NCT05926167

Observational Trial Evaluating Elevated Factor VIII Related Labs as a Biomarker for Incomplete Relapse Recovery

VALIDATE II Study: Observational Trial Evaluating Elevated Factor VIII Related Labs in Multiple Sclerosis Relapse as a Marker for Incomplete Recovery

• Status: Unknown
• Phase: —
• Study type: Observational
• Enrollment: 9 (estimated)
• Sponsor: Dignity Health Medical Foundation · Academic / Other
• Sex: All
• Age: 18 Years – 70 Years
• Healthy volunteers: Not accepted

Summary

Nine Multiple Sclerosis (MS) patients suffering an acute relapse from the outpatient or inpatient settings will be consented to be followed prospectively for three months post relapse, in an effort to identify markers of incomplete relapse recovery. Factor VIII-related labs will be drawn for three months without influencing standard of care treatment decisions. During this time, patients will be followed with clinical and diagnostic assessments in addition to blood tests including: Expanded Disability Status Scale (EDSS), Multiple Sclerosis Functional Composite (MSFC), recovery surveys, and MRIs of the brain, cervical spine, and thoracic spine with and without contrast. Clinical, imaging, and Factor VIII-related lab data individually or in aggregate will be correlated with relapse presence, severity, and extent of recovery following standard treatment interventions.

Detailed description

The identification of blood tests that detect patients having disability provoking Multiple Sclerosis (MS) relapses in real time could lead to a new era of MS relapse treatment. The cross-talk between inflammatory and coagulation pathways in multiple sclerosis has been recognized on some level for decades. Accidental and anecdotal clinical observations suggest that excessive activation of the intrinsic coagulation pathway, namely with increased Factor VIII activity, occurs with some MS relapses, and that these are the relapses that don't recover with current standard of care treatments, leaving behind permanent disability. To test this hypothesis that real time abnormally increased levels of Factor VIII activity related labs can identify disabling relapses in real time, nine adult patients with relapsing remitting MS with or without secondary progression will be consented and enrolled in this observational, longitudinal clinical trial. Patients will receive standard of care treatment per the investigator's discretion for their relapse, but will have additional blood tests testing Factor VIII activity, Factor VIII antigen, serum neurofilament among others drawn on days 1, 8, 22 and 90 of the study. The investigator and the patient will not know these lab test results. Patients will also have mris of the brain, cervical spine, and thoracic spine done with and without contrast as soon as possible upon study entry. On Days 1,8,22, and 90, in addition to blood tests, the patient will complete an EDSS and MSFC (that looks at ambulation, hand/arm coordination, and cognitive function, comprised of the 25 foot timed walking tests, with and without any assistive devices if possible), and fill out a recovery survey. Individual blood tests, aggregate blood tests, mri findings, EDSS and MSFC individual and summed scores will be scrutinized and evaluated for the ability to identify a MS relapse and to identify a treatment-resistant MS relapse

Conditions

• Multiple Sclerosis
• Relapse

Interventions

Type	Name	Description
DIAGNOSTIC_TEST	blood tests drawn days 1, 8, 22 and 90 of the study	Blood tests related to Factor VIII activity that are not a part of standard of care

Timeline

• Start date: 2023-09-12
• Primary completion: 2024-05-01
• Completion: 2024-12-01
• First posted: 2023-07-03
• Last updated: 2023-09-14