Study of Inebilizumab in Pediatric Subjects With Neuromyelitis Optica Spectrum Disorder

NCT05549258 | Recruiting Phase 2 DMC FDA Drug

• 2 other identifiers: VIB0551.P2.S2.NMO2023-510007-22
• Study Type: interventional
• Target: 15
• Locations: 10 countries
• Sites: 18

Brief 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).

Conditions

Neuromyelitis Optica Spectrum Disorder

Keywords

NMOSD

Outcome Measures

Primary Outcomes (16)

• Maximum Observed Concentration (Cmax) of Inebilizumab

  Day 1 to Week 28

• Area Under the Concentration Versus Time Curve of Inebilizumab from Time 0 to 14 Days Post-dose (AUC0-14d)

  Day 1 to pre-dose on Day 15

• Area Under the Concentration Versus Time Curve of Inebilizumab from Time 0 Extrapolated to Infinity (AUC0-Inf)

  Day 1 to Week 80

• Systemic Clearance (CL) of Inebilizumab

  Day 1 to Week 80

• Terminal Elimination Half-life (t½) of Inebilizumab

  Day 1 to Week 80

• Volume of Distribution at Steady State (VSS) of Inebilizumab

  Day 1 to Week 80

• Change from Baseline in Peripheral Cluster of Differentiation (CD)20-positive B-cell Counts

  Week 1, Week 2, Week 28, Week 80

• Number of Participants Experiencing Treatment-emergent Adverse Events (TEAEs), Serious Adverse Events (SAEs), and Adverse Events of Special Interest (AESIs)

  Day 1 to Week 80

• Change from Baseline in Serum Chemistry

  Week 1, Week 2, Week 28, Week 80

• Change from Baseline in Hematology

  Week 1, Week 2, Week 28, Week 80

• Change from Baseline in Serum Immunoglobulins

  Week 1, Week 2, Week 28, Week 80

• Change from Baseline in Systolic Blood Pressure

  Week 1, Week 2, Week 28, Week 80

• Change from Baseline in Diastolic Blood Pressure

  Week 1, Week 2, Week 28, Week 80

• Change from Baseline in Pulse Rate

  Week 1, Week 2, Week 28, Week 80

• Change from Baseline in Respiratory Rate

  Week 1, Week 2, Week 28, Week 80

• Change from Baseline in Body Temperature

  Week 1, Week 2, Week 28, Week 80

Secondary Outcomes (8)

• Disease Activity: Time to First Relapse

  Day 1 to Week 80

• Disease Activity: Proportion of Relapse-free Participants

  Day 1 to Week 80

• Disease Activity: Annualized Relapse Rate

  Day 1 to Week 80

• Health-Related Quality of Life (HRQoL) change from baseline in Euro Quality of Life-5 Dimension Youth score

  Day 1 to Week 80

• HRQoL change from baseline in Pediatric Quality of Life Inventory

  Day 1 to Week 80

Study Arms (1)

Inebilizumab

EXPERIMENTAL

Infusion of Inebilizumab

Drug: Inebilizumab

Interventions

Inebilizumab DRUG

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

Inebilizumab

Eligibility Criteria

• Age: 2 Years - 17 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17)

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

Sponsors & Collaborators

• Amgen: lead

Study Sites (19)

UCSD Altman Clinical and Translational Research Institute Building

La Jolla, California, 92037-1337, United States

RECRUITING

Loma Linda University Children's Hospital - PIN

Loma Linda, California, 92354, United States

RECRUITING

Massachusetts General Hospital

Boston, Massachusetts, 02115, United States

RECRUITING

University of Texas Southwestern Medical Center

Dallas, Texas, 78701, United States

RECRUITING

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

Parque Patricios, Ciudad Autónoma de BuenosAires, C1245AAM, Argentina

RECRUITING

Hospital Santa Izabel-Rua Floriano Peixoto 300

Salvador, Estado de Bahia, 40050-410, Brazil

RECRUITING

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

Porto Alegre/RS, 90610-000, Brazil

RECRUITING

CPQuali Pesquisa Clínica Sao Paulo

São Paulo, 01228-000, Brazil

RECRUITING

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

São Paulo, 05403-000, Brazil

RECRUITING

Hospital For Sick Children

Toronto, Ontario, M5G 1X8, Canada

RECRUITING

Centre Hospitalier Universitaire de Bicêtre

Le Kremlin-Bicêtre, Val-de-Marne, 94275, France

RECRUITING

Erasmus MC Sophia Children's Hospital-Wytemaweg 80

Rotterdam, South Holland, 3015 GD, Netherlands

RECRUITING

Uniwersyteckie Centrum Kliniczne w Gdansku - Smoluchowskiego 17

Gdansk, 80-952, Poland

RECRUITING

Clinic for Neurology and Psychiatry for Children and Youth

Belgrade, Belgrade, 11000, Serbia

RECRUITING

Hospital Sant Joan de Deu - PIN

Espluges de Llobregat, Barcelona, 08950, Spain

RECRUITING

Karolinska Universitetssjukhuset Solna

Stockholm, Stockholm County, 17176, Sweden

RECRUITING

Evelina London Children's Hospital

London, London, City of, SE1 7EH, United Kingdom

RECRUITING

Great Ormond Street Hospital - PPDS

London, London, City of, WC1N 3JH, United Kingdom

RECRUITING

Birmingham Women's and Children's NHS Foundation Trust

Birmingham, West Midlands, B4 6NH, United Kingdom

RECRUITING

Related Links

• AmgenTrials clinical trials website

MeSH Terms

Conditions

Neuromyelitis Optica

Interventions

inebilizumab

Condition Hierarchy (Ancestors)

Myelitis, Transverse; Demyelinating Autoimmune Diseases, CNS; Autoimmune Diseases of the Nervous System; Nervous System Diseases; Optic Neuritis; Optic Nerve Diseases; Cranial Nerve Diseases; Demyelinating Diseases; Eye Diseases; Autoimmune Diseases; Immune System Diseases

Study Officials

• MD

  Amgen

  STUDY DIRECTOR

Central Study Contacts

Amgen Call Center

CONTACT

866-572-6436; medinfo@amgen.com

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR
• Expanded Access: Yes

Study Record Dates

• First Submitted: July 22, 2022
• First Posted: September 22, 2022
• Study Start: July 3, 2023
• Primary Completion (Estimated): April 13, 2027
• Study Completion (Estimated): April 13, 2027
• Last Updated: December 5, 2025

Record last verified: 2025-12

Data Sharing

• IPD Sharing: Will share
• Shared Documents: STUDY PROTOCOL, SAP, ICF, CSR
• Time Frame: Data sharing requests relating to this study will be considered beginning 18 months after the study has ended and either 1) the product and indication have been granted marketing authorization in both the US and Europe or 2) clinical development for the product and/or indication discontinues and the data will not be submitted to regulatory authorities. There is no end date for eligibility to submit a data sharing request for this study.
• Access Criteria: Qualified researchers may submit a request containing the research objectives, the Amgen product(s) and Amgen study/studies in scope, endpoints/outcomes of interest, statistical analysis plan, data requirements, publication plan, and qualifications of the researcher(s). In general, Amgen does not grant external requests for individual patient data for the purpose of re-evaluating safety and efficacy issues already addressed in the product labelling. Requests are reviewed by a committee of internal advisors. If not approved, a Data Sharing Independent Review Panel will arbitrate and make the final decision. Upon approval, information necessary to address the research question will be provided under the terms of a data sharing agreement. This may include anonymized individual patient data and/or available supporting documents, containing fragments of analysis code where provided in analysis specifications. Further details are available at the URL below.

Locations

SE (1); GB (3); PL (1); NL (1); FR (1); AR (1); US (4); ES (1); BR (4); CA (1)