Spanish Natural History Study for LAMA2 Muscular Dystrophy
NCT ID: NCT06924125
Last Updated: 2025-04-11
Study Results
The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.
Basic Information
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RECRUITING
100 participants
OBSERVATIONAL
2021-07-27
2030-07-01
Brief Summary
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Detailed Description
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Conditions
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Study Design
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COHORT
PROSPECTIVE
Study Groups
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All patients
Compatible clinical presentation and identification of 2 pathogenic variants in LAMA2, or muscle biopsy with decreased laminin alpha2 protein and at least one pathogenic variant
Motor function scales
Evaluation of patients motor function using motor scales (MFM32, CHOP)
MUSCLE ULTRASOUND
Ultrasound guided evaluation of 28 muscles evaluated accross different body regions, assessed using the Heckmatt gradinf system (semiquantitative scale).
Muscle Elastography
Assess the mechanical properties of muscles, such as stiffness and elasticity.
Complete physical examination
Complete physical evaluations including muscle power and goniometry measurements
Ventilatory/ respiratory and other support assessment
Assessment of ventilatory, respiratory, and other support needs to evaluate the necessity of assistive devices
Oromotor function and nutrition
Assessment of bulbar funcionality: feeding devices, nutritional status.
Motor Milestone Assessments
Motor milestones age of acquisition and loss
Interventions
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Motor function scales
Evaluation of patients motor function using motor scales (MFM32, CHOP)
MUSCLE ULTRASOUND
Ultrasound guided evaluation of 28 muscles evaluated accross different body regions, assessed using the Heckmatt gradinf system (semiquantitative scale).
Muscle Elastography
Assess the mechanical properties of muscles, such as stiffness and elasticity.
Complete physical examination
Complete physical evaluations including muscle power and goniometry measurements
Ventilatory/ respiratory and other support assessment
Assessment of ventilatory, respiratory, and other support needs to evaluate the necessity of assistive devices
Oromotor function and nutrition
Assessment of bulbar funcionality: feeding devices, nutritional status.
Motor Milestone Assessments
Motor milestones age of acquisition and loss
Eligibility Criteria
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Inclusion Criteria
* Signed informed consent by the Legal Authority Responsible and/or assent by the subject (starting from 6 years old)
0 Minutes
100 Years
ALL
No
Sponsors
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ASOCIACIÓN IMPÚLSATE PARA LA CURA DE LOS NIÑOS CON DÉFICIT DE MEROSINA
UNKNOWN
Hospital Universitari Vall d'Hebron Research Institute
OTHER
Responsible Party
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Locations
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University Hospital Vall d'Hebron
Barcelona, Barcelona, Spain
Countries
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Central Contacts
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Facility Contacts
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Other Identifiers
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PR(AMI)/433/2021
Identifier Type: -
Identifier Source: org_study_id
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