Clinical trial NCT05346354: Efficacy and Safety Study of Ravulizumab IV in Pediatric Participants With NMOSD
Official title: A Phase 2/3, Open-label, Historical-controlled, Single-arm, Multicenter Study to Evaluate the Efficacy, Pharmacokinetics, Pharmacodynamics, and Safety of Ravulizumab in Children and Adolescents With Aquaporin-4 Antibody Positive (AQP4-Ab [+]) Neuromyelitis Optica Spectrum Disorder (NMOSD)
Study type: INTERVENTIONAL
Overall status: RECRUITING
Phase: PHASE2
PHASE3
Conditions: Neuromyelitis Optica Spectrum Disorder
Keywords: Neuromyelitis Optica Spectrum Disorder
Ravulizumab
ALXN1210
CNS Autoimmune Disorders
NMO
NMOSD
Lead sponsor: Alexion Pharmaceuticals, Inc.
Sponsor class: INDUSTRY
First posted: 2022-04-26
Enrollment: 12
Sex: ALL
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NON_RANDOMIZED
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

Brief summary: The primary purpose of this study is to evaluate the safety and efficacy of ravulizumab in pediatric participants with Neuromyelitis Optica Spectrum Disorder (NMOSD).


Eligibility criteria: Inclusion Criteria:

* Participants must be anti-AQP4 Ab-positive and have a diagnosis of NMOSD as defined by the 2015 international consensus diagnostic criteria.
* Complement inhibitor treatment-naïve participants must have had at least 1 attack or relapse in the last 12 months prior to the Screening Period.
* Expanded Disability Status Scale (EDSS) score ≤ 7.
* Eculizumab-experienced participants must be clinically stable per Investigator for 30 days and have been treated with eculizumab in Study ECU-NMO-303 for at least 90 days prior to screening with no missed doses within 2 months prior to Day 1.
* Participants who enter the study receiving supportive IST(s) (eg, corticosteroid, azathioprine \[AZA\], mycophenolate mofetil \[MMF\], methotrexate \[MTX\], tacrolimus \[TAC\], cyclosporin \[CsA\], or cyclophosphamide \[CYC\]) for the prevention of relapse, either in combination or monotherapy, must be on a stable dosing regimen of adequate duration prior to Screening and remain on a stable dosing regimen during the Screening Period.
* To reduce the risk of meningococcal infection (Neisseria meningitidis), all participants must be vaccinated against meningococcal infection.
* Documented vaccination for Hib and S pneumoniae at least 14 days prior to Day 1 according to national/local guidelines for the applicable age group.

Exclusion Criteria:

* Use of rituximab within 3 months prior to screening.
* Currently treated with a biologic medications (other than eculizumab) that may affect immune system functioning, or has stopped treatment with a biologic medication that may affect immune system functioning, and 5 half lives of the medication have not elapsed by the time of the Screening Visit.
* Use of intravenous immunoglobulin (IVIg) or plasma exchange (PE) within 3 weeks prior to Screening.
* Participation in another investigational drug or investigational device study (other than Study ECU-NMO-303) within 5 half lives of that investigational product (if known) or 30 days before initiation of the first dose of study drug, whichever is longer.
* Use of immunomodulatory therapies for multiple sclerosis within 3 months prior to Screening.

Primary outcomes:
measure: Change From Baseline in the Annualized Relapse Rate at Week 50
timeFrame: Baseline, Week 50
measure: Time to First Adjudicated On-trial Relapse through Week 50
timeFrame: Baseline through Week 50
Secondary outcomes:
measure: Change From Baseline in Expanded Disability Status Scale Score At Week 50
timeFrame: Baseline, Week 50
measure: Change From Baseline in Hauser Ambulation Index at Week 50
timeFrame: Baseline, Week 50
measure: Change From Baseline in Visual Acuity at Week 50
timeFrame: Baseline, Week 50
measure: Change From Baseline in Confrontational Visual Fields at Week 50
timeFrame: Baseline, Week 50
measure: Change From Baseline in Color Vision at Week 50
timeFrame: Baseline, Week 50
measure: Serum Ravulizumab Concentration
timeFrame: Predose and postdose (at end of infusion) on Day 1, Weeks 2, 10, 18, 26, and 42, and predose on Week 50
measure: Change from Baseline in Free Serum Complement Component 5 (C5) Concentration Over Time Through Week 50
timeFrame: Baseline through Week 50
Other outcomes:
Locations:
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: Saint Louis
country: United States
facility: Research Site
state: Missouri
city: Durham
country: United States
facility: Research Site
state: North Carolina
city: Philadelphia
country: United States
facility: Research Site
state: Pennsylvania
city: Edmonton
country: Canada
facility: Research Site
state: Alberta
city: Toronto
country: Canada
facility: Research Site
state: Ontario
city: Montreal
country: Canada
facility: Research Site
state: Quebec
city: Le Kremlin-Bicêtre
country: France
facility: Research Site
city: Marseille
country: France
facility: Research Site
city: Montpellier
country: France
facility: Research Site
city: Bochum
country: Germany
facility: Research Site
city: Giessen
country: Germany
facility: Research Site
city: Catania
country: Italy
facility: Research Site
city: Chieti
country: Italy
facility: Research Site
city: Gallarate
country: Italy
facility: Research Site
city: Roma
country: Italy
facility: Research Site
city: Yokohama-shi
country: Japan
facility: Research Site
city: Goyang-si
country: Korea, Republic of
facility: Research Site
city: Esplugues de Llobregat
country: Spain
facility: Research Site
Study officials:
References: