Neuromyelitis OpticaPhase 3NCT01892345
A Randomized Controlled Trial of Eculizumab in AQP4 Antibody-positive Participants With NMO (PREVENT Study)
A Phase 3 study testing Eculizumab in people with Neuromyelitis Optica and Neuromyelitis Optica Spectrum Disorder, run by Alexion Pharmaceuticals, Inc. at 70 sites in 18 countries.
Trial summary
The objectives of this time-to-event study were to assess the efficacy and safety of eculizumab as compared with placebo in participants with neuromyelitis optica spectrum disorder (NMOSD) who were anti-aquaporin-4 (AQP4) antibody-positive. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Neuromyelitis Optica, Neuromyelitis Optica Spectrum Disorder
- What is being tested?
- Eculizumab
- Type of treatment
- Vaccines, Injection or infusion
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 143 participants
- Where
- 70 sitesin 18 countries
- Study dates
- 11 Apr 2014to 17 Jul 2018
- Phase
- Phase 3
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Male or female participants ≥ 18 years old.
- Diagnosis of NMO or NMOSD.
- AQP4 antibody seropositive.
- 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.
- Expanded Disability Status Scale score ≤ 7.
- 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.
- 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.
Who cannot join
- Use of rituximab within 3 months prior to Screening.
- Use of mitoxantrone within 3 months prior to Screening.
- Use of intravenous immunoglobulin within 3 weeks prior to Screening.
Trial locations and contacts
70 sites in 18 countries.
70 sites in 18 countries
United States19 sites
University of Maryland Medical Center
Baltimore, Maryland, 21201
John Hopkins University School of Medicine
Baltimore, Maryland, 21287
The Research Center of Southern California
Carlsbad, California, 92011
Multiple Sclerosis Treatment Center of Dallas
Dallas, Texas, 75246
Fort Wayne Neurological Center
Fort Wayne, Indiana, 46804
The Ohio State University, Wexner Medical Center, CarePoint at Gahanna
Gahanna, Ohio, 43230
University of Kansas Medical Center
Kansas City, Kansas, 66160
Baptist Health Lexington
Lexington, Kentucky, 40503
University of Miami McKnight Brain Institute
Miami, Florida, 33136
Multiple Sclerosis Comprehensive Care Center, NYU Langone Medical Center
New York, New York, 10016
Neurological Services of Orlando
Orlando, Florida, 32806
Hospital of the University of Pennsylvania
Philadelphia, Pennsylvania, 19104
University of Pittsburgh
Pittsburgh, Pennsylvania, 15213
Mayo Clinic - Rochester
Rochester, Minnesota, 55905
University of Utah Health Care
Salt Lake City, Utah, 84132
Mayo Clinic Arizona
Scottsdale, Arizona, 85259
Swedish Neuroscience Institute
Seattle, Washington, 98122
Washington University School of Medicine
St Louis, Missouri, 63110
Georgetown University Hospital
Washington D.C., District of Columbia, 20007
Japan8 sites
Tokyo Medical and Dental University
Bunkyo-ku, Tokyo, 113-8519
Chiba University Hospital
Chiba, Chiba, 260-8677
Kyushu University Hospital
Fukuoka, 812-8582
Kyoto Min-iren Chuo Hospital
Kyoto, Kyoto, 604-8453
Hyogo College of Medicine Hospital
Nishinomiya-shi, Hyōgo, 663-8501
Tohoku University Hospital
Sendai, Miyagi, 980-8574
National Center Hospital, NCNP
Tokyo, 187-8551
Yamaguchi University Hospital
Ube-shi, Yamaguchi, 755-8505
Turkey (Türkiye)6 sites
Hacettepe University Medical Faculty
Ankara, 06100
Istanbul University Cerrahpasa Medical Faculty
Istanbul, 34098
Istanbul Bilim Universty Medical Fac.
Istanbul, 34381
Dokuz Eylul University Medicine Faculty
Izmir, 35340
Kocaeli University Medical Faculty
Kocaeli, 41380
Ondokuz Mayis Univ. Med. Fac.
Samsun, 55139
Russia5 sites
Republican Clinical Hospital for Rehabilitation of Healthcare Ministry of Republic of Tatarstan
Kazan', 420021
FSBHI 'Siberian Clinical Center of FMBA'
Krasnoyarsk, 630037
Federal State Budget Institution of Healthcare - Siberian District Medical Center of FMBA of Russia
Novosibirsk, 630067
SBEIHPE "Rostov SMU of MoH of RF"
Rostov-on-Don, 344022
First Pavlov State Medical University of St.Petersburg
Saint Petersburg, 197022
South Korea5 sites
National Cancer Center
Goyang-si, Gyeonggi-do, 10408
Korea University Anam Hospital
Seoul, 02841
Seoul University National Hospital
Seoul, 03080
Severance Hospital, Yonsei University
Seoul, 03722
Samsung Medical Center
Seoul, 06351
Argentina4 sites
Hospital General de Agudos Juan Antonio Fernandez
Ciudad Autonoma, Buenos Aires, Buenos Aires, 1425
Hospital J. M. Ramos Mejia
Ciudad Autonoma, Buenos Aires, Buenos Aires, C1221
Hospital Universitario Austral
Pilar, Buenos Aires, B1629ODT
Fundacion Rosarina de Neuro Rehabilitacion
Rosario, Santa Fe Province, S2000BZL
Italy4 sites
Universitaria Policlinico di Catania
Catania, 95123
Azienda Ospedaliera Universitaria Federico II
Naples, 80131
Azienda Ospedaliera San Camillo Forlanini
Rome, 00151
Neurological Centre of Latium Dipartimento di Neuroscienze
Rome, 00178
Germany3 sites
Universitaetsklinikum Heidelberg, Abteilung Neuroonkologie
Heidelberg, Baden-Wurttemberg, 69120
Klinikum rechts der Isar der TU Muenchen, Neurologische Klinik und Poliklinik
Munich, Bavaria, 81675
Universitaetsmedizin Rostock, Klinik für Neurologie
Rostock, 18147
Spain3 sites
Hospital de Cruces
Barakaldo, Bizkaia, 48903
Hospital Universitario Reina Sofia
Córdoba, 14011
Hospital Universitario Clinico San Carlos
Madrid, 28040
Thailand3 sites
Navamindradhiraj University, Vajira Hospital
Dusit, 10300
Thammasat University Hospital
Pathum Thani, 12120
Sunprasitthiprasong Hospital
Ubon Ratchathani, 34000
Australia2 sites
University of Sydney, Brain and Mind Center
Camperdown, New South Wales, 2050
St. Vincent's Hospital Melbourne
Fitzroy, Victoria, 3065
United Kingdom2 sites
The Walton Centre
Liverpool, L9 7LJ
John Radcliffe Hospital
Oxford, OX3 9DU
Czechia1 site
Vseobecna fakultni nemocnice Neurologicka klinika
Prague, 128 21
About this study
Official 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)
The objectives of this time-to-event study were to assess the efficacy and safety of eculizumab as compared with placebo in participants with neuromyelitis optica spectrum disorder (NMOSD) who were anti-aquaporin-4 (AQP4) antibody-positive.
- Being tested
- Eculizumab, Placebo
- Keywords
- Neuromyelitis Optica, Neuromyelitis Optica Spectrum Disorder, Devic's disease, Transverse Myelitis, Optic Neuritis, relapse, eculizumab, soliris, NMO-IgG, CNS Autoimmune Disorders, Demyelinating Disorders, NMO, NMOSD
- Medical subject headings
- Neuromyelitis Optica, Myelitis, Transverse, Optic Neuritis, Recurrence, Demyelinating Diseases
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
EculizumabDrug
Also called Soliris
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
PlaceboDrug
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
| Study group | Role | What they receive |
|---|---|---|
| Eculizumab | Experimental | 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. Receives: Drug: Eculizumab |
| Placebo | Placebo comparator | 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. Receives: Drug: Placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
Participants With An Adjudicated On-trial Relapse
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. An adjudicated On-trial Relapse was defined by the protocol and positively adjudicated by the relapse adjudication committee.
Measured: Baseline, Up To 211 Weeks (End of Study)
Secondary outcomes (6)
Adjudicated On-trial Annualized Relapse Rate (ARR)
The adjudicated On-trial ARR was computed as the total number of relapses divided by the total number of patient years in the study period. A central independent committee was used to adjudicate all On-trial Relapses as determined by the treating physician. Results reported as adjusted adjudicated On-trial ARR based on a Poisson regression adjusted for randomization strata and historical ARR in 24 months prior to Screening.
Measured: Baseline, Up To 211 Weeks (End of Study)
Change From Baseline In EDSS At End Of Study
Disease-related disability was measured by the EDSS. 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.
Measured: Baseline, Up To 211 Weeks (End of Study)
Change From Baseline In Modified Rankin Scale (mRS) Score At End Of Study
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 disability) to 6 (death) in whole-point increments. A decrease in score indicates improvement.
Measured: Baseline, Up To 211 Weeks (End of Study)
Change From Baseline In Hauser Ambulation Index (HAI) Score At End of Study
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 ambulatory with no assistance) and 9 being the worst (restricted to wheel chair; unable to transfer self independently). A decrease in score indicates improvement.
Measured: Baseline, Up To 211 Weeks (End of Study)
Change From Baseline In European Quality Of Life (EuroQoL) Health 5-Dimension Questionnaire (EQ-5D) Visual Analogue Scale At End Of Study
The EuroQoL EQ-5D is a generic, standardized, self-administered instrument that provides a simple, descriptive profile and a single index value for health status. Assessments were made using the EQ-5D Visual Analogue Scale, which captures the self-rating of current health status using a visual "thermometer" with the endpoints of 100 (best imaginable health state) at the top and zero (worst imaginable health state) at the bottom. An increase in score indicates improvement.
Measured: Baseline, Up To 211 Weeks (End of Study)
Change From Baseline In EuroQoL EQ-5D Index Score At End Of Study
The EuroQoL EQ-5D is a generic, standardized, self-administered instrument that provides a simple, descriptive profile and a single index value for health status. Index scores range from less than 0 to 1, with higher scores representing a better health status.
Measured: Baseline, Up To 211 Weeks (End of Study)
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted4 Jul 2013 (estimated)
- Study start11 Apr 2014
- Primary completion17 Jul 2018
- Study completion17 Jul 2018
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Publications (6)Papers the sponsor has linked to this study.
- Other trials for Neuromyelitis OpticaStudies of the same condition, open and under way.
- More trials run by Alexion Pharmaceuticals, Inc.The sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 3
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 143 participants (actual)
Sponsor and investigators
- Lead sponsor
- Alexion Pharmaceuticals, Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01892345
- Sponsor's protocol ID
- ECU-NMO-301
- Status last verified
- Jun 2019
- First submitted
- 20 Jun 2013
- Last updated
- 26 Jun 2019
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
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.
PubMed 23623397Pittock SJ, Berthele A, Fujihara K, Kim HJ, Levy M, Palace J, Nakashima I, Terzi M, Totolyan N, Viswanathan S, Wang KC, Pace A, Fujita KP, Armstrong R, Wingerchuk DM. Eculizumab in Aquaporin-4-Positive Neuromyelitis Optica Spectrum Disorder. N Engl J Med. 2019 Aug 15;381(7):614-625. doi: 10.1056/NEJMoa1900866. Epub 2019 May 3.
PubMed 31050279Singh P, Gao X, Kleijn HJ, Bellanti F, Pelto R. Eculizumab Pharmacokinetics and Pharmacodynamics in Patients With Neuromyelitis Optica Spectrum Disorder. Front Neurol. 2021 Nov 3;12:696387. doi: 10.3389/fneur.2021.696387. eCollection 2021.
PubMed 34803867Pittock 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.
PubMed 34498507Kim 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.
PubMed 33676197Palace J, Wingerchuk DM, Fujihara K, Berthele A, Oreja-Guevara C, Kim HJ, Nakashima I, Levy M, Terzi M, Totolyan N, Viswanathan S, Wang KC, Pace A, Yountz M, Miller L, Armstrong R, Pittock S; PREVENT Study Group. Benefits of eculizumab in AQP4+ neuromyelitis optica spectrum disorder: Subgroup analyses of the randomized controlled phase 3 PREVENT trial. Mult Scler Relat Disord. 2021 Jan;47:102641. doi: 10.1016/j.msard.2020.102641. Epub 2020 Nov 26.
PubMed 33310418
Study at a glance
From the registry record, last updated by the sponsor on 26 Jun 2019. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official 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)
- ClinicalTrials.gov ID
- NCT01892345
- Acronym / sponsor ID
- ECU-NMO-301
- Keywords
- Neuromyelitis Optica, Neuromyelitis Optica Spectrum Disorder, Devic's disease, Transverse Myelitis, Optic Neuritis, relapse, eculizumab, soliris, NMO-IgG
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- Terminated
- Enrolment
- 143 participants (actual)
- Lead sponsor
- Alexion Pharmaceuticals, Inc.Industry (pharma, biotech, devices)
- Study start
- 11 Apr 2014 (actual)
- Primary completion
- 17 Jul 2018 (actual)
- Study completion
- 17 Jul 2018 (actual)
- First posted
- 4 Jul 2013 (estimated)
- Results
- Posted 26 Jun 2019
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
The objectives of this time-to-event study were to assess the efficacy and safety of eculizumab as compared with placebo in participants with neuromyelitis optica spectrum disorder (NMOSD) who were anti-aquaporin-4 (AQP4) antibody-positive.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Eculizumab | Experimental | Drug: EculizumabBiological/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 | Placebo comparator | Drug: PlaceboPlacebo 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. |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (7 listed)
- Male or female participants ≥ 18 years old.
- Diagnosis of NMO or NMOSD.
- AQP4 antibody seropositive.
- 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.
- Expanded Disability Status Scale score ≤ 7.
Key exclusion criteria (3 listed)
- Use of rituximab within 3 months prior to Screening.
- Use of mitoxantrone within 3 months prior to Screening.
- Use of intravenous immunoglobulin within 3 weeks prior to Screening.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| EculizumabAlso: SolirisInduction Phase: 900 mg IV weekly for 4 weeks, followed by 1200 mg for the fifth dose; Maintenance Phase: 1200 mg IV every 2 weeks | Drug | Eculizumab |
| PlaceboInduction 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 | Drug | Placebo |
Primary outcome
Participants With An Adjudicated On-trial Relapse
Time frame: Baseline, Up To 211 Weeks (End of Study)
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. An adjudicated On-trial Relapse was defined by the protocol and positively adjudicated by the relapse adjudication committee.
6 secondary outcomes
Adjudicated On-trial Annualized Relapse Rate (ARR)
Time frame: Baseline, Up To 211 Weeks (End of Study)
Change From Baseline In EDSS At End Of Study
Time frame: Baseline, Up To 211 Weeks (End of Study)
Change From Baseline In Modified Rankin Scale (mRS) Score At End Of Study
Time frame: Baseline, Up To 211 Weeks (End of Study)
Change From Baseline In Hauser Ambulation Index (HAI) Score At End of Study
Time frame: Baseline, Up To 211 Weeks (End of Study)
Change From Baseline In European Quality Of Life (EuroQoL) Health 5-Dimension Questionnaire (EQ-5D) Visual Analogue Scale At End Of Study
Time frame: Baseline, Up To 211 Weeks (End of Study)
Change From Baseline In EuroQoL EQ-5D Index Score At End Of Study
Time frame: Baseline, Up To 211 Weeks (End of Study)
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 26 Jun 2019.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Background: PubMed 23623397Pittock 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.
- Results publication: PubMed 31050279Pittock SJ, Berthele A, Fujihara K, Kim HJ, Levy M, Palace J, Nakashima I, Terzi M, Totolyan N, Viswanathan S, Wang KC, Pace A, Fujita KP, Armstrong R, Wingerchuk DM. Eculizumab in Aquaporin-4-Positive Neuromyelitis Optica Spectrum Disorder. N Engl J Med. 2019 Aug 15;381(7):614-625. doi: 10.1056/NEJMoa1900866. Epub 2019 May 3.
- Derived from this study: PubMed 34803867Singh P, Gao X, Kleijn HJ, Bellanti F, Pelto R. Eculizumab Pharmacokinetics and Pharmacodynamics in Patients With Neuromyelitis Optica Spectrum Disorder. Front Neurol. 2021 Nov 3;12:696387. doi: 10.3389/fneur.2021.696387. eCollection 2021.
- Derived from this study: PubMed 34498507Pittock 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.
- Derived from this study: PubMed 33676197Kim 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.
- Derived from this study: PubMed 33310418Palace J, Wingerchuk DM, Fujihara K, Berthele A, Oreja-Guevara C, Kim HJ, Nakashima I, Levy M, Terzi M, Totolyan N, Viswanathan S, Wang KC, Pace A, Yountz M, Miller L, Armstrong R, Pittock S; PREVENT Study Group. Benefits of eculizumab in AQP4+ neuromyelitis optica spectrum disorder: Subgroup analyses of the randomized controlled phase 3 PREVENT trial. Mult Scler Relat Disord. 2021 Jan;47:102641. doi: 10.1016/j.msard.2020.102641. Epub 2020 Nov 26.
- Alexion Clinical Trials WebsiteLink provided by the sponsor.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
The record lists no contact. Use the official record to reach the sponsor.
70 sites in 18 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing Eculizumab in people with Neuromyelitis Optica and Neuromyelitis Optica Spectrum Disorder, run by Alexion Pharmaceuticals, Inc. at 70 sites in 18 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 7 inclusion and 3 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: stopped early, as of the sponsor's last update on 26 Jun 2019. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 70 sites in United States, Japan, Turkey (Türkiye), Russia, South Korea and 13 more countries. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Yes. Results are posted in the registry (first posted 26 Jun 2019). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Participants With An Adjudicated On-trial Relapse (time frame: Baseline, Up To 211 Weeks (End of Study)). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT01892345, which also lists 6 linked publications.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 26 Jun 2019.