NCT06354790|Recruiting
Natural History Study of Children With LAMA2-related Dystrophies
LAMA2
A Prospective, Longitudinal, Interventional Natural History Study of Children With LAMA2-related Dystrophies
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LAMA2
A Prospective, Longitudinal, Interventional Natural History Study of Children With LAMA2-related Dystrophies
Sponsor study identifier
observational
40
1 country
4
Study Start
December 5, 2024
Actual (reported)Primary Completion
December 4, 2027
EstimatedPlanned date reported by the registry; this milestone is not confirmed as achieved.
Study Completion
December 31, 2027
EstimatedPlanned date reported by the registry; this milestone is not confirmed as achieved.
The goal of this natural history study is to characterize the disease course, characteristics in paediatric population of LAMA2-RD (related dystrophies) patients. The aim of the study is to establish a well-described cohort of patients in France with LAMA2-RD for prospective follow-up and recruitment for future clinical trials. Participants will be follow up during a two years period regarding exhaustive aspects of the pathology:
Registry-reported information. Elapsed time and enrollment totals alone do not measure study progress or recruitment pace.
December 31, 2027
EstimatedPlanned date reported by the registry; this milestone is not confirmed as achieved.
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Some registry-reported names shown below are not yet linked in this graph.
Dates and qualifiers reported by the registry. Estimated dates do not confirm that a milestone has occurred.
March 20, 2024
Actual (reported)April 9, 2024
Actual (reported)December 5, 2024
Actual (reported)December 4, 2027
EstimatedPlanned date reported by the registry; this milestone is not confirmed as achieved.
December 31, 2027
EstimatedPlanned date reported by the registry; this milestone is not confirmed as achieved.
Change in Motor function Measurement (MFM32) score
Through study completion, an average of 2 years
Change in Motor Milestone Checklist
Acquisitions and losses of motor functions (ex: Head control, sitting, crawling, standing, walking, climbing stairs, jumping,running, hopping,...)
Through study completion, an average of 2 years
Change in Revised Upper Limb Module (RULM) score
Through study completion, an average of 2 years
Change in grip strength measured by dynamometer tool
Through study completion, an average of 2 years
Change in pinch strength measured by dynamometer tool
Through study completion, an average of 2 years
Change in arm flexion/extension strength measured by dynamometer tool
Change in Wechsler Preschool and Primary Scale of Intelligence-IV (WPPSI-IV) results
Through study completion, an average of 2 years
Change in Wechsler Intelligence Scale for Children-V (WISC-V) results
Through study completion, an average of 2 years
Change in PedsQL questionnaire results
Through study completion, an average of 2 years
Change in CGI-S questionnaire results
Through study completion, an average of 2 years
Change in CGI-I questionnaire results
Through study completion, an average of 2 years
Evaluation of patients motor function using motor scales (MFM32, RULM), Timed functioned tests (6MWT, Rise from floor, 4SCT, 10mWT), dynamometric strength evaluation (grip, pinch, flexion/extension)
Patients cognitive evaluation (WPPSI-IV, WISC-V)
40 young patients aged between 2 and 15 years old (inclusive) at time of consent, with a confirmed LAMA2 diagnosis (muscle biopsy with absence of merosin (laminin-211) and at least one pathogenic variant in the LAMA2 gene)
Centre de Référence GNMH, Pédiatrie Hôpital Raymond-Poincaré
Garches, France
Service de MPR pédiatrique L'Escale - HCL
Lyon, France
Département de neuropédiatrie Pôle Femme Mère Enfant CHU de Montpellier - Hôpital Gui de Chauliac
Erwan GASNIER, PhD
CONTACT
First Submitted
March 20, 2024
First Posted
April 9, 2024
Study Start
December 5, 2024
Actual (reported)Primary Completion
December 4, 2027
EstimatedPlanned date reported by the registry; this milestone is not confirmed as achieved.
Translation and Validation of the MQOL Self-questionnaire Into French for Assessing Quality of Life in Patients With Meningiomas
Through study completion, an average of 2 years
Change in 6 Minutes Walking Test
Through study completion, an average of 2 years
Change in 4 Stairs Climbing Test (4SCT)
Through study completion, an average of 2 years
Change in 10m Walking Test
Through study completion, an average of 2 years
Change in Rise from Floor Test
Through study completion, an average of 2 years
Change in patient's Forced Vital Capacity (FVC) results
Through study completion, an average of 2 years
Change in patient's Peak Cough Flow (PCF) results
Through study completion, an average of 2 years
Change in patient's Maximum Expiratory Pressure (MEP) results
Through study completion, an average of 2 years
Change in patient's Maximal Inspiratory Pressure (MIP) results
Through study completion, an average of 2 years
Change in patient's Sniff Nasal Inspiratory Pressure (SNIP) results
Through study completion, an average of 2 years
Change in patient's muscle fat replacement measured by Magnetic Nuclear Resonance
Through study completion, an average of 2 years
Change in patient's cross-sectional area of the residual muscle measured by MNR
Through study completion, an average of 2 years
Evaluation of patients' respiratory function (FVC, PCF, MIP, MEP, SNIP)
Evaluation of patients' cardiac function (ECG, Echo-cardiography)
Evaluation of patients quality of life with questionnaires and PROM
Evaluation of spinal deformities by X-ray
Evaluation of a qualitative whole-body muscle part and a quantitative lower limb muscle part by MRI
Collection of blood and urinary sample for biomarkers research.
Registry-reported names are shown in full. Names without a link have not yet been matched to a Syfrah profile.
Contact the study team to confirm eligibility.
Montpellier, France
Plateforme d'essais cliniques pédiatriques iMotion
Paris, France
Study Completion
December 31, 2027
EstimatedPlanned date reported by the registry; this milestone is not confirmed as achieved.
Last Updated
December 12, 2024
Record last verified: 2024-12
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Source: ClinicalTrials.gov.
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