Trial · NCT01997229

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

NCT01997229, Phase 3, Completed, Alexion Pharmaceuticals, Inc.; results first posted 2018-02-07

3 sourcestrials-2026-11-12-0.5.0

Cite this record

ConductScience Library. Safety and Efficacy of Eculizumab in Refractory Generalized Myasthenia Gravis (REGAIN Study). Trials, record NCT01997229. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT01997229

On this page

Trial

As registered on ClinicalTrials.gov, in the graph's capture of the registry.

Title: Safety and Efficacy of Eculizumab in Refractory Generalized Myasthenia Gravis (REGAIN Study)
NCT number: NCT01997229
Status: Completed
Phases: Phase 3
Enrollment: 125 participants, actual
Lead sponsor: Alexion Pharmaceuticals, Inc.
Conditions: Refractory Generalized Myasthenia Gravis
Interventions: Eculizumab, Placebo
First posted: 2013-11-28
Last updated: 2019-07-16
Other registries' IDs: eudract:2013-003589-15
Results first posted: 2018-02-07

Source ClinicalTrials.gov

Registered outcomes1

Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.

List	Outcome	Time frame	Description
Primary	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).

Source ClinicalTrials.gov

Outcomes1

Each outcome the trial posted results for, in ClinicalTrials.gov's words.

Type	Outcome	Time frame	Population	Units	Measured as
PRIMARY	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)		scores on a scale	LEAST_SQUARES_MEAN

Source ClinicalTrials.gov

Results by group2

Each measurement the trial posted, group by group, in the words and numbers it posted.

Outcome	Group	Category	Value	Spread	Units
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)	Eculizumab		56.6	Standard Error 4.53	scores on a scale
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)	Placebo		68.3	Standard Error 4.49	scores on a scale

Source ClinicalTrials.gov

Participant flow6

How many started, completed or left each group, period by period.

Period	Milestone	Group	Participants
Overall Study	STARTED	Eculizumab	62
Overall Study	STARTED	Placebo	63
Overall Study	COMPLETED	Eculizumab	57
Overall Study	COMPLETED	Placebo	61
Overall Study	NOT COMPLETED	Eculizumab	5
Overall Study	NOT COMPLETED	Placebo	2

Source ClinicalTrials.gov

Adverse events86

Each adverse event the trial posted, group by group: the participants affected out of those at risk.

Type	Event	Organ system	Group	Affected	At risk
other	Contusion	Injury, poisoning and procedural complications	Eculizumab	5	62
other	Contusion	Injury, poisoning and procedural complications	Placebo	2	63
other	Dizziness	Nervous system disorders	Eculizumab	5	62
other	Dizziness	Nervous system disorders	Placebo	5	63
other	Headache	Nervous system disorders	Eculizumab	10	62
other	Headache	Nervous system disorders	Placebo	12	63
other	Myasthenia gravis	Nervous system disorders	Eculizumab	1	62
other	Myasthenia gravis	Nervous system disorders	Placebo	5	63
other	Paraesthesia	Nervous system disorders	Eculizumab	3	62
other	Paraesthesia	Nervous system disorders	Placebo	4	63

1 to 10 of 86Source ClinicalTrials.gov

See all 86 adverse events

Adverse event totals6

The totals the trial posted for each group.

Total	Group	Affected	At risk
Total, all-cause mortality	Eculizumab	0	62
Total, all-cause mortality	Placebo	0	63
Total, other adverse events	Eculizumab	52	62
Total, other adverse events	Placebo	53	63
Total, serious adverse events	Eculizumab	9	62
Total, serious adverse events	Placebo	18	63

Source ClinicalTrials.gov

Papers10

The papers the trial lists on ClinicalTrials.gov.

PMID	Listed as	Source
23512355	BACKGROUND	PubMed
29066163	RESULT	PubMed
30905021	DERIVED	PubMed
32189108	DERIVED	PubMed
32426038	DERIVED	PubMed
32700461	DERIVED	PubMed
33229455	DERIVED	PubMed
33329303	DERIVED	PubMed
34590717	DERIVED	PubMed
34795626	DERIVED	PubMed

Grants0

The grants NIH RePORTER links to the trial, newest funded first: NIH's, and some of AHRQ's, CDC's, FDA's and VA's. Each shows its title, principal investigators, organization and funder in its newest year.

None in this release.

Trial comparisons2

Comparisons of the trial's two groups, calculated by ConductScience from the counts it posted.

Comparison	Source
Eculizumab vs Placebo: deaths from any cause	Trial comparisons
Eculizumab vs Placebo: serious adverse events	Trial comparisons

Tests0

The ConductCognition tests this trial's registered outcomes name, as ScienceGraph matched them in trial-tests-2026-11-12-0.2.1.

None in this release.

Data dates

The day each source was copied for this release.

ClinicalTrials.gov: 2026-11-12; ClinicalTrials.gov
Results, from AACT: 2026-03-23; AACT
NIH grant links: 2026-11-13; NIH RePORTER

Sources 3

ClinicalTrials.gov's posted results, through CTTI's AACTFreely available. Acknowledgement requested
ClinicalTrials.govNo license stated
NIH RePORTER's clinical studies and project filesPublic domain

Each source's terms

ClinicalTrials.gov's posted results, through CTTI's AACTClinicalTrials.gov data via Aggregate Analysis of ClinicalTrials.gov (AACT), CTTI. CTTI makes its materials freely available and requests acknowledgement. https://aact.ctti-clinicaltrials.org/documentation/299
ClinicalTrials.govnone stated
NIH RePORTER's clinical studies and project filesPublic domain (NIH RePORTER)

ClinicalTrials.gov's data carry an international copyright outside the United States: https://clinicaltrials.gov/about-site/terms-conditions.

For software

Read this trial from software.

The record and each of its lists are JSON, twenty rows a page. No key.

GET https://conductscience.org/api/trials/v1/NCT01997229

GET https://conductscience.org/api/trials/v1/NCT01997229/events?page=2

API reference For AI assistants