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The objectives of this time-to-event study were to assess the efficacy and safety of eculizumab as compared with placebo in participants with neuromyelitis optica spectrum disorder (NMOSD) who were anti-aquaporin-4 (AQP4) antibody-positive.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Eculizumab | Experimental | Biological/Vaccine: Eculizumab; Induction Period: Participants received eculizumab (900 milligrams [mg]) via intravenous (IV) infusion once a week (every 7 ± 2 days) for 4 weeks followed by eculizumab 1200 mg for the fifth dose (Week 4). This was followed by the Maintenance Period: Participants received eculizumab (1200 mg) via IV infusion every 2 weeks (every 14 ± 2 days) from the sixth dose (Week 6) onwards. |
|
| Placebo | Placebo Comparator | Placebo contains the same buffer components without the active ingredient. Induction Period: Participants received matching placebo (900 mg) via IV infusion once a week (every 7 ± 2 days) for 4 weeks, followed by matching placebo (1200 mg) for the fifth dose (Week 4). This was followed by the Maintenance Period: Participants received matching placebo (1200 mg) via IV infusion every 2 weeks (every 14 ± 2 days) from the sixth dose (Week 6) onwards. |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Eculizumab | Drug | Induction Phase: 900 mg IV weekly for 4 weeks, followed by 1200 mg for the fifth dose; Maintenance Phase: 1200 mg IV every 2 weeks |
|
| Measure | Description | Time Frame |
|---|---|---|
| Participants With An Adjudicated On-trial Relapse | An On-trial Relapse was defined as a new onset of neurologic symptoms or worsening of existing neurologic symptoms with an objective change (clinical sign) on neurologic examination that persisted for more than 24 hours as confirmed by the treating physician. An adjudicated On-trial Relapse was defined by the protocol and positively adjudicated by the relapse adjudication committee. | Baseline, Up To 211 Weeks (End of Study) |
| Measure | Description | Time Frame |
|---|---|---|
| Adjudicated On-trial Annualized Relapse Rate (ARR) | The adjudicated On-trial ARR was computed as the total number of relapses divided by the total number of patient years in the study period. A central independent committee was used to adjudicate all On-trial Relapses as determined by the treating physician. Results reported as adjusted adjudicated On-trial ARR based on a Poisson regression adjusted for randomization strata and historical ARR in 24 months prior to Screening. |
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Key Inclusion Criteria:
Key Exclusion Criteria:
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| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Mayo Clinic Arizona | Scottsdale | Arizona | 85259 | United States | ||
| The Research Center of Southern California |
| PubMed Identifier | Type | Citation | Retractions |
|---|---|---|---|
| 23623397 | Background | Pittock SJ, Lennon VA, McKeon A, Mandrekar J, Weinshenker BG, Lucchinetti CF, O'Toole O, Wingerchuk DM. Eculizumab in AQP4-IgG-positive relapsing neuromyelitis optica spectrum disorders: an open-label pilot study. Lancet Neurol. 2013 Jun;12(6):554-62. doi: 10.1016/S1474-4422(13)70076-0. Epub 2013 Apr 26. | |
| 31050279 | Result |
| Label | URL |
|---|---|
| Alexion Clinical Trials Website | View source |
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Main inclusion criteria were: participants aged ≥ 18 years old with NMO/NMOSD, AQP4 antibody-positive, historical relapse of at least 2 in last 12 months or 3 in last 24 months with at least 1 in 12 months prior to screening, Expanded Disability Status Scale score ≤ 7. If receiving immunosuppressive therapies, must be on a stable maintenance dose.
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| ID | Title | Description |
|---|---|---|
| FG000 | Eculizumab | Induction Period: Participants received eculizumab (900 milligrams [mg]) via intravenous (IV) infusion once a week (every 7 ± 2 days) for 4 weeks followed by eculizumab 1200 mg for the fifth dose (Week 4). Maintenance Period: Participants received eculizumab (1200 mg) via IV infusion every 2 weeks (every 14 ± 2 days) from the sixth dose (Week 6) onwards. |
| Title | Milestones | Reasons Not Completed | |||||
|---|---|---|---|---|---|---|---|
| Overall Study |
|
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| Type | Includes Protocol | Includes SAP | Includes ICF | Document Label | Document Date | Document Uploaded Date | Document File Name |
|---|---|---|---|---|---|---|---|
| Prot | Yes | No | No | Study Protocol | Jul 1, 2016 | Jun 7, 2019 |
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| Placebo | Drug | Induction Phase: matching placebo (900 mg) IV weekly for 4 weeks, followed by matching placebo (1200 mg) for the fifth dose; Maintenance Phase: matching placebo (1200 mg) IV every 2 weeks |
|
| Baseline, Up To 211 Weeks (End of Study) |
| Change From Baseline In EDSS At End Of Study | Disease-related disability was measured by the EDSS. The EDSS is an ordinal clinical rating scale that ranges from 0 (normal neurologic examination) to 10 (death) in half-point increments. A decrease in score indicates improvement. | Baseline, Up To 211 Weeks (End of Study) |
| Change From Baseline In Modified Rankin Scale (mRS) Score At End Of Study | Disease-related disability was measured by the mRS score. The mRS is a commonly used scale for measuring the degree of disability or dependence in the daily activities of people who have suffered from a neurological disability. The scale ranges from 0 (no disability) to 6 (death) in whole-point increments. A decrease in score indicates improvement. | Baseline, Up To 211 Weeks (End of Study) |
| Change From Baseline In Hauser Ambulation Index (HAI) Score At End of Study | The HAI evaluates gait and was used to assess the time and effort used by the participant to walk 25 feet (8 meters). The scale ranges from 0 to 9, with 0 being the best score (asymptomatic; fully ambulatory with no assistance) and 9 being the worst (restricted to wheel chair; unable to transfer self independently). A decrease in score indicates improvement. | Baseline, Up To 211 Weeks (End of Study) |
| Change From Baseline In European Quality Of Life (EuroQoL) Health 5-Dimension Questionnaire (EQ-5D) Visual Analogue Scale At End Of Study | The EuroQoL EQ-5D is a generic, standardized, self-administered instrument that provides a simple, descriptive profile and a single index value for health status. Assessments were made using the EQ-5D Visual Analogue Scale, which captures the self-rating of current health status using a visual "thermometer" with the endpoints of 100 (best imaginable health state) at the top and zero (worst imaginable health state) at the bottom. An increase in score indicates improvement. | Baseline, Up To 211 Weeks (End of Study) |
| Change From Baseline In EuroQoL EQ-5D Index Score At End Of Study | The EuroQoL EQ-5D is a generic, standardized, self-administered instrument that provides a simple, descriptive profile and a single index value for health status. Index scores range from less than 0 to 1, with higher scores representing a better health status. | Baseline, Up To 211 Weeks (End of Study) |
| Carlsbad |
| California |
| 92011 |
| United States |
| Georgetown University Hospital | Washington D.C. | District of Columbia | 20007 | United States |
| University of Miami McKnight Brain Institute | Miami | Florida | 33136 | United States |
| Neurological Services of Orlando | Orlando | Florida | 32806 | United States |
| Fort Wayne Neurological Center | Fort Wayne | Indiana | 46804 | United States |
| University of Kansas Medical Center | Kansas City | Kansas | 66160 | United States |
| Baptist Health Lexington | Lexington | Kentucky | 40503 | United States |
| University of Maryland Medical Center | Baltimore | Maryland | 21201 | United States |
| John Hopkins University School of Medicine | Baltimore | Maryland | 21287 | United States |
| Mayo Clinic - Rochester | Rochester | Minnesota | 55905 | United States |
| Washington University School of Medicine | St Louis | Missouri | 63110 | United States |
| Multiple Sclerosis Comprehensive Care Center, NYU Langone Medical Center | New York | New York | 10016 | United States |
| The Ohio State University, Wexner Medical Center, CarePoint at Gahanna | Gahanna | Ohio | 43230 | United States |
| Hospital of the University of Pennsylvania | Philadelphia | Pennsylvania | 19104 | United States |
| University of Pittsburgh | Pittsburgh | Pennsylvania | 15213 | United States |
| Multiple Sclerosis Treatment Center of Dallas | Dallas | Texas | 75246 | United States |
| University of Utah Health Care | Salt Lake City | Utah | 84132 | United States |
| Swedish Neuroscience Institute | Seattle | Washington | 98122 | United States |
| Hospital General de Agudos Juan Antonio Fernandez | Ciudad Autonoma, Buenos Aires | Buenos Aires | 1425 | Argentina |
| Hospital J. M. Ramos Mejia | Ciudad Autonoma, Buenos Aires | Buenos Aires | C1221 | Argentina |
| Hospital Universitario Austral | Pilar | Buenos Aires | B1629ODT | Argentina |
| Fundacion Rosarina de Neuro Rehabilitacion | Rosario | Santa Fe Province | S2000BZL | Argentina |
| University of Sydney, Brain and Mind Center | Camperdown | New South Wales | 2050 | Australia |
| St. Vincent's Hospital Melbourne | Fitzroy | Victoria | 3065 | Australia |
| Clinical Hospital Centre Zagreb | Zagreb | 10000 | Croatia |
| Vseobecna fakultni nemocnice Neurologicka klinika | Prague | 128 21 | Czechia |
| Århus Universitetshospital | Aarhus | 8000 | Denmark |
| Universitaetsklinikum Heidelberg, Abteilung Neuroonkologie | Heidelberg | Baden-Wurttemberg | 69120 | Germany |
| Klinikum rechts der Isar der TU Muenchen, Neurologische Klinik und Poliklinik | Munich | Bavaria | 81675 | Germany |
| Universitaetsmedizin Rostock, Klinik für Neurologie | Rostock | 18147 | Germany |
| Prince of Wales Hospital | Shatin | Hong Kong |
| Universitaria Policlinico di Catania | Catania | 95123 | Italy |
| Azienda Ospedaliera Universitaria Federico II | Naples | 80131 | Italy |
| Azienda Ospedaliera San Camillo Forlanini | Rome | 00151 | Italy |
| Neurological Centre of Latium Dipartimento di Neuroscienze | Rome | 00178 | Italy |
| Chiba University Hospital | Chiba | Chiba | 260-8677 | Japan |
| Hyogo College of Medicine Hospital | Nishinomiya-shi | Hyōgo | 663-8501 | Japan |
| Kyoto Min-iren Chuo Hospital | Kyoto | Kyoto | 604-8453 | Japan |
| Tohoku University Hospital | Sendai | Miyagi | 980-8574 | Japan |
| Tokyo Medical and Dental University | Bunkyo-ku | Tokyo | 113-8519 | Japan |
| Yamaguchi University Hospital | Ube-shi | Yamaguchi | 755-8505 | Japan |
| Kyushu University Hospital | Fukuoka | 812-8582 | Japan |
| National Center Hospital, NCNP | Tokyo | 187-8551 | Japan |
| Hospital Kuala Lumpur | Kuala Lumpur | 50586 | Malaysia |
| Republican Clinical Hospital for Rehabilitation of Healthcare Ministry of Republic of Tatarstan | Kazan' | 420021 | Russia |
| FSBHI 'Siberian Clinical Center of FMBA' | Krasnoyarsk | 630037 | Russia |
| Federal State Budget Institution of Healthcare - Siberian District Medical Center of FMBA of Russia | Novosibirsk | 630067 | Russia |
| SBEIHPE "Rostov SMU of MoH of RF" | Rostov-on-Don | 344022 | Russia |
| First Pavlov State Medical University of St.Petersburg | Saint Petersburg | 197022 | Russia |
| National Cancer Center | Goyang-si | Gyeonggi-do | 10408 | South Korea |
| Korea University Anam Hospital | Seoul | 02841 | South Korea |
| Seoul University National Hospital | Seoul | 03080 | South Korea |
| Severance Hospital, Yonsei University | Seoul | 03722 | South Korea |
| Samsung Medical Center | Seoul | 06351 | South Korea |
| Hospital de Cruces | Barakaldo | Bizkaia | 48903 | Spain |
| Hospital Universitario Reina Sofia | Córdoba | 14011 | Spain |
| Hospital Universitario Clinico San Carlos | Madrid | 28040 | Spain |
| Cheng Hsin General Hospital | Taipei | 112 | Taiwan |
| Navamindradhiraj University, Vajira Hospital | Dusit | 10300 | Thailand |
| Thammasat University Hospital | Pathum Thani | 12120 | Thailand |
| Sunprasitthiprasong Hospital | Ubon Ratchathani | 34000 | Thailand |
| Hacettepe University Medical Faculty | Ankara | 06100 | Turkey (Türkiye) |
| Istanbul University Cerrahpasa Medical Faculty | Istanbul | 34098 | Turkey (Türkiye) |
| Istanbul Bilim Universty Medical Fac. | Istanbul | 34381 | Turkey (Türkiye) |
| Dokuz Eylul University Medicine Faculty | Izmir | 35340 | Turkey (Türkiye) |
| Kocaeli University Medical Faculty | Kocaeli | 41380 | Turkey (Türkiye) |
| Ondokuz Mayis Univ. Med. Fac. | Samsun | 55139 | Turkey (Türkiye) |
| The Walton Centre | Liverpool | L9 7LJ | United Kingdom |
| John Radcliffe Hospital | Oxford | OX3 9DU | United Kingdom |
| Pittock SJ, Berthele A, Fujihara K, Kim HJ, Levy M, Palace J, Nakashima I, Terzi M, Totolyan N, Viswanathan S, Wang KC, Pace A, Fujita KP, Armstrong R, Wingerchuk DM. Eculizumab in Aquaporin-4-Positive Neuromyelitis Optica Spectrum Disorder. N Engl J Med. 2019 Aug 15;381(7):614-625. doi: 10.1056/NEJMoa1900866. Epub 2019 May 3. |
| 34803867 | Derived | Singh P, Gao X, Kleijn HJ, Bellanti F, Pelto R. Eculizumab Pharmacokinetics and Pharmacodynamics in Patients With Neuromyelitis Optica Spectrum Disorder. Front Neurol. 2021 Nov 3;12:696387. doi: 10.3389/fneur.2021.696387. eCollection 2021. |
| 34498507 | Derived | Pittock SJ, Fujihara K, Palace J, Berthele A, Kim HJ, Oreja-Guevara C, Nakashima I, Levy M, Shang S, Yountz M, Miller L, Armstrong R, Wingerchuk DM; PREVENT Study Group. Eculizumab monotherapy for NMOSD: Data from PREVENT and its open-label extension. Mult Scler. 2022 Mar;28(3):480-486. doi: 10.1177/13524585211038291. Epub 2021 Sep 9. |
| 33676197 | Derived | Kim HJ, Nakashima I, Viswanathan S, Wang KC, Shang S, Miller L, Yountz M, Wingerchuk DM, Pittock SJ, Levy M, Berthele A, Totolyan N, Palace J, Barnett MH, Fujihara K; PREVENT Study Group. Eculizumab in Asian patients with anti-aquaporin-IgG-positive neuromyelitis optica spectrum disorder: A subgroup analysis from the randomized phase 3 PREVENT trial and its open-label extension. Mult Scler Relat Disord. 2021 May;50:102849. doi: 10.1016/j.msard.2021.102849. Epub 2021 Feb 20. |
| 33310418 | Derived | Palace J, Wingerchuk DM, Fujihara K, Berthele A, Oreja-Guevara C, Kim HJ, Nakashima I, Levy M, Terzi M, Totolyan N, Viswanathan S, Wang KC, Pace A, Yountz M, Miller L, Armstrong R, Pittock S; PREVENT Study Group. Benefits of eculizumab in AQP4+ neuromyelitis optica spectrum disorder: Subgroup analyses of the randomized controlled phase 3 PREVENT trial. Mult Scler Relat Disord. 2021 Jan;47:102641. doi: 10.1016/j.msard.2020.102641. Epub 2020 Nov 26. |
| FG001 |
| Placebo |
Induction Period: Participants received matching placebo (900 mg) via IV infusion once a week (every 7 ± 2 days) for 4 weeks, followed by matching placebo (1200 mg) for the fifth dose (Week 4). Maintenance Period: Participants received matching placebo (1200 mg) via IV infusion every 2 weeks (every 14 ± 2 days) from the sixth dose (Week 6) onwards. |
| Received At Least 1 Dose Of Study Drug | Full Analysis Set (FAS) |
|
| COMPLETED |
|
| NOT COMPLETED |
|
|
Full Analysis Set (FAS): all participants who were randomized to treatment and who received at least 1 dose of study drug.
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| ID | Title | Description |
|---|---|---|
| BG000 | Eculizumab | Induction Period: Participants received eculizumab (900 mg) via IV infusion once a week (every 7 ± 2 days) for 4 weeks followed by eculizumab 1200 mg for the fifth dose (Week 4). Maintenance Period: Participants received eculizumab (1200 mg) via IV infusion every 2 weeks (every 14 ± 2 days) from the sixth dose (Week 6) onwards. |
| BG001 | Placebo | Induction Period: Participants received matching placebo (900 mg) via IV infusion once a week (every 7 ± 2 days) for 4 weeks, followed by matching placebo (1200 mg) for the fifth dose (Week 4). Maintenance Period: Participants received matching placebo (1200 mg) via IV infusion every 2 weeks (every 14 ± 2 days) from the sixth dose (Week 6) onwards. |
| BG002 | Total | Total of all reporting groups |
| Units | Counts |
|---|---|
| Participants |
|
| Title | Description | Population Description | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Denominator Units Selected | Denominators | Classes | ||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Age, Continuous | Age at first dose (years) | Mean | Standard Deviation | years |
| ||||||||||||||
| Sex: Female, Male | Count of Participants | Participants |
| ||||||||||||||||
| Ethnicity (NIH/OMB) | Count of Participants | Participants |
| ||||||||||||||||
| Race/Ethnicity, Customized | Count of Participants | Participants |
| ||||||||||||||||
| Overall Stratification Groupings (4 strata) at Randomization | EDSS = Expanded Disability Status Scale; IST = Immunosuppressive Therapy High EDSS (≥ 2.5 to ≤ 7): Same IST = High EDSS (≥ 2.5 to ≤ 7) and Continuing on the Same IST(s) since last relapse. High EDSS (≥ 2.5 to ≤ 7): Change in IST = High EDSS (≥ 2.5 to ≤ 7) and Changes in IST(s) since last relapse. | Count of Participants | Participants |
|
| 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 | Participants With An Adjudicated On-trial Relapse | An On-trial Relapse was defined as a new onset of neurologic symptoms or worsening of existing neurologic symptoms with an objective change (clinical sign) on neurologic examination that persisted for more than 24 hours as confirmed by the treating physician. An adjudicated On-trial Relapse was defined by the protocol and positively adjudicated by the relapse adjudication committee. | Full Analysis Set (FAS): all participants who were randomized to treatment, received at least 1 dose of study drug. | Posted | Count of Participants | Participants | Baseline, Up To 211 Weeks (End of Study) |
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| Secondary | Adjudicated On-trial Annualized Relapse Rate (ARR) | The adjudicated On-trial ARR was computed as the total number of relapses divided by the total number of patient years in the study period. A central independent committee was used to adjudicate all On-trial Relapses as determined by the treating physician. Results reported as adjusted adjudicated On-trial ARR based on a Poisson regression adjusted for randomization strata and historical ARR in 24 months prior to Screening. | Full Analysis Set (FAS): all participants who were randomized to treatment and who received at least 1 dose of study drug. | Posted | Number | 95% Confidence Interval | relapses/years on study | Baseline, Up To 211 Weeks (End of Study) |
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| Secondary | Change From Baseline In EDSS At End Of Study | Disease-related disability was measured by the EDSS. The EDSS is an ordinal clinical rating scale that ranges from 0 (normal neurologic examination) to 10 (death) in half-point increments. A decrease in score indicates improvement. | Full Analysis Set (FAS): all participants who were randomized to treatment and who received at least 1 dose of study drug. | Posted | Mean | Standard Deviation | units on a scale | Baseline, Up To 211 Weeks (End of Study) |
| ||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
| Secondary | Change From Baseline In Modified Rankin Scale (mRS) Score At End Of Study | Disease-related disability was measured by the mRS score. The mRS is a commonly used scale for measuring the degree of disability or dependence in the daily activities of people who have suffered from a neurological disability. The scale ranges from 0 (no disability) to 6 (death) in whole-point increments. A decrease in score indicates improvement. | Full Analysis Set (FAS): all participants who were randomized to treatment and who received at least 1 dose of study drug. | Posted | Mean | Standard Deviation | units on a scale | Baseline, Up To 211 Weeks (End of Study) |
| ||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
| Secondary | Change From Baseline In Hauser Ambulation Index (HAI) Score At End of Study | The HAI evaluates gait and was used to assess the time and effort used by the participant to walk 25 feet (8 meters). The scale ranges from 0 to 9, with 0 being the best score (asymptomatic; fully ambulatory with no assistance) and 9 being the worst (restricted to wheel chair; unable to transfer self independently). A decrease in score indicates improvement. | Full Analysis Set (FAS): all participants who were randomized to treatment and who received at least 1 dose of study drug. | Posted | Mean | Standard Deviation | units on a scale | Baseline, Up To 211 Weeks (End of Study) |
| ||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
| Secondary | Change From Baseline In European Quality Of Life (EuroQoL) Health 5-Dimension Questionnaire (EQ-5D) Visual Analogue Scale At End Of Study | The EuroQoL EQ-5D is a generic, standardized, self-administered instrument that provides a simple, descriptive profile and a single index value for health status. Assessments were made using the EQ-5D Visual Analogue Scale, which captures the self-rating of current health status using a visual "thermometer" with the endpoints of 100 (best imaginable health state) at the top and zero (worst imaginable health state) at the bottom. An increase in score indicates improvement. | Full Analysis Set (FAS): all participants who were randomized to treatment and who received at least 1 dose of study drug. | Posted | Mean | Standard Deviation | units on a scale | Baseline, Up To 211 Weeks (End of Study) |
| ||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
| Secondary | Change From Baseline In EuroQoL EQ-5D Index Score At End Of Study | The EuroQoL EQ-5D is a generic, standardized, self-administered instrument that provides a simple, descriptive profile and a single index value for health status. Index scores range from less than 0 to 1, with higher scores representing a better health status. | Full Analysis Set (FAS): all participants who were randomized to treatment and who received at least 1 dose of study drug. | Posted | Mean | Standard Deviation | units on a scale | Baseline, Up To 211 Weeks (End of Study) |
|
From Day 1 to End of Study (211 Weeks)
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| 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 Period: Participants received eculizumab (900 mg) via IV infusion once a week (every 7 ± 2 days) for 4 weeks followed by eculizumab 1200 mg for the fifth dose (Week 4). Maintenance Period: Participants received eculizumab (1200 mg) via IV infusion every 2 weeks (every 14 ± 2 days) from the sixth dose (Week 6) onwards. | 1 | 96 | 30 | 96 | 86 | 96 |
| EG001 | Placebo | Induction Period: Participants received matching placebo (900 mg) via IV infusion once a week (every 7 ± 2 days) for 4 weeks, followed by matching placebo (1200 mg) for the fifth dose (Week 4). Maintenance Period: Participants received matching placebo (1200 mg) via IV infusion every 2 weeks (every 14 ± 2 days) from the sixth dose (Week 6) onwards. | 0 | 47 | 26 | 47 | 43 | 47 |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Leukocytosis | Blood and lymphatic system disorders | MedDRA 21.0 | Systematic Assessment |
| |
| Pancytopenia | Blood and lymphatic system disorders | MedDRA 21.0 | Systematic Assessment |
| |
| Thrombocytopenia | Blood and lymphatic system disorders | MedDRA 21.0 | Systematic Assessment |
| |
| Atrial fibrillation | Cardiac disorders | MedDRA 21.0 | Systematic Assessment |
| |
| Cardiac failure congestive | Cardiac disorders | MedDRA 21.0 | Systematic Assessment |
| |
| Myocardial ischaemia | Cardiac disorders | MedDRA 21.0 | Systematic Assessment |
| |
| Vertigo | Ear and labyrinth disorders | MedDRA 21.0 | Systematic Assessment |
| |
| Cataract | Eye disorders | MedDRA 21.0 | Systematic Assessment |
| |
| Conjunctival haemorrhage | Eye disorders | MedDRA 21.0 | Systematic Assessment |
| |
| Visual impairment | Eye disorders | MedDRA 21.0 | Systematic Assessment |
| |
| Abdominal pain | Gastrointestinal disorders | MedDRA 21.0 | Systematic Assessment |
| |
| Abdominal pain upper | Gastrointestinal disorders | MedDRA 21.0 | Systematic Assessment |
| |
| Nausea | Gastrointestinal disorders | MedDRA 21.0 | Systematic Assessment |
| |
| Pancreatitis | Gastrointestinal disorders | MedDRA 21.0 | Systematic Assessment |
| |
| Umbilical hernia | Gastrointestinal disorders | MedDRA 21.0 | Systematic Assessment |
| |
| Vomiting | Gastrointestinal disorders | MedDRA 21.0 | Systematic Assessment |
| |
| Pain | General disorders | MedDRA 21.0 | Systematic Assessment |
| |
| Pyrexia | General disorders | MedDRA 21.0 | Systematic Assessment |
| |
| Cholecystitis acute | Hepatobiliary disorders | MedDRA 21.0 | Systematic Assessment |
| |
| Appendicitis | Infections and infestations | MedDRA 21.0 | Systematic Assessment |
| |
| Bartholin's abscess | Infections and infestations | MedDRA 21.0 | Systematic Assessment |
| |
| Bronchitis | Infections and infestations | MedDRA 21.0 | Systematic Assessment |
| |
| Cellulitis | Infections and infestations | MedDRA 21.0 | Systematic Assessment |
| |
| Gallbladder empyema | Infections and infestations | MedDRA 21.0 | Systematic Assessment |
| |
| Gastroenteritis viral | Infections and infestations | MedDRA 21.0 | Systematic Assessment |
| |
| Herpes zoster | Infections and infestations | MedDRA 21.0 | Systematic Assessment |
| |
| Infectious pleural effusion | Infections and infestations | MedDRA 21.0 | Systematic Assessment |
| |
| Influenza | Infections and infestations | MedDRA 21.0 | Systematic Assessment |
| |
| Pneumococcal infection | Infections and infestations | MedDRA 21.0 | Systematic Assessment |
| |
| Pneumonia | Infections and infestations | MedDRA 21.0 | Systematic Assessment |
| |
| Renal abscess | Infections and infestations | MedDRA 21.0 | Systematic Assessment |
| |
| Sepsis | Infections and infestations | MedDRA 21.0 | Systematic Assessment |
| |
| Urinary tract infection | Infections and infestations | MedDRA 21.0 | Systematic Assessment |
| |
| Viral upper respiratory tract infection | Infections and infestations | MedDRA 21.0 | Systematic Assessment |
| |
| Cervical vertebral fracture | Injury, poisoning and procedural complications | MedDRA 21.0 | Systematic Assessment |
| |
| Contusion | Injury, poisoning and procedural complications | MedDRA 21.0 | Systematic Assessment |
| |
| Fall | Injury, poisoning and procedural complications | MedDRA 21.0 | Systematic Assessment |
| |
| Hip fracture | Injury, poisoning and procedural complications | MedDRA 21.0 | Systematic Assessment |
| |
| Pubis fracture | Injury, poisoning and procedural complications | MedDRA 21.0 | Systematic Assessment |
| |
| Rib fracture | Injury, poisoning and procedural complications | MedDRA 21.0 | Systematic Assessment |
| |
| Back pain | Musculoskeletal and connective tissue disorders | MedDRA 21.0 | Systematic Assessment |
| |
| Muscular weakness | Musculoskeletal and connective tissue disorders | MedDRA 21.0 | Systematic Assessment |
| |
| Pain in extremity | Musculoskeletal and connective tissue disorders | MedDRA 21.0 | Systematic Assessment |
| |
| Adenocarcinoma | Neoplasms benign, malignant and unspecified (incl cysts and polyps) | MedDRA 21.0 | Systematic Assessment |
| |
| Myelitis transverse | Nervous system disorders | MedDRA 21.0 | Systematic Assessment |
| |
| Neuromyelitis optica spectrum disorder | Nervous system disorders | MedDRA 21.0 | Systematic Assessment |
| |
| Paraesthesia | Nervous system disorders | MedDRA 21.0 | Systematic Assessment |
| |
| Somnolence | Nervous system disorders | MedDRA 21.0 | Systematic Assessment |
| |
| Syncope | Nervous system disorders | MedDRA 21.0 | Systematic Assessment |
| |
| Confusional state | Psychiatric disorders | MedDRA 21.0 | Systematic Assessment |
| |
| Panic attack | Psychiatric disorders | MedDRA 21.0 | Systematic Assessment |
| |
| Haematuria | Renal and urinary disorders | MedDRA 21.0 | Systematic Assessment |
| |
| Cough | Respiratory, thoracic and mediastinal disorders | MedDRA 21.0 | Systematic Assessment |
| |
| Dyspnoea | Respiratory, thoracic and mediastinal disorders | MedDRA 21.0 | Systematic Assessment |
| |
| Dyspnoea exertional | Respiratory, thoracic and mediastinal disorders | MedDRA 21.0 | Systematic Assessment |
| |
| Pleurisy | Respiratory, thoracic and mediastinal disorders | MedDRA 21.0 | Systematic Assessment |
| |
| Pulmonary embolism | Respiratory, thoracic and mediastinal disorders | MedDRA 21.0 | Systematic Assessment |
| |
| Respiratory disorder | Respiratory, thoracic and mediastinal disorders | MedDRA 21.0 | Systematic Assessment |
| |
| Respiratory failure | Respiratory, thoracic and mediastinal disorders | MedDRA 21.0 | Systematic Assessment |
| |
| Urinary retention | Renal and urinary disorders | MedDRA 21.0 | Systematic Assessment |
| |
| Catheterisation venous | Surgical and medical procedures | MedDRA 21.0 | Systematic Assessment |
| |
| Orthostatic hypotension | Vascular disorders | MedDRA 21.0 | Systematic Assessment |
| |
| Superior mesenteric artery syndrome | Vascular disorders | MedDRA 21.0 | Systematic Assessment |
| |
| Venous occlusion | Vascular disorders | MedDRA 21.0 | Systematic Assessment |
|
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Leukopenia | Blood and lymphatic system disorders | MedDRA 21.0 | Systematic Assessment |
| |
| Lymphopenia | Blood and lymphatic system disorders | MedDRA 21.0 | Systematic Assessment |
| |
| Cataract | Eye disorders | MedDRA 21.0 | Systematic Assessment |
| |
| Abdominal pain upper | Gastrointestinal disorders | MedDRA 21.0 | Systematic Assessment |
| |
| Constipation | Gastrointestinal disorders | MedDRA 21.0 | Systematic Assessment |
| |
| Diarrhoea | Gastrointestinal disorders | MedDRA 21.0 | Systematic Assessment |
| |
| Dyspepsia | Gastrointestinal disorders | MedDRA 21.0 | Systematic Assessment |
| |
| Gastrooesophageal reflux disease | Gastrointestinal disorders | MedDRA 21.0 | Systematic Assessment |
| |
| Nausea | Gastrointestinal disorders | MedDRA 21.0 | Systematic Assessment |
| |
| Vomiting | Gastrointestinal disorders | MedDRA 21.0 | Systematic Assessment |
| |
| Asthenia | General disorders | MedDRA 21.0 | Systematic Assessment |
| |
| Chest discomfort | General disorders | MedDRA 21.0 | Systematic Assessment |
| |
| Fatigue | General disorders | MedDRA 21.0 | Systematic Assessment |
| |
| Oedema peripheral | General disorders | MedDRA 21.0 | Systematic Assessment |
| |
| Pain | General disorders | MedDRA 21.0 | Systematic Assessment |
| |
| Pyrexia | General disorders | MedDRA 21.0 | Systematic Assessment |
| |
| Bronchitis | Infections and infestations | MedDRA 21.0 | Systematic Assessment |
| |
| Conjunctivitis | Infections and infestations | MedDRA 21.0 | Systematic Assessment |
| |
| Cystitis | Infections and infestations | MedDRA 21.0 | Systematic Assessment |
| |
| Hordeolum | Infections and infestations | MedDRA 21.0 | Systematic Assessment |
| |
| Influenza | Infections and infestations | MedDRA 21.0 | Systematic Assessment |
| |
| Nasopharyngitis | Infections and infestations | MedDRA 21.0 | Systematic Assessment |
| |
| Pharyngitis | Infections and infestations | MedDRA 21.0 | Systematic Assessment |
| |
| Pneumonia | Infections and infestations | MedDRA 21.0 | Systematic Assessment |
| |
| Sinusitis | Infections and infestations | MedDRA 21.0 | Systematic Assessment |
| |
| Upper respiratory tract infection | Infections and infestations | MedDRA 21.0 | Systematic Assessment |
| |
| Urinary tract infection | Infections and infestations | MedDRA 21.0 | Systematic Assessment |
| |
| Contusion | Injury, poisoning and procedural complications | MedDRA 21.0 | Systematic Assessment |
| |
| Fall | Injury, poisoning and procedural complications | MedDRA 21.0 | Systematic Assessment |
| |
| Weight decreased | Investigations | MedDRA 21.0 | Systematic Assessment |
| |
| Decreased appetite | Metabolism and nutrition disorders | MedDRA 21.0 | Systematic Assessment |
| |
| Arthralgia | Musculoskeletal and connective tissue disorders | MedDRA 21.0 | Systematic Assessment |
| |
| Back pain | Musculoskeletal and connective tissue disorders | MedDRA 21.0 | Systematic Assessment |
| |
| Muscle spasms | Musculoskeletal and connective tissue disorders | MedDRA 21.0 | Systematic Assessment |
| |
| Musculoskeletal pain | Musculoskeletal and connective tissue disorders | MedDRA 21.0 | Systematic Assessment |
| |
| Myalgia | Musculoskeletal and connective tissue disorders | MedDRA 21.0 | Systematic Assessment |
| |
| Pain in extremity | Musculoskeletal and connective tissue disorders | MedDRA 21.0 | Systematic Assessment |
| |
| Dizziness | Nervous system disorders | MedDRA 21.0 | Systematic Assessment |
| |
| Headache | Nervous system disorders | MedDRA 21.0 | Systematic Assessment |
| |
| Hypoaesthesia | Nervous system disorders | MedDRA 21.0 | Systematic Assessment |
| |
| Paraesthesia | Nervous system disorders | MedDRA 21.0 | Systematic Assessment |
| |
| Depression | Psychiatric disorders | MedDRA 21.0 | Systematic Assessment |
| |
| Insomnia | Psychiatric disorders | MedDRA 21.0 | Systematic Assessment |
| |
| Cough | Respiratory, thoracic and mediastinal disorders | MedDRA 21.0 | Systematic Assessment |
| |
| Nasal congestion | Respiratory, thoracic and mediastinal disorders | MedDRA 21.0 | Systematic Assessment |
| |
| Oropharyngeal pain | Respiratory, thoracic and mediastinal disorders | MedDRA 21.0 | Systematic Assessment |
| |
| Rhinitis allergic | Respiratory, thoracic and mediastinal disorders | MedDRA 21.0 | Systematic Assessment |
| |
| Alopecia | Skin and subcutaneous tissue disorders | MedDRA 21.0 | Systematic Assessment |
| |
| Pruritus | Skin and subcutaneous tissue disorders | MedDRA 21.0 | Systematic Assessment |
| |
| Rash | Skin and subcutaneous tissue disorders | MedDRA 21.0 | Systematic Assessment |
|
After rigorous review of blinded study data, the Sponsor terminated the study at 23 adjudicated events, not the protocol-specified 24. This was not driven by safety or efficacy concerns, but rather by uncertainty in estimating final event occurrence.
Publication rights are tied to the completion of the multi-center publication.
| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Alexion Pharmaceuticals, Inc. | Alexion Pharmaceuticals, Inc. | 855-752-2356 | clinicaltrials@alexion.com |
| Prot_000.pdf |
| SAP | No | Yes | No | Statistical Analysis Plan | Jun 1, 2018 | Jun 7, 2019 | SAP_001.pdf |
| ID | Term |
|---|---|
| D009471 | Neuromyelitis Optica |
| D009188 | Myelitis, Transverse |
| D009902 | Optic Neuritis |
| D012008 | Recurrence |
| D003711 | Demyelinating Diseases |
| ID | Term |
|---|---|
| D020278 | Demyelinating Autoimmune Diseases, CNS |
| D020274 | Autoimmune Diseases of the Nervous System |
| D009422 | Nervous System Diseases |
| D009901 | Optic Nerve Diseases |
| D003389 | Cranial Nerve Diseases |
| D005128 | Eye Diseases |
| D001327 | Autoimmune Diseases |
| D007154 | Immune System Diseases |
| D009187 | Myelitis |
| D002494 | Central Nervous System Infections |
| D007239 | Infections |
| D020361 | Paraneoplastic Syndromes, Nervous System |
| D009423 | Nervous System Neoplasms |
| D009371 | Neoplasms by Site |
| D009369 | Neoplasms |
| D010257 | Paraneoplastic Syndromes |
| D002493 | Central Nervous System Diseases |
| D013118 | Spinal Cord Diseases |
| D019636 | Neurodegenerative Diseases |
| D000090862 | Neuroinflammatory Diseases |
| D020969 | Disease Attributes |
| D010335 | Pathologic Processes |
| D013568 | Pathological Conditions, Signs and Symptoms |
Not provided
Not provided
| ID | Term |
|---|---|
| C481642 | eculizumab |
Not provided
Not provided
Not provided
| Male |
|
| Not Hispanic or Latino |
|
| Unknown or Not Reported |
|
| Black or African American |
|
| White |
|
| Other or Unknown |
|
| High EDSS (≥ 2.5 to ≤ 7) and Treatment Naive |
|
| High EDSS (≥ 2.5 to ≤ 7): Same IST |
|
| High EDSS (≥ 2.5 to ≤ 7): Change in IST |
|
|
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