Efficacy and Safety of Belimumab in Neuromyelitis Optica Spectrum Disorders

NMO Spectrum Disorder Clinical Trial

Official title:

Efficacy and Safety of Belimumab in Neuromyelitis Optica Spectrum Disorders (BEAT NMO)

Clinical Trial Details — Status: Recruiting

Administrative data

• NCT number: NCT05154734
• Other study ID #: IRB2021-YX-187-01
• Status: Recruiting
• Phase: Phase 1/Phase 2
• Start date: October 30, 2021
• Est. completion date: July 30, 2023

Study information

• Verified date: March 2023
• Source: Tianjin Medical University General Hospital
• Contact: Qiang Liu, M.D.,Ph.D.
• Phone: +86 15022439149
• Email: qliu[@]tmu.edu.com
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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. Clincial trials may be needed to observe its efficacy and safety.

Description:

The investigators primarily aim to observe the time to first relapse 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.

Recruitment information / eligibility

• Status: Recruiting
• Enrollment: 12
• Est. completion date: July 30, 2023
• Est. primary completion date: June 30, 2023
• Accepts healthy volunteers: Accepts Healthy Volunteers
• Gender: All
• Age group: 18 Years and older

Eligibility

Inclusion Criteria:

1. Male or female patients = 18 years old

2. Diagnosis of NMO or NMO spectrum disorder according to the 2015 International diagnostic criteria for neuromyelitis optic

3. Clinical evidence of either at least one attack requiring rescue therapy (intravenous corticosteroids,intravenous immunoglobulin,plasma exchange,or a combination of these therapies) or at least two attacks requiring rescue therapy in the 2 years before screening.

4. EDSS <= 6.0

5. 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

Related Conditions & MeSH terms

• Neuromyelitis Optica
• NMO Spectrum Disorder

Intervention

Drug:
• 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.

Locations

Country	Name	City	State
China	Tianjin Medical University General Hospital	Tianjin	Tianjin

Sponsors (1)

Lead Sponsor	Collaborator
Tianjin Medical University General Hospital

Country where clinical trial is conducted

China, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	First time to relapse	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	Worsening in EDSS	The Expanded Disability Status Scale (EDSS) is a rating system that is frequently used for classifying and standardizing the severity and progression. EDSS ranges from 0 to 10.	Worsening from baseline in EDSS to 52 weeks
Secondary	Number of New, and/or Enlarging T2 Hyperintense Lesions as Detected by Optic nerve,brain and spinal cord Magnetic Resonance Imaging (MRI)	The total number of new and/or enlarging T2 lesions for all participants was calculated as the sum of the individual number of lesions at Weeks 12, 24, and 52	From baseline to 52 weeks
Secondary	Counts of peripheral blood B cell subsets	Compare peripheral blood plasma cells before and one year after initial intervention	From baseline to 52 weeks
Secondary	Determination of serum AQP4 antibodies	Compare serum AQP4-ab titers before and one year after initial intervention	From baseline to 52 weeks
Secondary	Incidence of treatment-emergent adverse events [safety and tolerability]	Adverse events related to belimumab are recorded	From baseline to 52 weeks