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Trial record 1 of 2 for:    ECU-NMO-301
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An Open Label Extension Trial of Eculizumab in Relapsing NMO Patients

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ClinicalTrials.gov Identifier: NCT02003144
Recruitment Status : Completed
First Posted : December 6, 2013
Results First Posted : August 23, 2022
Last Update Posted : August 23, 2022
Sponsor:
Information provided by (Responsible Party):
Alexion Pharmaceuticals

Study Description
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Brief Summary:
The purpose of this study is to determine whether eculizumab long-term use is safe and effective in patients with relapsing NMO.

Condition or disease Intervention/treatment Phase
Neuromyelitis Optica Neuromyelitis Optica Spectrum Disorder Biological: eculizumab Phase 3

Detailed Description:
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.
Study Design
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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 119 participants
Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: A Phase III, Open-label, Extension Trial of ECU-NMO-301 to Evaluate the Safety and Efficacy of Eculizumab in Patients With Relapsing Neuromyelitis Optica (NMO)
Actual Study Start Date : January 12, 2015
Actual Primary Completion Date : July 12, 2021
Actual Study Completion Date : July 12, 2021

Resource links provided by the National Library of Medicine

Drug Information available for: Eculizumab

Arms and Interventions
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Arm Intervention/treatment
Experimental: Eculizumab
Eculizumab intravenous infusion every two weeks.
 Biological: eculizumab
Other Name: Soliris


Outcome Measures
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Primary Outcome Measures :
  1. Number of Participants With Treatment Emergent Adverse Events and Serious Adverse Events [ Time Frame: Baseline up to end of study (up to 6.5 years) ]
    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.

  2. Number of Participants With At Least 1 Post Baseline Columbia-Suicide Severity Rating Scale (C-SSRS) Assessment (Suicide-Related Thoughts or Behaviours) Abnormality [ Time Frame: Baseline up to end of study (up to 6.5 years) ]
    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.

  3. Number of Participants With An On-trial Relapse as Determined by The Treating Physician [ Time Frame: Baseline up to end of study (up to 6.5 years) ]
    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.

  4. On-Trial Annualized Relapse Rate (ARR) as Determined by The Treating Physician [ Time Frame: Baseline up to end of study (up to 6.5 years) ]
    The On-trial ARR was computed as the total number of relapses divided by the total number of participant years in the study period.


Secondary Outcome Measures :
  1. Change From Baseline in Expanded Disability Status Scale (EDSS) Score [ Time Frame: Baseline, Weeks 52, 104 and 156 ]
    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.

  2. Change From Baseline in Modified Rankin Scale (mRS) Score [ Time Frame: Baseline, Weeks 52, 104 and 156 ]
    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.

  3. Change From Baseline in Hauser Ambulation Index (HAI) in Participants With Abnormal Baseline Ambulatory Function [ Time Frame: Baseline, Weeks 52, 104 and 156 ]
    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.

  4. Change From Baseline in European Quality of Life (EuroQoL) 5-Dimension Visual Analog Scale (EQ-5D VAS) Health Status Score [ Time Frame: Baseline, Weeks 52, 104 and 156 ]
    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.

  5. Change From Baseline in Kurtzke Visual Functional System Scores (FSS) in Participants With Abnormal Baseline Visual Function [ Time Frame: Baseline, Weeks 52, 104 and 156 ]
    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.


Eligibility Criteria
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Ages Eligible for Study:   18 Years and older   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Key Inclusion Criteria:

  1. Patient completed the ECU-NMO-301 trial
  2. Patient has given written informed consent

Key Exclusion Criteria:

  1. Patients who have withdrawn from the ECU-NMO-301 trial as a result of an AE related to trial drug
  2. Female patients who are pregnant, breastfeeding, or intend to conceive during the course of the trial
Contacts and Locations
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Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02003144


Locations
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Sponsors and Collaborators
Alexion Pharmaceuticals
  Study Documents (Full-Text)

Documents provided by Alexion Pharmaceuticals:
Study Protocol  [PDF] March 22, 2018
Statistical Analysis Plan  [PDF] July 8, 2021

More Information
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Publications:

Publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):

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Responsible Party: Alexion Pharmaceuticals
ClinicalTrials.gov Identifier: NCT02003144    
Other Study ID Numbers: ECU-NMO-302
2013-001151-12 ( EudraCT Number )
First Posted: December 6, 2013    Key Record Dates
Results First Posted: August 23, 2022
Last Update Posted: August 23, 2022
Last Verified: July 2022
Keywords provided by Alexion Pharmaceuticals:
Long-term safety study
Extension trial
Eculizumab
Neuromyelitis Optica Spectrum Disorder
Devic's disease
Transverse Myelitis
 Optic Neuritis
Relapse
NMO-IgG
CNS Autoimmune Disorders
Demyelinating Disorders
Additional relevant MeSH terms:
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Neuromyelitis Optica
Myelitis, Transverse
Demyelinating Autoimmune Diseases, CNS
Autoimmune Diseases of the Nervous System
Nervous System Diseases
Optic Neuritis
Optic Nerve Diseases
Cranial Nerve Diseases
Demyelinating Diseases
 Eye Diseases
Autoimmune Diseases
Immune System Diseases
Eculizumab
Complement Inactivating Agents
Immunosuppressive Agents
Immunologic Factors
Physiological Effects of Drugs