Efficacy and Safety of Belimumab in Neuromyelitis Optica Spectrum Disorders

NCT ID: NCT05154734

Last Updated: 2024-10-16

Basic Information

• Recruitment Status: COMPLETED
• Clinical Phase: PHASE1/PHASE2
• Total Enrollment: 14 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2021-12-12
• Study Completion Date: 2024-04-09

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.

Detailed Description

The investigators primarily aim to observe the number of attacks from initiation of belimumab treatment.

The secondary outcomes are to determine: The safety profile of belimumab in participants with NMO and whether belimumab improves Expanded Disability Status Scale (EDSS), et al.

Conditions

NMO Spectrum Disorder

Study Design

• Allocation Method: NA
• Intervention Model: SINGLE_GROUP
• Primary Study Purpose: BASIC_SCIENCE
• Blinding Strategy: NONE

Study Groups

Belimumab

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.

Group Type: EXPERIMENTAL

Belimumab

Intervention Type: 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.

Interventions

Belimumab

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.

Intervention Type: DRUG

Eligibility Criteria

Inclusion Criteria

1. Male or female patients ≥ 18 years old

2. Diagnosis of NMO or NMO spectrum disorder according to the 2015 International Panel for Neuromyelitis Optica Diagnosis criteria

3. 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.

4. EDSS <= 6.0

5. Patients were seropositive for AQP4-IgG

6. Able and willing to give written informed consent and comply with the requirements of the study protocol.

Exclusion Criteria

1. 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)

2. Participation in another interventional trial within the last 3 months

3. Tumor disease currently or within last 5 years

4. Pregnant, breastfeeding, or child-bearing potential during the course of the study

5. Clinically relevant heart, liver, kidney or bone marrow function disorder

• Minimum Eligible Age: 18 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: Yes

Sponsors

Tang-Du Hospital

OTHER

Sponsor Role: collaborator

Tianjin Medical University General Hospital

OTHER

Sponsor Role: lead

Responsible Party

Qiang Liu

Professor of Department of Neurology

Responsibility Role: PRINCIPAL_INVESTIGATOR

Locations

Tianjin Medical University General Hospital

Tianjin, Tianjin Municipality, China

Countries

China

Other Identifiers

IRB2021-YX-187-01

Identifier Type: -

Identifier Source: org_study_id