A Study of the Safety and Activity of Eculizumab in Pediatric Participants With Relapsing Neuromyelitis Optica Spectrum Disorder

October 13, 2023 updated by: Alexion

A Phase 2/3 Open-Label, Single-Arm Trial to Evaluate the Safety and Activity of Eculizumab in Pediatric Patients With Relapsing Neuromyelitis Optica Spectrum Disorder

The objective of this study is to evaluate the safety and efficacy of eculizumab in pediatric participants (aged 2 to < 18 years) with relapsing neuromyelitis optica spectrum disorder (NMOSD).

Study Overview

Status

Active, not 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 Locations

  • Canada
    • Alberta
      • Edmonton, Alberta, Canada
        • Clinical Trial Site
    • Ontario
      • Toronto, Ontario, Canada
        • Clinical Trial Site
  • Germany
    • Goettingen
      • Regensburger Straße, Goettingen, Germany
        • Clinical Trial Site
  • Italy
      • Genoa, Italy
        • Clinical Trial Site
      • Rome, Italy
        • Clinical Trial Site
  • Japan
      • Yokohama, Japan
        • Clinical Trial Site
  • Korea, Republic of
      • Seoul, Korea, Republic of
        • Clinical Trial Site
  • Spain
      • Barcelona, Spain
        • Clinical Trial Site
      • Sevilla, Spain
        • Clinical Trial Site
  • United States
    • California
      • San Francisco, California, United States, 94016
        • Clinical Trial Site
    • District of Columbia
      • Washington, District of Columbia, United States, 20001
        • Clinical Trial Site
    • Florida
      • Miami, Florida, United States, 33101
        • Clinical Trial Site
    • Georgia
      • Atlanta, Georgia, United States, 30301
        • Clinical Trial Site
    • Maryland
      • Rockville, Maryland, United States, 20847
        • Clinical Trial Site
    • Massachusetts
      • Boston, Massachusetts, United States, 02101
        • Clinical Trial Site
    • North Carolina
      • Chapel Hill, North Carolina, United States, 27514
        • Clinical Trial Site
    • Pennsylvania
      • Philadelphia, Pennsylvania, United States, 19019
        • 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:

  1. Male or female participants aged 2 years to < 18 years with body weight ≥ 10 kilograms (kg).
  2. Vaccinated against Neisseria meningitidis within 3 years prior to, or at the time of initiating eculizumab. Participants who initiate study drug treatment less than 2 weeks after receiving a meningococcal vaccine must receive appropriate prophylactic antibiotics until 2 weeks after the vaccination.
  3. Documented vaccination against haemophilus influenzae type b and streptococcus pneumoniae infections at least 2 weeks prior to dosing as per local and country-specific immunization guidelines for the appropriate age group.
  4. Anti-aquaporin-4 antibody-positive and diagnosis of NMOSD as defined by the 2015 International Panel for Neuromyelitis Optica Diagnosis criteria.
  5. Historical Relapse Rate of at least 2 relapses in the last 2 years, and with at least 1 relapse in the year prior to Screening.
  6. EDSS score ≤ 7.
  7. Participants who enter the study receiving supportive immunosuppressive therapies (ISTs) for the prevention of relapse, either in combination or monotherapy, must be on a stable dosing regimen of adequate duration.
  8. Female participants of childbearing potential must have a negative pregnancy test (serum human chorionic gonadotropin) at Screening and follow protocol-specified contraception guidance for avoiding pregnancy while on treatment and for 5 months after the last dose of eculizumab.
  9. Male participants with a female spouse/partner of childbearing potential or a pregnant or breastfeeding spouse or partner must agree to use double barrier contraception (male condom plus appropriate barrier method for the female partner) while on treatment and for at least 5 months after the last dose of eculizumab.

Exclusion Criteria:

  1. Parent or legal guardian is an Alexion employee.
  2. Pregnant, breastfeeding, or intending to conceive during the course of the study.
  3. Participants known to be human immunodeficiency virus positive or with congenital immunodeficiency.
  4. Unresolved meningococcal or other serious infection.
  5. Any unresolved acute or chronic systemic bacterial or other infection that is clinically significant in the opinion of the Investigator and has not been treated with appropriate antibiotics.
  6. Use of rituximab or other biologicals such as tocilizumab within 6 months prior to Screening.
  7. Use of mitoxantrone within 3 months prior to Screening.
  8. Use of intravenous immunoglobulin or plasma exchange within 3 weeks prior to Screening.
  9. Use of immunomodulatory therapies for multiple sclerosis within 3 months prior to Screening.
  10. Has previously received treatment with eculizumab or other complement inhibitors.

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: Eculizumab

All participants will receive open-label eculizumab by intravenous infusion during the Primary Treatment Period, starting on Day 1 and for a total of 52/53 weeks. The dosing regimen will be based on the participant's body weight. As body weight changes during the study, the participant's weight cohort and dose may change accordingly.

After completing the 52/53-week Primary Treatment Period, participants may continue receiving eculizumab in the Extension Treatment Period for 104 weeks.
Drug: Eculizumab
Following a weight-based weekly dose of eculizumab during an induction phase, participants will receive weight-based doses of eculizumab every 2 weeks during the Primary Treatment Period and Extension Treatment Period.
Other Names:
  • Soliris

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Time Frame
Change From Baseline In The Annualized Relapse Rate At 52/53 Weeks
Time Frame: Baseline, Week 52/53
Baseline, Week 52/53
Time To First Relapse
Time Frame: Baseline up to Week 52/53
Baseline up to Week 52/53

Secondary Outcome Measures

Outcome Measure
Time Frame
Change From Baseline In Expanded Disability Status Scale (EDSS) Score At 52/53 Weeks In Participants ≥5 Years Of Age
Time Frame: Baseline, Week 52/53
Baseline, Week 52/53
Change From Baseline In The Hauser Ambulatory Index Score At 52/53 Weeks
Time Frame: Baseline, Week 52/53
Baseline, Week 52/53
Change From Baseline In Pediatric Quality Of Life Inventory (PedsQL) At 52/53 Weeks In Participants ≥5 Years Of Age
Time Frame: Baseline, Week 52/53
Baseline, Week 52/53
Change From Baseline In Pediatric Quality Of Life Inventory Parent Proxy (PedsQL Parent Proxy) At 52/53 Weeks In Participants < 5 Years Of Age
Time Frame: Baseline, Week 52/53
Baseline, Week 52/53
Change From Baseline In Visual Acuity As Measured By The Snellen Or LEA Symbols Eye Chart Examination At 52/53 Weeks
Time Frame: Baseline, Week 52/53
Baseline, Week 52/53
Change From Baseline In Confrontational Visual Fields As Measured During Ophthalmologic Examination At 52/53 Weeks
Time Frame: Baseline, Week 52/53
Baseline, Week 52/53
Change From Baseline In Color Vision As Measured During Ophthalmologic Examination At 52/53 Weeks
Time Frame: Baseline, Week 52/53
Baseline, Week 52/53
Serum Eculizumab Concentration Over Time
Time Frame: Baseline through Week 52/53
Baseline through Week 52/53
Serum Free Complement Protein 5 Concentrations Over Time
Time Frame: Baseline through Week 52/53
Baseline through Week 52/53
In Vitro Hemolytic Activity Over Time
Time Frame: Baseline through Week 52/53
Baseline through Week 52/53

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)

January 14, 2020

Primary Completion (Actual)

July 31, 2023

Study Completion (Estimated)

July 31, 2025

Study Registration Dates

First Submitted

November 5, 2019

First Submitted That Met QC Criteria

November 5, 2019

First Posted (Actual)

November 7, 2019

Study Record Updates

Last Update Posted (Actual)

October 16, 2023

Last Update Submitted That Met QC Criteria

October 13, 2023

Last Verified

October 1, 2023

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • ECU-NMO-303
  • 2019-001829-26 (EudraCT Number)

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

NO

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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