Phase 2
N=14
An Open Label Study of the Effects of Eculizumab in Neuromyelitis Optica
Neuromyelitis Optica · Devic's Disease
Bottom Line
View on ClinicalTrials.gov: NCT00904826 ↗Enrolled (actual)
14
Serious AEs
7.1%
Results posted
Nov 2013
Primary outcome: Primary: Median Number of Neuromyelitis Optica (NMO) Attacks Per Year — 3; 0 attacks per year — p=<0.0001
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 2
- Interventions
- Eculizumab (Drug)
- Age
- Adult, Older Adult · 18+ yrs
- Sex
- All
- Sponsor
- Mayo Clinic
- Primary completion
- Dec 2011
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Median Number of Neuromyelitis Optica (NMO) Attacks Per Year |
3; 0 | <0.0001 sig |
| SECONDARY Number Subjects Experiencing an NMO Attack in 12 Months of Eculizumab Treatment |
2 | — |
| SECONDARY Change in Expanded Disability Status Scale (EDDS) Score |
-0.7 | 0.0078 sig |
| SECONDARY Number of Subjects With Change in Visual Acuity in at Least One Eye by at Least One Point |
5 | — |
| SECONDARY Number of Subjects With Change in Ambulation by at Least 1 Point |
3 | — |
| SECONDARY Mean Serum Concentration of Eculizumab |
206; 187; 230; 246 | — |
| SECONDARY Percentage Hemolysis |
88.5; 0.4; 0; 0.2; 0.4; 0.9 | <0.0001 sig |
| SECONDARY Mean Eculizumab Concentration in Cerebrospinal Fluid (CSF) |
34.7 | — |
| SECONDARY Mean Complement Protein 5 (C5) Concentration in CSF |
144; 60.8 | 0.0019 sig |
Summary
The purpose of this study is to determine if the drug eculizumab reduces the attack rate and improves outcome in patients with neuromyelitis optica.
Eligibility Criteria
Inclusion Criteria
- Diagnosis of NMO, as defined by 2006 criteria OR NMO seropositive spectrum disorder (Recurrent ON or longitudinally extensive transverse myelitis (LETM)). All patients must be NMO-IgG seropositive.
- Clinical evidence of at least 2 relapses in last 6 months or 3 relapses in the last 12 months (with at least 1 relapse occurring in the preceding 6 months).
- Age ≥18 years
- Corrected visual acuity 20/100 or better in at least one eye. If fails item # 4 then entry allowed but only if last attack was myelitis and only attacks of myelitis are considered as outcome measurement.
- Ambulatory (with or without walker). If fails item # 5 then entry allowed but only if last attack was ON and only attacks of ON are considered as outcome measurement.
- Provision of written informed consent (see attached) to participate in the study.
- N. meningitidis vaccination at least 14 days prior to receiving the first eculizumab infusion. If patient in midst of an acute relapse, then relapse will be treated with standard therapy and vaccination given only after a minimum of 4 weeks post attack onset.
Exclusion Criteria
Candidates will be excluded from study entry if any of the following criteria are met at the time of randomization:
- Progressive neurological deterioration unrelated to relapses of ON or myelitis.
- Pregnant, breastfeeding, or intending to conceive during the course of the study
- Patients will not participate in any other clinical therapeutic study or will not have participated in any other experimental treatment study within 30 days of screening
- Patients with a history of splenectomy, because of a potential increased risk of developing meningococcal infection.
Data sourced from ClinicalTrials.gov (NCT00904826). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.