15 Participants Needed

Inebilizumab for Neuromyelitis Optica

Recruiting at 29 trial locations
AC
HT
Overseen ByHorizon Therapeutics
No Placebo GroupAll trial participants will receive the active study treatment (no placebo)
Prior Safety DataThis treatment has passed at least one previous human trial
Approved in 3 JurisdictionsThis treatment is already approved in other countries

Trial Summary

Will I have to stop taking my current medications?

The trial requires that you stop taking certain medications before starting, such as rituximab, IVIG, and others listed in the exclusion criteria. If you're on any of these, you may need to stop them for a specific period before joining the trial.

Is inebilizumab safe for humans?

Inebilizumab, also known as Uplizna, has been generally well tolerated in clinical trials for neuromyelitis optica spectrum disorder (NMOSD), with common side effects including urinary tract infections and joint pain. It has been used safely in humans for over four years in studies, with the most frequent side effects being infusion reactions and respiratory infections.12345

How does the drug inebilizumab work for neuromyelitis optica?

Inebilizumab is unique because it targets and depletes B cells that express a specific marker called CD19, which helps reduce attacks in neuromyelitis optica. It is given as an intravenous infusion and has been shown to lower the risk of attacks and hospitalizations in patients with this condition.13467

What is the purpose of this trial?

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).

Research Team

M

MD

Principal Investigator

Amgen

Eligibility Criteria

This trial is for kids aged 2 to <18 with Neuromyelitis Optica Spectrum Disorder (NMOSD) who've had at least one relapse in the past year or two in the last two years. They must test positive for AQP4-IgG antibodies and meet specific diagnostic criteria. Kids can't join if they've recently taken certain meds, received live vaccines or blood transfusions, have uncontrolled autoimmune diseases, significant organ dysfunction, severe allergies to multiple drugs/foods, or low B-cell counts.

Inclusion Criteria

I have NMOSD and tested positive for anti-AQP4-IgG.
I am between 2 and 17 years old.
I have had at least one NMOSD flare-up in the past year or two in the last two years.

Exclusion Criteria

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
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
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
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Timeline

Screening

Participants are screened for eligibility to participate in the trial

up to 4 weeks

Treatment

Participants receive Inebilizumab administered intravenously over 28 weeks

28 weeks
9 visits (in-person)

Follow-up

Participants are monitored for safety and effectiveness after treatment

52 weeks
4 visits (in-person)

Treatment Details

Interventions

  • Inebilizumab
Trial Overview The study tests Inebilizumab's effects on children with NMOSD. It's an open-label Phase 2 trial where all participants receive the drug; researchers will look at how it moves through and affects the body (PK/PD), as well as its safety profile.
Participant Groups
1Treatment groups
Experimental Treatment
Group I: InebilizumabExperimental Treatment1 Intervention
Infusion of Inebilizumab

Inebilizumab is already approved in United States, European Union, Canada for the following indications:

🇺🇸
Approved in United States as Uplizna for:
  • Neuromyelitis optica spectrum disorder (NMOSD) in adults who are anti-aquaporin-4 (AQP4) antibody positive
🇪🇺
Approved in European Union as Uplizna for:
  • Neuromyelitis optica spectrum disorder (NMOSD) in adults who are anti-aquaporin-4 (AQP4) antibody positive
🇨🇦
Approved in Canada as Uplizna for:
  • Neuromyelitis optica spectrum disorder (NMOSD) in adults who are anti-aquaporin-4 (AQP4) antibody positive

Find a Clinic Near You

Who Is Running the Clinical Trial?

Amgen

Lead Sponsor

Trials
1,508
Recruited
1,433,000+
Founded
1980
Headquarters
Thousand Oaks, USA
Known For
Human Therapeutics
Top Products
Enbrel, Prolia, Neulasta, Otezla
Robert A. Bradway profile image

Robert A. Bradway

Amgen

Chief Executive Officer since 2012

MBA from Harvard Business School

Paul Burton profile image

Paul Burton

Amgen

Chief Medical Officer since 2023

MD from University of London, PhD in Molecular and Cellular Biology from Imperial College London

Horizon Therapeutics Ireland DAC

Lead Sponsor

Trials
21
Recruited
2,500+

Findings from Research

Inebilizumab is a humanized anti-CD19 monoclonal antibody that effectively targets and depletes CD19-expressing B cells, which is crucial for treating autoimmune diseases.
In June 2020, inebilizumab received its first approval in the USA for treating neuromyelitis optica spectrum disorder (NMOSD) in adults who are positive for AQP4-IgG, marking a significant milestone in its development.
Inebilizumab: First Approval.Frampton, JE.[2021]
In a long-term study of 75 adults with neuromyelitis optica spectrum disorder treated with inebilizumab for over 4 years, the annualized attack rate was very low at 0.052 attacks per person per year, indicating strong efficacy in preventing attacks.
Inebilizumab was well tolerated with only 2.7% of participants experiencing serious adverse events, and no deaths reported, suggesting it is a safe treatment option for this condition.
Long-term efficacy and safety of inebilizumab in neuromyelitis optica spectrum disorder: Analysis of aquaporin-4-immunoglobulin G-seropositive participants taking inebilizumab for ⩾4 years in the N-MOmentum trial.Rensel, M., Zabeti, A., Mealy, MA., et al.[2022]
Inebilizumab-cdon (Uplizna™) is an FDA-approved treatment for neuromyelitis optica spectrum disorder (NMOSD), demonstrating a safe and effective profile in clinical trials.
The drug works by depleting CD19+ B cells through mechanisms like antibody-dependent cell cytotoxicity (ADCC) and phagocytosis, which are crucial for its therapeutic action.
Inebilizumab-cdon: USFDA Approved for the Treatment of NMOSD (Neuromyelitis Optica Spectrum Disorder).Ali, F., Sharma, K., Anjum, V., et al.[2022]

References

Inebilizumab: First Approval. [2021]
Long-term efficacy and safety of inebilizumab in neuromyelitis optica spectrum disorder: Analysis of aquaporin-4-immunoglobulin G-seropositive participants taking inebilizumab for ⩾4 years in the N-MOmentum trial. [2022]
3.United Arab Emiratespubmed.ncbi.nlm.nih.gov
Inebilizumab-cdon: USFDA Approved for the Treatment of NMOSD (Neuromyelitis Optica Spectrum Disorder). [2022]
Inebilizumab: A Review in Neuromyelitis Optica Spectrum Disorder. [2023]
Inebilizumab in AQP4-Ab-positive neuromyelitis optica spectrum disorder. [2021]
Association between B-cell depletion and attack risk in neuromyelitis optica spectrum disorder: An exploratory analysis from N-MOmentum, a double-blind, randomised, placebo-controlled, multicentre phase 2/3 trial. [2022]
Pharmacodynamic modelling and exposure-response assessment of inebilizumab in subjects with neuromyelitis optica spectrum disorders. [2022]
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