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NCT Number: NCT06497374

FcRn Antagonists (Efgartigimod) for Acute NMOSD Attack

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.

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Key information

Conditions

Neuromyelitis Optica Autoimmune Diseases Autoimmune Diseases of the Nervous System Cranial Nerve Diseases Demyelinating Autoimmune Diseases, CNS Demyelinating Diseases Eye Diseases Immune System Diseases Myelitis, Transverse NMO Spectrum Disorder Nervous System Diseases Neuromyelitis Optica Spectrum Disorder Optic Nerve Diseases Optic Neuritis

Age range

18 year–75 year

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 2

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

Inclusion criteria

  • 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.
  • Confirmation of an acute attack of neuromyelitis optica spectrum disorders either with an acute optic neuritis and/or acute myelitis, defined as a worsening in the patient's signs and symptoms of neurological/visual impairment, an increase in the EDSS score, and symptoms lasting more than 24 hours and occurring more than 1 month since the last attack. Combination of imaging and clinical evaluation will be used to assess relapse and rule out a pseudorelapse.
  • New lesions or enhanced lesions need to be found in MRI.
  • Subjects who were receiving immunosuppressive therapy prior to the screening period will be required to agree to discontinue immunosuppression.
  • Treatment is stable at least 3 months.

Exclusion criteria

  • 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.
  • Received high-dose intravenous methylprednisolone within 4 weeks prior to the screening period.
  • Received intravenous immunoglobulin, plasma exchange, or immunoadsorption treatment within 4 weeks prior to the screening period.
  • Received a vaccination within the first 4 weeks of the screening period or planned during the study.
  • Using of a monoclonal antibody or investigational drug not mentioned above that has immunomodulatory effects within 3 months or 5 half-lives (whichever is longer) prior to the screening period.
  • Subject is known to be allergic to any component of the study drug or any other FcRn drug or contrast medium for enhanced MRI.
  • Subject is known to have contraindications to taking methylprednisolone (for subjects in A and B group).
  • Subjects with clinically significant active infections (including unresolved or inadequately treated infections, including active tuberculosis) as assessed by the investigator.
  • Subjects with positive screening tests for hepatitis B and C who have received live or live attenuated vaccine within 6 weeks prior to baseline.
  • Subjects is known unable to taken MRI.
  • Subjects is known to have other ophthalmic disease that affect vision as assessed by the investigator.

Treatment and study plan

Efgartigimod Alfa Injection

Drug

Efgartigimod+IVMP; IVMP; Efgartigimod

High-dose intravenous methylprednisolone

Drug

Efgartigimod+IVMP; IVMP; Efgartigimod

Primary outcomes

  1. Change in Neurological Disability - Expanded Disability Scale Score

    Time frame: Acute nadir to week4

    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.

Secondary outcomes

  1. Change of FS score and MRC score at Week 12.

    Time frame: Baseline to 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.

  2. Change of EDSS score, FS score, and MRC score at Week 4 and 24.

    Time frame: Baseline to Week 4 and Week 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.

  3. Change of EQ-5D-5L score.

    Time frame: Baseline to Week 4, 12 and 24.

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

  4. Change of high-contrast visual acuity in subjects with acute optic neuritis.

    Time frame: Baseline to Week 4, 12 and 24.

    To evaluate changes in vision outcomes (High contrast visual acuity ) in subjects with acute optic neuritis post-treatment at Week 4, 12, 24.

  5. Change of OCT measurements in subjects with acute optic neuritis.

    Time frame: Baseline to Week 4, 12 and 24.

    To evaluate changes in vision outcomes (OCT measures) in subjects with acute optic neuritis post-treatment at Week 4, 12, 24.

  6. The proportion of participants requiring rescue treatment during the study period.

    Time frame: Baseline to Week 24.

    To evaluate the proportion of subjects requiring rescue treatment during the study period.

  7. The incidence of adverse events in the subjects during the study period.

    Time frame: Baseline to Week 24.

    To evaluate the safety and tolerability of efgartigimod +IVMP versus IVMP alone.

  8. The vital signs, clinical laboratory indices, and electrocardiogram records of the subjects during the study period.

    Time frame: Baseline to Week 24.

    To evaluate the vital signs, clinical laboratory parameters and electrocardiograms during the study.

Other outcomes

  1. Change of serum total IgG levels.

    Time frame: Baseline to Week 4, 12 and 24.

    To evaluate changes in serum levels of total IgG in participants post-treatment at Week 4, 12, 24.

  2. Change of serum AQP4-IgG antibody levels.

    Time frame: Baseline to Week 4, 12 and 24.

    To evaluate changes in serum levels of AQP4-IgG antibodies in participants post-treatment at Week 4, 12, 24.

  3. Change of serum GFAP and NfL.

    Time frame: Baseline to Week 4, 12 and 24.

    To evaluate changes in serum GFAP and NfL levels in participants after treatment at Week 4, 12 and 24.

  4. Change of the size and extent of MRI lesions.

    Time frame: Baseline to Week 4, 12 and 24.

    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.

Study contacts

Contact information is provided by the study sponsor or research team.

Fu-Dong Shi

CONTACT

[email protected]

+8602260814587

Sponsors and collaborators

Lead sponsor

Tianjin Medical University General Hospital

Other

Registry information

Official study title

FcRn Antagonists (Efgartigimod) for Acute Neuromyelitis Optica Spectrum Disorders (NMOSD) Attack: a Phase 2, Randomized Controlled Trial.

Acronym: FACT

Important dates

Study start
2024
Primary completion
2025
Study completion
2026
First posted
Jul 11, 2024
Registry last updated
Jul 11, 2024

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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