Neuromyelitis Optica Spectrum DisorderPhase 1/2NCT05504694
Ofatumumab in AQP4-IgG Seropositive NMOSD
A Phase 1/2 study testing Ofatumumab in people with Neuromyelitis Optica Spectrum Disorder, run by Tang-Du Hospital at 1 site in China.
Trial summary
This is an open-label, single-arm, multicentre prospective pilot study to assess the efficacy and safety of ofatumumab in patients with AQP4-IgG seropositive neuromyelitis optica spectrum disorder (NMOSD) in China. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Neuromyelitis Optica Spectrum Disorder
- What is being tested?
- Ofatumumab
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 5 participants
- Where
- 1 siteChina
- Study dates
- 28 Jun 2022to 31 Jul 2026
- Phase
- Phase 1/2
- Are there results?
- Not yet: the study is under way
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
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Diagnosis of NMOSD according to the 2015 International Panel Diagnostic Criteria for NMOSD with AQP4-IgG.
- Clinical evidence of at least 2 relapses (including first attack) in past 24 months with at least 1 relapse occurring in the preceding 12 months.
- Adults aged ≥18 years old.
- Expanded disability status scale (EDSS) score between 0 and 7.5 (inclusive).
- Provision of written informed consent to participate in this study.
- Only oral corticosteroids were permitted at screening (≤10mg equivalent per day), which should be terminated within one month.
- Effective contraception was used for female patients with fertility during the treatment or at least 3 months after stopping medication.
Who cannot join
- Progressive neurological deterioration unrelated to relapses of NMOSD, or presence of neurological findings suspected with PML.
- Pregnant or breastfeeding patients and those with family planning during the study period.
- Patients participating in any other clinical therapeutic study at the screening or within 30 days of screening.
- Patients with splenectomy or history of no spleen, and those with planned surgery (excluding minor surgery) during the study period.
- Presence of uncontrolled severe concurrent diseases; long-term glucocorticoids or immunosuppressants use due to other autoimmune diseases, or presence of other chronic diseases that cannot receiving immunosuppression.
- Active infection at within 4 weeks before baseline.
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- Positive for HBV or HCV.
- Evidence of latent or active tuberculosis (TB).
- Have received any live or live-attenuated vaccine within 6 weeks before baseline.
- History of malignancy in past 5 years, including solid tumor, malignant hematopathy and carcinoma in situ.
- History of severe allergic reactions to biological agents.
- Inability to provide written informed consent.
Trial locations and contacts
1 site in 1 country. Contact the study team or a site near you to ask about taking part.
China1 site, 1 recruiting
- Recruiting
Tangdu Hospital
Xi'an, Shaanxi
Jun Guo, M.D., 86-29-8477 8844, guojun_81@163.com
Yan Jia, M.S., 86-29-8471 7483, neurologist_jiayan@163.com
About this study
Official title: Efficacy and Safety of Ofatumumab in AQP4-IgG Seropositive NMOSD: an Open-label, Single-arm, Multicentre Prospective Pilot Study
This is an open-label, single-arm, multicentre prospective pilot study to assess the efficacy and safety of ofatumumab in patients with AQP4-IgG seropositive neuromyelitis optica spectrum disorder (NMOSD) in China.
Read the detailed description
Neuromyelitis optica spectrum disorder (NMOSD) is a rare but severe demyelinating disorder that affects mainly adult patients. It is associated with a pathological B cell-mediated humoral immune response against the aquaporin-4 (AQP4) water channel. Monoclonal antibodies against CD20 have been shown to be effective for prevention of relapses in patients with NMOSD, and therefore been recommended as first-line therapy for this disorder. Ofatumumab (OFA), a fully humanized anti-CD20 monoclonal antibody, has been approved for multiple sclerosis treatment. However, prospective multicenter studies are needed to determine the efficacy and safety of ofatumumab in treating NMOSD.
- Conditions
- Neuromyelitis Optica Spectrum Disorder
- Being tested
- Ofatumumab
- Keywords
- Neuromyelitis Optica Spectrum Disorder, Aquaporin 4, Ofatumumab
- Medical subject headings
- Neuromyelitis Optica
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
OfatumumabDrug
Also called Arzerra
The enrolled patients will receive ofatumumab (20 mg/0.4 ml) subcutaneously administered at baseline, Day 7, Day 14 and monthly thereafter. Patients will receive ofatumumab therapy for a total of 48 weeks.
The first 4 infusions will be administered at study center site; subsequent infusions will be given in the patient's home with a nurse online interview to administer the infusion.
| Study group | Role | What they receive |
|---|---|---|
| Ofatumumab | Experimental | The enrolled patients will receive ofatumumab (20 mg/0.4 ml) subcutaneously administered at baseline, Day 7, Day 14 and monthly thereafter. Patients will receive ofatumumab therapy for a total of 48 weeks. Receives: Drug: Ofatumumab |
What is being measured
The primary outcome is the main question the study is designed to answer.
Change from baseline in annual relapse rate (ARR) at last follow-up visit
Pre-treatment ARR was determined at baseline by the total number of attacks divided by disease course from onset to baseline; post-treatment ARR is determined at 12 months after treatment by the number of relapses divided by 12 months.
Measured: baseline, 12 months
Secondary outcomes (8)
Change from baseline in Expanded Disability Status Scale (EDSS) score
Patients are followed up and EDSS score is determined. In general, the minimum and maximum scores of EDSS are 0 and 10, respectively, with higher scores meaning a worse outcome.
Measured: baseline, 3 months, 6 months, 9 months, 12 months
Change from baseline in lesion burden on MRI T2-weighted images
MRI is conducted to measure the lesion burden on T2-weighted images.
Measured: baseline, 6 months, 12 months
Change from baseline in optic coherence tomography (OCT) measures
OCT is done to measure peripapillary retinal nerve fiber layer (RNFL) thickness, macular ganglion cell-inner plexiform layer (GCIPL) thickness, and macular inner nuclear layer (INL) thickness.
Measured: baseline, 6 months, 12 months
Change from baseline in the frequencies of circulating B cell subsets
Circulating B cell monitoring is conducted to evaluate the effectiveness of B-cell-depletion therapy. FACS is used to measure the frequencies of CD19+ B cells, CD19+CD27+ memory B cells, and CD19+CD38+CD27+ plasmablasts.
Measured: baseline, 1 week, 2 weeks, 1 month, 3 months, 6 months, 9 months, 12 months
Change from baseline in immune landscape
The dynamic changes of immune cells and cytokines are monitored, such as Th1 cells, Th2 cells, Th17 cells, NK cells, IL-1β, IL-2, IL-4, etc.
Measured: baseline, 1 week, 2 weeks, 1 month, 3 months, 6 months, 9 months, 12 months
Biochemical indicators monitoring
Blood routine test, liver and kidney function, immunoglobulins, complement, and serum AQP4-IgG titer.
Measured: baseline, 1 month, 3 months, 6 months, 9 months, 12 months
Assessment of functional questionnaire
SF-36, Functional Assessment of Chronic Illness Therapy (FACIT), EuroQol Health State (EQ-5D), Visual Analogue Pain Scale (VAPS), Timed 25-foot Walk Test, etc.
Measured: baseline, 1 month, 3 months, 6 months, 9 months, 12 months
Adverse events
Ofatumumab-related adverse events (AEs) are evaluated and the rate of AEs is recorded.
Measured: 1 week, 2 weeks, 1 month, 3 months, 6 months, 9 months, 12 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted17 Aug 2022
- Study start28 Jun 2022
- Primary completion30 Apr 2026 (estimated)
- Study completion31 Jul 2026 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Neuromyelitis Optica Spectrum DisorderStudies of the same condition, open and under way.
- More trials run by Tang-Du HospitalThe sponsor's other registered studies.
- 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
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 5 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Tang-Du Hospital
- Sponsor type
- University, hospital or other
- Collaborators
- Henan Provincial People's Hospital
- Responsible party
- Principal investigator, Jun Guo, MD, Tang-Du Hospital
- Investigators
- Jun Guo, M.D., Principal investigator, Tang-Du Hospital
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05504694
- Sponsor's protocol ID
- K202206-13
- Status last verified
- Jan 2026
- First submitted
- 16 Aug 2022
- Last updated
- 30 Jan 2026
- 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 30 Jan 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Efficacy and Safety of Ofatumumab in AQP4-IgG Seropositive NMOSD: an Open-label, Single-arm, Multicentre Prospective Pilot Study
- ClinicalTrials.gov ID
- NCT05504694
- Acronym / sponsor ID
- K202206-13
- Conditions
- Neuromyelitis Optica Spectrum Disorder
- Keywords
- Neuromyelitis Optica Spectrum Disorder, Aquaporin 4, Ofatumumab
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 5 participants (estimated)
- Lead sponsor
- Tang-Du HospitalUniversity, hospital or other
- Collaborators
- Henan Provincial People's Hospital
- Study start
- 28 Jun 2022 (actual)
- Primary completion
- 30 Apr 2026 (estimated)
- Study completion
- 31 Jul 2026 (estimated)
- First posted
- 17 Aug 2022 (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.
This is an open-label, single-arm, multicentre prospective pilot study to assess the efficacy and safety of ofatumumab in patients with AQP4-IgG seropositive neuromyelitis optica spectrum disorder (NMOSD) in China.
Detailed description
Neuromyelitis optica spectrum disorder (NMOSD) is a rare but severe demyelinating disorder that affects mainly adult patients. It is associated with a pathological B cell-mediated humoral immune response against the aquaporin-4 (AQP4) water channel. Monoclonal antibodies against CD20 have been shown to be effective for prevention of relapses in patients with NMOSD, and therefore been recommended as first-line therapy for this disorder. Ofatumumab (OFA), a fully humanized anti-CD20 monoclonal antibody, has been approved for multiple sclerosis treatment. However, prospective multicenter studies are needed to determine the efficacy and safety of ofatumumab in treating NMOSD.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Ofatumumab | Experimental | Drug: OfatumumabThe enrolled patients will receive ofatumumab (20 mg/0.4 ml) subcutaneously administered at baseline, Day 7, Day 14 and monthly thereafter. Patients will receive ofatumumab therapy for a total of 48 weeks. |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (7 listed)
- Diagnosis of NMOSD according to the 2015 International Panel Diagnostic Criteria for NMOSD with AQP4-IgG.
- Clinical evidence of at least 2 relapses (including first attack) in past 24 months with at least 1 relapse occurring in the preceding 12 months.
- Adults aged ≥18 years old.
- Expanded disability status scale (EDSS) score between 0 and 7.5 (inclusive).
- Provision of written informed consent to participate in this study.
Key exclusion criteria (12 listed)
- Progressive neurological deterioration unrelated to relapses of NMOSD, or presence of neurological findings suspected with PML.
- Pregnant or breastfeeding patients and those with family planning during the study period.
- Patients participating in any other clinical therapeutic study at the screening or within 30 days of screening.
- Patients with splenectomy or history of no spleen, and those with planned surgery (excluding minor surgery) during the study period.
- Presence of uncontrolled severe concurrent diseases; long-term glucocorticoids or immunosuppressants use due to other autoimmune diseases, or presence of other chronic diseases that cannot receiving immunosuppression.
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 |
|---|---|---|
| OfatumumabAlso: ArzerraThe enrolled patients will receive ofatumumab (20 mg/0.4 ml) subcutaneously administered at baseline, Day 7, Day 14 and monthly thereafter. Patients will receive ofatumumab therapy for a total of 48 weeks. The first 4 infusions will be administered at study center site; subsequent infusions will be given in the patient's home with a nurse online interview to administer the infusion. | Drug | Ofatumumab |
Primary outcome
Change from baseline in annual relapse rate (ARR) at last follow-up visit
Time frame: baseline, 12 months
Pre-treatment ARR was determined at baseline by the total number of attacks divided by disease course from onset to baseline; post-treatment ARR is determined at 12 months after treatment by the number of relapses divided by 12 months.
8 secondary outcomes
Change from baseline in Expanded Disability Status Scale (EDSS) score
Time frame: baseline, 3 months, 6 months, 9 months, 12 months
Change from baseline in lesion burden on MRI T2-weighted images
Time frame: baseline, 6 months, 12 months
Change from baseline in optic coherence tomography (OCT) measures
Time frame: baseline, 6 months, 12 months
Change from baseline in the frequencies of circulating B cell subsets
Time frame: baseline, 1 week, 2 weeks, 1 month, 3 months, 6 months, 9 months, 12 months
Change from baseline in immune landscape
Time frame: baseline, 1 week, 2 weeks, 1 month, 3 months, 6 months, 9 months, 12 months
Biochemical indicators monitoring
Time frame: baseline, 1 month, 3 months, 6 months, 9 months, 12 months
Assessment of functional questionnaire
Time frame: baseline, 1 month, 3 months, 6 months, 9 months, 12 months
Adverse events
Time frame: 1 week, 2 weeks, 1 month, 3 months, 6 months, 9 months, 12 months
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 30 Jan 2026.
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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.
Jun Guo, M.D. principal investigator
Tang-Du Hospital
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 Ofatumumab in people with Neuromyelitis Optica Spectrum Disorder, run by Tang-Du 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 7 inclusion and 12 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: looking for participants now, as of the sponsor's last update on 30 Jan 2026. 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?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Jun Guo, M.D. (86-29-8477 8844, guojun_81@163.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Change from baseline in annual relapse rate (ARR) at last follow-up visit (time frame: baseline, 12 months). 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 30 Jan 2026.