Neuromyelitis OpticaPhase 2FACTNCT06497374
FcRn Antagonists (Efgartigimod) for Acute NMOSD Attack
A Phase 2 study testing Efgartigimod Alfa Injection and High-dose intravenous methylprednisolone in people with Neuromyelitis Optica and Neuromyelitis Optica Spectrum Disorder, run by Tianjin Medical University General Hospital.
Trial 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… Read more about this study
- Who is it for?
- Ages 18 to 75 years
- Conditions the study is for
- Neuromyelitis Optica, Neuromyelitis Optica Spectrum Disorder, NMO Spectrum Disorder
- What is being tested?
- Efgartigimod Alfa Injection and High-dose intravenous methylprednisolone
- Type of treatment
- Pill or oral medicine, Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 63 participants
- Where
- No sites listed yet
- Study dates
- 5 Jul 2024to 30 Jun 2026
- Phase
- Phase 2
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 to 75 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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.
Who cannot join
- 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.
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- 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.
Trial locations and contacts
No sites are listed yet.
About this study
Official title: FcRn Antagonists (Efgartigimod) for Acute Neuromyelitis Optica Spectrum Disorders (NMOSD) Attack: a Phase 2, Randomized Controlled 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.
- Being tested
- Efgartigimod Alfa Injection, High-dose intravenous methylprednisolone
- Medical subject headings
- Neuromyelitis Optica
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Efgartigimod Alfa InjectionDrug
Efgartigimod+IVMP; IVMP; Efgartigimod
High-dose intravenous methylprednisoloneDrug
Efgartigimod+IVMP; IVMP; Efgartigimod
| Study group | Role | What they receive |
|---|---|---|
| Efgartigimod+IVMP group | Experimental | Patients will receive efgartigimod alpha via intravenous infusion at a dose of 10 mg/kg, each infusion lasting approximately 2 hours, on Week 0, 1, 2 and 3. Efgartigimod should be administered no later than the second day after initiation of high-dose intravenous methylprednisolone therapy. This is delivered intravenously at a dosage of 1,000 mg/day for five consecutive days, which is then reduced to 500 mg/day for the next three days, followed by 240 mg/day for another three days, and then 120 mg/day for an additional three days. Subsequently, the treatment shifts to oral prednisone, starting with 60 mg daily for seven days, then decreasing to 50 mg daily for the next seven days, followed by 40 mg daily for another seven days. Afterward, the prednisone dosage is reduced by 5 mg every two weeks until it reaches 10 mg. After Week 4, Inebilizumab to prevent relapse will be introduced according to the indication. Receives: Drug: Efgartigimod Alfa Injection; Drug: High-dose intravenous methylprednisolone |
| IVMP group | Experimental | Patients will be treated with injectable methylprednisolone sodium succinate as described in Arm A. After Week 4, Inebilizumab to prevent relapse will be introduced according to the indication. Receives: Drug: High-dose intravenous methylprednisolone |
| Efgartigimod group | Other | Patients will be treated with efgartigimod alpha injection via intravenous infusion at a dosage of 10 mg/kg, with each session lasting approximately 2 hours, on Week 0, 1, 2 and 3. After Week 4, Inebilizumab to prevent relapse will be introduced according to the indication. Receives: Drug: Efgartigimod Alfa Injection |
What is being measured
The primary outcome is the main question the study is designed to answer.
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.
Measured: Acute nadir to week4
Secondary outcomes (8)
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.
Measured: 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.
Measured: 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.
Measured: 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.
Measured: 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.
Measured: 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.
Measured: 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.
Measured: 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.
Measured: Baseline to Week 24.
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted11 Jul 2024
- Study start5 Jul 2024 (estimated)
- Primary completion31 Dec 2025 (estimated)
- Study completion30 Jun 2026 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Neuromyelitis OpticaStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Enrolment
- 63 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Tianjin Medical University General Hospital
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Fu-Dong Shi, Beijing Tiantan Hospital
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06497374
- Sponsor's protocol ID
- 20240426
- Acronym
- FACT
- Status last verified
- Jul 2024
- First submitted
- 16 Jun 2024
- Last updated
- 11 Jul 2024
- Results posted
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 11 Jul 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- FcRn Antagonists (Efgartigimod) for Acute Neuromyelitis Optica Spectrum Disorders (NMOSD) Attack: a Phase 2, Randomized Controlled Trial.
- ClinicalTrials.gov ID
- NCT06497374
- Acronym / sponsor ID
- FACT / 20240426
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Unknown status
- Enrolment
- 63 participants (estimated)
- Lead sponsor
- Tianjin Medical University General HospitalUniversity, hospital or other
- Study start
- 5 Jul 2024 (estimated)
- Primary completion
- 31 Dec 2025 (estimated)
- Study completion
- 30 Jun 2026 (estimated)
- First posted
- 11 Jul 2024 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 3
| Arm | Type | Receives |
|---|---|---|
| Efgartigimod+IVMP group | Experimental | Drug: Efgartigimod Alfa Injection, Drug: High-dose intravenous methylprednisolonePatients will receive efgartigimod alpha via intravenous infusion at a dose of 10 mg/kg, each infusion lasting approximately 2 hours, on Week 0, 1, 2 and 3. Efgartigimod should be administered no later than the second day after initiation of high-dose intravenous methylprednisolone therapy. This is delivered intravenously at a dosage of 1,000 mg/day for five consecutive days, which is then reduced to 500 mg/day for the next three days, followed by 240 mg/day for another three days, and then 120 mg/day for an additional three days. Subsequently, the treatment shifts to oral prednisone, starting with 60 mg daily for seven days, then decreasing to 50 mg daily for the next seven days, followed by 40 mg daily for another seven days. Afterward, the prednisone dosage is reduced by 5 mg every two weeks until it reaches 10 mg. After Week 4, Inebilizumab to prevent relapse will be introduced according to the indication. |
| IVMP group | Experimental | Drug: High-dose intravenous methylprednisolonePatients will be treated with injectable methylprednisolone sodium succinate as described in Arm A. After Week 4, Inebilizumab to prevent relapse will be introduced according to the indication. |
| Efgartigimod group | Other | Drug: Efgartigimod Alfa InjectionPatients will be treated with efgartigimod alpha injection via intravenous infusion at a dosage of 10 mg/kg, with each session lasting approximately 2 hours, on Week 0, 1, 2 and 3. After Week 4, Inebilizumab to prevent relapse will be introduced according to the indication. |
Who may qualify
- Ages
- 18 to 75 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (8 listed)
- 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.
Key exclusion criteria (14 listed)
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Efgartigimod Alfa InjectionEfgartigimod+IVMP; IVMP; Efgartigimod | Drug | Efgartigimod group, Efgartigimod+IVMP group |
| High-dose intravenous methylprednisoloneEfgartigimod+IVMP; IVMP; Efgartigimod | Drug | Efgartigimod+IVMP group, IVMP group |
Primary outcome
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.
8 secondary outcomes
Change of FS score and MRC score at Week 12.
Time frame: Baseline to week 12.
Change of EDSS score, FS score, and MRC score at Week 4 and 24.
Time frame: Baseline to Week 4 and Week 24.
Change of EQ-5D-5L score.
Time frame: Baseline to Week 4, 12 and 24.
Change of high-contrast visual acuity in subjects with acute optic neuritis.
Time frame: Baseline to Week 4, 12 and 24.
Change of OCT measurements in subjects with acute optic neuritis.
Time frame: Baseline to Week 4, 12 and 24.
The proportion of participants requiring rescue treatment during the study period.
Time frame: Baseline to Week 24.
The incidence of adverse events in the subjects during the study period.
Time frame: Baseline to Week 24.
The vital signs, clinical laboratory indices, and electrocardiogram records of the subjects during the study period.
Time frame: Baseline to Week 24.
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 11 Jul 2024.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Efgartigimod Alfa Injection and High-dose intravenous methylprednisolone in people with Neuromyelitis Optica and Neuromyelitis Optica Spectrum Disorder, run by Tianjin Medical University General Hospital.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 8 inclusion and 14 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: status not confirmed, as of the sponsor's last update on 11 Jul 2024. Status can change between updates, so confirm with the study contact before making plans.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
Contact Fu-Dong Shi (+8602260814587, fshi@tmu.edu.cn), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Change in Neurological Disability - Expanded Disability Scale Score (time frame: Acute nadir to week4). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 11 Jul 2024.