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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: EUCTR
Last refreshed on: 25 November 2019
Main ID:  EUCTR2005-002740-26-GB
Date of registration: 01/12/2005
Prospective Registration: Yes
Primary sponsor: Ester Neuroscience Ltd
Public title: 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
Scientific title: 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: 21/02/2006
Target sample size: 30
Recruitment status: Not Recruiting
URL:  https://www.clinicaltrialsregister.eu/ctr-search/search?query=eudract_number:2005-002740-26
Study type:  Interventional clinical trial of medicinal product
Study design: 
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
 
Phase:  Human pharmacology (Phase I): no Therapeutic exploratory (Phase II): yes Therapeutic confirmatory - (Phase III): no Therapeutic use (Phase IV): no
Countries of recruitment
United Kingdom
Contacts
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Key inclusion & exclusion criteria
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
Health Condition(s) or Problem(s) studied
Myasthenia Gravis
MedDRA version: 8.1 Level: LLT Classification code 10028417 Term: Myasthenia gravis
Intervention(s)

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-

Primary Outcome(s)
Secondary Objective: - To assess the quality of life using the ADL-MG

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.

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.
Secondary Outcome(s)
Secondary ID(s)
MG-02
Source(s) of Monetary Support
Secondary Sponsor(s)
Ethics review
Status: Approved
Approval date:
Contact:
Results
Results available:
Date Posted:
Date Completed:
URL:
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