NCT04956822

Brief Summary

This was a cross-sectional study in which patients were divided into 20 patients with amyotrophic lateral sclerosis, 20 patients with peroneal muscular dystrophy, 20 patients with Kennedy's disease and 30 healthy controls, in which patients with amyotrophic lateral sclerosis continued to be followed up for 1 year and the results of 4 cross-sectional examinations were taken.

Trial Health

43
At Risk

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Trial has exceeded expected completion date
Enrollment
90

participants targeted

Target at P50-P75 for all trials

Timeline
Completed

Started Jun 2018

Longer than P75 for all trials

Geographic Reach
1 country

1 active site

Status
unknown

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

Study Start

First participant enrolled

June 1, 2018

Completed
3.1 years until next milestone

First Submitted

Initial submission to the registry

June 27, 2021

Completed
12 days until next milestone

First Posted

Study publicly available on registry

July 9, 2021

Completed
1.4 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

December 1, 2022

Completed
Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

December 1, 2022

Completed
Last Updated

July 9, 2021

Status Verified

June 1, 2021

Enrollment Period

4.5 years

First QC Date

June 27, 2021

Last Update Submit

July 4, 2021

Conditions

Amyotrophic Lateral Sclerosis

Outcome Measures

Primary Outcomes (2)

  • The number of motor unit

    Motor unit estimation index (MUNIX) is a non-invasive electrophysiological technique that uses compound muscle action potential (CMAP) and surface electromyographic interference pattern (SIP) methods to evaluate the number of motor units (Mus) (motor unit number size, MUSIX).

    1 year

  • The size of motor unit

    Motor unit estimation index (MUNIX) is a non-invasive electrophysiological technique that uses compound muscle action potential (CMAP) and surface electromyographic interference pattern (SIP) methods to evaluate the number and size of motor units (Mus) (motor unit number size, MUSIX).

    1 year

Study Arms (4)

ALS

Amyotrophic lateral sclerosis group

Other: Basic informationOther: Functional scoresDevice: MINUX

CMT

peroneal muscular dystrophy group

Other: Basic informationOther: Functional scoresDevice: MINUX

KD

Kennedy's disease group

Other: Basic informationOther: Functional scoresDevice: MINUX

Control

Healthy control group

Other: Basic informationOther: Functional scoresDevice: MINUX

Interventions

Basic informationOTHER

Demographic data registration, medical history taking, physical examination. Electrocardiogram. Pulmonary function tests.

ALSCMTControlKD
Functional scoresOTHER

depending on the diseases enrolled, ALS and KD chose ALS-FRS functional scores and CMT chose CMTNS2 functional scores

ALSCMTControlKD
MINUXDEVICE

MUNIX examination was performed on all subjects. A total of eight bilaterally symmetrical muscles were selected, bilateral abductor little finger or abductor pollicis brevis, bilateral biceps or deltoid muscles, bilateral tibialis anterior muscles, and bilateral femurs. For quadriceps, choose a muscle with less muscle atrophy.

ALSCMTControlKD

Eligibility Criteria

Sexall
Healthy VolunteersYes
Age GroupsChild (0-17), Adult (18-64), Older Adult (65+)
Sampling MethodProbability Sample
Study Population

ALS is a degenerative disease of the nervous system, with an incidence of 2-3 in 100,000. It usually begins in middle age and mainly presents with progressive atrophy and weakness of skeletal muscles throughout the body. After 3 to 5 years, it dies of respiratory muscle involvement. At present, there are no biomarkers for early diagnosis and no effective treatment.

You may qualify if:

  • ALS patient group: 20 patients with confirmed or proposed ALS meeting the 1998 revised El Escorial diagnostic criteria for limb onset ALS.
  • CMT group: 20 patients with peroneal muscular dystrophy meeting the genetically confirmed diagnosis, who signed an informed consent form.
  • KD group: 20 patients with genetically confirmed Kennedy's disease consistent with genetic diagnosis, signed informed consent.
  • healthy controls:
  • age-matched healthy adults who volunteered to participate;
  • signed informed consent.

You may not qualify if:

  • signs of sensory impairment;
  • significant sphincter dysfunction;
  • visual and oculomotor impairment;
  • autonomic dysfunction;
  • signs of extravertebral symptoms;
  • severe cortical dysfunction;
  • ALS-like syndrome.

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (1)

Peking University Third Hospital

Beijing, China

RECRUITING

MeSH Terms

Conditions

Amyotrophic Lateral Sclerosis

Condition Hierarchy (Ancestors)

Spinal Cord DiseasesCentral Nervous System DiseasesNervous System DiseasesMotor Neuron DiseaseNeurodegenerative DiseasesTDP-43 ProteinopathiesNeuromuscular DiseasesProteostasis DeficienciesMetabolic DiseasesNutritional and Metabolic Diseases

Study Officials

  • Xiaoxuan Liu

    Peking University Third Hospital

    PRINCIPAL INVESTIGATOR

Central Study Contacts

Xiaoxuan Liu

CONTACT

13910982101zhangys0317@126.com

Xiaoxuan Liu

CONTACT

zhangys0317@126.com

Study Design

Study Type
observational
Observational Model
CASE CONTROL
Time Perspective
CROSS SECTIONAL
Sponsor Type
OTHER
Responsible Party
SPONSOR

Study Record Dates

First Submitted

June 27, 2021

First Posted

July 9, 2021

Study Start

June 1, 2018

Primary Completion

December 1, 2022

Study Completion

December 1, 2022

Last Updated

July 9, 2021

Record last verified: 2021-06

Data Sharing

IPD Sharing
Will not share

Locations

CN(1)