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

Neuromyelitis Optica Clinical Trial

Official title:

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

Clinical Trial Details — Status: Active, not recruiting

Administrative data

• NCT number: NCT04155424
• Other study ID #: ECU-NMO-303
• Secondary ID: 2019-001829-26
• Status: Active, not recruiting
• Phase: Phase 2/Phase 3
• Start date: January 14, 2020
• Est. completion date: July 31, 2025

Study information

• Verified date: October 2023
• Source: Alexion
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial Summary

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).

Recruitment information / eligibility

• Status: Active, not recruiting
• Enrollment: 12
• Est. completion date: July 31, 2025
• Est. primary completion date: July 31, 2023
• Accepts healthy volunteers: No
• Gender: All
• Age group: 2 Years to 17 Years

Eligibility

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.

Related Conditions & MeSH terms

• Neuromyelitis Optica
• Neuromyelitis Optica Spectrum Disorder

Intervention

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.

Locations

Country	Name	City	State
Canada	Clinical Trial Site	Edmonton	Alberta
Canada	Clinical Trial Site	Toronto	Ontario
Germany	Clinical Trial Site	Regensburger Straße	Goettingen
Italy	Clinical Trial Site	Genoa
Italy	Clinical Trial Site	Rome
Japan	Clinical Trial Site	Yokohama
Korea, Republic of	Clinical Trial Site	Seoul
Spain	Clinical Trial Site	Barcelona
Spain	Clinical Trial Site	Sevilla
United States	Clinical Trial Site	Atlanta	Georgia
United States	Clinical Trial Site	Boston	Massachusetts
United States	Clinical Trial Site	Chapel Hill	North Carolina
United States	Clinical Trial Site	Miami	Florida
United States	Clinical Trial Site	Philadelphia	Pennsylvania
United States	Clinical Trial Site	Rockville	Maryland
United States	Clinical Trial Site	San Francisco	California
United States	Clinical Trial Site	Washington	District of Columbia

Sponsors (1)

Lead Sponsor	Collaborator
Alexion

Countries where clinical trial is conducted

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

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Change From Baseline In The Annualized Relapse Rate At 52/53 Weeks		Baseline, Week 52/53
Primary	Time To First Relapse		Baseline up to Week 52/53
Secondary	Change From Baseline In Expanded Disability Status Scale (EDSS) Score At 52/53 Weeks In Participants =5 Years Of Age		Baseline, Week 52/53
Secondary	Change From Baseline In The Hauser Ambulatory Index Score At 52/53 Weeks		Baseline, Week 52/53
Secondary	Change From Baseline In Pediatric Quality Of Life Inventory (PedsQL) At 52/53 Weeks In Participants =5 Years Of Age		Baseline, Week 52/53
Secondary	Change From Baseline In Pediatric Quality Of Life Inventory Parent Proxy (PedsQL Parent Proxy) At 52/53 Weeks In Participants < 5 Years Of Age		Baseline, Week 52/53
Secondary	Change From Baseline In Visual Acuity As Measured By The Snellen Or LEA Symbols Eye Chart Examination At 52/53 Weeks		Baseline, Week 52/53
Secondary	Change From Baseline In Confrontational Visual Fields As Measured During Ophthalmologic Examination At 52/53 Weeks		Baseline, Week 52/53
Secondary	Change From Baseline In Color Vision As Measured During Ophthalmologic Examination At 52/53 Weeks		Baseline, Week 52/53
Secondary	Serum Eculizumab Concentration Over Time		Baseline through Week 52/53
Secondary	Serum Free Complement Protein 5 Concentrations Over Time		Baseline through Week 52/53
Secondary	In Vitro Hemolytic Activity Over Time		Baseline through Week 52/53