A Phase I Clinical Trial of BAT4406F Injection in Patients With Neuromyelitis Optica Spectrum Disorders

A Phase I Clinical Trial of BAT4406F Injection on the Safety, Tolerability, and Pharmacokinetics in Patients With Neuromyelitis Optica Spectrum Disorders

This study is a phase I clinical study of the safety, tolerability, and pharmacokinetics of BAT4406F injection in patients with neuromyelitis optica spectrum disorders.

Study Overview

• Status: Completed
• Conditions: Neuromyelitis Optica Spectrum Disorders
• Intervention / Treatment: Drug: BAT4406F

Detailed Description

This is a Phase 1, open-label, dose-escalation study in NMOSD patients in which subjects will receive BAT4406F injection via intravenous infusion. A 3 + 3 design will be utilized to define a maximum tolerated dose (MTD). The overall objective is to assess the safety, tolerability, and pharmacokinetics of BAT4406F injection in NMOSD patients.

• Study Type: Interventional
• Enrollment (Actual): 15
• Phase: Phase 1

Contacts and Locations

Study Locations

• China
  • Shanghai
    • Shanghai, Shanghai, China
      • Huashan Hospital Affiliated to Fudan University

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 14 years to 61 years (Adult, Older Adult)
• Accepts Healthy Volunteers: No

Description

Inclusion Criteria:

• Compliance with the NMOSD diagnostic criteria developed by the 2015 International NMO Diagnostic Team (IPND);
• 18-65 years old , male or female;
• At least 2 relapses occurred within 2 years before screening, or at least 1 relapse within 1 year before screening;
• Discontinue the immunosuppressive agents such as azathioprine within 28 days before the baseline;
• EDSS score ≤ 6;
• Men and women with fertility must agree to use effective methods of contraception during treatment and within 12 months of treatment completion;
• Agree to participate in the trial and sign the informed consent in writing.

Exclusion Criteria:

• Any monoclonal antibody treatment was used within 6 months prior to dosing;
• Having been treated with anti-CD20 monoclonal antibody;
• Live vaccine received within 4 weeks before screening;
• Having participated in another clinical study within 1 month or 5 half-lives of the drug prior to the baseline (whichever is longer);
• A history of allergies to monoclonal antibodies; severe allergic reaction to certain foods or drugs;
• Abnormal liver function, kidney function and bone marrow reserve;
• HIV-positive history or HIV-positive at screening; hepatitis B and/or hepatitis C history or hepatitis B surface antigen-positive at screening; or hepatitis C virus (HCV) antibody positive; treponema pallidum antibody positive when enrolled;
• History of infections that investigators have identified as unsuitable for testing;
• Patients with a clear history of heart disease ;
• Have a history of mental disorders;
• Pregnant or lactating women, and female subjects who have a positive pregnancy test at screening;
• None of the investigators or their relatives participating in the study could be enrolled.

Study Plan

How is the study designed?

Design Details

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

Number of Arms

1

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: BAT4406F	Drug: BAT4406F; Open-label dose escalation starting from 20mg. Route of administration: intravenous infusion.

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Dose-limiting toxicity（DLT）	Safety and tolerability endpoint	4weeks
Maximum tolerated dosed (MTD)	Safety and tolerability endpoint	up to 6 months
Area under the curve (AUC)	Pharmacokinetic endpoint	up to 6 months
Maximum serum drug concentration (Cmax)	Pharmacokinetic endpoint	up to 6 months
Half-life period(t1/2)	Pharmacokinetic endpoint	up to 6 months
Maximum serum drug time (Tmax)	Pharmacokinetic endpoint	up to 6 months
CD19+ B lymphocyte ratio	Pharmacodynamics endpoint	up to 6 months

Collaborators and Investigators

• Sponsor: Bio-Thera Solutions
• Investigators: Principal Investigator: Xiangjun Chen, Huashan Hospital; Principal Investigator: Jing Zhang, Huashan Hospital

Study record dates

Study Major Dates

• Study Start (Actual): September 1, 2020
• Primary Completion (Actual): March 13, 2023
• Study Completion (Actual): December 25, 2023

Study Registration Dates

• First Submitted: September 26, 2019
• First Submitted That Met QC Criteria: October 29, 2019
• First Posted (Actual): October 31, 2019

Study Record Updates

• Last Update Posted (Actual): July 10, 2025
• Last Update Submitted That Met QC Criteria: July 7, 2025
• Last Verified: March 1, 2023

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Nervous System Diseases; Autoimmune Diseases; Immune System Diseases; Eye Diseases; Demyelinating Autoimmune Diseases, CNS; Autoimmune Diseases of the Nervous System; Demyelinating Diseases; Myelitis, Transverse; Optic Neuritis; Optic Nerve Diseases; Cranial Nerve Diseases; Neuromyelitis Optica
• Other Study ID Numbers: BAT-4406F-001-CR

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No
• product manufactured in and exported from the U.S.: No