Clinical Study of B001 Injection in Subjects With Neuromyelitis Optic Spectrum Disorder (NMOSD)

Official title

A Phase Ib Clinical Study to Evaluate the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics and Preliminary Efficacy of B001 in Subjects With Aquaporin-4 Antibody (AQP4-IgG) Positive Neuromyelitis Optic Spectrum Disorder (NMOSD)

Overview

The objectives of this phase Ib study are to evaluate the efficacy, safety, pharmacokinetics, pharmacodynamics and immunogenic profiles of B001 in subjects with aquaporin-4 antibody (AQP4-IgG) positive NMOSD.

Interventions

• Drug B001 injection
  B001 injection 50mg/5mL Intravenous solution
• Biological Placebo
  Placebo 5mL Intravenous solution

Primary outcome measures

• Dose-limiting toxicity (DLT) [Time frame: Up to 18 days.]
• Evaluate incidence of treatment-emergent adverse events [Safety and Tolerability]. [Time frame: Up to 1 year]

Secondary outcome measures (12)

• Maximum serum concentration (Cmax) of B001. [Time frame: Through study completion, up to 2 years]
• Time of maximum serum concentration (Tmax) of B001. [Time frame: Through study completion, up to 2 years]
• Area under the serum concentration-time curve (AUC) of the Dosing Interval (0-14D) of B001. [Time frame: Through study completion, up to 2 years]
• Area under the serum concentration-time curve (AUC) of the Dosing Interval (0-Last) of B001. [Time frame: Through study completion, up to 2 years]
• Area under the serum concentration-time curve (AUC) of the Dosing Interval (0-infinity) of B001. [Time frame: Through study completion, up to 2 years]
• Accumulation ratio of maximum serum concentration (Rac_Cmax) of B001. [Time frame: Through study completion, up to 2 years]
• Accumulation ratio of area under the serum concentration-time curve (Rac_AUC) of the Dosing Interval (0-14D) of B001. [Time frame: Through study completion, up to 2 years]
• Terminal rate constant(λz) of B001. [Time frame: Through study completion, up to 2 years]
• Half-life (t1/2) of B001. [Time frame: Through study completion, up to 2 years]
• Total clearance(CL) of B001. [Time frame: Through study completion, up to 2 years]
• Volume of distribution(Vz) of B001. [Time frame: Through study completion, up to 2 years]
• Percentage of area under the serum concentration-time curve (AUC 0-infinity) obtained by extrapolation (%AUCex) of B001. [Time frame: Through study completion, up to 2 years]

Eligibility criteria

Inclusion criteria

• NMOSD as defined by either of the following 2015 criteria with anti-AQP4 antibody (Ab) seropositive status at screening
• Clinical evidence of at least 1 documented relapse in last 12 months prior to screening
• Expanded Disability Status Scale (EDSS) score from 0 to 7.5 inclusive at screening
• Age 18 to 70 years, inclusive at the time of informed consent

Exclusion criteria

• Any previous treatment with anti-CD20, eculizumab, anti-BLyS monoclonal antibody (e.g., belimumab), any other treatment for prevention of multiple sclerosis (MS) relapse (e.g., interferon, natalizumab, glatiramer acetate, fingolimod, teriflunomide or dimethyl fumarate) within 6 months prior to baseline.
• Received immunosuppression such as azathioprine, mycophenolate mofetil, methotrexate, cyclophosphamide, tacrolimus, mitoxantrone, cyclosporine A, etc, and rug therapy, biological agents such as satralizumab, tocilizumab, eculizumab, etc, 3 months prior to the first administration.
• Evidence of serious uncontrolled concomitant diseases that may preclude participant participation, as described; Other nervous system disease, cardiovascular disease, hematologic/hematopoiesis disease, respiratory disease, muscular disease, endocrine disease, renal/urologic disease, digestive system disease, congenital or acquired severe immunodeficiency.
• Known active infection within 3 months prior to baseline
• Pregnancy or lactation.
• History of severe allergic reaction to a biologic agent
• Evidence of chronic active hepatitis B or C
• Evidence of active tuberculosis
• Following laboratory abnormalities at screening\*:

• White blood cells (WBC) <4.0 x10\^3/microliter (μL)
• Absolute neutrophil count (ANC)
• Absolute lymphocyte count <0.5 x10\^3/μL
• Platelet count <80 x 10\^9/ L
• Aspartate aminotransferase (AST) or alanine aminotransferase
• History of drug or alcohol abuse within 6 months prior to baseline
• Receipt of any live or live attenuated vaccine within 4 weeks prior to baseline
• Uncontrolled systemic diseases, including hypertension that cannot be effectively controlled after treatment (systolic blood pressure ≥150 mmHg and/or diastolic blood pressure ≥100 mmHg), diabetes, gastrointestinal diseases, etc.; or the investigator believes that there is anything inappropriate reasons for selection.

Criteria are shown verbatim from the registry (in English). Final eligibility is always assessed by the study center.

Healthy volunteers: No

Study design

Allocation

Randomized

Model

Parallel assignment

Masking

Double blind

Primary purpose

Treatment

Study locations

China · 4 centers

• Beijing Tiantan Hospital Capital Medical University — Beijing
• First Hospital of Shanxi Medical University — Taiyuan
• Tangdu hospital,fourth military medical university — Xi’an
• Tianjin Medical University General Hospital — Tianjin

Publications

• Jia D, Wang H, Jiang W, Shen Y, Guo J, Zhao D, Zhang M, Meng H, Xue H, Song Y, Yao Q, Xie N, Zhang C. A novel recombinant anti-cluster of differentiation 20 humanized monoclonal antibody (B001) for the treatment of neuromyelitis optica spectrum disorder: a phase 1, multicenter randomized, double-blind trial. Front Immunol. 2026 Apr 16;17:1676908. doi: 10.3389/fimmu.2026.1676908. eCollection 2026. PMID 42079612

Identifiers

NCT: NCT05145361 · B001-103

Primary sources (government registries)

• ClinicalTrials.gov (US NIH)

View this study on ClinicalTrials.gov ↗