A Clinical Study of B001 Injection in the Treatment of Neuromyelitis Optica Spectrum Disorders(NMOSD)

A Multicenter, Randomized, Double-blind, Placebo-controlled Phase II/III Clinical Study to Evaluate the Efficacy and Safety of B001 Injection in Aquaporin-4 Antibody Positive Patients With Neuromyelitis Optica Spectrum Disorder

To evaluate the efficacy and safety of B001 injection in aquaporin-4 antibody positive patients with neuromyelitis optica spectrum disorder.

Study Overview

• Status: Not yet recruiting
• Conditions: Neuromyelitis Optica Spectrum Disorders
• Intervention / Treatment: Drug: B001; Other: Placebo

• Study Type: Interventional
• Enrollment (Estimated): 132
• Phase: Phase 2; Phase 3

Contacts and Locations

• Study Contact: Name: FuDong Shi; Phone Number: 0086-010-59978555; Email: ttyyirb@163.com

Study Locations

• China
  • Beijing, China
    • Beijing Tiantan Hospital，Capital Medical University
    • Contact: Fudong Shi
  • Beijing, China
    • Peking University First Hospital
    • Contact: Feng Gao
  • Beijing, China
    • Beijing Anzhen Hospital，Capital Medical University
    • Contact: Guangzhi Liu
  • Changsha, China
    • The second Xiangya Hospital of Central South University
    • Contact: Xiaomei Wu
  • Fuzhou, China
    • The First Affiliated Hospital of Fujian Medical University
    • Contact: Aiyu Lin
  • Guangzhou, China
    • Guangzhou First People's Hospital
    • Contact: Honghao Wang
  • Guangzhou, China
    • The First Affiliated Hospital, Sun Yat-sen University
    • Contact: Jinsheng Zeng
  • Hangzhou, China
    • The Second Affiliated Hospital of Zhejiang University School of Medicine
    • Contact: Zhiying Wu
  • Jinan, China
    • The First Affiliated Hospital Of Shandong First Medical University
    • Contact: Ruisheng Duan
  • Kunming, China
    • First People's Hospital of Yunnan Province
    • Contact: Qiang Meng
  • Shenyang, China
    • The First Hospital of China Medical University
    • Contact: Zhe Wu
  • Shijiazhuang, China
    • The Second Hospital of Hebei Medical University
    • Contact: Bin Li
  • Taiyuan, China
    • First Hospital of Shanxi Medical University
    • Contact: Meini Zhang
  • Tianjin, China
    • Tianjin Medical University General Hospital
    • Contact: Li Yang
  • Wenzhou, China
    • The First Affiliated Hospital of Wenzhou Medical University
    • Contact: Xu Zhang
  • Wuhan, China
    • Renmin Hospital of Wuhan University
    • Contact: Ting Chen
  • Wuhan, China
    • Tongji Hospital
    • Contact: Daishi Tian
  • Xi'an, China
    • The Second Affiliated Hospital of the Chinese People's Liberation Army Air Force Medical University
    • Contact: Jun Guo
  • Yantai, China
    • Yantai Mountain Hospital in Yantai City
    • Contact: Jianhua Tang
  • Zhengzhou, China
    • Henan Provincial People's Hospital
    • Contact: Yue Huang
  • Zhengzhou, China
    • The First Affiliated Hospital of Zhengzhou University
    • Contact: Yuming Xu

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Adult; Older Adult
• Accepts Healthy Volunteers: No

Description

Inclusion Criteria:

1. Confirmed NMOSD patients;
2. Experienced at least 1 recurrence of NMOSD within 1 year or at least 2 recurrence within 2 years prior to screening;
3. The blood pregnancy tests were negative within 7 days before the first dose in fertile female subjects. The fertile subjects and the male subjects whose partner are fertile woman agree to use reliable contraception during the study period and within 6 months after the study drug discontinuation;
4. Volunteer to participate in clinical research; Fully understand and know the study and sign the informed consent; Willing to follow and able to complete all test procedures.

Exclusion Criteria:

1. Subjects with severe NMOSD were judged by the researcher to be unfit to participate in this study;
2. Subjects with chronic active immune system diseases undergoing systematic treatment;
3. Received anti-CD20 or other cell depletion therapy within 6 months before first dose;
4. Received the prescribed drug treatment at the prescribed time before first dose;
5. Subjects who have participated in any drug clinical trials within 28 days prior to the first dose;
6. Received hematopoietic stem cell transplantation、 lymphatic irradiation before first dose;
7. Pregnant or lactating women; Subjects with birth plans during the trial;
8. Subjects who had undergone major surgery within 2 months prior to screening or were scheduled to undergo major surgery during the trial;
9. Subjects with severe, progressive, or uncontrolled disease who have been assessed by the investigator to be at increased risk of participating in the study;
10. A history of gastrointestinal perforation and/or fistula within 6 months prior to screening;
11. Subjects who received a live or attenuated vaccine within 4 weeks prior to initial administration, or who plan to receive vaccination during the study period;
12. Subjects with severe or persistent infection currently or within the 3 months prior to screening;
13. Subjects with positive gamma interferon release test;
14. Subjects who currently have active hepatitis or have a history of severe liver disease;
15. Uncontrolled systemic disease, or any other reason the investigator deems inappropriate for inclusion;
16. Abnormal laboratory test results;
17. Subjects with a history of alcohol or drug abuse in the 6 months prior to screeing, or who are abusing;
18. Subjects with symptoms of severe mental illness who are clinically uncooperative;
19. Subjects who were unable to undergo magnetic resonance imaging during the study and who had other conditions that the investigator considered inappropriate to enroll.

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: Randomized
• Interventional Model: Parallel Assignment
• Masking: Quadruple

Number of Arms

2

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Placebo Comparator: Placebo	Other: Placebo; The subjects will receive IV dose of placebo matched to B001 on Day 1 and Day 15 of the RCP
Experimental: B001 injection	Drug: B001; The subjects will receive IV dose of B001 on Day 1 and Day 15 of RCP

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Time to first NMOSD Attack During RCP	The NMOSD attack is defined as the presence of new or worsening symptom(s) related to NMOSD.	Approximately 48 weeks

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Change in Expanded Disability Status Scale (EDSS) Score	EDSS and its associated functional system (FS) score provide a system for quantifying disability and monitoring changes in the level of disability over time. EDSS consists of 7 FS (visual FS, brainstem FS, pyramidal FS, cerebellar FS, sensory FS, bowel and bladder FS, and cerebral FS) which are used to derive EDSS score ranging from 0 (normal neurological exam) to 10 (death from MS).	Approximately 48 weeks
Annualized relapse rate (ARR)	Annualized attack rate is defined as total number of attacks divided by total person years.	Approximately 3 years
Incidence of Treatment-Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs)	An adverse event (AE) is any untoward medical occurrence in a participant who received study drug without regard to possibility of causal relationship. Serious adverse event is any AE that resulted in death, life threatening, inpatient hospitalization or prolongation of existing hospitalization, persistent or significant disability or incapacity, is a congenital anomaly/birth defect in offspring of a study participant, is an important medical event that may jeopardize the participant or may require medical intervention. TEAEs are defined as events present at baseline that worsened in intensity after administration of study drug or events absent at baseline that emerged after administration of study drug.	Approximately 3 years
Change in Vision Acuity/ Low contrast visual acuity (VA/LCVA)	Change in VA/LCVA	Approximately 48 weeks
Change in Opticospinal Impairment Scale (OSIS)	Change in OSIS	Approximately 48 weeks

Collaborators and Investigators

• Sponsor: Shanghai Jiaolian Drug Research and Development Co., Ltd
• Collaborators: Shanghai Pharmaceuticals Holding Co., Ltd

Study record dates

Study Major Dates

• Study Start (Estimated): June 20, 2024
• Primary Completion (Estimated): October 31, 2027
• Study Completion (Estimated): February 28, 2029

Study Registration Dates

• First Submitted: May 9, 2024
• First Submitted That Met QC Criteria: May 13, 2024
• First Posted (Actual): May 14, 2024

Study Record Updates

• Last Update Posted (Actual): May 16, 2024
• Last Update Submitted That Met QC Criteria: May 14, 2024
• Last Verified: May 1, 2024

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Nervous System Diseases; Immune System Diseases; Demyelinating Autoimmune Diseases, CNS; Autoimmune Diseases of the Nervous System; Demyelinating Diseases; Autoimmune Diseases; Eye Diseases; Optic Nerve Diseases; Cranial Nerve Diseases; Myelitis, Transverse; Optic Neuritis; Neuromyelitis Optica
• Other Study ID Numbers: SPH-B001-301

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No