Study of Inebilizumab in Pediatric Subjects With Neuromyelitis Optica Spectrum Disorder

NCT ID: NCT05549258

Last Updated: 2025-12-05

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

RECRUITING

Clinical Phase

PHASE2

Total Enrollment

15 participants

Study Classification

INTERVENTIONAL

Study Start Date

2023-07-03

Study Completion Date

2027-04-13

Brief Summary

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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).

Detailed Description

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Approximately 15 participants 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.

Acquired from Horizon in 2023.

Conditions

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Neuromyelitis Optica Spectrum Disorder

Study Design

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Allocation Method

NA

Intervention Model

SINGLE_GROUP

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

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Inebilizumab

Infusion of Inebilizumab

Group Type EXPERIMENTAL

Inebilizumab

Intervention Type DRUG

Inebilizumab administered intravenously (IV) over a total of 28 weeks.

Interventions

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Inebilizumab

Inebilizumab administered intravenously (IV) over a total of 28 weeks.

Intervention Type DRUG

Eligibility Criteria

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Inclusion Criteria

* 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.

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 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:

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.

Receipt of any of the following:

1. 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).
2. Bacillus Calmette Guérin vaccine within one year of screening.
3. Blood transfusion within 4 weeks prior to screening or during screening.

* Clinically significant serious active or chronic viral, bacterial, or fungal infection that requires treatment with anti-infectives, hospitalization, or, in the Investigator's opinion, represents an additional risk to the participant, within 2 months prior to Day 1.
* Known history of congenital or acquired immunodeficiency (e.g., due to human immunodeficiency virus \[HIV\] infection, splenectomy, immunosuppression-related or idiopathic T-cell deficiencies) that predisposes the participant to infection.
* Positive test for chronic hepatitis B infection at screening, defined as either:

a. Positive hepatitis B surface antigen (HBsAg), or b. Positive hepatitis B core (HBc) antibody (anti-HBc) plus negative hepatitis B surface (HBs) antibody (anti-HBs).

* Positive test for hepatitis C virus antibody.
* Negative test for varicella zoster virus (VZV)-IgG.
* History of cancer, apart from squamous cell or basal cell carcinoma of the skin treated with documented success of curative therapy \> 3 months prior to Day 1.
* History of active or latent tuberculosis (TB), or a positive QuantiFERON®-TB Gold test at screening, unless treatment for TB was completed per local guidelines. Participants with latent TB or a positive QuantiFERON®-TB Gold test who are actively on anti-TB treatment can enroll if they have completed at least one month of anti-TB treatment and intend to complete the full course of anti-TB treatment. Participants with an indeterminate QuantiFERON®-TB Gold test result can enroll if a repeat QuantiFERON®-TB Gold test is negative or a tuberculin skin test is negative.
* For participants who may undergo MRI scans:

1. Unable to undergo an MRI scan (e.g., hypersensitivity to Gd-containing MRI contrast agents, implanted pacemakers, defibrillators, or other metallic objects on or inside the body that limit performing MRI scans), or
2. Unable to tolerate or comply with the MRI procedure.
Minimum Eligible Age

2 Years

Maximum Eligible Age

17 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Amgen

INDUSTRY

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Principal Investigators

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MD

Role: STUDY_DIRECTOR

Amgen

Locations

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UCSD Altman Clinical and Translational Research Institute Building

La Jolla, California, United States

Site Status RECRUITING

Loma Linda University Children's Hospital - PIN

Loma Linda, California, United States

Site Status RECRUITING

Massachusetts General Hospital

Boston, Massachusetts, United States

Site Status RECRUITING

University of Texas Southwestern Medical Center

Dallas, Texas, United States

Site Status RECRUITING

Hospital de Pediatría S.A.M.I.C.- Prof. Dr. Juan P. Garrahan

Parque Patricios, Ciudad Autónoma de BuenosAires, Argentina

Site Status RECRUITING

Hospital Santa Izabel-Rua Floriano Peixoto 300

Salvador, Estado de Bahia, Brazil

Site Status RECRUITING

Hospital Sao Lucas Da Pontificia Universidade Catolica Do Rio Grande Do Sul (PUCRS)

Porto Alegre/RS, , Brazil

Site Status RECRUITING

CPQuali Pesquisa Clínica Sao Paulo

São Paulo, , Brazil

Site Status RECRUITING

Hospital das Clínicas da Faculdade de Medicina da Universidade de São Paulo

São Paulo, , Brazil

Site Status RECRUITING

Hospital For Sick Children

Toronto, Ontario, Canada

Site Status RECRUITING

Centre Hospitalier Universitaire de Bicêtre

Le Kremlin-Bicêtre, Val-de-Marne, France

Site Status RECRUITING

Erasmus MC Sophia Children's Hospital-Wytemaweg 80

Rotterdam, South Holland, Netherlands

Site Status RECRUITING

Uniwersyteckie Centrum Kliniczne w Gdansku - Smoluchowskiego 17

Gdansk, , Poland

Site Status RECRUITING

Clinic for Neurology and Psychiatry for Children and Youth

Belgrade, Belgrade, Serbia

Site Status RECRUITING

Hospital Sant Joan de Deu - PIN

Espluges de Llobregat, Barcelona, Spain

Site Status RECRUITING

Karolinska Universitetssjukhuset Solna

Stockholm, Stockholm County, Sweden

Site Status RECRUITING

Evelina London Children's Hospital

London, London, City of, United Kingdom

Site Status RECRUITING

Great Ormond Street Hospital - PPDS

London, London, City of, United Kingdom

Site Status RECRUITING

Birmingham Women's and Children's NHS Foundation Trust

Birmingham, West Midlands, United Kingdom

Site Status RECRUITING

Countries

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United States Argentina Brazil Canada France Netherlands Poland Serbia Spain Sweden United Kingdom

Central Contacts

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Amgen Call Center

Role: CONTACT

866-572-6436

Related Links

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http://www.amgentrials.com

AmgenTrials clinical trials website

Other Identifiers

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2023-510007-22

Identifier Type: EUDRACT_NUMBER

Identifier Source: secondary_id

VIB0551.P2.S2.NMO

Identifier Type: -

Identifier Source: org_study_id

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