Summary

Eligibility
for people ages 2-17 (full criteria)
Location
at La Jolla, California and other locations
Dates
study started
estimated completion
Principal Investigator
by Jennifer Sharon Graves, MD, PhD
Headshot of Jennifer Sharon Graves
Jennifer Sharon Graves

Description

Summary

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).

Official Title

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

Details

Approximately 15 subjects to be enrolled and receive Inebilizumab administered intravenously over 28 weeks. The maximum trial duration per participant is approximately 80 weeks, including up to 4 week screening period, 9 visits during a 28 week open-label treatment period, and approximately 4 visits during a 52 week follow-up period. Safety evaluations will be performed regularly throughout the course of the study.

Keywords

Neuromyelitis Optica Spectrum Disorder, Neuromyelitis Optica, Inebilizumab

Eligibility

You can join if…

Open to people ages 2-17

  • 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.

You CAN'T join if...

  • 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:
  • 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

Locations

  • UC San Diego Altman Clinical and Translational Research Institute (ACTRI) Building not yet accepting patients
    La Jolla California 92037-1337 United States
  • University of Texas Southwestern Medical Center not yet accepting patients
    Dallas Texas 78701 United States

Lead Scientist at UCSD

  • Jennifer Sharon Graves, MD, PhD
    Associate Professor Of Clinical, Neurosciences, Vc-health Sciences-schools. Authored (or co-authored) 65 research publications

Details

Status
accepting new patients at some sites,
but this study is not currently recruiting here
Start Date
Completion Date
(estimated)
Sponsor
Horizon Therapeutics Ireland DAC
ID
NCT05549258
Phase
Phase 2 Neuromyelitis Optica Spectrum Disorders Research Study
Study Type
Interventional
Participants
Expecting 15 study participants
Last Updated