A Phase II Double Blind, Cross-Over Study to Compare the Safety and Efficacy of 10, 20 and 40 mg Monarsen (EN101) administered to Patients with Myasthenia Gravis
- Conditions
- Myasthenia GravisMedDRA version: 8.1 Level: LLT Classification code 10028417 Term: Myasthenia gravis
- Registration Number
- EUCTR2005-002740-26-GB
- Lead Sponsor
- Ester Neuroscience Ltd
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- ot Recruiting
- Sex
- Not specified
- Target Recruitment
- 30
To be considered eligible to participate in this study, a patient must meet the inclusion criteria listed below:
(1) Clinical diagnosis of Myasthenia Gravis according to the MGFA (MG Foundation of America) classification being defined as a minimum of class II in whom it is safe to interrupt Mestinon treatment.
(2) Patients (male or female) aged 18 years or above.
(3) Patients must be seropositive for AChR antibodies (greater than 0.5 nM) or seropositive for anti MUSK antibodies. (Levels of anti MUSK greater then 0.05 fMol/L)
(4) Laboratory Test of TSH and T4, within the normal range.
(5) Previously treated with Mestinon (at least 3 tab. a day of 60mg each) until 12-18 hours prior to study initiation, with or without concomitant prednisolone or immunosuppressive treatment, which has been stable for at least 2 months.
(6) Have hepatic and renal function, as well as coagulation parameters as documented by the following laboratory parameters, within local laboratory normal limits:
–AST
–Bilirubin
–Creatinine
–Platelets
–Prothrombin time
–Activated partial thromboplastin time (aPTT)
(7) Women of childbearing potential are excluded
Post-menopausal women (ie women who have been post-menopausal for at least 1 year) and women who have had a hysterectomy can be included.
Men in relationship with women of childbearing potential, must agree to use effective contraceptive methods during the course of the study and for a 3 month follow-up period.
(8) Have ability to understand the requirements of the study, have provided written informed consent, and agree to abide by the study restrictions and to return for the required assessments.
(9) QMG score (while on Mestinon) > 3 points and QMG score while on washout > 3 points greater than score while on Mestinon.
Are the trial subjects under 18? no
Number of subjects for this age range:
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range
F.1.3 Elderly (>=65 years) yes
F.1.3.1 Number of subjects for this age range
To be eligible for entry into the study, the patient must not meet any of the exclusion criteria listed below:
(1) Patients have one of the following symptoms: acute respiratory failure/insufficiency, major difficulty in swallowing, functional disability responsible for the discontinuation of physical activity, within 1 month prior to screening.
(2) Patients have exclusive mild ocular MG.
(3) Body weight >100 kg
(4) Patients in whom disease is so severe that it would be unethical to delay conventional therapy.
(5) Have participated in any experimental protocol within the preceding one month.
(6) Have received an IVIG infusion or plasmapheresis treatment, within 8 weeks prior to study initiation.
(7) Abnormal CBC count (Hgb less than 12 g/dl, WBC less than 4,000/ml, or platelet count less than 130,000/ml).
(8) Have developed other autoimmune disease within one month prior to study initiation.
(9) Have uncontrolled hypertension, severe hepatic or renal disease, or other severe general or psychiatric disease.
(10) Be pregnant or lactating.
Study & Design
- Study Type
- Interventional clinical trial of medicinal product
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method
- Secondary Outcome Measures
Name Time Method