A RANDOMIZED, DOUBLE-BLIND, PLACEBO-CONTROLLED, MULTI-CENTER TRIAL TO EVALUATE THE SAFETY AND EFFICACY OF ECULIZUMAB IN PATIENTS WITH RELAPSING NEUROMYELITIS OPTICA (NMO) - ECU-NMO-301

• Register: EUCTR
• Last refreshed on: 21 December 2021
• Main ID: EUCTR2013-001150-10-IT
• Date of registration: 09/07/2013
• Prospective Registration: Yes
• Primary sponsor: Alexion Pharmaceuticals, Inc.
• Public title: A RANDOMIZED, DOUBLE-BLIND, PLACEBO-CONTROLLED, MULTI-CENTER TRIAL TO EVALUATE THE SAFETY AND EFFICACY OF ECULIZUMAB IN PATIENTS WITH RELAPSING NEUROMYELITIS OPTICA (NMO) - ECU-NMO-301
• Scientific title: A RANDOMIZED, DOUBLE-BLIND, PLACEBO-CONTROLLED, MULTI-CENTER TRIAL TO EVALUATE THE SAFETY AND EFFICACY OF ECULIZUMAB IN PATIENTS WITH RELAPSING NEUROMYELITIS OPTICA (NMO) - ECU-NMO-301
• Date of first enrolment: 17/09/2013
• Target sample size: 93
• Recruitment status: Not Recruiting
• URL: https://www.clinicaltrialsregister.eu/ctr-search/search?query=eudract_number:2013-001150-10
• Study type: Interventional clinical trial of medicinal product
• Study design: Controlled: yes; Randomised: yes; Open: no; Single blind: no; Double blind: yes; Parallel group: yes; Cross over: no; Other: no; If controlled, specify comparator, Other Medicinial Product: no; Placebo: yes; Other: no; Number of treatment arms in the trial: 2
• Phase: Human pharmacology (Phase I): no Therapeutic exploratory (Phase II): no Therapeutic confirmatory - (Phase III): yes Therapeutic use (Phase IV): no

Countries of recruitment

Argentina, Australia, Austria, Belgium, Brazil, Canada, Chile, Croatia, Czech Republic, Denmark, Finland, France, Germany, Hong Kong, Italy, Japan, Korea, Republic of, Peru, Russian Federation, Saudi Arabia, Singapore, Spain, Sweden, Switzerland, Taiwan, Turkey, United Arab Emirates, United Kingdom, United States

Contacts

• Name: European Clinical Trial Information
• Affiliation: ALEXION EUROPE SAS

• Name: European Clinical Trial Information
• Affiliation: ALEXION EUROPE SAS

Key inclusion & exclusion criteria

Inclusion criteria:
- Male or female patient = 18 years old
- Diagnosis of NMO or NMO spectrum disorder
- NMO-IgG seropositive at screening visit
- Historical Relapse of at least 2 in last 6 months or 3 in the last 12 months with the most recent attack occurring =30 days and =90 days prior to Randomization
- EDSS score =7
- If patients enter the trial receiving an IST, they must be on the IST on which they had the most recent relapse and must remain at the same dose thoughout the trial
- 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 84
F.1.3 Elderly (>=65 years) yes
F.1.3.1 Number of subjects for this age range 9

Exclusion criteria:
- Use of rituximab or mitoxantrone within 3 months prior to randomization
- Use of IVIg within 3 weeks prior to screening
- Has received treatment with eculizumab at any time prior to enrolling in this study


Age minimum:
Age maximum:
Gender:
Female: yes
Male: yes

Health Condition(s) or Problem(s) studied

Therapeutic area: Diseases [C] - Nervous System Diseases [C10]

Relapsing neuromyelitis optica
MedDRA version: 16.0 Level: PT Classification code 10029322 Term: Neuromyelitis optica System Organ Class: 10029205 - Nervous system disorders

Intervention(s)

Trade Name: SOLIRIS
Pharmaceutical Form: Concentrate for solution for infusion
INN or Proposed INN: ECULIZUMAB
CAS Number: 219685-50-4
Current Sponsor code: h5G1.1-mAb
Concentration unit: mg/ml milligram(s)/millilitre
Concentration type: equal
Concentration number: 10-
Pharmaceutical form of the placebo: Concentrate for solution for infusion
Route of administration of the placebo: Intravenous use

Primary Outcome(s)

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.

Primary end point(s): Time to first relapse

Secondary Objective: - Safety and tolerability of eculizumab compared with placebo
- Efficacy of eculizumab compared with placebo
- Describe the pharmacokinetics (PK) and pharmacodynamics (PD) of eculizumab

Timepoint(s) of evaluation of this end point: Relapse evaluation visits:
- Within 24/48 hours after relapse
- Week +1 after relapse
- Week +4 after relapse
- Week +6 after relapse

Completion of the trial after 29 relapses in 29 distinct patients.

Secondary Outcome(s)

Secondary end point(s): - Change from baseline in EDSS score at the End of Study Period
- Annualized relapse rate
- Change from baseline in EQ-5D at the End of the Study Period
- Change from baseline in modified Rankin Scale (mRS) score at the End of the Study Period
- Change from baseline in ambulatory function as measured by Hauser Ambulation Index at the End of the Study Period.

Timepoint(s) of evaluation of this end point: evaluation at:
- Screening visit
- Weeks 1, 4, 8, 12, 24, 36, 48, 60, 72, 84, 96, 104
- Relapse evaluation visits
- End of trial visit

Secondary ID(s)

ECU-NMO-301

2013-001150-10-DE

Source(s) of Monetary Support

ALEXION PHARMACEUTICALS INC

Ethics review

Status: Approved
Approval date: 17/09/2013
Contact:

Results

• Results available: Yes
• Date Posted: 21/06/2019
• Date Completed: 17/07/2018
• URL: https://www.clinicaltrialsregister.eu/ctr-search/trial/2013-001150-10/results