Trial Outcomes & Findings for Safety and Efficacy of Eculizumab in Refractory Generalized Myasthenia Gravis (REGAIN Study) (NCT NCT01997229)

NCT ID: NCT01997229

Last Updated: 2019-07-16

Results Overview

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).

Recruitment status

COMPLETED

Study phase

PHASE3

Target enrollment

125 participants

Primary outcome timeframe

End of study (Week 26)

Results posted on

2019-07-16

Participant Flow

Participant milestones

Participant milestones
Measure	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 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).
Overall Study STARTED	62	63
Overall Study COMPLETED	57	61
Overall Study NOT COMPLETED	5	2

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

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

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Eculizumab n=62 Participants 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 n=63 Participants 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).	Total n=125 Participants Total of all reporting groups
Age, Continuous	47.5 years STANDARD_DEVIATION 15.66 • n=5 Participants	46.9 years STANDARD_DEVIATION 17.98 • n=7 Participants	47.2 years STANDARD_DEVIATION 16.80 • n=5 Participants
Sex: Female, Male Female	41 Participants n=5 Participants	41 Participants n=7 Participants	82 Participants n=5 Participants
Sex: Female, Male Male	21 Participants n=5 Participants	22 Participants n=7 Participants	43 Participants n=5 Participants

PRIMARY outcome

Timeframe: 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).

Outcome measures

Outcome measures
Measure	Eculizumab n=62 Participants 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 n=63 Participants 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).
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)	56.6 scores on a scale Standard Error 4.53	68.3 scores on a scale Standard Error 4.49

Adverse Events

Eculizumab

Serious events: 9 serious events

Other events: 52 other events

Deaths: 0 deaths

Placebo

Serious events: 18 serious events

Other events: 53 other events

Deaths: 0 deaths

Serious adverse events

Other adverse events

Additional Information

Jing-Jing Wang, MD

Alexion Pharmaceuticals, Inc.

Phone: +1 (475) 230-2596

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee Publication rights are tied to the completion of the multi-center publication.
Publication restrictions are in place

Restriction type: OTHER

Measure	Eculizumab n=62 participants at risk 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 n=63 participants at risk 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).
Vascular disorders Deep vein thrombosis	0.00% 0/62 • 26 weeks (Study Period) + 8 weeks (Follow-up Period for subjects who discontinued the Study Period or completed the Study Period but did not enroll in the ECU-MG-302 extension trial)	1.6% 1/63 • 26 weeks (Study Period) + 8 weeks (Follow-up Period for subjects who discontinued the Study Period or completed the Study Period but did not enroll in the ECU-MG-302 extension trial)
Injury, poisoning and procedural complications Intentional overdose	0.00% 0/62 • 26 weeks (Study Period) + 8 weeks (Follow-up Period for subjects who discontinued the Study Period or completed the Study Period but did not enroll in the ECU-MG-302 extension trial)	1.6% 1/63 • 26 weeks (Study Period) + 8 weeks (Follow-up Period for subjects who discontinued the Study Period or completed the Study Period but did not enroll in the ECU-MG-302 extension trial)
Investigations Lymphocyte count decreased	0.00% 0/62 • 26 weeks (Study Period) + 8 weeks (Follow-up Period for subjects who discontinued the Study Period or completed the Study Period but did not enroll in the ECU-MG-302 extension trial)	1.6% 1/63 • 26 weeks (Study Period) + 8 weeks (Follow-up Period for subjects who discontinued the Study Period or completed the Study Period but did not enroll in the ECU-MG-302 extension trial)
Neoplasms benign, malignant and unspecified (incl cysts and polyps) Metastases to bone	1.6% 1/62 • 26 weeks (Study Period) + 8 weeks (Follow-up Period for subjects who discontinued the Study Period or completed the Study Period but did not enroll in the ECU-MG-302 extension trial)	0.00% 0/63 • 26 weeks (Study Period) + 8 weeks (Follow-up Period for subjects who discontinued the Study Period or completed the Study Period but did not enroll in the ECU-MG-302 extension trial)
Neoplasms benign, malignant and unspecified (incl cysts and polyps) Prostate cancer	1.6% 1/62 • 26 weeks (Study Period) + 8 weeks (Follow-up Period for subjects who discontinued the Study Period or completed the Study Period but did not enroll in the ECU-MG-302 extension trial)	0.00% 0/63 • 26 weeks (Study Period) + 8 weeks (Follow-up Period for subjects who discontinued the Study Period or completed the Study Period but did not enroll in the ECU-MG-302 extension trial)
Respiratory, thoracic and mediastinal disorders Apnoea	0.00% 0/62 • 26 weeks (Study Period) + 8 weeks (Follow-up Period for subjects who discontinued the Study Period or completed the Study Period but did not enroll in the ECU-MG-302 extension trial)	1.6% 1/63 • 26 weeks (Study Period) + 8 weeks (Follow-up Period for subjects who discontinued the Study Period or completed the Study Period but did not enroll in the ECU-MG-302 extension trial)
Respiratory, thoracic and mediastinal disorders Pulmonary embolism	0.00% 0/62 • 26 weeks (Study Period) + 8 weeks (Follow-up Period for subjects who discontinued the Study Period or completed the Study Period but did not enroll in the ECU-MG-302 extension trial)	1.6% 1/63 • 26 weeks (Study Period) + 8 weeks (Follow-up Period for subjects who discontinued the Study Period or completed the Study Period but did not enroll in the ECU-MG-302 extension trial)
Blood and lymphatic system disorders Lymphopenia	1.6% 1/62 • 26 weeks (Study Period) + 8 weeks (Follow-up Period for subjects who discontinued the Study Period or completed the Study Period but did not enroll in the ECU-MG-302 extension trial)	0.00% 0/63 • 26 weeks (Study Period) + 8 weeks (Follow-up Period for subjects who discontinued the Study Period or completed the Study Period but did not enroll in the ECU-MG-302 extension trial)
Nervous system disorders Myasthenia gravis	8.1% 5/62 • 26 weeks (Study Period) + 8 weeks (Follow-up Period for subjects who discontinued the Study Period or completed the Study Period but did not enroll in the ECU-MG-302 extension trial)	12.7% 8/63 • 26 weeks (Study Period) + 8 weeks (Follow-up Period for subjects who discontinued the Study Period or completed the Study Period but did not enroll in the ECU-MG-302 extension trial)
Nervous system disorders Myasthenia gravis crisis	1.6% 1/62 • 26 weeks (Study Period) + 8 weeks (Follow-up Period for subjects who discontinued the Study Period or completed the Study Period but did not enroll in the ECU-MG-302 extension trial)	0.00% 0/63 • 26 weeks (Study Period) + 8 weeks (Follow-up Period for subjects who discontinued the Study Period or completed the Study Period but did not enroll in the ECU-MG-302 extension trial)
General disorders General physical health deterioration	0.00% 0/62 • 26 weeks (Study Period) + 8 weeks (Follow-up Period for subjects who discontinued the Study Period or completed the Study Period but did not enroll in the ECU-MG-302 extension trial)	1.6% 1/63 • 26 weeks (Study Period) + 8 weeks (Follow-up Period for subjects who discontinued the Study Period or completed the Study Period but did not enroll in the ECU-MG-302 extension trial)
General disorders Pyrexia	3.2% 2/62 • 26 weeks (Study Period) + 8 weeks (Follow-up Period for subjects who discontinued the Study Period or completed the Study Period but did not enroll in the ECU-MG-302 extension trial)	0.00% 0/63 • 26 weeks (Study Period) + 8 weeks (Follow-up Period for subjects who discontinued the Study Period or completed the Study Period but did not enroll in the ECU-MG-302 extension trial)
Gastrointestinal disorders Gastritis	0.00% 0/62 • 26 weeks (Study Period) + 8 weeks (Follow-up Period for subjects who discontinued the Study Period or completed the Study Period but did not enroll in the ECU-MG-302 extension trial)	1.6% 1/63 • 26 weeks (Study Period) + 8 weeks (Follow-up Period for subjects who discontinued the Study Period or completed the Study Period but did not enroll in the ECU-MG-302 extension trial)
Gastrointestinal disorders Intestinal perforation	1.6% 1/62 • 26 weeks (Study Period) + 8 weeks (Follow-up Period for subjects who discontinued the Study Period or completed the Study Period but did not enroll in the ECU-MG-302 extension trial)	0.00% 0/63 • 26 weeks (Study Period) + 8 weeks (Follow-up Period for subjects who discontinued the Study Period or completed the Study Period but did not enroll in the ECU-MG-302 extension trial)
Hepatobiliary disorders Cholecystitis acute	0.00% 0/62 • 26 weeks (Study Period) + 8 weeks (Follow-up Period for subjects who discontinued the Study Period or completed the Study Period but did not enroll in the ECU-MG-302 extension trial)	1.6% 1/63 • 26 weeks (Study Period) + 8 weeks (Follow-up Period for subjects who discontinued the Study Period or completed the Study Period but did not enroll in the ECU-MG-302 extension trial)
Metabolism and nutrition disorders Hyperglycaemia	0.00% 0/62 • 26 weeks (Study Period) + 8 weeks (Follow-up Period for subjects who discontinued the Study Period or completed the Study Period but did not enroll in the ECU-MG-302 extension trial)	1.6% 1/63 • 26 weeks (Study Period) + 8 weeks (Follow-up Period for subjects who discontinued the Study Period or completed the Study Period but did not enroll in the ECU-MG-302 extension trial)
Infections and infestations Bacteraemia	1.6% 1/62 • 26 weeks (Study Period) + 8 weeks (Follow-up Period for subjects who discontinued the Study Period or completed the Study Period but did not enroll in the ECU-MG-302 extension trial)	0.00% 0/63 • 26 weeks (Study Period) + 8 weeks (Follow-up Period for subjects who discontinued the Study Period or completed the Study Period but did not enroll in the ECU-MG-302 extension trial)
Infections and infestations Diverticulitis	1.6% 1/62 • 26 weeks (Study Period) + 8 weeks (Follow-up Period for subjects who discontinued the Study Period or completed the Study Period but did not enroll in the ECU-MG-302 extension trial)	0.00% 0/63 • 26 weeks (Study Period) + 8 weeks (Follow-up Period for subjects who discontinued the Study Period or completed the Study Period but did not enroll in the ECU-MG-302 extension trial)
Infections and infestations Endocarditis	1.6% 1/62 • 26 weeks (Study Period) + 8 weeks (Follow-up Period for subjects who discontinued the Study Period or completed the Study Period but did not enroll in the ECU-MG-302 extension trial)	0.00% 0/63 • 26 weeks (Study Period) + 8 weeks (Follow-up Period for subjects who discontinued the Study Period or completed the Study Period but did not enroll in the ECU-MG-302 extension trial)
Infections and infestations Gastroenteritis	0.00% 0/62 • 26 weeks (Study Period) + 8 weeks (Follow-up Period for subjects who discontinued the Study Period or completed the Study Period but did not enroll in the ECU-MG-302 extension trial)	1.6% 1/63 • 26 weeks (Study Period) + 8 weeks (Follow-up Period for subjects who discontinued the Study Period or completed the Study Period but did not enroll in the ECU-MG-302 extension trial)
Infections and infestations Tonsillitis	0.00% 0/62 • 26 weeks (Study Period) + 8 weeks (Follow-up Period for subjects who discontinued the Study Period or completed the Study Period but did not enroll in the ECU-MG-302 extension trial)	1.6% 1/63 • 26 weeks (Study Period) + 8 weeks (Follow-up Period for subjects who discontinued the Study Period or completed the Study Period but did not enroll in the ECU-MG-302 extension trial)
Infections and infestations Upper respiratory tract infection	0.00% 0/62 • 26 weeks (Study Period) + 8 weeks (Follow-up Period for subjects who discontinued the Study Period or completed the Study Period but did not enroll in the ECU-MG-302 extension trial)	3.2% 2/63 • 26 weeks (Study Period) + 8 weeks (Follow-up Period for subjects who discontinued the Study Period or completed the Study Period but did not enroll in the ECU-MG-302 extension trial)
Infections and infestations Urinary tract infection bacterial	0.00% 0/62 • 26 weeks (Study Period) + 8 weeks (Follow-up Period for subjects who discontinued the Study Period or completed the Study Period but did not enroll in the ECU-MG-302 extension trial)	1.6% 1/63 • 26 weeks (Study Period) + 8 weeks (Follow-up Period for subjects who discontinued the Study Period or completed the Study Period but did not enroll in the ECU-MG-302 extension trial)
Infections and infestations Varicella	0.00% 0/62 • 26 weeks (Study Period) + 8 weeks (Follow-up Period for subjects who discontinued the Study Period or completed the Study Period but did not enroll in the ECU-MG-302 extension trial)	1.6% 1/63 • 26 weeks (Study Period) + 8 weeks (Follow-up Period for subjects who discontinued the Study Period or completed the Study Period but did not enroll in the ECU-MG-302 extension trial)