Safety and Efficacy of Eculizumab in Refractory Generalized Myasthenia Gravis (REGAIN Study)

• Register: ClinicalTrials.gov
• Last refreshed on: 12 December 2020
• Main ID: NCT01997229
• Date of registration: 18/11/2013
• Prospective Registration: Yes
• Primary sponsor: Alexion Pharmaceuticals
• Public title: Safety and Efficacy of Eculizumab in Refractory Generalized Myasthenia Gravis (REGAIN Study)
• Scientific title: A Randomized, Double-Blind, Placebo-Controlled, Multi-Center Study to Evaluate the Safety and Efficacy of Eculizumab in Subjects With Refractory Generalized Myasthenia Gravis (gMG)
• Date of first enrolment: December 2013
• Target sample size: 125
• Recruitment status: Completed
• URL: https://clinicaltrials.gov/show/NCT01997229
• Study type: Interventional
• Study design: Allocation: Randomized. Intervention model: Parallel Assignment. Primary purpose: Treatment. Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor).
• Phase: Phase 3

Countries of recruitment

Argentina, Australia, Belgium, Brazil, Canada, Czech Republic, Czechia, Denmark, Finland, France, Germany, Greece, Hungary, Italy, Japan, Korea, Republic of, Netherlands, Spain, Sweden, Turkey, United Kingdom, United States

Key inclusion & exclusion criteria

Key Inclusion Criteria:

- Male or female patients =18 years old

- Diagnosis of MG made by the following tests:

1. Positive serologic test for anti-AChR Abs as confirmed at screening, and

2. One of the following:

1. History of abnormal neuromuscular transmission test demonstrated by
single-fiber electromyography (SFEMG) or repetitive nerve stimulation, or

2. History of positive anticholinesterase test, e.g. edrophonium chloride test,
or

3. Subject has demonstrated improvement in MG signs on oral cholinesterase
inhibitors, as assessed by the treating physician.

- MGFA Clinical Classification Class II to IV at screening.

- MG-ADL total score must be =6 at screening and Randomization (Day 1).

- Subjects who have:

1. Failed treatment with at least two immunosuppressive agents. Or,

2. Failed treatment with at least one immunosuppressive agent and require chronic
plasma exchange or IVIg

Key Exclusion Criteria:

- History of thymoma or other neoplasms of the thymus

- History of thymectomy within 12 months prior to screening

- MGFA Class I or MG crisis at screening (MGFA Class V)

- Use of rituximab within 6 months prior to screening

- Use of IVIg or PE within 4 weeks prior to Randomization (Day 1)



Age minimum: 18 Years
Age maximum: N/A
Gender: All

Health Condition(s) or Problem(s) studied

Refractory Generalized Myasthenia Gravis

Intervention(s)

Biological: Eculizumab

Drug: Placebo

Primary Outcome(s)

Myasthenia Gravis Activities of Daily Living Profile (MG-ADL): Change From Baseline in MG-ADL Total Score at Week 26 by Worst-Rank Analysis of Covariance (ANCOVA) [Time Frame: End of study (Week 26)]

Secondary ID(s)

ECU-MG-301

2013-003589-15

Source(s) of Monetary Support

Please refer to primary and secondary sponsors

Results

• Results available: Yes
• Date Posted: 07/02/2018
• URL: https://clinicaltrials.gov/ct2/show/results/NCT01997229