Tocilizumab in patients with neuromyelitis optica

Not Applicable

• Conditions: euromyelitis optica; D009471

• Registration Number: JPRN-jRCTs031180152
• Lead Sponsor: Yamamura Takashi

Brief Summary

This is an exploratory clinical study investigating the safety profiles and efficacy of tocilizumab for refractory anti-AQP4-Ab+ neuromyelitis optica patients. In total, 20 patients were enrolled. The safety profiles included 16 infections, mostly mild and there were no unexpected severe adverse events. Clinical efficacy were apparent with significant reduction of annual relapse rates after tocilizumab treatment.

Detailed Description

Not available

Study Timeline

• Results First Posted: 2014-04-15
• Study Start: 2019-02-25
• Study Completion: 2020-10-30
• Last Update Posted: 17 October 2023

Recruitment & Eligibility

• Status: Complete
• Sex: All
• Target Recruitment: 20

Inclusion Criteria

Diagnosis of neuromyelitis optica (NMO), as defined by 2006 criteria published in NEUROLOGY vol. 66 or NMO seropositive spectrum disorder (recurrent optic neuritis or longitudinally extensive transverse myelitis). All patients must be anti-aquaporin 4 antibody seropositive.

Patients with intractable NMO who are resistant to the standard therapy or experiencing sever adverse reactions of the current therapy.

History of at least one relapse during the 1 year prior to initiation of this study.

Provision of written informed consent to participate in this study.

Exclusion Criteria

1) Patients with severe infection
tuberculosis, pneumocystis carinii pneumonia, nontuberculous mycobacterial infection (diagnosed by tuberculin test, beta-D glucan,chest X-ray)
When the tuberculin test shows intermediate or strong positivity, or the patients have a clinical history of contact with a patient with tuberculosis, isoniazid or rifampicin at a dose of 300 mg once daily for 6 to 9 months with pyridoxal at a dose of 10mg once daily initiated 3 weeks prior to tocilizumab.
Immunocompromised host
2) hepatitis B (positivity for HBS Ag and HBC Ab, hepatitis C (positivity for HCV Ab), or chronic active EB virus infection.
Patients with a history of tuberculosis infection or patients with pleural thickness or old tuberculin in chest X-ray.
3) Patients with a history of hypersensitivity to this drug.
4) Patients with a history of intestinal diverticulum.
5) Patients with interstitial pneumonitis.
6) Patients with leukopenia (white blood cell number less than 4000/mm3), lymphopenia (lymphocyte number less than 500/mm3) or seropositivity for beta-D-glucan in peripheral blood.
7) Pregnant women or patients with possible pregnancy.
8) Patients aged older than 20 and younger than 66 years.
9) Patients with a history of allergic reaction to tocilizumab.
10) Patients who cannot provide consent to participate in this study by themselves.
11) Patients whose EDSS are more than 7.

Study & Design

• Study Type: Interventional
• Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
1) Adverse events after the initiation of TCZ<br>2) Relapsing rate after the initiation of TCZ, as compared to pretreatment period

Secondary Outcome Measures

Name	Time	Method
1) EDSS/FS<br>2) Neurological findings (neurological examination, visual acuity, VAS(1), Critical flicker frequency(2), VEP(3), SSEP(4), ABR(5), Motor functional and sensory functional test)<br>3) New lesions in brain and spinal cord MRI<br>4) Cerebral spinal fluid cytokine profiles<br>5) Anti-aquaporin 4 antibody titer<br>6) Plasmablast frequency<br>7) T cell cytokine profile <br>8) Gut microbiota