Study of the Clinical and Radiological Impact of Ravulizumab in People With Neuromyelitis Optica Spectrum Disorder

NCT06398158 | Recruiting DMC FDA Drug

• 1 other identifier: STU-2023-0744
• Study Type: observational
• Target: 35
• Locations: 1 country
• Sites: 1

Brief Summary

This is an observational study to:

• evaluate the on-treatment clinical performance of ravulizumab in relation to the pre-treatment time period (time period prior to exposure),
• enhance knowledge regarding conventional MRI outcomes in people with NMOSD treated with ravulizumab,
• identify factors suggestive of subclinical disease progression through conventional MRI sequences,
• determine if treatment with ravulizumab, impacts longitudinal 3D conformational MRI measures at the dorsal medulla and other regions of the CNS, and
• identify biomarkers (e.g., serum neurofilament light chain (sNfL), conventional and novel MRI markers, etc.) related to disease activity.

Conditions

Neuromyelitis Optica

Outcome Measures

Primary Outcomes (1)

• Number of participants with clinical relapses before and after treatment with ravulizumab

  Compare the number of participants with adjudicated clinical relapses both pre- and post-ravulizumab treatment

  52 weeks - 78 weeks

Study Arms (1)

NMOSD

35 people with aquaporin-4 IgG antibody positive neuromyelitis optica spectrum disorder is planned with all individuals treated with commercially supplied ravulizumab at the recommendation of the healthcare provider, with use consistent with the approved indication and labeling. Approximately 50% of the cohort is anticipated to be Black/African American based on existing internal data.

Drug: Ravulizumab

Interventions

Ravulizumab DRUG

All study participants will receive commercially covered ravulizumab consistent with the indication, dose and frequency contained within the approved label.

NMOSD

Eligibility Criteria

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

Study Population

The study population will consist of male and female adult participants recruited from The Multiple Sclerosis and Neuroimmunology Clinic at The University of Texas Southwestern Medical Center in Dallas, Texas.

You may qualify if:

• Signed informed consent available prior to conduct of any study associated activities
• Men and women \> 18 years of age
• Aquaporin-4 IgG positive people with neuromyelitis optica spectrum disorder treated with commercially available ravulizumab in a manner consistent with the approved indication
• Expanded Disability Status Scale score of \<7.0

You may not qualify if:

• Individuals who are intolerant to MRI
• Individuals previously exposed to eculizumab with treatment discontinuation due to lack of effective disease control (i.e., clinical relapse or demonstration of MRI advancement after 12 weeks of sustained treatment exposure)
• Unresolved meningococcal disease
• History of an active infection
• Existing participation in neuromyelitis optical spectrum disorder interventional clinical studies
• Pregnant or lactating women

Sponsors & Collaborators

• University of Texas Southwestern Medical Center: lead
• Alexion Pharmaceuticals, Inc.: collaborator

Study Sites (1)

UT Southwestern Medical Center

Dallas, Texas, 75390, United States

RECRUITING

Biospecimen

Retention: SAMPLES WITH DNA

Whole blood samples will be routinely collected during the course of the study, in addition to acute clinical relapses to assess the temporal course of change in serum neurofilament light chain, glial fibrillary acidic protein, tau, and ubiquitin carboxyl-terminal hydrolase levels. Data captured from the samples collected may be utilized in the future to identify or verify putative prognostic and predictive markers associated with disease and markers of therapeutic response to treatment. Baseline and dynamic (within study) clinical disease characteristics and associated biomarker data may be utilized to predict subsequent disease advancement along with severity, identifying high-risk subgroups and identify predictors of response to treatment.

MeSH Terms

Conditions

Neuromyelitis Optica

Interventions

ravulizumab

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

• Darin T. Okuda, MD

  University of Texas Southwestern Medical Center

  PRINCIPAL INVESTIGATOR

• Peter Sguigna, MD

  University of Texas Southwestern Medical Center

  PRINCIPAL INVESTIGATOR

Central Study Contacts

Diem Tran, PhD

CONTACT

214-645-9165; diemh.tran@utsouthwestern.edu

Jose Santoyo

CONTACT

214-645-1878; jose.santoyo@utsouthwestern.edu

Study Design

• Study Type: observational
• Observational Model: COHORT
• Time Perspective: PROSPECTIVE
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Professor

Study Record Dates

• First Submitted: April 16, 2024
• First Posted: May 3, 2024
• Study Start: July 10, 2024
• Primary Completion (Estimated): December 31, 2026
• Study Completion (Estimated): June 30, 2027
• Last Updated: August 5, 2025

Record last verified: 2025-07

Data Sharing

• IPD Sharing: Will not share

Locations

US (1)