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

Phase 3

Completed

• Conditions: Refractory Generalized Myasthenia Gravis
• Interventions: Biological: Eculizumab; Drug: Placebo

• Registration Number: NCT01997229
• Lead Sponsor: Alexion Pharmaceuticals, Inc.

Brief Summary

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

Detailed Description

Not available

Study Timeline

• Study First Submitted: 2013-11-18
• Study First Submitted QC: 2013-11-22
• Study First Posted: 2013-11-28
• Study Start: 2013-12
• Primary Completion: 2016-02
• Study Completion: 2016-06
• Disposition First Submitted: 2017-03-01
• Disposition First Submitted QC: 2017-03-01
• Disposition First Posted: 2017-03-03
• Results First Submitted: 2017-11-21
• Results First Submitted QC: 2018-01-12
• Results First Posted: 2018-02-07
• Status Verified: 2019-07
• Last Update Submitted: 2019-07-12
• Last Update Posted: 2019-07-16

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 125

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)

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: PARALLEL

Arm && Interventions

Group	Intervention	Description
Eculizumab	Eculizumab	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).
Placebo	Placebo	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).

Primary Outcome Measures

Name	Time	Method
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)	End of study (Week 26)	In the Worst-Rank analysis, the 125 total patients were ranked from best outcome (rank/score of 1) to worst outcome (rank/score of 125).

Trial Locations

Locations (114)

University of Alabama; 🇺🇸 Birmingham, Alabama, United States

University of California San Francisco-Fresno; 🇺🇸 Fresno, California, United States

University of Southern California; 🇺🇸 Los Angeles, California, United States

University of California-Irvine; 🇺🇸 Orange, California, United States

University of California Davis Health System; 🇺🇸 Sacramento, California, United States

California Pacific Medical Center; 🇺🇸 San Francisco, California, United States

Stanford University School of Medicine; 🇺🇸 Stanford, California, United States

Yale New Haven Hospital; 🇺🇸 New Haven, Connecticut, United States

University of Florida Health Science Center; 🇺🇸 Jacksonville, Florida, United States

University of Miami School of Medicine; 🇺🇸 Miami, Florida, United States

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