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The purpose of this study is to determine whether eculizumab is safe and effective in the treatment of patients with generalized myasthenia gravis despite treatment with various immunosuppressants, such as prednisone, methotrexate, Cellcept, cyclosporine, and cyclophosphamide, that are currently available.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| 1 | Experimental | eculizumab |
|
| 2 | Placebo Comparator | Placebo |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| eculizumab | Drug | eculizumab 600 mg IV weekly for 4 doses followed by eculizumab 900 mg IV every two weeks for 7 doses |
|
| Measure | Description | Time Frame |
|---|---|---|
| Quantitative Myasthenia Gravis (QMG): The Primary Efficacy Endpoint in This Study Was the Percentage of Patients With a 3-point Reduction From Baseline in the QMG Total Score for Disease Severity. | The QMG scoring system is considered to be an objective evaluation of muscle strength based on quantitative testing of sentinel muscle groups. The MGFA task force has recommended that the QMG score be used in prospective studies of therapy for MG. | 16 weeks |
| Measure | Description | Time Frame |
|---|---|---|
| Mean Change From Baseline in QMG Total Score | The QMG scoring system is considered to be an objective evaluation of muscle strength based on quantitative testing of sentinel muscle groups. The Myasthenia Gravis Foundation of America task force has recommended that the QMG score be used in prospective studies of therapy for MG. The QMG scoring system consists of 13 items. Each item is graded 0 to 3, with 3 being the most severe. The range of total QMG score is 0-39. |
Not provided
Inclusion Criteria:
Exclusion Criteria:
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| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| University of Alabama at Birmingham | Birmingham | Alabama | 35233 | United States | ||
| University of California, Irvine |
| PubMed Identifier | Type | Citation | Retractions |
|---|---|---|---|
| 23512355 | Result | 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. |
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Patients received standard of care during the Screening Period. Patients were randomized to a treatment sequence to receive eculizumab in Period 1 followed by placebo in Period 2 or placebo in Period 1 followed by eculizumab in Period 2. Patients were permitted to continue on background immunosuppressive therapy throughout the study.
There were 26 IRB/IEC-approved study sites. Patients were screened at 13 Institutional Review Board/Independent Ethics Committee approved study sites; and 14 patients were randomized at 8 sites.
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| ID | Title | Description |
|---|---|---|
| FG000 | Eculizumab to Placebo Sequence | Eculizumab: eculizumab 600 mg IV weekly (4 doses) followed by 900 mg IV every other week (7 doses) Placebo: matching placebo IV weekly (4 doses) followed by IV every other week (7 doses) Period 1: patients received eculizumab for 16 weeks. Wash-out period for 5 weeks. Period 2 (cross-over treatment period): patients received placebo for 16 weeks. |
| Title | Milestones | Reasons Not Completed | |||||
|---|---|---|---|---|---|---|---|
| Screening Period |
|
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
| Placebo | Drug | Placebo IV weekly for 4 doses then every two weeks for 7 doses |
|
| 16 weeks |
| Change From Baseline in the MGFA Post-Intervention Status (PIS) | The MGFA PIS is designed to assess the clinical state of MG patients at any time after treatment of MG is initiated. Change in status categories of Improved, Unchanged, Worse, Exacerbation, and Died of MG was to be assessed and recorded at every visit from Visits 3 to 24 (Weeks 1 to 16). Minimal manifestations were to be assessed at these visits. | 16 weeks |
| Change From Baseline in the MG-Activity of Daily Living Profile (MG-ADL) | The MG-ADL is an 8-point questionnaire that focuses on relevant symptoms and functional performance of activities of daily living (ADL) in MG patients. The 8 items of the MG-ADL were derived from symptom-based components of the original 13-item QMG to assess disability secondary to ocular (2 items), bulbar (3 items), respiratory (1 item), and gross motor or limb (2 items) impairment related to effects from MG. In this functional status instrument, each response is graded 0 (normal) to 3 (most severe). The range of total MG-ADL score is 0 - 24. MG-ADL was to be performed at every study visit. The recall period for MG-ADL was since the preceding study visit (1 or 2 weeks). | 16 weeks |
| Change From Baseline in the QoL Instrument, SF-36. | The SF-36 is a multi-purpose, short-form health survey with 36 questions. It yields an 8-scale profile of functional health and well-being scores (physical functioning, role-physical, bodily pain, general health, mental health, role-emotional, social functioning and vitality) as well as psychometrically-based physical and mental health summary measures. It is a generic measure, as opposed to one that targets a specific age, disease or treatment group. The lower the score the more disability; the higher the score the less disability. Norm-based scoring involving a linear T-score transformation method was used so that scores for each of the health domain scales and component summary measures have a mean of 50 and a standard deviation of 10 based on the 1998 US general population. Thus, scores above and below 50 are above and below the average, respectively, in the 1998 US general. | 16 weeks |
| Change From Baseline in Respiratory Function Tests to Characterize the Degree of Involvement of Respiratory Muscles. | Change from Baseline in Forced Vital Capacity | 16 weeks |
| Change From Baseline in Respiratory Function Tests to Characterize the Degree of Involvement of Respiratory Muscles. | Change from Baseline in Negative Inspiratory Force. NIF is a measurement of respiratory muscle strength and ventilator reserve. NIF is represented by centimeters of water pressure (cmH2O). A normal NIF measurement is negative 60 cmH2O, or as 100% predicted value. | 16 weeks |
| Change From Baseline to the End of Treatment (16 Weeks) in the Two Most Affected QMG Items for Disease Severity (Individual Test Item: Double Vision) | The QMG scoring system is considered to be an objective evaluation of muscle strength based on quantitative testing of sentinel muscle groups. The MGFA task force has recommended that the QMG score be used in prospective studies of therapy for MG. All individual QMG items are scored 0 to 3, with 3 being the most severe. Negative values imply an improvement in QMG Item Score. | 16 weeks |
| Change From Baseline to the End of Treatment (16 Weeks) in the Two Most Affected QMG Items for Disease Severity (Individual Test Item: Ptosis) | The QMG scoring system is considered to be an objective evaluation of muscle strength based on quantitative testing of sentinel muscle groups. The MGFA task force has recommended that the QMG score be used in prospective studies of therapy for MG. All individual QMG items are scored 0 to 3, with 3 being the most severe. Negative values imply an improvement in QMG Item Score. | 16 weeks |
| Orange |
| California |
| 92868 |
| United States |
| University of California - Davis | Sacramento | California | 95817 | United States |
| University of Florida & Shands Neuroscience Institute | Jacksonville | Florida | 32209 | United States |
| Emory University | Atlanta | Georgia | 30322 | United States |
| Wishard Hospital | Indianapolis | Indiana | 46202 | United States |
| University of Kansas Medical Center | Kansas City | Kansas | 66160 | United States |
| Johns Hopkins University School of Medicine | Baltimore | Maryland | 21287 | United States |
| Massachusetts General Hospital | Boston | Massachusetts | 02114 | United States |
| Caritas St. Elizabeths' Medical Center | Boston | Massachusetts | 02135 | United States |
| Wayne State University | Detroit | Michigan | 48201 | United States |
| Mount Sinai School of Medicine | New York | New York | 10029-6574 | United States |
| University of North Carolina at Chapel Hill | Chapel Hill | North Carolina | 27599-1651 | United States |
| Duke University Medical Center | Durham | North Carolina | 27710 | United States |
| University Hospitals - Case Medical Center | Cleveland | Ohio | 44106-5098 | United States |
| The Ohio State University | Columbus | Ohio | 43210 | United States |
| The Warren Alpert Medical School of Brown University | Providence | Rhode Island | 02905 | United States |
| Vanderbilt University Medical Center | Nashville | Tennessee | 37232 | United States |
| University of Texas Southwestern Medical School | Dallas | Texas | 75390 | United States |
| The University of Vermont College of Medicine | Burlington | Vermont | 05405 | United States |
| University of Virginia | Charlottesville | Virginia | 22908-0394 | United States |
| The Northern Alberta Clinical trials and Research Centre | Edmonton | Alberta | Canada |
| Institute of Neurological Sciences, Department of Neurology, Southern General Hospital, | Glasgow | United Kingdom |
| Institute of Neurology | London | United Kingdom |
| Department of Clinical Neurology, West Wing, John Radcliffe Hospital | Oxford | United Kingdom |
| FG001 | Placebo to Eculizumab Sequence | Placebo: matching placebo IV weekly (4 doses) followed by IV every other week (7 doses). Eculizumab: eculizumab 600 mg IV weekly (4 doses) followed by 900 mg IV every other week (7 doses). Period 1: patients received placebo for 16 weeks. Wash-out period for 5 weeks. Period 2 (cross-over treatment period): patients received eculizumab for 16 weeks. |
| FG002 | Not Randomized/Screen Failures | Not randomized; not treatment cohort |
| COMPLETED |
|
| NOT COMPLETED |
|
|
| Treatment Period 1 |
|
| Washout Period |
|
| Treatment Period 2 |
|
|
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| ID | Title | Description |
|---|---|---|
| BG000 | Overall Study | Eculizumab: Eculizumab [600 mg IV weekly (4 doses) followed by 900 mg IV every other week (7 doses)]. Period 1: patients received eculizumab for 16 weeks; Period 2: wash-out period for 5 weeks (the cross-over treatment period). Patients then received placebo for 16 weeks. Placebo: Matching placebo [IV weekly (4 doses) followed by IV every other week (7 doses)] Period 1: patients received placebo for 16 weeks; Period 2: wash-out period for 5 weeks (the cross-over treatment period). Patients then received eculizumab for 16 weeks. |
| Units | Counts |
|---|---|
| Participants |
|
| Title | Description | Population Description | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Denominator Units Selected | Denominators | Classes | |||||||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Age, Continuous | Mean | Standard Deviation | years |
| ||||||||||||||||||||||
| Sex: Female, Male | Count of Participants | Participants |
| |||||||||||||||||||||||
| Race/Ethnicity, Customized | Number | participants |
| |||||||||||||||||||||||
| Region of Enrollment | Number | participants |
| |||||||||||||||||||||||
| Myasthenia Gravis Foundation of America (MGFA) classification at Screening | MGFA Classification identifies the subgroup patients with MG who share distinct clinical features or severity of disease: Class I (ocular MG), classes II, III and IV generalized MG with mild, moderate and severe disease, respectively; Class V MG crisis. Separate subclasses under classes II, III and IV are designed: "a" if the predominant weakness is affecting limb/axial weakness or both; subclass "b" if the predominant weakness is affecting oropharyngeal or respiratory muscles or both. | Number | participants |
| ||||||||||||||||||||||
| Number of MG crisis/exacerbation prior to Screening | The number of a MG crisis/exacerbation prior to Screening provides pertinent information about disease morbidity and severity. | Mean | Standard Deviation | Events |
| |||||||||||||||||||||
| Prednisone use at Screening | An inclusion criterion was that patients must have failed treatment or failed to achieve significant clinical benefit with at least 2 immunomodulators after 1 year of treatment. | Number | participants |
| ||||||||||||||||||||||
| Immunosuppression therapy other than prednisone at Screening | An inclusion criterion was that patients must have failed treatment or failed to achieve significant clinical benefit with at least 2 immunomodulators after 1 year of treatment. | Number | participants |
| ||||||||||||||||||||||
| Cholinesterase inhibitor use at Screening | Number | participants |
| |||||||||||||||||||||||
| Quantitative Myasthenia Gravis (QMG) at Screening | The QMG scoring system is considered to be an objective evaluation of therapy for MG and is based on quantitative testing of muscle strength and endurance/fatigue of sentinel muscle groups. The QMG consists of 13 items: ocular (2 items), facial (1 item), bulbar (2 items), gross motor (6 items), axial (1 item) and respiratory (1 item); each graded 0 to 3, with 3 being the most severe. The range of total QMG score is 0 - 39. | Mean | Standard Deviation | QMG score |
| |||||||||||||||||||||
| Myasthenia Gravis-Activity of Daily Living (MG-ADL) | The MG-ADL is a questionnaire that focuses on relevant symptoms and functional performance of activities of daily living in MG patients. The MG-ADL consists of 8 items: ocular (2 items), bulbar (3 items), respiratory (1 item) and gross motor or limb (2 items). Each response is graded 0 (normal) to 3 (most severe). The range of total MG-ADL score is 0 (normal) to 24 (most severe). | Mean | Full Range | units on a scale |
|
| Type | Title | Description | Population Description | Reporting Status | Anticipated Posting Date | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Time Frame | Units Analyzed | Denominator Units Selected | Arm/Group Information | Denominators | Classes | Analyses | |||||||||||||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Primary | Quantitative Myasthenia Gravis (QMG): The Primary Efficacy Endpoint in This Study Was the Percentage of Patients With a 3-point Reduction From Baseline in the QMG Total Score for Disease Severity. | The QMG scoring system is considered to be an objective evaluation of muscle strength based on quantitative testing of sentinel muscle groups. The MGFA task force has recommended that the QMG score be used in prospective studies of therapy for MG. | Posted | Number | percentage of patients | 16 weeks |
|
|
| ||||||||||||||||||||||||||||||
| Secondary | Mean Change From Baseline in QMG Total Score | The QMG scoring system is considered to be an objective evaluation of muscle strength based on quantitative testing of sentinel muscle groups. The Myasthenia Gravis Foundation of America task force has recommended that the QMG score be used in prospective studies of therapy for MG. The QMG scoring system consists of 13 items. Each item is graded 0 to 3, with 3 being the most severe. The range of total QMG score is 0-39. | Posted | Mean | Standard Deviation | units on a scale | 16 weeks |
| |||||||||||||||||||||||||||||||
| Secondary | Change From Baseline in the MGFA Post-Intervention Status (PIS) | The MGFA PIS is designed to assess the clinical state of MG patients at any time after treatment of MG is initiated. Change in status categories of Improved, Unchanged, Worse, Exacerbation, and Died of MG was to be assessed and recorded at every visit from Visits 3 to 24 (Weeks 1 to 16). Minimal manifestations were to be assessed at these visits. | Posted | Number | participants | 16 weeks |
| ||||||||||||||||||||||||||||||||
| Secondary | Change From Baseline in the MG-Activity of Daily Living Profile (MG-ADL) | The MG-ADL is an 8-point questionnaire that focuses on relevant symptoms and functional performance of activities of daily living (ADL) in MG patients. The 8 items of the MG-ADL were derived from symptom-based components of the original 13-item QMG to assess disability secondary to ocular (2 items), bulbar (3 items), respiratory (1 item), and gross motor or limb (2 items) impairment related to effects from MG. In this functional status instrument, each response is graded 0 (normal) to 3 (most severe). The range of total MG-ADL score is 0 - 24. MG-ADL was to be performed at every study visit. The recall period for MG-ADL was since the preceding study visit (1 or 2 weeks). | Comparison of MG Activities of Daily Living (Total score) at the Last Visit Between Eculizumab and Placebo Cohorts. | Posted | Mean | Standard Deviation | units on a scale | 16 weeks |
| ||||||||||||||||||||||||||||||
| Secondary | Change From Baseline in the QoL Instrument, SF-36. | The SF-36 is a multi-purpose, short-form health survey with 36 questions. It yields an 8-scale profile of functional health and well-being scores (physical functioning, role-physical, bodily pain, general health, mental health, role-emotional, social functioning and vitality) as well as psychometrically-based physical and mental health summary measures. It is a generic measure, as opposed to one that targets a specific age, disease or treatment group. The lower the score the more disability; the higher the score the less disability. Norm-based scoring involving a linear T-score transformation method was used so that scores for each of the health domain scales and component summary measures have a mean of 50 and a standard deviation of 10 based on the 1998 US general population. Thus, scores above and below 50 are above and below the average, respectively, in the 1998 US general. | Comparison of SF-36 at the Last Visit Between Eculizumab and Placebo Cohorts. | Posted | Mean | Standard Deviation | units on a scale | 16 weeks |
| ||||||||||||||||||||||||||||||
| Secondary | Change From Baseline in Respiratory Function Tests to Characterize the Degree of Involvement of Respiratory Muscles. | Change from Baseline in Forced Vital Capacity | Comparison of FVC at the Last Visit Between Eculizumab and Placebo Cohorts. | Posted | Mean | Standard Deviation | percentage of predicted | 16 weeks |
| ||||||||||||||||||||||||||||||
| Secondary | Change From Baseline in Respiratory Function Tests to Characterize the Degree of Involvement of Respiratory Muscles. | Change from Baseline in Negative Inspiratory Force. NIF is a measurement of respiratory muscle strength and ventilator reserve. NIF is represented by centimeters of water pressure (cmH2O). A normal NIF measurement is negative 60 cmH2O, or as 100% predicted value. | Comparison of NIF at the Last Visit Between Eculizumab and Placebo Cohorts. | Posted | Mean | Standard Deviation | percentage of predicted | 16 weeks |
| ||||||||||||||||||||||||||||||
| Secondary | Change From Baseline to the End of Treatment (16 Weeks) in the Two Most Affected QMG Items for Disease Severity (Individual Test Item: Double Vision) | The QMG scoring system is considered to be an objective evaluation of muscle strength based on quantitative testing of sentinel muscle groups. The MGFA task force has recommended that the QMG score be used in prospective studies of therapy for MG. All individual QMG items are scored 0 to 3, with 3 being the most severe. Negative values imply an improvement in QMG Item Score. | The Investigators selected the 2 most affected items (double vision, ptosis) out of the 13 items in the QMG scoring system for each of their patients based on their clinical evaluation at Baseline. The count is provided when the item was selected as the most affected by the Investigator, for participants who were treated in the respective sequence. | Posted | Mean | Standard Deviation | units on a scale | 16 weeks |
|
| |||||||||||||||||||||||||||||
| Secondary | Change From Baseline to the End of Treatment (16 Weeks) in the Two Most Affected QMG Items for Disease Severity (Individual Test Item: Ptosis) | The QMG scoring system is considered to be an objective evaluation of muscle strength based on quantitative testing of sentinel muscle groups. The MGFA task force has recommended that the QMG score be used in prospective studies of therapy for MG. All individual QMG items are scored 0 to 3, with 3 being the most severe. Negative values imply an improvement in QMG Item Score. | The Investigators selected the 2 most affected items (double vision, ptosis) out of the 13 items in the QMG scoring system for each of their patients based on their clinical evaluation at Baseline. The count is provided when the item was selected as the most affected by the Investigator, for participants who were treated in the respective sequence. | Posted | Mean | Standard Deviation | units on a scale | 16 weeks |
|
|
46 weeks (Screening Period [up to 4 weeks], Treatment Period 1 [16 weeks], Washout Period [5 weeks], Treatment Period 2 [16 weeks], Follow-up Period [5 weeks]) for adverse events (AEs); 42 weeks for treatment emergent AEs (TEAEs).
TEAEs were collected at every visit and follow-up
Not provided
| ID | Title | Description | Deaths (Affected) | Deaths (At Risk) | Serious Events (Affected) | Serious Events (At Risk) | Other Events (Affected) | Other Events (At Risk) |
|---|---|---|---|---|---|---|---|---|
| EG000 | Placebo | Placebo Placebo: Placebo IV weekly for 4 doses then every two weeks for 7 doses Events that occurred during the Washout Period were attributed to treatment assignment during Treatment Period 1. | 1 | 13 | 11 | 13 | ||
| EG001 | Eculizumab | Eculizumab Eculizumab: eculizumab 600 mg IV weekly for 4 doses followed by eculizumab 900 mg IV every two weeks for 7 doses Events that occurred during the Washout Period were attributed to treatment assignment during Treatment Period 1. | 1 | 13 | 13 | 13 |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Myasthenia Gravis | Nervous system disorders | MedDRA 11.0 | Systematic Assessment |
| |
| Myasthenia Gravis Crisis | Nervous system disorders | MedDRA 11.0 | Systematic Assessment |
|
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Leukopenia | Blood and lymphatic system disorders | MedDRA 11.0 | Systematic Assessment |
| |
| Diplopia | Eye disorders | MedDRA 11.0 | Systematic Assessment |
| |
| Eye Discharge | Eye disorders | MedDRA 11.0 | Systematic Assessment |
| |
| Lacrimation Increased | Eye disorders | MedDRA 11.0 | Systematic Assessment |
| |
| Ocular Hyperaemia | Eye disorders | MedDRA 11.0 | Systematic Assessment |
| |
| Abdominal Discomfort | Gastrointestinal disorders | MedDRA 11.0 | Systematic Assessment |
| |
| Abdominal Pain | Gastrointestinal disorders | MedDRA 11.0 | Systematic Assessment |
| |
| Abdominal Pain Upper | Gastrointestinal disorders | MedDRA 11.0 | Systematic Assessment |
| |
| Constipation | Gastrointestinal disorders | MedDRA 11.0 | Systematic Assessment |
| |
| Dental Caries | Gastrointestinal disorders | MedDRA 11.0 | Systematic Assessment |
| |
| Diarrhoea | Gastrointestinal disorders | MedDRA 11.0 | Systematic Assessment |
| |
| Dyspepsia | Gastrointestinal disorders | MedDRA 11.0 | Systematic Assessment |
| |
| Haematochezia | Blood and lymphatic system disorders | MedDRA 11.0 | Systematic Assessment |
| |
| Lip Dry | Blood and lymphatic system disorders | MedDRA 11.0 | Systematic Assessment |
| |
| Nausea | Blood and lymphatic system disorders | MedDRA 11.0 | Systematic Assessment |
| |
| Oesophageal Food Impaction | Gastrointestinal disorders | MedDRA 11.0 | Systematic Assessment |
| |
| Salivary Gland Enlargement | Gastrointestinal disorders | MedDRA 11.0 | Systematic Assessment |
| |
| Vomiting | Gastrointestinal disorders | MedDRA 11.0 | Systematic Assessment |
| |
| Application Site Pruritus | General disorders | MedDRA 11.0 | Systematic Assessment |
| |
| Chest Discomfort | General disorders | MedDRA 11.0 | Systematic Assessment |
| |
| Chest Pain | General disorders | MedDRA 11.0 | Systematic Assessment |
| |
| Cyst | General disorders | MedDRA 11.0 | Systematic Assessment |
| |
| Fatigue | General disorders | MedDRA 11.0 | Systematic Assessment |
| |
| Influenza Like Illness | General disorders | MedDRA 11.0 | Systematic Assessment |
| |
| Pain | General disorders | MedDRA 11.0 | Systematic Assessment |
| |
| Pyrexia | General disorders | MedDRA 11.0 | Systematic Assessment |
| |
| Vessel Puncture Site Haematoma | General disorders | MedDRA 11.0 | Systematic Assessment |
| |
| Hepatic Steatosis | Hepatobiliary disorders | MedDRA 11.0 | Systematic Assessment |
| |
| Bronchitis | Infections and infestations | MedDRA 11.0 | Systematic Assessment |
| |
| Cellulitis | Infections and infestations | MedDRA 11.0 | Systematic Assessment |
| |
| Fungal Infection | Infections and infestations | MedDRA 11.0 | Systematic Assessment |
| |
| Gastroenteritis Viral | Infections and infestations | MedDRA 11.0 | Systematic Assessment |
| |
| Influenza | Infections and infestations | MedDRA 11.0 | Systematic Assessment |
| |
| Nasopharyngitis | Infections and infestations | MedDRA 11.0 | Systematic Assessment |
| |
| Onychomycosis | Infections and infestations | MedDRA 11.0 | Systematic Assessment |
| |
| Oral Infection | Infections and infestations | MedDRA 11.0 | Systematic Assessment |
| |
| Rhinitis | Infections and infestations | MedDRA 11.0 | Systematic Assessment |
| |
| Sinusitis | Infections and infestations | MedDRA 11.0 | Systematic Assessment |
| |
| Upper Respiratory Tract Infection | Infections and infestations | MedDRA 11.0 | Systematic Assessment |
| |
| Viral Infection | Infections and infestations | MedDRA 11.0 | Systematic Assessment |
| |
| Viral Upper Respiratory Tract Infection | Infections and infestations | MedDRA 11.0 | Systematic Assessment |
| |
| Contusion | Injury, poisoning and procedural complications | MedDRA 11.0 | Systematic Assessment |
| |
| Limb Injury | Injury, poisoning and procedural complications | MedDRA 11.0 | Systematic Assessment |
| |
| Muscle Strain | Injury, poisoning and procedural complications | MedDRA 11.0 | Systematic Assessment |
| |
| Procedural Nausea | Injury, poisoning and procedural complications | MedDRA 11.0 | Systematic Assessment |
| |
| Scratch | Injury, poisoning and procedural complications | MedDRA 11.0 | Systematic Assessment |
| |
| Tendon Rupture | Injury, poisoning and procedural complications | MedDRA 11.0 | Systematic Assessment |
| |
| Tooth Fracture | Injury, poisoning and procedural complications | MedDRA 11.0 | Systematic Assessment |
| |
| Wound | Injury, poisoning and procedural complications | MedDRA 11.0 | Systematic Assessment |
| |
| Heart Rate Increased | Investigations | MedDRA 11.0 | Systematic Assessment |
| |
| Lymphocyte Count Decreased | Investigations | MedDRA 11.0 | Systematic Assessment |
| |
| Weight Decreased | Investigations | MedDRA 11.0 | Systematic Assessment |
| |
| Weight Increased | Investigations | MedDRA 11.0 | Systematic Assessment |
| |
| White Blood Cell Count Decreased | Investigations | MedDRA 11.0 | Systematic Assessment |
| |
| Type 2 Diabetes Mellitus | Metabolism and nutrition disorders | MedDRA 11.0 | Systematic Assessment |
| |
| Arthralgia | Musculoskeletal and connective tissue disorders | MedDRA 11.0 | Systematic Assessment |
| |
| Back Pain | Musculoskeletal and connective tissue disorders | MedDRA 11.0 | Systematic Assessment |
| |
| Bone Pain | Musculoskeletal and connective tissue disorders | MedDRA 11.0 | Systematic Assessment |
| |
| Muscle Spasms | Musculoskeletal and connective tissue disorders | MedDRA 11.0 | Systematic Assessment |
| |
| Musculoskeletal Pain | Musculoskeletal and connective tissue disorders | MedDRA 11.0 | Systematic Assessment |
| |
| Musculoskeletal Stiffness | Musculoskeletal and connective tissue disorders | MedDRA 11.0 | Systematic Assessment |
| |
| Myalgia | Musculoskeletal and connective tissue disorders | MedDRA 11.0 | Systematic Assessment |
| |
| Neck Pain | Musculoskeletal and connective tissue disorders | MedDRA 11.0 | Systematic Assessment |
| |
| Pain In Extremity | Musculoskeletal and connective tissue disorders | MedDRA 11.0 | Systematic Assessment |
| |
| Acrochordon | Neoplasms benign, malignant and unspecified (incl cysts and polyps) | MedDRA 11.0 | Systematic Assessment |
| |
| Carpal Tunnel Syndrome | Nervous system disorders | MedDRA 11.0 | Systematic Assessment |
| |
| Cholinergic Syndrome | Nervous system disorders | MedDRA 11.0 | Systematic Assessment |
| |
| Dizziness | Nervous system disorders | MedDRA 11.0 | Systematic Assessment |
| |
| Headache | Nervous system disorders | MedDRA 11.0 | Systematic Assessment |
| |
| Memory Impairment | Nervous system disorders | MedDRA 11.0 | Systematic Assessment |
| |
| Paraesthesia | Nervous system disorders | MedDRA 11.0 | Systematic Assessment |
| |
| Presyncope | Nervous system disorders | MedDRA 11.0 | Systematic Assessment |
| |
| Sensory Disturbance | Nervous system disorders | MedDRA 11.0 | Systematic Assessment |
| |
| Sinus Headache | Nervous system disorders | MedDRA 11.0 | Systematic Assessment |
| |
| Insomnia | Psychiatric disorders | MedDRA 11.0 | Systematic Assessment |
| |
| Haematuria | Renal and urinary disorders | MedDRA 11.0 | Systematic Assessment |
| |
| Nephrolithiasis | Renal and urinary disorders | MedDRA 11.0 | Systematic Assessment |
| |
| Erectile Dysfunction | Reproductive system and breast disorders | MedDRA 11.0 | Systematic Assessment |
| |
| Menstrual Disorder | Reproductive system and breast disorders | MedDRA 11.0 | Systematic Assessment |
| |
| Polymenorrhoea | Reproductive system and breast disorders | MedDRA 11.0 | Systematic Assessment |
| |
| Cough | Respiratory, thoracic and mediastinal disorders | MedDRA 11.0 | Systematic Assessment |
| |
| Dysphonia | Respiratory, thoracic and mediastinal disorders | MedDRA 11.0 | Systematic Assessment |
| |
| Dyspnoea | Respiratory, thoracic and mediastinal disorders | MedDRA 11.0 | Systematic Assessment |
| |
| Hyperventilation | Respiratory, thoracic and mediastinal disorders | MedDRA 11.0 | Systematic Assessment |
| |
| Pharyngolaryngeal Pain | Respiratory, thoracic and mediastinal disorders | MedDRA 11.0 | Systematic Assessment |
| |
| Respiratory Tract Congestion | Respiratory, thoracic and mediastinal disorders | MedDRA 11.0 | Systematic Assessment |
| |
| Dermatitis Allergic | Skin and subcutaneous tissue disorders | MedDRA 11.0 | Systematic Assessment |
| |
| Eczema | Skin and subcutaneous tissue disorders | MedDRA 11.0 | Systematic Assessment |
| |
| Erythema | Skin and subcutaneous tissue disorders | MedDRA 11.0 | Systematic Assessment |
| |
| Hyperhidrosis | Skin and subcutaneous tissue disorders | MedDRA 11.0 | Systematic Assessment |
| |
| Pruritus Generalised | Skin and subcutaneous tissue disorders | MedDRA 11.0 | Systematic Assessment |
| |
| Rash | Skin and subcutaneous tissue disorders | MedDRA 11.0 | Systematic Assessment |
| |
| Skin Lesion | Skin and subcutaneous tissue disorders | MedDRA 11.0 | Systematic Assessment |
| |
| Haematoma | Vascular disorders | MedDRA 11.0 | Systematic Assessment |
|
Small sample size limited ability to detect intercohort differences. Carryover effect from Treatment Period (TP) 1 warrants cautious interpretation of TP 2 data.
Study terminated early; 13 patients received eculizumab or placebo in TP2.
Not provided
Not provided
| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Alexion Pharmaceuticals | Alexion Pharmaceuticals | 855-752-2356 | clinicaltrials@alexion.com |
| ID | Term |
|---|---|
| D009157 | Myasthenia Gravis |
| 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 |
Not provided
Not provided
| ID | Term |
|---|---|
| C481642 | eculizumab |
Not provided
Not provided
Not provided
| Black |
|
| Measurements |
|---|
|
| IIIa |
|
| IVa |
|
Placebo
Placebo: Placebo IV weekly for 4 doses then every two weeks for 7 doses
|
|
|
|
|
|
Eculizumab
Eculizumab: eculizumab 600 mg IV weekly for 4 doses followed by eculizumab 900 mg IV every two weeks for 7 doses
| OG003 | Placebo Both Periods | Placebo Placebo: Placebo IV weekly for 4 doses then every two weeks for 7 doses |
|
|
|
| OG002 |
| Eculizumab Both Periods |
Eculizumab Eculizumab: eculizumab 600 mg IV weekly for 4 doses followed by eculizumab 900 mg IV every two weeks for 7 doses |
| OG003 | Placebo Both Periods | Placebo Placebo: Placebo IV weekly for 4 doses then every two weeks for 7 doses |
|
|
|
| Units | Counts |
|---|---|
| Participants |
|
|
|
Placebo
Placebo: Placebo IV weekly for 4 doses then every two weeks for 7 doses
|
|
|
|
|
|