Clinical trial NCT04201262: An Efficacy and Safety Study of Ravulizumab in Adult Participants With NMOSD
Official title: A Phase 3, External Placebo-Controlled, Open-Label, Multicenter Study to Evaluate the Efficacy and Safety of Ravulizumab in Adult Patients With Neuromyelitis Optica Spectrum Disorder (NMOSD)
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE3
Conditions: Neuromyelitis Optica
Neuromyelitis Optica Spectrum Disorder
Keywords: Neuromyelitis Optica
Neuromyelitis Optica Spectrum Disorder
Devic's Disease
Transverse Myelitis
Optic Neuritis
Relapse
Ravulizumab
Ultomiris
ALXN1210
CNS Autoimmune Disorders
Demyelinating Disorders
NMO
NMOSD
Lead sponsor: Alexion Pharmaceuticals, Inc.
Sponsor class: INDUSTRY
First posted: 2019-12-17
Enrollment: 58
Sex: ALL
Minimum age (years): 18
Maximum age (years): 100
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Intervention model description: External Placebo-Controlled, Open-Label Design
Primary purpose: TREATMENT

Brief summary: The primary purpose of this study is to evaluate the efficacy and safety of ravulizumab for the treatment of adult participants with NMOSD.


Eligibility criteria: Inclusion Criteria:

1. Anti-aquaporin-4 antibody-positive and a diagnosis of NMOSD as defined by the 2015 international consensus diagnostic criteria.
2. At least 1 attack or relapse in the last 12 months prior to the Screening Period.
3. Expanded Disability Status Scale score ≤7.
4. Participants who enter the study receiving supportive immunosuppressive therapy must be on a stable dosing regimen of adequate duration prior to Screening.
5. Body weight ≥40 kilograms.
6. Contraceptive use by men or women should be consistent with local regulations regarding the methods of contraception for those participating in clinical studies.

Exclusion Criteria:

1. History of Neisseria meningitidis infection.
2. Human immunodeficiency virus (HIV) infection (evidenced by HIV-1 or HIV-2 antibody titer).
3. Previously or currently treated with a complement inhibitor.
4. Use of rituximab or mitoxantrone within 3 months prior to Screening.
5. Use of IV immunoglobulin within 3 weeks prior to Screening.

Primary outcomes:
measure: Number of Participants With an Adjudicated On-trial Relapse in the Primary Treatment Period
timeFrame: Baseline up to 2.25 years (end of the Primary Treatment Period)
Secondary outcomes:
measure: Adjudicated On-trial Annualized Relapse Rate (ARR) in the Primary Treatment Period
timeFrame: Baseline up to 2.25 years (end of the Primary Treatment Period)
measure: Number of Participants With Clinically Important Change From Baseline in Hauser Ambulation Index (HAI) Score at the End of Primary Treatment Period
timeFrame: Baseline up to 2.25 years (end of the Primary Treatment Period)
measure: Change From Baseline in European Quality of Life Health 5-dimension Questionnaire (EQ-5D) Index Score at the End of Primary Treatment Period
timeFrame: Baseline, up to 2.25 years (end of the Primary Treatment Period)
measure: Change From Baseline in EQ-5D Visual Analog Scale (VAS) Score at the End of Primary Treatment Period
timeFrame: Baseline, up to 2.25 years (end of the Primary Treatment Period)
measure: Number of Participants With Clinically Important Worsening From Baseline in Expanded Disability Status Scale (EDSS) Score at the End of Primary Treatment Period
timeFrame: Baseline up to 2.25 years (end of the Primary Treatment Period)
measure: Number of Participants With Treatment-emergent Adverse Events (TEAEs), Treatment-emergent Serious Adverse Events (TESAEs), and TEAEs Leading to Study Drug Discontinuation in the Primary Treatment Period
timeFrame: Baseline up to 2.25 years (end of the Primary Treatment Period)
measure: Serum Ravulizumab Concentration
timeFrame: Predose and end of infusion (EOI) at Week 26
measure: Change From Baseline in Serum Free C5 Concentration at Week 26 and 50
timeFrame: Baseline, Week 26 (Predose and EOI)
measure: Number of Participants With Anti-drug Antibodies (ADAs) During the Primary Treatment Period
timeFrame: Baseline, Weeks 26, 50, 82, and 106
Other outcomes:
Locations:
city: Aurora
country: United States
facility: Research Site
state: Colorado
city: Fort Collins
country: United States
facility: Research Site
state: Colorado
city: Washington
country: United States
facility: Research Site
state: District of Columbia
city: Miami
country: United States
facility: Research Site
state: Florida
city: Boston
country: United States
facility: Research Site
state: Massachusetts
city: Rochester
country: United States
facility: Research Site
state: Minnesota
city: Jackson
country: United States
facility: Research Site
state: Mississippi
city: Saint Louis
country: United States
facility: Research Site
state: Missouri
city: Cincinnati
country: United States
facility: Research Site
state: Ohio
city: Houston
country: United States
facility: Research Site
state: Texas
city: Camperdown
country: Australia
facility: Research Site
city: Fitzroy
country: Australia
facility: Research Site
city: Vienna
country: Austria
facility: Research Site
city: Burnaby
country: Canada
facility: Research Site
state: British Columbia
city: Aarhus
country: Denmark
facility: Research Site
city: Strasbourg
country: France
facility: Research Site
city: Berlin
country: Germany
facility: Research Site
city: Leipzig
country: Germany
facility: Research Site
city: Muenchen
country: Germany
facility: Research Site
city: Cefalu
country: Italy
facility: Research Site
city: Gallarate
country: Italy
facility: Research Site
city: Napoli
country: Italy
facility: Research Site
city: Roma
country: Italy
facility: Research Site
city: Rome
country: Italy
facility: Research Site
city: Chiba-shi
country: Japan
facility: Research Site
city: Fukuoka City
country: Japan
facility: Research Site
city: Kawagoe-shi
country: Japan
facility: Research Site
city: Niigata-shi
country: Japan
facility: Research Site
city: Sendai-shi
country: Japan
facility: Research Site
city: Sendai-shi
country: Japan
facility: Research Site
city: Goyang-si
country: Korea, Republic of
facility: Research Site
city: Seoul
country: Korea, Republic of
facility: Research Site
city: Seoul
country: Korea, Republic of
facility: Research Site
city: Seoul
country: Korea, Republic of
facility: Research Site
city: Seoul
country: Korea, Republic of
facility: Research Site
city: Katowice
country: Poland
facility: Research Site
city: Lódz
country: Poland
facility: Research Site
city: Barcelona
country: Spain
facility: Research Site
city: Madrid
country: Spain
facility: Research Site
city: Malaga
country: Spain
facility: Research Site
city: Oxford
country: United Kingdom
facility: Research Site
Study officials:
References: