Efficacy and Safety of Belimumab in Neuromyelitis Optica Spectrum Disorders

NCT05154734 | Completed Phase 1

• 1 other identifier: IRB2021-YX-187-01
• Study Type: interventional
• Target: 14
• Locations: 1 country
• Sites: 1

Brief Summary

Neuromyelitis Optica Spectrum Disorders (NMOSD) is associated with a pathological humoral immune response against the aquaporin-4(AQP-4) water channel. Belimumab (Benlysta ®) is a human immunoglobulin G1λ monoclonal antibody that inhibits B-cell survival and differentiation by neutralizing soluble B lymphocyte stimulator. Belimumab may benefit some patients with NMOSD due to the important role of B cells in the pathogenesis of NMOSD. Clinical trials may be needed to observe its efficacy and safety.

Conditions

NMO Spectrum Disorder

Outcome Measures

Primary Outcomes (1)

• The number of attacks

  An acute attack was defined as a new neurological worsening lasting for at least 24 hours and occurring more than 30 days after the previous attack.

  From baseline to one year after

Secondary Outcomes (5)

• Worsening in EDSS

  Worsening from baseline in EDSS to 52 weeks

• Number of New, and/or Enlarging T2 Hyperintense Lesions as Detected by Optic nerve,brain and spinal cord Magnetic Resonance Imaging (MRI)

  From baseline to 52 weeks

• Counts of peripheral blood B cell subsets

  From baseline to 52 weeks

• Determination of serum AQP4 antibodies

  From baseline to 52 weeks

• Incidence of treatment-emergent adverse events [safety and tolerability]

  From baseline to 52 weeks

Study Arms (1)

Belimumab

EXPERIMENTAL

Belimumab will be intravenously administered with a dose of 10mg/kg on Days 0,14 and28, then every 28 days until week 48, with a final evaluation at week 52.

Drug: Belimumab

Interventions

Belimumab DRUG

Belimumab will be intravenously administered with a dose of 10mg/kg on Days 0,14 and28, then every 28 days until week 48, with a final evaluation at week 52.

Belimumab

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: Yes
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Male or female patients ≥ 18 years old
• Diagnosis of NMO or NMO spectrum disorder according to the 2015 International Panel for Neuromyelitis Optica Diagnosis criteria
• Clinical evidence of either at least one attack requiring rescue therapy (intravenous corticosteroids, intravenous immunoglobulin, plasma exchange, or a combination of these therapies) in the year before screening or at least two attacks requiring rescue therapy in the 2 years before screening.
• EDSS \<= 6.0
• Patients were seropositive for AQP4-IgG
• Able and willing to give written informed consent and comply with the requirements of the study protocol.

You may not qualify if:

• Current evidence or known history of clinically significant infection (Herpes simplex virus, varicella-zoster virus, cytomegalovirus, Epstein-Barr virus, human immunodeficiency virus, Hepatitis viruses, Syphilis, etc)
• Participation in another interventional trial within the last 3 months
• Tumor disease currently or within last 5 years
• Pregnant, breastfeeding, or child-bearing potential during the course of the study
• Clinically relevant heart, liver, kidney or bone marrow function disorder

Sponsors & Collaborators

• Tianjin Medical University General Hospital: lead
• Tang-Du Hospital: collaborator

Study Sites (1)

Tianjin Medical University General Hospital

Tianjin, Tianjin Municipality, 300052, China

Location

MeSH Terms

Conditions

Neuromyelitis Optica

Interventions

belimumab

Condition Hierarchy (Ancestors)

Myelitis, Transverse; Demyelinating Autoimmune Diseases, CNS; Autoimmune Diseases of the Nervous System; Nervous System Diseases; Optic Neuritis; Optic Nerve Diseases; Cranial Nerve Diseases; Demyelinating Diseases; Eye Diseases; Autoimmune Diseases; Immune System Diseases

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NA
• Masking: NONE
• Purpose: BASIC SCIENCE
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Professor of Department of Neurology

Study Record Dates

• First Submitted: December 7, 2021
• First Posted: December 13, 2021
• Study Start: December 12, 2021
• Primary Completion: June 30, 2023
• Study Completion: April 9, 2024
• Last Updated: October 16, 2024

Record last verified: 2024-10

Locations

CN (1)