NMO Spectrum DisorderPhase 1/2NCT05154734
Efficacy and Safety of Belimumab in Neuromyelitis Optica Spectrum Disorders
A Phase 1/2 study testing Belimumab in people with NMO Spectrum Disorder, run by Tianjin Medical University General Hospital at 1 site in China.
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… Read more about this study
- Who is it for?
- Ages 18 years and olderHealthy volunteers can take part
- Conditions the study is for
- NMO Spectrum Disorder
- What is being tested?
- Belimumab
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 14 participants
- Where
- 1 siteChina
- Study dates
- 12 Dec 2021to 9 Apr 2024
- Phase
- Phase 1/2
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- Healthy volunteers accepted
- Adult, Older adult
Who may be able to join
- 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.
Who cannot join
- 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
Trial locations and contacts
1 site in 1 country.
China1 site
Tianjin Medical University General Hospital
Tianjin, Tianjin Municipality, 300052
About this study
Official title: Efficacy and Safety of Belimumab in Neuromyelitis Optica Spectrum Disorders (BEAT NMO)
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.
Read the 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
- Being tested
- Belimumab
- Medical subject headings
- Neuromyelitis Optica
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
BelimumabDrug
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.
| Study group | Role | What they receive |
|---|---|---|
| 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. Receives: Drug: Belimumab |
What is being measured
The primary outcome is the main question the study is designed to answer.
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.
Measured: From baseline to one year after
Secondary outcomes (5)
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.
Measured: 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)
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
Measured: From baseline to 52 weeks
Counts of peripheral blood B cell subsets
Compare peripheral blood plasma cells before and one year after initial intervention
Measured: From baseline to 52 weeks
Determination of serum AQP4 antibodies
Compare serum AQP4-ab titers before and one year after initial intervention
Measured: From baseline to 52 weeks
Incidence of treatment-emergent adverse events [safety and tolerability]
Adverse events related to belimumab are recorded
Measured: From baseline to 52 weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted13 Dec 2021
- Study start12 Dec 2021
- Primary completion30 Jun 2023
- Study completion9 Apr 2024
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for NMO Spectrum DisorderStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1/2
- Primary purpose
- Basic science
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 14 participants (actual)
Sponsor and investigators
- Lead sponsor
- Tianjin Medical University General Hospital
- Sponsor type
- University, hospital or other
- Collaborators
- Tang-Du Hospital
- Responsible party
- Principal investigator, Qiang Liu, Tianjin Medical University General Hospital
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05154734
- Sponsor's protocol ID
- IRB2021-YX-187-01
- Status last verified
- Oct 2024
- First submitted
- 7 Dec 2021
- Last updated
- 16 Oct 2024
- Results posted
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 16 Oct 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Efficacy and Safety of Belimumab in Neuromyelitis Optica Spectrum Disorders (BEAT NMO)
- ClinicalTrials.gov ID
- NCT05154734
- Acronym / sponsor ID
- IRB2021-YX-187-01
- Conditions
- NMO Spectrum Disorder
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 14 participants (actual)
- Lead sponsor
- Tianjin Medical University General HospitalUniversity, hospital or other
- Collaborators
- Tang-Du Hospital
- Study start
- 12 Dec 2021 (actual)
- Primary completion
- 30 Jun 2023 (actual)
- Study completion
- 9 Apr 2024 (actual)
- First posted
- 13 Dec 2021 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Basic science
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Belimumab | Experimental | Drug: BelimumabBelimumab 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. |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (6 listed)
- 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
Key exclusion criteria (5 listed)
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| BelimumabBelimumab 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 |
Primary outcome
The number of attacks
Time frame: From baseline to one year after
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.
5 secondary outcomes
Worsening in EDSS
Time frame: 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)
Time frame: From baseline to 52 weeks
Counts of peripheral blood B cell subsets
Time frame: From baseline to 52 weeks
Determination of serum AQP4 antibodies
Time frame: From baseline to 52 weeks
Incidence of treatment-emergent adverse events [safety and tolerability]
Time frame: From baseline to 52 weeks
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 16 Oct 2024.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
The record lists no contact. Use the official record to reach the sponsor.
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1/2 study testing Belimumab in people with NMO Spectrum Disorder, run by Tianjin Medical University General Hospital at 1 site in China.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 6 inclusion and 5 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 16 Oct 2024. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in China. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this combined phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
The number of attacks (time frame: From baseline to one year after). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 16 Oct 2024.