Safety and Efficacy Study of Eculizumab in Patients With Refractory Generalized Myasthenia Gravis

• Register: ClinicalTrials.gov
• Last refreshed on: 12 December 2020
• Main ID: NCT00727194
• Date of registration: 30/07/2008
• Prospective Registration: Yes
• Primary sponsor: Alexion Pharmaceuticals
• Public title: Safety and Efficacy Study of Eculizumab in Patients With Refractory Generalized Myasthenia Gravis
• Scientific title: A Randomized, Double-Blind, Placebo-Controlled, Cross-Over, Multi-Center Study of Eculizumab in Patients With Generalized Myasthenia Gravis (gMG) Who Have Moderate to Severe Muscle Weakness Despite Treatment With Immunosuppressants
• Date of first enrolment: October 2008
• Target sample size: 14
• Recruitment status: Terminated
• URL: https://clinicaltrials.gov/show/NCT00727194
• Study type: Interventional
• Study design: Allocation: Randomized. Intervention model: Crossover Assignment. Primary purpose: Treatment. Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor).
• Phase: Phase 2

Countries of recruitment

Canada, United Kingdom, United States

Key inclusion & exclusion criteria

Inclusion Criteria:

- Generalized MG

- MGFA Clinical Classification Class II, III or IVa.

- QMG total score =12

- Minimum score of two (2) in four (4) or more test items in the QMG

- Able to give informed consent.

- Have failed at least two immunosuppressants after one year of treatment

- A positive serologic test for binding anti-acetylcholine receptor Abs at Screening and
one of the following a) history of abnormal neuromuscular transmission test
demonstrated by single-fiber electromyography or repetitive nerve stimulation, or b)
history of positive anticholinesterase test, eg, edrophonium chloride test, or c)
patient has demonstrated improvement in MG signs on acetylcholinesterase inhibitors as
assessed by treating physician.

Exclusion Criteria:

- History of thymoma or other neoplasms of the thymus.

- History of thymectomy within 12 months prior to screening.

- Pregnancy or lactation

- Current or chronic use of plasmapheresis/plasma exchange

- IVIG treatment within 8 weeks prior to screening.

- Use of etanercept within 2 months prior to screening.

- Use of rituximab (RITUXAN®) within 6 months prior to screening.

- MGFA Class I, IVb, and V

- Crisis or impending crisis



Age minimum: 18 Years
Age maximum: 80 Years
Gender: All

Health Condition(s) or Problem(s) studied

Myasthenia Gravis

Intervention(s)

Drug: eculizumab

Drug: Placebo

Primary Outcome(s)

Quantitative Myasthenia Gravis (QMG): The Primary Efficacy Endpoint in This Study Was the Percentage of Patients With a 3-point Reduction From Baseline in the QMG Total Score for Disease Severity. [Time Frame: 16 weeks]

Secondary Outcome(s)

Change From Baseline in the MGFA Post-Intervention Status (PIS) [Time Frame: 16 weeks]

Change From Baseline to the End of Treatment (16 Weeks) in the Two Most Affected QMG Items for Disease Severity (Individual Test Item: Double Vision) [Time Frame: 16 weeks]

Change From Baseline in the QoL Instrument, SF-36. [Time Frame: 16 weeks]

Mean Change From Baseline in QMG Total Score [Time Frame: 16 weeks]

Change From Baseline in Respiratory Function Tests to Characterize the Degree of Involvement of Respiratory Muscles. [Time Frame: 16 weeks]

Change From Baseline to the End of Treatment (16 Weeks) in the Two Most Affected QMG Items for Disease Severity (Individual Test Item: Ptosis) [Time Frame: 16 weeks]

Change From Baseline in the MG-Activity of Daily Living Profile (MG-ADL) [Time Frame: 16 weeks]

Secondary ID(s)

C08-001

Source(s) of Monetary Support

Please refer to primary and secondary sponsors

Results

• Results available: Yes
• Date Posted: 12/12/2016
• URL: https://clinicaltrials.gov/ct2/show/results/NCT00727194