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

Alexion Pharmaceuticals

Status and phase: Completed; Phase 3

Conditions: Refractory Generalized Myasthenia Gravis

Treatments: Biological: Eculizumab; Drug: Placebo

Study type: Interventional

Funder types: Industry

Identifiers

NCT01997229

2013-003589-15 (EudraCT Number)

ECU-MG-301

Details and patient eligibility

About

The purpose of this study is to determine if eculizumab is safe and effective for the treatment of refractory generalized Myasthenia Gravis.

Enrollment: 125 patients

Sex: All

Ages: 18+ years old

Volunteers: No Healthy Volunteers

Inclusion and 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)

Trial design

Primary purpose: Treatment

Allocation: Randomized

Interventional model: Parallel Assignment

Masking: Quadruple Blind

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125 participants in 2 patient groups, including a placebo group

Eculizumab

Experimental group

Description:

Biological/Vaccine: Eculizumab; Induction phase: 3 vials of study drug (equivalent to 900 mg of eculizumab) weekly for 4 doses (every 7 days ± 2 days) followed by 4 vials of study drug (equivalent to 1200 mg of eculizumab) 1 week later for the fifth dose (Week 4); Maintenance phase: 4 vials of study drug (equivalent to 1200 mg of eculizumab) every 2 weeks (14 days ± 2 days) from the fifth dose onwards (Week 6 through Week 26).

Treatment:

Biological: Eculizumab

Placebo

Placebo Comparator group

Description:

Placebo contains the same buffer components without the active ingredient; Induction phase: 3 vials of study drug (placebo) weekly for 4 doses (every 7 days ± 2 days) followed by 4 vials of study drug (placebo) 1 week later for the fifth dose (Week 4); Maintenance phase: 4 vials of study drug (placebo) every 2 weeks (14 days ± 2 days) from the fifth dose onwards (Week 6 through Week 26).

Treatment:

Drug: Placebo