Efficacy and Safety Study of Ravulizumab IV in Pediatric Participants With NMOSD

Neuromyelitis Optica Spectrum Disorder Clinical Trial

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)

Clinical Trial Details — Status: Recruiting

Administrative data

• NCT number: NCT05346354
• Other study ID #: ALXN1210-NMO-317
• Status: Recruiting
• Phase: Phase 2/Phase 3
• Start date: June 23, 2022
• Est. completion date: March 30, 2028

Study information

• Verified date: August 2023
• Source: Alexion
• Contact: Alexion Pharmaceuticals, Inc.
• Phone: 855-752-2356
• Email: clinicaltrials[@]alexion.com
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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).

Recruitment information / eligibility

• Status: Recruiting
• Enrollment: 12
• Est. completion date: March 30, 2028
• Est. primary completion date: March 1, 2026
• Accepts healthy volunteers: No
• Gender: All
• Age group: 2 Years to 17 Years

Eligibility

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 6 months prior to Day 1.
• 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.

Related Conditions & MeSH terms

• Neuromyelitis Optica
• Neuromyelitis Optica Spectrum Disorder

Intervention

Drug:
• Ravulizumab
Participants will receive a weight-based loading dose of ravulizumab on Day 1, followed by weight-based maintenance treatment with ravulizumab on Day 15 and every 8 weeks (q8w) after or once every 4 weeks (q4w) depending on weight. During the Extension Period, participants will continue to receive weight-based maintenance doses of ravulizumab IV on Day 351 and q8w or q4w, depending on weight.

Locations

Country	Name	City	State
Canada	Clinical Trial Site	Edmonton	Alberta
Canada	Clinical Trial Site	Montreal	Quebec
Canada	Clinical Trial Site	Toronto	Ontario
France	Clinical Trial Site	Le Kremlin-Bicêtre
France	Clinical Trial Site	Montpellier
Italy	Clinical Trial Site	Rome
Japan	Clinical Trial Site	Yokohama	Kanagawa
Korea, Republic of	Clinical Trial Site	Goyang-si	Gyeonggi-do
Spain	Clinical Trial Site	Barcelona
United States	Clinical Trial Site	Boston	Massachusetts
United States	Clinical Trial Site	Miami	Florida

Sponsors (1)

Lead Sponsor	Collaborator
Alexion

Countries where clinical trial is conducted

United States,  Canada,  France,  Italy,  Japan,  Korea, Republic of,  Spain, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Change From Baseline in the Annualized Relapse Rate at Week 50		Baseline, Week 50
Primary	Time to First Adjudicated On-trial Relapse through Week 50		Baseline through Week 50
Secondary	Change From Baseline in Expanded Disability Status Scale Score At Week 50	The score ranges are 0 to 10, higher score indicates worse outcome.	Baseline, Week 50
Secondary	Change From Baseline in Hauser Ambulation Index at Week 50	The score ranges are 0 to 9, higher score indicates worse outcome.	Baseline, Week 50
Secondary	Change From Baseline in Visual Acuity at Week 50		Baseline, Week 50
Secondary	Change From Baseline in Confrontational Visual Fields at Week 50		Baseline, Week 50
Secondary	Change From Baseline in Color Vision at Week 50		Baseline, Week 50
Secondary	Serum Ravulizumab Concentration		Predose and postdose (at end of infusion) on Day 1, Weeks 2, 10, 18, 26, and 42, and predose on Week 50
Secondary	Change from Baseline in Free Serum Complement Component 5 (C5) Concentration Over Time Through Week 50		Baseline through Week 50