Evaluating B001 Injection for Neuromyelitis Optic Spectrum Disorder

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)

This study is testing a new injection called B001 to see if it can help people with neuromyelitis optic spectrum disorder feel better and reduce their symptoms.

Quick facts

• Phase: Early Phase 1
• Study type: Interventional
• Enrollment: 45 (estimated)
• Ages: 18 Years and up
• Sex: All
• Sponsor: Shanghai Pharmaceuticals Holding Co., Ltd (Industry-sponsored)
• Drugs / interventions: eculizumab, belimumab, natalizumab, satralizumab, tocilizumab, methotrexate, cyclophosphamide
• Locations: 4 sites (Beijing, Beijing Municipality and 3 other locations)
• Trial ID: NCT05145361 on ClinicalTrials.gov

What this trial studies

This phase Ib clinical trial aims to assess the efficacy, safety, pharmacokinetics, pharmacodynamics, and immunogenicity of B001 injection in patients with aquaporin-4 antibody positive neuromyelitis optic spectrum disorder (NMOSD). Participants will receive either the B001 injection or a placebo, and their responses will be monitored over the course of the study. The trial focuses on individuals who have experienced at least one relapse in the past year and have specific disability scores. The goal is to determine how well B001 can manage symptoms and improve patient outcomes.

Who should consider this trial

Good fit: Ideal candidates for this study are adults aged 18 to 70 who have been diagnosed with NMOSD and are seropositive for anti-AQP4 antibodies.

Not a fit: Patients who have received certain immunosuppressive therapies or specific monoclonal antibodies within the last few months may not benefit from this study.

Why it matters

Potential benefit: If successful, this treatment could provide a new therapeutic option for patients suffering from NMOSD, potentially reducing relapses and improving quality of life.

How similar studies have performed: While this approach is relatively novel, similar studies targeting NMOSD have shown promise in the past, indicating potential for success.

Eligibility criteria

Inclusion Criteria:

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

Exclusion Criteria:

1. 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.
2. 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.
3. 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.
4. Known active infection within 3 months prior to baseline
5. Pregnancy or lactation.
6. History of severe allergic reaction to a biologic agent
7. Evidence of chronic active hepatitis B or C
8. Evidence of active tuberculosis
9. Following laboratory abnormalities at screening\*:

   1. White blood cells (WBC) \<4.0 x10\^3/microliter (μL)
   2. Absolute neutrophil count (ANC)
   3. Absolute lymphocyte count \<0.5 x10\^3/μL
   4. Platelet count \<80 x 10\^9/ L
   5. Aspartate aminotransferase (AST) or alanine aminotransferase
10. History of drug or alcohol abuse within 6 months prior to baseline
11. Receipt of any live or live attenuated vaccine within 4 weeks prior to baseline
12. 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.

Where this trial is running

Beijing, Beijing Municipality and 3 other locations

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

Study contacts

• Principal investigator: Fu-Dong Shi, MD,PhD — Tianjin Medical University General Hospital
• Study coordinator: Fu-Dong Shi, MD,PhD

Conditions NMO Spectrum Disorder