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

• Register: ClinicalTrials.gov
• Last refreshed on: 23 October 2023
• Main ID: NCT04155424
• Date of registration: 05/11/2019
• Prospective Registration: Yes
• Primary sponsor: Alexion
• Public title: A Study of the Safety and Activity of Eculizumab in Pediatric Participants With Relapsing Neuromyelitis Optica Spectrum Disorder
• Scientific 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
• Date of first enrolment: January 14, 2020
• Target sample size: 12
• Recruitment status: Active, not recruiting
• URL: https://clinicaltrials.gov/ct2/show/NCT04155424
• Study type: Interventional
• Study design: Allocation: N/A. Intervention model: Single Group Assignment. Primary purpose: Treatment. Masking: None (Open Label).
• Phase: Phase 2/Phase 3

Countries of recruitment

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

Key inclusion & exclusion criteria

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.



Age minimum: 2 Years
Age maximum: 17 Years
Gender: All

Health Condition(s) or Problem(s) studied

Neuromyelitis Optica

Neuromyelitis Optica Spectrum Disorder

Intervention(s)

Drug: Eculizumab

Primary Outcome(s)

Change From Baseline In The Annualized Relapse Rate At 52/53 Weeks [Time Frame: Baseline, Week 52/53]

Time To First Relapse [Time Frame: Baseline up to Week 52/53]

Secondary Outcome(s)

Change From Baseline In Confrontational Visual Fields As Measured During Ophthalmologic Examination At 52/53 Weeks [Time Frame: 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]

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]

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]

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]

Change From Baseline In Color Vision As Measured During Ophthalmologic Examination At 52/53 Weeks [Time Frame: Baseline, Week 52/53]

Change From Baseline In The Hauser Ambulatory Index Score At 52/53 Weeks [Time Frame: Baseline, Week 52/53]

In Vitro Hemolytic Activity Over Time [Time Frame: Baseline through Week 52/53]

Serum Free Complement Protein 5 Concentrations Over Time [Time Frame: Baseline through Week 52/53]

Serum Eculizumab Concentration Over Time [Time Frame: Baseline through Week 52/53]

Secondary ID(s)

ECU-NMO-303

2019-001829-26

Source(s) of Monetary Support

Please refer to primary and secondary sponsors