Back to resultsEfficacy and Safety of Mycophenolate Mofetil in subjectswithSjogren's Syndrome
Primary Purpose
Sjogren's Syndrome
Status
Unknown status
Phase
Phase 2
Locations
Study Type
Interventional
Intervention
Mycophenolate mofetil
Sponsored by
About this trial
This is an interventional treatment trial for Sjogren's Syndrome
Eligibility Criteria
Inclusion Criteria:
- Diagnosis of primary Sjogren's syndrome based on the 2002 American-European Consensus criteria
- Aged 20 to 75 years
- Stable doses of oral corticosteroids(≦5mg/d) for at least 4 weeks before enrollment
Intolerance or inadequate response to hydroxychloroquine and (pilocarpine or cevimeline), defined as less than 50mm on at least 2 of VAS including:
- global assessment : 0mm (very bad) to 100mm (very good)
- pain: 0mm (very bad) to 100mm (very good)
- fatigue: 0mm (very bad) to 100mm (very good)
- xerostomia: 0mm (very bad) to 100mm (very good)
- Adequate contraception for patients of childbearing potential
Exclusion Criteria:
- Receiving biologics during the 6 previous months or any other immunosuppressant (methotrexate, cyclophosphamide, cyclosporine, azathioprine, mycophenolate mofetil (MMF), mycophenolate sodium, leflunomide, penicillamine) during the previous month
Any one of laboratory abnormalities:
- Serum creatinine ≥2 mg/dl
- aspartate aminotransferase (AST) or alanine transaminase (ALT) more than 1.5 x upper normal range of the laboratory
- Leukopenia (WBC<4000/μl)
- Hb ≤ 9 g/dl (5.6 mmol/l) for males and 8.5 g/dl (5.3 mmol/l) for females
- Neutrophil less than 1.5 x 109/l
- Platelet count less than 150 x 109/l
- History of other autoimmune diseases
- Use topical cyclosporine eyedrops, antihistamine, anticholinergic, antidepressant, or antipsychotic drug with possible effects on ocular dryness or oral dryness within 1 month
- Pregnant or lactating women
- Previous or current malignancies adequately controlled less than 5 years, hepatitis B, hepatitis C, HIV infection, tuberculosis, or diabetes
- Subjects with serious infections requiring hospitalization within the last 12 months
- Subjects with herpes zoster or cytomegalovirus that resolved less than 2 months before enrollment
- Subjects who have received any live vaccines within 3 months
- Underlying cardiac, pulmonary, metabolic, renal, hepatic, gastrointestinal, haematological or neurological conditions, chronic or latent infectious diseases or immune deficiency which places the patient at an unacceptable risk for participation in the study
- History of recurring or chronic infections or underlying conditions which may further predispose patients to serious infection
- Subjects who are impaired, incapacitated, or incapable of completing study-related assessments
- History of allergy to mycophenolate sodium
- Nausea, vomiting, diarrhea within 1 week before enrollment
- History of psychosis, seizure, retinopathy
- Infection 2 weeks before enrollment
- Heart rate < 60/min at rest
Sites / Locations
Arms of the Study
Arm 1
Arm 2
Arm Type
Experimental
Experimental
Arm Label
Mycophenolate mofetil standard
Mycophenolate sodium low
Arm Description
mycophenolate mofetil 250mg 2# twice per day (BID)
mycophenolate mofetil 250mg 1# twice per day (BID)
Outcomes
Primary Outcome Measures
Change of Composite Index of Sjogren's syndrome
Secondary Outcome Measures
ocular dryness
We will calculate the change of ocular dryness from baseline to week 28 (0mm [very bad] to 100mm [very good])
physician visual analog scale (VAS)
We will calculate the change of physician VAS from baseline to week 28 (0mm [very bad] to 100mm [very good])
Schirmer's test
We will calculate the change of Schirmer's test results from baseline to week 28
Saxon's test
We will calculate the change of Saxon's test results from baseline to week 28
heart rate
resting heart rate
blood pressure
resting blood pressure
leukocyte count
WBC count
Hb level
Hb level
platelet count
platelet count
Full Information
NCT ID
NCT02691949
First Posted
February 16, 2016
Last Updated
September 21, 2016
Sponsor
Kaohsiung Medical University
1. Study Identification
Unique Protocol Identification Number
NCT02691949
Brief Title
Efficacy and Safety of Mycophenolate Mofetil in subjectswithSjogren's Syndrome
Official Title
Efficacy and Safety of Mycophenolate Mofetil in subjectswithSjogren's Syndrome
Study Type
Interventional
2. Study Status
Record Verification Date
September 2016
Overall Recruitment Status
Unknown status
Study Start Date
February 2016 (undefined)
Primary Completion Date
April 2018 (Anticipated)
Study Completion Date
April 2018 (Anticipated)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Principal Investigator
Name of the Sponsor
Kaohsiung Medical University
4. Oversight
Data Monitoring Committee
No
5. Study Description
Brief Summary
Past literature showed encouraging effects of mycophenolate on dryness symptoms and quality of life in patients with Sjogren's syndrome. Mycophenolate also has excellent immunomodulation effects in lupus nephritis. Currently Mycophenolate is only used in lupus nephritis and organ transplant. It is unknown whether low dosage of mycophenolate mofetil could be used to improve ocular dryness and oral dryness in patients with Sjogren's syndrome.
Detailed Description
Sjogren's syndrome is one of the most common autoimmune diseases in Taiwan. It is characterized by keratoconjunctivitis sicca and xerostomia. Although it is well established that Sjogren's syndrome is caused by infiltration and destruction of lacrimal gland and salivary gland by lymphocytic cells, effective treatment of patients' symptoms is lacking. Hydroxychloroquine is the most well-studied medication in Sjogren's syndrome. However, recent clinical trials showed disappointing effects of hydroxychloroquine in Sjogren's syndrome. Thus there is an unmet need to find effective treatment for patient's bothering symptoms.
Mycophenolate is a selective inhibitor of inosinemonophosphate dehydrogenase which leads to inhibition of the de novo pathway of nucleotide synthesis. The antiproliferative effect of mycophenolate mainly affects activated T and B lymphocytes because the proliferation of these cells is critically dependent on the de novo purine synthesis compared with other eukaryotic cells. Since these lymphocytes have been suggested to play a pivotal role in the inflammation and immunopathogenesis of Sjogren's syndrome, mycophenolate might be a promising agent in the treatment of Sjogren's syndrome.
Past literature showed encouraging effects of mycophenolate on dryness symptoms and quality of life in patients with Sjogren's syndrome. Mycophenolate also has excellent immunomodulation effects in lupus nephritis. Currently mycophenolate is only used in lupus nephritis and organ transplant. It is unknown whether low dosage of mycophenolate could be used to improve ocular dryness and oral dryness in patients with Sjogren's syndrome.
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Sjogren's Syndrome
7. Study Design
Primary Purpose
Treatment
Study Phase
Phase 2
Interventional Study Model
Parallel Assignment
Masking
None (Open Label)
Allocation
Randomized
Enrollment
54 (Anticipated)
8. Arms, Groups, and Interventions
Arm Title
Mycophenolate mofetil standard
Arm Type
Experimental
Arm Description
mycophenolate mofetil 250mg 2# twice per day (BID)
Arm Title
Mycophenolate sodium low
Arm Type
Experimental
Arm Description
mycophenolate mofetil 250mg 1# twice per day (BID)
Intervention Type
Drug
Intervention Name(s)
Mycophenolate mofetil
Intervention Description
mycophenolate mofetil 1# BID-2# BID
Primary Outcome Measure Information:
Title
Change of Composite Index of Sjogren's syndrome
Time Frame
baseline, 28 week
Secondary Outcome Measure Information:
Title
ocular dryness
Description
We will calculate the change of ocular dryness from baseline to week 28 (0mm [very bad] to 100mm [very good])
Time Frame
baseline, 28 week
Title
physician visual analog scale (VAS)
Description
We will calculate the change of physician VAS from baseline to week 28 (0mm [very bad] to 100mm [very good])
Time Frame
baseline, 28 week
Title
Schirmer's test
Description
We will calculate the change of Schirmer's test results from baseline to week 28
Time Frame
baseline, 28 week
Title
Saxon's test
Description
We will calculate the change of Saxon's test results from baseline to week 28
Time Frame
baseline, 28 week
Title
heart rate
Description
resting heart rate
Time Frame
baseline, 28 week
Title
blood pressure
Description
resting blood pressure
Time Frame
baseline, 28 week
Title
leukocyte count
Description
WBC count
Time Frame
baseline, 28 week
Title
Hb level
Description
Hb level
Time Frame
baseline, 28 week
Title
platelet count
Description
platelet count
Time Frame
baseline, 28 week
10. Eligibility
Sex
All
Minimum Age & Unit of Time
20 Years
Maximum Age & Unit of Time
75 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria:
Diagnosis of primary Sjogren's syndrome based on the 2002 American-European Consensus criteria
Aged 20 to 75 years
Stable doses of oral corticosteroids(≦5mg/d) for at least 4 weeks before enrollment
Intolerance or inadequate response to hydroxychloroquine and (pilocarpine or cevimeline), defined as less than 50mm on at least 2 of VAS including:
global assessment : 0mm (very bad) to 100mm (very good)
pain: 0mm (very bad) to 100mm (very good)
fatigue: 0mm (very bad) to 100mm (very good)
xerostomia: 0mm (very bad) to 100mm (very good)
Adequate contraception for patients of childbearing potential
Exclusion Criteria:
Receiving biologics during the 6 previous months or any other immunosuppressant (methotrexate, cyclophosphamide, cyclosporine, azathioprine, mycophenolate mofetil (MMF), mycophenolate sodium, leflunomide, penicillamine) during the previous month
Any one of laboratory abnormalities:
Serum creatinine ≥2 mg/dl
aspartate aminotransferase (AST) or alanine transaminase (ALT) more than 1.5 x upper normal range of the laboratory
Leukopenia (WBC<4000/μl)
Hb ≤ 9 g/dl (5.6 mmol/l) for males and 8.5 g/dl (5.3 mmol/l) for females
Neutrophil less than 1.5 x 109/l
Platelet count less than 150 x 109/l
History of other autoimmune diseases
Use topical cyclosporine eyedrops, antihistamine, anticholinergic, antidepressant, or antipsychotic drug with possible effects on ocular dryness or oral dryness within 1 month
Pregnant or lactating women
Previous or current malignancies adequately controlled less than 5 years, hepatitis B, hepatitis C, HIV infection, tuberculosis, or diabetes
Subjects with serious infections requiring hospitalization within the last 12 months
Subjects with herpes zoster or cytomegalovirus that resolved less than 2 months before enrollment
Subjects who have received any live vaccines within 3 months
Underlying cardiac, pulmonary, metabolic, renal, hepatic, gastrointestinal, haematological or neurological conditions, chronic or latent infectious diseases or immune deficiency which places the patient at an unacceptable risk for participation in the study
History of recurring or chronic infections or underlying conditions which may further predispose patients to serious infection
Subjects who are impaired, incapacitated, or incapable of completing study-related assessments
History of allergy to mycophenolate sodium
Nausea, vomiting, diarrhea within 1 week before enrollment
History of psychosis, seizure, retinopathy
Infection 2 weeks before enrollment
Heart rate < 60/min at rest
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Jeng-Hsien Yen
Organizational Affiliation
Kaohsiung Medical University
Official's Role
Principal Investigator
First Name & Middle Initial & Last Name & Degree
Wen-Chan Tsai
Organizational Affiliation
Kaohsiung Medical University
Official's Role
Study Director
First Name & Middle Initial & Last Name & Degree
Tsan-Teng Ou, MD
Organizational Affiliation
Kaohsiung Medical University
Official's Role
Study Director
First Name & Middle Initial & Last Name & Degree
Cheng-Chin Wu, MD
Organizational Affiliation
Kaohsiung Medical University
Official's Role
Study Director
First Name & Middle Initial & Last Name & Degree
Wan-Yu Sung, MD
Organizational Affiliation
Kaohsiung Medical University
Official's Role
Study Director
First Name & Middle Initial & Last Name & Degree
Chia-Chun Tseng, MD
Organizational Affiliation
Kaohsiung Medical University
Official's Role
Study Director
12. IPD Sharing Statement
Plan to Share IPD
No
IPD Sharing Plan Description
We won't share our data
Citations:
PubMed Identifier
17986340
Citation
Willeke P, Schluter B, Becker H, Schotte H, Domschke W, Gaubitz M. Mycophenolate sodium treatment in patients with primary Sjogren syndrome: a pilot trial. Arthritis Res Ther. 2007;9(6):R115. doi: 10.1186/ar2322.
Results Reference
result
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Efficacy and Safety of Mycophenolate Mofetil in subjectswithSjogren's Syndrome
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