An Open-Label Multicenter Study to Evaluate the Pharmacokinetics, Pharmacodynamics, and Safety of Inebilizumab in Pediatric Subjects With Neuromyelitis Optica Spectrum Disorder

VIB0551.P2.S2.NMO

A Phase 2, open-label, multicenter study to evaluate the pharmacokinetics (PK), pharmacodynamics (PD), and safety of inebilizumab in eligible pediatric participants 2 to \< 18 years of age with recently active neuromyelitis optica spectrum disorder (NMOSD) who are seropositive for autoantibodies against aquaporin-4 (AQP4-immunoglobulin \[Ig\]G).

2022-08-25

2027-04-13

2027-04-13

Recruiting

Early phase I

15

Inclusion Criteria: * Male or female subjects age 2 to \< 18 years at the time of screening. * Positive serum anti-AQP4-IgG result at screening and diagnosed with NMOSD according to the criteria of Wingerchuk et al, 2015. * Documented history of one or more NMOSD acute relapses within the last year, or 2 or more NMOSD acute relapses within 2 years prior to screening. Exclusion Criteria: * Any condition that, in the opinion of the Investigator, would interfere with the evaluation or administration of the Investigational Product or interpretation of subject safety or study results * Concurrent/previous enrollment in another clinical study involving an investigational treatment within 4 weeks or 5 published half-lives of the investigational treatment, whichever is the longer, prior to Day 1 * Evidence of significant hepatic, renal, or metabolic dysfunction or significant hematological abnormality (one repeat test may be conducted to confirm results within the same screening period): * B-cell counts \< one-half of the lower limit of normal (LLN) for age according to the central laboratory * Receipt of the following at any time prior to Day 1: 1. Alemtuzumab 2. Total lymphoid irradiation 3. Bone marrow transplant 4. T-cell vaccination therapy * Receipt of rituximab or any experimental B-cell depleting agent within 6 months prior to screening unless B-cell counts have returned to ≥ one-half the LLN * Receipt of intravenous immunoglobulin (IVIG) within one month prior to Day 1 * Receipt of any of the following within 2 months prior to Day 1: 1. Cyclosporine 2. Methotrexate 3. Mitoxantrone 4. Cyclophosphamide 5. Tocilizumab 6. Satralizumab 7. Eculizumab * Receipt of natalizumab (Tysabri®) within 6 months prior to Day 1 * Severe drug allergic history or anaphylaxis to 2 or more food products or medicine (including known sensitivity to acetaminophen/paracetamol, diphenhydramine or equivalent antihistamine, and methylprednisolone or equivalent glucocorticoid) * Diagnosed with a concurrent autoimmune disease that is uncontrolled (unless approved by the medical monitor) * Recent receipt of live/attenuated vaccine or blood transfusion

{'studyType': 'INTERVENTIONAL', 'phases': ['PHASE2'], 'designInfo': {'allocation': 'NA', 'interventionModel': 'SINGLE_GROUP', 'primaryPurpose': 'TREATMENT', 'maskingInfo': {'masking': 'NONE'}}, 'enrollmentInfo': {'count': 15, 'type': 'ESTIMATED'}}

2024-05-01