Clinical trial NCT05154370: China National Registry of Neuro-Inflammatory Diseases
Official title: China National Registry of Neuro-Inflammatory Diseases: a Prospective Cohort Study
Study type: OBSERVATIONAL
Overall status: RECRUITING
Conditions: Multiple Sclerosis
NMO Spectrum Disorder
Clinically Isolated Syndrome
CNS Demyelinating Autoimmune Diseases
Acute Disseminated Encephalomyelitis
Primay Angiitis of the Central Nervous System
Autoimmune Glial Fibrillary Acidic Protein Astrocytopathy
Lead sponsor: Beijing Tiantan Hospital
Sponsor class: OTHER
First posted: 2021-12-13
Enrollment: 10000
Sex: ALL
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Observational model: COHORT
Time perspective: PROSPECTIVE
Sampling method: NON_PROBABILITY_SAMPLE
Study population: 10000 IDD patients are planned to be recruited from many sites throughout the country.

Brief summary: Central nervous system (CNS) idiopathic inflammatory demyelinating diseases (IDD) are mainly diseases caused by autoimmune factors that result in CNS demyelination damage and loss. It tends to accumulate in the brain, spinal cord and optic nerves. Multiple sclerosis (MS), clinically isolated syndrome (CIS), neuromyelitis optica spectrum disorder (NMOSD), myelin oligodendrocyte glycoprotein antibody-associated disease (MOGAD) and acute disseminated encephalomyelitis (ADEM) are all common IDDs of the CNS. Besides, primary angiitis of the central nervous system (PACNS), autoimmune glial fibrillary acidic protein astrocytopathy (GFAP-A), etc. may also be included because they are important differential diagnoses. This study will establish a large prospective cohort study database of Chinese IDD, which will record detailed electronic information on IDD patients, including demographic and socioeconomic data, medical history, clinical information, medication, and relevant examination results. The long-term observational study will be used to understand the natural history of disease, disability progression rates, imaging and biological indicators, long-term treatment approaches and prognosis of Chinese patients with IDD, to find predictive markers for IDD progression and prognosis, and to identify factors that influence the treatment and prognosis of patients with IDD.

Detailed description: Large prospective cohort studies allow long-term observation and analysis of the clinical status and disease activity of IDD patient population, and are the best way to understand IDD's natural course, clinical outcome, and drug efficacy in specific populations. Globally, several large prospective follow-up cohort studies have been conducted, providing important information on the disease characteristics of IDD patients. However, there is no large nationwide registry study of CNS IDD in China, and therefore there is a relative lack of data on the natural course of disease and drug efficacy in the Chinese IDD patient population, as well as a lack of standardized follow-up management of Chinese IDD patients. This study will establish a large prospective cohort study database of Chinese IDD, which will record detailed electronic information on IDD patients. We aim to establish a national (multicenter) disease registry for central nervous system idiopathic inflammatory demyelinating diseases in China, and to establish a unified standardized follow-up management process and treatment guidelines for patients with IDD in China; To provide real world data on the disease status of Chinese IDD patients; To understand the disease progression characteristics of IDD in China; To search for biological and imaging markers that predict the relapse, progression, and prognosis of IDD; to investigate the efficacy, safety, compliance and switch of different disease-modifying drugs (DMDs) in the long-term treatment of Chinese patients with IDD.

The study is a prospective observational (non-interventional) national multicenter cohort study to collect clinical data from IDD patients who have signed informed consent, to routinely and regularly follow up IDD patients on multiple clinical indicators, and to assess clinical outcomes. All information is to be completed prospectively from the time point the patient visited the hospital (except for Basic patient information and information about previous disease that are required at enrollment follow-up).Once the project started, the study sites are not allowed to discontinue the study on their own until the end of study is announced. In addition, to ensure the continuity of the data, each site needs to appoint designated clinical data collectors to collect data from qualified inpatient clinical cases consecutively, so as to ensure the consecutive registration of each inpatient case who meets the inclusion and exclusion criteria.

As the purpose of this study is to establish a national multicenter disease registry to provide disease-related information on patients with IDD in China, and the primary and secondary endpoints of the study being descriptive endpoints, therefore no formal calculation of sample size is needed. 10000 IDD patients are planned to be recruited.

Eligibility criteria: Inclusion Criteria:

* 1. No requirement for age and sex
* 2. Need to meet the diagnosis of at least one IDD (clinically isolated syndrome (CIS)/multiple sclerosis (MS)/neuromyelitis optica spectrum disorder (NMOSD)/MOG antibody-associated disease (MOGAD)/acute disseminated encephalomyelitis (ADEM).
* 3. Signed informed consent form.

Exclusion Criteria:

* Those with severe mental disease unable to cooperate with the examination and/or follow-up.
* Any patient (or the patient's legal representative) who is unable or refuses to sign informed consent.

Primary outcomes:
measure: Annual relapse rate (ARR) between baseline and follow-up in patients with IDD
timeFrame: baseline up to 5 years
measure: Change in EDSS scores of patients with IDD between baseline and follow-up over time
timeFrame: baseline, Month 6, Month12, Month18, Month24, Month30, Month36, Month42, Month48, Month54, Month60
Secondary outcomes:
measure: The brain structural change over time between the baseline MRI and the follow-up MRIs
timeFrame: baseline, year1, year2, year3, year4, year5.
measure: The spinal cord change over time between the baseline MRI and the follow-up MRIs.
timeFrame: baseline, year1, year2, year3, year4, year5.
measure: Change of the central vein sign at 7T MRI.
timeFrame: baseline, year1, year2, year3, year4, year5.
measure: Change of rim of iron at 7T MRI.
timeFrame: baseline, year1, year2, year3, year4, year5.
measure: Change in High-contrast Letter Acuity (HCLA) over time at baseline and during follow-up in patients with IDD
timeFrame: baseline, year1, year2, year3, year4, year5.
measure: Change in Low-contrast Letter Acuity (LCLA) over time at baseline and during follow-up in patients with IDD
timeFrame: baseline, year1, year2, year3, year4, year5.
measure: Percentage Change in Spectral-domain Optical Coherence Tomography (SD-OCT) Average Retinal Nerve Fiber Layer (RNFL) Thickness at baseline and every year.
timeFrame: baseline, year1, year2, year3, year4, year5.
measure: Percentage change in SD-OCT Average Retinal Ganglion Cell Layer/Inner Plexiform Retinal Layer (RGCL/IPL) every year.
timeFrame: baseline, year1, year2, year3, year4, year5.
measure: Changes in cognitive function of patients with IDD at baseline and over time during follow-up
timeFrame: baseline, year1, year2, year3, year4, year5.
measure: Number of Participants with Adverse Events as a Measure of Safety and Tolerability
timeFrame: From baseline to 5 years
measure: NF-L level in serum.
timeFrame: baseline, year1, year2, year3, year4, year5.
measure: Determination of serum autoimmune antibodies
timeFrame: baseline, year1, year2, year3, year4, year5.
Other outcomes:
Locations:
city: Beijing
country: China
facility: Beijing Tiantan Hospital
state: Beijing
Study officials:
References: