Observational Safety Study in Women With Neuromyelitis Optica Spectrum Disorder (NMOSD) Exposed to UPLIZNA® During Pregnancy

An Observational Pregnancy Safety Study in Women With Neuromyelitis Optica Spectrum Disorder (NMOSD) Exposed to UPLIZNA® (Inebilizumab-cdon) During Pregnancy

This is an observational study to monitor female participants exposed to UPLIZNA during pregnancy. This study requires voluntary reporting of pregnancies in female participants with NMOSD exposed to UPLIZNA during pregnancy or within 6 months preceding conception. Pregnancy-related data, potential confounding factors and information related to pregnancy outcome will be collected. The schedule of office visits and all treatment regimens will be determined by the treating healthcare provider. Duration of the study is 10 years, at minimum.

Study Overview

• Status: Recruiting
• Conditions: Pregnancy Related; Neuromyelitis Optica Spectrum Disorder
• Intervention / Treatment: Drug: UPLIZNA

Detailed Description

Acquired from Horizon in 2024.

• Study Type: Observational
• Enrollment (Estimated): 60

Expanded Access

Available outside the clinical trial. See expanded access record.

Contacts and Locations

• Study Contact: Name: Amgen Call Center; Phone Number: 866-572-6436; Email: medinfo@amgen.com

Study Locations

• United States
  • Colorado
    • Aurora, Colorado, United States, 80045
      • Recruiting
      • University of Colorado Denver
      • Principal Investigator: Dr. Amanda Piquet, MD
      • Contact: Amanda Piquet, MD; Phone Number: 303-724-4644; Email: AMANDA.PIQUET@CUANSCHUTZ.EDU

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Child; Adult; Older Adult
• Accepts Healthy Volunteers: No

• Sampling Method: Non-Probability Sample

Study Population

The study population will consist of participants who have been exposed to UPLIZNA during pregnancy and have signed an informed consent form (ICF).

Description

Inclusion Criteria:

• Provide informed consent
• Are a female of reproductive potential with a confirmed or suspected diagnosis of NMOSD
• Have been exposed to UPLIZNA during pregnancy as defined by receipt of any dose during pregnancy or within 6 months preceding conception

Note: Other Protocol Defined Inclusion/Exclusion Criteria Apply

Study Plan

How is the study designed?

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Time Frame
Number of newborns with Major Congenital Malformations (MCMs)	Minimum of 10 years
Number of newborns with Preterm birth	Minimum of 10 years
Number of newborns with low birth weight	Minimum of 10 years
Number of stillbirths	Minimum of 10 years

Secondary Outcome Measures

Outcome Measure	Time Frame
Number of newborns with Minor Congenital Malformations	Minimum of 10 years
Number of infants with developmental milestone abnormalities	Minimum of 10 years
Number of infants with neurologic abnormalities	Minimum of 10 years
Number of infants with immune system development abnormalities	Minimum of 10 years
Number of Spontaneous Abortions	Minimum of 10 years
Number of Induced or Elective Abortions	Minimum of 10 years

Collaborators and Investigators

• Sponsor: Amgen
• Investigators: Study Director: MD, Amgen

Publications and helpful links

Helpful Links

• AmgenTrials clinical trials website

Study record dates

Study Major Dates

• Study Start (Estimated): October 15, 2026
• Primary Completion (Estimated): October 31, 2032
• Study Completion (Estimated): October 31, 2032

Study Registration Dates

• First Submitted: June 9, 2023
• First Submitted That Met QC Criteria: June 9, 2023
• First Posted (Actual): June 18, 2023

Study Record Updates

• Last Update Posted (Actual): August 31, 2026
• Last Update Submitted That Met QC Criteria: August 28, 2026
• Last Verified: August 1, 2026

More Information

Terms related to this study

• Keywords: Pregnancy; Neuromyelitis Optica Spectrum Disorder; inebilizumab; UPLIZNA; birth complications
• Additional Relevant MeSH Terms: Nervous System Diseases; Autoimmune Diseases; Immune System Diseases; Eye Diseases; Demyelinating Autoimmune Diseases, CNS; Autoimmune Diseases of the Nervous System; Demyelinating Diseases; Myelitis, Transverse; Optic Neuritis; Optic Nerve Diseases; Cranial Nerve Diseases; Neuromyelitis Optica
• Other Study ID Numbers: VIB0551.P4.S4; EUPAS105613 (Other Identifier: European Union Post Authorization Studies)

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: YES
• IPD Plan Description: De-identified individual patient data for variables necessary to address the specific research question in an approved data sharing request.
• IPD Sharing 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.
• IPD Sharing 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.
• IPD Sharing Supporting Information Type: STUDY_PROTOCOL; SAP; ICF; CSR

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: Yes
• Studies a U.S. FDA-regulated device product: No
• product manufactured in and exported from the U.S.: Yes