EXTENSION STUDY OF ECU-MG-301 TO EVALUATE ECULIZUMAB IN REFRACTORY GENERALIZED MYASTHENIA GRAVIS (GMG) SUBJECTS

• Register: EUCTR
• Last refreshed on: 30 April 2019
• Main ID: EUCTR2013-002191-41-FI
• Date of registration: 25/09/2015
• Prospective Registration: Yes
• Primary sponsor: Alexion Pharmaceuticals, Inc.
• Public title: EXTENSION STUDY OF ECU-MG-301 TO EVALUATE ECULIZUMAB IN REFRACTORY GENERALIZED MYASTHENIA GRAVIS (GMG) SUBJECTS
• Scientific title: A PHASE III, OPEN-LABEL, EXTENSION TRIAL OF ECU-MG-301 TO EVALUATE THE SAFETY AND EFFICACY OF ECULIZUMAB IN SUBJECTS WITH REFRACTORY GENERALIZED MYASTHENIA GRAVIS (gMG)
• Date of first enrolment: 01/10/2015
• Target sample size: 92
• Recruitment status: Not Recruiting
• URL: https://www.clinicaltrialsregister.eu/ctr-search/search?query=eudract_number:2013-002191-41
• Study type: Interventional clinical trial of medicinal product
• Study design: Controlled: no; Randomised: no; Open: no; Single blind: no; Double blind: no; Parallel group: no; Cross over: no; Other: yes; Other trial design description: induction blinded phase followed by an open label maintenance phase; If controlled, specify comparator, Other Medicinial Product: no; Placebo: no; Other: no; Number of treatment arms in the trial: 1
• Phase: Human pharmacology (Phase I): no Therapeutic exploratory (Phase II): no Therapeutic confirmatory - (Phase III): yes Therapeutic use (Phase IV): no

Countries of recruitment

Argentina, Australia, Austria, Belgium, Brazil, Canada, Czech Republic, Denmark, Finland, France, Germany, Greece, Hungary, Ireland, Israel, Italy, Japan, Korea, Republic of, Netherlands, Norway, Russian Federation, Spain, Sweden, Switzerland, Turkey, United Kingdom

Contacts

• Name: European Clinical Trial Information
• Affiliation: ALEXION EUROPE SAS

• Name: European Clinical Trial Information
• Affiliation: ALEXION EUROPE SAS

Key inclusion & exclusion criteria

Inclusion criteria:
1. Subject has completed the ECU-MG-301 trial.
2. Subject has given written informed consent.
3. Subject is willing and able to comply with the protocol requirements
for the duration of the trial.
4. Female subjects of child-bearing potential must have a negative
pregnancy test (serum human chorionic gonadotropin [HCG]). All
subjects must practice an effective, reliable and medically approved
contraceptive regimen during the trial and for up to 5 months following
discontinuation of treatment.
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 83
F.1.3 Elderly (>=65 years) yes
F.1.3.1 Number of subjects for this age range 9

Exclusion criteria:
1. Subjects who withdrew from the ECU-MG-301 trial as a result of an AE
related to trial drug.
2. Female subjects who are pregnant, breastfeeding or intend to
conceive during the course of the trial.
3. Any medical condition or circumstances that, in the opinion of the
Investigator, might interfere with the subject's participation in the trial,
pose any added risk for the subject, or confound the assessment of the
subjects.


Age minimum:
Age maximum:
Gender:
Female: yes
Male: yes

Health Condition(s) or Problem(s) studied

Therapeutic area: Diseases [C] - Nervous System Diseases [C10]

Generalized Myasthenia Gravis
MedDRA version: 20.0 Level: HLT Classification code 10071942 Term: Myasthenia gravis and related conditions System Organ Class: 100000004859

Intervention(s)

Trade Name: SOLIRIS
Product Name: Soliris
Pharmaceutical Form: Concentrate for solution for infusion
INN or Proposed INN: ECULIZUMAB
CAS Number: 219685-50-4
Current Sponsor code: h5G1.1-mAb
Concentration unit: mg/ml milligram(s)/millilitre
Concentration type: equal
Concentration number: 10-
Pharmaceutical form of the placebo: Concentrate for solution for infusion
Route of administration of the placebo: Intravenous use

Primary Outcome(s)

Main Objective: To evaluate the long-term safety of eculizumab in subjects with
refractory gMG.

Secondary Objective: - To evaluate the long-term efficacy of eculizumab in subjects with
refractory gMG as measured by the improvement or maintenance of the
MG-specific Activities of Daily Living profile (MG-ADL).
- To evaluate the long-term efficacy of eculizumab by additional
efficacy measures including: Quantitative Myasthenia Gravis (QMG)
score, Myasthenia Gravis Composite (MGC) score, and Improvement or
maintenance in primary symptoms that are most clinically meaningful to
the subjects.
- To characterize the effect of eculizumab on quality of life measures.
- To describe the pharmacokinetics (PK) and pharmacodynamics (PD) of
eculizumab in subjects with gMG.

Primary end point(s): Change from baseline in the MG-ADL total score.

Timepoint(s) of evaluation of this end point: From week 1 to 182

Secondary Outcome(s)

Secondary end point(s): 1. Change from baseline in QMG total score.
2. Proportion of subjects with at least a 3-point reduction in the MG-ADL
total score from baseline and with no rescue therapy.
3. Proportion of subjects with at least a 5-point reduction in the QMG
total score from baseline and with no rescue therapy.
4. Change from baseline in the MGC scale total score.
5. Change from baseline in MG-QOL-15.

Timepoint(s) of evaluation of this end point: From week 1 to 182

Secondary ID(s)

ECU-MG-302

2013-002191-41-GB

NCT02301624

Source(s) of Monetary Support

ALEXION PHARMACEUTICALS INC

Ethics review

Status: Approved
Approval date:
Contact: