NCT00904826

Brief 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.

Trial Health

87
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
14

participants targeted

Target at below P25 for phase_1

Timeline
Completed

Started Apr 2009

Typical duration for phase_1

Geographic Reach
1 country

2 active sites

Status
completed

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

Study Start

First participant enrolled

April 1, 2009

Completed
2 months until next milestone

First Submitted

Initial submission to the registry

May 18, 2009

Completed
2 days until next milestone

First Posted

Study publicly available on registry

May 20, 2009

Completed
2.5 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

December 1, 2011

Completed
Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

December 1, 2011

Completed
1.9 years until next milestone

Results Posted

Study results publicly available

November 4, 2013

Completed
Last Updated

November 4, 2013

Status Verified

August 1, 2013

Enrollment Period

2.7 years

First QC Date

May 18, 2009

Results QC Date

August 30, 2013

Last Update Submit

August 30, 2013

Conditions

Neuromyelitis OpticaDevic's Disease

Keywords

NMODevic's diseaseNeuromyelitis opticaNMO-IgGAquaporin-4 antibodyEculizumabComplement

Outcome Measures

Primary Outcomes (1)

  • Median Number of Neuromyelitis Optica (NMO) Attacks Per Year

    baseline, after 12 months of treatment

Secondary Outcomes (8)

  • Number Subjects Experiencing an NMO Attack in 12 Months of Eculizumab Treatment

    12 months

  • Change in Expanded Disability Status Scale (EDDS) Score

    baseline, 12 months

  • Number of Subjects With Change in Visual Acuity in at Least One Eye by at Least One Point

    12 months

  • Number of Subjects With Change in Ambulation by at Least 1 Point

    12 months

  • Mean Serum Concentration of Eculizumab

    6 weeks, 3 months, 6 months, 9 months, 12 months

  • +3 more secondary outcomes

Study Arms (1)

Eculizumab

EXPERIMENTAL

The patient will receive eculizumab at a dose of 600mg intravenously (an infusion given into the vein) each week for 4 weeks, then 900mg intravenously at the fifth week, then 900mg every 2 weeks for 48 weeks. Subjects will receive therapy for a total of 12 months.

Drug: Eculizumab

Interventions

EculizumabDRUG

The patient will receive eculizumab at a dose of 600mg intravenously (an infusion given into the vein) each week for 4 weeks, then 900mg intravenously at the fifth week, then 900mg every 2 weeks for 48 weeks. The first infusion will be given at Mayo Clinic site; subsequent infusions will be administered in the subject's home by a company which will send a nurse to administer the infusion. Subjects will receive therapy for a total of 12 months.

Also known as: Soliris
Eculizumab

Eligibility Criteria

Age18 Years+
Sexall
Healthy VolunteersNo
Age GroupsAdult (18-64), Older Adult (65+)

You may qualify if:

  • 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.

You may not qualify if:

  • 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.

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (2)

Mayo Clinic

Scottsdale, Arizona, 85259, United States

Location

Mayo Clinic

Rochester, Minnesota, 55905, United States

Location

Related Publications (8)

  • Wingerchuk DM, Lennon VA, Lucchinetti CF, Pittock SJ, Weinshenker BG. The spectrum of neuromyelitis optica. Lancet Neurol. 2007 Sep;6(9):805-15. doi: 10.1016/S1474-4422(07)70216-8.

    PMID: 17706564BACKGROUND

  • Lennon VA, Wingerchuk DM, Kryzer TJ, Pittock SJ, Lucchinetti CF, Fujihara K, Nakashima I, Weinshenker BG. A serum autoantibody marker of neuromyelitis optica: distinction from multiple sclerosis. Lancet. 2004 Dec 11-17;364(9451):2106-12. doi: 10.1016/S0140-6736(04)17551-X.

    PMID: 15589308BACKGROUND

  • Lennon VA, Kryzer TJ, Pittock SJ, Verkman AS, Hinson SR. IgG marker of optic-spinal multiple sclerosis binds to the aquaporin-4 water channel. J Exp Med. 2005 Aug 15;202(4):473-7. doi: 10.1084/jem.20050304. Epub 2005 Aug 8.

    PMID: 16087714BACKGROUND

  • Wingerchuk DM, Lennon VA, Pittock SJ, Lucchinetti CF, Weinshenker BG. Revised diagnostic criteria for neuromyelitis optica. Neurology. 2006 May 23;66(10):1485-9. doi: 10.1212/01.wnl.0000216139.44259.74.

    PMID: 16717206BACKGROUND

  • Hinson SR, Pittock SJ, Lucchinetti CF, Roemer SF, Fryer JP, Kryzer TJ, Lennon VA. Pathogenic potential of IgG binding to water channel extracellular domain in neuromyelitis optica. Neurology. 2007 Dec 11;69(24):2221-31. doi: 10.1212/01.WNL.0000289761.64862.ce. Epub 2007 Oct 10.

    PMID: 17928579BACKGROUND

  • Rother RP, Rollins SA, Mojcik CF, Brodsky RA, Bell L. Discovery and development of the complement inhibitor eculizumab for the treatment of paroxysmal nocturnal hemoglobinuria. Nat Biotechnol. 2007 Nov;25(11):1256-64. doi: 10.1038/nbt1344.

    PMID: 17989688BACKGROUND

  • Hinson SR, McKeon A, Fryer JP, Apiwattanakul M, Lennon VA, Pittock SJ. Prediction of neuromyelitis optica attack severity by quantitation of complement-mediated injury to aquaporin-4-expressing cells. Arch Neurol. 2009 Sep;66(9):1164-7. doi: 10.1001/archneurol.2009.188.

    PMID: 19752309BACKGROUND

  • Pittock SJ, Lennon VA, McKeon A, Mandrekar J, Weinshenker BG, Lucchinetti CF, O'Toole O, Wingerchuk DM. Eculizumab in AQP4-IgG-positive relapsing neuromyelitis optica spectrum disorders: an open-label pilot study. Lancet Neurol. 2013 Jun;12(6):554-62. doi: 10.1016/S1474-4422(13)70076-0. Epub 2013 Apr 26.

    PMID: 23623397RESULT

MeSH Terms

Conditions

Neuromyelitis Optica

Interventions

eculizumab

Condition Hierarchy (Ancestors)

Myelitis, TransverseDemyelinating Autoimmune Diseases, CNSAutoimmune Diseases of the Nervous SystemNervous System DiseasesOptic NeuritisOptic Nerve DiseasesCranial Nerve DiseasesDemyelinating DiseasesEye DiseasesAutoimmune DiseasesImmune System Diseases

Results Point of Contact

Title
Dr. Sean J. Pittock
Organization
Mayo Clinic
pittock.sean@mayo.edu
507-284-4741

Study Officials

  • Sean J. Pittock, M.D.

    Mayo Clinic

    PRINCIPAL INVESTIGATOR

Publication Agreements

PI is Sponsor Employee
Yes
Restrictive Agreement
No

Study Design

Study Type
interventional
Phase
phase 1
Allocation
NA
Masking
NONE
Purpose
TREATMENT
Intervention Model
SINGLE GROUP
Sponsor Type
OTHER

Study Record Dates

First Submitted

May 18, 2009

First Posted

May 20, 2009

Study Start

April 1, 2009

Primary Completion

December 1, 2011

Study Completion

December 1, 2011

Last Updated

November 4, 2013

Results First Posted

November 4, 2013

Record last verified: 2013-08

Locations

US(2)