Efficacy and Safety of Bortezomib as add-on Treatment in Relapsing Neuromyelitis Optica Spectrum Disorder

• Register: ClinicalTrials.gov
• Last refreshed on: 22 April 2024
• Main ID: NCT02893111
• Date of registration: 27/08/2016
• Prospective Registration: No
• Primary sponsor: Tianjin Medical University General Hospital
• Public title: Efficacy and Safety of Bortezomib as add-on Treatment in Relapsing Neuromyelitis Optica Spectrum Disorder
• Scientific title: Single-center, Open Label Trial of Bortezomib as add-on Treatment in Relapsing Neuromyelitis Optica Spectrum Disorder
• Date of first enrolment: December 2015
• Target sample size: 5
• Recruitment status: Completed
• URL: https://clinicaltrials.gov/ct2/show/NCT02893111
• Study type: Interventional
• Study design: Allocation: N/A. Intervention model: Single Group Assignment. Primary purpose: Treatment. Masking: None (Open Label).
• Phase: Phase 2

Countries of recruitment

China

Key inclusion & exclusion criteria

Inclusion Criteria:

- Age =18 years

- Diagnosis of NMOSD, as defined by 2015 criteria OR NMOSD 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)

- The B cell count must be normal (5-20% of total lymphocytes) in subjects before
administration of bortezomib

- Provision of written informed consent to participate in the study

- Corrected visual acuity 20/100 or better in at least one eye; otherwise, last attack
was myelitis and only attacks of myelitis are outcomes

- Ambulatory (with or without walker); otherwise, last attack was optic neuritis and
only attacks of optic neuritis are outcomes

Exclusion Criteria:

- Current evidence or known history of clinically significant infection (HSV, VZV, CMV,
EBV, HIV, Hepatitis viruses, Syphilis, etc)

- Pregnant, breastfeeding, or child-bearing potential 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

- Participation in another interventional trial within the last 3 months

- Pre-existent sensory or motor polyneuropathy = degree 2 (NCI CTC AE criteria), within
14 days before screening

- Heart or kidney insufficiency

- Tumor disease currently or within last 5 years

- Clinically relevant liver, kidney or bone marrow function disorder



Age minimum: 18 Years
Age maximum: 75 Years
Gender: Female

Health Condition(s) or Problem(s) studied

Neuromyelitis Optica Spectrum Disorder

Intervention(s)

Drug: Bortezomib

Primary Outcome(s)

Annual relapse rate (ARR) of NMOSD Attacks [Time Frame: Baseline, after 12 months of initial treatment]

Secondary Outcome(s)

Number of Subjects With Change in Visual Acuity in at Least One Eye by at Least One Point [Time Frame: Baseline, 12 months]

Retinal nerve fiber layer (RNFL) [Time Frame: Baseline, 12 months]

Change in Expanded Disability Status Scale (EDDS) Score [Time Frame: Baseline, 12 months]

Number of Participants with Adverse Events [Time Frame: Baseline, 12 months]

Cognition [Time Frame: Baseline, 12 months]

Timed 25-foot Walk [Time Frame: Baseline, 12 months]

MRI brain and spine [Time Frame: Baseline, 12 months]

Immunological assessments [Time Frame: Baseline, 12 months]

Secondary ID(s)

IRB2016-YX-021

Source(s) of Monetary Support

Please refer to primary and secondary sponsors