Mycophenolate Sodium Treatment in Patients With Primary Sjogren's Syndrome

NCT ID: NCT00542763

Last Updated: 2007-10-11

Basic Information

• Recruitment Status: COMPLETED
• Clinical Phase: PHASE1
• Total Enrollment: 12 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2005-04-30
• Study Completion Date: 2007-09-30

Brief 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. 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.

Conditions

Primary Sjogren's Syndrome

Keywords

Sjogren's syndrome; Mycophenolate sodium

Study Design

• Allocation Method: NA
• Intervention Model: SINGLE_GROUP
• Primary Study Purpose: TREATMENT
• Blinding Strategy: NONE

Interventions

Mycophenolate sodium

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.

Intervention Type: DRUG

Other Intervention Names

Myfortic

Eligibility Criteria

Inclusion Criteria

• 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

Exclusion Criteria

• 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

• Minimum Eligible Age: 18 Years
• Maximum Eligible Age: 75 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Novartis

INDUSTRY

Sponsor Role: collaborator

University Hospital Muenster

OTHER

Sponsor Role: lead

Principal Investigators

Markus Gaubitz, MD

Role: PRINCIPAL_INVESTIGATOR

University Hospital Muenster

Locations

University Hospital Muenster

Münster, North Rhine-Westphalia, Germany

Countries

Germany

References

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.

Reference Type: BACKGROUND

PMID: 16932698 (View on PubMed)

Gaubitz 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.

Reference Type: BACKGROUND

PMID: 10602445 (View on PubMed)

Vitali 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.

Reference Type: BACKGROUND

PMID: 12006334 (View on PubMed)

Willeke P, Schotte H, Schluter B, Erren M, Becker H, Dyong A, Mickholz E, Domschke W, Gaubitz M. Interleukin 1beta and tumour necrosis factor alpha secreting cells are increased in the peripheral blood of patients with primary Sjogren's syndrome. Ann Rheum Dis. 2003 Apr;62(4):359-62. doi: 10.1136/ard.62.4.359.

Reference Type: BACKGROUND

PMID: 12634239 (View on PubMed)

Other Identifiers

myf-01-049

Identifier Type: -

Identifier Source: org_study_id