Clinical trial NCT02003144: An Open Label Extension Trial of Eculizumab in Relapsing NMO Patients
Official 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)
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE3
Conditions: Neuromyelitis Optica
Neuromyelitis Optica Spectrum Disorder
Keywords: Long-term safety study
Extension trial
Eculizumab
Neuromyelitis Optica Spectrum Disorder
Devic's disease
Transverse Myelitis
Optic Neuritis
Relapse
NMO-IgG
CNS Autoimmune Disorders
Demyelinating Disorders
Lead sponsor: Alexion Pharmaceuticals, Inc.
Sponsor class: INDUSTRY
First posted: 2013-12-06
Enrollment: 119
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

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

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.

Eligibility criteria: 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 Patients who have withdrawn from the ECU-NMO-301 trial as a result of an AE related to trial drug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

Primary outcomes:
measure: Number of Participants With Treatment Emergent Adverse Events and Serious Adverse Events
timeFrame: Baseline up to end of study (up to 6.5 years)
measure: Number of Participants With At Least 1 Post Baseline Columbia-Suicide Severity Rating Scale (C-SSRS) Assessment (Suicide-Related Thoughts or Behaviours) Abnormality
timeFrame: Baseline up to end of study (up to 6.5 years)
measure: Number of Participants With An On-trial Relapse as Determined by The Treating Physician
timeFrame: Baseline up to end of study (up to 6.5 years)
measure: On-Trial Annualized Relapse Rate (ARR) as Determined by The Treating Physician
timeFrame: Baseline up to end of study (up to 6.5 years)
Secondary outcomes:
measure: Change From Baseline in Expanded Disability Status Scale (EDSS) Score
timeFrame: Baseline, Weeks 52, 104 and 156
measure: Change From Baseline in Modified Rankin Scale (mRS) Score
timeFrame: Baseline, Weeks 52, 104 and 156
measure: Change From Baseline in Hauser Ambulation Index (HAI) in Participants With Abnormal Baseline Ambulatory Function
timeFrame: Baseline, Weeks 52, 104 and 156
measure: Change From Baseline in European Quality of Life (EuroQoL) 5-Dimension Visual Analog Scale (EQ-5D VAS) Health Status Score
timeFrame: Baseline, Weeks 52, 104 and 156
measure: Change From Baseline in Kurtzke Visual Functional System Scores (FSS) in Participants With Abnormal Baseline Visual Function
timeFrame: Baseline, Weeks 52, 104 and 156
Other outcomes:
Locations:
city: Scottsdale
country: United States
facility: Mayo Clinic Arizona
state: Arizona
city: Oceanside
country: United States
facility: The Research Center of Southern California
state: California
city: Washington
country: United States
facility: Georgetown University Hospital
state: District of Columbia
city: Miami
country: United States
facility: University of Miami McKnight Brain Institute
state: Florida
city: Orlando
country: United States
facility: Neurological Services of Orlando
state: Florida
city: Fort Wayne
country: United States
facility: Allied Physicians Inc. of Fort Wayne
state: Indiana
city: Kansas City
country: United States
facility: University of Kansas Medical Center
state: Kansas
city: Nicholasville
country: United States
facility: Baptist Health Lexington
state: Kentucky
city: Baltimore
country: United States
facility: Johns Hopkins University Medical Center
state: Maryland
city: Rochester
country: United States
facility: Mayo Clinic - Rochester
state: Minnesota
city: Saint Louis
country: United States
facility: Washington University School of Medicine
state: Missouri
city: New York
country: United States
facility: Multiple Sclerosis Comprehensive Care Center NYU Langone Medical Center
state: New York
city: New York
country: United States
facility: Mount Sinai School of Medicine
state: New York
city: Columbus
country: United States
facility: Ohio Health Reserach Institute
state: Ohio
city: Philadelphia
country: United States
facility: University of Pennsylvania School of Medicine
state: Pennsylvania
city: Pittsburgh
country: United States
facility: University of Pittsburgh
state: Pennsylvania
city: Dallas
country: United States
facility: Multiple Sclerosis Treatment Center of Dallas
state: Texas
city: San Antonio
country: United States
facility: The University of Texas Health Science
state: Texas
city: Salt Lake City
country: United States
facility: University of Utah
state: Utah
city: Pilar
country: Argentina
facility: Hospital Universitario Austral
state: Buenos Aires
city: Rosario
country: Argentina
facility: Fundacion Rosarina de Neuro Rehabilitacion
state: Santa Fe
city: Ciudad Autonoma, Buenos Aires,
country: Argentina
facility: Hospital General de Agudos Dr. J. M. Ramos Mejia
city: Ciudad Autonoma, Buenos Aires
country: Argentina
facility: Hospital General de Agudos Juan Antonio Fernandez
city: Camperdown
country: Australia
facility: Brain and Mind Research Institute
state: New South Wales
city: Fitzroy
country: Australia
facility: St. Vincent's Hospital
state: Victoria
city: Ottawa
country: Canada
facility: The Ottawa Hospital
state: Ontario
city: Floridablanca
country: Colombia
facility: Fundacion Cardiovascular de Colombia
state: Santander
city: Zagreb
country: Croatia
facility: Clinical Hospital Centre Zagreb
city: Praha
country: Czechia
facility: VFN v Praze
city: Teplice
country: Czechia
facility: Krajska zdravotni, a.s. - Nemocnice
city: Arhus
country: Denmark
facility: Århus Universitetshospital
city: Heidelberg
country: Germany
facility: University Hospital Heidelberg
state: Baden Wuerttemberg
city: Munich
country: Germany
facility: Neurologische Klinik und Poliklinik
state: Bayern
city: Dusseldorf
country: Germany
facility: University Hospital Heinrich Heine University
state: Nordrhein Westfalen
city: Rostock
country: Germany
facility: Universitaetsmedizin Rostock
city: Shatin
country: Hong Kong
facility: Prince of Wales Hospital
city: Catania
country: Italy
facility: Policlinico di Catania
city: Napoli
country: Italy
facility: Azienda Ospedaliera Universitaria
city: Padova
country: Italy
facility: Azienda Ospedaliera di Padova
city: Rome
country: Italy
facility: Azienda Ospedaliera Sant'Andrea-Università di Roma La Sapienza
city: Chiba-shi
country: Japan
facility: Chiba University Hospital
state: Chiba-Ken
city: Nishinomiya-shi
country: Japan
facility: Hyogo College of Medicine Hospital
state: HyogoKen
city: Kyoto-shi
country: Japan
facility: Kyoto Min-iren Chuo Hospital
state: Kyoto-Fu
city: Sendai-shi
country: Japan
facility: Tohoku University Hospital
state: Miyagi-Ken
city: Ube-shi
country: Japan
facility: Yamaguchi University Hospital
state: Yamaguchi-Ken
city: Fukuoka
country: Japan
facility: Kyushu University Hospital
city: Tokio
country: Japan
facility: National Center Hospital, NCNP
city: Goyang-si
country: Korea, Republic of
facility: National Cancer Center
state: Gyeonggi-do
city: Seoul
country: Korea, Republic of
facility: Seoul University National Hospital
city: Seoul
country: Korea, Republic of
facility: Samsung Medical Center
city: Seoul
country: Korea, Republic of
facility: Korea University Anam Hospital
city: Seoul
country: Korea, Republic of
facility: Severance Hospital, Yonsei University
city: Kuching
country: Malaysia
facility: Hospital Umum Sarawak
state: Sarawak
city: Kuala Lumpur
country: Malaysia
facility: Hospital Kuala Lumpur
city: Kazan
country: Russian Federation
facility: Republican Clinical Hospital for Rehabilitation of Healthcare Ministry of Republic of Tatarstan
city: Krasnoyarsk
country: Russian Federation
facility: SBEI "Krasnoyarsk SMU n.a. Prof. V.F. Voyno-Yasenetsky"
city: Novosibirsk
country: Russian Federation
facility: Federal State Budget Institution of Healthcare - Siberian District Medical Center of FMBA of Russia
city: Rostov-on Don
country: Russian Federation
facility: SEIHPE "Rostov SMU of MoH of RF"
city: Barakaldo
country: Spain
facility: Hospital de Cruces
state: Bizkaia
city: Cordoba
country: Spain
facility: Hospital Universitario Reina Sofia
city: Madrid
country: Spain
facility: Hospital Universitario Clinico San Carlos
city: Taipei
country: Taiwan
facility: Cheng Hsin General Hospital
city: Pathum Thani
country: Thailand
facility: Thammasat University Hospital
city: Ankara
country: Turkey
facility: Hacettepe University Medical Faculty
city: Edirne
country: Turkey
facility: Trakya University Medical Faculty
city: Izmir
country: Turkey
facility: Dokuz Eylul University Medicine Faculty
city: Kocaeli
country: Turkey
facility: Kocaeli University Medical Faculty
city: Samsun
country: Turkey
facility: Ondokuz Mayis Univ. Med. Fac.
city: Liverpool
country: United Kingdom
facility: The Walton Centre
Study officials:
References:
citation: 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.
citation: 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.
citation: 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.