Neuromyelitis Optica Clinical Trial
Official title:
An Open Label Study of the Effects of Eculizumab in Neuromyelitis Optica
Verified date | August 2013 |
Source | Mayo Clinic |
Contact | n/a |
Is FDA regulated | No |
Health authority | United States: Institutional Review Board |
Study type | Interventional |
The purpose of this study is to determine if the drug eculizumab reduces the attack rate and improves outcome in patients with neuromyelitis optica.
Status | Completed |
Enrollment | 14 |
Est. completion date | December 2011 |
Est. primary completion date | December 2011 |
Accepts healthy volunteers | No |
Gender | Both |
Age group | 18 Years and older |
Eligibility |
Inclusion Criteria: 1. 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. 2. 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). 3. Age =18 years 4. 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. 5. 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. 6. Provision of written informed consent (see attached) to participate in the study. 7. 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: 1. Progressive neurological deterioration unrelated to relapses of ON or myelitis. 2. Pregnant, breastfeeding, or intending to conceive during the course of the study 3. 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 4. Patients with a history of splenectomy, because of a potential increased risk of developing meningococcal infection. |
Endpoint Classification: Safety/Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment
Country | Name | City | State |
---|---|---|---|
United States | Mayo Clinic | Rochester | Minnesota |
United States | Mayo Clinic | Scottsdale | Arizona |
Lead Sponsor | Collaborator |
---|---|
Mayo Clinic | Alexion Pharmaceuticals |
United States,
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. — View Citation
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. Epub 2007 Oct 10. — View Citation
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. Epub 2005 Aug 8. — View Citation
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. — View Citation
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 — View Citation
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. Erratum in: Nat Biotechnol. 2007 Dec;25(12):1488. — View Citation
Wingerchuk DM, Lennon VA, Lucchinetti CF, Pittock SJ, Weinshenker BG. The spectrum of neuromyelitis optica. Lancet Neurol. 2007 Sep;6(9):805-15. Review. — View Citation
Wingerchuk DM, Lennon VA, Pittock SJ, Lucchinetti CF, Weinshenker BG. Revised diagnostic criteria for neuromyelitis optica. Neurology. 2006 May 23;66(10):1485-9. — View Citation
Type | Measure | Description | Time frame | Safety issue |
---|---|---|---|---|
Primary | Median Number of Neuromyelitis Optica (NMO) Attacks Per Year | baseline, after 12 months of treatment | No | |
Secondary | Number Subjects Experiencing an NMO Attack in 12 Months of Eculizumab Treatment | 12 months | No | |
Secondary | Change in Expanded Disability Status Scale (EDDS) Score | The EDSS is an ordinal clinical rating scale ranging from 0 (normal neurologic examination) to 10 (death) in half-point increments. | baseline, 12 months | No |
Secondary | Number of Subjects With Change in Visual Acuity in at Least One Eye by at Least One Point | Visual acuity was measured using the the Visual Acuity subscale of the Opticospinal Impairment Score (OSIS) for Exacerbations. This subscale ranges from 0 (normal) to 8 (no light perception). | 12 months | No |
Secondary | Number of Subjects With Change in Ambulation by at Least 1 Point | Ambulation was measured by the Hauser Ambulation Index, which ranges from 0 (asymptomatic; fully active) to 9 (restricted to wheelchair; unable to transfer self independently.) | 12 months | No |
Secondary | Mean Serum Concentration of Eculizumab | 6 weeks, 3 months, 6 months, 9 months, 12 months | No | |
Secondary | Percentage Hemolysis | Percentage of hemolysis is a measure of complement activity. Less than 20% lysis is deemed to be complete complement inhibition. | baseline, 6 weeks, 3 months, 6 months, 9 months, 12 months | No |
Secondary | Mean Eculizumab Concentration in Cerebrospinal Fluid (CSF) | 3 months | No | |
Secondary | Mean Complement Protein 5 (C5) Concentration in CSF | baseline, 3 months | No |
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