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

August 16, 2023 updated by: Alexion

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)

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

Study Overview

Status

Recruiting

Conditions

Intervention / Treatment

Study Type

Interventional

Enrollment (Estimated)

12

Phase

  • Phase 2
  • Phase 3

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Contact

Study Locations

  • Canada
    • Alberta
      • Edmonton, Alberta, Canada, T6G 1C9
        • Recruiting
        • Clinical Trial Site
    • Ontario
      • Toronto, Ontario, Canada, M5G 1X8
        • Recruiting
        • Clinical Trial Site
    • Quebec
      • Montreal, Quebec, Canada, H3T 1C5
        • Recruiting
        • Clinical Trial Site
  • France
      • Le Kremlin-Bicêtre, France, 94270
        • Recruiting
        • Clinical Trial Site
      • Montpellier, France, 34295
        • Recruiting
        • Clinical Trial Site
  • Italy
      • Rome, Italy, 00165
        • Recruiting
        • Clinical Trial Site
  • Japan
    • Kanagawa
      • Yokohama, Kanagawa, Japan, 232-0024
        • Recruiting
        • Clinical Trial Site
  • Korea, Republic of
    • Gyeonggi-do
      • Goyang-si, Gyeonggi-do, Korea, Republic of, 10408
        • Recruiting
        • Clinical Trial Site
  • Spain
      • Barcelona, Spain, 08950
        • Recruiting
        • Clinical Trial Site
  • United States
    • Florida
      • Miami, Florida, United States, 33136
        • Recruiting
        • Clinical Trial Site
    • Massachusetts
      • Boston, Massachusetts, United States, 02114
        • Recruiting
        • Clinical Trial Site

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.

Eligibility Criteria

Ages Eligible for Study

2 years to 17 years (Child)

Accepts Healthy Volunteers

No

Description

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.

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: N/A
  • Interventional Model: Single Group Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Experimental: Ravulizumab

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.
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.

Other Names:
  • Ultomiris
  • ALXN1210

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Time Frame
Change From Baseline in the Annualized Relapse Rate at Week 50
Time Frame: Baseline, Week 50
Baseline, Week 50
Time to First Adjudicated On-trial Relapse through Week 50
Time Frame: Baseline through Week 50
Baseline through Week 50

Secondary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Change From Baseline in Expanded Disability Status Scale Score At Week 50
Time Frame: Baseline, Week 50
The score ranges are 0 to 10, higher score indicates worse outcome.
Baseline, Week 50
Change From Baseline in Hauser Ambulation Index at Week 50
Time Frame: Baseline, Week 50
The score ranges are 0 to 9, higher score indicates worse outcome.
Baseline, Week 50
Change From Baseline in Visual Acuity at Week 50
Time Frame: Baseline, Week 50
Baseline, Week 50
Change From Baseline in Confrontational Visual Fields at Week 50
Time Frame: Baseline, Week 50
Baseline, Week 50
Change From Baseline in Color Vision at Week 50
Time Frame: Baseline, Week 50
Baseline, Week 50
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
Predose and postdose (at end of infusion) on Day 1, Weeks 2, 10, 18, 26, and 42, and predose on Week 50
Change from Baseline in Free Serum Complement Component 5 (C5) Concentration Over Time Through Week 50
Time Frame: Baseline through Week 50
Baseline through Week 50

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Alexion

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 Major Dates

Study Start (Actual)

June 23, 2022

Primary Completion (Estimated)

March 1, 2026

Study Completion (Estimated)

March 30, 2028

Study Registration Dates

First Submitted

April 20, 2022

First Submitted That Met QC Criteria

April 20, 2022

First Posted (Actual)

April 26, 2022

Study Record Updates

Last Update Posted (Actual)

August 18, 2023

Last Update Submitted That Met QC Criteria

August 16, 2023

Last Verified

August 1, 2023

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • ALXN1210-NMO-317

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

YES

IPD 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.

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

Yes

Studies a U.S. FDA-regulated device product

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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