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Note: This record shows only 22 elements of the WHO Trial Registration Data Set. To view changes that have been made to the source record, or for additional information about this trial, click on the URL below to go to the source record in the primary register. |
Register:
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EUCTR |
Last refreshed on:
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25 November 2019 |
Main ID: |
EUCTR2005-002740-26-GB |
Date of registration:
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01/12/2005 |
Prospective Registration:
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Yes |
Primary sponsor: |
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Public title:
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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
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Scientific title:
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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 |
Date of first enrolment:
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21/02/2006 |
Target sample size:
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30 |
Recruitment status: |
Not Recruiting |
URL:
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https://www.clinicaltrialsregister.eu/ctr-search/search?query=eudract_number:2005-002740-26 |
Study type:
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Interventional clinical trial of medicinal product |
Study design:
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Controlled: yes Randomised: yes Open: no Single blind: no Double blind: yes Parallel group: no Cross over: yes Other: no If controlled, specify comparator, Other Medicinial Product: no Placebo: no Other: no
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Phase:
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Human pharmacology (Phase I): no
Therapeutic exploratory (Phase II): yes
Therapeutic confirmatory - (Phase III): no
Therapeutic use (Phase IV): no
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Countries of recruitment
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United Kingdom
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Key inclusion & exclusion criteria
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Inclusion criteria: 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
Exclusion criteria: 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.
Age minimum:
Age maximum:
Gender:
Female: yes Male: yes
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Health Condition(s) or Problem(s) studied
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Myasthenia Gravis
MedDRA version: 8.1
Level: LLT
Classification code 10028417
Term: Myasthenia gravis
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Intervention(s)
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Product Name: MONARSEN Product Code: EN101 Pharmaceutical Form: Oral solution Current Sponsor code: EN101 Other descriptive name: MONARSEN Concentration unit: mg milligram(s) Concentration type: equal Concentration number: 10- Current Sponsor code: EN101 Other descriptive name: MONARSEN Concentration unit: mg milligram(s) Concentration type: equal Concentration number: 20- Current Sponsor code: EN101 Other descriptive name: MONARSEN Concentration unit: mg milligram(s) Concentration type: equal Concentration number: 40-
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Primary Outcome(s)
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Secondary Objective: - To assess the quality of life using the ADL-MG
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Primary end point(s): Efficacy The study is not designed to achieve a certain power to detect effectiveness of the study drug. The efficacy analysis will therefore involve evaluating the degree of success with which the study drug ameliorates symptoms that appear when Monarsen treatment is given.
Safety Changes from the screening period (prior to removal from Mestinon) of vital signs, hematology, clinical chemistry, urinalysis, ECG parameters and physical exam to the end of the study will be examined. Treatment emergent and/or clinically significant changes from normal to abnormal values in key laboratory parameters will be identified.
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Main Objective: - To assess the efficacy of three doses of Monarsen given once daily for one week. Efficacy will be assessed by evaluating changes in the QMG score. - Safety will be assessed by evaluating adverse events and laboratory tests during a 3 week treatment period (dosing on alternative weeks with Mestinon, over 5 weeks) and a 4 week follow up period.
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Source(s) of Monetary Support
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Ethics review
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Status: Approved
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Results
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Results available:
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