Long-term, Open-label, Safety Study of Inebilizumab in Neuromyelitis Optica Spectrum Disorder (NMOSD)

NCT06180278 | Active Not Recruiting Phase 4 FDA Drug

• 1 other identifier: HZNP-UPL-401
• Study Type: interventional
• Target: 32
• Locations: 7 countries
• Sites: 11

Brief Summary

The purpose of this study is to understand the effects of long-term treatment with inebilizumab on circulating levels of immunoglobulins, B-cell counts, and other safety measures, and to further monitor repletion of immunoglobins and B-cell counts in participants with NMOSD who discontinue treatment. The objectives include:

1. 1.To establish the nadir in circulating immunoglobulins (Ig) during chronic treatment with inebilizumab and ascertain the time needed to ensure restoration of pre-treatment baseline serum levels of IgG and IgM after discontinuation of treatment
2. 2.To characterize B-cell counts throughout treatment with inebilizumab and after discontinuation until repletion of Immunoglobulin (Ig levels)
3. 3.To assess long-term safety of inebilizumab
4. 4.To assess other long-term effects of inebilizumab

Conditions

Neuromyelitis Optica Spectrum Disorder

Keywords

Immunoglobulins; B-cell count; inebilizumab; Uplizna

Outcome Measures

Primary Outcomes (2)

• Change from baseline in serum Ig levels (total Ig, IgG, IgM, IgA, IgE) over time

  This will be assessed via serum samples, drawn at the site and processed at a central laboratory, and analyses performed to assess change from baseline over time, as measured by each parameter in mg/dL

  Up to 42 months

• Change from baseline in peripheral CD20+ B-cell counts over time

  This will be assessed via serum samples, drawn at the site and processed at a central laboratory, and analyses performed to assess change from baseline over time, as measured by each parameter in cells/mL

  Up to 42 months

Secondary Outcomes (11)

• Change from baseline in hematology over time

  Up to 42 months

• Change from baseline in clinical chemistry over time

  Up to 42 months

• Incidence of serious infections

  Up to 42 months

• Incidence of viral reactivation

  Up to 42 months

• Incidence of progressive multifocal leukoencephalopathy (PML)

  Up to 42 months

Study Arms (1)

Participants with NMOSD exposed to inebilizumab

OTHER

Participants with NMOSD who previously enrolled in N-MOmentum study, who participated for at least 2 years in the open label phase (OLP) of the study, and participants newly initiating inebilizumab treatment will have hematology, chemistry, B-cell count, serum immunoglobulin (Ig) levels, adverse events, concomitant medications list, NMOSD attacks information, antidrug antibody (ADA) status and titers collected.

Other: Blood tests; Drug: Inebilizumab

Interventions

Blood tests OTHER

Participants will have blood tests done at each scheduled visit (approximately every 6 months).

Participants with NMOSD exposed to inebilizumab

Inebilizumab DRUG

Participants with NMOSD who previously enrolled in N-MOmentum study, who participated for at least 2 years in the open label phase (OLP) of the study, and participants newly initiating inebilizumab treatment at the discretion of their treating physician will have hematology, chemistry, B-cell count, serum immunoglobulin (Ig) levels, adverse events, concomitant medications list, NMOSD attacks information, antidrug antibody (ADA) status and titers collected.

Also known as: UPLIZNA

Participants with NMOSD exposed to inebilizumab

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Age 18 years or above, and able to provide written informed consent and any locally required authorization (e.g., Health Insurance Portability and Accountability Act \[HIPAA\] in the United States of America (USA), European Union \[EU\] Data Privacy Directive in the EU) obtained from the subject/legal representative prior to performing any protocol-related procedures, including screening evaluations.
• Have completed at least 2 years in the open-label period of the N-MOmentum study or are newly initiating inebilizumab treatment at a participating site.
• Females of childbearing potential who are sexually active with a nonsterilized male partner must use a highly effective method of contraception as outlined in the protocol (subjects in the Czech Republic only must use 1 additional method of contraception) from screening, and must agree to continue using such precautions for 6 months after treatment with inebilizumab; cessation of contraception after this point should be discussed with a responsible physician.
• Nonsterilized males who are sexually active with a female partner of childbearing potential must use a male condom plus spermicide (subjects in the Czech Republic only must use 1 additional method of contraception) from Day 1 for 3 months after receipt of last treatment with inebilizumab.
• Sterilized males, without the appropriate post-vasectomy documentation on the absence of sperm in the ejaculate, who are sexually active with a female partner of childbearing potential must use a condom and spermicide from Day 1 for 3 months after receipt of the last treatment with inebilizumab.

You may not qualify if:

• Have any condition that would place the participant at unacceptable risk of complications, interfere with evaluation of inebilizumab or confound the interpretation of participant safety or study results.
• Received rituximab or any other B-cell depleting agent after exit from N-MOmentum study, or within the last 12 months prior to screening for non N-MOmentum participants.
• Known history of allergy or reaction to any component of inebilizumab formulation or history of anaphylaxis following any biologic therapy
• Have a severe clinically significant infection, including active chronic infection such as hepatitis B
• Have active or untreated latent tuberculosis
• Have a history of progressive multifocal leukoencephalopathy (PML)
• Is severely immunocompromised state
• Have active malignancies

Sponsors & Collaborators

• Amgen: lead

Study Sites (11)

Wayne State University School of Medicine

Detroit, Michigan, 48201-2153, United States

Location

Baylor College of Medicine

Houston, Texas, 77030-4202, United States

Location

Prince of Wales Hospital (PWH) - The Chinese University of Hong Kong (CUHK) - Ophtalmology

Hong Kong, 0000, China

Location

Vseobecna fakultni nemocnice v Praze - Neurologicka klinika

Prague, 128 21, Czechia

Location

Szegedi Tudományegyetem, à OK, Szent-Györgyi Albert Klinikai Központ - Neurológiai Osztály

Szeged, Csongrád megye, 6725, Hungary

Location

Uniwersyteckie Centrum Kliniczne WUM - Oddzial Kliniczny Neurologii

Warsaw, Masovian Voivodeship, 02-057, Poland

Location

M.A.-Lek A.M. Maciejowscy S.C. Centrum Terapii SM

Katowice, Silesian Voivodeship, 40-751, Poland

Location

National Cancer Center - Neurology Clinic

Goyang-si, Gyeonggido [Kyonggi-do], 10408, South Korea

Location

Seoul National University Hospital

Seoul, Seoul Teugbyeolsi [Seoul-T'ukp, 03080, South Korea

Location

Samsung Medical Center - Pediatric Neurology

Seoul, Seoul Teugbyeolsi [Seoul-T'ukp, 6351, South Korea

Location

Khon Kaen University, Srinagarind Hospital - Division of Neurology, Depart

Khon Kaen, 40002, Thailand

Location

Related Links

• AmgenTrials clinical trials website

MeSH Terms

Conditions

Neuromyelitis Optica

Interventions

Hematologic Tests; 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

Intervention Hierarchy (Ancestors)

Clinical Laboratory Techniques; Diagnostic Techniques and Procedures; Diagnosis; Investigative Techniques

Study Officials

• MD

  Amgen

  STUDY DIRECTOR

Study Design

• Study Type: interventional
• Phase: phase 4
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR
• Expanded Access: Yes

Study Record Dates

• First Submitted: November 17, 2023
• First Posted: December 22, 2023
• Study Start: April 2, 2024
• Primary Completion (Estimated): June 26, 2028
• Study Completion (Estimated): June 26, 2028
• Last Updated: November 21, 2025

Record last verified: 2025-11

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

CN (1); CZ (1); US (2); HU (1); KR (3); TH (1); PL (2)