Safety and Efficacy of Eculizumab in Refractory Generaliz... | NCT01997229

NCT01997229

ID	Type	Description	Link
2013-003589-15	EudraCT Number

Status Module

Oversight Module

Design Module

Interventional Study Design

Biospecimen

No data available

No data is available for this block.

Arms/Interventions Module

Arm Groups

Label	Type	Description	Intervention Names
Eculizumab	Experimental	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).	Biological: Eculizumab
Placebo	Placebo Comparator	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).	Drug: Placebo

Name	Type	Description	Arm Group Labels	Other Names
Eculizumab	Biological	Induction Phase: 900 mg IV weekly X 4 Maintenance Phase: 1200 mg IV every 2 weeks during Weeks 4-26	Eculizumab

Outcomes Module

Primary Outcomes

Measure	Description	Time Frame
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)	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).	End of study (Week 26)

Eligibility Module

Facility	Status	City	State	ZIP	Country	Contacts
University of Alabama		Birmingham	Alabama	35233	United States
University of California San Francisco-Fresno

References Module

Citations

PubMed Identifier	Type	Citation	Retractions
23512355	Background	Howard JF Jr, Barohn RJ, Cutter GR, Freimer M, Juel VC, Mozaffar T, Mellion ML, Benatar MG, Farrugia ME, Wang JJ, Malhotra SS, Kissel JT; MG Study Group. A randomized, double-blind, placebo-controlled phase II study of eculizumab in patients with refractory generalized myasthenia gravis. Muscle Nerve. 2013 Jul;48(1):76-84. doi: 10.1002/mus.23839. Epub 2013 Apr 30.
29066163

Label	URL
Alexion Clinical Trials Webpage	View source

Results Section

Participant Flow Module

Arm/Group Information

ID	Title	Description
FG000	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).

Baseline Characteristics Module

Outcome Measures Module

Adverse Events Module

More Info Module

Arm/Group Information

ID	Title	Description
BG000	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).
BG001	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).
BG002	Total	Total of all reporting groups

Baseline Measures

Title

Description

Population Description

Parameter Type

Dispersion Type

Unit of Measure

Calculate Percentage

Denominator Units Selected

Denominators

Classes

Age, Continuous

Mean

Standard Deviation

years

Title

Denominators

Categories

Title	Measurements
	OG00056.6± 4.53 OG00168.3± 4.49

Group IDs	Group Description	Statistical Method	Statistical Comment	P-Value	P-Value Comment	Parameter Type	Parameter Value	Dispersion Type	Dispersion Value	Confidence Interval Sides	Confidence Interval %	CI Lower Limit	CI Upper Limit	CI Lower Limit Comment	CI Upper Limit Comment	Estimate Comment	Tested Non-Inferiority	Non-Inferiority Type	Non-Inferiority Comment	Other Analysis Description
OG000 OG001		ANCOVA		0.0698		Mean Difference (Net)	-11.7			2-Sided	95	-24.33	0.96					Superiority

Arm/Groups

ID	Title	Description	Deaths (Affected)	Deaths (At Risk)	Serious Events (Affected)	Serious Events (At Risk)	Other Events (Affected)	Other Events (At Risk)
EG000	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).	0	62	9	62	52	62
EG001	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).	0	63	18	63	53	63

Serious Adverse Events

Term	Organ System	Source Vocabulary	Statistical Information
Deep vein thrombosis	Vascular disorders	MedDRA (18.1)	EG0000 affected62 at risk EG0011 affected63 at risk
Intentional overdose	Injury, poisoning and procedural complications	MedDRA (18.1)	EG0000 affected62 at risk EG0011 affected63 at risk
Lymphocyte count decreased	Investigations	MedDRA (18.1)	EG0000 affected62 at risk EG0011 affected63 at risk
Metastases to bone	Neoplasms benign, malignant and unspecified (incl cysts and polyps)	MedDRA (18.1)	EG0001 affected62 at risk EG0010 affected63 at risk
Prostate cancer	Neoplasms benign, malignant and unspecified (incl cysts and polyps)	MedDRA (18.1)	EG0001 affected62 at risk EG0010 affected63 at risk
Apnoea	Respiratory, thoracic and mediastinal disorders	MedDRA (18.1)	EG0000 affected62 at risk EG0011 affected63 at risk
Pulmonary embolism	Respiratory, thoracic and mediastinal disorders	MedDRA (18.1)	EG0000 affected62 at risk EG0011 affected63 at risk
Lymphopenia	Blood and lymphatic system disorders	MedDRA (18.1)	EG0001 affected62 at risk EG0010 affected63 at risk
Myasthenia gravis	Nervous system disorders	MedDRA (18.1)	EG0005 affected62 at risk EG0018 affected63 at risk
Myasthenia gravis crisis	Nervous system disorders	MedDRA (18.1)	EG0001 affected62 at risk EG0010 affected63 at risk
General physical health deterioration	General disorders	MedDRA (18.1)	EG0000 affected62 at risk EG0011 affected63 at risk
Pyrexia	General disorders	MedDRA (18.1)	EG0002 affected62 at risk EG0010 affected63 at risk
Gastritis	Gastrointestinal disorders	MedDRA (18.1)	EG0000 affected62 at risk EG0011 affected63 at risk
Intestinal perforation	Gastrointestinal disorders	MedDRA (18.1)	EG0001 affected62 at risk EG0010 affected63 at risk
Cholecystitis acute	Hepatobiliary disorders	MedDRA (18.1)	EG0000 affected62 at risk EG0011 affected63 at risk
Hyperglycaemia	Metabolism and nutrition disorders	MedDRA (18.1)	EG0000 affected62 at risk EG0011 affected63 at risk
Bacteraemia	Infections and infestations	MedDRA (18.1)	EG0001 affected62 at risk EG0010 affected63 at risk
Diverticulitis	Infections and infestations	MedDRA (18.1)	EG0001 affected62 at risk EG0010 affected63 at risk
Endocarditis	Infections and infestations	MedDRA (18.1)	EG0001 affected62 at risk EG0010 affected63 at risk
Gastroenteritis	Infections and infestations	MedDRA (18.1)	EG0000 affected62 at risk EG0011 affected63 at risk
Tonsillitis	Infections and infestations	MedDRA (18.1)	EG0000 affected62 at risk EG0011 affected63 at risk
Upper respiratory tract infection	Infections and infestations	MedDRA (18.1)	EG0000 affected62 at risk EG0012 affected63 at risk
Urinary tract infection bacterial	Infections and infestations	MedDRA (18.1)	EG0000 affected62 at risk EG0011 affected63 at risk
Varicella	Infections and infestations	MedDRA (18.1)	EG0000 affected62 at risk EG0011 affected63 at risk

Other Adverse Events

Term	Organ System	Source Vocabulary	Statistical Information
Contusion	Injury, poisoning and procedural complications	MedDRA (18.1)	EG0005 affected62 at risk EG0012 affected63 at risk
Dizziness	Nervous system disorders	MedDRA (18.1)	EG0005 affected62 at risk EG0015 affected63 at risk
Headache	Nervous system disorders	MedDRA (18.1)	EG00010 affected62 at risk EG00112 affected63 at risk
Myasthenia gravis	Nervous system disorders	MedDRA (18.1)	EG0001 affected62 at risk EG0015 affected63 at risk
Paraesthesia	Nervous system disorders	MedDRA (18.1)	EG0003 affected62 at risk EG0014 affected63 at risk
Chills	General disorders	MedDRA (18.1)	EG0001 affected62 at risk EG0014 affected63 at risk
Oedema peripheral	General disorders	MedDRA (18.1)	EG0004 affected62 at risk EG0012 affected63 at risk
Insomnia	Psychiatric disorders	MedDRA (18.1)	EG0002 affected62 at risk EG0015 affected63 at risk
Diarrhoea	Gastrointestinal disorders	MedDRA (18.1)	EG0008 affected62 at risk EG0018 affected63 at risk
Nausea	Gastrointestinal disorders	MedDRA (18.1)	EG0008 affected62 at risk EG0019 affected63 at risk
Vomiting	Gastrointestinal disorders	MedDRA (18.1)	EG0003 affected62 at risk EG0015 affected63 at risk
Pruritus	Skin and subcutaneous tissue disorders	MedDRA (18.1)	EG0001 affected62 at risk EG0014 affected63 at risk
Back pain	Musculoskeletal and connective tissue disorders	MedDRA (18.1)	EG0005 affected62 at risk EG0016 affected63 at risk
Myalgia	Musculoskeletal and connective tissue disorders	MedDRA (18.1)	EG0005 affected62 at risk EG0012 affected63 at risk
Pain in extremity	Musculoskeletal and connective tissue disorders	MedDRA (18.1)	EG0004 affected62 at risk EG0012 affected63 at risk
Nasopharyngitis	Infections and infestations	MedDRA (18.1)	EG0009 affected62 at risk EG00110 affected63 at risk
Oral herpes	Infections and infestations	MedDRA (18.1)	EG0005 affected62 at risk EG0010 affected63 at risk
Upper respiratory tract infection	Infections and infestations	MedDRA (18.1)	EG00010 affected62 at risk EG00110 affected63 at risk
Urinary tract infection	Infections and infestations	MedDRA (18.1)	EG0004 affected62 at risk EG0015 affected63 at risk

ID	Term
D020361	Paraneoplastic Syndromes, Nervous System
D009423	Nervous System Neoplasms
D009371	Neoplasms by Site
D009369	Neoplasms
D010257	Paraneoplastic Syndromes
D020274	Autoimmune Diseases of the Nervous System
D009422	Nervous System Diseases
D019636	Neurodegenerative Diseases
D020511	Neuromuscular Junction Diseases
D009468	Neuromuscular Diseases
D001327	Autoimmune Diseases
D007154	Immune System Diseases