Motor Neuron DiseasesW-MoSCANCT07838883
Rare Disease Registry Database for Movement Disorders in Southern Anhui Province
An observational study of Motor Neuron Diseases and Spinocerebellar Ataxias, run by First Affiliated Hospital of Wannan Medical College at 1 site in China.
Trial summary
According to the World Health Organization (WHO) definition, a rare disease is a disease affecting fewer than 6.5 people per 10,000; in China, it generally refers to diseases with a prevalence of less than 1/10,000 (i.e., with a total of no more than 140,000 patients). Read more about this study
- Who is it for?
- Any age
- Conditions the study is for
- Motor Neuron Diseases, Spinocerebellar Ataxias, Hereditary Muscular Dystrophy
- How many people
- 180 participants
- Where
- 1 siteChina
- Study dates
- 30 Sep 2026to Sep 2029
- How long will I take part?
- 10 Years
- Phase
- Observational
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 Any age
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Study population: These rare movement-related neurological diseases are all characterized primarily by gait abnormalities; although muscle weakness, ataxia, poor muscle endurance, and spastic paraplegia are their typical presentations, respectively, they share overlapping motor neurological symptoms and similar early clinical manifestations.
Who may be able to join
- Adults aged 19 years or older who are capable of giving informed consent.
- Definitive diagnosis of one of the following diseases: (1) amyotrophic lateral sclerosis (diagnosed according to the revised El Escorial criteria, Awaji criteria, or Gold Coast criteria); (2) spinocerebellar ataxia; (3) muscular dystrophy; (4) hereditary spastic paraplegia.
- Others: primary lateral sclerosis, progressive muscular atrophy, ALS-FTD syndrome, progressive bulbar palsy, benign focal muscular atrophy, and other motor neuron diseases.
Who cannot join
- Patients with concomitant systemic diseases;
- Vulnerable research subjects: minors and patients with cognitive impairment;
- Participants from whom clinical information and human biological samples cannot be collected;
- Other patients judged by the investigators to be unsuitable for participation in the study.
Trial locations and contacts
1 site in 1 country. Contact the study team or a site near you to ask about taking part.
China1 site, 1 recruiting
- Recruiting
First affiliated hospital of Wannan Medical University
Wuhu, Anhui, 241001
Xianjin Shang, Prof., 86 15155300503, shangxj2013@wnmc.edu.cn
About this study
According to the World Health Organization (WHO) definition, a rare disease is a disease affecting fewer than 6.5 people per 10,000; in China, it generally refers to diseases with a prevalence of less than 1/10,000 (i.e., with a total of no more than 140,000 patients). Such diseases are mostly hereditary or congenital in origin. Patients with rare diseases often present with complex symptoms, most of which are neurological. Although a large number of rare diseases have been identified, the small number of patients with each disease and the diversity of symptoms make early diagnosis difficult and render large-sample clinical trials challenging. Consequently, most rare diseases lack curative treatments; available therapies are of limited efficacy or prohibitive cost, ultimately leading to severe disability or death and imposing a substantial socioeconomic burden. This study establishes a registry for motor neuron diseases (MND), spinocerebellar ataxias (SCAs), hereditary muscular dystrophy (HMD), and hereditary spastic paraplegia (HSP). Owing to the low incidence, complex clinical diagnosis, unclear pathogenic mechanisms, and strong genetic heterogeneity, epidemiological data on rare motor neuron diseases are limited, further increasing the difficulty of biomarker screening and targeted therapeutic development. Therefore, there is an urgent need to establish a comprehensive registry and to obtain reliable evidence on risk factors and early diagnosis through research. This study aims to establish a registry of rare neurological diseases in the southern Anhui Province and to build a high-quality biobank of human biological resources.
- Conditions
- Motor Neuron Diseases, Spinocerebellar Ataxias, Hereditary Muscular Dystrophy, Hereditary Spastic Paraplegia
- Medical subject headings
- Motor Neuron Disease, Spinocerebellar Ataxias, Spastic Paraplegia, Hereditary
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
| Study group | Role | What they receive |
|---|---|---|
| Motor Neuron Diseases | Patient diagnosis, clinical assessment, treatment, and biological sample collection | |
| spinocerebellar ataxia(SCAs) | Patient diagnosis, clinical assessment, treatment, and biological sample collection | |
| hereditary muscular dystrophy | Patient diagnosis, clinical assessment, treatment, and biological sample collection | |
| hereditary spastic paraplegia | Patient diagnosis, clinical assessment, treatment, and biological sample collection |
What is being measured
The primary outcome is the main question the study is designed to answer.
Amyotrophic Lateral Sclerosis Functional Rating Scale (K-ALSFRS-R)
Total score: 0 to 48 points 48 points: completely normal function 0 points: complete loss of function in the corresponding dimension. The lower the score, the more severe the functional disability.
Measured: through study completion, an average of 3 months
Hereditary spastic paraplegia (HSP): Spastic Paraplegia Rating Scale (SPRS)
Including 13 items, each item is scored from 0 to 4 (0 = normal, 4 = most serious impairment) Total score: 0 to 52 points; the higher the score, the more serious the condition
Measured: through study completion, an average of one year
Scale for the Assessment and Rating of Ataxia (SARA)
Including 8 items, each item has a different maximum score. Total score: 0 to 40 points; the higher the score, the more severe the ataxia. 0 points: no ataxia. 40 points: the most severe ataxia.
Measured: through study completion, an average of one year
6-minute walk test for hereditary muscular dystrophy
The subjects walked continuously for 6 minutes as fast and safely as possible in a flat hard corridor; they can use the patient's daily walkers, crutches, and orthopedic braces; they are allowed to stop and rest in the middle (timer does not stop), and the total walking meters PMC is recorded at the end of 6 minutes.
Measured: through study completion, an average of 1 year
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted24 Sep 2026
- Study start30 Sep 2026
- Primary completionSep 2029 (estimated)
- Study completionSep 2029 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (3)Papers the sponsor has linked to this study.
- Other trials for Motor Neuron DiseasesStudies 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
- Observational
- Observational model
- Cohort
- Time perspective
- Prospective
- Enrolment
- 180 participants (estimated)
- Target duration
- 10 Years
Sponsor and investigators
- Lead sponsor
- First Affiliated Hospital of Wannan Medical College
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07838883
- Sponsor's protocol ID
- W-MoSCA2026200
- Acronym
- W-MoSCA
- Status last verified
- Oct 2026
- First submitted
- 17 Sep 2026
- Last updated
- 23 Oct 2026
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Publications
Cao Y, Zheng H, Zhu Z, Yao L, Tian W, Cao L. Clinical and Genetic Spectrum in a Large Cohort of Hereditary Spastic Paraplegia. Mov Disord. 2024 Apr;39(4):651-662. doi: 10.1002/mds.29728. Epub 2024 Jan 31.
PubMed 38291924Tong YR, Geng C, Guan YZ, Zhao YH, Ren HT, Yao FX, Ling C, Wang DC, Chen L, Cui LY, Zhang SY, Dai Y. A Comprehensive Analysis of 2013 Dystrophinopathies in China: A Report From National Rare Disease Center. Front Neurol. 2020 Sep 30;11:572006. doi: 10.3389/fneur.2020.572006. eCollection 2020.
PubMed 33101180Xu L, Chen L, Wang S, Feng J, Liu L, Liu G, Wang J, Zhan S, Gao P, Fan D. Incidence and prevalence of amyotrophic lateral sclerosis in urban China: a national population-based study. J Neurol Neurosurg Psychiatry. 2020 May;91(5):520-525. doi: 10.1136/jnnp-2019-322317. Epub 2020 Mar 5.
PubMed 32139654