An Open Label Study of the Effects and Safety of Zanubrutinib in NMOSDs Adult Patients

NCT05356858 | Terminated Phase 2

• 1 other identifier: BGB-3111-2005-IIT
• Study Type: interventional
• Target: 6
• Locations: 1 country
• Sites: 1

Brief Summary

This is an open-label study, to evaluate the efficacy and safety of a BTK inhibitor zanubrutinib in participants with NMOSDs.

Conditions

Neuromyelitis Optica

Keywords

NMOSD; BTK inhibitor,; zanubrutinib

Outcome Measures

Primary Outcomes (1)

• Relapse-free rate at week 48

  Proportion of subjects who are relapse-free at week 48

  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

  up to week12,24,36 and 48

• Changes in Serum AQP4-IgG titer at week 12, 24,36 and 48 from baseline

  up to week12,24,36 and 48

• Changes in Peripheral blood lymphocyte subset at week 12, 24,36 and 48 from baseline

  up to week12,24,36 and 48

• Plasma concentraion of zanubrutinib

  up to week 48

• Number of participants with all grade Adverse Events, Serious Adverse Events and who discontinued study therapy due to AEs

  up to week 48

Other Outcomes (2)

• Concentraion of zanubrutinib in Cerebrospinal fluid at week 12

  up to week 12

• BTK accupancy of peripheral blood mononuclear cells

  up to week 48

Study Arms (1)

zanubrutinib

EXPERIMENTAL

zanubrutinib orally, 80mg bid for 1 year

Drug: zanubrutinib

Interventions

zanubrutinib DRUG

zanubrutinib orally, 80mg bid for 1 year

zanubrutinib

Eligibility Criteria

• Age: 18 Years - 75 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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:
• 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.

You may not qualify if:

• 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).
• 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.

Sponsors & Collaborators

• Xuanwu Hospital, Beijing: lead

Study Sites (1)

XuanWu Hospital

Beijing, Beijing Municipality, 100053, China

Location

MeSH Terms

Conditions

Neuromyelitis Optica

Interventions

zanubrutinib

Condition Hierarchy (Ancestors)

Myelitis, Transverse; Demyelinating Autoimmune Diseases, CNS; Autoimmune Diseases of the Nervous System; Nervous System Diseases; Optic Neuritis; Optic Nerve Diseases; Cranial Nerve Diseases; Demyelinating Diseases; Eye Diseases; Autoimmune Diseases; Immune System Diseases

Study Officials

• Junwei Hao, MD

  Xuanwu Hospital, Beijing

  STUDY CHAIR

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: April 26, 2022
• First Posted: May 2, 2022
• Study Start: May 7, 2022
• Primary Completion: February 24, 2024
• Study Completion: February 24, 2024
• Last Updated: April 27, 2026

Record last verified: 2026-04

Data Sharing

• IPD Sharing: Will not share

Locations

CN (1)