Trial of Mycophenolic Acid Versus Azathioprine in the Treatment of Corticosteroid-refractory Myasthenia Gravis

Myasthenia Gravis Clinical Trial

— Myfortic  

Official title:

Randomized, Double-blind, Double-dummy Trial of Mycophenolic Acid Versus Azathioprine in the Treatment of Corticosteroid-refractory Myasthenia Gravis

Clinical Trial Details — Status: Active, not recruiting

Administrative data

• NCT number: NCT00997412
• Other study ID #: CERL080ATW07T
• Status: Active, not recruiting
• Phase: N/A
• First received: October 16, 2009
• Last updated: October 16, 2009
• Start date: May 2009
• Est. completion date: May 2011

Study information

• Verified date: October 2009
• Source: Qualitix Clinical Research Co., Ltd.
• Contact: n/a
• Is FDA regulated: No
• Health authority: Taiwan: Institutional Review Board
• Study type: Interventional

Clinical Trial Summary

This is an randomized, double-blind, double-dummy trial, and the objective is to compare the efficacy and safety of Mycophenolic acid (MA) and Azathioprine (AZA), immunosuppressive drugs, in myasthenia gravis patients. This prospective study will enroll 40 myasthenia gravis (MG) patients who are poor controlled under prior steroid therapy. All subjects should be randomly assigned to MA group and AZA group that will receive routine pyridostigmine and prednisolone in combination with MA or AZA.

Description:

This will be a double-dummy study to keep the blinded quality.

• MA group: 1 tablet AZA placebo and 4 tables MA (180 mg/tab,720 mg/day) twice daily.

• AZA group: 1 tablet AZA (50mg/tab) and 4 tables MA placebo twice daily.

• When patients achieve minimal manifestation (MM, i.e. complete remission), which lead to normal daily routine, the dose of pyridostigmine should reduce to 240 mg/day (4 tablets) or less. The dose of steroid should be stepped down by 10 mg qod (every other day) for every 2 weeks until the dose achieves 40 mg qod. After that, the dose should be stepped down by 5 mg qod for every month.

• When disease progresses and is no longer maintaining minimal manifestation, the dose of steroid will be stepped up by 10 mg qod for every 2 weeks until achieve clinical stable remission. The taper rule of steroid could start again 1 month after stabilization.

• Every patient will be treated for 1 year. If the patient could not achieve MM within 1 year, the blind of individual patient will be opened and the patients will be crossed over to another medical treatment. The efficacy and safety of second medication will be observed openly until the end of study.

• When the muscle weakness worsens under established study schedule, plasmapheresis could be conducted to improve the condition rapidly.

Recruitment information / eligibility

• Status: Active, not recruiting
• Enrollment: 40
• Est. completion date: May 2011
• Est. primary completion date: May 2011
• Accepts healthy volunteers: No
• Gender: Both
• Age group: 20 Years to 70 Years

Eligibility

Inclusion Criteria:

• Male or female age between 20-70 (including 20 and 70 years old).

• Osserman II and III Myasthenia Gravis.

• Positive serum anti-acetylcholine receptor antibodies.

• Poor control of disease with daily dose of prednisone = 30 mg or 0.5 mg/kg at 3 months before enrollment.

• Without immunosuppressive therapy other than steroid.

Exclusion Criteria:

• Ocular MG or minimal clinical syndrome that would not require the therapy of steroids.

• Negative serum anti-acetylcholine receptor antibodies.

• Use immunosuppressants other than steroids in the preceding year.

• Previous use other investigational medication within 3 months or current participate other clinical study.

• Poor renal function: serum creatinine > 3.0 mg/dl or estimated creatinine clearance < 30 ml/min

• Females who are pregnancy or breast-feeding.

• Recent history, within 5 years, of malignancy

• Unwilling or unable to participate the necessary continuous visits and examinations.

Study Design

Allocation: Randomized, Endpoint Classification: Safety/Efficacy Study, Intervention Model: Crossover Assignment, Masking: Double Blind (Subject, Investigator), Primary Purpose: Treatment

Related Conditions & MeSH terms

• Myasthenia Gravis

Intervention

Drug:
• Mycophenolic acid
180 mg/tablet, 4 tablets twice daily
• AZA
1 tablet AZA (50 mg/tab) and 4 tablets MA placebo twice daily

Locations

Country	Name	City	State
n/a

Sponsors (1)

Lead Sponsor	Collaborator
Qualitix Clinical Research Co., Ltd.

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	The ratio of two arms patients achieve minimal manifestation (MM, i.e. complete remission)		One year after treatment	Yes
Secondary	Osserman clinical classification		One year after treatment	Yes
Secondary	Myasthenia gravis (MG) score		One year after treatment	Yes