RECRUITING

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

Conditions

Neuromyelitis Optica Spectrum Disorder Ravulizumab ALXN1210 CNS Autoimmune Disorders NMO NMOSD

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Study Overview

This clinical trial focuses on testing the efficacy of different digital interventions to promote re-engagement in cancer-related long-term follow-up care for adolescent and young adult (AYA) survivors of childhood cancer.

Description

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

Quick Facts

Study Start:2022-06-23

Study Completion:2028-03-31

Study Type:Not specified

Phase:Not Applicable

Enrollment:Not specified

Status:RECRUITING

Study ID

NCT05346354

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Ages Eligible for Study:Not specified

Sexes Eligible for Study:ALL

Accepts Healthy Volunteers:No

Standard Ages:CHILD, ADULT, OLDER_ADULT

Inclusion Criteria	Exclusion 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.	* 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.

Inclusion Criteria

Exclusion 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.

* 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.

Contacts and Locations

Study Contact

Alexion Pharmaceuticals, Inc. (Sponsor)

CONTACT

1-855-752-2356

clinicaltrials@alexion.com

Study Locations (Sites)

Research Site

Washington, District of Columbia, 20010

United States

Research Site

Miami, Florida, 33136

United States

Research Site

Boston, Massachusetts, 02114

United States

Research Site

Saint Louis, Missouri, 63110

United States

Research Site

Durham, North Carolina, 27705

United States

Research Site

Philadelphia, Pennsylvania, 19104

United States

Collaborators and Investigators

Sponsor: Alexion Pharmaceuticals, Inc.

Study Record Dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Registration Dates

Study Start Date2022-06-23

Study Completion Date2028-03-31

Study Record Updates

Study Start Date2022-06-23

Study Completion Date2028-03-31

Terms related to this study

Keywords Provided by Researchers

Neuromyelitis Optica Spectrum Disorder
Ravulizumab
ALXN1210
CNS Autoimmune Disorders
NMO
NMOSD

Additional Relevant MeSH Terms

Neuromyelitis Optica Spectrum Disorder