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STUDY OF ECULIZUMAB IN RELAPSING NEUROMYELITIS OPTICA SUBJECTS

Phase 1
Conditions
neuromyelitis optica
MedDRA version: 19.0Level: LLTClassification code 10029322Term: Neuromyelitis opticaSystem Organ Class: 100000004852
Therapeutic area: Diseases [C] - Nervous System Diseases [C10]
Registration Number
EUCTR2013-001150-10-DK
Lead Sponsor
Alexion Pharmaceuticals, Inc.
Brief Summary

Not available

Detailed Description

Not available

Recruitment & Eligibility

Status
ot Recruiting
Sex
All
Target Recruitment
132
Inclusion Criteria

- Male or female patient = 18 years old
- Diagnosis of NMO or NMO spectrum disorder
- NMO-IgG seropositive
- Historical Relapse of at least 2 relapses in the last 12 months or 3 relapses in the last 24 months, with at least 1 relapse in the 12 months prior to the Screening
- EDSS score =7
- Immunosuppressive therapy is allowed provided patients have been on
a stable maintenance dose prior to the Screening and remain on a stable dose for the duration of the study
- Patients must be willing and able to give written informed consent
Are the trial subjects under 18? no
Number of subjects for this age range:
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 119
F.1.3 Elderly (>=65 years) yes
F.1.3.1 Number of subjects for this age range 13

Exclusion Criteria

- Use of rituximab or mitoxantrone within 3 months prior to screening
- Use of IVIg within 3 weeks prior to screening

Study & Design

Study Type
Interventional clinical trial of medicinal product
Study Design
Not specified
Primary Outcome Measures
NameTimeMethod
Main Objective: Efficacy of eculizumab treatment as compared with placebo in relapsing NMO patients based on the time to first relapse and relapse risk reduction.;Secondary Objective: - Safety and tolerability of eculizumab compared with placebo<br>- Efficacy of eculizumab compared with placebo<br>- Describe the pharmacokinetics (PK) and pharmacodynamics (PD) of eculizumab <br><br>;Primary end point(s): Time to first adjudicated On-Trial Relapse ;Timepoint(s) of evaluation of this end point: Relapse evaluation visits:<br>- Within 24/48 hours after relapse<br>- Week +1 after relapse<br>- Week +4 after relapse<br>- Week +6 after relapse<br><br>Completion of the trial after 24 adjudicated On-Trial Relapse events in 24 distinct patients.
Secondary Outcome Measures
NameTimeMethod
Secondary end point(s): - Change from baseline in EDSS score at the End of Study Period<br>- Adjudicated annualized relapse rate<br>- Change from baseline in EQ-5D at the End of the Study Period <br>- Change from baseline in modified Rankin Scale (mRS) score at the End of the Study Period <br>- Change from baseline in ambulatory function as measured by Hauser Ambulation Index at the End of the Study Period.<br>;Timepoint(s) of evaluation of this end point: evaluation at:<br>- Screening visit<br>- Weeks 1, 4, 8, 12, 24, 36, 48, 60, 72, 84, 96, 104<br>- Relapse evaluation visits<br>- End of trial visit
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