Back to resultsEfficacy and Safety Study of Ravulizumab IV in Pediatric Participants With NMOSD
Primary Purpose
Neuromyelitis Optica Spectrum Disorder
Status
Recruiting
Phase
Phase 2
Locations
International
Study Type
Interventional
Intervention
Ravulizumab
Sponsored by
About this trial
This is an interventional treatment trial for Neuromyelitis Optica Spectrum Disorder focused on measuring Neuromyelitis Optica Spectrum Disorder, Ravulizumab, ALXN1210, CNS Autoimmune Disorders, NMO, 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 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.
Sites / Locations
- Clinical Trial SiteRecruiting
- Clinical Trial SiteRecruiting
- Clinical Trial SiteRecruiting
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- Clinical Trial SiteRecruiting
Arms of the Study
Arm 1
Arm Type
Experimental
Arm Label
Ravulizumab
Arm Description
During the Primary Treatment Period, all participants will receive weight-based dosing of ravulizumab IV for a total of 50 weeks of treatment. During the Extension Period, participants will continue to receive weight-based dosing of ravulizumab IV for up to 104 weeks.
Outcomes
Primary Outcome Measures
Change From Baseline in the Annualized Relapse Rate at Week 50
Time to First Adjudicated On-trial Relapse through Week 50
Secondary Outcome Measures
Change From Baseline in Expanded Disability Status Scale Score At Week 50
The score ranges are 0 to 10, higher score indicates worse outcome.
Change From Baseline in Hauser Ambulation Index at Week 50
The score ranges are 0 to 9, higher score indicates worse outcome.
Change From Baseline in Visual Acuity at Week 50
Change From Baseline in Confrontational Visual Fields at Week 50
Change From Baseline in Color Vision at Week 50
Serum Ravulizumab Concentration
Change from Baseline in Free Serum Complement Component 5 (C5) Concentration Over Time Through Week 50
Full Information
1. Study Identification
Unique Protocol Identification Number
NCT05346354
Brief Title
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
2. Study Status
Record Verification Date
August 2023
Overall Recruitment Status
Recruiting
Study Start Date
June 23, 2022 (Actual)
Primary Completion Date
March 1, 2026 (Anticipated)
Study Completion Date
March 30, 2028 (Anticipated)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Alexion
4. Oversight
Studies a U.S. FDA-regulated Drug Product
Yes
Studies a U.S. FDA-regulated Device Product
No
Data Monitoring Committee
Yes
5. Study Description
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).
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Neuromyelitis Optica Spectrum Disorder
Keywords
Neuromyelitis Optica Spectrum Disorder, Ravulizumab, ALXN1210, CNS Autoimmune Disorders, NMO, NMOSD
7. Study Design
Primary Purpose
Treatment
Study Phase
Phase 2, Phase 3
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
12 (Anticipated)
8. Arms, Groups, and Interventions
Arm Title
Ravulizumab
Arm Type
Experimental
Arm Description
During the Primary Treatment Period, all participants will receive weight-based dosing of ravulizumab IV for a total of 50 weeks of treatment.
During the Extension Period, participants will continue to receive weight-based dosing of ravulizumab IV for up to 104 weeks.
Intervention Type
Drug
Intervention Name(s)
Ravulizumab
Other Intervention Name(s)
ALXN1210, Ultomiris
Intervention Description
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.
Primary Outcome Measure Information:
Title
Change From Baseline in the Annualized Relapse Rate at Week 50
Time Frame
Baseline, Week 50
Title
Time to First Adjudicated On-trial Relapse through Week 50
Time Frame
Baseline through Week 50
Secondary Outcome Measure Information:
Title
Change From Baseline in Expanded Disability Status Scale Score At Week 50
Description
The score ranges are 0 to 10, higher score indicates worse outcome.
Time Frame
Baseline, Week 50
Title
Change From Baseline in Hauser Ambulation Index at Week 50
Description
The score ranges are 0 to 9, higher score indicates worse outcome.
Time Frame
Baseline, Week 50
Title
Change From Baseline in Visual Acuity at Week 50
Time Frame
Baseline, Week 50
Title
Change From Baseline in Confrontational Visual Fields at Week 50
Time Frame
Baseline, Week 50
Title
Change From Baseline in Color Vision at Week 50
Time Frame
Baseline, Week 50
Title
Serum Ravulizumab Concentration
Time Frame
Predose and postdose (at end of infusion) on Day 1, Weeks 2, 10, 18, 26, and 42, and predose on Week 50
Title
Change from Baseline in Free Serum Complement Component 5 (C5) Concentration Over Time Through Week 50
Time Frame
Baseline through Week 50
10. Eligibility
Sex
All
Minimum Age & Unit of Time
2 Years
Maximum Age & Unit of Time
17 Years
Accepts Healthy Volunteers
No
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 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.
Central Contact Person:
First Name & Middle Initial & Last Name or Official Title & Degree
Alexion Pharmaceuticals, Inc.
Phone
855-752-2356
Email
clinicaltrials@alexion.com
Facility Information:
Facility Name
Clinical Trial Site
City
Miami
State/Province
Florida
ZIP/Postal Code
33136
Country
United States
Individual Site Status
Recruiting
Facility Name
Clinical Trial Site
City
Boston
State/Province
Massachusetts
ZIP/Postal Code
02114
Country
United States
Individual Site Status
Recruiting
Facility Name
Clinical Trial Site
City
Edmonton
State/Province
Alberta
ZIP/Postal Code
T6G 1C9
Country
Canada
Individual Site Status
Recruiting
Facility Name
Clinical Trial Site
City
Toronto
State/Province
Ontario
ZIP/Postal Code
M5G 1X8
Country
Canada
Individual Site Status
Recruiting
Facility Name
Clinical Trial Site
City
Montreal
State/Province
Quebec
ZIP/Postal Code
H3T 1C5
Country
Canada
Individual Site Status
Recruiting
Facility Name
Clinical Trial Site
City
Le Kremlin-Bicêtre
ZIP/Postal Code
94270
Country
France
Individual Site Status
Recruiting
Facility Name
Clinical Trial Site
City
Montpellier
ZIP/Postal Code
34295
Country
France
Individual Site Status
Recruiting
Facility Name
Clinical Trial Site
City
Rome
ZIP/Postal Code
00165
Country
Italy
Individual Site Status
Recruiting
Facility Name
Clinical Trial Site
City
Yokohama
State/Province
Kanagawa
ZIP/Postal Code
232-0024
Country
Japan
Individual Site Status
Recruiting
Facility Name
Clinical Trial Site
City
Goyang-si
State/Province
Gyeonggi-do
ZIP/Postal Code
10408
Country
Korea, Republic of
Individual Site Status
Recruiting
Facility Name
Clinical Trial Site
City
Barcelona
ZIP/Postal Code
08950
Country
Spain
Individual Site Status
Recruiting
12. IPD Sharing Statement
Plan to Share IPD
Yes
IPD Sharing Plan Description
Alexion has a public commitment to allow requests for access to study data and will be supplying a protocol, CSR, and plain language summaries.
Learn more about this trial
Efficacy and Safety Study of Ravulizumab IV in Pediatric Participants With NMOSD
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