About this trial

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.

Eligibility criteria

This trial does not accept healthy volunteers

Qualifiers

Male or female, ages 18 to 75.

Meet the 2015 International Panel of Experts (IPND) diagnostic criteria for neuromyelitis optica spectrum disorders.

Acute EDSS nadir of 2.5-7.5, and a change of at least 0.5 points from baseline due to an acute relapse event.

Confirmation of serum AQP4-IgG antibody positivity using the CBA assay.

Disqualifiers

Other core clinical symptoms besides optic neuritis and myelitis.

Severe neuromyelitis optica spectrum disorder attack, which in the judgment of the investigator is not appropriate for this study. Severe is defined as requiring assisted ventilation or likely to require assisted ventilation during the study based on the judgment of the investigator.

Subjects with total IgG levels ≤ 6 g/L at screening.

Subjects with a B-cell count ≤ 5% of the lower limit of normal at screening.

Trial design

Design model

Parallel

Treatments tested in this trial

Efgartigimod Alfa Injection
Drug
Efgartigimod+IVMP; IVMP; Efgartigimod
High-dose intravenous methylprednisolone
Drug
Efgartigimod+IVMP; IVMP; Efgartigimod

Treatment groups

63 Participants

are divided into 3 treatment groups

Group A: Efgartigimod+IVMP groupExperimental treatment 2 interventions

Group B: IVMP groupExperimental treatment 1 intervention

Group C: Efgartigimod groupOther 1 intervention

Trial outcomes

Primary outcomes

1

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.

Time frame

Acute nadir to week4

Secondary outcomes

1

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.

Time frame

Baseline to week 12.

2

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.

Time frame

Baseline to Week 4 and Week 24.

3

Change of EQ-5D-5L score.

To evaluate changes in quality of life in subjects at Week 4, 12, 24.

Time frame