Ignite Creation Date:
2024-05-06 @ 8:28 PM
Last Modification Date:
2024-10-26 @ 3:28 PM
Study NCT ID:
NCT06398158
Status:
RECRUITING
Last Update Posted:
2024-06-27
First Post:
2024-04-16
Brief Title:
Study of the Clinical and Radiological Impact of Ravulizumab in People With Neuromyelitis Optica Spectrum Disorder
Sponsor:
University of Texas Southwestern Medical Center
Organization:
University of Texas Southwestern Medical Center
Study Overview
Official Title:
Clinical and Radiological Outcomes in People With Aquaporin-4 IgG Positive Neuromyelitis Optica Spectrum Disorder Treated With Ravulizumab
Status:
RECRUITING
Status Verified Date:
2024-07
Last Known Status:
None
Delayed Posting:
No
If Stopped, Why?:
Not Stopped
Has Expanded Access:
False
If Expanded Access, NCT#:
N/A
Has Expanded Access, NCT# Status:
N/A
Acronym:
AMAZE
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 eg serum neurofilament light chain sNfL conventional and novel MRI markers etc related to disease activity
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 eg serum neurofilament light chain sNfL conventional and novel MRI markers etc related to disease activity
Detailed Description:
This is a single-center prospective observational study with all participants recruited from The University of Texas Southwestern Medical Center in Dallas Texas Enrollment of 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
The study is composed of the following
1 Study screening Study screening will occur up to 45 days with a minimum of 3 days to allow for the scheduling of study related events During this time the eligibility of participants will be evaluated and diagnosis of aquaporin 4-IgG positive neuromyelitis optica spectrum confirmed by both the Core Clinical Committee and Core Imaging Committee If a subject is declared a screen failure all reasons for ineligibility will be captured Ineligible subjects may be rescreened at a later time
2 Open-label treatment with commercially covered ravulizumab Enrolled subjects will be treated with ravulizumab in a manner consistent with the FDA approved labeling All prescreening laboratory tests vaccinations and clinical surveillance studies will be ordered and managed by the Primary Clinical Team
Treatment with ravulizumab will continue throughout the course of the study unless one of the following events occur a An individual loses commercial coverage for ravulizumab b Discontinuation of the medication by the clinical team is recommended due to an adverse reaction intolerability or inadequate disease control The decision regarding treatment will be determined by the Primary Clinical Team and the patient c The participant prematurely discontinues treatment for personal reasons
In the event that the medication is discontinued prematurely participants will continue to be followed throughout the 52-78 week period with all clinical radiological and serological assessments performed
3 Clinical surveillance Clinical safety surveillance will be performed throughout the study and managed by the Primary Clinical Team In the event of new or concerning clinical symptoms participants will be evaluated within a 24-hour period via telehealth or in-person visit Clinical evaluations serological assessments and repeat MRI data per protocol will be performed within a 48-hour period The acute management of subjects will be led by the Primary Clinical Team All clinical data associated with acute clinical events will be sent to the Core Clinical Committee and relapses adjudicated by consensus MRI data acquired at the time of the event will be evaluated by the Core Imaging Committee and interval change in conventional imaging outcomes ie new andor newly enlarging T2-hyperintense lesions gadolinium enhancement adjudicated by consensus
4 Safety surveillance Safety assessments in association with treatment with ravulizumab will be managed by the Primary Clinical Team The use of concomitant medications will be tracked during the entire course of the study These will include prescribed medications for both NMOSD and non-NMOSD including the use of glucocorticosteroid treatment administered orally or parenterally along with over-the-counter agents
The study is composed of the following
1 Study screening Study screening will occur up to 45 days with a minimum of 3 days to allow for the scheduling of study related events During this time the eligibility of participants will be evaluated and diagnosis of aquaporin 4-IgG positive neuromyelitis optica spectrum confirmed by both the Core Clinical Committee and Core Imaging Committee If a subject is declared a screen failure all reasons for ineligibility will be captured Ineligible subjects may be rescreened at a later time
2 Open-label treatment with commercially covered ravulizumab Enrolled subjects will be treated with ravulizumab in a manner consistent with the FDA approved labeling All prescreening laboratory tests vaccinations and clinical surveillance studies will be ordered and managed by the Primary Clinical Team
Treatment with ravulizumab will continue throughout the course of the study unless one of the following events occur a An individual loses commercial coverage for ravulizumab b Discontinuation of the medication by the clinical team is recommended due to an adverse reaction intolerability or inadequate disease control The decision regarding treatment will be determined by the Primary Clinical Team and the patient c The participant prematurely discontinues treatment for personal reasons
In the event that the medication is discontinued prematurely participants will continue to be followed throughout the 52-78 week period with all clinical radiological and serological assessments performed
3 Clinical surveillance Clinical safety surveillance will be performed throughout the study and managed by the Primary Clinical Team In the event of new or concerning clinical symptoms participants will be evaluated within a 24-hour period via telehealth or in-person visit Clinical evaluations serological assessments and repeat MRI data per protocol will be performed within a 48-hour period The acute management of subjects will be led by the Primary Clinical Team All clinical data associated with acute clinical events will be sent to the Core Clinical Committee and relapses adjudicated by consensus MRI data acquired at the time of the event will be evaluated by the Core Imaging Committee and interval change in conventional imaging outcomes ie new andor newly enlarging T2-hyperintense lesions gadolinium enhancement adjudicated by consensus
4 Safety surveillance Safety assessments in association with treatment with ravulizumab will be managed by the Primary Clinical Team The use of concomitant medications will be tracked during the entire course of the study These will include prescribed medications for both NMOSD and non-NMOSD including the use of glucocorticosteroid treatment administered orally or parenterally along with over-the-counter agents
Study Oversight
Has Oversight DMC:
None
Is a FDA Regulated Drug?:
True
Is a FDA Regulated Device?:
False
Is an Unapproved Device?:
None
Is a PPSD?:
None
Is a US Export?:
True
Is an FDA AA801 Violation?:
None