Study of Inebilizumab in Pediatric Subjects With Neuromyelitis Optica Spectrum Disorder

Recruiting now · Phase 2

Conditions studied: Neuromyelitis Optica Spectrum Disorder

In brief

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).

Key facts

Study ID

NCT05549258

Run by

Amgen

People needed

15

Starts

2023-07-03

Expected to finish

2027-04-13

Last updated by the study team

2025-12-05

Who can join

Age: 2 and older, up to 17. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

• Male or female participants, minimum body weight of 15 kg, 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.

You may not qualify if…

• Any condition that, in the opinion of the Investigator, would interfere with the evaluation or administration of the Investigational Product or interpretation of participant 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:
• Alemtuzumab
• Total lymphoid irradiation
• Bone marrow transplant
• 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:
• Cyclosporine
• Methotrexate
• Mitoxantrone
• Cyclophosphamide
• Tocilizumab
• Satralizumab
• 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.
• Receipt of any of the following:
• Any live or attenuated vaccine within 4 weeks prior to Day 1 (administration of killed vaccines and nucleoside-modified mRNA-based vaccines is acceptable; the Sponsor recommends that Investigators ensure all participants are up to date on required vaccinations prior to study entry).

Where it is running

• UCSD Altman Clinical and Translational Research Institute Building — La Jolla, California, United States (enrolling)
• Loma Linda University Children's Hospital - PIN — Loma Linda, California, United States (enrolling)
• Massachusetts General Hospital — Boston, Massachusetts, United States (enrolling)
• University of Texas Southwestern Medical Center — Dallas, Texas, United States (enrolling)
• Hospital de Pediatría S.A.M.I.C.- Prof. Dr. Juan P. Garrahan — Parque Patricios, Ciudad Autónoma de BuenosAires, Argentina (enrolling)
• Hospital Santa Izabel-Rua Floriano Peixoto 300 — Salvador, Estado de Bahia, Brazil (enrolling)
• Hospital Sao Lucas Da Pontificia Universidade Catolica Do Rio Grande Do Sul (PUCRS) — Porto Alegre/RS, Brazil (enrolling)
• CPQuali Pesquisa Clínica Sao Paulo — São Paulo, Brazil (enrolling)
• Hospital das Clínicas da Faculdade de Medicina da Universidade de São Paulo — São Paulo, Brazil (enrolling)
• Hospital For Sick Children — Toronto, Ontario, Canada (enrolling)
• Centre Hospitalier Universitaire de Bicêtre — Le Kremlin-Bicêtre, Val-de-Marne, France (enrolling)
• Erasmus MC Sophia Children's Hospital-Wytemaweg 80 — Rotterdam, South Holland, Netherlands (enrolling)
• Uniwersyteckie Centrum Kliniczne w Gdansku - Smoluchowskiego 17 — Gdansk, Poland (enrolling)
• Clinic for Neurology and Psychiatry for Children and Youth — Belgrade, Belgrade, Serbia (enrolling)
• Hospital Sant Joan de Deu - PIN — Espluges de Llobregat, Barcelona, Spain (enrolling)
• Karolinska Universitetssjukhuset Solna — Stockholm, Stockholm County, Sweden (enrolling)
• Evelina London Children's Hospital — London, London, City of, United Kingdom (enrolling)
• Great Ormond Street Hospital - PPDS — London, London, City of, United Kingdom (enrolling)
• Birmingham Women's and Children's NHS Foundation Trust — Birmingham, West Midlands, United Kingdom (enrolling)

Full record on ClinicalTrials.gov

Trial information comes from ClinicalTrials.gov and is refreshed daily. TrialsForMe does not provide medical care and does not run the studies it lists.