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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/study/NCT01892345




Registration number
NCT01892345
Ethics application status
Date submitted
20/06/2013
Date registered
4/07/2013
Date last updated
26/06/2019

Titles & IDs
Public title
A Randomized Controlled Trial of Eculizumab in AQP4 Antibody-positive Participants With NMO (PREVENT Study)
Scientific title
A Randomized, Double-Blind, Placebo-Controlled, Multi-Center Trial to Evaluate the Safety and Efficacy of Eculizumab in Patients With Relapsing Neuromyelitis Optica (NMO)
Secondary ID [1] 0 0
ECU-NMO-301
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
Treatment: Drugs - Eculizumab
Treatment: Drugs - Placebo

Experimental: Eculizumab - 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 comparator: Placebo - 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.


Treatment: Drugs: Eculizumab
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

Treatment: Drugs: Placebo
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
Intervention code [1] 0 0
Treatment: Drugs
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Participants With An Adjudicated On-trial Relapse
Timepoint [1] 0 0
Baseline, Up To 211 Weeks (End of Study)
Secondary outcome [1] 0 0
Adjudicated On-trial Annualized Relapse Rate (ARR)
Timepoint [1] 0 0
Baseline, Up To 211 Weeks (End of Study)
Secondary outcome [2] 0 0
Change From Baseline In EDSS At End Of Study
Timepoint [2] 0 0
Baseline, Up To 211 Weeks (End of Study)
Secondary outcome [3] 0 0
Change From Baseline In Modified Rankin Scale (mRS) Score At End Of Study
Timepoint [3] 0 0
Baseline, Up To 211 Weeks (End of Study)
Secondary outcome [4] 0 0
Change From Baseline In Hauser Ambulation Index (HAI) Score At End of Study
Timepoint [4] 0 0
Baseline, Up To 211 Weeks (End of Study)
Secondary outcome [5] 0 0
Change From Baseline In European Quality Of Life (EuroQoL) Health 5-Dimension Questionnaire (EQ-5D) Visual Analogue Scale At End Of Study
Timepoint [5] 0 0
Baseline, Up To 211 Weeks (End of Study)
Secondary outcome [6] 0 0
Change From Baseline In EuroQoL EQ-5D Index Score At End Of Study
Timepoint [6] 0 0
Baseline, Up To 211 Weeks (End of Study)

Eligibility
Key inclusion criteria
Key

1. Male or female participants = 18 years old.
2. Diagnosis of NMO or NMOSD.
3. AQP4 antibody seropositive.
4. Historical relapse of at least 2 relapses in the last 12 months or 3 relapses in the last 24 months with at least 1 relapse in the 12 months prior to the screening.
5. Expanded Disability Status Scale score = 7.
6. If a participant entered the study receiving immunosuppressive therapy (IST) for relapse prevention, the participant must have been on a stable maintenance dose of IST(s), as defined by the treating physician, prior to Screening and must have remained on that dose for the duration of the study, unless the participant experienced a relapse.
7. Female participants of childbearing potential were to have a negative pregnancy test (serum human chorionic gonadotropin). Participants were required to practice an effective, reliable, and medically approved contraceptive regimen during the study and for up to 5 months following discontinuation of treatment.

Key
Minimum age
18 Years
Maximum age
No limit
Sex
Both males and females
Can healthy volunteers participate?
No
Key exclusion criteria
1. Use of rituximab within 3 months prior to Screening.
2. Use of mitoxantrone within 3 months prior to Screening.
3. Use of intravenous immunoglobulin within 3 weeks prior to Screening.

Study design
Purpose of the study
Treatment
Allocation to intervention
Randomised controlled trial
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
Blinded (masking used)
Who is / are masked / blinded?
The people receiving the treatment/s
The people administering the treatment/s
The people assessing the outcomes
The people analysing the results/data
Intervention assignment
Parallel
Other design features
Phase
Phase 3
Type of endpoint/s
Statistical methods / analysis

Recruitment
Recruitment status
Stopped early
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
11/04/2014
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
Actual
17/07/2018
Sample size
Target
Accrual to date
Final
143
Recruitment in Australia
Recruitment state(s)
NSW,VIC
Recruitment hospital [1] 0 0
University of Sydney, Brain and Mind Center - Camperdown
Recruitment hospital [2] 0 0
St. Vincent's Hospital Melbourne - Fitzroy
Recruitment postcode(s) [1] 0 0
2050 - 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
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Minnesota
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United States of America
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Missouri
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United States of America
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New York
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United States of America
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Ohio
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United States of America
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Pennsylvania
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United States of America
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Texas
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United States of America
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Utah
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United States of America
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Washington
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Argentina
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Buenos Aires
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Argentina
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Santa Fe
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Croatia
State/province [19] 0 0
Zagreb
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Czechia
State/province [20] 0 0
Praha
Country [21] 0 0
Denmark
State/province [21] 0 0
Århus
Country [22] 0 0
Germany
State/province [22] 0 0
Baden Wuerttemberg
Country [23] 0 0
Germany
State/province [23] 0 0
Bayern
Country [24] 0 0
Germany
State/province [24] 0 0
Rostock
Country [25] 0 0
Hong Kong
State/province [25] 0 0
Shatin
Country [26] 0 0
Italy
State/province [26] 0 0
Catania
Country [27] 0 0
Italy
State/province [27] 0 0
Napoli
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Italy
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Rome
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Japan
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Chiba
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Japan
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Hyogo
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Japan
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Kyoto
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Japan
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Miyagi
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Japan
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Tokyo
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Japan
State/province [34] 0 0
Yamaguchi
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Japan
State/province [35] 0 0
Fukuoka
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Japan
State/province [36] 0 0
Tokio
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Korea, Republic of
State/province [37] 0 0
Gyeonggi-do
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Korea, Republic of
State/province [38] 0 0
Seoul
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Malaysia
State/province [39] 0 0
Kuala Lumpur
Country [40] 0 0
Russian Federation
State/province [40] 0 0
Kazan
Country [41] 0 0
Russian Federation
State/province [41] 0 0
Krasnoyarsk
Country [42] 0 0
Russian Federation
State/province [42] 0 0
Novosibirsk
Country [43] 0 0
Russian Federation
State/province [43] 0 0
Rostov-on Don
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Russian Federation
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St. Petersburg
Country [45] 0 0
Spain
State/province [45] 0 0
Bizkaia
Country [46] 0 0
Spain
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Cordoba
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Spain
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Madrid
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Taiwan
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Taipei
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Thailand
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Dusit
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Thailand
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Pathumthani
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Thailand
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Ubon Ratchathani
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Turkey
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Ankara
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Turkey
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Istanbul
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Turkey
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Izmir
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Turkey
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Kocaeli
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Turkey
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Samsun
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United Kingdom
State/province [57] 0 0
Liverpool
Country [58] 0 0
United Kingdom
State/province [58] 0 0
Oxford

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

Ethics approval
Ethics application status

Summary
Brief summary
Trial website
Trial related presentations / publications
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

No information has been provided regarding IPD availability


What supporting documents are/will be available?




Results publications and other study-related documents

TypeCitations or Other Details
Journal Pittock SJ, Berthele A, Fujihara K, Kim HJ, Levy M... [More Details]

Results are available at https://clinicaltrials.gov/study/NCT01892345