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| ID | Type | Description | Link |
|---|---|---|---|
| 2013-001151-12 | EudraCT Number |
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The purpose of this study is to determine whether eculizumab long-term use is safe and effective in patients with relapsing NMO.
This study is an open label extension study to confirm the long term safety and efficacy of eculizumab in subjects with relapsing NMO who have completed the initial double-blind, randomized, placebo-controlled trial ECU-NMO-301.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Eculizumab | Experimental | Eculizumab intravenous infusion every two weeks. |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| eculizumab | Biological |
|
|
| Measure | Description | Time Frame |
|---|---|---|
| Number of Participants With Treatment Emergent Adverse Events and Serious Adverse Events | An adverse event (AE) was defined as any unfavorable and unintended sign (including an abnormal laboratory finding, for example), symptom, or disease temporally associated with the use of a medicinal product, whether or not considered related to the medicinal product. Treatment-emergent adverse events (TEAEs) were defined as an AE with onset on or after the first study drug dose in Study ECU-NMO-302. A serious adverse event (SAE) was defined as an untoward medical occurrence that at any dose either results in death, is life-threatening, requires inpatient hospitalization or prolongation of existing hospitalization, results in persistent or significant disability/incapacity, is a congenital anomaly/birth defect, or is an important medical event. A summary of serious and all other non-serious adverse events, regardless of causality, is located in the Reported Adverse Events module. | Baseline up to end of study (up to 6.5 years) |
| Number of Participants With At Least 1 Post Baseline Columbia-Suicide Severity Rating Scale (C-SSRS) Assessment (Suicide-Related Thoughts or Behaviours) Abnormality | The C-SSRS is a validated questionnaire to capture occurrence, severity, and frequency of suicide-related thoughts and behaviours, and has a binary response (yes/no). Suicidal Ideation: a "yes" answer to any one of 5 suicidal ideation questions: Wish to be Dead; Non-specific Active Suicidal Thoughts; Active Suicidal Ideation with Any Methods (Not Planned) without Intent to Act; Active Suicidal Ideation with Some Intent to Act, without Specific Plan; and Active Suicidal Ideation with Specific Plan and Intent. Suicidal Behaviour: a "yes" answer to any of 5 suicidal behaviour questions: Preparatory Acts or Behaviour, Aborted Attempt, Interrupted Attempt, Actual Attempt (non-fatal), and Completed Suicide. Suicidal Ideation or Behaviour: a "yes" answer to the following question: Self-injurious behaviour without suicidal intent. | Baseline up to end of study (up to 6.5 years) |
| Number of Participants With An On-trial Relapse as Determined by The Treating Physician | 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. |
| Measure | Description | Time Frame |
|---|---|---|
| Change From Baseline in Expanded Disability Status Scale (EDSS) Score | Disease-related disability was measured by the EDSS. The EDSS quantifies disability in 8 Functional Systems (FS) and allows neurologists to assign a Functional System Score (FSS) in each of these. The Functional Systems are pyramidal, cerebellar, brainstem, sensory, bowel and bladder, visual, cerebral, and other. 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 was defined as the last available assessment prior to the first study drug infusion in Study ECU-NMO-302. |
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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. | |
| 34498507 | Derived |
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Participants who completed Study ECU-NMO-301 (NCT01892345) were eligible to participate in Study ECU-NMO-302. This is an open-label study in which all participants were administered intravenous eculizumab. However, to maintain the blind of Study ECU-NMO-301, all participants underwent a 4-week Blind Induction Phase before entering the Open-label Maintenance Phase.
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| ID | Title | Description |
|---|---|---|
| FG000 | Placebo/Eculizumab | Blind Induction Phase: Participants who had received blinded treatment with placebo in Study ECU-NMO-301 were administered eculizumab (900 milligrams [mg]) plus matching placebo via intravenous (IV) infusion on Day 1 and Weeks 1 through 3. Open-Label Maintenance Phase: Participants received open-label eculizumab (1200 mg) via IV infusion every 2 weeks starting at Week 4 and continued for up to 6.5 years. |
| Title | Milestones | Reasons Not Completed | |||||
|---|---|---|---|---|---|---|---|
| Blind Induction Phase |
|
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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 | Mar 22, 2018 | Jun 13, 2022 |
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| Baseline up to end of study (up to 6.5 years) |
| On-Trial Annualized Relapse Rate (ARR) as Determined by The Treating Physician | The On-trial ARR was computed as the total number of relapses divided by the total number of participant years in the study period. | Baseline up to end of study (up to 6.5 years) |
| Baseline, Weeks 52, 104 and 156 |
| Change From Baseline in Modified Rankin Scale (mRS) Score | 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 symptoms at all) to 6 (death) in whole-point increments. A decrease in score indicates improvement. Baseline was defined as the last available assessment prior to the first study drug infusion in Study ECU-NMO-302. | Baseline, Weeks 52, 104 and 156 |
| Change From Baseline in Hauser Ambulation Index (HAI) in Participants With Abnormal Baseline Ambulatory Function | 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 active) and 9 being the worst (restricted to wheelchair; unable to transfer self independently). A decrease in score indicates improvement. Baseline is defined as the last available assessment prior to the first study drug infusion in Study ECU-NMO-302. | Baseline, Weeks 52, 104 and 156 |
| Change From Baseline in European Quality of Life (EuroQoL) 5-Dimension Visual Analog Scale (EQ-5D VAS) Health Status Score | The EQ-5D is a generic, standardized participant self-administered health status instrument. EQ-5D general health status can also be measured by a visual analog scale (EQ-5D VAS). EQ-5D-VAS recorded the participant's self-rated health on a vertical visual analog scale (VAS) that allowed the participants to indicate their health state that ranged from 0 (worst imaginable) to 100 (best imaginable). Baseline is defined as the last available assessment prior to the first study drug infusion in Study ECU-NMO-302. | Baseline, Weeks 52, 104 and 156 |
| Change From Baseline in Kurtzke Visual Functional System Scores (FSS) in Participants With Abnormal Baseline Visual Function | The EDSS assesses multiple Kurtzke functional systems in the context of a standard neurological exam, including visual function. The visual score ranges from 0 to 6. A score of 0 implies the participant has normal visual function. Higher scores represent worse disability. Baseline is defined as the last available assessment prior to the first study drug infusion in Study EC-NMO-302. | Baseline, Weeks 52, 104 and 156 |
| Oceanside |
| California |
| 92056 |
| 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 |
| Allied Physicians Inc. of Fort Wayne | Fort Wayne | Indiana | 46805 | United States |
| University of Kansas Medical Center | Kansas City | Kansas | 66160 | United States |
| Baptist Health Lexington | Nicholasville | Kentucky | 40503 | United States |
| Johns Hopkins University Medical Center | 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 |
| Mount Sinai School of Medicine | New York | New York | 10029 | United States |
| Ohio Health Reserach Institute | Columbus | Ohio | 43214 | United States |
| University of Pennsylvania School of Medicine | Philadelphia | Pennsylvania | 19104 | United States |
| University of Pittsburgh | Pittsburgh | Pennsylvania | 15232 | United States |
| Multiple Sclerosis Treatment Center of Dallas | Dallas | Texas | 75246 | United States |
| The University of Texas Health Science | San Antonio | Texas | 78229 | United States |
| University of Utah | Salt Lake City | Utah | 84108 | United States |
| Hospital Universitario Austral | Pilar | Buenos Aires | B1629ODT | Argentina |
| Fundacion Rosarina de Neuro Rehabilitacion | Rosario | Santa Fe Province | S2000BZL | Argentina |
| Hospital General de Agudos Juan Antonio Fernandez | Ciudad Autonoma, Buenos Aires | C1425AGP | Argentina |
| Hospital General de Agudos Dr. J. M. Ramos Mejia | Ciudad Autonoma, Buenos Aires, | C1221ADC | Argentina |
| Brain and Mind Research Institute | Camperdown | New South Wales | Australia |
| St. Vincent's Hospital | Fitzroy | Victoria | 3065 | Australia |
| The Ottawa Hospital | Ottawa | Ontario | K1H 8L6 | Canada |
| Fundacion Cardiovascular de Colombia | Floridablanca | Santander Department | Colombia |
| Clinical Hospital Centre Zagreb | Zagreb | 10000 | Croatia |
| VFN v Praze | Prague | Czechia |
| Krajska zdravotni, a.s. - Nemocnice | Teplice | 415 01 | Czechia |
| Århus Universitetshospital | Aarhus | 8000 | Denmark |
| University Hospital Heidelberg | Heidelberg | Baden-Wurttemberg | 69120 | Germany |
| Neurologische Klinik und Poliklinik | Munich | Bavaria | 81675 | Germany |
| University Hospital Heinrich Heine University | Düsseldorf | North Rhine-Westphalia | 40225 | Germany |
| Universitaetsmedizin Rostock | Rostock | 18147 | Germany |
| Prince of Wales Hospital | Shatin | Hong Kong |
| Policlinico di Catania | Catania | 95123 | Italy |
| Azienda Ospedaliera Universitaria | Naples | 80131 | Italy |
| Azienda Ospedaliera di Padova | Padova | 35128 | Italy |
| Azienda Ospedaliera Sant'Andrea-Università di Roma La Sapienza | Rome | 00178 | Italy |
| Chiba University Hospital | Chiba | Chiba | 260-8677 | Japan |
| Hyogo College of Medicine Hospital | Nishinomiya-shi | HyogoKen | Japan |
| Kyoto Min-iren Chuo Hospital | Kyoto | Kyoto | 604-8453 | Japan |
| Tohoku University Hospital | Sendai | Miyagi | 980-8574 | Japan |
| Yamaguchi University Hospital | Ube-shi | Yamaguchi | 755-8505 | Japan |
| Kyushu University Hospital | Fukuoka | 812-8582 | Japan |
| National Center Hospital, NCNP | Tokyo | Japan |
| Hospital Umum Sarawak | Kuching | Sarawak | 93586 | Malaysia |
| Hospital Kuala Lumpur | Kuala Lumpur | 50586 | Malaysia |
| Republican Clinical Hospital for Rehabilitation of Healthcare Ministry of Republic of Tatarstan | Kazan' | 420021 | Russia |
| SBEI "Krasnoyarsk SMU n.a. Prof. V.F. Voyno-Yasenetsky" | Krasnoyarsk | 660037 | Russia |
| Federal State Budget Institution of Healthcare - Siberian District Medical Center of FMBA of Russia | Novosibirsk | 630068 | Russia |
| SEIHPE "Rostov SMU of MoH of RF" | Rostov-on-Don | Russia |
| National Cancer Center | Goyang-si | Gyeonggi-do | 410-769 | South Korea |
| Seoul University National Hospital | Seoul | 110744 | South Korea |
| Samsung Medical Center | Seoul | 135-710 | South Korea |
| Korea University Anam Hospital | Seoul | 136-705 | South Korea |
| Severance Hospital, Yonsei University | Seoul | 136-705 | South Korea |
| Hospital de Cruces | Barakaldo | Bizkaia | 48903 | Spain |
| Hospital Universitario Reina Sofia | Córdoba | 14404 | Spain |
| Hospital Universitario Clinico San Carlos | Madrid | 28040 | Spain |
| Cheng Hsin General Hospital | Taipei | Taiwan |
| Thammasat University Hospital | Pathum Thani | Thailand |
| Hacettepe University Medical Faculty | Ankara | 06100 | Turkey (Türkiye) |
| Trakya University Medical Faculty | Edirne | Turkey (Türkiye) |
| Dokuz Eylul University Medicine Faculty | Izmir | 35340 | Turkey (Türkiye) |
| Kocaeli University Medical Faculty | Kocaeli | Turkey (Türkiye) |
| Ondokuz Mayis Univ. Med. Fac. | Samsun | Turkey (Türkiye) |
| The Walton Centre | Liverpool | L97LJ | United Kingdom |
| 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. |
| FG001 | Eculizumab/Eculizumab | Blind Induction Phase: Participants who had received blinded treatment with eculizumab in Study ECU-NMO-301 were administered eculizumab (1200 mg) via IV infusion on Day 1 and Week 2 and placebo at Weeks 1 and 3. Open-Label Maintenance Phase: Participants received open-label eculizumab (1200 mg) via IV infusion every 2 weeks starting at Week 4 and continued for up to 6.5 years. |
| Received at Least 1 Dose of Study Drug |
|
| COMPLETED |
|
| NOT COMPLETED |
|
| Open Label Maintenance Phase |
|
|
The Extension Full Analysis Set consisted of all participants who had received at least 1 dose of eculizumab in Study ECU-NMO-302 and have a post-IP-infusion efficacy assessment.
Not provided
| ID | Title | Description |
|---|---|---|
| BG000 | Placebo/Eculizumab | Blind Induction Phase: Participants who had received blinded treatment with placebo in Study ECU-NMO-301 were administered eculizumab (900 mg) plus matching placebo via IV infusion on Day 1 and Weeks 1 through 3. Open-Label Maintenance Phase: Participants received open-label eculizumab (1200 mg) via IV infusion every 2 weeks starting at Week 4 and continued for up to 6.5 years. |
| BG001 | Eculizumab/Eculizumab | Blind Induction Phase: Participants who had received blinded treatment with eculizumab in Study ECU-NMO-301 were administered eculizumab (1200 mg) via IV infusion on Day 1 and Week 2 and placebo at Weeks 1 and 3. Open-Label Maintenance Phase: Participants received open-label eculizumab (1200 mg) via IV infusion every 2 weeks starting at Week 4 and continued for up to 6.5 years. |
| 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 | 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 |
|
| 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 | Number of Participants With Treatment Emergent Adverse Events and Serious Adverse Events | An adverse event (AE) was defined as any unfavorable and unintended sign (including an abnormal laboratory finding, for example), symptom, or disease temporally associated with the use of a medicinal product, whether or not considered related to the medicinal product. Treatment-emergent adverse events (TEAEs) were defined as an AE with onset on or after the first study drug dose in Study ECU-NMO-302. A serious adverse event (SAE) was defined as an untoward medical occurrence that at any dose either results in death, is life-threatening, requires inpatient hospitalization or prolongation of existing hospitalization, results in persistent or significant disability/incapacity, is a congenital anomaly/birth defect, or is an important medical event. A summary of serious and all other non-serious adverse events, regardless of causality, is located in the Reported Adverse Events module. | The Extension Safety Set consisted of all participants who had received at least 1 dose of eculizumab in Study ECU-NMO-302. | Posted | Count of Participants | Participants | Baseline up to end of study (up to 6.5 years) |
|
|
| ||||||||||||||||||||||||||||||||||||
| Primary | Number of Participants With At Least 1 Post Baseline Columbia-Suicide Severity Rating Scale (C-SSRS) Assessment (Suicide-Related Thoughts or Behaviours) Abnormality | The C-SSRS is a validated questionnaire to capture occurrence, severity, and frequency of suicide-related thoughts and behaviours, and has a binary response (yes/no). Suicidal Ideation: a "yes" answer to any one of 5 suicidal ideation questions: Wish to be Dead; Non-specific Active Suicidal Thoughts; Active Suicidal Ideation with Any Methods (Not Planned) without Intent to Act; Active Suicidal Ideation with Some Intent to Act, without Specific Plan; and Active Suicidal Ideation with Specific Plan and Intent. Suicidal Behaviour: a "yes" answer to any of 5 suicidal behaviour questions: Preparatory Acts or Behaviour, Aborted Attempt, Interrupted Attempt, Actual Attempt (non-fatal), and Completed Suicide. Suicidal Ideation or Behaviour: a "yes" answer to the following question: Self-injurious behaviour without suicidal intent. | The Extension Safety Set consisted of all participants who had received at least 1 dose of eculizumab in Study ECU-NMO-302. | Posted | Count of Participants | Participants | Baseline up to end of study (up to 6.5 years) |
| ||||||||||||||||||||||||||||||||||||||
| Primary | Number of Participants With An On-trial Relapse as Determined by The Treating Physician | 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. | The Extension Full Analysis Set consisted of all participants who had received at least 1 dose of eculizumab in Study ECU-NMO-302 and had a post-IP-infusion efficacy assessment. | Posted | Count of Participants | Participants | Baseline up to end of study (up to 6.5 years) |
| ||||||||||||||||||||||||||||||||||||||
| Primary | On-Trial Annualized Relapse Rate (ARR) as Determined by The Treating Physician | The On-trial ARR was computed as the total number of relapses divided by the total number of participant years in the study period. | The Extension Full Analysis Set consisted of all participants who had received at least 1 dose of eculizumab in Study ECU-NMO-302 and had a post-IP-infusion efficacy assessment. | Posted | Mean | Standard Deviation | relapses/years on study | Baseline up to end of study (up to 6.5 years) |
| |||||||||||||||||||||||||||||||||||||
| Secondary | Change From Baseline in Expanded Disability Status Scale (EDSS) Score | Disease-related disability was measured by the EDSS. The EDSS quantifies disability in 8 Functional Systems (FS) and allows neurologists to assign a Functional System Score (FSS) in each of these. The Functional Systems are pyramidal, cerebellar, brainstem, sensory, bowel and bladder, visual, cerebral, and other. 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 was defined as the last available assessment prior to the first study drug infusion in Study ECU-NMO-302. | The Extension Full Analysis Set consisted of all participants who had received at least 1 dose of eculizumab in Study ECU-NMO-302 and had a post-IP-infusion efficacy assessment. Here, Number Analyzed signifies those participants who were evaluable at specified time points. | Posted | Mean | Standard Deviation | units on a scale | Baseline, Weeks 52, 104 and 156 |
| |||||||||||||||||||||||||||||||||||||
| Secondary | Change From Baseline in Modified Rankin Scale (mRS) Score | 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 symptoms at all) to 6 (death) in whole-point increments. A decrease in score indicates improvement. Baseline was defined as the last available assessment prior to the first study drug infusion in Study ECU-NMO-302. | The Extension Full Analysis Set consisted of all participants who had received at least 1 dose of eculizumab in Study ECU-NMO-302 and had a post-IP-infusion efficacy assessment. Here, Number Analyzed signifies those participants who were evaluable at specified time points. | Posted | Mean | Standard Deviation | units on a scale | Baseline, Weeks 52, 104 and 156 |
| |||||||||||||||||||||||||||||||||||||
| Secondary | Change From Baseline in Hauser Ambulation Index (HAI) in Participants With Abnormal Baseline Ambulatory Function | 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 active) and 9 being the worst (restricted to wheelchair; unable to transfer self independently). A decrease in score indicates improvement. Baseline is defined as the last available assessment prior to the first study drug infusion in Study ECU-NMO-302. | The Extension Full Analysis Set consisted of all participants who had received at least 1 dose of eculizumab in Study ECU-NMO-302 and had a post-IP-infusion efficacy assessment. Here, Number of Participants Analyzed signifies those participants who were evaluable for this outcome measure and Number Analyzed signifies those participants who were evaluable at specified time points. | Posted | Mean | Standard Deviation | units on a scale | Baseline, Weeks 52, 104 and 156 |
| |||||||||||||||||||||||||||||||||||||
| Secondary | Change From Baseline in European Quality of Life (EuroQoL) 5-Dimension Visual Analog Scale (EQ-5D VAS) Health Status Score | The EQ-5D is a generic, standardized participant self-administered health status instrument. EQ-5D general health status can also be measured by a visual analog scale (EQ-5D VAS). EQ-5D-VAS recorded the participant's self-rated health on a vertical visual analog scale (VAS) that allowed the participants to indicate their health state that ranged from 0 (worst imaginable) to 100 (best imaginable). Baseline is defined as the last available assessment prior to the first study drug infusion in Study ECU-NMO-302. | The Extension Full Analysis Set consisted of all participants who had received at least 1 dose of eculizumab in Study ECU-NMO-302 and had a post-IP-infusion efficacy assessment. Here, Number Analyzed signifies those participants who were evaluable at specified time points. | Posted | Mean | Standard Deviation | units on a scale | Baseline, Weeks 52, 104 and 156 |
| |||||||||||||||||||||||||||||||||||||
| Secondary | Change From Baseline in Kurtzke Visual Functional System Scores (FSS) in Participants With Abnormal Baseline Visual Function | The EDSS assesses multiple Kurtzke functional systems in the context of a standard neurological exam, including visual function. The visual score ranges from 0 to 6. A score of 0 implies the participant has normal visual function. Higher scores represent worse disability. Baseline is defined as the last available assessment prior to the first study drug infusion in Study EC-NMO-302. | The Extension Full Analysis Set consisted of all participants who had received at least 1 dose of eculizumab in Study ECU-NMO-302 and had a post-IP-infusion efficacy assessment. Here, Number of Participants analyzed signifies those participants who were evaluable for this outcome measure and Number Analyzed signifies those participants who were evaluable at specified time points. | Posted | Mean | Standard Deviation | units on a scale | Baseline, Weeks 52, 104 and 156 |
|
Baseline up to end of study (up to 6.5 years)
The Extension Safety Set consisted of all participants who had received at least 1 dose of eculizumab in Study ECU-NMO-302.
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/Eculizumab | Blind Induction Phase: Participants who had received blinded treatment with placebo in Study ECU-NMO-301 were administered eculizumab (900 mg) plus matching placebo via IV infusion on Day 1 and Weeks 1 through 3. Open-Label Maintenance Phase: Participants received open-label eculizumab (1200 mg) via IV infusion every 2 weeks starting at Week 4 and continued for up to 6.5 years. | 0 | 41 | 14 | 41 | 40 | 41 |
| EG001 | Eculizumab/Eculizumab | Blind Induction Phase: Participants who had received blinded treatment with eculizumab in Study ECU-NMO-301 were administered eculizumab (1200 mg) via IV infusion on Day 1 and Week 2 and placebo at Weeks 1 and 3. Open-Label Maintenance Phase: Participants received open-label eculizumab (1200 mg) via IV infusion every 2 weeks starting at Week 4 and continued for up to 6.5 years. | 0 | 78 | 26 | 78 | 70 | 78 |
| EG002 | Eculizumab (Combined Total) | All participants who received at least 1 dose of eculizumab in the extension study. Participants received open-label eculizumab (1200 mg) every 2 weeks starting at Week 4 and continued for up to 6.5 years. | 0 | 119 | 40 | 119 | 110 | 119 |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Lymphadenopathy | Blood and lymphatic system disorders | MedDRA v24.0 | Non-systematic Assessment |
| |
| Ventricular fibrillation | Cardiac disorders | MedDRA v24.0 | Non-systematic Assessment |
| |
| Glaucoma | Eye disorders | MedDRA v24.0 | Non-systematic Assessment |
| |
| Irritable bowel syndrome | Gastrointestinal disorders | MedDRA v24.0 | Non-systematic Assessment |
| |
| Asthenia | General disorders | MedDRA v24.0 | Non-systematic Assessment |
| |
| Chest pain | General disorders | MedDRA v24.0 | Non-systematic Assessment |
| |
| Oedema peripheral | General disorders | MedDRA v24.0 | Non-systematic Assessment |
| |
| Cholecystitis acute | Hepatobiliary disorders | MedDRA v24.0 | Non-systematic Assessment |
| |
| Urinary tract infection | Infections and infestations | MedDRA v24.0 | Non-systematic Assessment |
| |
| Pneumonia | Infections and infestations | MedDRA v24.0 | Non-systematic Assessment |
| |
| Pyelonephritis acute | Infections and infestations | MedDRA v24.0 | Non-systematic Assessment |
| |
| Urosepsis | Infections and infestations | MedDRA v24.0 | Non-systematic Assessment |
| |
| Abscess limb | Infections and infestations | MedDRA v24.0 | Non-systematic Assessment |
| |
| Bacterial sepsis | Infections and infestations | MedDRA v24.0 | Non-systematic Assessment |
| |
| COVID-19 | Infections and infestations | MedDRA v24.0 | Non-systematic Assessment |
| |
| COVID-19 pneumonia | Infections and infestations | MedDRA v24.0 | Non-systematic Assessment |
| |
| Device related infection | Infections and infestations | MedDRA v24.0 | Non-systematic Assessment |
| |
| Escherichia pyelonephritis | Infections and infestations | MedDRA v24.0 | Non-systematic Assessment |
| |
| Gastroenteritis | Infections and infestations | MedDRA v24.0 | Non-systematic Assessment |
| |
| Gonorrhoea | Infections and infestations | MedDRA v24.0 | Non-systematic Assessment |
| |
| Infective tenosynovitis | Infections and infestations | MedDRA v24.0 | Non-systematic Assessment |
| |
| Osteomyelitis | Infections and infestations | MedDRA v24.0 | Non-systematic Assessment |
| |
| Pneumonia bacterial | Infections and infestations | MedDRA v24.0 | Non-systematic Assessment |
| |
| Pneumonia influenzal | Infections and infestations | MedDRA v24.0 | Non-systematic Assessment |
| |
| Psoas abscess | Infections and infestations | MedDRA v24.0 | Non-systematic Assessment |
| |
| Pyelonephritis | Infections and infestations | MedDRA v24.0 | Non-systematic Assessment |
| |
| Respiratory tract infection | Infections and infestations | MedDRA v24.0 | Non-systematic Assessment |
| |
| Staphylococcal bacteraemia | Infections and infestations | MedDRA v24.0 | Non-systematic Assessment |
| |
| Streptococcal infection | Infections and infestations | MedDRA v24.0 | Non-systematic Assessment |
| |
| Urinary tract infection enterococcal | Infections and infestations | MedDRA v24.0 | Non-systematic Assessment |
| |
| Femoral neck fracture | Injury, poisoning and procedural complications | MedDRA v24.0 | Non-systematic Assessment |
| |
| Clavicle fracture | Injury, poisoning and procedural complications | MedDRA v24.0 | Non-systematic Assessment |
| |
| Head injury | Injury, poisoning and procedural complications | MedDRA v24.0 | Non-systematic Assessment |
| |
| Spinal compression fracture | Injury, poisoning and procedural complications | MedDRA v24.0 | Non-systematic Assessment |
| |
| Wrist fracture | Injury, poisoning and procedural complications | MedDRA v24.0 | Non-systematic Assessment |
| |
| Blood pressure increased | Investigations | MedDRA v24.0 | Non-systematic Assessment |
| |
| Hypophosphataemia | Metabolism and nutrition disorders | MedDRA v24.0 | Non-systematic Assessment |
| |
| Intervertebral disc protrusion | Musculoskeletal and connective tissue disorders | MedDRA v24.0 | Non-systematic Assessment |
| |
| Osteonecrosis | Musculoskeletal and connective tissue disorders | MedDRA v24.0 | Non-systematic Assessment |
| |
| Muscular weakness | Musculoskeletal and connective tissue disorders | MedDRA v24.0 | Non-systematic Assessment |
| |
| Pain in extremity | Musculoskeletal and connective tissue disorders | MedDRA v24.0 | Non-systematic Assessment |
| |
| Spondylolisthesis | Musculoskeletal and connective tissue disorders | MedDRA v24.0 | Non-systematic Assessment |
| |
| Systemic lupus erythematosus | Musculoskeletal and connective tissue disorders | MedDRA v24.0 | Non-systematic Assessment |
| |
| Tenosynovitis | Musculoskeletal and connective tissue disorders | MedDRA v24.0 | Non-systematic Assessment |
| |
| Acrochordon | Neoplasms benign, malignant and unspecified (incl cysts and polyps) | MedDRA v24.0 | Non-systematic Assessment |
| |
| Cholesteatoma | Neoplasms benign, malignant and unspecified (incl cysts and polyps) | MedDRA v24.0 | Non-systematic Assessment |
| |
| Neuromyelitis optica spectrum disorder | Nervous system disorders | MedDRA v24.0 | Non-systematic Assessment |
| |
| Optic neuritis | Nervous system disorders | MedDRA v24.0 | Non-systematic Assessment |
| |
| Aphasia | Nervous system disorders | MedDRA v24.0 | Non-systematic Assessment |
| |
| Hemiparaesthesia | Nervous system disorders | MedDRA v24.0 | Non-systematic Assessment |
| |
| Mental impairment | Nervous system disorders | MedDRA v24.0 | Non-systematic Assessment |
| |
| Muscle spasticity | Nervous system disorders | MedDRA v24.0 | Non-systematic Assessment |
| |
| Neuromuscular blockade | Nervous system disorders | MedDRA v24.0 | Non-systematic Assessment |
| |
| Neuromyelitis optica pseudo relapse | Nervous system disorders | MedDRA v24.0 | Non-systematic Assessment |
| |
| Anembryonic gestation | Pregnancy, puerperium and perinatal conditions | MedDRA v24.0 | Non-systematic Assessment |
| |
| Catatonia | Psychiatric disorders | MedDRA v24.0 | Non-systematic Assessment |
| |
| Delirium | Psychiatric disorders | MedDRA v24.0 | Non-systematic Assessment |
| |
| Urinary retention | Renal and urinary disorders | MedDRA v24.0 | Non-systematic Assessment |
| |
| Endometriosis | Reproductive system and breast disorders | MedDRA v24.0 | Non-systematic Assessment | The N for this adverse event has been adjusted to the number of females in the study as it is a sex-specific event. |
|
| Bronchiectasis | Respiratory, thoracic and mediastinal disorders | MedDRA v24.0 | Non-systematic Assessment |
| |
| Chronic obstructive pulmonary disease | Respiratory, thoracic and mediastinal disorders | MedDRA v24.0 | Non-systematic Assessment |
| |
| Nasal septum deviation | Respiratory, thoracic and mediastinal disorders | MedDRA v24.0 | Non-systematic Assessment |
| |
| Pulmonary embolism | Respiratory, thoracic and mediastinal disorders | MedDRA v24.0 | Non-systematic Assessment |
| |
| Venous thrombosis limb | Vascular disorders | MedDRA v24.0 | Non-systematic Assessment |
|
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Anaemia | Blood and lymphatic system disorders | MedDRA v24.0 | Non-systematic Assessment |
| |
| Iron deficiency anaemia | Blood and lymphatic system disorders | MedDRA v24.0 | Non-systematic Assessment |
| |
| Diarrhoea | Gastrointestinal disorders | MedDRA v24.0 | Non-systematic Assessment |
| |
| Constipation | Gastrointestinal disorders | MedDRA v24.0 | Non-systematic Assessment |
| |
| Nausea | Gastrointestinal disorders | MedDRA v24.0 | Non-systematic Assessment |
| |
| Dyspepsia | Gastrointestinal disorders | MedDRA v24.0 | Non-systematic Assessment |
| |
| Toothache | Gastrointestinal disorders | MedDRA v24.0 | Non-systematic Assessment |
| |
| Dental caries | Gastrointestinal disorders | MedDRA v24.0 | Non-systematic Assessment |
| |
| Pyrexia | General disorders | MedDRA v24.0 | Non-systematic Assessment |
| |
| Fatigue | General disorders | MedDRA v24.0 | Non-systematic Assessment |
| |
| Oedema peripheral | General disorders | MedDRA v24.0 | Non-systematic Assessment |
| |
| Peripheral swelling | General disorders | MedDRA v24.0 | Non-systematic Assessment |
| |
| Nasopharyngitis | Infections and infestations | MedDRA v24.0 | Non-systematic Assessment |
| |
| Upper respiratory tract infection | Infections and infestations | MedDRA v24.0 | Non-systematic Assessment |
| |
| Urinary tract infection | Infections and infestations | MedDRA v24.0 | Non-systematic Assessment |
| |
| Influenza | Infections and infestations | MedDRA v24.0 | Non-systematic Assessment |
| |
| Cystitis | Infections and infestations | MedDRA v24.0 | Non-systematic Assessment |
| |
| Oral herpes | Infections and infestations | MedDRA v24.0 | Non-systematic Assessment |
| |
| Bronchitis | Infections and infestations | MedDRA v24.0 | Non-systematic Assessment |
| |
| Herpes zoster | Infections and infestations | MedDRA v24.0 | Non-systematic Assessment |
| |
| Pharyngitis | Infections and infestations | MedDRA v24.0 | Non-systematic Assessment |
| |
| Periodontitis | Infections and infestations | MedDRA v24.0 | Non-systematic Assessment |
| |
| Pneumonia | Infections and infestations | MedDRA v24.0 | Non-systematic Assessment |
| |
| Sinusitis | Infections and infestations | MedDRA v24.0 | Non-systematic Assessment |
| |
| Contusion | Injury, poisoning and procedural complications | MedDRA v24.0 | Non-systematic Assessment |
| |
| Thermal burn | Injury, poisoning and procedural complications | MedDRA v24.0 | Non-systematic Assessment |
| |
| Fall | Injury, poisoning and procedural complications | MedDRA v24.0 | Non-systematic Assessment |
| |
| Blood pressure increased | Investigations | MedDRA v24.0 | Non-systematic Assessment |
| |
| Arthralgia | Musculoskeletal and connective tissue disorders | MedDRA v24.0 | Non-systematic Assessment |
| |
| Back pain | Musculoskeletal and connective tissue disorders | MedDRA v24.0 | Non-systematic Assessment |
| |
| Pain in extremity | Musculoskeletal and connective tissue disorders | MedDRA v24.0 | Non-systematic Assessment |
| |
| Muscle spasms | Musculoskeletal and connective tissue disorders | MedDRA v24.0 | Non-systematic Assessment |
| |
| Muscular weakness | Musculoskeletal and connective tissue disorders | MedDRA v24.0 | Non-systematic Assessment |
| |
| Musculoskeletal pain | Musculoskeletal and connective tissue disorders | MedDRA v24.0 | Non-systematic Assessment |
| |
| Headache | Nervous system disorders | MedDRA v24.0 | Non-systematic Assessment |
| |
| Dizziness | Nervous system disorders | MedDRA v24.0 | Non-systematic Assessment |
| |
| Hypoaesthesia | Nervous system disorders | MedDRA v24.0 | Non-systematic Assessment |
| |
| Paraesthesia | Nervous system disorders | MedDRA v24.0 | Non-systematic Assessment |
| |
| Insomnia | Psychiatric disorders | MedDRA v24.0 | Non-systematic Assessment |
| |
| Urinary incontinence | Renal and urinary disorders | MedDRA v24.0 | Non-systematic Assessment |
| |
| Cough | Respiratory, thoracic and mediastinal disorders | MedDRA v24.0 | Non-systematic Assessment |
| |
| Oropharyngeal pain | Respiratory, thoracic and mediastinal disorders | MedDRA v24.0 | Non-systematic Assessment |
| |
| Rhinorrhoea | Respiratory, thoracic and mediastinal disorders | MedDRA v24.0 | Non-systematic Assessment |
| |
| Asthma | Respiratory, thoracic and mediastinal disorders | MedDRA v24.0 | Non-systematic Assessment |
| |
| Rash | Skin and subcutaneous tissue disorders | MedDRA v24.0 | Non-systematic Assessment |
| |
| Dermatitis | Skin and subcutaneous tissue disorders | MedDRA v24.0 | Non-systematic Assessment |
| |
| Skin lesion | Skin and subcutaneous tissue disorders | MedDRA v24.0 | Non-systematic Assessment |
|
Not provided
Not provided
| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Alexion Pharmaceuticals Inc. | Alexion Pharmaceuticals Inc. | +1 855-752-2356 | clinicaltrials@alexion.com |
| Prot_000.pdf |
| SAP | No | Yes | No | Statistical Analysis Plan | Jul 8, 2021 | Jun 13, 2022 | 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
| Pregnancy |
|
| Physician Decision |
|
| Lost to Follow-up |
|
| Adverse Event |
|
| Male |
|
| Not Hispanic or Latino |
|
| Unknown or Not Reported |
|
| Asian |
|
| Black or African American |
|
| White |
|
| Other |
|
| Unknown or Not Reported |
|
| OG001 |
| Eculizumab/Eculizumab |
Blind Induction Phase: Participants who had received blinded treatment with eculizumab in Study ECU-NMO-301 were administered eculizumab (1200 mg) via IV infusion on Day 1 and Week 2 and placebo at Weeks 1 and 3. Open-Label Maintenance Phase: Participants received open-label eculizumab (1200 mg) via IV infusion every 2 weeks starting at Week 4 and continued for up to 6.5 years. |
|
|
|
|
| Units | Counts |
|---|---|
| Participants |
|
|
Blind Induction Phase: Participants who had received blinded treatment with eculizumab in Study ECU-NMO-301 were administered eculizumab (1200 mg) via IV infusion on Day 1 and Week 2 and placebo at Weeks 1 and 3. Open-Label Maintenance Phase: Participants received open-label eculizumab (1200 mg) via IV infusion every 2 weeks starting at Week 4 and continued for up to 6.5 years.
|
|
|
|
Blind Induction Phase: Participants who had received blinded treatment with eculizumab in Study ECU-NMO-301 were administered eculizumab (1200 mg) via IV infusion on Day 1 and Week 2 and placebo at Weeks 1 and 3. Open-Label Maintenance Phase: Participants received open-label eculizumab (1200 mg) via IV infusion every 2 weeks starting at Week 4 and continued for up to 6.5 years.
|
|
Blind Induction Phase: Participants who had received blinded treatment with eculizumab in Study ECU-NMO-301 were administered eculizumab (1200 mg) via IV infusion on Day 1 and Week 2 and placebo at Weeks 1 and 3. Open-Label Maintenance Phase: Participants received open-label eculizumab (1200 mg) via IV infusion every 2 weeks starting at Week 4 and continued for up to 6.5 years.
|
|
|
|