An Open Label Extension Trial of Eculizumab in Relapsing NMO Patients

July 25, 2022 updated by: Alexion Pharmaceuticals

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)

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

Study Overview

Status

Status

Indicates the current recruitment status or the expanded access status.
Completed

Conditions

Conditions

The disease, disorder, syndrome, illness, or injury that is being studied. On ClinicalTrials.gov, conditions may also include other health-related issues, such as lifespan, quality of life, and health risks.

Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.

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 Type

Study Type

Describes the nature of a clinical study. Study types include interventional studies (also called clinical trials), observational studies (including patient registries), and expanded access.
Interventional

Enrollment (Actual)

Enrollment

The number of participants in a clinical study. The "anticipated" enrollment is the target number of participants that the researchers need for the study.
119

Phase

Phase

The stage of a clinical trial studying a drug or biological product, based on definitions developed by the U.S. Food and Drug Administration (FDA). The phase is based on the study's objective, the number of participants, and other characteristics. There are five phases: Early Phase 1 (formerly listed as Phase 0), Phase 1, Phase 2, Phase 3, and Phase 4. Not Applicable is used to describe trials without FDA-defined phases, including trials of devices or behavioral interventions.
  • Phase 3

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • Argentina
      • Ciudad Autonoma, Buenos Aires, Argentina, C1425AGP
        • Hospital General de Agudos Juan Antonio Fernandez
      • Ciudad Autonoma, Buenos Aires,, Argentina, C1221ADC
        • Hospital General de Agudos Dr. J. M. Ramos Mejia
    • Buenos Aires
      • Pilar, Buenos Aires, Argentina, B1629ODT
        • Hospital Universitario Austral
    • Santa Fe
      • Rosario, Santa Fe, Argentina, S2000BZL
        • Fundacion Rosarina de Neuro Rehabilitacion
  • Australia
    • New South Wales
      • Camperdown, New South Wales, Australia
        • Brain and Mind Research Institute
    • Victoria
      • Fitzroy, Victoria, Australia, 3065
        • St. Vincent's Hospital
  • Canada
    • Ontario
      • Ottawa, Ontario, Canada, K1H 8L6
        • The Ottawa Hospital
  • Colombia
    • Santander
      • Floridablanca, Santander, Colombia
        • Fundacion Cardiovascular de Colombia
  • Croatia
      • Zagreb, Croatia, 10000
        • Clinical Hospital Centre Zagreb
  • Czechia
      • Praha, Czechia
        • VFN v Praze
      • Teplice, Czechia, 415 01
        • Krajska zdravotni, a.s. - Nemocnice
  • Denmark
      • Arhus, Denmark, 8000
        • Århus Universitetshospital
  • Germany
      • Rostock, Germany, 18147
        • Universitaetsmedizin Rostock
    • Baden Wuerttemberg
      • Heidelberg, Baden Wuerttemberg, Germany, 69120
        • University Hospital Heidelberg
    • Bayern
      • Munich, Bayern, Germany, 81675
        • Neurologische Klinik und Poliklinik
    • Nordrhein Westfalen
      • Dusseldorf, Nordrhein Westfalen, Germany, 40225
        • University Hospital Heinrich Heine University
  • Hong Kong
      • Shatin, Hong Kong
        • Prince of Wales Hospital
  • Italy
      • Catania, Italy, 95123
        • Policlinico di Catania
      • Napoli, Italy, 80131
        • Azienda Ospedaliera Universitaria
      • Padova, Italy, 35128
        • Azienda Ospedaliera di Padova
      • Rome, Italy, 00178
        • Azienda Ospedaliera Sant'Andrea-Università di Roma La Sapienza
  • Japan
      • Fukuoka, Japan, 812-8582
        • Kyushu University Hospital
      • Tokio, Japan
        • National Center Hospital, NCNP
    • Chiba-Ken
      • Chiba-shi, Chiba-Ken, Japan, 260-8677
        • Chiba University Hospital
    • HyogoKen
      • Nishinomiya-shi, HyogoKen, Japan
        • Hyogo College of Medicine Hospital
    • Kyoto-Fu
      • Kyoto-shi, Kyoto-Fu, Japan, 604-8453
        • Kyoto Min-iren Chuo Hospital
    • Miyagi-Ken
      • Sendai-shi, Miyagi-Ken, Japan, 980-8574
        • Tohoku University Hospital
    • Yamaguchi-Ken
      • Ube-shi, Yamaguchi-Ken, Japan, 755-8505
        • Yamaguchi University Hospital
  • Korea, Republic of
      • Seoul, Korea, Republic of, 135-710
        • Samsung Medical Center
      • Seoul, Korea, Republic of, 136-705
        • Korea University Anam Hospital
      • Seoul, Korea, Republic of, 110744
        • Seoul University National Hospital
      • Seoul, Korea, Republic of, 136-705
        • Severance Hospital, Yonsei University
    • Gyeonggi-do
      • Goyang-si, Gyeonggi-do, Korea, Republic of, 410-769
        • National Cancer Center
  • Malaysia
      • Kuala Lumpur, Malaysia, 50586
        • Hospital Kuala Lumpur
    • Sarawak
      • Kuching, Sarawak, Malaysia, 93586
        • Hospital Umum Sarawak
  • Russian Federation
      • Kazan, Russian Federation, 420021
        • Republican Clinical Hospital for Rehabilitation of Healthcare Ministry of Republic of Tatarstan
      • Krasnoyarsk, Russian Federation, 660037
        • SBEI "Krasnoyarsk SMU n.a. Prof. V.F. Voyno-Yasenetsky"
      • Novosibirsk, Russian Federation, 630068
        • Federal State Budget Institution of Healthcare - Siberian District Medical Center of FMBA of Russia
      • Rostov-on Don, Russian Federation
        • SEIHPE "Rostov SMU of MoH of RF"
  • Spain
      • Cordoba, Spain, 14404
        • Hospital Universitario Reina Sofia
      • Madrid, Spain, 28040
        • Hospital Universitario Clínico San Carlos
    • Bizkaia
      • Barakaldo, Bizkaia, Spain, 48903
        • Hospital de Cruces
  • Taiwan
      • Taipei, Taiwan
        • Cheng Hsin General Hospital
  • Thailand
      • Pathum Thani, Thailand
        • Thammasat University Hospital
  • Turkey
      • Ankara, Turkey, 06100
        • Hacettepe University Medical Faculty
      • Edirne, Turkey
        • Trakya University Medical Faculty
      • Izmir, Turkey, 35340
        • Dokuz Eylul University Medicine Faculty
      • Kocaeli, Turkey
        • Kocaeli University Medical Faculty
      • Samsun, Turkey
        • Ondokuz Mayis Univ. Med. Fac.
  • United Kingdom
      • Liverpool, United Kingdom, L97LJ
        • The Walton Centre
  • United States
    • Arizona
      • Scottsdale, Arizona, United States, 85259
        • Mayo Clinic Arizona
    • California
      • Oceanside, California, United States, 92056
        • The Research Center of Southern California
    • District of Columbia
      • Washington, District of Columbia, United States, 20007
        • Georgetown University Hospital
    • Florida
      • Miami, Florida, United States, 33136
        • University of Miami McKnight Brain Institute
      • Orlando, Florida, United States, 32806
        • Neurological Services of Orlando
    • Indiana
      • Fort Wayne, Indiana, United States, 46805
        • Allied Physicians Inc. of Fort Wayne
    • Kansas
      • Kansas City, Kansas, United States, 66160
        • University of Kansas Medical Center
    • Kentucky
      • Nicholasville, Kentucky, United States, 40503
        • Baptist Health Lexington
    • Maryland
      • Baltimore, Maryland, United States, 21287
        • Johns Hopkins University Medical Center
    • Minnesota
      • Rochester, Minnesota, United States, 55905
        • Mayo Clinic - Rochester
    • Missouri
      • Saint Louis, Missouri, United States, 63110
        • Washington University School of Medicine
    • New York
      • New York, New York, United States, 10029
        • Mount Sinai School of Medicine
      • New York, New York, United States, 10016
        • Multiple Sclerosis Comprehensive Care Center NYU Langone Medical Center
    • Ohio
      • Columbus, Ohio, United States, 43214
        • Ohio Health Reserach Institute
    • Pennsylvania
      • Philadelphia, Pennsylvania, United States, 19104
        • University of Pennsylvania School of medicine
      • Pittsburgh, Pennsylvania, United States, 15232
        • University of Pittsburgh
    • Texas
      • Dallas, Texas, United States, 75246
        • Multiple Sclerosis Treatment Center of Dallas
      • San Antonio, Texas, United States, 78229
        • The University of Texas Health Science
    • Utah
      • Salt Lake City, Utah, United States, 84108
        • University of Utah

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Eligibility Criteria

The key requirements that people who want to participate in a clinical study must meet or the characteristics they must have. Eligibility criteria consist of both inclusion criteria (which are required for a person to participate in the study) and exclusion criteria (which prevent a person from participating). Types of eligibility criteria include whether a study accepts healthy volunteers, has age or age group requirements, or is limited by sex.

Ages Eligible for Study

18 years and older (Adult, Older Adult)

Accepts Healthy Volunteers

No

Genders Eligible for Study

All

Description

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

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: N/A
  • Interventional Model: Single Group Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / Arm

Participant Group / Arm

A group or subgroup of participants in a clinical trial that receives a specific intervention/treatment, or no intervention, according to the trial's protocol.
Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.
Experimental: Eculizumab
Eculizumab intravenous infusion every two weeks.
Biological: eculizumab
Other Names:
  • Soliris

What is the study measuring?

Primary Outcome Measures

Primary Outcome Measures

In a clinical study's protocol, the planned outcome measure that is the most important for evaluating the effect of an intervention/treatment. Most clinical studies have one primary outcome measure, but some have more than one.
Outcome Measure
Measure Description
Time Frame
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.
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
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.
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
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.
Baseline up to end of study (up to 6.5 years)
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.
Baseline up to end of study (up to 6.5 years)

Secondary Outcome Measures

Secondary Outcome Measures

In a clinical study's protocol, a planned outcome measure that is not as important as the primary outcome measure for evaluating the effect of an intervention but is still of interest. Most clinical studies have more than one secondary outcome measure.
Outcome Measure
Measure Description
Time Frame
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.
Baseline, Weeks 52, 104 and 156
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.
Baseline, Weeks 52, 104 and 156
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.
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
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.
Baseline, Weeks 52, 104 and 156
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.
Baseline, Weeks 52, 104 and 156

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Sponsor

The organization or person who initiates the study and who has authority and control over the study.
Alexion Pharmaceuticals

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

General Publications

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

Study Start

The actual date on which the first participant was enrolled in a clinical study. The "anticipated" study start date is the date that the researchers think will be the study start date.
January 12, 2015

Primary Completion (Actual)

Primary Completion

The date on which the last participant in a clinical study was examined or received an intervention to collect final data for the primary outcome measure. Whether the clinical study ended according to the protocol or was terminated does not affect this date. For clinical studies with more than one primary outcome measure with different completion dates, this term refers to the date on which data collection is completed for all the primary outcome measures. The "anticipated" primary completion date is the date that the researchers think will be the primary completion date for the study.
July 12, 2021

Study Completion (Actual)

Study Completion

The date on which the last participant in a clinical study was examined or received an intervention/treatment to collect final data for the primary outcome measures, secondary outcome measures, and adverse events (that is, the last participant's last visit). The "anticipated" study completion date is the date that the researchers think will be the study completion date.
July 12, 2021

Study Registration Dates

First Submitted

First Submitted

The date on which the study sponsor or investigator first submitted a study record to ClinicalTrials.gov. There is typically a delay of a few days between the first submitted date and the record's availability on ClinicalTrials.gov (the first posted date).
November 18, 2013

First Submitted That Met QC Criteria

First Submitted That Met QC Criteria

The date on which the study sponsor or investigator first submits a study record that is consistent with National Library of Medicine (NLM) quality control (QC) review criteria. The sponsor or investigator may need to revise and submit a study record one or more times before NLM's QC review criteria are met. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
December 2, 2013

First Posted (Estimate)

First Posted

The date on which the study record was first available on ClinicalTrials.gov after National Library of Medicine (NLM) quality control (QC) review has concluded. There is typically a delay of a few days between the date the study sponsor or investigator submitted the study record and the first posted date.
December 6, 2013

Study Record Updates

Last Update Posted (Actual)

Last Update Posted

The most recent date on which changes to a study record were made available on ClinicalTrials.gov. There may be a delay between when the changes were submitted to ClinicalTrials.gov by the study's sponsor or investigator (the last update submitted date) and the last update posted date.
August 23, 2022

Last Update Submitted That Met QC Criteria

Last Update Submitted That Met QC Criteria

The most recent date on which the study sponsor or investigator submitted changes to a study record that are consistent with National Library of Medicine (NLM) quality control (QC) review criteria. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
July 25, 2022

Last Verified

Last Verified

The most recent date on which the study sponsor or investigator confirmed the information about a clinical study on ClinicalTrials.gov as accurate and current. If a study with a recruitment status of recruiting; not yet recruiting; or active, not recruiting has not been confirmed within the past 2 years, the study's recruitment status is shown as unknown.
July 1, 2022

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

Other Study ID Numbers

Identifiers or ID numbers other than the NCT number that are assigned to a clinical study by the study's sponsor, funders, or others. These numbers may include unique identifiers from other trial registries and National Institutes of Health grant numbers.
  • ECU-NMO-302
  • 2013-001151-12 (EudraCT Number)

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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