Neuromyelitis OpticaPhase 2NCT05356858
An Open Label Study of the Effects and Safety of Zanubrutinib in NMOSDs Adult Patients
A Phase 2 study testing zanubrutinib in people with Neuromyelitis Optica, run by Xuanwu Hospital, Beijing at 1 site in China.
Trial summary
This is an open-label study, to evaluate the efficacy and safety of a BTK inhibitor zanubrutinib in participants with NMOSDs. Read more about this study
- Who is it for?
- Ages 18 to 75 years
- Conditions the study is for
- Neuromyelitis Optica
- What is being tested?
- zanubrutinib
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 6 participants
- Where
- 1 siteChina
- Study dates
- 7 May 2022to 24 Feb 2024
- Phase
- Phase 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 to 75 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Patients must meet the NMOSD diagnostic criteria set by the international NMO Diagnostic Group (IPND) in 2015.
- Serum AQP4-IgG positive.
- Clinical evidence of at least 2 documented relapse (including first attack) in the last 2 years, with at least 1 relapse within 12 months prior to screening.
- Extended Disability Status Scale (EDSS) score ≤7.5 at screening.
- Age 18 to 75 years inclusive, weight at least 35 kg at the time of informed consent.
- If the patients were using the following baseline treatment for relapse prevention, they must be treated at a steady dose for at least 4 weeks prior to enrollment:
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- Azathioprine, metecophenol ester and other immunosuppressive agents
- Oral corticosteroid (≦30mg/ day prednisone tablet or equivalent dose of other hormones)
- (patients or their legal representatives) can provide written informed consent indicating that they understand and agree to comply with the requirements of the study protocol.
Who cannot join
- Continuous treatment with strong or moderate CYP3A inhibitors or inducers is required during the study period. Patients were excluded if they had taken a potent or moderate CYP3A inhibitor or inducer within 7 days prior to administration of the study drug (or had stopped taking these drugs for less than 5 half-lives).
- Previously treated with BTK inhibitors (e.g., ibrutinib).
- Allergic to the study drug or any of the ingredient.
- Desease relaps (including first episode) within the previous 30 days.
- Pregnancy or lactation.
- Previous or current malignancy, except locally recurrent cancers that have received radical treatment (e.g. excised basal or squamous cell skin cancer, cervical or breast cancer in situ).
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- Currently central nervous system (CNS) disease that may affect the evaluation of NMOSD.
- Serious and uncontrolled conditions considered by the investigator that could affect safety, compliance and endpoint evaluation, or need for use of a drug not permitted in the protocol.
- Disease that could affected drug absorption, distribution, metabolism, and excretion determined by the investigator.
- Any major clinical infection lead to hospitalization or parenteral antibiotic treatment within 1 month prior to screening; Or other infections that may be aggravated due to the study determined by the investigator.
- Active, latent or undertreated mycobacterium tuberculosis (TB) infection
- Known primary immunodeficiency or underlying disease such as human immunodeficiency virus (HIV) infection.
- Hepatitis B or C virus infection by serological test.
- Received B-cell targeted therapy (e.g. Rituximab) within 6 months prior to the initial administration of the study drug.
- Received biologics such as tozizumab within 12 weeks prior to initial administration of the study drug.
- Received live attenuated vaccine during the screening and study periods, or any live virus vaccine within 8 weeks prior to initial administration.
- Abnormal and clinically significant in ECG examination during screening.
- Uncontrolled hypertension (SBP>160 mmHg or DBP ≥ 95 mmHg)
- Grade 3 or 4 heart Failure, (NYHA scale).
- Severe liver insufficiency (Child-pugh C).
- Aspartate aminotransferase (AST)>3 times the upper limit of normal (ULN) and/or alanine aminotransferase (ALT)>3ULN and/or bilirubin >2ULN.
- Estimated creatinine clearance <30 mL/min or requiring dialysis.
- Inability to receive MRI scans
- A history of clinically significant CNS trauma
- Received experimental drug or other experimental treatment within 4 weeks prior to screening or during 5 pharmacokinetic half-lives or duration of biological effects, whichever is longer.
- Participate in another clinical study.
- Accept any of the following:
- BCG vaccination within 1 year prior to screening.
- Prior bone marrow transplant, hematopoietic stem cell transplant, or systemic radiation therapy.
- Received intravenous gamma globulin within 30 days prior to screening.
- Plasmapheresis or leukocyte separation within 90 days prior to screening
- Abnormal white blood cell count, neutrophil count, lymphocyte count, or platelet count during the screening and were considered unsuitable for study by investigator
- Inability to swallow capsules or medical conditions that significantly affect gastrointestinal function
- A history of severe hemorrhagic disorders such as hemophilia A, hemophilia B, von willebrand disease, or a history of spontaneous bleeding requiring blood transfusion or other medical intervention.
- History of stroke or intracranial hemorrhage within 6 months prior to screening
- Current alcohol, drug or chemical abuse, or history of such abuse within 1 year prior to screening.
- Anticoagulants or a combination of anticoagulants and antiplatelet agents is ongoing or planned.
- Any other circumstances in which the investigator or sponsor considers the patient unsuitable for study participation.
Trial locations and contacts
1 site in 1 country.
About this study
Official title: An Open Label Trial Evaluating the Efficacy and Safety of Bruton's Tyrosine Kinase (BTK) Inhibitor Zanubrutinib in Adult Patients With Neuromyelitis Optical Spectrum Disorders (NMOSDs)
This is an open-label study, to evaluate the efficacy and safety of a BTK inhibitor zanubrutinib in participants with NMOSDs.
Read the detailed description
This is an open, single-center clinical study to evaluate the efficacy and safety of Zanubrutinib in the treatment of recurrent neuromyelitis optica spectrum disease. Patients were required to be diagnosed with neuromyelitis optic spectrum disease according to the NMOSD diagnostic criteria established by the international NMO Diagnostic Group (IPND) in 2015, and to have had at least two relapses (including first episode) within two years while at least one relapse occurring within the 12 months prior to screening. The AQP4 antibody must be positive during screening.
- Conditions
- Neuromyelitis Optica
- Being tested
- zanubrutinib
- Keywords
- NMOSD, BTK inhibitor,, zanubrutinib
- Medical subject headings
- Neuromyelitis Optica
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
zanubrutinibDrug
zanubrutinib orally, 80mg bid for 1 year
| Study group | Role | What they receive |
|---|---|---|
| zanubrutinib | Experimental | zanubrutinib orally, 80mg bid for 1 year Receives: Drug: zanubrutinib |
What is being measured
The primary outcome is the main question the study is designed to answer.
Relapse-free rate at week 48
Proportion of subjects who are relapse-free at week 48
Measured: up to week 48
Secondary outcomes (5)
Changes in the expanded disability status scale (EDSS) score from baseline at week 12,24,36 and 48
EDSS is an ordered scale with values ranging from 0 points (normal neurological examination) to 10 points (death) in increments of 0.5 points. Higher scores indicate increased disability, and negative changes from baseline indicate improvement
Measured: up to week12,24,36 and 48
Changes in Serum AQP4-IgG titer at week 12, 24,36 and 48 from baseline
Changes in Serum AQP4-IgG titer at week 12, 24,36 and 48 from baseline
Measured: up to week12,24,36 and 48
Changes in Peripheral blood lymphocyte subset at week 12, 24,36 and 48 from baseline
Changes in Peripheral blood lymphocyte subset at week 12, 24,36 and 48 from baseline
Measured: up to week12,24,36 and 48
Plasma concentraion of zanubrutinib
Concentraion of zanubrutinib in peripheral blood plasma
Measured: up to week 48
Number of participants with all grade Adverse Events, Serious Adverse Events and who discontinued study therapy due to AEs
Evaluate Number of participants with all grade Adverse Events and Serious Adverse Events determined by NCI-CTCAE V5.0，and tolerance base on percentage of patients who discontinued study therapy due to AEs
Measured: up to week 48
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted2 May 2022
- Study start7 May 2022
- Primary completion24 Feb 2024
- Study completion24 Feb 2024
Why it stopped: Difficult in participant recruiting
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Neuromyelitis OpticaStudies of the same condition, open and under way.
- More trials run by Xuanwu Hospital, BeijingThe 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 2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 6 participants (actual)
Sponsor and investigators
- Lead sponsor
- Xuanwu Hospital, Beijing
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- Junwei Hao, MD, Study chair, Xuanwu Hospital, Beijing
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05356858
- Sponsor's protocol ID
- BGB-3111-2005-IIT
- Status last verified
- Apr 2026
- First submitted
- 26 Apr 2022
- Last updated
- 27 Apr 2026
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 27 Apr 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- An Open Label Trial Evaluating the Efficacy and Safety of Bruton's Tyrosine Kinase (BTK) Inhibitor Zanubrutinib in Adult Patients With Neuromyelitis Optical Spectrum Disorders (NMOSDs)
- ClinicalTrials.gov ID
- NCT05356858
- Acronym / sponsor ID
- BGB-3111-2005-IIT
- Conditions
- Neuromyelitis Optica
- Keywords
- NMOSD, BTK inhibitor,, zanubrutinib
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- TerminatedReason stopped: Difficult in participant recruiting
- Enrolment
- 6 participants (actual)
- Lead sponsor
- Xuanwu Hospital, BeijingUniversity, hospital or other
- Study start
- 7 May 2022 (actual)
- Primary completion
- 24 Feb 2024 (actual)
- Study completion
- 24 Feb 2024 (actual)
- First posted
- 2 May 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 study, to evaluate the efficacy and safety of a BTK inhibitor zanubrutinib in participants with NMOSDs.
Detailed description
This is an open, single-center clinical study to evaluate the efficacy and safety of Zanubrutinib in the treatment of recurrent neuromyelitis optica spectrum disease. Patients were required to be diagnosed with neuromyelitis optic spectrum disease according to the NMOSD diagnostic criteria established by the international NMO Diagnostic Group (IPND) in 2015, and to have had at least two relapses (including first episode) within two years while at least one relapse occurring within the 12 months prior to screening. The AQP4 antibody must be positive during screening.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| zanubrutinib | Experimental | Drug: zanubrutinibzanubrutinib orally, 80mg bid for 1 year |
Who may qualify
- Ages
- 18 to 75 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (9 listed)
- Patients must meet the NMOSD diagnostic criteria set by the international NMO Diagnostic Group (IPND) in 2015.
- Serum AQP4-IgG positive.
- Clinical evidence of at least 2 documented relapse (including first attack) in the last 2 years, with at least 1 relapse within 12 months prior to screening.
- Extended Disability Status Scale (EDSS) score ≤7.5 at screening.
- Age 18 to 75 years inclusive, weight at least 35 kg at the time of informed consent.
Key exclusion criteria (38 listed)
- Continuous treatment with strong or moderate CYP3A inhibitors or inducers is required during the study period. Patients were excluded if they had taken a potent or moderate CYP3A inhibitor or inducer within 7 days prior to administration of the study drug (or had stopped taking these drugs for less than 5 half-lives).
- Previously treated with BTK inhibitors (e.g., ibrutinib).
- Allergic to the study drug or any of the ingredient.
- Desease relaps (including first episode) within the previous 30 days.
- Pregnancy or lactation.
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 |
|---|---|---|
| zanubrutinibzanubrutinib orally, 80mg bid for 1 year | Drug | zanubrutinib |
Primary outcome
Relapse-free rate at week 48
Time frame: up to week 48
Proportion of subjects who are relapse-free at week 48
5 secondary outcomes
Changes in the expanded disability status scale (EDSS) score from baseline at week 12,24,36 and 48
Time frame: up to week12,24,36 and 48
Changes in Serum AQP4-IgG titer at week 12, 24,36 and 48 from baseline
Time frame: up to week12,24,36 and 48
Changes in Peripheral blood lymphocyte subset at week 12, 24,36 and 48 from baseline
Time frame: up to week12,24,36 and 48
Plasma concentraion of zanubrutinib
Time frame: up to week 48
Number of participants with all grade Adverse Events, Serious Adverse Events and who discontinued study therapy due to AEs
Time frame: up to week 48
Results and safety
The study was stopped early (reason given: Difficult in participant recruiting). Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 27 Apr 2026.
- Individual participant dataSharing planned: No
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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.
Junwei Hao, MD study chair
Xuanwu Hospital, Beijing
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing zanubrutinib in people with Neuromyelitis Optica, run by Xuanwu Hospital, Beijing 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 9 inclusion and 38 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: stopped early, as of the sponsor's last update on 27 Apr 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 phase mean?
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 was stopped early (reason given: Difficult in participant recruiting). Results are not available in the source record.
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?
Relapse-free rate at week 48 (time frame: up to week 48). 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 27 Apr 2026.