Natural History and Clinical Features of Amyotrophic Lateral Sclerosis (ALS)

A Study on the Natural History and Clinical Features of Amyotrophic Lateral Sclerosis (ALS) in Mainland China

1. Describe the distribution of ALS in mainland China, to explore the differences in the number of ALS in different times, regions and populations in order to further explore the causes affecting the distribution of ALS;
2. To investigate the cause of ALS in mainland China in the crowd disease development process and the corresponding characteristics change ;
3. To explore the effect of prognosis of ALS;

Study Overview

• Status: Recruiting
• Conditions: Amyotrophic Lateral Sclerosis
• Intervention / Treatment: Other: Amyotrophic lateral sclerosis

Detailed Description

With the development of supportive measures, the natural history of ALS has changed. Researchers compared the natural history of ALS patients from 1999-2004 and 1984-1998 and found that the median survival time was significantly longer in the former than in the latter (4.32 years vs. 3.22 years) and that the disease progression was slower in the former, even after adjusting for other confounding factors. Although previous studies have provided reference for the diagnosis and treatment of ALS, the etiology of ALS is still unknown, and the relevant clinical features and natural history of ALS still lack the verification of large samples. Therefore, the research on the natural history of ALS is of great significance to further increase the understanding of ALS and provide new evidence for the diagnosis and treatment of ALS.

• Study Type: Observational
• Enrollment (Anticipated): 3000

Contacts and Locations

• Study Contact: Name: Lu Tang, Msc; Phone Number: 13811854649; Email: tanglu@bjmu.edu.cn

Study Locations

• China
  • Beijing, China
    • Recruiting
    • Peking University Third Hospital
    • Contact: Dongsheng Fan, PHD. MD

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: ADULT; OLDER_ADULT; CHILD
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

• Sampling Method: Non-Probability Sample

Study Population

ALS patients in mainland China.

Description

Inclusion Criteria:

- Patients were diagnosed with ALS recruited from all participant clinical centers.The diagnosis of ALS was made using the revised El Escorial criteria for definite, probable, lab-supported, and possible.

Exclusion Criteria:

• Decline to follow-up.

Study Plan

How is the study designed?

Number of groups / cohorts

1

Cohorts and Interventions

Group / Cohort

Intervention / Treatment

Amyotrophic lateral sclerosis patients; Although previous studies have provided reference for the diagnosis and treatment of ALS, the etiology of ALS is still unknown, and the relevant clinical features and natural history of ALS still lack the verification of large samples. Therefore, the research on the natural history of ALS is of great significance to further increase the understanding of ALS and provide new evidence for the diagnosis and treatment of ALS

Other: Amyotrophic lateral sclerosis; AMYOTROPHIC lateral sclerosis (ALS) is a degenerative disorder of motor neurons in the cortex, brainstem and spinal cord1,2. Its cause is unknown and it is uniformly fatal, typically within five years3

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Overall Survival	Time interval from disease diagnosis to death	5 years to 10 years after recruitment
Indicators of disease progression	1) in the diagnosis of disease progression (⊿ FS) = (diagnosis of FRS - R score - when follow-up FRS - R)/diagnosis and follow-up time 2）Weight loss =(weight at diagnosis - weight at follow-up)/time to follow-up 3) BMI decreased = (BMI) at the time of diagnosis of BMI - follow-up/diagnosis and follow-up time 4) FVC decreased = (FVC - during the diagnosis to follow-up FVC)/diagnosis and follow-up time 5) between the above indexes can also be in all follow-up multiple numerical calculation, observing the trend of the change over time	1 year to 10 years after recruitment

Collaborators and Investigators

• Sponsor: Peking University Third Hospital

Study record dates

Study Major Dates

• Study Start (ACTUAL): November 1, 2019
• Primary Completion (ANTICIPATED): December 1, 2030
• Study Completion (ANTICIPATED): December 1, 2030

Study Registration Dates

• First Submitted: June 29, 2020
• First Submitted That Met QC Criteria: June 29, 2020
• First Posted (ACTUAL): July 2, 2020

Study Record Updates

• Last Update Posted (ACTUAL): July 2, 2020
• Last Update Submitted That Met QC Criteria: June 29, 2020
• Last Verified: June 1, 2020

More Information

Terms related to this study

• Keywords: amyotrophic lateral sclerosis; Epidemiology
• Additional Relevant MeSH Terms: Pathologic Processes; Metabolic Diseases; Central Nervous System Diseases; Nervous System Diseases; Neuromuscular Diseases; Neurodegenerative Diseases; Spinal Cord Diseases; TDP-43 Proteinopathies; Proteostasis Deficiencies; Sclerosis; Motor Neuron Disease; Amyotrophic Lateral Sclerosis
• Other Study ID Numbers: PUTH2019388

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No