FcRn Antagonists (Efgartigimod) for Acute NMOSD Attack
NCT Number: NCT06497374
Acronym: FACT
Status: NOT_YET_RECRUITING
Study Type: INTERVENTIONAL
Phases: PHASE2
Conditions: Neuromyelitis Optica|Neuromyelitis Optica Spectrum Disorder|NMO Spectrum Disorder
Interventions: DRUG: Efgartigimod Alfa Injection|DRUG: High-dose intravenous methylprednisolone
Sponsor: Tianjin Medical University General Hospital
Sex: ALL
Age: ADULT, OLDER_ADULT
Enrollment: 63
Study Design: Allocation: RANDOMIZED|Intervention Model: PARALLEL|Masking: NONE|Primary Purpose: TREATMENT
Start Date: 2024-07-05
Completion Date: 2026-06-30

Summary:
NMOSD is an autoimmune disease of the central nervous system that predominantly affects the spinal cord and optic nerves. The objectives of this study are to assess the efficacy and safety of FcRn antagonists (efgartigmod) for treatment of patients with neuromyelitis optica spectrum disorders during acute phase who are anti-aquaporin-4 (AQP4) antibody-positive. The potential of efgartigimod, an IgG1 Fc fragment that competes with IgG for FcRn binding, thereby lowering IgG levels, warrants further investigation as a treatment for acute neuromyelitis optica spectrum disorders attacks. This study aims to evaluate the therapeutic potential of efgartigmod in acute NMOSD attack.

Primary Outcome Measures: Change in Neurological Disability - Expanded Disability Scale Score, The Kurtzke Expanded Disability Status Scale (EDSS) was developed to measure the disability status of subjects with demyelinating disease. It allows an objective quantification of the level of functioning that could be widely and reproducibly used by researchers and health care providers. The EDSS provides a total score on a scale that ranges from 0 to 10 where 0 is normal and 10 is deceased. Increasing disability is reflected in an increasing EDSS score., Acute nadir to week4
Secondary Outcome Measures: Change of FS score and MRC score at Week 12., To evaluate changes in neurologic outcomes in subjects with acute attacks in subjects with AQP4-IgG antibody-positive neuromyelitis optica spectrum disorders who are treated with efgartigimod + IVMP versus IVMP alone at Week 12.

The Functional System (FS) was developed to measure the function of seven systems, including visual functions (score 0-6), brainstem functions (score 0-5), pyramical functions (score 0-6), cerebellar functions (score 0-5, X is the condition that pyramidal weakness (BMRC grade 3 or worse in limb strength) or sensory deficits interfere with cerebellar testing), sensory functions (score 0-6), bowel and bladder functions (score 0-6), cerebral functions (score 0-5). Increasing disability of different functional system is reflected in an increasing FS score.

Muscle Power Assessment (MRC) was developed to assess the muscle power of limbs. A score of 0-5 was used to grade the power. Increasing disability of muscle power is reflected in an decreasing MRC score., Baseline to week 12.|Change of EDSS score, FS score, and MRC score at Week 4 and 24., To evaluate changes in neurologic outcomes in subjects with acute attacks in subjects with AQP4-IgG antibody-positive neuromyelitis optica spectrum disorders who are treated with efgartigimod + IVMP versus IVMP alone at Week 4 and Week 24. The EDSS score, FS score MRC score was described as in outcome 2., Baseline to Week 4 and Week 24.|Change of EQ-5D-5L score., To evaluate changes in quality of life in subjects at Week 4, 12, 24., Baseline to Week 4, 12 and 24.|Change of high-contrast visual acuity in subjects with acute optic neuritis., To evaluate changes in vision outcomes (High contrast visual acuity ) in subjects with acute optic neuritis post-treatment at Week 4, 12, 24., Baseline to Week 4, 12 and 24.|Change of OCT measurements in subjects with acute optic neuritis., To evaluate changes in vision outcomes (OCT measures) in subjects with acute optic neuritis post-treatment at Week 4, 12, 24., Baseline to Week 4, 12 and 24.|The proportion of participants requiring rescue treatment during the study period., To evaluate the proportion of subjects requiring rescue treatment during the study period., Baseline to Week 24.|The incidence of adverse events in the subjects during the study period., To evaluate the safety and tolerability of efgartigimod +IVMP versus IVMP alone., Baseline to Week 24.|The vital signs, clinical laboratory indices, and electrocardiogram records of the subjects during the study period., To evaluate the vital signs, clinical laboratory parameters and electrocardiograms during the study., Baseline to Week 24.
Other Outcome Measures: Change of serum total IgG levels., To evaluate changes in serum levels of total IgG in participants post-treatment at Week 4, 12, 24., Baseline to Week 4, 12 and 24.|Change of serum AQP4-IgG antibody levels., To evaluate changes in serum levels of AQP4-IgG antibodies in participants post-treatment at Week 4, 12, 24., Baseline to Week 4, 12 and 24.|Change of serum GFAP and NfL., To evaluate changes in serum GFAP and NfL levels in participants after treatment at Week 4, 12 and 24., Baseline to Week 4, 12 and 24.|Change of the size and extent of MRI lesions., To evaluate changes in spinal cord imaging in participants with acute myelitis following a course of efgartigimod + IVMP versus IVMP alone at Week 4, 12 and 24., Baseline to Week 4, 12 and 24.