: PubMed 16932698Mavragani CP, Moutsopoulos NM, Moutsopoulos HM. The management of Sjogren's syndrome. Nat Clin Pract Rheumatol. 2006 May;2(5):252-61. doi: 10.1038/ncprheum0165.
- Background: PubMed 10602445Gaubitz M, Schorat A, Schotte H, Kern P, Domschke W. Mycophenolate mofetil for the treatment of systemic lupus erythematosus: an open pilot trial. Lupus. 1999;8(9):731-6. doi: 10.1191/096120399678840927.
- Background: PubMed 12006334Vitali C, Bombardieri S, Jonsson R, Moutsopoulos HM, Alexander EL, Carsons SE, Daniels TE, Fox PC, Fox RI, Kassan SS, Pillemer SR, Talal N, Weisman MH; European Study Group on Classification Criteria for Sjogren's Syndrome. Classification criteria for Sjogren's syndrome: a revised version of the European criteria proposed by the American-European Consensus Group. Ann Rheum Dis. 2002 Jun;61(6):554-8. doi: 10.1136/ard.61.6.554.
- BackgroundWilleke P, Domschke W, Gaubitz M: Mycophenolate Mofetil for the treatment of primary Sjogren's Syndrome: A case report. Ann Rheum Dis, 62 (Suppl. 1):352,2003
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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.
Markus Gaubitz, MD principal investigator
University Hospital Muenster
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing Mycophenolate sodium in people with Primary Sjogren's Syndrome, run by University Hospital Muenster at 1 site in Germany.
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 6 inclusion and 7 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 11 Oct 2007. 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 Germany. 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 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
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?
Efficacy of mycophenolate sodium for sicca syndrome and changes in laboratory values associated with the disease (time frame: Basline, week 12 and week 24). 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 NCT00542763, which also lists 4 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 11 Oct 2007.