Ofatumumab in AQP4-IgG Seropositive NMOSD

January 28, 2026 updated by: Jun Guo, MD, Tang-Du Hospital

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.

Study Overview

Status

Recruiting

Conditions

Intervention / Treatment

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 Type

Interventional

Enrollment (Estimated)

5

Phase

  • Phase 2
  • Phase 1

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Contact

Study Contact Backup

Study Locations

  • China
    • Shaanxi
      • Xi'an, Shaanxi, China
        • Recruiting
        • Tangdu Hospital
        • Principal Investigator:
          • Jun Guo, M.D.
        • Contact:
        • Contact:

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

14 years and older (Adult, Older Adult)

Accepts Healthy Volunteers

No

Description

Inclusion Criteria:

  • 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.

Exclusion Criteria:

  • 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.
  • 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.

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: N/A
  • Interventional Model: Single Group Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Experimental: Ofatumumab
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.
Drug: Ofatumumab

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.

Other Names:
  • Arzerra

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
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.
baseline, 12 months

Secondary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Change from baseline in Expanded Disability Status Scale (EDSS) score
Time Frame: baseline, 3 months, 6 months, 9 months, 12 months
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.
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
MRI is conducted to measure the lesion burden on T2-weighted images.
baseline, 6 months, 12 months
Change from baseline in optic coherence tomography (OCT) measures
Time Frame: baseline, 6 months, 12 months
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.
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
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.
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
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.
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
Blood routine test, liver and kidney function, immunoglobulins, complement, and serum AQP4-IgG titer.
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
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.
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
Ofatumumab-related adverse events (AEs) are evaluated and the rate of AEs is recorded.
1 week, 2 weeks, 1 month, 3 months, 6 months, 9 months, 12 months

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Tang-Du Hospital

Collaborators

Henan Provincial People's Hospital

Investigators

  • Principal Investigator: Jun Guo, M.D., Tang-Du Hospital

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

June 28, 2022

Primary Completion (Estimated)

April 30, 2026

Study Completion (Estimated)

July 31, 2026

Study Registration Dates

First Submitted

August 16, 2022

First Submitted That Met QC Criteria

August 16, 2022

First Posted (Actual)

August 17, 2022

Study Record Updates

Last Update Posted (Actual)

January 30, 2026

Last Update Submitted That Met QC Criteria

January 28, 2026

Last Verified

January 1, 2026

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • K202206-13

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

No

Studies a U.S. FDA-regulated device product

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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