--- title: A Randomized Controlled Trial of Eculizumab in AQP4 Antibody-positive Participants With NMO (PREVENT Study) nct_id: NCT01892345 overall_status: TERMINATED phase: PHASE3 sponsor: Alexion Pharmaceuticals, Inc. study_type: INTERVENTIONAL primary_condition: Neuromyelitis Optica countries: United States, Argentina, Australia, Croatia, Czechia, Denmark, Germany, Hong Kong, Italy, Japan, Malaysia, Russia, South Korea, Spain, Taiwan, Thailand, Turkey (Türkiye), United Kingdom canonical_url: "https://parkinsonspathways.com/agent/trials/NCT01892345.md" clinicaltrials_gov: "https://clinicaltrials.gov/study/NCT01892345" ct_last_update_post_date: 2019-06-26 last_seen_at: "2026-05-12T07:12:38.385Z" source: ClinicalTrials.gov (mirrored, no enrichment) --- # A Randomized Controlled Trial of Eculizumab in AQP4 Antibody-positive Participants With NMO (PREVENT 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) **NCT ID:** [NCT01892345](https://clinicaltrials.gov/study/NCT01892345) ## Key Facts - **Status:** TERMINATED - **Phase:** PHASE3 - **Study Type:** INTERVENTIONAL - **Target Enrollment:** 143 - **Lead Sponsor:** Alexion Pharmaceuticals, Inc. - **Conditions:** Neuromyelitis Optica, Neuromyelitis Optica Spectrum Disorder - **Start Date:** 2014-04-11 - **Completion Date:** 2018-07-17 - **CT.gov Last Update:** 2019-06-26 ## Brief 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. ## Eligibility - **Minimum age:** 18 Years - **Sex:** ALL - **Healthy Volunteers:** No ``` Key Inclusion Criteria: 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 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. ``` ## Arms - **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. - **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. ## Interventions - **Eculizumab** (DRUG) — 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 - **Placebo** (DRUG) — 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 ## Primary Outcomes - **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. ## 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))_ ## Locations (70) - Mayo Clinic Arizona, Scottsdale, Arizona, United States - The Research Center of Southern California, Carlsbad, California, United States - Georgetown University Hospital, Washington D.C., District of Columbia, United States - University of Miami McKnight Brain Institute, Miami, Florida, United States - Neurological Services of Orlando, Orlando, Florida, United States - Fort Wayne Neurological Center, Fort Wayne, Indiana, United States - University of Kansas Medical Center, Kansas City, Kansas, United States - Baptist Health Lexington, Lexington, Kentucky, United States - University of Maryland Medical Center, Baltimore, Maryland, United States - John Hopkins University School of Medicine, Baltimore, Maryland, United States - Mayo Clinic - Rochester, Rochester, Minnesota, United States - Washington University School of Medicine, St Louis, Missouri, United States - Multiple Sclerosis Comprehensive Care Center, NYU Langone Medical Center, New York, New York, United States - The Ohio State University, Wexner Medical Center, CarePoint at Gahanna, Gahanna, Ohio, United States - Hospital of the University of Pennsylvania, Philadelphia, Pennsylvania, United States - University of Pittsburgh, Pittsburgh, Pennsylvania, United States - Multiple Sclerosis Treatment Center of Dallas, Dallas, Texas, United States - University of Utah Health Care, Salt Lake City, Utah, United States - Swedish Neuroscience Institute, Seattle, Washington, United States - Hospital General de Agudos Juan Antonio Fernandez, Ciudad Autonoma, Buenos Aires, Buenos Aires, Argentina - Hospital J. M. Ramos Mejia, Ciudad Autonoma, Buenos Aires, Buenos Aires, Argentina - Hospital Universitario Austral, Pilar, Buenos Aires, Argentina - Fundacion Rosarina de Neuro Rehabilitacion, Rosario, Santa Fe Province, Argentina - University of Sydney, Brain and Mind Center, Camperdown, New South Wales, Australia - St. Vincent's Hospital Melbourne, Fitzroy, Victoria, Australia - Clinical Hospital Centre Zagreb, Zagreb, Croatia - Vseobecna fakultni nemocnice Neurologicka klinika, Prague, Czechia - Århus Universitetshospital, Aarhus, Denmark - Universitaetsklinikum Heidelberg, Abteilung Neuroonkologie, Heidelberg, Baden-Wurttemberg, Germany - Klinikum rechts der Isar der TU Muenchen, Neurologische Klinik und Poliklinik, Munich, Bavaria, Germany - Universitaetsmedizin Rostock, Klinik für Neurologie, Rostock, Germany - Prince of Wales Hospital, Shatin, Hong Kong - Universitaria Policlinico di Catania, Catania, Italy - Azienda Ospedaliera Universitaria Federico II, Naples, Italy - Azienda Ospedaliera San Camillo Forlanini, Rome, Italy - Neurological Centre of Latium Dipartimento di Neuroscienze, Rome, Italy - Chiba University Hospital, Chiba, Chiba, Japan - Hyogo College of Medicine Hospital, Nishinomiya-shi, Hyōgo, Japan - Kyoto Min-iren Chuo Hospital, Kyoto, Kyoto, Japan - Tohoku University Hospital, Sendai, Miyagi, Japan - Tokyo Medical and Dental University, Bunkyo-ku, Tokyo, Japan - Yamaguchi University Hospital, Ube-shi, Yamaguchi, Japan - Kyushu University Hospital, Fukuoka, Japan - National Center Hospital, NCNP, Tokyo, Japan - Hospital Kuala Lumpur, Kuala Lumpur, Malaysia - Republican Clinical Hospital for Rehabilitation of Healthcare Ministry of Republic of Tatarstan, Kazan', Russia - FSBHI 'Siberian Clinical Center of FMBA', Krasnoyarsk, Russia - Federal State Budget Institution of Healthcare - Siberian District Medical Center of FMBA of Russia, Novosibirsk, Russia - SBEIHPE "Rostov SMU of MoH of RF", Rostov-on-Don, Russia - First Pavlov State Medical University of St.Petersburg, Saint Petersburg, Russia - National Cancer Center, Goyang-si, Gyeonggi-do, South Korea - Korea University Anam Hospital, Seoul, South Korea - Seoul University National Hospital, Seoul, South Korea - Severance Hospital, Yonsei University, Seoul, South Korea - Samsung Medical Center, Seoul, South Korea - Hospital de Cruces, Barakaldo, Bizkaia, Spain - Hospital Universitario Reina Sofia, Córdoba, Spain - Hospital Universitario Clinico San Carlos, Madrid, Spain - Cheng Hsin General Hospital, Taipei, Taiwan - Navamindradhiraj University, Vajira Hospital, Dusit, Thailand - Thammasat University Hospital, Pathum Thani, Thailand - Sunprasitthiprasong Hospital, Ubon Ratchathani, Thailand - Hacettepe University Medical Faculty, Ankara, Turkey (Türkiye) - Istanbul University Cerrahpasa Medical Faculty, Istanbul, Turkey (Türkiye) - Istanbul Bilim Universty Medical Fac., Istanbul, Turkey (Türkiye) - Dokuz Eylul University Medicine Faculty, Izmir, Turkey (Türkiye) - Kocaeli University Medical Faculty, Kocaeli, Turkey (Türkiye) - Ondokuz Mayis Univ. Med. Fac., Samsun, Turkey (Türkiye) - The Walton Centre, Liverpool, United Kingdom - John Radcliffe Hospital, Oxford, United Kingdom ## Recent Field Changes (last 30 days) - `status.overallStatus` — added _(2026-05-12)_ - `status.primaryCompletionDate` — added _(2026-05-12)_ - `status.completionDate` — added _(2026-05-12)_ - `status.lastUpdatePostDate` — added _(2026-05-12)_ - `design.phases` — added _(2026-05-12)_ - `design.enrollmentCount` — added _(2026-05-12)_ - `eligibility.criteria` — added _(2026-05-12)_ - `eligibility.minAge` — added _(2026-05-12)_ - `eligibility.sex` — added _(2026-05-12)_ - `outcomes.primary` — added _(2026-05-12)_ - `outcomes.secondary` — added _(2026-05-12)_ - `armsInterventions.arms` — added _(2026-05-12)_ - `armsInterventions.interventions` — added _(2026-05-12)_ - `sponsor.lead` — added _(2026-05-12)_ - `results.hasResults` — added _(2026-05-12)_ - `locations.mayo clinic arizona|scottsdale|arizona|united states` — added _(2026-05-12)_ - `locations.the research center of southern california|carlsbad|california|united states` — added _(2026-05-12)_ - `locations.georgetown university hospital|washington d.c.|district of columbia|united states` — added _(2026-05-12)_ - `locations.university of miami mcknight brain institute|miami|florida|united states` — added _(2026-05-12)_ - `locations.neurological services of orlando|orlando|florida|united states` — added _(2026-05-12)_ - `locations.fort wayne neurological center|fort wayne|indiana|united states` — added _(2026-05-12)_ - `locations.university of kansas medical center|kansas city|kansas|united states` — added _(2026-05-12)_ - `locations.baptist health lexington|lexington|kentucky|united states` — added _(2026-05-12)_ - `locations.university of maryland medical center|baltimore|maryland|united states` — added _(2026-05-12)_ - `locations.john hopkins university school of medicine|baltimore|maryland|united states` — added _(2026-05-12)_ - `locations.mayo clinic - rochester|rochester|minnesota|united states` — added _(2026-05-12)_ - `locations.washington university school of medicine|st louis|missouri|united states` — added _(2026-05-12)_ - `locations.multiple sclerosis comprehensive care center, nyu langone medical center|new york|new york|united states` — added _(2026-05-12)_ - `locations.the ohio state university, wexner medical center, carepoint at gahanna|gahanna|ohio|united states` — added _(2026-05-12)_ - `locations.hospital of the university of pennsylvania|philadelphia|pennsylvania|united states` — added _(2026-05-12)_ - `locations.university of pittsburgh|pittsburgh|pennsylvania|united states` — added _(2026-05-12)_ - `locations.multiple sclerosis treatment center of dallas|dallas|texas|united states` — added _(2026-05-12)_ - `locations.university of utah health care|salt lake city|utah|united states` — added _(2026-05-12)_ - `locations.swedish neuroscience institute|seattle|washington|united states` — added _(2026-05-12)_ - `locations.hospital general de agudos juan antonio fernandez|ciudad autonoma, buenos aires|buenos aires|argentina` — added _(2026-05-12)_ - `locations.hospital j. m. ramos mejia|ciudad autonoma, buenos aires|buenos aires|argentina` — added _(2026-05-12)_ - `locations.hospital universitario austral|pilar|buenos aires|argentina` — added _(2026-05-12)_ - `locations.fundacion rosarina de neuro rehabilitacion|rosario|santa fe province|argentina` — added _(2026-05-12)_ - `locations.university of sydney, brain and mind center|camperdown|new south wales|australia` — added _(2026-05-12)_ - `locations.st. vincent's hospital melbourne|fitzroy|victoria|australia` — added _(2026-05-12)_ - `locations.clinical hospital centre zagreb|zagreb||croatia` — added _(2026-05-12)_ - `locations.vseobecna fakultni nemocnice neurologicka klinika|prague||czechia` — added _(2026-05-12)_ - `locations.århus universitetshospital|aarhus||denmark` — added _(2026-05-12)_ - `locations.universitaetsklinikum heidelberg, abteilung neuroonkologie|heidelberg|baden-wurttemberg|germany` — added _(2026-05-12)_ - `locations.klinikum rechts der isar der tu muenchen, neurologische klinik und poliklinik|munich|bavaria|germany` — added _(2026-05-12)_ - `locations.universitaetsmedizin rostock, klinik für neurologie|rostock||germany` — added _(2026-05-12)_ - `locations.prince of wales hospital|shatin||hong kong` — added _(2026-05-12)_ - `locations.universitaria policlinico di catania|catania||italy` — added _(2026-05-12)_ - `locations.azienda ospedaliera universitaria federico ii|naples||italy` — added _(2026-05-12)_ - `locations.azienda ospedaliera san camillo forlanini|rome||italy` — added _(2026-05-12)_ --- *Canonical: https://parkinsonspathways.com/agent/trials/NCT01892345.md* *Source data (authoritative): https://clinicaltrials.gov/study/NCT01892345* *This page is a raw mirror with no AI summary, no editorial enrichment, and no Parkinson's-specific filtering.*