Mycophenolate Sodium (Myfortic®) in the Treatment of Corticosteroid-refractory Autoimmune Uveitis:Pilot Study

• ClinicalTrials.gov Identifier: NCT01261169
• Recruitment Status: Unknown
• First Posted: December 16, 2010
• Last Update Posted: December 16, 2010
• Sponsor: Genovate Biotechnology Co., Ltd.,
• Collaborator: Novartis
• Information provided by: Genovate Biotechnology Co., Ltd.,

Tracking Information

• First Submitted Date: December 14, 2010
• First Posted Date: December 16, 2010
• Last Update Posted Date: December 16, 2010
• Study Start Date: January 2009
• Primary Completion Date: Not Provided
• Current Primary Outcome Measures: Not Provided
• Original Primary Outcome Measures: Not Provided
• Change History: No Changes Posted
• Current Secondary Outcome Measures: Not Provided
• Original Secondary Outcome Measures: Not Provided
• Current Other Pre-specified Outcome Measures: Not Provided
• Original Other Pre-specified Outcome Measures: Not Provided

Descriptive Information

• Brief Title: Mycophenolate Sodium (Myfortic®) in the Treatment of Corticosteroid-refractory Autoimmune Uveitis:Pilot Study
• Official Title: Mycophenolate Sodium (Myfortic®) in the Treatment of Corticosteroid-refractory Autoimmune Uveitis:Pilot Study
• Brief Summary: This study is designed to explore the use of Myfortic® in patients with steroid-refractory uveitis. The aim of the study will be to show the therapeutic effect of Myfortic® in managing uveitis patients.

Detailed Description

This study is a single arm prospective evaluation in which patients with uveitis are treated with Myfortic® in a dose of 360 mg bid for six months. From day 0, Myfortic will be combined with 1 mg/kg prednisone. Prednisone will be tapered from week 1 based on the clinical findings of the ophthalmologist.

There will be no other stratification of patients. After the screening, patients will be treated with Myfortic® for 6 months. Patients will be seen at month 1, 3, and 6. Thereafter patients will be followed up at month 12. Efficacy and safety assessments will be performed at each visit.

• Study Type: Observational
• Study Design: Observational Model: Cohort; Time Perspective: Prospective
• Target Follow-Up Duration: Not Provided
• Biospecimen: Not Provided
• Sampling Method: Probability Sample
• Study Population: About 40 patients with uveitis
• Condition: Uveitis

Intervention

Drug: Myfortic

Myfortic in uveitis

Study Groups/Cohorts

Myfortic

Intervention: Drug: Myfortic

• Publications *: Not Provided

Recruitment Information

• Recruitment Status: Unknown status
• Estimated Enrollment (submitted: December 14, 2010): 40
• Original Estimated Enrollment: Same as current
• Estimated Study Completion Date: December 2011
• Primary Completion Date: Not Provided

Eligibility Criteria

Inclusion Criteria:

1. Men or women ≥ 18 years of age with corticosteroid-refractory uveitis due to one of the inflammatory conditions listed below:

   1. Ocular sarcoidosis: a granulomatous uveitis with posterior inflammation in a patient in which sarcoidosis has been established by means of a biopsy, cytology, or a positive scan.
   2. Intermediate uveitis: patients with clinical features as defined by the IUSG23. However, it is important that the patient has no evidence of a systemic infection such as Lyme Borreliosis and that there is no history of neurological symptoms likely to be associated with multiple sclerosis. All patients with intermediate uveitis should have a negative MRI with gadolinium contrast, prior to being enrolled.
   3. Behçet's syndrome: the international classification criteria of the International Society for Behçet's Disease (ISBD) will be used to define these patients.
   4. Idiopathic Retinal Vasculitis where systemic or infectious causes have been eliminated. In particular patients will not have evidence of Wegener's granulomatosis, SLE, PAN, polymyositis, dermatomyositis or other systemic vasculitic disorders. Patients must also lack evidence for a systemic infection, and have been adequately screened. For the purpose of this study, Eale's disease will be excluded from the idiopathic retinal vasculitis group. However, both non-occlusive and occlusive types of retinal vasculitis can be included in the study.
   5. Vogt-Koyanagi-Harada disease (VKH) as defined according to the international Workgroup definition of VKH.
   6. Sympathetic ophthalmia: a granulomatous uveitis involving the choroid and retina, characterized by multiple white-yellow lesions often in the periphery, which can coalesce particularly in the circumpapillary region. It is associated with trauma or multiple prior surgeries.
   7. Idiopathic panuveitis: all non-infectious panuveititides that can not be related to the diseases mentioned above.
   8. Corticosteroid-refractory uveitis patient is defined as the patient who receive steroid treatment and cannot lower the dosage of steroid to less than 10mg per day in 3 months due to the clinical condition.
2. Disease that is 5 years or less in duration, with a significant flare in the past 24 months requiring intensification of anti-inflammatory therapy (predsinolone). A significant flare is defined as a drop of 2 lines of vision on an ETDRS chart or equivalent, or increase in vitreous flare by 2 grades.
3. Visual acuity of 0.1 or better in at least one eye.
4. Men and women of childbearing potential must use adequate birth control measures (e.g., abstinence, oral contraceptives, intrauterine device, barrier method with spermicidal cream, or surgical sterilization) for the duration of the study and should continue such precautions for 6 weeks after receiving the last administration.

5. The screening laboratory test results must meet the following criteria:

   1. Hemoglobin ≥ 10.5 g/dL
   2. WBC ≥ 3.0 x 103/μL
   3. Neutrophils ≥ 1.5 x 103/μL
   4. Platelets ≥ 1 x 103/μL
   5. SGOT (AST) and alkaline phosphatase levels must be within 3 times the upper limit of normal range for the laboratory conducting the test (3xULN).
   6. Creatinine clearance > 25 ml/min
6. Must have a normal chest radiograph within 3 months prior to first therapy with no evidence of malignancy, infection or TB.
7. Patient must be able to adhere to the study visit schedule and other protocol requirements.
8. The patient must be capable of giving informed consent and the consent must be obtained prior to any screening procedures.

Exclusion Criteria:

1. Inability to visualize the fundus due to corneal or lenticular opacities.
2. Patients requiring ocular surgery within the initial 3 months of treatment, or who have had surgery in the prior 3 months.
3. Women who are pregnant, nursing, or planning pregnancy within 6 months after screening (i.e., approximately 6 weeks following last treatment).
4. Use of any investigational drug within 1 month prior to screening or within 5 half-lives of the investigational agent, whichever is longer.
5. Recent history of systemic immunosuppressive therapy, other than steroids for ocular disease within 2 months.
6. Creatinine clearance of < 25ml/min or serum creatinine level higher than 1.5 mg/dl
7. Patients with known hypersensitivity to prednisone, Myfortic® or drugs with similar chemical structures.
8. Patients with any clinically significant infection.
9. Documented HIV infection.
10. Patients with active TB or evidence of latent TB.
11. Patients with positive Lues serology and or significant Lues infection.
12. Patients with opportunistic infections, including but not limited to evidence of active cytomegalovirus, active Pneumocystis carinii, Aspergillosis, Histoplasmosis or atypical mycobacterium infection, etc, within the previous 6 months.
13. Current signs or symptoms of severe, progressive or uncontrolled renal, hepatic,hematologic, gastrointestinal, endocrine, pulmonary, cardiac, neurologic, or cerebral disease.
14. Presence of a transplanted organ (with the exception of a corneal transplant 3 months prior to screening).
15. Malignancy within the past 5 years (except for treated squamous or basal cell carcinoma of the skin without evidence of recurrence).
16. History of lymphoproliferative disease including lymphoma, or signs and symptoms suggestive of possible lymphoproliferative disease, such as lymphadenopathy of unusual size or location (such as nodes in the posterior triangle of the neck, infra-clavicular, epitrochlear, or periaortic areas), or splenomegaly.
17. Known recent substance abuse (drugs or alcohol).
18. Poor tolerability of venipuncture or lack of adequate venous access for required blood sampling during the study period.
19. Recent live vaccinations

Sex/Gender

• Sexes Eligible for Study: All

• Ages: 18 Years and older (Adult, Older Adult)
• Accepts Healthy Volunteers: No
• Contacts: Contact information is only displayed when the study is recruiting subjects
• Listed Location Countries: Taiwan

Administrative Information

• NCT Number: NCT01261169
• Other Study ID Numbers: CERL080ATW06T
• Has Data Monitoring Committee: No
• U.S. FDA-regulated Product: Not Provided
• IPD Sharing Statement: Not Provided
• Responsible Party: Yih-Shiou Hwang
• Study Sponsor: Genovate Biotechnology Co., Ltd.,
• Collaborators: Novartis

Investigators

• Principal Investigator: Hwang Yih Shiou, MD; CGMH

• PRS Account: Genovate Biotechnology Co., Ltd.,
• Verification Date: December 2010