ECU-NMO-302 Phase 3 Open-Label Extension Study of Eculizumab for NMO
Research type
Research Study
Full title
A Phase III, Open-Label, Extension Trial of ECU-NMO-301 to Evaluate the Safety and Efficacy of Eculizumab in Patients With Relapsing Neuromyelitis Optica (NMO)
IRAS ID
133324
Contact name
Saif Huda
Contact email
Sponsor organisation
Alexion Pharmaceuticals, Inc.
Eudract number
2013-001151-12
Clinicaltrials.gov Identifier
116,207, IND
Duration of Study in the UK
4 years, 0 months, 1 days
Research summary
Summary of Research
The purpose of this study is to evaluate the long-term safety of a study drug, called eculizumab, in patients with relapsing Neuromyelitis Optica (NMO). NMO is a rare disease of the central nervous system that mainly affects the optic nerves and spinal cord. Relapsing is when a patient’s health worsens after a period of improvement. Eculizumab is not approved for the treatment of NMO; it is considered to be experimental for use in patients with NMO.
This is an extension trial to the ECU-NMO-301 trial. A maximum of 132 patients will be enrolled in the ECU-NMO-301 trial. Patients who complete the ECU-NMO-301 trial may potentially enter this extension trial. Participants must be 18 years old and above. The study is running globally.
This is an open-label trial. Patients who exit the ECU-NMO-301 trial due to relapse will have their first extension trial visit once the Week 6 Relapse Evaluation Visit is completed and no later than 2 weeks (14 days ±2 days) after the last study drug dose; patients who exit the ECU-NMO-301 trial due to trial completion will have their first extension visit 2 weeks (14 days ±2 days) after the End of the Study Period (EOS) Visit.
The trial duration is 4 years including enrolment. Trial duration for an individual patient will vary depending on when the patient enters the trial; but the maximum time is 4 years.
Currently there is no therapy approved for the treatment of NMO. Given the seriousness of the disease, limitations of currently available therapies, and the limited options for treatment, there remains a significant unmet medical need for an effective and safe treatment for NMO.
Summary of Results
Final results from Study ECU-NMO-302 show a robust treatment effect of eculizumab on relapse risk reduction in patients with NMOSD. Results from this long-term extension study continue to support the safety and efficacy of eculizumab in patients with NMOSD. Consistent with data from other indications for which eculizumab is approved, the safety profile and therapeutic effect of eculizumab in patients with NMOSD is maintained with long-term exposure.
REC name
East Midlands - Leicester South Research Ethics Committee
REC reference
14/EM/0030
Date of REC Opinion
18 Feb 2014
REC opinion
Further Information Favourable Opinion