Amyotrophic Lateral Sclerosis Registry

NCT05892822 | Unknown DMC

• 1 other identifier: KY2023-013-02
• Study Type: observational
• Target: 1,000
• Locations: 1 country
• Sites: 1

Brief Summary

This study takes amyotrophic lateral sclerosis (ALS) patients as the main research object. Through collecting genetics, imaging and clinical symptoms for Exploratory research, we will construct the gene spectrum of ALS in China, explore unknown pathogenic genes, explore the characteristic image characteristics of ALS, and establish the iPSCs library of ALS, providing resources and basis for the research of pathogenesis and treatment targets of ALS.

Conditions

Amyotrophic Lateral Sclerosis

Keywords

Amyotrophic lateral sclerosis

Outcome Measures

Primary Outcomes (2)

• Gene Mutation Characteristics of ALS in Chinese

  Constructing a Chinese ALS genetic information database by collecting genetic information from patients.

  day 1

• The disease development of ALS in the Chinese

  Record the progress of patients' clinical symptoms from baseline through 2-year follow-up.

  month 24

Secondary Outcomes (3)

• Exploring the imaging characteristics of ALS in the Chinese

  day 1, month 3, month 6, month 9, month 12, month 15, month 18, month 21, month 24

• Establishing an iPSCs library for ALS in the Chinese

  day 1

• Analysis of the correlation between electrophysiology characteristics and imaging characteristics of ALS in Chinese

  day 1, month 3, month 6, month 9, month 12, month 15, month 18, month 21, month 24

Study Arms (1)

Amyotrophic Lateral Sclerosis

This study is an observational study without grouping.

Other: Amyotrophic Lateral Sclerosis

Interventions

Amyotrophic Lateral Sclerosis OTHER

All ALS patients included in this group.

Amyotrophic Lateral Sclerosis

Eligibility Criteria

• Age: 18 Years - 80 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)
• Sampling Method: Non-Probability Sample

Study Population

The study aims to include an estimated 1000 ALS patients in China.

You may qualify if:

• years old ≤ age ≤ 80 years old;
• Patients are diagnosed definite or probable ALS according to the Awaji diagnostic criteria;
• Sign an informed consent form.

You may not qualify if:

• Unable to cooperate with research or complete follow-up due to geographical or other reasons.

Sponsors & Collaborators

• yilong Wang: lead

Study Sites (1)

Beijing Tiantan Hospital

Beijing, 100050, China

Location

Related Publications (6)

• Kiernan MC, Vucic S, Cheah BC, Turner MR, Eisen A, Hardiman O, Burrell JR, Zoing MC. Amyotrophic lateral sclerosis. Lancet. 2011 Mar 12;377(9769):942-55. doi: 10.1016/S0140-6736(10)61156-7. Epub 2011 Feb 4.

  PMID: 21296405 BACKGROUND

• Meng L, Bian A, Jordan S, Wolff A, Shefner JM, Andrews J. Profile of medical care costs in patients with amyotrophic lateral sclerosis in the Medicare programme and under commercial insurance. Amyotroph Lateral Scler Frontotemporal Degener. 2018 Feb;19(1-2):134-142. doi: 10.1080/21678421.2017.1363242. Epub 2017 Sep 11.

  PMID: 28891333 BACKGROUND

• van Rheenen W, van der Spek RAA, Bakker MK, van Vugt JJFA, Hop PJ, Zwamborn RAJ, de Klein N, Westra HJ, Bakker OB, Deelen P, Shireby G, Hannon E, Moisse M, Baird D, Restuadi R, Dolzhenko E, Dekker AM, Gawor K, Westeneng HJ, Tazelaar GHP, van Eijk KR, Kooyman M, Byrne RP, Doherty M, Heverin M, Al Khleifat A, Iacoangeli A, Shatunov A, Ticozzi N, Cooper-Knock J, Smith BN, Gromicho M, Chandran S, Pal S, Morrison KE, Shaw PJ, Hardy J, Orrell RW, Sendtner M, Meyer T, Basak N, van der Kooi AJ, Ratti A, Fogh I, Gellera C, Lauria G, Corti S, Cereda C, Sproviero D, D'Alfonso S, Soraru G, Siciliano G, Filosto M, Padovani A, Chio A, Calvo A, Moglia C, Brunetti M, Canosa A, Grassano M, Beghi E, Pupillo E, Logroscino G, Nefussy B, Osmanovic A, Nordin A, Lerner Y, Zabari M, Gotkine M, Baloh RH, Bell S, Vourc'h P, Corcia P, Couratier P, Millecamps S, Meininger V, Salachas F, Mora Pardina JS, Assialioui A, Rojas-Garcia R, Dion PA, Ross JP, Ludolph AC, Weishaupt JH, Brenner D, Freischmidt A, Bensimon G, Brice A, Durr A, Payan CAM, Saker-Delye S, Wood NW, Topp S, Rademakers R, Tittmann L, Lieb W, Franke A, Ripke S, Braun A, Kraft J, Whiteman DC, Olsen CM, Uitterlinden AG, Hofman A, Rietschel M, Cichon S, Nothen MM, Amouyel P; SLALOM Consortium; PARALS Consortium; SLAGEN Consortium; SLAP Consortium; Traynor BJ, Singleton AB, Mitne Neto M, Cauchi RJ, Ophoff RA, Wiedau-Pazos M, Lomen-Hoerth C, van Deerlin VM, Grosskreutz J, Roediger A, Gaur N, Jork A, Barthel T, Theele E, Ilse B, Stubendorff B, Witte OW, Steinbach R, Hubner CA, Graff C, Brylev L, Fominykh V, Demeshonok V, Ataulina A, Rogelj B, Koritnik B, Zidar J, Ravnik-Glavac M, Glavac D, Stevic Z, Drory V, Povedano M, Blair IP, Kiernan MC, Benyamin B, Henderson RD, Furlong S, Mathers S, McCombe PA, Needham M, Ngo ST, Nicholson GA, Pamphlett R, Rowe DB, Steyn FJ, Williams KL, Mather KA, Sachdev PS, Henders AK, Wallace L, de Carvalho M, Pinto S, Petri S, Weber M, Rouleau GA, Silani V, Curtis CJ, Breen G, Glass JD, Brown RH Jr, Landers JE, Shaw CE, Andersen PM, Groen EJN, van Es MA, Pasterkamp RJ, Fan D, Garton FC, McRae AF, Davey Smith G, Gaunt TR, Eberle MA, Mill J, McLaughlin RL, Hardiman O, Kenna KP, Wray NR, Tsai E, Runz H, Franke L, Al-Chalabi A, Van Damme P, van den Berg LH, Veldink JH. Common and rare variant association analyses in amyotrophic lateral sclerosis identify 15 risk loci with distinct genetic architectures and neuron-specific biology. Nat Genet. 2021 Dec;53(12):1636-1648. doi: 10.1038/s41588-021-00973-1. Epub 2021 Dec 6.

  PMID: 34873335 BACKGROUND

• Oskarsson B, Gendron TF, Staff NP. Amyotrophic Lateral Sclerosis: An Update for 2018. Mayo Clin Proc. 2018 Nov;93(11):1617-1628. doi: 10.1016/j.mayocp.2018.04.007. Epub 2018 Jul 4.

  PMID: 30401437 BACKGROUND

• Sances S, Bruijn LI, Chandran S, Eggan K, Ho R, Klim JR, Livesey MR, Lowry E, Macklis JD, Rushton D, Sadegh C, Sareen D, Wichterle H, Zhang SC, Svendsen CN. Modeling ALS with motor neurons derived from human induced pluripotent stem cells. Nat Neurosci. 2016 Apr;19(4):542-53. doi: 10.1038/nn.4273.

  PMID: 27021939 BACKGROUND

• Rowe RG, Daley GQ. Induced pluripotent stem cells in disease modelling and drug discovery. Nat Rev Genet. 2019 Jul;20(7):377-388. doi: 10.1038/s41576-019-0100-z.

  PMID: 30737492 BACKGROUND

Biospecimen

Retention: SAMPLES WITH DNA

In order to explore unknown pathogenic genes and therapeutic targets, this study will collect patient genetics to construct a Chinese ALS gene spectrum and establish an iPSCs library.

MeSH Terms

Conditions

Amyotrophic Lateral Sclerosis

Condition Hierarchy (Ancestors)

Spinal Cord Diseases; Central Nervous System Diseases; Nervous System Diseases; Motor Neuron Disease; Neurodegenerative Diseases; TDP-43 Proteinopathies; Neuromuscular Diseases; Proteostasis Deficiencies; Metabolic Diseases; Nutritional and Metabolic Diseases

Study Officials

• Yilong Wang, M.D.

  Beijing Tiantan Hospital

  PRINCIPAL INVESTIGATOR

Central Study Contacts

Yilong Wang, M.D.

CONTACT

13911666571; yilong528@gmail.com

Weiqi Chen, M.D.

CONTACT

15652813380; weiqichen@aliyun.com

Study Design

• Study Type: observational
• Observational Model: CASE ONLY
• Time Perspective: PROSPECTIVE
• Target Duration: 24 Months
• Sponsor Type: OTHER
• Responsible Party: SPONSOR INVESTIGATOR
• PI Title: Vice-President of Beijing Tiantan Hospital,Chief Scientist of Neurology Center

Study Record Dates

• First Submitted: May 16, 2023
• First Posted: June 7, 2023
• Study Start: June 15, 2023
• Primary Completion: December 31, 2024
• Study Completion: December 31, 2024
• Last Updated: June 7, 2023

Record last verified: 2023-05

Data Sharing

• IPD Sharing: Will not share

Locations

CN (1)