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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/study/NCT02003144




Registration number
NCT02003144
Ethics application status
Date submitted
18/11/2013
Date registered
6/12/2013
Date last updated
23/08/2022

Titles & IDs
Public title
An Open Label Extension Trial of Eculizumab in Relapsing NMO Patients
Scientific 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)
Secondary ID [1] 0 0
2013-001151-12
Secondary ID [2] 0 0
ECU-NMO-302
Universal Trial Number (UTN)
Trial acronym
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Neuromyelitis Optica 0 0
Neuromyelitis Optica Spectrum Disorder 0 0
Condition category
Condition code
Inflammatory and Immune System 0 0 0 0
Autoimmune diseases
Neurological 0 0 0 0
Other neurological disorders
Eye 0 0 0 0
Diseases / disorders of the eye

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
Experimental: Eculizumab - Eculizumab intravenous infusion every two weeks.
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Number of Participants With Treatment Emergent Adverse Events and Serious Adverse Events
Timepoint [1] 0 0
Baseline up to end of study (up to 6.5 years)
Primary outcome [2] 0 0
Number of Participants With At Least 1 Post Baseline Columbia-Suicide Severity Rating Scale (C-SSRS) Assessment (Suicide-Related Thoughts or Behaviours) Abnormality
Timepoint [2] 0 0
Baseline up to end of study (up to 6.5 years)
Primary outcome [3] 0 0
Number of Participants With An On-trial Relapse as Determined by The Treating Physician
Timepoint [3] 0 0
Baseline up to end of study (up to 6.5 years)
Primary outcome [4] 0 0
On-Trial Annualized Relapse Rate (ARR) as Determined by The Treating Physician
Timepoint [4] 0 0
Baseline up to end of study (up to 6.5 years)
Secondary outcome [1] 0 0
Change From Baseline in Expanded Disability Status Scale (EDSS) Score
Timepoint [1] 0 0
Baseline, Weeks 52, 104 and 156
Secondary outcome [2] 0 0
Change From Baseline in Modified Rankin Scale (mRS) Score
Timepoint [2] 0 0
Baseline, Weeks 52, 104 and 156
Secondary outcome [3] 0 0
Change From Baseline in Hauser Ambulation Index (HAI) in Participants With Abnormal Baseline Ambulatory Function
Timepoint [3] 0 0
Baseline, Weeks 52, 104 and 156
Secondary outcome [4] 0 0
Change From Baseline in European Quality of Life (EuroQoL) 5-Dimension Visual Analog Scale (EQ-5D VAS) Health Status Score
Timepoint [4] 0 0
Baseline, Weeks 52, 104 and 156
Secondary outcome [5] 0 0
Change From Baseline in Kurtzke Visual Functional System Scores (FSS) in Participants With Abnormal Baseline Visual Function
Timepoint [5] 0 0
Baseline, Weeks 52, 104 and 156

Eligibility
Key inclusion criteria
Key

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

Key
Minimum age
18 Years
Maximum age
No limit
Sex
Both males and females
Can healthy volunteers participate?
No
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 design
Purpose of the study
Treatment
Allocation to intervention
Not applicable
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Masking / blinding
Open (masking not used)
Who is / are masked / blinded?



Intervention assignment
Single group
Other design features
Phase
Phase 3
Type of endpoint/s
Statistical methods / analysis

Recruitment
Recruitment status
Completed
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
12/01/2015
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
Actual
12/07/2021
Sample size
Target
Accrual to date
Final
119
Recruitment in Australia
Recruitment state(s)
NSW,VIC
Recruitment hospital [1] 0 0
Brain and Mind Research Institute - Camperdown
Recruitment hospital [2] 0 0
St. Vincent's Hospital - Fitzroy
Recruitment postcode(s) [1] 0 0
- Camperdown
Recruitment postcode(s) [2] 0 0
3065 - Fitzroy
Recruitment outside Australia
Country [1] 0 0
United States of America
State/province [1] 0 0
Arizona
Country [2] 0 0
United States of America
State/province [2] 0 0
California
Country [3] 0 0
United States of America
State/province [3] 0 0
District of Columbia
Country [4] 0 0
United States of America
State/province [4] 0 0
Florida
Country [5] 0 0
United States of America
State/province [5] 0 0
Indiana
Country [6] 0 0
United States of America
State/province [6] 0 0
Kansas
Country [7] 0 0
United States of America
State/province [7] 0 0
Kentucky
Country [8] 0 0
United States of America
State/province [8] 0 0
Maryland
Country [9] 0 0
United States of America
State/province [9] 0 0
Minnesota
Country [10] 0 0
United States of America
State/province [10] 0 0
Missouri
Country [11] 0 0
United States of America
State/province [11] 0 0
New York
Country [12] 0 0
United States of America
State/province [12] 0 0
Ohio
Country [13] 0 0
United States of America
State/province [13] 0 0
Pennsylvania
Country [14] 0 0
United States of America
State/province [14] 0 0
Texas
Country [15] 0 0
United States of America
State/province [15] 0 0
Utah
Country [16] 0 0
Argentina
State/province [16] 0 0
Buenos Aires
Country [17] 0 0
Argentina
State/province [17] 0 0
Santa Fe
Country [18] 0 0
Argentina
State/province [18] 0 0
Ciudad Autonoma, Buenos Aires,
Country [19] 0 0
Argentina
State/province [19] 0 0
Ciudad Autonoma, Buenos Aires
Country [20] 0 0
Canada
State/province [20] 0 0
Ontario
Country [21] 0 0
Colombia
State/province [21] 0 0
Santander
Country [22] 0 0
Croatia
State/province [22] 0 0
Zagreb
Country [23] 0 0
Czechia
State/province [23] 0 0
Praha
Country [24] 0 0
Czechia
State/province [24] 0 0
Teplice
Country [25] 0 0
Denmark
State/province [25] 0 0
Arhus
Country [26] 0 0
Germany
State/province [26] 0 0
Baden Wuerttemberg
Country [27] 0 0
Germany
State/province [27] 0 0
Bayern
Country [28] 0 0
Germany
State/province [28] 0 0
Nordrhein Westfalen
Country [29] 0 0
Germany
State/province [29] 0 0
Rostock
Country [30] 0 0
Hong Kong
State/province [30] 0 0
Shatin
Country [31] 0 0
Italy
State/province [31] 0 0
Catania
Country [32] 0 0
Italy
State/province [32] 0 0
Napoli
Country [33] 0 0
Italy
State/province [33] 0 0
Padova
Country [34] 0 0
Italy
State/province [34] 0 0
Rome
Country [35] 0 0
Japan
State/province [35] 0 0
Chiba-Ken
Country [36] 0 0
Japan
State/province [36] 0 0
HyogoKen
Country [37] 0 0
Japan
State/province [37] 0 0
Kyoto-Fu
Country [38] 0 0
Japan
State/province [38] 0 0
Miyagi-Ken
Country [39] 0 0
Japan
State/province [39] 0 0
Yamaguchi-Ken
Country [40] 0 0
Japan
State/province [40] 0 0
Fukuoka
Country [41] 0 0
Japan
State/province [41] 0 0
Tokio
Country [42] 0 0
Korea, Republic of
State/province [42] 0 0
Gyeonggi-do
Country [43] 0 0
Korea, Republic of
State/province [43] 0 0
Seoul
Country [44] 0 0
Malaysia
State/province [44] 0 0
Sarawak
Country [45] 0 0
Malaysia
State/province [45] 0 0
Kuala Lumpur
Country [46] 0 0
Russian Federation
State/province [46] 0 0
Kazan
Country [47] 0 0
Russian Federation
State/province [47] 0 0
Krasnoyarsk
Country [48] 0 0
Russian Federation
State/province [48] 0 0
Novosibirsk
Country [49] 0 0
Russian Federation
State/province [49] 0 0
Rostov-on Don
Country [50] 0 0
Spain
State/province [50] 0 0
Bizkaia
Country [51] 0 0
Spain
State/province [51] 0 0
Cordoba
Country [52] 0 0
Spain
State/province [52] 0 0
Madrid
Country [53] 0 0
Taiwan
State/province [53] 0 0
Taipei
Country [54] 0 0
Thailand
State/province [54] 0 0
Pathum Thani
Country [55] 0 0
Turkey
State/province [55] 0 0
Ankara
Country [56] 0 0
Turkey
State/province [56] 0 0
Edirne
Country [57] 0 0
Turkey
State/province [57] 0 0
Izmir
Country [58] 0 0
Turkey
State/province [58] 0 0
Kocaeli
Country [59] 0 0
Turkey
State/province [59] 0 0
Samsun
Country [60] 0 0
United Kingdom
State/province [60] 0 0
Liverpool

Funding & Sponsors
Primary sponsor type
Commercial sector/industry
Name
Alexion Pharmaceuticals, Inc.
Address
Country

Ethics approval
Ethics application status

Summary
Brief summary
The purpose of this study is to determine whether eculizumab long-term use is safe and effective in patients with relapsing NMO.
Trial website
https://clinicaltrials.gov/study/NCT02003144
Trial related presentations / publications
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.
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.
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.
Public notes

Contacts
Principal investigator
Name 0 0
Address 0 0
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for public queries
Name 0 0
Address 0 0
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for scientific queries



Summary Results

For IPD and results data, please see https://clinicaltrials.gov/study/NCT02003144